Ashley Duenas

HERCULES: Evidence must drive decisions—not be retrofitted to them. Yesterday, Duchenne UK convened and led a cross-sector workshop to confront a shared challenge: How do we generate and interpret evidence for paediatric, progressive, disabling, life-limiting conditions—when current HTA frameworks aren’t built for them? While HERCULES began in Duchenne, the issues we’re tackling go far beyond any single condition. That’s why we’re expanding—across paediatric, progressive, disabling, life limiting rare diseases, across systems, and across lived realities. 📉 The reality: 75% of rare diseases begin in childhood, and 80% are disabling (EURORDIS Rare Barometer) ABPI data (2020–2023) shows: 25% of rare disease medicines with MHRA approval never reached NICE Of those that did, 20% were failed or terminated The rest faced years of delay HTA frameworks routinely miss: The cost and impact of disability The invisible labour and emotional toll on parent carers The functional loss and impact of upper body decline—including cardiac, respiratory, ventilation, and gut The complexity of paediatric evidence generation 🧠 Attendees included: HTA bodies and regulators Health economists and academic methodologists Clinicians and trialists Patient advocates and lived experience experts Industry partners working in severe paediatric rare disease 💡 To address this, HERCULES is launching four targeted workstreams: 🧠 Project TITAN – Capturing the cost and impact of disability 🫂 Project HERA – Recognising the role and burden of carers 🦾 Project ATLAS – Valuing upper body function, including cardiac, respiratory, ventilation, and gut 👶 Project CHIRON – Strengthening paediatric evidence generation and interpretation Together, these projects will build a shared evidence framework—fit for the complexity, urgency, and ethical weight of decision-making in rare paediatric conditions. 📣 Want to get involved? We’re inviting collaborators across HTA, academia, clinical practice, and advocacy to help shape and fund the next phase. If you’re working on methods, metrics, or lived experience in rare disease—get in touch - with me, Emily Reuben OBE and Josie Godfrey #DuchenneUK #RareDisease #HTA #ProjectHERCULES #ProjectTITAN #ProjectHERA #ProjectATLAS #ProjectCHIRON #Paediatrics #EvidenceGeneration #CarerBurden #DisabilityCosts #UpperBodyFunction #PolicyDiplomacy #HealthEconomics #InclusionMatters

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Rare disease research and development is complex. But we can take steps now that will create a better future. Below are the 10 recommendations from our recent report with Genetic Alliance UK to help #ChangeTheRareFuture for those living with rare conditions. Find the full report here: https://lnkd.in/enrFifAs

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The UK’s life sciences sector is at a pivotal moment... Despite Merck’s and AstraZeneca’s recent announcements, there are reasons for optimism. The convergence of AI and health innovation is creating new opportunities for growth - especially in the spaces where universities, hospitals and investors collaborate. Our CEO, Anne Lane, has written an op-ed in the Financial Times about her optimism for the sector. She highlights that, at UCLB, we’ve seen firsthand how research breakthroughs can become commercial success stories: £3bn external in investment since 2020, and 100+ spinout companies creating 2300+ jobs.  But the potential goes far beyond this. To truly unlock economic growth, the UK needs more early-stage funding to help academic research become market-ready, more scale-up venture capital and an easier-to-access NHS, to provide the market ‘pull’ for homegrown health innovations coming out of our universities. Read Anne's thoughts on what needs to change - and why the UK can still lead the next wave of medical breakthroughs (£): https://lnkd.in/eZGYiSc7

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NHS Wales has launched its new Healthcare Science Framework, mapping career pathways and opportunities in the profession. The IBMS contributed to the development of the Pathology and Genomics pathway to ensure it reflects the realities of biomedical science in Wales. You can read more about the framework and how it supports biomedical scientists here: https://bit.ly/4fHgkBi

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We’re partnering with Stand Up To Cancer to offer up to £2.5m per award for research programmes to support data-driven solutions to common challenges in children’s and young people’s cancers. This funding call invites proposals that foster new interdisciplinary partnerships and develop novel methods for working with and getting the most from children’s and young people’s data, whether from existing sources or newly generated datasets, to advance research in this area. We welcome applications from international, interdisciplinary teams based across multiple institutions. Collaboration with industry is encouraged. Learn more about what we’re looking for and tips on how to put together a strong application at our webinar on 22 September from 1:00 pm. Head to our website for more information here - https://cruk.ink/4njVxWU Register for the webinar here - https://cruk.ink/4gdAcw9 #CCAM

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