Emerging Biopharma’s

Contribution to
Innovation
A SSESSING THE IMPAC T

JUNE

20 19
Introduction
The majority of biomedical innovation is developed by emerging biopharma
companies, many of which have never marketed a therapy before. Over time,
those companies either successfully bring their products to market or, in many
cases, their assets or whole companies are acquired by others. These emerging
biopharma (EBP) companies are at the root of early-stage drug development and
their performance, the environment in which they operate, and their relationship
to other stakeholders in the health system play a critical role in determining the
future of many novel therapies and health technologies.

This report is intended to inform executives from these This study was produced independently by the IQVIA
emerging biopharma companies (EBPs), their investors, Institute for Human Data Science as a public service,

the large pharma company executives who engage with without industry or government funding. We gratefully

EBPs and often purchase their assets, as well as policy- acknowledge the contributions of Taskin Ahmed,
Aurelio Arias, Heather Cartwright, Carlo Ciapparelli,
makers focused on the overall innovation ecosystem. It
Steven DeVrieze, Kobby Essien, Onil Ghotkar, Shabnam
also has a global focus – as innovation knows no borders
Hanassab, Nora Hannigan, Seth Houston, Graham
– and includes novel analytics and an informative
Lewis, Michelle Liu, Mary Lu, Jay Margolis, Arth Mathur,
segmentation of EBPs based on the commercial routes
Bill McClellan, Brian Mi, Max Newton, Ester Oben Etah,
they employ to bring drugs to market, as there are a
Siobhan Palmer, Frank Papaianni, Natasha Piper, Urvashi
multitude of strategies being pursued in this area.
Porwal, Amish Puri, Sam Riches, Sarah Rickwood, Rohin
Sethi, Durgesh Soni, Gene Tatham, Alan Thomas, Terri
The report provides clarity on the current landscape of
Wallace and dozens of others at IQVIA.
EBP companies and their emerging product pipelines, as
well as associated clinical trial activity and levels of trial Find Out More
success. In addition, the report focuses on the financial If you wish to receive future reports from the IQVIA
deals, strategies, and organizational archetypes that lead Institute for Human Data Science or join our mailing list,
to EBPs effectively developing and/or marketing novel visit iqviainstitute.org

products. Finally, the report provides an assessment of MURRAY AITKEN

the overall outlook for EBPs, with a focus on how key Executive Director
trends will shape future achievements. IQVIA Institute for Human Data Science

©2019 IQVIA and its affiliates. All reproduction rights, quotations, broadcasting, publications reserved. No part of this publication may be reproduced or
transmitted in any form or by any means, electronic or mechanical, including photocopy, recording, or any information storage and retrieval system, without
express written consent of IQVIA and the IQVIA Institute.
Table of contents
Executive summary 2

Emerging biopharma companies 4

Research and development activities 7

Commercialization performance 17

Strategic transactions 28

Profile of the 2008 Series A financing cohort 36

Looking ahead 43

Notes on sources 52

Useful resources 53

Definitions 54

About the authors 55

About the Institute 57

Executive summary
Emerging biopharma (EBP) is a segment of companies Commercialization performance
driving a large portion of innovation and development EBPs increasingly contribute to innovation, and these
in the life sciences. EBP companies are defined as companies were the original patentees for 29 of the
having less than $200 million in estimated annual current top 100 drugs, which account for 40% of sales in
spending on R&D, or under $500 million in global the United States in 2018. For drugs launched in 2018,
revenue. Emerging biopharma companies employ many EBPs originated and launched 42% of the new drugs, a
strategies involving development of novel compounds, higher percentage than in past years and up from 26% in
value-added medicines, and/or generics, or even license 2017. These companies have a greater focus on orphan
products from other larger companies for regional drugs than other companies, with over half of EBP-
marketing rights. While some are not involved in R&D launched drugs in 2018 receiving orphan designation
and may not be targets for partnering or M&A activities and almost a quarter were approved based on a single-
in the traditional sense, they do represent investment arm trial.
opportunities for some.
The median time for EBP companies to launch new
Research and development activities drugs was 16.6 years in 2018, over 30% slower than
There are 3,212 companies defined as emerging other company segments. Emerging biopharma
biopharma in 2018. Emerging biopharma companies now originated products generally reach the market faster
represent 73% of late-stage research, up from 52% in if they were launched by other company segments.
2003, and the number of molecules under development This may be more indicative of the value seen by the
by EBPs grew by 15% in each of the past two years. EBP companies purchasing the assets, which was borne
companies also represent 84% of early-phase research. out in subsequent trials and faster approval, but truly
In total, there are 8,706 products or programs in active understanding what drives product performance
development, ranging from discovery to registration, requires an assessment of how it addresses clinical
with 80% from EBP companies. Oncology products unmet need and how different each product is from
developed by emerging pharma companies have competing treatments. Across hundreds of launches
increased 74% since 2013, driven by targeted agents. over the ten years to 2017, EBP companies were
EBP companies are developing more than 90% of Next- the originators of more products in areas of high
Generation Biotherapeutics (NGB), which include cell, unmet need, and also those with greater product
gene and RNAi technologies, among others. The top 30 differentiation, and many of these drugs were
emerging biopharma companies employ both traditional partnered with or acquired by larger companies for
and cutting-edge technologies that span across therapy launch. Of note, EBPs generally achieve lower average
areas and include the most highly innovative new sales when launching new active substances than
mechanisms in development. other companies.

EBP companies conducted 65% of all clinical trials in 2018 Strategic transactions
and are now running more trials than larger companies The lifecycle of a company or a compound in
across all phases. Products developed by emerging biopharmaceuticals often begins with an academic
biopharma companies have a composite success rate of research institution or a venture capital investment.
17% – greater than other company segments. Looking ahead to the innovations that will likely emerge

2 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

in the next decade and beyond, the volume of venture Ten years after initial financing, 51% of companies
capital activity in life sciences offers a useful barometer were privately held and 17% had gone public. Of those
of investor interest and provided research remains 28 publicly traded companies, five now have market
productive at historic levels, the output of research in capitalization of over $1 billion dollars. Forty-three
the future. Venture financing has been rising in absolute companies have been acquired since initial financing;
terms and in the number of deals. more than half by other emerging
biopharma companies.
Collaborative deals between EBPs and larger
companies have continued, and partnering deals have Over half of financed companies have molecules in late-
increasingly had payments later in the agreement with stage development, and six drugs from five companies
more milestones and lower up-front payments. EBPs were approved within ten years of financing.
partnering with large companies accounted for nine of
Looking ahead
the top ten partnering deals in 2018, and seven of the top
The environment is changing rapidly around clinical
ten M&A deals in 2018 involved an emerging biopharma
development and the marketing of drugs, and EBP
company. There was a 78% increase in deals in 2018
companies will be operating in a highly dynamic and
when drugs were at pre-registration stage, or 62 deals
complicated market for the foreseeable future. Eight
compared with 36 in 2017. Large pharma continues to
key trends are influencing aspects of trial design,
acquire or license assets between themselves and with
duration and success with important implications for EBP
EBPs, and in 2018, among 45 companies assessed, 415
companies. In addition, the commercial environment is
deals were transacted for an aggregate disclosed value of
increasingly under pressure related to the high level of
$272 billion.
existing healthcare spending across most economies.
Profile of the 2008 Series A financing cohort The companies with the greatest success in the coming
There were 168 startups which received series A funding years will be those that take the best advantage of
in 2008, and ten years later they have achieved a three key factors: the use of data and analytics, the
variety of outcomes providing a useful benchmark on adoption of technology and a critical need to employ
performance for startups which have received funding flexible business models. Some companies may choose
more recently. to partner to maximize value around technology or
analytics, others may make their entire focus the
Startups received funding in 2008 across a range of
development of tools and approaches to sell to others.
therapy areas, including high profile NGBs, and at
The companies that can find the best mix of these
varied funding levels, with half occurring in three U.S.
factors aligned to the assets they have in development
states. The largest area of focus for these companies
and the capabilities they have in their organizations will
was oncology, mirroring its rise in importance to the
find the greatest success in the coming years.
overall pipeline since 2008. They also received varied
initial funding levels, suggesting that some investors
were seeing a proof of concept that required milestones
to prove value, while others had identified a significant
value and funded it to a greater extent.

iqviainstitute.org | 3
Emerging biopharma companies

It is helpful to use a set of common definitions to • In general, emerging biopharma companies are
fully understand the role that emerging biopharma defined as those with $50-400 million in sales or
companies play in the United States and global health under $200 million in R&D spending noted from public
systems. Defining companies by revenue and pipeline companies reporting or presence of R&D activity in the
activity offers a simple yet effective way of mapping past four years if spending levels were not reported
types of companies. (private companies). In practice however, some
companies which has less than $400 million in the past
For the purposes of this report, the following segments
five years could have revenues up to $500 million due
are discussed, and placement within those segments are
to high performing launch products. In this report, the
determined based on the 2018 calendar year:
sales threshold for EBP companies was extended to
• Emerging biopharma (EBP) companies are defined $500 million to maintain cohort stability over a 5-year
as those with either R&D spend <$200 million or period/time for analytical purposes.
prescription sales up to $500 million. Companies with
*N
 ote: Company segmentation has been informed
any active pipeline since 2014 were included.
by a number of sources including IQVIA MIDAS for
• Small companies have global prescription sales sales revenue and IQVIA Pipeline Intelligence for R&D
between $500 million to $5 billion in the calendar year. activity. Other sources include Clarivate Analytics
Cortellis for additional information on the presence of
• Mid-size companies have global prescription sales
R&D activity and EvaluatePharma for R&D spending
between $5 and $10 billion in the calendar year.
where available (generally for public companies).

• Large companies are those with global prescription

sales exceeding $10 billion in the calendar year.

Emerging Biopharma Sales <$500Mn, R&D Spend <$200Mn

Small Pharma Sales $500Mn – <$5Bn

Mid-sized Pharma Sales $5Bn – <$10Bn

Large Pharma Sales >$10Bn

4 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

EMERGING BIOPHARMA COMPANIES

The universe of biopharma companies is diverse and includes a large

set of companies defined as emerging biopharma

Exhibit 1: Global Medicine Sales and Number of R&D Pipeline Drugs per Company in 2018 by Segment
Global Sales 2018 Const US$Mn

50,000

5,000 Large Pharma

25 Companies, $637Bn; 1,845 Pipeline Products
Mid-sized Pharma
9 Companies, $50Bn; 181 Pipeline Products
500 Small Pharma
74 Companies, $159Bn; 446 Pipeline Products

Emerging Biopharma
3,212 companies, $139Bn; 8,572 Pipeline Products
50
0 20 40 60 80 100 120 160 180
Number of Pipeline Drugs, Discovery to Filing

Source: IQVIA Pipeline Intelligence, Jan 2019; IQVIA MIDAS, Dec 2018

• There are 3,212 companies defined as emerging • The number of development projects correlates directly
biopharma in 2018, including a subset of companies with company size. Although EBPs are numerous, they
with revenues below $50 million, some of which have an average of three development projects each,
focus on generics or are non-pharmaceutical while large companies average 74, and mid and small
companies acting as distributors or intermediaries companies average 20 and six, respectively.
in some markets.
• The significant number of drug projects per large
• In this report, these smaller companies are considered pharma are a reflection or the strategy of some
emerging biopharma companies, in part because many large companies to acquire drug candidates and
of them have mixed strategies involving development technologies from EBP companies.
of novel compounds, value-added medicines, as well
• Some EBPs have over 20 pipeline projects, similar to
as generics or licensing of products from other larger
their small- and mid-sized company counterparts.
companies for regional marketing rights. While some
are not involved in R&D and may not be targets for • Large biopharma companies have significant
partnering or M&A activities in the traditional sense, research programs but oftentimes source assets from
they do represent investment opportunities for some. thousands of emerging firms.

Chart notes: IQVIA MIDAS revenues reported by marketing company and linked by corporation ownership but not including co-marketing or royalty payments
and may understate revenues for some companies and overstate for others. Some pipeline products may be attributed to multiple companies across segments.
EBPs in this chart include some smaller generic companies, distributors, parallel traders. Some others have no novel compound research but are instead focused
on novel formulations, Value Added Medicines (VAMs), biosimilars, but are included in the overall statistics. Further analyses in the report in areas of company
launch and commercialization performance does not include such companies.

iqviainstitute.org | 5
EMERGING BIOPHARMA COMPANIES

Emerging biopharma companies make up 80% of the current

development pipeline and earned 14% of revenue in 2018

Exhibit 2: Company Segments as a Percentage of Pipeline Projects and Global Sales in 2018

Global Medicine Spending Const US$ = $995Bn

Large 15% Large 64%

Mid 2%
Small 3% Mid 5%

Small 16%

EPB 80%

EPB 14%

Total Pipeline Projects Discovery to Filing, N = 8,706

Source: IQVIA Pipeline Intelligence, Jan 2019; IQVIA MIDAS, Dec 2018

• Including both early phase and late phase research, process of growing rapidly to eventually have sales
160
EBP companies account for 80% of R&D activity. above $10 billion.

• These companies include a range of strategies from • Large pharma companies have 64% of revenue
pure innovators to companies focused on developing globally, taking advantage of their larger resources to
generics, reformulations and biosimilars, all of which select and invest in early-phase research that shows
still require some regulatory review of their phase I, the most promise.
phase II or phase III trials.
• Large pharma companies represent the largest
• Companies which have revenues between $500 million segment of partners and/or purchasers of companies
and $10 billion dollars represent only 5% of pipeline and products originated by EBP companies.
but 21% of sales, as the combination of the small and
• The pipeline activity by large companies includes
mid-sized segments.
those assets originated in their own labs as well
• These companies are often focused on specific niches as those already acquired from academia or other
by therapy area, geography, or commercialization smaller companies.
approach, but a subset of these companies are in the

Chart notes: Pipeline products being developed by multiple companies are attributed to the company in the larger revenue segment. Company segment share of
sales or pipeline are represented in terms of the share of the area of the triangle. EBPs in this chart include some smaller generic companies, distributors, parallel
traders. Some of these companies have no novel compound research but are instead focused on novel formulations, Value Added Medicines (VAMs), biosimilars, but
are included in the overall statistics. Further analyses of company performance around launch and commercialization rarely include such companies.

