Value assessment of orphan drugs

Value assessment of orphan drugs

Health Technology Assessment (HTA) is playing an increasingly crucial role in the process of appraising and reimbursing medical technologies across Europe. While HTA generally relies on a robust estimation of the clinical cost-effectiveness of a new technology, the clinical and economic evidence required for this purpose is often not available for orphan drugs, partly because of challenges related to the recruitment of patients in clinical trials.

In the RARE-Bestpractices project the London School of Economics (LSE) is responsible of mapping out the policies currently in place in selected EU countries on orphan drugs appraisals and exploring the implications these policies have for coverage decisions. A review of the available literature and jurisdiction-specific guidelines on the value assessment of orphan drugs was performed and presented in a peer-reviewed article (RARE DISEASES AND ORPHAN DRUGS. An International Journal of Public Health, Vol.1, No.3). The ongoing research is directed to understand how appraisals are conducted, the critical factors leading to positive and negative recommendations and how do assessments feed into clinical practice guidelines. For this reason, 17 case studies on 17 drug-indication pairs are being conducted based on appraisal documents and publicly available guidance from England, France, Germany, Italy, Poland, Scotland, Spain and Sweden (under Work Package 5).  

Based on the evidence collected, LSE will explore best practices and share these across EU Member States.

Read here the full article about the LSE study:


Panos Kanavos and Victoria Tzouma

LSE Health, London School of Economics and Political Science

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