Improving Healthcare Access for Sickle Cell Patients

🚀 A Roadmap to Affordable Genetic Medicines! 🧬 🌟 With gene therapies like CASGEVY for sickle cell disease now a reality, we stand at the frontier of revolutionizing medicine! But the biggest challenge? Costs over $4 million per patient when these advanced therapies are used! 💰😳 A new paper (see link below) outlines a comprehensive plan to reduce costs tenfold while expanding access to life-saving therapies. Here’s what’s included: 1️⃣ New Business Models: Traditional, for-profit pharmaceutical development is profit-driven, but this roadmap introduces public-private partnerships and nonprofit models to ensure affordability without sacrificing innovation. This way, financial sustainability is prioritized without placing the burden on patients. 🤝 2️⃣ Innovative Manufacturing: By developing distributed manufacturing (including point-of-care production at hospitals), we can reduce the logistical and financial challenges of centralized gene therapy manufacturing. Imagine therapies being created on-site, cutting both costs and time! 🏥⚙️ 3️⃣ Collaborative Licensing: Universities, where most of the research originates, can negotiate licensing terms to guarantee lower prices for underserved populations. "Most-favoured nation" clauses in these agreements ensure that US patients don’t pay more than others globally. 🌍📑 4️⃣ Regulatory Innovation: The roadmap also proposes simplified regulatory pathways, especially for therapies targeting ultra-rare diseases. This includes risk-based comparability assessments and platform technologies, which would speed up the regulatory process and reduce costs. ✅ 5️⃣ Flexible Pricing Models: Beyond reducing manufacturing costs, the proposed cost-plus pricing model ties the final price of therapies to the actual cost of development and deployment, ensuring that therapies remain accessible. 💸🔧 6️⃣ Access for Low- and Middle-Income Countries (LMICs): A critical focus is on global access. Licensing agreements could include requirements to develop affordable versions of therapies for LMICs, or allow non-exclusive licenses in regions where access is challenging. 🌎🌱 By embracing a collaborative, mission-driven approach, we can unlock the full potential of genetic therapies for all patients, regardless of geography or income! Let’s work towards a future where cutting-edge treatments are no longer a luxury, but a right. 🙌 #GeneTherapy #CellTherapy #HealthcareInnovation #AffordableHealthcare #GlobalAccess #BiotechRevolution 🔗 Read the full study: https://lnkd.in/dpY_Ziy5

I have spent the last decade developing and leading high-touch, team-oriented, advanced primary care models for populations of people with a variety of complex chronic conditions, focused on advancing care at the bedside and the sidewalk (in the community). What I have learned, and the data support, is that well designed programs that combine: - technology to enable multi-disciplinary teams, -population-based analytics, -guideline directed evidence-based care, -and novel locations of delivery including virtual care, hybrid virtual care, and home visits, can dramatically improve patient outcomes across various complex conditions and population groups. There is good reason to believe these strategies applied to complex disease populations, like those affected by sickle cell, would ensure that those who qualify from novel gene therapies can actually access this treatment, and those who don’t, still receive the best possible care and quality of life that decades of non-gene-therapy advancements and good old fashion social care provide. These care models, especially those with a heavy emphasis on telehealth, are particularly effective for the exact subpopulations in greatest need for improved access, rural and low-income communities, without direct access to an academic medical center. #complexcare #sicklecelldisease #rarediseases #healthcareinnovation

The era of in vivo base editing is here. One patient, one injection, one letter change in the HBB gene. And potentially, a cure for sickle cell disease. Eric Topol, MD’s recap of the first in vivo base editing treatment should stop anyone in biotech in their tracks. It’s not just a scientific breakthrough; it’s a signal of how fast we’re moving toward rewriting disease at the source. Baby KJ, the patient with sickle cell, didn't undergo a bone marrow transplant. There was no cell extraction. No months of prep. Just a single IV infusion using Beam’s base editing tech delivered via lipid nanoparticles. The implications are enormous. Sickle cell disease affects over 20 million people globally. In the U.S. alone, it drives over 100,000 emergency visits each year with average lifetime treatment costs exceeding $1.7M per patient over a lifetime. And now? A potential one-time intervention. 🔬 The shift from ex vivo to in vivo marks a critical inflection point: - No need to remove, edit, and reinsert cells. - Opens the door to broader access, lower costs, and faster deployment. - Reduces complexity across regulatory, logistical, and clinical dimensions. Beam Therapeutics, Verve Therapeutics and Prime Medicine, Inc. are not just leading-edge biotech companies, they’re building platforms that will reshape health economics as much as biology. As in vivo gene editing enters the clinic, the next decade will hinge on more than trial endpoints. We’ll need continuous, real-world data to understand how these therapies perform across diverse populations, age groups, and health systems. It’s a question we think about a lot at Edilytics. We’re building tools to support the long-term data infrastructure needed for therapies like this - so we can better understand how these interventions perform in the real world, over years and across populations. One base change can change a life. The ripple effects deserve to be understood just as precisely.

Janiera Jackson, 2024 Alumni of Drexel University Nurse Anesthesia Program, a practicing CRNA and selection of scholarly project through a health equity lens impacting Black marginalized patients with Sickle Cell Disease Scholarly Project: Sub-anesthetic Ketamine's Role in Improving Patient Outcomes during a Sickle Cell Vaso-Occlusive Crisis "You are your own best thing" ~ Toni Morrison Standing on business for mine, always. ✊🏾🖤🤎✊🏾 It was imperative for me to do my senior research project on a topic that can potentially impact the health of black people. One of the main reasons I am in healthcare is help tether the bridge that closes the health equity gap in this country. Sickle cell disease may only affect 100,00 Americans, but it is the most common inherited hematologic disorder in the country. The disease mostly affects African Americans and those of Sub-Saharan African descent. For perspective about 1 and 13 African American babies are born with sickle cell trait. The primary morbidity or the disease is pain (acute or chronic). It is not uncommon for these patients to be prescribed opioids at home to manage their pain. During vaso-occlusive crisis (VOC) however the pain experienced is more intense and refractory to opioids. This leads to ED visits for VOC. Sometimes ED providers have a negative attitude when treating these patients with more opioids. This contributes to under and poorly treated pain in this population. Ketamine is a non-opioid anesthetic that is a great drug to be introduced in this conversation of treating SCD-VOC. SCD patients also experience opioid induced hyperalgesia (OIH) which is a maladaptive response of the CNS to chronic pain or under treated acute pain. On a cellular level OIH has an up-regulation of NMDA receptors and a down regulation of Mu receptors. And guess what, Ketamine is a NMDA antagonist! Making it a great contender for refractory pain/OIH/SCD-VOC. There is still much research to be done but Ketamine deserves a seat at the table to help this marginalized patient population achieve better analgesia. #blackwomen #crna #drexeluniversity #SickleCellAwareness