6 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

Research and development activities

• Emerging biopharma companies now represent 73% • The composite success rate for products developed by
of late-stage research, up from 52% in 2003. emerging biopharma companies is 17% – greater than
other company segments.
• Emerging biopharma companies also represent 84%
of early-phase research and a sharply rising share of • The top 30 emerging biopharma companies employ
late-phase research. both traditional and cutting-edge technologies that
span across therapy areas.
• The late-stage development pipeline of emerging
biopharma has steadily expanded with 15% growth in • These EBPs tend to have three major organizational
the number of products in both 2017 and 2018. archetypes that may correlate with their approaches to
development and commercialization strategies. These
• With 8,706 products in active development, ranging
include “US-Based Standalone Companies”, largely
from discovery to registration, emerging biopharma
developing assets to license to other companies; “Hub
are developing treatments that span a diverse range
and Spoke” companies with centralized corporate
of drug classes.
functions and separate subsidiaries to optimize
• Oncology late-phase pipelines have increased 74% in operations and develop assets across a wide range
the past five years driven by targeted agents. of therapy areas; and “Ex-US Companies” that are
typically regionally focused companies working on
• Emerging biopharma companies are developing more
biobetter and/or biosimilars or that in-license assets
than 90% of Next-Generation Biotherapeutics in the
for marketing in emerging markets.
late-stage pipeline.
• The top 30 emerging biopharma companies by
• EBP companies also ran 65% of all clinical trials in
pipeline employ both traditional and cutting-edge
2018 and are now running more trials than larger
technologies that span across therapy areas.
companies across all phases.

iqviainstitute.org | 7
RESEARCH AND DEVELOPMENT ACTIVITIES

Emerging biopharma companies now represent 73% of late-stage

research, up from 52% in 2003

Exhibit 3: Percentage of Late-Stage Pipeline by Company Segment, 2003–2018

5%
3%

19%
3%
6% 2% 6%

5% 6%
27%
31%
52% 61%
36% 65% 73%

2003 2008 2013 2018

n = 1,383 n = 2,049 n = 2,083 n = 2,891

EPB Large Mid Small

Source: IQVIA Pipeline Intelligence, Jan 2019

• Emerging biopharma companies accounted for 73% the fastest-growing areas of oncology and orphan
of the total late-stage R&D pipeline in 2018, compared drugs, and because they increasingly can develop their
with 61% in 2008. innovations without the need to partner or be acquired.

• Large pharma companies have seen their share drop • In the past, the majority of EBP assets were sold
from 27% to 19% from 2017 to 2018. or licensed before launch. However, in 2018, EBPs
themselves launched 47% novel therapies.1
• The share of mid-sized and small pharma companies
developing novel products has remained steady since • Since 2013, the absolute number of active R&D
2003, with small pharma developing approximately compounds has increased 37%, and this will likely
5–6% of products and mid-sized pharma developing support a continued increase in the number of
from 2–5%. EBP-launched drugs over the next five years.

• Emerging biopharma companies are increasing their

pipeline share, because they are the most active in

Chart notes: Late-stage pipeline is defined as active programs (activity in past three years) in Phase II through registration. Research programs are considered
active following an update for three years unless specific information indicates that research has stopped. Drugs are noted in relation to the most-advanced
research phase across indications and geographies. If multiple companies were involved in a project, the larger segment takes precedence. Percentages may not
sum to 100% due to rounding.
1
IQVIA Institute. The Changing Landscape of Research and Development: Innovation, Drivers of Change, and Evolution of Clinical Trial Productivity. Apr 2019.
Available from: https://www.iqvia.com/institute/reports/the-changing-landscape-of-research-and-development

8 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

RESEARCH AND DEVELOPMENT ACTIVITIES

Emerging biopharma companies represent 84% of early-phase

research but a sharply rising share of late-phase research

Exhibit 4: Emerging Biopharma Share of Early, Late and Overall R&D Pipeline 2003–2018

84%
82% 81%
79% 80% 80% 80% 80% 80%
76%
73% 74% 74% 74% 80%
71% 78%
68% 76% 75% 75% 76%
74% 75% 74%
72% 73%
71% 71% 70% 71%
70%
67% 68%
67%
65% 65% 65%
63%
61% 61% 61% 62%
59% 60%
57%
52% 54%

2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

EPB Share of Discovery – Phase I EPB Share of Overall Pipeline EPB Share of Phase II – Registration

Source: IQVIA Pipeline Intelligence, Jan 2019

• Over the last 15 years, emerging biopharma companies • Late-stage development, from Phase II to registration,
have seen steady growth in their share of the overall has also seen an increase in share, growing from
R&D product pipeline. They reached an 80% share of the 52% in 2003 to 73% in 2018. This increase mirrors
total pipeline in 2018, up from 65% in 2003. an increase in the number of emerging biopharma
companies launching novel products, growing from
• Emerging biopharma companies have a growing
33% in 2010 to 47% in 2018.1
impact on early-stage development - from discovery
through Phase I – with their share of pipeline products
increasing from 68% in 2003 to 84% in 2018.

Chart notes: Research programs are considered active following an update for three years unless specific information indicates that research has stopped. Drugs
are noted in relation to the most-advanced research phase across indications and geographies. If multiple companies are involved in a drug program, the company
from the larger segment has been shown. The EBP segment shown here do not have partners from Large, Mid or Small-sized pharma companies involved in their
programs, but that does not mean there are not commercialization agreements in place that could ultimately result in a comarketing or copromotion.
1
IQVIA Institute. The Changing Landscape of Research and Development: Innovation, Drivers of Change, and Evolution of Clinical Trial Productivity. Apr 2019.
Available from: https://www.iqvia.com/institute/reports/the-changing-landscape-of-research-and-development

iqviainstitute.org | 9
 RESEARCH AND DEVELOPMENT ACTIVITIES

The late-stage development pipeline of emerging biopharma has

steadily expanded with 15% growth in both 2017 and 2018

Exhibit 5: Number of Late-Stage Emerging Biopharma Pipeline Products by Therapeutic Drug Class, 2008–2018

2,000

1,500
Number of Products

1,000

500

0
2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

Oncology Neurology - Other/Behavioral Infectious Diseases* GI Products

Pain Dermatology Immunology Endocrinology
Vaccines Nervous System Disorders All Others
Source: IQVIA Pipeline Intelligence, Dec 2018; IQVIA Institute, Mar 2019

• The number of molecules in development by emerging • Despite product attrition between 2017 and 2018 in
biopharma companies in Phase II or later increased infectious disease, research in this area is robust,
by 15.3% in 2018 to a total of 2,120 molecules, and by representing 7% of the late-stage pipeline and growing
57.5% from 2013–2018, at a CAGR of approximately 43% since 2013.
9.5% over the same period.
• Pain products have increased the most out of all
• Almost 30% of late-stage emerging biopharma pipeline therapy classes since 2017, an increase of 40% through
products were oncology therapies in 2018. Oncology 2018. Pain products made up 6% of the pipeline in
products developed by emerging pharma companies 2018 and have increased 76% from 2013. Notably,
have grown by 20% since 2017 and 74% since 2013. development in pain has been shifting towards
non-narcotic medicines, as pressures to limit and avoid
• The category “Neurology - other/behavioral,” which
opioid use have strengthened in response to
are therapies for indications such as spinal muscular
the opioid crisis.
atrophy, cognitive disorders**, insomnia and epilepsy,
made up 8% of the pipeline in 2018 and have grown 69%
since 2013, with 31 new products added in 2018 alone.

Chart notes: Late-stage pipeline is defined as active programs (activity in past three years) in Phase II through registered. Pipeline products are categorized
by their most-advanced indication, and additional indications for pipeline drugs still in earlier phases or for already marketed drugs are not counted.
Infectious disease* = infectious disease products excluding vaccines; GI = Gastrointestinal; **Cognitive disorders under “Neurology – Other/Behavioral” drug
class do not contain anti-Alzheimer’s therapies.

10 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

RESEARCH AND DEVELOPMENT ACTIVITIES

Emerging biopharma are developing treatments that span a

diverse range of drug classes

Exhibit 6: Number of Products and Emerging Biopharma Share of Early and Late-Stage R&D Pipeline, 2018
Antituberculars Other Hormones Contraception
Labour Inhibitors Diagnostic Equip & Allergen Tests
Allergy, Systemic & Nasal
100% Anti-Parasitics,
BPH Ear Treatments
IV&IM Immunoglobulins Antimalarials, Hypothalamic Hormones
Thyroid & Anti-Thyroid Insecticides Erectile Dysfunction
Erythropoietins
Labour Inducers
Emerging Biopharma Share of Late-Phase

Other Alimentary
90% Corticosteroids, Plain & Combo All Other Urological
Ophthalmology, General
Hematopoietic Growth Factors
Anti-Inflammatory Enzymes Miotics+Antiglaucoma Preps Growth Hormones
GU Anti-infectives Hypertension Cancer Detox Ag, Anti-Nauseants
Osteoporosis Antidiabetics Dermatologics
Antivirals, Herpes
80% Interferons, Excl Ms Market
Lipid Regulators
Epilepsy/Parkinson’s Other CNS Cough, Cold, Flu
Pain Sex Hormones
Blood Coagulation ADHD
Anti-Obesity Preps
Antigout Preps All Other Therapeutics GI Products Anti-Ulcerants
70% Anti-Alzheimer’s AMD
Anti-Anaemics Multiple Sclerosis Anticoagulants
Other Cardiovasculars Other Haematologicals Syst Antifungals
Urinary Viral Hepatitis Antibacterials
Incontinence
60% Autoimmune Diseases HIV Antivirals Resp Antivirals Ex Flu
Vaccines (Pure, Comb, Other)
Mental Health Oncologics
Imaging
Immunosuppressants Immunoglobulins & Antitoxic Sera
50% Bubble Size =
e r Total # of Drugs
e er as ate Respiratory Agents
as at Ph e Discovery to Reg
- Ph Gre rly- Gr
40% te e e
La har Ea har
S S
1500
0% 20% 40% 60% 80% 100%
Emerging Biopharma Share of Early-Phase 250
5
Source: IQVIA Pipeline Intelligence, Dec 2018; IQVIA Institute, Apr 2019

• There are currently 8,706 products in active • Small yet critical areas of development, such as
development, ranging from discovery to registration. hypothalamic hormones and erythropoietins, are
Emerging biopharma companies currently hold assets dominated by emerging biopharma companies across
across the development spectrum, with greater the pipeline, with shares between 90–100% in both
concentration in certain therapeutic areas with varying early and late-stage development.
degrees of demand.
• Emerging biopharma companies have a majority of the
• Emerging biopharma companies comprise a large late-phase yet little to none of the early-phase pipeline
portion of both early-stage and late-stage oncology for antituberculars, allergy, thyroid and genitourinary
development, at 69% and 72%, respectively. anti-infective assets. These are small areas of
development, consisting of only 12 products.
• The next largest areas of pipeline development
include pain, other CNS therapeutics, dermatologics, • Similarly, emerging biopharma companies hold a
immunosuppressants and gastrointestinal (GI) majority share of 17 blood coagulation products in
products. EBP shares for the early and late-stage late-phase development, though little to no share of
pipeline in these areas are on average 70%. those in early-stage development.

Chart notes: Pipeline products are categorized by their most-advanced indication, and additional indications for pipeline drugs still in earlier phases or for
already marketed drugs are not counted. Bubble size represents total pipeline, not just EBP products.

iqviainstitute.org | 11
RESEARCH AND DEVELOPMENT ACTIVITIES

Oncology late-phase pipelines have increased 74% in the past five

years driven by targeted agents

Exhibit 7: Number of EBP-Developed Oncology Products in the Late-Stage R&D Pipeline by Drug Type

610

65
+74%

221
+10%
351
59
+107%
107

324
+75%
185

2013 2018

Targeted Biologics Targeted Small Molecule Others

Source: IQVIA Pipeline Intelligence, Dec 2018; IQVIA Institute, Apr 2019

• The number of late-stage pipeline oncology therapies • Targeted therapies, including immuno-oncology
developed by emerging biopharma companies grew therapies and small-molecule kinase inhibitors,
from 351 in 2013 to 610 in 2018, an expansion of 74%, represent a paradigm shift in the treatment of cancer.
due in large part to the growing number of targeted Robust growth in this area of oncology products
therapies in the pipeline. suggests that the range of mechanisms and novel
technologies being explored by emerging biopharma
• In 2018, 89% of late-stage oncology products being
companies is increasing.
developed by emerging biopharma companies were
targeted therapeutics, up from 83% of the pipeline
in 2013.

• Targeted biologic therapies increased 75% from 2013

to 2018, while the number of targeted small molecules
more than doubled. Other therapies, including
radiotherapies and hormonal, have increased more
modestly by 10% over the past five years.

Chart notes: Therapeutic Oncology pipeline where emerging biopharma companies are the only companies involved in development. Late phase pipeline
includes trials in Phase II or higher for the most advanced indication. Phase I/II trials are included as Phase II. Others includes radiotherapies, hormonal
therapies and cytotoxic therapies.

12 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

RESEARCH AND DEVELOPMENT ACTIVITIES

Emerging biopharma companies are developing more than 90% of

Next-Generation Biotherapeutics in the late-stage pipeline

Exhibit 8: Number of Next-Generation Biotherapeutic Pipeline Products in Late-Stage Pipeline by Company

Segment, 2008–2018

250

200

150

100

50

0
2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

EBP Non-EBP

Source: IQVIA Pipeline Intelligence, Dec 2018; IQVIA Institute, Mar 2019

• Next-Generation Biotherapeutics (NGB) – defined as • Emerging biopharma companies accounted for 92%
cell, gene and nucleotide therapies – make up less of the late-stage NGB pipeline, as the startups who
than 10% of the total late-stage R&D pipeline, but have have pioneered the dozens of cell and gene therapy
more than doubled in number over the past three approaches have continued to innovate and retain
years, as new pathways for disease treatment and control of their technologies.
potential cures command growing attention
• Rather than a distinct drug as the output of research,
and investment.1
some of these technologies will produce a patient
• The total number of NGBs in the late-stage pipeline treatment personalization methodology that must
reached 269 by the end of 2018, up from 182 in 2017, as then be scaled up for the market. It is likely that large
a number of products moved from Phase I to Phase II. proportions of these therapeutics will ultimately be
licensed to larger companies for commercialization.

Chart notes: Late-stage pipeline is defined as active programs (activity in past three years) in Phase II through Registered. Next-Generation Biotherapeutics
defined as cell and gene therapies or nucleotide therapies with mechanisms including: cell therapy, dendritic cell therapy, NK cell therapy, T-cell therapy, CAR-T-
cell therapy, T-cell receptor therapy, stem cell therapy, bacterial cell therapy, CIK cell therapy, CIK-CAR therapy, whole cell vaccine, dendritic cell vaccine, bacterial
cell vaccine, DNA vaccine, RNA vaccine, exon skipping, nucleic acid-based, gene therapy, oligonucleotide, antisense, RNAi, microRNA mimic, gene editing,
CRISPR-Cas9, zinc finger nuclease, RNA therapy, and mRNA therapy.
1
IQVIA Institute. The Changing Landscape of Research and Development: Innovation, Drivers of Change, and Evolution of Clinical Trial Productivity. Apr 2019.
Available from: https://www.iqvia.com/institute/reports/the-changing-landscape-of-research-and-development

iqviainstitute.org | 13
RESEARCH AND DEVELOPMENT ACTIVITIES

Emerging biopharma ran 65% of clinical trials in 2018 and are now
running more trials than larger companies across all phases

Exhibit 9: Number of Clinical Trials by Phase and Company Segment

All Phases 1,500 Phase I

5,025 1,000
4,756 4,683
500
4,215 0
4,051 4,098 4,005
3,799 3,902

1,735
1,500 Phase II
1,884

1,812
1,000
1,943

3,290 500
1,991

2,872

2,871
2,016
2,311

0
2,480
2,490

2,272

1,500
1,911
1,783
1,694

Phase III
1,561

1,618

1,000
500
0
2010 2011 2012 2013 2014 2015 2016 2017 2018 2010 2011 2012 2013 2014 2015 2016 2017 2018

EBP Non-EBP

Source: Clarivate Analytics Cortellis, May 2019; IQVIA Institute, May 2019

• From 2010 to 2018, the number of clinical trials run • The percentage of emerging biopharma run clinical
by emerging biopharma companies has more than trials has increased across all therapy areas. In
doubled to 3,290 trials, representing 65% of trials particular, the share of emerging biopharma
started in 2018. trials out of all trials has increased significantly in
endocrinology, psychiatry, respiratory, rheumatology
• The greatest increases in the number of emerging
and transplantation, with growth rates of over 100%
biopharma trials has been in Phase I and Phase II, with
from 2010 to 2018.
2.5 times more trials and 2.1 times, respectively.
• The EBP share of oncology trials has increased from
• The number of Phase III trials being run by emerging
43% in 2010 to 65% in 2018, demonstrating that
biopharma has increased more slowly, but has still
emerging biopharma companies are becoming more
grown 75% since 2010.
capable of running these more complex studies.

