An expert group, the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL) has proposed nine principles to guide value assessment, pricing, and funding of rare disease drugs, which it claims will promote greater sustainability.
One of the main principles for this value-based health policy measure is that healthcare stakeholders should incorporate the perspective of patients and benefit of the wider society into decision-making.
“Rare diseases are an important public health issue and it is important that patients with these
diseases have access to safe and effective therapies, in the same way as others suffering from more common diseases. The need for greater consistency, clarity and certainty will become ever more important as the number of rare disease treatments grows. Payers, policy makers, and manufacturers will need to take a long-term perspective and build frameworks and processes that are capable of addressing the scale of the challenge.”
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