Pharma’s interest in treatments for rare diseases has continued to intensify as the orphan sector sees rapid growth and attractive profit margins. Developers of orphan drugs can obtain substantial support from the European Medicines Agency during the development process as well as a reduction in fees for review, and in some EU5 countries (Germany and the United Kingdom) drugs may launch upon approval. However, in those and in the other EU5 markets, patient access and uptake ultimately depend on a positive health technology assessment (HTA), the basis for negotiating favorable reimbursement terms with individual countries. Due to the increasing budgetary impact of orphan drug, European payers have become more restrained when assessing these agents, with stricter or narrower HTA outcomes becoming more common as well as less favorable reimbursement terms for orphan drugs. 

 


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