Chart notes: Phase II includes Phases I/II, II, IIa, IIb. Phase III includes Phase II/III and III. Terminated trials were excluded from the analysis. Totals for 2018
may be reflecting delayed filing of those trials into trial databases. Non-EBP includes companies with R&D spend > $1.5Bn or Rx Sales >$5Bn and companies
with R&D spend between $200Mn - $1.5Bn OR Rx sales between $400Mn - $5Bn. Emerging biopharma (EBP) companies have R&D spend < $200Mn or Rx sales
between $50Mn - $400Mn or number of trials since 2014 > 0. EBP companies in the older years may understate EBP, as the segmentation in this analysis was
conducted in 2018 and does not account for companies’ transactions in prior periods.

14 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

RESEARCH AND DEVELOPMENT ACTIVITIES

The composite success rate for products developed by emerging

biopharma companies is 17% – greater than other company segments
99

Exhibit 10: Average R&D Composite Success Rate and Average Success Rates per Phase, 2014–2018

91% 92%

70% 72%

63%
15%

48% 4%
42%
37%

5%
17%
12%

Phase I Phase II Phase III Pre-Registration Composite

EBP Non-EBP

Source: IQVIA Pipeline Intelligence, Mar 2019; IQVIA Institute, Mar 2019

• The composite success rate of clinical development in Phase II, reflecting the critical role emerging
from Phase I trials to regulatory submission – based on biopharma companies are playing in early-stage
the percent of drugs successfully progressing to each development in R&D.
next stage of development – was 11.4% in 2018 for all
• Larger pharma companies are more likely to acquire or
company segments, down from 14.4% in 2017.1
partner with an emerging biopharma company in later
• Emerging biopharma companies achieved a stages, as the potential of the pipeline candidate has
composite success rate of 17.2%, higher than the been established.
12.1% of other company types, reflecting their greater
• Phase III and pre-registration rates are slightly lower
success across Phase I development relative to other
for emerging biopharma companies. This could
company segments.
indicate that emerging biopharma companies have
• There was a 15.5% difference in success rates fewer resources available to move new products
between emerging biopharma companies and other through later-stage development.
company segments in Phase I, and a 4.3% difference

Chart notes: Numbers may not sum to totals due to rounding.

1
IQVIA Institute. The Changing Landscape of Research and Development: Innovation, Drivers of Change, and Evolution of Clinical Trial Productivity. Apr 2019.
Available from: https://www.iqvia.com/institute/reports/the-changing-landscape-of-research-and-development

iqviainstitute.org | 15
RESEARCH AND DEVELOPMENT ACTIVITIES

The top 30 emerging biopharma companies employ both traditional

and cutting-edge technologies that span across therapy areas

Exhibit 11: Top Emerging Biopharma Companies by Focus in Each Organizational Archetype

US-Based Standalone Companies Hub and Spoke Other

Antibody
/Immunology Nucleic Acids - Evotec (29) - Alphamab (29)
- Inovio (26) - Ionis Pharmaceuticals Takes equity in companies - Bio-Thera Solutions
- Kymab (23) (59) in major therapy areas, (21)
- Agenus (21) - Sarepta including metabolic - Lee’s Pharmaceutical
- Seattle Genetics (21) - Therapeutics (18) diseases, inflammation, (20)
- MacroGenics (19) and oncology - HEC Pharm (19)
Small Molecule - Cellix Bio (24)
Gene Technologies - Galápagos (22) - Fortress Biotech (19)
- 3SBio (19)
- Ligand Pharmaceuticals Therapy area-agnostic
(30) company, investing in
Antibody - Sosei Heptares (22)
- Intrexon (19) technologies in addition
/Immunology to pharmaceuticals
- CRISPR - Yuhan (34)
- Sorrento
Therapeutics (18) - Daewoong
Therapeutics (21) - Roivant Sciences (18)
- Precision Pharmaceuticals (32)
Biosciences (17) Multiple therapy areas, - GC Pharma (23)
diverse interests, - Jeil Pharma Holdings
integrated holding (21)
Nucleic Acids
company
- Moderna Therapeutics (19)
- WAVE Life Sciences (18) - BIOCAD (22)

Source: IQVIA Pipeline Intelligence; IQVIA Institute, May 2019

• There are currently 3,212 emerging biopharma • “Hub and Spoke” companies are also U.S.-based but
companies collectively investigating 6,965 assets. distinctly have centralized corporate functions, but
Of these, 47% are investigating a single asset, while separate subsidiaries for assets. These companies
another 40% are investigating 2–10 assets each. have invested in a wide range of therapy areas, and
capitalize on their management and organizational
• The top 30 emerging biopharma companies by
hub model to optimize operations.
number of compounds/programs in any stage of R&D
account for 703 products, or 8.1% of all products under • Another twelve companies are ex-US based companies
investigation. developing biobetters and/or biosimilars, or in-
licensing assets for marketing in emerging markets.
• All of the U.S.-based companies are currently
developing medicines and indicate that they will • Oncology is the major focus for 21 of the top 30
pursue licensing or partnership to commercialize companies but areas of focus also include therapy areas
them, though some have pursued marketing of their such as infectious disease, rare diseases and vaccines.
assets directly. A prime example of this archetype is
• Companies in the top 30 specializing in gene
Ionis, the largest of any emerging biopharma company
technologies, including gene therapy, gene editing and
with 59 assets in development.
epigenetics, are found exclusively in the United States.

Chart notes: Top 30 companies were determined by number of products in any stage of development, ranging from discovery to registration. Does not take
into account assets being investigated for multiple indications in different therapy areas.

16 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

 Commercialization performance

• Emerging biopharma have patented 29 of the top 100 • For newly launched active substances originated by
drugs, which account for 40% of sales in the United EBP companies, more address areas of high unmet
States in 2018. need; 69% of EBP originated drugs were focused on
areas of high unmet need compared to 65% of drugs
• Emerging biopharma companies originated and
from other companies.
launched 42% of new drugs in 2018, a higher
percentage than in past years, up from 26% in 2017. • Launch performance varies significantly based
on unmet need in the market and level of product
• Of the 59 new active substance (NAS) launches in 2018,
differentiation
64% were originated by emerging biopharma.
• Emerging biopharmas generally achieve lower average
• Over half of EBP-launched drugs had orphan
sales when launching new active substances than
designation and almost a quarter were approved
other companies.
based on a single-arm trial.
• Average quarterly sales uptake at one year after launch
• The median time for EBP companies to launch new
is 2.6 times higher for larger companies launching
drugs was 16.6 years in 2018, over 30% slower than
emerging biopharma-originated products than for
other company segments.
emerging biopharma companies who develop and
• EBP-originated products generally reach the market launch their own assets, widening to 6.5 times higher
faster if they were acquired. Particularly assets that 18 months later.
were initially owned by an EBP but submitted and
subsequently launched by a large pharma company
spent less time in development compared to those
owned, developed and launched by an EBP.

iqviainstitute.org | 17
COMMERCIALIZATION PERFORMANCE

Emerging biopharma have patented 29 of the top 100 drugs, which

account for 40% of sales in the United States in 2018

Exhibit 12: Sales of the Top 100 Drugs in the United States in 2018 by Originator Company Segment

Number of Drugs Aggregate Sales

N=100 N=$216.1Bn
100%

80%
129.2
71
60%

40%

20% 86.9
29

0%

EBP Originated Non-EBP Originated

Source: IQVIA MIDAS, Dec 2018; IQVIA Institute, Apr 2019

• Emerging biopharma companies originated 29 of the by sales is the so-called ‘quad’ pill for HIV, cobicistat/
top 100 drugs in the United States in 2018 but were elvitegravir/emtricitabine/tenofovir alafenamide
never the company to launch the drugs. (Stribild), with $4.5 billion in 2018 sales in the United
States and ranked 9th overall.
• These larger selling drugs have universally been
launched or marketed by larger companies. • The immuno-oncology checkpoint inhibitors
pembrolizumab (Keytruda) and nivolumab (Opdivo),
• Emerging biopharma originated drugs have a larger
ranked 11th and 12th in sales in the United States in
share of sales at 40%, as they generally average higher
2018, and this illustrates an important dynamic related
sales per product than non-emerging biopharma
to emerging companies, as the history of both drugs
originated drugs.
dates back to smaller emerging companies at one
• Four of the top five drugs in the United States in 2018 point, but arguably the compounds emerged into
were originated by an emerging biopharma company public view at different stages.
but launched by a larger company.
• Nivolumab was developed by the emerging biopharma
• Emerging biopharma originated drugs account company Medarex, while pembrolizumab was
for 55% of the sales in the top 20 and include such developed at Organon, which was a larger company
leading drugs as adalimumab (Humira) and etanercept by the time the compound emerged and was then
(Enbrel), the top two drugs by sales overall and the top purchased in a series of mergers to ultimately be
two autoimmune biologics. marketed by Merck.
• Of launches in the past five years originated by
emerging biopharma companies, the highest ranking

18 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

COMMERCIALIZATION PERFORMANCE

Emerging biopharma companies originated and launched 42% of new

drugs, a higher percentage than in past years and up from 26% in 2017

Exhibit 13: Companies Originating and Filing FDA Regulatory Submissions for NASs and Percent of Launches by
NAS Launch Year
42%
38%
29% 31% 31%
80 26%
25% 24%
19% 20%
25% 25%
22%
21% 19% 22%
60 15% 17%
12% 10%
Number of NASs

40

20

0
2009 2010 2011 2012 2013 2014 2015 2016 2017 2018
Non-EBP for Origination and Launch Non-EBP Originated and EBP Launched
EBP Originated and Another Company Launched EBP Originated and Launched
Percent EBP Originated and Another Company Launched Percent EBP Originated and Launched

Source: IQVIA Institute, May 2019

• In 2018, 64% of the 59 NAS launches were originated • Of the products originated by EBPs, they are retaining
by emerging biopharma. and launching more of them, launching 55% of
originated products in the past five years compared to
• EBPs also originated and launched 42% of new drugs in
41% in the prior five years.
2018, up from 26% in 2017.
• While larger company launches have varied in number
• An increasing number of launches were originated by
significantly, EBP companies have been steadily
EBP companies with 50%, 110 of the 219 launches in
launching products, and in increasing numbers,
the past five years compared to 43% of the 158 in the
reflecting the significant share of the pipeline they
prior five years.
represent and a greater level of strategic interest in
• The 62% increase in the absolute number of EBP marketing their assets themselves.
originated launches in the past five years compared
to the prior five years reflects a sustained level of
investment in these companies over the last few decades.

Chart notes: New Active Substance (NAS) is a new molecular or biologic entity or combination where at least one element is new; NAS launches in the United
States by year of launch regardless of timing of FDA approval. Chart growth lines exclude percent of EBP launched but originated by other companies.

iqviainstitute.org | 19
 COMMERCIALIZATION PERFORMANCE

Over half of EBP-launched drugs had orphan designation and almost

a quarter were approved based on a single-arm trial

Exhibit 14: Percent of EBP- and Non-EBP-Launched NASs in the United States 2015–2018 with Each Feature

Characteristics Approval Attribute Attribute of Approval Trials

Orphan 51% Priority57% Approval Based on 30%

39% Review 59% Phase I or II Trial 32%
Biologic 29% Breakthrough 20%
33% Therapy 35% Single-Arm Trial 24%
13%
Predictive 14% Fast Track 26%
Biomarker 26% 20%
Accelerated 9%
26% Approval 16%

Percent of Non-EBP-Launched Percent of EBP-Launched

N = 82 N = 91
Source: IQVIA Institute, May 2019

• A higher percentage of new drugs launched by • A lower percentage of drugs launched by emerging
emerging biopharma companies from 2015-2018 were biopharma companies had predictive biomarkers,
orphan drugs, were approved based on single arm were biologics, had accelerated approval or were
trials or had fast-track status, as compared with those breakthrough therapies than those launched by non-
launched by non-EBP companies. EBP companies.

• Over half of the 91 NASs launched by EBPs (n=46) in • Non-EBP companies launched nearly double the
this period were orphan drugs, suggesting the scope percentage of NASs with predictive biomarkers
of commercial activities and their likelihood of success (26%, n=21) versus EBPs (14%, n=13) suggesting that
may be more attainable or appealing to small EBP larger companies may see greater value in precision-
companies with limited resources. medicine assets and acquire them at an early-stage
from EBP originators.
• Emerging biopharma companies launched twice
the number of NASs approved based on single-arm • Non-EBP companies launched 61% more breakthrough
trials (n=22), versus launched by non-EBP companies therapies (n=29) than non EBPs (n=18) from 2015-2018.
(n=11), likely driven by the high rates of orphan drugs
• Non-EBP companies launched 13 drugs with
developed where control arms are less utilized.
accelerated approval compared with the 8 drugs
launched by EBPs in this period.

Chart notes: A New Active Substance (NAS) is a new molecular or biologic entity or combination where at least one element is new; includes NASs launched
in the United States in 2015-2018 regardless of the timing of FDA approval. Orphans include drugs with one or more orphan indications approved by the
FDA at product launch. Products are not reclassified as orphan if they subsequently receive an approval for an orphan designated indication. Biologics are
defined by IQVIA as clearly identifiable molecules of biologic origin, including but not limited to products created with recombinant DNA technology and
without necessarily adhering to classifications by regulatory bodies that are sometimes inconsistent with this approach. For regulatory designations (priority,
breakthrough, fast track, accelerated approval, approval based on Phase I or II trial, or single arm trials) these are based on announcements by the FDA.

20 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

COMMERCIALIZATION PERFORMANCE

The median time for EBP companies to launch new drugs was
16.6 years in 2018, over 30% slower than other company segments

Exhibit 15: Median Time Per Year from First Patent Filing to Launch by NAS Launch Year, United States

19

17

15
Time in Years

13

11

5
2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

EBP Launched EBP Originated and Another Company Launched Non-EBP for Origination and Launch

Source: IQVIA Patent Intelligence; IQVIA Institute, May 2019

• Development of new drugs remains a slow process. • In 2018, products originated by an emerging
In the United States in 2018, NASs took a median of biopharma company but launched by a different
13.7 years to launch from the time of their patent company had a median time from first patent to
filing, when assessed across all company segments.1 launch of 12.6 years. This value is similar to products
However, the median time from patent to launch for not originated or launched by emerging biopharma
products launched by emerging biopharma companies companies, at 11.9 years.
is 16.6 years.
• Overall, these patent to launch times suggest that
• From 2013 through 2018, median patent to launch emerging biopharma companies face challenges
timelines have increased 26% for products that when launching products, while other companies
were launched by emerging biopharma companies, have access to resources that allow them to reach the
however, when viewed since 2009, median patent to market sooner.
launch for products within this segmentation has been
relative stable.

