Robin Durand
Project Lead, Rare Diseases International
From outcome-based agreements to venture philanthropy, innovative financing is changing who carries the risk in rare disease care.
Redistributing uncertainty
Rare diseases carry inherent risk. Patients often wait years for a diagnosis, only to face continued uncertainty about the effectiveness of treatments. Manufacturers risk decades of research in therapies without a guaranteed return. Payers must decide whether to fund treatments with uncertain long-term benefits.
Each rare condition affects a small population, making it difficult for any stakeholder to plan with confidence. Yet, each risk throughout the rare disease care pathway has traditionally fallen on a single actor. Innovative financing can change this dynamic by redistributing risk and allocating it to those best placed to absorb it, creating value no stakeholder could achieve alone.
This system has raised around USD $70 million for patients
living with conditions like Gaucher and Pompe disease
Risk-sharing in practice
In Brazil, outcome-based agreements for spinal muscular atrophy therapies link payment to real-world clinical results,1 shifting the risk of treatment failure from health systems onto manufacturers. This gives patients earlier access while generating real-world evidence. The UK’s National Institute for Health and Care Excellence (NICE) applies the same logic,2 paying upfront but tying final payment to routine-care results, protecting budgets without delaying access.
In Singapore, the Government matches every dollar donated to the national Rare Disease Fund threefold and offers a 250% tax deduction, so the full cost never lands on any one donor, company or ministry. This system has raised around USD $70 million for patients living with conditions like Gaucher and Pompe disease.
Venture philanthropy — charities investing like venture capitalists — takes on risk before treatments are developed. In 2000, the Cystic Fibrosis Foundation gave Vertex Pharmaceuticals $40 million to pursue CF’s underlying cause in exchange for royalty rights,3 a bet no conventional investor would have made. The therapy succeeded, and the Foundation reinvested its royalty rights into further research.
A positive-sum deal
Sharing risk protects budgets while creating value. The 2025 World Health Assembly Resolution on Rare Diseases placed rare disease sustainable financing on the global agenda. We know that innovative risk-sharing can work. Now, it is time to scale these models and improve equitable access to care and treatments for rare diseases.
[1] Dolon Institute, 2026. Access Barriers and Solutions to Orphan Medicinal Products in LMICs.
[2] NICE. Managed access. tinyurl.com/k4kxjss3.
[3] Cystic Fibrosis Foundation, 2014. Cystic Fibrosis Foundation Royalty Sale Will Be Transformational for People with CF.
