Rare diseases: How do we build global policy momentum?

Rare diseases remain one of the largest areas of unmet need in global health, affecting more than 300 million people worldwide. When families and caregivers are included, the circle of direct impact expands to more than 1 billion people, resulting in immense human and economic costs across societies and healthcare systems. The challenge is compounded by a scarcity of real-world evidence to support the search for new treatments, disparities in access and affordability of diagnosis, treatment and care, and a lack of knowledge sharing that leads to duplication of efforts to address the disease burden.

This Financial Times Digital Dialogue, which took place in partnership with Biogen ahead of the first anniversary of the WHO Rare Diseases Resolution, brought together policy makers, healthcare professionals and patient representatives to discuss practical actions to translate global ambition into local change. Speakers discussed opportunities for strengthening rare disease systems, ensuring that solutions also take account of the longer-term needs of patients as medical advances lead to longer and better lives.

POLICY TO PRACTICE 

How can national governments build capacity, fund and create self-sustaining rare disease programmes?

ACCESS AND EQUITY

How do we improve access and ensure ‘no one is left behind’?

EVIDENCE FOR INVESTMENT

How can policy makers build the economic case for investment in rare diseases: the role of real-word evidence?

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