Chart notes: Compares the date of patent filing for a medicine to FDA approval for a specific indication. EBP launched includes products that were originated
by an emerging biopharma company or another company segment.
1
IQVIA Institute. The Changing Landscape of Research and Development: Innovation, Drivers of Change, and Evolution of Clinical Trial Productivity. Apr 2019.
Available from: https://www.iqvia.com/institute/reports/the-changing-landscape-of-research-and-development

iqviainstitute.org | 21
COMMERCIALIZATION PERFORMANCE

Emerging biopharma originated products progressed to launch with

a variety of pathways, development events and durations

Exhibit 16: New Active Substances Launched in the U.S. and Originated by Emerging Biopharma

te
xi
bo

lfa

ab
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ar

su
ab

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ib
e
e

em
e
rm

ib

id
id

rib
lin

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in
m

se

t
ib

il

ta
in

am

ez
et

ad
ud

at
ic
ib
ni
yc
lu

pa

ia
en

vi

as
ct
lc

om

an
lis

am
it i
de

eg
xa

al
ac
rs

ut
zo
ca

te
id

al
ric

ve

gv

em
ta

av
lo
na

ap

al
az
os

ro

ig

st
el

la

du
ne
ba

pe
ba

ap
er

fo
m
et

ta

pl

el
la

la
iv

fr
Agios

Dyax / Shire

Kai Pharmaceuticals / Amgen

Shionogi / Roche

Aerie

Tetraphase

Array BioPharma / Loxo

Incyte / Lilly

Lead Therapeutics / BioMarin / Pfizer

Infinity / Verastem

Achaogen

BioMarin

Aragon / Janssen

Rinat Neurosciences / Teva

Amicus

Rigel
Enzon / Sigma Tau/ Leadiant
2018

2015

2010

2005

2000 Emerging Biopharma Rare Pediatric Disease Designation Phase I

Large Pharma Priority Review Phase II

Multiple Emerging Biopharma Together Orphan Designation Phase III

Complete Response Letter Submission by Large Pharma
Large and Emerging Together
Fast Track Designation Submission by EBP

1995 Breakthrough Designation QIDP Designation

Source: IQVIA Patent Intelligence; IQVIA Pipeline Intelligence; IQVIA Institute, May 2019

22 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

COMMERCIALIZATION PERFORMANCE

Emerging biopharma originated products progressed to launch with

a variety of pathways, development events and durations continued

e
at

flo e m
et

la at niu
um

g
ac
g

pa
vo te pa

de ilic co
in
di

n
bo
n

ro vec

e
ab
ib

in
at

Spark Therapeutics lea bo

ab
an
bu rvo e

ci
el
so

Wakunaga / Rib-X/ Melinta los zir

ol
ci

ne
a n
in

ac
rim

xa
om
m

or
na

az
um
a m

ab
e
ix

et

cy um

di
ox
su
ne etig

im

id
m ro
fe

ol

vi

tr

ix
m

xi
iz
en

cn
ve

co
ag

at

ac

su

di
tr
p

fe
ni

al
r

c
be

so
oz
av

se
re

m
te

lu
bi

ib

lo
el
Theravance / Mylan & Theravance

Neurocrine Biosciences / AbbVie & Neurocrine Biosciences

Array BioPharma

Kyowa Hakko Kirin / Ultragenyx

Ardana / AetenaZentaris

Shionogi

Cor Therapeutics / Portola

Toyama / Ferrer/ Medimetriks

UOP LLC / ZS Pharma / AstraZeneca

TaiMed / Biogen

Symbiox

Nordmark Werke / US World Meds

Yamanouchi / Astellas / Eisai / Dova
Siga Technologies

2018 2018

2015 2015

2010 2010

2005 2005

2000 1975

1995 1970

1969

1967

Chart notes: A New Active Substance (NAS) is a new molecular or biologic entity or combination where at least one element is new. There were no mid-sized or
small companies involved in the assets tracked on the prior two pages. Designations flagged on the prior pages are FDA designations including rare pediatric,
fast track, breakthrough, orphan, and qualified infectious disease (QIDP). Priority review are noted when they were announced. Phase I, II, III start have
been included where identified as the first phase start across indications and geographies. Submission is based on the date companies announced filing with
the FDA. Complete response letters are provided by FDA as a conclusion to an application, but allow the applicant to resolve the issues and resubmit, which
occurred in all three instances analyzed. Companies who jointly filed with FDA have been indicated with combined coloring in the timeline.

iqviainstitute.org | 23
COMMERCIALIZATION PERFORMANCE

Emerging biopharma originated products progressed to launch with

a variety of pathways, development events and durations continued

• A subset of 33 NAS products across therapy areas • Certain attributes, such as orphan designation or
are shown, demonstrating differences between breakthrough designation, are not awarded until the
the development and regulatory journey of assets end of the development cycle, and therefore have little
developed and launched by EBPs versus those impact on the overall development timeline. Similarly,
licensed/sold to a large company. Ten of these fast track designation is awarded relatively late in the
products originated with an EBP, but were launched developmental timeline and does not have a strong

by a large company, while the remaining 23 products impact on the overall timeline, though it may increase

were developed and launched by an EBP. the probability of approval.

• Assets initially owned by an EBP but submitted and • While priority review lessens the time from submission
to launch, there is little effect on the overall
subsequently launched by a large pharma company
development time, as products with both short and
generally spent less time in development compared
long developmental timelines received priority review.
to those owned, developed and launched by an EBP.
Large pharma companies entered into partnerships • Additionally, the receipt of a complete response letter,
and/or acquisitions with EBPs in late-stage while lengthening the time from initial submission to
development, when risks associated with early-stage launch, does not necessarily significantly impact the
development were resolved. development timeline.

• Phases I and II appear to be the biggest determinants • Products with high unmet need, such as netarsudil
of time to launch, as Phase III timeline is relatively for glaucoma, and eravacycline for certain antibiotic-
consistent. resistant infections, demonstrated a relatively quick
and uncomplicated developmental journey, despite
• Partnerships between EBPs or an EBP and large
a lack of designations designed to lessen time to
pharma only occurred in Phase III or at submission
approval and launch.
in this subset, when developmental and regulatory
timelines are less variable. • Novel products, such as the gene therapy voretigene
neparvovec, do not necessarily experience significant
• The development time of products with more than development time increases or decreases, as
two companies involved appears to lengthen, as seen development of this product is consistent with the
with ozenoxacin, sodium zirconium cyclosilicate median time from first patent to launch.
and delafloxacin.
• The variation in time between submission and launch
• Patent shelf delays developmental time, however, once reflects both the length of time for regulatory review
development begins, there is little to no effect on time and, in some cases, delays while reimbursement issues
from Phase I to launch. for the product are resolved prior to the launch.

24 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

COMMERCIALIZATION PERFORMANCE

For newly launched active substances originated by EBP companies,

more address areas of high unmet need

Exhibit 17: U.S. New Active Substance Launches by Originating Company Type, 2008–2017

Low Market Unmet Need High

High
6% 8% 7% 32% 28% 30%

Product Differentiation
Low
24% 30% 27% 37% 37% 36%

EBP Originated Non-EBP Originated Total % Quadrant % of Type

Source: IQVIA. Creating a framework for a successful launch: Planning and preparing. Jul 2018
Available from: https://www.iqvia.com/blogs/2018/07/creating-a-framework-for-a-successful-launch

• Of the 291 NAS assessed launches between 2008 and companies have demonstrated an ability to identify and
2017, 139 were originated by an emerging biopharma demonstrate the value of these products. EBP companies
company, and 152 by other companies. filed the specific patents on 53% of the 262 non-NAS
products launched between 2008 and 2017 (not shown).
• EBPs originate more differentiated, high unmet need
medicines, 32% of all of their launches and 45 products • Sixty percent of non-NAS launches were in areas with
overall compared to 43 medicines or 28% of non-EBP lower unmet need and with relatively low product
originated drugs. differentiation, and EBPs launched 92 of these 165
drugs, suggesting an important sub-segment of EBP
• Overall, 69% of EBP originated drugs were focused on
companies are pursuing niche markets with more
areas of high unmet need compared to 65% of drugs
complicated marketing messages.
from other companies.
• Of the 15 non-NASs, which were both highly
• Of the most differentiated and unmet need drugs
differentiated and filled a high unmet need, EBPs
originated by EBPs, 60% (27) of them were licensed or
originated 12 of them and launched seven of them,
sold to and launched by other companies.
licensing or selling the other five, and other companies
• EBPs retained 45 of the 85 drugs they developed, developed and launched the other three.
which were of lower product differentiation, perhaps
• Of the 64 drugs with high unmet need but lower
reflecting less market interest in these assets.
product differentiation, EBPs originated 41 of them
• Non-NAS products are generally reformulations and launched 26, as well as launching a further seven
or combinations of existing medicines, and EBP originated by other companies.

Chart notes: A New Active Substance (NAS) is a new molecular or biologic entity or combination where at least one element is new.

iqviainstitute.org | 25
COMMERCIALIZATION PERFORMANCE

Launch performance varies significantly based on unmet need in

the market and level of product differentiation

Exhibit 18: EBP Launch Archetypes and Average First-Year Sales 2008–2017

Low Market Unmet Need High

High
Product Differentiation
MARKET SHAPING SCIENCE SELLS
$74Mn $128Mn
LAUNCH
ARCHETYPES

WHO BENEFITS EMPHASIZE THE DIFFERENCE

$30Mn $46Mn

Low
Source: IQVIA. Creating a framework for a successful launch: Planning and preparing. Jul 2018.
Available from: https://www.iqvia.com/blogs/2018/07/creating-a-framework-for-a-successful-launch

• In analyzing launches, the characteristics of the action relative to the current standard of care were all
therapy area and the product highlight some distinct assessed to determine a product differentiation result.
differences in the market environment and potential
• For non-emerging biopharma launches, the average
of the products. The names of the resulting quadrants
first-year sales for the “Science Sells” group is $174
suggests common market approaches to drugs fitting
million, 36% higher than emerging biopharma
those attributes.
companies, which average $128 million. In the ‘Market
• “Unmet Need” in a therapy area includes the efficacy Shaping’ group, first-year sales are $105 million, 42%
of the standard of care, as well as some of the existing higher than EBPs at $74 million.
challenges in treating patients, such as side effects,
dosing, overall quality of life and the disease’s burden • The “Emphasize the Difference” products, when launched

to the health system. by larger companies averaged 63% higher first year sales
at $75 million compared to $46 million by EBPs.
• “Product Differentiation” was assessed based on
each launched product’s clinical improvement • “Who Benefits” products, which are undifferentiated with
over the standard of care at the time of launch, as low overall unmet need, launched by EBP companies
demonstrated in clinical trials. Tolerability, dosing averaged $30 million in first-year sales, less than half of
advantages and the novelty of the mechanism of the $74 million averaged by larger companies.

Chart notes: Viral hepatitis excluded from averages.

26 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

 COMMERCIALIZATION PERFORMANCE

Emerging biopharmas generally achieve lower average sales when

launching new active substances than other companies

Exhibit 19: New Active Substances Launched 2014–2018 Originated or Launched by EBP Companies

70
Average Quarterly Sales US$Mn

60

50

40

30

20

10

0
0 1 2 3 4 5 6 7 8 9 10
Quarters Since Launch

EBP Originated and Other Companies Launched EBP Originated and Other Companies Launched Excluding
EBP Originated and Launched Ledipasvir/Sofosbuvir (Harvoni)
Originated by Other Companies and Launched by EBP
Source: IQVIA MIDAS, Dec 2018

• Some of the most successful new drugs of the past • A quarter of the NASs launched by emerging
five years were discovered by emerging biopharma biopharma companies have been in areas of low
companies and later launched by larger companies. unmet need and low product differentiation,
correlating with lower average sales.
• For launches in the United States since 2013, excluding
ledipasvir/sofosbuvir (Harvoni), the average quarterly • For emerging biopharma-launched products, only six
sales uptake at one year after launch is 2.6 times have a quarterly sales period above $20 million in the
higher for larger companies launching emerging five years after launch, with three achieving that within
biopharma-originated products than for emerging a year of launch.
biopharma companies who develop and launch their
own assets, and 6.5 times higher just 18 months later.

• An increasing number of emerging biopharma

launches are orphan drugs, often with low sales, as
target populations are often small and hard
to diagnose.

iqviainstitute.org | 27
Strategic transactions

• U.S. venture capital activity in life sciences has been • Licensing deals for therapeutics typically occur earlier
rising in absolute terms and the number of deals. in a product’s development, though 2018 had some
notable late-phase deals.
• In 2018, 1,308 life science venture capital deals were
closed with an overall value of over $23 billion, an all- • Discovery stage and Phase I grew 10% and 20%,
time high resulting from a sharp increase in the past respectively, from 2017 to 2018, and Phase III licensing
five years, represented by a five-year CAGR of 15%. activity decreased by 19% from 2017 to 2018, with 50
deals signed in 2018 compared to 62 in 2017.
• Collaborative R&D deals in 2018 all involved large
companies partnering with EBPs. • There was a 78% increase in deals at pre-registration,
up to 62 deals from 36 in 2017.
• The number of collaborative deals has been declining
while absolute deal values have risen. • Seven of the top ten deals in 2018 involved an emerging
biopharma company.
• The number of disclosed collaborative R&D deals fell by
12% from 2017 to 2018, while the aggregate total value • Deals announced in 2018 included 415 in-licensing or
of those deals increased to $47.3 billion, with the mean in-bound partnership agreements totaling $272 billion
total deal value increasing 8% in 2018 to $569 million. in agreed payments

• EBPs partnering with large companies accounted for • On average, companies carried out 9.2 deals each, with
nine of the top ten deals in 2018. deals summing to an average of $6 billion per company,
and the median deal amount at $2.6 billion.

28 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

STRATEGIC TRANSACTIONS

U.S. venture capital activity in life sciences has been rising in

absolute terms and the number of deals

Exhibit 20: U.S. Venture Capital Deal Value in $Bn and Number of Deals Closed

1,289 1,273 1,308

1,240 23.3
1,168 1,141
1,094
1,039
963
16.6
873 871
828 14.9

12.2 12.6

9.6 9.9
9.3 8.8 8.7
7.9 7.8

2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

Deal Value (US$Bn) Number of Deals Closed

Source: National Venture Capital Association. Accessed Dec 2018. Available from: https://nvca.org/research/research-resources/

• In 2018, 1,308 life science venture capital deals were • Growth in 2018 was, in part, due to a strong period
closed with an overall value of over $23 billion. of performance on public markets, including seven of
the ten largest IPOs in Q4 coming from the healthcare
• Life science venture capital deal values have grown
sector.2
sharply in the past five years, with a five-year CAGR
of 15%. • The increase in number and magnitude of venture
capital deals has created a positive funding
• Venture capital deals have been rising steadily
environment for emerging biopharma companies,
since 2007, following a dip in 2016 in venture capital
allowing them to delay the decision to partner or
investment, in part due to uncertainties around the
develop an asset alone.
U.S. election.1

• Despite a drop in 2016, the number of deals have

rebounded since then and are up 15% – now higher
than any other year – while the corresponding deal
value nearly doubled from 2016.

Chart notes: CAGR = Compound annual growth rate.

