Paradigm shifts and strategic recommendations from Volv Global, Sanofi, Fondation Ipsen, Unitechpharma, and brave2change
Orphan drug development faces barriers: approximately 95% of known rare diseases have no approved treatment. Around 400 million people worldwide are living with a rare condition, and for the majority, the search for a therapy that addresses their disease remains open – often indefinitely. The barriers are real, and they are well understood. What has been lacking, until now, is a structured, cross-sector account of what must change and how.
Drive For Change: Paradigm Shifts and Strategic Recommendations to Overcome Barriers in Orphan Drug Development is that account.
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What the paper addresses
Authored by leaders from Volv Global, Sanofi, Fondation Ipsen, Unitechpharma, and brave2change, and grounded in cross-industry workshops at the World Orphan Drug Congress 2023 spanning 25 countries, Drive For Change identifies three structural paradigms that must shift simultaneously if orphan drug development is to advance at the scale patients deserve.
How patient populations are understood. Conventional prevalence estimates routinely undercount undiagnosed patients – understating market size, weakening the business case, and undermining trial design. Machine learning applied to real-world data at population scale can change this materially. Volv Global’s own deployments have identified patient populations several times larger than prior sponsor estimates, and produced diagnosis rate increases of 50% to 100% in disease areas where rates had been static for a decade.
How rare disease biology is understood. Pipeline herding concentrates development effort in well-understood indications, leaving the majority of rare diseases without even a candidate therapy. The paper sets out what it takes to build scientific and commercial confidence in less-charted disease areas.
How the cost and burden of disease is evaluated. Current HTA frameworks systematically undercount the true economic impact of rare disease – excluding indirect costs, misdiagnosis expenditure, and productivity losses. The paper proposes a more complete valuation framework, and makes the case for linking reimbursement to demonstrated real-world outcomes.
Why now
Several major pharmaceutical companies have recently scaled back or withdrawn from rare disease programmes. Drive For Change engages directly with that reality – and with the evidence that the addressable patient population is almost always larger than sponsors currently believe. The investment case is stronger than it appears. This paper shows why, and what it takes to act on it.
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Contributing authors
Léon van Wouwe – Clinical Innovation Director, Volv Global (Lead Author)
Dr Kristina An Haack, MD – Senior Global Project Head, Sanofi
Dr Mahdi Farhan – Unitechpharma SA
Professor James A. Levine, MD PhD – President, Fondation Ipsen
Bernd Rosenbichler – Founder, brave2change
Dr Vahid Esmaeili – Data Science & Digital Health Director, Volv Global
Christopher Rudolf – CEO & Founder, Volv Global
The authors also express their warm appreciation to Gina Cioffi Loud, Public Affairs Lead, US Rare Diseases, Chiesi Global Rare Diseases, for her significant editorial contribution to this work.
Drive For Change is available to download now, free of charge. If you work in rare disease development, access, or advocacy, this paper was written with you in mind.
