OVERVIEW OF REGULATORY INITIATIVES IN THE EUROPEAN UNION TO STIMULATE RESEARCH AND ACCELERATE ACCESS TO ORPHAN DRUGS AND OTHER HIGH MEDICAL NEED PRODUCTS
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The European Union (EU) is continuously working towards further promoting and facilitating registration and development of OMPs. The goal of this article is to present a broad overview of existing legislation, programmes and incentives in the EU that allow for accelerated access of orphan drugs and high medical need products, as well as give some historical context and how these programs are interconnected. The authors conducted an explorative review of publicly available sources of regulatory intelligence policy and information on orphan drug and rare disease policies as well as information on accelerated assessment programs in the EU. The authors identified and looked into the following programs and initiatives: orphan drug legislation, accelerated assessment, conditional approval, Priority Medicine (PRIME), compassionate use programs, off-label use and RTU, Medicines Adaptive Pathways to Patients (MAPPs), ADAPT-SMART, HTA initiatives (EUnetHTA, Parallel Consultation), STAMP, IMI and IMI2 and the European Framework Programmes (incl. FP7 IDEAL, Asterix and Inspire). It can be concluded that promoting innovation and early access to medicines has been firmly put on the agenda in the EU, with various programs and initiatives, either national or EU-wide. Hurdles in regulatory science remain present, especially for rare diseases, but fundamental research as done in FP7 could lead to more efficient orphan drug development. Barriers in the field of HTA, pricing and reimbursement still need to be addressed to enhance patient access. The true effectiveness of all these programs will only become clear over time.
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