1
KPMG Enterprise. Venture Pulse 2016. Global Analysis of Venture Funding. 2017 Jan 12. Available from: https://assets.kpmg/content/dam/kpmg/xx/
pdf/2017/01/venture-pulse-q4-2016-report.pdf
2
National Venture Capitol Association. NCVA blog. 2019 Jan 15. Available from: https://nvca.org/blog/8-takeaways-8-graphics-historic-2018-venture-capital/

iqviainstitute.org | 29
STRATEGIC TRANSACTIONS

Collaborative R&D deals in 2018 all involved large companies

partnering with EBPs

Exhibit 21: Top 10 Therapeutic Collaborative R&D Deals of 2018 by Total Potential Deal Value

TOTAL DEAL UPFRONT DEVELOPMENT

COMPANIES INTEREST AREA
VALUE PAYMENT PHASE
$96 Mn in an Natural killer cell engager-based
upfront payment cancer immunotherapeutics using
$5,096 Mn Affimed, Genentech Discovery
and near-term Affimed’s ROCK® (Redirected
committed funding Optimised Cell Killing) platform

$200 Mn ($100 Mn Dicerna RNAi therapies for cardio-metabolic

$3,705 Mn cash and $100 Mn Pharmaceuticals, disease, neurodegeneration and pain Discovery
equity investment) Eli Lilly utilising Dicerna’s GalXC™ platform

Sangamo
Cell therapies for cancer treatment
Therapeutics, Kite
$3,150 Mn $150 Mn using Sangamo’s zinc finger nuclease Discovery
Pharma/Gilead
(ZFN) genome editing technology
Sciences

Tango Immuno-oncology therapies derived

$1,750 Mn $50 Mn Therapeutics, from Tango’s functional genomics- Discovery
Gilead Sciences based discovery platform

Immatics Bispecific cancer immunotherapies

$1,704 Mn $54 Mn Biotechnologies, discovered using Immatics’ Discovery
Genmab Xpresident ® technology

Sutro Biopharma, Immune-modulating therapies for

$1,670 Mn $60 Mn Discovery
Merck & Co. cancer and autoimmune disorders

$110 Mn ($80 Mn Inhibitors of transforming growth

Scholar Rock,
$1,530 Mn cash and $30 Mn factor beta (TGFβ) activation for the Discovery
Gilead Sciences
equity investment) treatment of fibrotic diseases

OSE Immuno-
€1,130 Mn therapeutics, OSE-172 for the treatment of advanced
€15 Mn ($18.4 Mn) Preclinical
($1,389 Mn) Boehringer solid tumors
Ingelheim
$125 Mn in an
SQZ
upfront payment Antigen presenting cell therapies for
$1,375 Mn Biotechnologies, Discovery
and near-term the treatment of oncology indications
Roche
milestones

Off-the-shelf chimeric antigen

Fate Therapeutics,
$1,320 Mn $10 Mn receptor T-cell (CAR-T) therapies for Discovery
Ono Pharmaceutical
cancer treatment

Source: IQVIA. IQVIA Pharma Deals - Review of 2018. Mar 2019. Available from: https://www.iqvia.com/library/white-papers/iqvia-pharma-deals--review-of-2018

30 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

STRATEGIC TRANSACTIONS

The number of collaborative deals has been declining,

while absolute deal values have risen

Exhibit 22: Number of Collaborative R&D Deals and Aggregate Value and Mean Total Deal Value of Collaborative
R&D Deals

900 50,000 600

Total Value of Collaborative R&D Deals (US$Mn)

800 45,000
500

Mean Total Deal Value (US$Mn)

700 40,000

600 35,000 400

Number of Deals

500 30,000
300
400 25,000

300 20,000 200

200 15,000
100
100 10,000

0 0 0
2014 2015 2016 2017 2018 2014 2015 2016 2017 2018

Aggregate Total Deal Value Mean Total Deal Value

Source: IQVIA. IQVIA Pharma Deals - Review of 2018. Mar 2019. Available from: https://www.iqvia.com/library/white-papers/iqvia-pharma-deals--review-of-2018

• The number of disclosed collaborative R&D deals fell • More recently, rather than collaborating on research
by 12% from 2017 to 2018, and the aggregate total and development, some big pharma firms prefer
value of those deals, excluding royalties, reached its to fund early-stage research at biotech companies
highest level of the last five years in 2018, peaking at via option-based deals that leave the R&D in the
$47.3 billion. hands of the biotech company until a defined point
in development. Like collaborative R&D deals, these
• The mean total deal value (excluding royalties) of those
agreements are usually high in total value but
collaborative R&D deals with disclosed financial terms
heavily backloaded.
increased 8% in 2018 to $569 million, also a five-year
high, and included 16 deals with a total value more than • One such deal in 2018, potentially worth $759 million,
$1 billion in 2018, up from 12 deals in 2017 at that value. is the collaboration agreement between Ionis
Pharmaceuticals and Roche to develop the antisense
• Although there were fewer collaborative R&D deals
therapy IONIS-FB-LRx for the treatment of
signed in 2018, on average they were of higher total
geographic atrophy (i.e., atrophic age-related macular
value, and moreover, the median total deal value for
degeneration [AMD]) and other complement-mediated
such deals has increased 157% over the past five years
diseases.
to $320 million in 2018 – only slightly lower than the
high of $322 million in 2015.
Chart notes: Collaborative deals are defined here as discovery or preclinical stage deals that involve two or more parties actively collaborating on R&D.
Disclosed value of deals excludes multicomponent deals where it is not possible to split out the financial terms of the research collaboration element.
TVD = total deal value

iqviainstitute.org | 31
STRATEGIC TRANSACTIONS

EBP partnering with large companies accounted for 9 of the top 10

deals in 2018

Exhibit 23: Top Partnering Deals (Excluding Settlements and Product Acquisitions) of 2018 by Upfront Consideration

TOTAL DEAL UPFRONT DEVELOPMENT

COMPANIES INTEREST AREA
VALUE PAYMENT PHASE
$1,850 Mn ($1,000 Nektar Joint development and
Mn cash upfront Therapeutics, commercialization of NKTR-214 in
$3,630 Mn Phase II
and $850 Mn equity Bristol-Myers combination with Opdivo® (nivolumab)
investment) Squibb and Opdivo plus Yervoy ® (ipilimumab)
$1,000 Mn ($375
Ionis
Mn cash upfront Antisense drug candidates for
$1,270 Mn Pharmaceuticals, Discovery
and $625 Mn equity neurological diseases
Biogen
investment)
Arena
Pharmaceuticals, Ralinepag for the treatment of
$1,200 Mn $800 Mn Phase III
United pulmonary arterial hypertension
Therapeutics

$750 Mn ($300 Co-development and co-

Mn cash upfront commercialization of Lenvima®
Launched,
$5,755 Mn and $450 Mn Eisai, Merck & Co. (lenvatinib mesylate), both as
Clinical stage
reimbursement for monotherapy and in combination with
R&D expenses) Keytruda® (pembrolizumab)

$500 Mn ($300 Mn
Cusatuzumab for oncology indications
cash upfront and Phase II,
$1,800 Mn Argenx, Cilag including acute myeloid leukaemia and
$200 Mn equity Phase I/II
high-risk myelodysplastic syndrome
investment)

ARO-HBV for the treatment of

$250 Mn ($175 Mn Arrowhead hepatitis B virus infection; RNAi
cash upfront and Pharmaceuticals, therapeutics using Arrowhead’s
$3,750 Mn Phase I/II, Discovery
$75 Mn equity Janssen TRiM™ (Targeted RNAi Molecule)
investment) Pharmaceuticals platform for undisclosed therapy
areas

$200 Mn ($100 Mn
Dicerna RNAi therapies for cardio-metabolic
cash upfront and
$3,705 Mn Pharmaceuticals, disease, neurodegeneration and pain Discovery
$100 Mn equity
Eli Lilly utilising Dicerna’s GalXC™ platform
investment)
$170 Mn ($110 Mn
Wave Life
cash upfront and Nucleic acid therapies for CNS Phase I/II, Preclinical,
$2,230 Mn Sciences, Takeda
$60 Mn equity disorders Discovery
Pharmaceutical
investment)
$150 Mn ($100 Mn
cash upfront and Therapies for a broad range of
$2,262.5 Mn Prothena, Celgene Discovery
$50 Mn equity neurodegenerative diseases
investment)

Ionis
Inotersen and IONIS-TTR-LRx for Pre-registration,
$1,880 Mn $150 Mn Pharmaceuticals,
transthyretin amyloidosis Preclinical
Akcea Therapeutics

Source: IQVIA. IQVIA Pharma Deals - Review of 2018. Mar 2019. Available from: https://www.iqvia.com/library/white-papers/iqvia-pharma-deals--review-
of-2018

32 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

STRATEGIC TRANSACTIONS

Licensing deals for therapeutics typically occur earlier in a product’s

development, though 2018 had some notable late-phase deals
99

Exhibit 24: Therapeutic Licensing Deals by Development Stage, 2017 Versus 2018

200
Number of Licensing Deals

150

100

50

0
Discovery Preclinical Phase I Phase II Phase III Pre-Registration Registered/ Launched Undisclosed
Approved

2017 2018

Source: IQVIA. IQVIA Pharma Deals - Review of 2018. Mar 2019. Available from: https://www.iqvia.com/library/white-papers/iqvia-pharma-deals--review-of-2018

• An analysis comparing the licensing activity by • Phase III licensing activity showed a 19% decrease
developmental stage for therapeutic programs in from 2017 to 2018, with 50 deals signed in 2018
2017 and 2018 demonstrates reduced licensing in compared to 62 in 2017.
most stages; however, there was an increase in
• Interestingly, there was a 78% increase in deals at pre-
licensing activity for assets in discovery, Phase I,
registration, up to 62 deals from 36 in 2017. A majority
and pre-registration.
of these were settlement deals on patent litigation for
• Discovery stage and Phase I grew 10% and 20%, generic equivalents.
respectively, from 2017 to 2018, while the level of
licensing activity for preclinical and Phase II programs
remained stable.

iqviainstitute.org | 33
STRATEGIC TRANSACTIONS

Seven of the top 10 deals in 2018 involved an emerging biopharma

company

Exhibit 25: Top Mergers and Acquisition (M&A) Deals in 2018 Ranked by Total Deal Value

TOTAL DEAL VALUE COMPANIES DEAL DRIVER

Increased footprint in US market, expanded portfolios in

£46 Bn ($62.3 Bn) Takeda Pharmaceutical, Shire
neuroscience and gastroenterology, rare disease franchise

$13 Bn GlaxoSmithKline (GSK), Novartis Full ownership of consumer healthcare joint venture

$11.6 Bn Sanofi, Bioverativ Haemophilia franchise, rare disease pipeline assets

$9 Bn Celgene, Juno Therapeutics Cellular immunotherapy platform

$8.7 Bn Novartis, AveXis Phase III gene therapy for spinal muscular atrophy

Fedratinib, a Phase III Janus kinase 2 (JAK2) inhibitor for

$7 Bn Celgene, Impact Biomedicines
myelofibrosis

Expanded oncology pipeline including Zejula® (niraparib), a

$5.1 Bn GSK, Tesaro
selective poly ADP-ribose polymerase PARP) inhibitor

Nanobody ® technology platform, strengthened pipeline in

€3.9 Bn ($4.8 Bn) Sanofi, Ablynx
rare blood disorders

€3.4 Bn ($4.2 Bn) Procter & Gamble, Merck KGaA Expansion of consumer health business

€3.03 Bn ($3.5 Bn) CVC Capital Partners, Recordati Growth in orphan disease and specialty care markets

Source: IQVIA. IQVIA Pharma Deals - Review of 2018. Mar 2019. Available from: https://www.iqvia.com/library/white-papers/iqvia-pharma-deals--review-
of-2018

34 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

STRATEGIC TRANSACTIONS

Deals announced in 2018 included 415 in-licensing or in-bound

partnership agreements totaling $272 billion in agreed payments

Exhibit 26: Companies by Aggregate Value of All Disclosed Deals in 2018

Bubble size: Aggregate

5.0 deal value US$Bn
4.5 75
Procter & Gamble
Average Deal Value US$Bn

4.0 25

3.5 CVC Capital Partners 5

Takeda 1
Colfax
3.0
Fortive
2.5
GSK
2.0 Sanofi
Aurora Lilly
1.5 Cannabis
Boston Scientific Celgene
1.0
Biogen AstraZeneca
Novartis
0.5 Gilead Roche
BMS Pfizer J&J
AbbVie Merck & Co.
0.0
0 5 10 15 20 25 30 35 40 45
Number of Deals with Monetary Disclosure
Source: IQVIA. IQVIA Pharma Deals - Review of 2018. Mar 2019. Available from: https://www.iqvia.com/library/white-papers/iqvia-pharma-deals--review-of-2018

• Large pharma continues to acquire or license assets • In contrast, GSK, Sanofi, and Celgene carried out fewer
between themselves and with EBPs. Of the 45 deals, with 11, 13, and 16, respectively, though average
companies assessed, 415 deals were transacted in value per deals ranged from $1.1 to $1.6 billion. These
2018, for an aggregate disclosed value of $272 billion. differences between large companies reflect differing
company strategy and business approaches.
• On average, companies carried out 9.2 deals each, with
deals summing to an average of $6 billion per company, • A small group of companies carried out a single deal
and the median deal amount at $2.6 billion. each in 2018, though these deals were some of the
largest. These include Proctor & Gamble’s acquisition
• Takeda’s acquisition of Shire was the largest deal,
of Merck’s consumer health business at $4.2 billion,
with Takeda spending $62 billion to acquire the rare
and CVC Capital Partners acquisition of Recordati, a
disease-focused Shire.
rare disease company, for $3.5 billion.
• Roche and Johnson & Johnson were the most prolific
• Similarly, Colfax acquired DJO Global, an orthopedic
deal-making companies, with 37 and 42 deals,
company for $3.15 billion, and Fortive acquired
respectively. However, their average deal values were
Johnson & Johnson’s advanced sterilization products
$396 million and $239 million, some of the lower
average aggregate deals seen. business for $2.8 billion.

Chart notes: Disclosed deals excluding out-licensing deals and divestments by such companies are included. Number of deals includes some without
disclosed deal-value and average deal value is the total disclosed value divided by all deals including those without disclosed value. For example, of the 21
deals involving Takeda, only seven had values disclosed in the public domain totaling $67.8 billion. Celgene and BMS are treated as separate companies and
their expected merger excluded from this analysis, as the deal remains ongoing.

iqviainstitute.org | 35
Profile of the 2008 Series A financing cohort

• Startups received funding in 2008 across a range of • Forty-three companies have been acquired since initial
therapy areas, including high profile, including next- financing; more than half by other emerging biopharma
generation biotherapeutics, and at varied funding companies.
levels, with half occurring in three U.S. states
• Over half of financed companies have molecules in
• The 168 companies received $1,685 million in financing late-stage development.
in 2008, with 87% of that funding U.S.-based, and
• Some companies are focused on drug-discovery
over half of the companies headquartered in either
platforms, and have licensed the drugs resulting from
California, Massachusetts or New Jersey.
the platform or partnered with other companies,
• Thirty-seven percent of the companies received demonstrating not all companies are focused on
more than $10 million in 2008, accounting for 80% of following assets from discovery to the market.
total funding.
• Six drugs from five companies were approved within
• Ten years after initial financing, 51% of companies were ten years of financing.
privately held.
• Five of the six drugs have launched in the United States,
• Seventeen percent of the companies (28/168) have all but one approved after May 2016.
gone public within ten years of their initial funding,
• Two of these drugs have more than $100 million in 2018
having received $413 million in initial financing and are
sales were launched in 2014 and 2016, while the other
now valued at over $14 billion.
drugs were launched more recently.
• Of those twenty-eight publicly traded companies,
five now have market capitalization of over
$1 billion dollars.

