Progress in R&D and technology, regulatory and monetary incentives for advancing the development of treatments for rare diseases, and ongoing focus by the pharmaceutical market on this space are key factors driving continued growth in the number of approved and emerging orphan drugs. However, with many orphan drugs priced at high premiums, and given the increasing armamentarium of such agents in the marketplace, the EU5 healthcare agencies must find ways to manage their ever-tightening budgets in order to best serve the areas of high unmet medical need that orphan drugs seek to address. This study analyzes the health technology assessment (HTA) and pricing and reimbursement (P&R) policies that are used to regulate market access for orphan drugs, specifically exploring the evolving dynamics affecting reimbursement and uptake of current and emerging orphan drugs for cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD).

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