Session 21

New partnership models for rare diseases

Future flagship financing and R&D solutions for accessible care

Organised by AOP Orphan

Rare disease patients are often left outside – either by lacking research and development or by public payers refusing to pay for the expensive treatment. After decades of frustratingly slow progress, it is time for a rethink to adopt alternative partnership and financing models that incentivise investment in orphan drug development and provide accessible patient care.

What new approaches can reward innovation whilst reducing financial risk in rare disease research? How can we move away from a system driven by shareholder interest and deliver orphan drugs as a public good to safeguard supply and access for patients? Why do current PPP models not seem to suffice in the rare disease space, and how can stakeholders better collaborate to share the risk in research and development?

This session looks to trigger public institutions, universities, and private companies to build more effective solidarity towards rare disease patients - to support new drug research with better knowledge, assessment and characterisation of treatment whilst ensuring affordable and continued market access. Join us to brainstorm and discuss ideas that question existing business models, explore new approaches to financing rare disease treatment, and to create a flagship for the future.