36 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

PROFILE OF THE 2008 SERIES A FINANCING COHORT

Startups received funding in 2008 across a range of therapy areas

and at varied funding levels, with half occurring in three U.S. states

Exhibit 27: Companies Receiving Series A Financing in 2008 by Feature

Therapy Area Location Funding Level Funding Level % of Total Funding
N = 168 N = 168 N = 168 N = $1.7Bn
MA 16% 11% 5%
20% 20%
27% CA 15%
27%
16%
NJ 38%
5%
11% 12%
1%
2% 26%
4% 10%
3% 9% Ex-US
13%
4%
5% 8% Others
6% 35 % 21% 30%

Oncology Anti-Infectives CO 6 OH 3 CT 1 $25–$60Mn

NGBs CNS WA 6 WI 2 VA 1 $15–$25Mn
NY 6 KY 2 NH 1
Autoimmune Cardiovascular PA 5 IL 2 OK 1 $10–$15Mn
Metabolic Ophthalmology NC 5 MD 2 TN 1 $5–$10Mn
Dermatology Diabetes TX 4 FL 1 RI 1 $0–$5Mn
MI 4 UT 1
NASH Other Not Disclosed
GA 3 HI 1

Source: BIO: Emerging Company Investment and Deal Trends, 2009-2018, May 2019; IQVIA Institute, May 2019

• The 168 companies that received Series A financing • Over half of the companies are headquartered in
in 2008 were focused in a range of therapies, some of either California, Massachusetts or New Jersey, with
which have since become very high-profile, including another 16 companies based in the northeast corridor
Next-Generation Biotherapeutics like stem cells, gene from Maryland to New England.
therapy and RNAi technologies.
• Other parts of the United States and the world have
• The largest area of focus for these companies was had less success in building investor interest in large
oncology, mirroring its rise in importance to the overall numbers of startups, with only 13% of companies
pipeline since 2008. outside the United States.

• Two companies were focused in non-alcoholic • Companies received widely varying amounts of initial
steatohepatitis (NASH) and non-alcoholic fatty liver financing, with 37% of the companies receiving more
disease (NAFLD), an area with a significant ongoing than $10 million in 2008, but accounting for 80% of
research program in the broader industry. total funding.

Chart notes: CNS = Central Nervous System. Initial investment in Series A financing was identified for 168 companies receiving funding in 2008. Funding
amounts were disclosed for 144 of the 168 companies and are included only for the initial financing in 2008. Two companies (Oceana and Lumavita) had
received some funding and had approved products prior to 2008. Subsequent financing after 2009 has not been included. Therapeutic area of focus has been
assessed based on the lead compound or platform at the time of financing.

iqviainstitute.org | 37
PROFILE OF THE 2008 SERIES A FINANCING COHORT

Ten years after initial financing, 51% of companies were

privately held

Exhibit 28: Initial Series A Financing in 2008 and Subsequent Company Structure/Ownership

Publicly Traded
$413Mn 28 17%
Financing Companies

Acquired
$533Mn 43 26%
Series A Financing Companies
Financing 2008
$1,685Mn 168
Privately Held
Financing Companies
$638Mn 86 51%
Financing Companies

Closed
$101Mn 11 7%
Financing Companies

Source: BIO: Emerging Company Investment and Deal Trends, 2009−2018, May 2019; IQVIA Institute, May 2019

• Overall, 168 companies received $1,685 million in • Forty-three companies have been acquired, 22 by
financing in 2008 or 2009, with 87% of that funding other emerging biopharma companies and 14 by
U.S.-based. large pharma companies, though some of the assets
that have changed hands in these transactions have
• Since the initial financing, 51% of companies are still
subsequently failed in trials.
privately held, 7%, or 11 companies have closed and
26% have been acquired. • Over half of the companies that received financing are
still privately owned.
• Twenty-eight companies (17%) have gone public within
ten years of their initial funding, having received • Eleven of the 168 companies have closed, having failed
$413 million in initial financing and are now valued at to progress their research, or find a purchaser or
over $14 billion. funding streams to continue operations.

Chart notes: Initial investment in Series A financing was identified for 168 companies receiving funding in 2008. Funding amounts were disclosed for 144
of the 168 companies and are included only for the initial financing in 2008. Two companies (Oceana and Lumavita) had received some funding and had
approved products prior to 2008. Subsequent financing after 2009 has not been included.

38 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

PROFILE OF THE 2008 SERIES A FINANCING COHORT

Twenty-eight companies are publicly traded, five of which now

have market capitalization of over $1 billion dollars

Exhibit 29: Publicly Traded Companies and Their Current Market Capitalization $Mn
100% = $14,340
3% $3,495

$2,739

30%

$1,174 $1,116 $1,061

67%

AGIO ICPT EPZM ESPR FATE

8/2013 10/2012 5/2013 7/2013 10/2013

Ticker & IPO date

Market Cap >$1Bn (5 Companies) Market Cap $100Mn−$1Bn (11 Companies) Market Cap <$100Mn (12 Companies)

Source: BIO: Emerging Company Investment and Deal Trends, 2009-2018, May 2019; IQVIA Institute, May 2019

• Of the 28 companies that have gone public, the five • Epizyme focuses on epigenetics and has five molecules
with market capitalization above $1 billion all had IPOs in development, and has partnerships with Celgene
between October 2012 and October 2013, and are now and GlaxoSmithKline.
valued at over $9.6 billion.
• Esperion focuses on non-statin treatments for patients
• Agios is focused on precision medicines related to with elevated LDL (low-density lipoprotein, so-called
cancer metabolism in leukemia, including Tibsovo bad cholesterol).
and Idhifa, two of the six drugs which have been
• Fate Therapeutics is an immunology company with a
subsequently approved from this group of companies.
focus in cancer and other immune disorders with five
• Intercept has a focus on non-viral liver diseases programs in Phase I and eight in preclinical research.
including NASH, NAFLD, primary sclerosing
• Eleven of the companies are now valued between $100
cholangitis (PSC) and biliary atresia, and has licensing
million and $1 billion, and 12 companies are collectively
partnerships for commercialization outside the
valued at $430 million.
United States.

Chart notes: Market capitalization values as of April 15, 2019. Initial investment in Series A financing was identified for 168 companies receiving funding in
2008 or continuing into 2009. Subsequent IPO has been identified for 28 companies.

iqviainstitute.org | 39
PROFILE OF THE 2008 SERIES A FINANCING COHORT

Forty-three companies have been acquired since initial financing;

more than half by other emerging biopharma companies

Exhibit 30: Acquired Companies by Segment of Acquiring Company

100% = 43 Cardioxyl BMS

VentiRx Celgene BMS*
Receptos Celgene BMS*
SARcode Bioscience Shire Takeda
Meritage Pharma Shire Takeda
FerroKin BioSciences Shire Takeda
35%
Calixa Therapeutics Cubist Merck & Co
Immune Design Merck & Co
51% Actimis Boehringer Ingelheim
Alnara Pharmaceuticals Eli Lilly
Calistoga Pharmaceuticals Gilead
Corimmun Janssen
Elastagen Allergan
Synovex Roche
14%

Large Pharma Small/Mid/Other Emerging Biopharma

Source: BIO: Emerging Company Investment and Deal Trends, 2009-2018, May 2019; IQVIA Institute, May 2019

• Forty-three of the 168 companies have been acquired • Privately-held Boehringer Ingelheim purchased
since their initial financing, more than half by another Actimis for their respiratory franchise in 2008, shortly
emerging biopharma and some of them multiple times. after the initial financing.
Large pharma companies have acquired 14 of the • Eli Lilly’s purchase of Alnara brought them liprotamase,
companies, five of them by three large companies that a cystic fibrosis enzyme treatment, which they
were themselves acquired by other large companies. subsequently sold to Anthera. Development was recently
• BMS and Celgene acquired Cardioxyl, VentiRx and discontinued after failing to reach a Phase III endpoint.
Receptos, focused in heart failure, solid tumors and a • Gilead has augmented their cancer portfolio with the
drug-discovery platform G protein-coupled receptor purchase of Seattle-based Calistoga.
(GPCR) technology, respectively. • Janssen acquired German Corimmun for their antibody
• Shire had acquired SARCode, Meritage and FerroKin and peptide-based cardiovascular treatments.
prior to being acquired by Takeda. The treatments • Allergan added to their aesthetics portfolio with scar-
include dry eye, GI, rare diseases and an iron-chelator remodeling and wound-repair treatments acquired
to address iron overload, common in patients receiving with Elastagen.
chronic transfusions. • Roche acquired Adheron, the new name of Synovex,
• Merck has acquired Calixa and Immune design, the which has been focused on antibody treatments for
first through their Cubist purchase. inflammation by interrupting the cell surface protein
CAD-11.

Chart notes: Initial investment in Series A financing was identified for 168 companies receiving funding in 2008. Subsequent acquisition has been identified
for 43 companies, which have been grouped by company segments. * BMS acquisition of Celgene has been announced but the final closing is not expected
until 3Q 2019.

40 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

PROFILE OF THE 2008 SERIES A FINANCING COHORT

Over half of financed companies have molecules in late-stage

development

Exhibit 31: Portfolio Evolution Since initial Financing

Approved Product
7 4%
Companies

Molecules in
Ph II, Ph III or Pre-Reg
Series A 54%
91
Financing 2008 Companies
168
Companies
Molecules in Phase I
57 34%
Companies

Molecules in Pre-Clinical
53 32%
Companies

Source: BIO: Emerging Company Investment and Deal Trends, 2009-2018, May 2019; IQVIA Institute, May 2019

• Five companies have approved products within ten • Some companies are focused in drug-discovery
years of their initial financing, one (Agios) with two platforms. They have licensed the drugs resulting from
products. Two other companies already had approved the platform, or partnered with other companies to
drugs prior to getting their financing in 2008 (Oceana develop those assets, while continuing to engage in
and Lumavita). early drug discovery. In this way, not all companies are
focused on following assets from discovery to
• More than half of the 168 companies now have
the market.
compounds in late-stage research.

• Two-thirds of the companies have early-stage research

ongoing, with some continuing their initial projects,
and others pursuing additional developments begun
after funding was received.

Chart notes: Companies with approved products (7) includes Oceana and Lumavita, which had approved products prior to receiving financing in 2008.
Companies may have products at multiple phases of development and segments are overlapping.

iqviainstitute.org | 41
PROFILE OF THE 2008 SERIES A FINANCING COHORT

Six drugs from five companies were approved within ten years
of financing

Exhibit 32: Approved Products

REPORTED NET
BRAND (MOLECULE) INDICATION(S) ORIGINAL COMPANY APPROVAL DATE
SALES (2018)
Apadaz
Attention-deficit/hyperactivity Launch expected
(benzhydrocodone KemPharm Inc. Feb 2018
disorder 2H 2019
/ acetaminophen)
Ocaliva Intercept Pharmaceuticals
Primary biliary cirrhosis May 2016 $177.8 Mn
(obeticholic acid) Inc.

Tibsovo (ivosidenib) Acute myeloid leukemia Agios Pharmaceuticals Inc. Jul 2018 $13.8 Mn
Agios Pharmaceuticals Inc.
Idhifa (Celgene has worldwide
Acute myeloid leukemia Aug 2017 $68 Mn
(enasidenib) development and
commercialization rights)
Tetraphase Pharmaceuticals
Xerava (eravacycline) Multidrug-resistant infection Aug 2018 $0.2 Mn
Inc
Chronic lymphocytic leukemia
Zydelig (CLL), small lymphocytic Calistoga Pharmaceuticals
Jul 2014 $133 Mn
(idelalisib) lymphoma and follicular Inc.
lymphoma (FL)
Source: BIO: Emerging Company Investment and Deal Trends, 2009-2018, May 2019; IQVIA Institute, May 2019

• There have been six drugs approved from this group • Ivosidenib (Tibsovo) was approved in July 2018, but
of companies and products, all at least six years after not launched in the United States until October, and
financing and in a substantially faster timeframe than achieved $13.8 million in 2018 sales in 2.5 months.
other drugs.
• Eravacycline, for multidrug-resistant infections, should
• Five of the six drugs have launched in the United be used rarely and has had a similar 2.5 months of
States, all but one approved after May 2016. sales since launching, achieving $0.2 million in sales.

• The two drugs with more than $100 million in 2018

sales were launched in 2014 and 2016, while the other
drugs were launched more recently.

Chart notes: Excludes Delfux from Oceana and Femifect from Lumavita, which were approved and launched prior to the companies receiving financing
in 2008.

42 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

 Looking ahead

• Eight key trends are influencing aspects of trial design, • Biomarkers will have the greatest impact on clinical
duration and success, including digital health and productivity, yielding 34% average increases across
mobile technologies, curated real-world data sources, therapy areas and trial phases and the greatest
predictive analytics and AI, shifts in types of drugs increases in success rates of 27%.
being tested, biomarker test availability, shifts in the
• Pools of pre-screened patients will yield a similarly high
regulatory landscape, increased focus on patient-
increase in productivity of 29% on average by driving
reported outcomes and pools of pre-screened patients/
the largest average improvement in effort of 11%.
direct-to-patient recruitment.
• Emerging biopharma companies assessing
• Their impact on trial design and complexity, duration
commercialization options and strategies for their
and success was explored through the IQVIA Clinical
assets will be facing a large and dynamic global market
Development Trends Impact Assessment completed by
for medicines designed for unmet patient needs.
IQVIA therapy area experts, which extrapolated how
these eight drivers of change would impact the effort, • Emerging biopharma companies will have many choices
success and productivity of trials in the next five years. across various areas, including big data and artificial
intelligence, to support and explore how they relate to
• The number of drugs in active late-stage clinical
their new medicines. Rather than divide their resources,
development has increased 39% over the past five
many view partnerships as the way to adapt to these
years, reflecting the increased level of investment in
environmental and generational shifts.
scientific innovation and raising the level of competition
in key therapy areas at the same time. Looking ahead, a
number of trends are expected to drive changes in the
way EPBs conduct clinical development.

iqviainstitute.org | 43
LOOKING AHEAD

Over the next five years, EBPs engaged in R&D and and their availability will significantly enhance all aspects
commercialization (directly or indirectly) will face a of drug development. The uncertainties surrounding
changing environment and need to adapt accordingly. patient population size and targeting, which often
These changes include trends that are reshaping confound developers and marketers, are becoming
clinical development, as well as trends affecting more surmountable through the use of real-world data,
the commercialization of medicines. In addition, predictive analytics and AI, and the interactive effects of
the financing and deal-making ecosystem in which a number of other advances.
companies decide to license, buy or sell, or partner
Companies which adopt digital health technologies
on assets will transform. As some companies adapt
will range from those who use technology to enhance
to events more rapidly and with greater affinity for
clinical development, patient experience, commercial
the critical factors that will drive success, they will
operations efficiency as well as safety and compliance.
outperform. Others will be better able to maximize value
The use of mobile apps in trials can help companies
through their understanding of the world in which the
engage with trial participants in a more integrated
purchasers of their assets operate.
and holistic way, retaining trialists despite potential
CLINICAL DEVELOPMENT challenges and enhancing trial speed while reducing
The number of drugs in active late-stage clinical costs. Digitally collected patient-reported outcomes are
development has increased 39% over the past five increasingly a way to optimize trial information, and in
years, reflecting the increased level of investment in some trials have improved overall survival; in cancer
scientific innovation and raising the level of competition trials, treatment escalations were able to be made more
in key therapy areas at the same time. Looking ahead, a swiftly, improving trial outcomes.
number of trends are expected to drive changes in the
In examining these key trends, all are expected to yield
way EPBs conduct clinical development (see Exhibit 33).
improvements in trial success rates, however, increases
These include the ongoing revamping of the regulatory in productivity will additionally be driven by decreases
landscape, which has added new breakthrough in trial complexity and duration (i.e., reductions in
designations in recent years, as well as allowing effort), resulting from most trends. Biomarkers and
regulators in the United States and other geographies the development of pools of pre-screened patients
the latitude to make decisions based on less information, to aid in trial recruitment are expected to have the
especially in the case of life-threatening or rare diseases. largest positive impacts on productivity – 34% and 29%,
Additionally, regulators may now assess real-world respectively – on average across all therapy areas. Pools
evidence, or include patient reported outcomes (PROs) of pre-screened patients will have the second greatest
in their decision-making. The expansion of the genomic impact, with a 17% increase in productivity, resulting
revolution is now driving the development of more drugs from the largest percentage drop in effort (-12%) and an
informed by biomarker tests, which further result in 18% increase in success. While predictive analytics and
optimized trial design and development in the presence PRO are among the trends which will yield the smallest
of pre-screened pools of patients. Biomarkers continue benefit to success on average across all therapy areas,
to be discovered, both as a result of drug discovery and in respiratory these were both predicted to yield large
through other research, and the wider range of tests increases to success yet the largest increases in effort.

