Orphan medicines: special treatment required?: This report was prompted by concerns that patients and their clinicians are not always able to access medicines for those with rare diseases. Ongoing reforms to the NHS and the development of a strategy for rare disease offer an opportunity to consider access to medicines for those with orphan or ultra-orphan conditions. This report provides an analysis of the complex framework that influences patients’ access to orphan drugs, focusing on the role of R&D, regulatory and payer/commissioner decisions, and the links between decision making along the pathway to access. It makes suggestions for better decision making and how to improve access to such drugs more widely.
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