44 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

LOOKING AHEAD

Eight key trends are driving change in clinical development

Exhibit 33: Trends Driving Change in Clinical Development

Digital health technologies, still immature, will require significant investment to deliver their full potential. EBP companies may
benefit from these technologies as they continue to evolve – likely through the use of outsourced clinical operations or alongside
a larger development partner more likely to invest in these new technologies. Assessing the value and potential of digital
technologies related to a specific development program could be a critical success factor when assessing trial outsourcing or
partnerships.

Increased focus on PROs will shed new light on patient outcomes (PROMs) and experience (PREMs) outside the clinical setting
to inform ongoing clinical decisions, serve as secondary endpoints, influence labeling, and accelerate trial times. A large
proportion of EBP launches are in areas much more commonly associated with PROs, where patient experience and function
are impacted or the delivery system, convenience or tolerability might offer a key differentiator, but only if supported by a
patient measures.

Real-world data is increasing exponentially in volume and complexity, but its use requires significant investment. To participate
and benefit from these changes, smaller companies may need to find a partner or vendor able to integrate these approaches,
but the impact could be transformative if pursued. It may accelerate trials by aiding in investigator/site selection, help optimize
trial design including right-sizing trials for treatment effect, and enable new trial designs.

Predictive analytics and AI offer the potential to radically reduce costs across the biopharmaceutical enterprise, but
leveraging these new technologies is a large investment and may not be core to the founding principles of a science-based
startup. EBP companies that can find applications closely aligned to their research mission may reap significant advantages,
and see increased probability of success and approval. For trials, these tools will identify new clinical hypotheses to test,
reduce trial design risks, speed enrollment by identifying protocol-ready patients, and help narrow trial patient populations to
pre-defined subgroups (i.e., precision medicine). It will also enable adaptive designs that lead to earlier approval with smaller
patient samples.

Shifts in drug types include the development of targeted therapies and Next-Generation Biotherapeutics will improve efficacy
and success rates overall and lead to trials for new indications lacking current options, and 92% of the current crop of late-stage
development in these next-generation areas are EBP companies. As many of these therapies have short treatment durations,
and some have shown curative results, costs are expected to be high, as is payer concern about overall costs. EBP companies
will be reaching the end of their development journey with key questions about how best to commercialize their assets.

Increased availability and ease of biomarker testing is core to the research focus of a whole range of EBP companies and has
been a democratizing force enabling these startups to continue to develop their assets for longer periods prior to eventual sale
or licensing. It has also been a key driver increasing the value of some assets where the benefit to a patient subpopulation is
much more directly assessable.

Availability of pools of pre-screened patients and direct-to-patient recruitment will facilitate trial recruitment and help
sites/trials hit accrual targets, but the investment in building these pools may mean that EBP companies struggle to compete
with better funded rivals. While these pre-screened pools are projected to be one of the larger factors driving better clinical
development productivity in the future, access to the pools may be more limited and come at the cost of surrendering an asset
to a larger partner.

Changes in the regulatory landscape will encourage the use of biomarkers and further precision medicine, drive use of novel
trial designs and endpoints, and provide means for accelerated drug approvals. It may also minimize work burden through the
use of risk-based monitoring, electronic records, and electronic signatures and speed drug approvals by increasing the use of
real-world data to expedite drug development, especially for drugs pursuing an unmet medical need indication. While some
of these approaches require a base level of competence, increasingly emerging companies are taking advantage of this newer
regulatory flexibility to bring their drugs to market and in some cases shorten the gap between EBPs and better funded larger
companies.

Source: IQVIA Institute, Mar 2019; Clinical Development Trends Impact Assessment, Jun-Jul 2018

iqviainstitute.org | 45
LOOKING AHEAD

Exhibit 34: Predicted Percentage Change in Productivity, Effort and Success per Trend from 2018 to 2023
34%
Availability of Biomarker Tests 27%
8%
29%
Availability of Pools of Pre-Screened Patients 18%
11%
16%
Use of Predictive Analytics and Artificial Intelligence 9%
4%
13%
11%
Changes in the Regulatory Landscape
2%
13%
11%
Application of Digital Health / Mobile Technologies 0%
12%
8% 5 Year % Improvement
Emergence of Curated Real-World Data Sources 4% Productivity
2%
8% 5 Year % Improvement
Shifts in Types of Drugs Being Tested -2% Success
2%
6% 5 Year % Improvement
Increased Focus on Patient-Reported Outcomes -4% Effort

Source: IQVIA Institute, Mar 2019; Clinical Development Trends Impact Assessment, Jun−Jul 2018

Several other factors are reshaping approaches to their assets when negotiating a licensing deal, company
clinical development with the potential to improve sale or partnership.
productivity – defined as success rates divided by trial
The types of therapies driving much of the deal-making
complexity and duration  – from current levels. Some
and asset-valuation in recent years have been shifting
types of drugs will be more challenging than others
from targeted oncologics to cell and gene therapies and
to develop, and drive an increase in ‘effort’ – defined
other next-generation biotherapeutics. The speed of
as complexity and duration  – similar to attempts to
development of some of these treatments could radically
incorporate more PROs into trials (see Exhibit 34).
alter the landscape for companies with a marketed
Larger pharma companies have greatly advanced their product, either potentially diminishing or reinforcing
focus on many of these issues, as they develop and the advantage of incumbent products and companies.
market many more drugs than smaller companies and How the first-mover develops relationships in a crowded
often have large internal infrastructures and resources. market and maintains that advantage over time is a
Smaller companies are often deciding which functions critical factor concerning all companies, and ultimately
are absolutely critical to their company as they grow, influences many of the negotiations around emerging
meaning prioritizing resources to address a future companies and products. If a gene-editing technology
trend is often beyond their means. However, emerging can be developed and approved in a few years, which may
companies can still benefit from the awareness of these well be the case over the next decade, it will have broad
trends, particularly as they could affect the valuation of implications for the companies marketing therapies in

46 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

LOOKING AHEAD

that disease, as well as insurers, providers and patients. compound, biologic or process may not be a single
Curative treatments have been relatively rare historically, learning process, but an ongoing one marked by building
but in the past ten years several transformative and consensus between investors, partners, and market
tolerable treatments have become available, often with stakeholders. An emerging company solely focused on
cure in short or even single dosing regimens. the science and the clinical aspects of their drug may be
ill-prepared to address these issues.
These kinds of treatments are expected to be more
common in the near future and promise transformation COMMERCIALIZATION
of their target diseases and the funding arrangements Emerging biopharma companies assessing
affecting all drugs. commercialization options and strategies for their assets
will be facing a large and dynamic global market for
In a future where payers are making complex tradeoffs,
medicines designed to meet unmet patient needs. Over
marginally beneficial late-to-market products of only
the next five years, the global demand for medicines is
modest incremental clinical benefit will lead to a different
expected to continue to grow, both in in volume through
and more challenging commercialization roadmap. A large
expanded access and insurance coverage, and due to
number of drugs developed have offered incremental
the continued flow of innovation and the use of newer
improvements; coupled with the coming increase in
treatments. Spending globally is expected to exceed
commercialization challenges for such products, there will
$1.5 trillion in 2023, adding $50-60 billion per year in
be a significant change in the risk profile of all launches.
global spending, with half driven by large developed
Understanding these shifting dynamics, the competencies
economies and a large proportion from novel medicines
necessary to navigate them and the true value of a
(see Exhibit 35).

Exhibit 35: Global Medicine Spending and Growth in Selected Regions, 2018–2023

UNITED STATES TOP 5 EUROPE

2018: $485Bn +5.2% 2018: $178Bn +3.9%
2014-18: 7.2% 5-year CAGR 2014-18: 4.7% 5-year CAGR
2019: $507Bn +4.6% 2019: $182Bn +2.8%
2023: $625–655Bn +4-7% 5-year CAGR 2023: $195–225Bn +1-4% 5-year CAGR
WORLDWIDE
2018: $1,205Bn +4.8%
2014-18: 6.3% 5-year CAGR
2019: $1,245Bn +4.5%
2023: $1,505–1,535Bn+3-6% 5-year CAGR
PHARMERGING JAPAN
2018: $286Bn +6.9% 2018: $86Bn -1.8%;
2014-18: 9.3% 5-year CAGR 2014-18: 1.0% 5-year CAGR
2019: $293Bn +7.0% 2019: $89Bn +0.9%
2023: $355–385Bn +5-8% 5-year CAGR 2023: $89–93Bn (-)3-0% 5-year CAGR

Source: IQVIA Market Prognosis, Sep 2018; IQVIA Institute, Dec 2018

iqviainstitute.org | 47
LOOKING AHEAD

Exhibit 36: Average Number of Global NAS Launches Annually per Period and Percentage of Launches by Type
Forecast

38% 50% 61% 65%

34% 37% 44% 45%
25% 25% 29% 35%
17% 23% 25% 30%

54
46
36
33

2004–2008 2009–2013 2014–2018 2019–2023

Average Annual Global NAS Launches Oncology Biologic Orphan Specialty

Source: IQVIA Institute. The Global Use of Medicine in 2019 and Outlook to 2023: Forecasts and Areas to Watch. Jan 2019.
Available from: https://www.iqvia.com/institute/reports/the-global-use-of-medicine-in-2019-and-outlook-to-2023

Many developed countries have targeted slowing pressure, as stakeholders use the presence of multiple
healthcare spending growth as a key priority, and as options to generate leverage in negotiations. As larger
a result the continued flow of new drugs presents a companies have recognized the need for assets in certain
funding challenge for those health systems (see Exhibit areas, the last few years have seen a frenzy of deal-
36). Existing mechanisms to adjudicate value range from making activity around immune-oncology therapies,
negotiation of prices through insurers to the use of health cell and gene therapies and others. Alongside this trend,
technology assessments (HTA), which generally result larger companies have been making lower overall up-
in pressure on companies commercializing new drugs. front payments, while total licensing deal values have
These are becoming particularly important, as a small been relatively stable. Faced with less appealing offers for
but growing group of products with extremely high prices their assets and with financing available, more emerging
for relatively few patients are reaching the market (see companies are choosing to finish the development and
Exhibit 37). This also creates opportunities to deliver value commercialize their assets on their own.
through different formulations addressing unmet needs
KEY ENABLERS
in smaller and sometimes lower-priced niches.
In the next five to ten years, three key enablers will be
Across a range of therapy areas there is an increasing important for EBPs to embrace to assure their success:
number of companies aspiring to develop and the use of data and analytics, the adoption of technology
commercialize new treatment options, which will be and a critical need to employ flexible business models.
supported by both sustained success rates and increased

48 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

 LOOKING AHEAD

a) Data and analytics applications b) Technology applications

The combination of technological advances, a decline in Engaging with patients or consumers “where they are”
the cost of computing power and a generation of data is an increasing refrain in modern healthcare, as it is
scientists coming of age is creating and uncovering in modern consumerism. In both cases, patients are
dramatic improvements in the quality and efficiency of increasingly existing in a digital and connected world.
clinical development and commercial operations. By engaging with patients digitally, through e-mail,
smartphones and apps and by collecting necessary health
Companies are using advanced analytic models to
information in optimal ways enabled by these newer
identify and model candidates in drug discovery, to
technologies, more information is being received from
harmonize and integrate unstructured data in multiple
more patients, potentially increasing our knowledge and
languages, as well as optimize pattern recognition
understanding of diseases.
and accelerate the findings from early research. When
artificial intelligence and machine learning are applied These digital engagements can drive short-term and
to the practical and logistical considerations of clinical relatively easy successes, like efficiency gains and drug
trial operations, site selection, patient stratification, and adherence improvements, but they suggest so much more
managing trial costs can all be improved dramatically is possible. The FDA has already approved therapies that
(see Exhibit 38).  use an app to encourage behavior change, and there will

Exhibit 37: Annual and Median Costs of U.S. Brands by Type and Launch Year US$
800,000 Forecast

700,000
Annual Cost of Treatment, US$

600,000

500,000

400,000

300,000

200,000

100,000

0
2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023

Annual Cost per Brand Median of Oncology Median of Specialty

Median of Non-Oncology Orphan Median of Traditional
Source: IQVIA National Sales Perspectives, Dec 2018; IQVIA Institute, Mar 2019

Chart notes: Annual costs based on invoice prices, with overall invoice-level spending divided by estimated numbers of patients. Patient estimates are based
on audited volumes assuming all patients use the drug according to the approved label. Products are included in medians based on segment assignments.
Oncology includes both orphan and non-orphan products. All other products that have orphan indications are grouped together and some products have
both orphan and non-orphan indications in this group. Specialty and traditional products exclude orphan or oncology products but are otherwise defined
according to IQVIA definitions. Projected median costs are based on simple extrapolation of the medians in the prior ten years.

iqviainstitute.org | 49
LOOKING AHEAD

Exhibit 38: Predictive Analytics and Artificial Intelligence Driving Value for Clinical Development

Advanced Analytics
Models
Predict efficacy Find
and safety based undiagnosed Stratify patients
Logistic Regression on molecular patients to for trial enroll-
w/and w/o LASSO structure to Guide site accelerate ment based on
guide drug identification and diagnosis and risk of disease Enable adaptive
XGBoost selection selection screening progression trials

Random Forest

Neutral Networks (DNN,

RNN, CNN) and NLP R&D Clinical Trials

Support Vector
Machine
Accelerate drug Generate new Optimize trials and Identify subgroups of
discovery hypotheses to test study planning patients within many
2-Stage Modeling clinically common disease
Approach states to allow
precision targeting of
Graphical Models therapies

Investigator Predict enrollment Predict patient

segmentation & and screening persistency
selection trajectory
Source: IQVIA Advanced Analytics, Feb 2019; IQVIA Institute, Mar 2019

be many more areas of research exploring the potential c) Flexible business models
of apps as therapies or in combination with drugs as As emerging companies plan for the future, they can
combined therapies. Improved patient engagement in adapt a flexible commercial model, with contracts
clinical trials with the potential for site-less trials and with partners, vendors and service providers which
improved safety monitoring are also being explored can be switched on or off as events dictate. A single
(see Exhibit 39). compound’s regulatory setback can prompt a derailing
few months of reorganization, cuts or even the closing of
Emerging biopharma companies will be overwhelmed
the company. A more modular and flexible model would
with choices of the areas to support and explore how they
only scale up once the success was confirmed.  This
relate to their new medicines. Rather than divide their
flexible approach has been more aspiration than reality
resources, many view partnerships as the way to adapt
for many companies because the technology, analytics,
to these environmental and generational shifts. Those
with potential therapies most affected by some of these and integration necessary to manage such a complex

trends can take advantage of their awareness to see value enterprise simply could not be managed this way even

where others have not. As some drug therapy areas are a decade ago. As companies increasingly explore such

saturated with older and generic treatments, the threshold structures, it becomes possible for more companies to
for innovation to be accepted in the market approaches develop and market an asset while handing over less of
impossible levels; but a new imagining of therapeutic the potential return to other companies. In some cases,
benefit, perhaps involving an app or a device, could be a even just the plausibility of going alone may be a useful
unique value proposition only an outsider could see. negotiating tactic in the sale of an asset (see Exhibit 40).  

50 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

LOOKING AHEAD

Exhibit 39: Digital Health Applications Transforming Clinical Development

Mobile Data Collection from Digital Health Tools Improving Clinical Development

Increased data sources

Novel endpoints Patient safety

Telemedicine and Connected and centralized
Virtual Physician Biometric Sensors Continuous data monitoring
Visits

Digital Virtual eConsent

biomarkers
Contextual metadata
Consumer Disease Consumer
Mobile Apps Management Wearables
Apps
Companion apps Direct-to-patient
Real-time data recruitment

In-home Connected Web-based

Virtual Assessments Interactive
Programs ePRO data Virtual trials and Reduced trial
patient-centric management
designs ‘work burden’
Source: IQVIA Institute. The Changing Landscape of Research and Development: Innovation, Drivers of Change, and Evolution of Clinical Trial Productivity.
Apr 2019. Available from: www.iqviainstitute.org/researchanddev2019

Exhibit 40: Advancing Asset Value Across Milestones

Critical Milestone Activities

Commercialization &
Pre-clinical Validation IND/CTA Filings Phase I Implementation Phase II/III Implementation Product Launch Lifecycle Management
Defining and framing the Designing your clinical Creating a target product Developing a robust trial Determining optimal Finalizing pricing and
value of your asset – with program and understand- profile (TPP), developing design, including pricing and reimbursement, distribution, analyzing your
real-world insights, ing the market for your regulatory strategy and efficient site and patient identifying key stakeholders – commercial infrastructure
alternative paths and product improving your clinical selection, and mapping out and creating brand and and requirements, and
study designs development plan potential launch plans marketing strategies expanding product usage
and labeling
Solutions Customized

DRUG DEVELOPMENT COMMERCIALIZATION

For Your Needs

ASSET VALUATION CLINICAL LAUNCH STRATEGY

STRATEGY & ANALYTICS & LIFECYCLE
& DUE DILIGENCE DEVELOPMENT & PLANNING
MANAGEMENT

• Define the value of an asset • Create a compelling target • Plan and implement highly • Prepare for strong • Optimize commercial
• Create a differentiated product profile and clinical targeted, data-driven product launch execution to maximize
value story development strategy clinical trials • Create the asset brand and product uptake and growth
• Drive investor and partnering • Align to regulatory, payer and • Advance assets from first- map all channels to trajectory post-launch
discussions for optimal commercial requirements in-man through global, effectively demonstrate value
outcomes pivotal Phase III studies

Source: IQVIA Biotech

iqviainstitute.org | 51
Notes on sources
THIS REPORT IS BASED ON THE IQVIA SERVICES IQVIA™ Pipeline Intelligence is a drug pipeline
DETAILED BELOW database containing up-to-date R&D information on
over 40,000 drugs, and over 9,000 in active development
IQVIA Patent Intelligence™ is a database of
worldwide. The database captures the full process of
biopharmaceutical patents or equivalents in over 130
R&D, covering activity from discovery stage through
countries and including over 3,000 molecules. Research
preclinical and clinical development, to approval
covers approved patent extensions in 51 countries, and
and launch.
covers all types of patents including product, process,
method of use and others. IQVIA™ Pharma Deals is a comprehensive life science
deals and alliances database that leverages worldwide
MIDAS™ is a unique platform for assessing worldwide
information sources to deliver the latest intelligence in
healthcare markets. It integrates IQVIA’s national audits
deals and alliances.
into a globally consistent view of the pharmaceutical
market, tracking virtually every product in hundreds
of therapeutic classes and provides estimated product
volumes, trends and market share through retail and
non-retail channels.

52 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

Useful resources
IQVIA Pharma Deals Review of 2018

This Review provides an insightful overview of deal activity in 2018, as well as White Paper

the outlook for deals in 2019. Key information provided includes the following: IQVIA™ PHARMA DEALS
Review of 2018

• Top M&A and partnership deals in 2018 MICHELLE LIU, Analyst, Global Market Insights, IQVIA
NATASHA PIPER, Analyst, Global Market Insights, IQVIA
HEATHER CARTWRIGHT, Senior Analyst, Global Market Insights, IQVIA

• Deal activity rankings of the top pharmaceutical companies TASKIN AHMED, Manager, Global Market Insights, IQVIA

• Deal activity by therapeutic area and development phase

• Deal value analysis of M&A, licensing and R&D deals

Key highlights:
Deal activity in the life sciences sector slowed in 2018, as political uncertainty,
leadership changes and changing regulatory and pricing landscapes took a toll.
As was the case in 2017, small companies had various financing options available
to them resulting in inflated company valuations, which thereby discouraged
pharmaceutical companies from certain types of dealmaking.

Download at: https://www.iqvia.com/library/white-papers/iqvia-pharma-

deals--review-of-2018

Launch Archetypes: The Bedrock of Successful Launch Strategies

From the moment that commercial planning begins, new product launches face White Paper

a gauntlet of challenges to which many succumb. In fact, over half fail to meet LAUNCH ARCHETYPES:
THE BEDROCK OF SUCCESSFUL
financial expectations based on a broad internal analysis. LAUNCH STRATEGIES
DARIN DECARLO, Senior Principal and Segment Lead, Commercial Services, US – IQVIA
NORA HANNIGAN, Engagement Manager, Launch Center of Excellence, Commercial Services, US – IQVIA

Brand teams can improve their chances of success in such a high-risk WILLIAM MCCLELLAN, Center of Excellence Leader, Launch Excellence, Commercial Services, US – IQVIA

environment by understanding their product’s Launch Archetype, as defined by

the mix of unmet need in the market and the product’s level of differentiation.

The IQVIA Launch Center of Excellence applies a proprietary scoring model

along these two dimensions to all new product launches and, consequently, has
identified four Launch Archetypes. This white paper discusses how marketers
can develop successful launch strategies by studying other marketed products
that share the same Archetype. It also provides perspective of the current
launch environment through the lens of Archetypes, highlighting trends and the
relationship between Archetype and market performance. Each Archetype
comes with its own set of considerations, marketing implications, and associated
launch challenges.

Download at: https://www.iqvia.com/locations/united-states/library/white-

papers/launch-archetypes-the-bedrock-of-successful-launches

iqviainstitute.org | 53
Definitions
Early-stage pipeline is defined as the developmental
• 
stages of discovery, preclinical, and Phase I.

• Late-stage pipeline includes developmental

stages of Phase II, Phase III, pre-registration, and
registration.

• Next-Generation Biotherapeutics are defined as

cell, gene and nucleotide therapies.

• Mechanisms include: cell therapy, dendritic cell

therapy, NK cell therapy, T-cell therapy, CAR-T- cell
therapy, T-cell receptor therapy, stem cell therapy,
bacterial cell therapy, CIK cell therapy, CIK-CAR
therapy, whole cell vaccine, dendritic cell vaccine,
bacterial cell vaccine, DNA vaccine, RNA vaccine,
exon skipping, nucleic acid-based, gene therapy,
oligonucleotide, antisense, RNAi, microRNA mimic,
gene editing, CRISPR-Cas9, zinc finger nuclease,
RNA therapy, and mRNA therapies

54 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

About the authors
MURRAY AITKEN MICHAEL KLEINROCK
Executive Director, IQVIA Institute Research Director, IQVIA Institute
for Human Data Science for Human Data Science

Murray Aitken is Executive Director, IQVIA Institute Michael Kleinrock serves as research director for
for Human Data Science, which provides policy setters the IQVIA Institute for Human Data Science, setting
and decisionmakers in the global health sector with the research agenda for the Institute, leading the
objective insights into healthcare dynamics. He led development of reports and projects focused on the
the IMS Institute for Healthcare Informatics, now the current and future role of human data science in
IQVIA Institute, since its inception in January 2011. healthcare in the United States and globally. Kleinrock
Murray previously was Senior Vice President, Healthcare leads the research development included in Institute
Insight, leading IMS Health’s thought leadership reports published throughout the year. The research is
initiatives worldwide. Before that, he served as Senior focused on advancing the understanding of healthcare
Vice President, Corporate Strategy, from 2004 to 2007. and the complex systems and markets around the world
Murray joined IMS Health in 2001 with responsibility that deliver it. Throughout his tenure at IMS Health,
for developing the company’s consulting and services which began in 1999, he has held roles in customer
businesses. Prior to IMS Health, Murray had a 14-year service, marketing, product management, and in 2006
career with McKinsey & Company, where he was a leader joined the Market Insights team, which is now the IQVIA
in the Pharmaceutical and Medical Products practice Institute for Human Data Science. He holds a B.A. degree
from 1997 to 2001. Murray writes and speaks regularly in History and Political Science from the University of
on the challenges facing the healthcare industry. He is Essex, Colchester, UK, and an M.A. in Journalism and
editor of Health IQ, a publication focused on the value Radio Production from Goldsmiths College, University of
of information in advancing evidence-based healthcare, London, UK.
and also serves on the editorial advisory board of
Pharmaceutical Executive. Murray holds a Master of
Commerce degree from the University of Auckland
in New Zealand, and received an M.B.A. degree with
distinction from Harvard University.

iqviainstitute.org | 55
 DEANNA NASS ALANA SIMORELLIS
Director of Publications, IQVIA Publications Manager, IQVIA
Institute for Human Data Science Institute for Human Data Science

Deanna Nass is the director of publications at the Alana is Publications Manager for the IQVIA Institute

IQVIA Institute for Human Data Science. She manages and helps manage aspects of IQVIA Institute research

the development and production lifecycles of IQVIA projects and publications, as well as conducting research

Institute reports and performs analyses of global and analysis within global healthcare. Alana came to

biopharmaceutical and healthcare trends. With a diverse IQVIA in 2016 having previously worked at Decision

background that spans from consulting and business Resources Group for over six years as a Principal

development to market analysis and writing industry Business Insights Analyst. At Decision Resources group,

publications, she brings a unique perspective of the Alana authored a number of publications within multiple

biopharma industry to the Institute. Deanna joined the disease areas that included Alzheimer’s disease, pain,

Institute in 2013 and IMS Health in 2004. Deanna holds a bipolar disorder, schizophrenia and major depression.

B.A. in Biology from Yale University with a specialization Alana has a Ph.D. in Chemistry from the University

in Neurobiology and a Certificate in International Affairs of Utah and completed a postdoctoral fellowship at

from New York University. Brandeis University, where part of her research involved
structural investigation of a protein associated with
Parkinson’s disease.

ELYSE MUÑOZ
Thought Leadership Manager, IQVIA Institute for Human Data Science

Elyse Muñoz is a Thought Leadership Manager for the IQVIA Institute, managing aspects of
IQVIA Institute research projects and conducting research and analysis within global healthcare.
Elyse joined IQVIA in 2017 as an associate consultant in the Competitive Intelligence consulting
group, where she developed rich clinical and commercial insights to serve clients. She worked in major therapy areas
including diabetes, cardiovascular disease and kidney dysfunction, as well as rare diseases such as hemophilia. Elyse
holds a Bachelor of Science from Arizona State University in Genetics, as well as a Ph.D. in Genetics from Pennsylvania
State University. Her research focused on the genetic makeup of the parasite that causes malaria to aid in the
development of targeted drugs to eradicate the disease.

56 | Emerging Biopharma’s Contribution to Innovation: Assessing the Impact

About the Institute
The IQVIA Institute for Human Data Science • Understanding the future role for biopharmaceuticals
contributes to the advancement of human health in human health, market dynamics, and implications
globally through timely research, insightful analysis and for manufacturers, public and private payers,
scientific expertise applied to granular non-identified providers, patients, pharmacists and distributors.
patient-level data.
• Researching the role of technology in health system
Fulfilling an essential need within healthcare, the products, processes and delivery systems and the
Institute delivers objective, relevant insights and business and policy systems that drive innovation.
research that accelerate understanding and innovation
Guiding Principles
critical to sound decision making and improved
The Institute operates from a set of Guiding Principles:
human outcomes. With access to IQVIA’s institutional
knowledge, advanced analytics, technology and • Healthcare solutions of the future require fact-based
unparalleled data, the Institute works in tandem with a scientific evidence, expert analysis of information,
broad set of healthcare stakeholders to drive a research technology, ingenuity and a focus on individuals.
agenda focused on Human Data Science, including
• Rigorous analysis must be applied to vast amounts of
government agencies, academic institutions, the life
timely, high quality and relevant data to provide value
sciences industry and payers.
and move healthcare forward.
Research Agenda
• Collaboration across all stakeholders in the
The research agenda for the Institute centers on
public and private sectors is critical to advancing
five areas considered vital to contributing to the
healthcare solutions.
advancement of human health globally:
• Insights gained from information and analysis should
• Improving decision-making across health systems
be made widely available to healthcare stakeholders.
through the effective use of advanced analytics and
methodologies applied to timely, relevant data. • Protecting individual privacy is essential, so research will
be based on the use of non-identified patient information
• Addressing opportunities to improve clinical
and provider information will be aggregated.
development productivity focused on innovative
treatments that advance healthcare globally. • Information will be used responsibly to advance
research, inform discourse, achieve better healthcare
• Optimizing the performance of health systems by
and improve the health of all people.
focusing on patient centricity, precision medicine
and better understanding disease causes, treatment
consequences and measures to improve quality and
cost of healthcare delivered to patients.

iqviainstitute.org | 57
 The IQVIA Institute for Human Data Science is committed to using
human data science to provide timely, fact-based perspectives on the
dynamics of health systems and human health around the world.
The cover artwork is a visual representation of this mission. Using
algorithms and data from the report itself, the final image presents
a new perspective on the complexity, beauty and mathematics of
human data science and the insights within the pages.

Artwork on this report cover was generated using data sets from
IQVIA Patent Intelligence™, IQVIA Pipeline Intelligence ™ and research
by the IQVIA Institute for Human Data Science. The data sets show
drugs launched in the U.S. 2017-2018 by the originating company and
the FDA filing company as well as by the type of company based on
their size (emerging biopharma, small, mid-sized, or large).

CONTACT US
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United States
[email protected]
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Copyright © 2019 IQVIA. All rights reserved. 05.2019.ENT