Research poster presentations – session I Health care use & policy studies - health care costs & management| Volume 15, ISSUE 7, PA308, November 01, 2012

PHP110 Gaining Reimbursement of Orphan Products in Europe: Challenges Due to Wide Variations in Evidence Requirements and Processes


      Understand country-specific evidence requirements for the national reimbursement of orphan products (disease prevalence: <5/10,000) in the UK, France, Spain, Italy and Germany.


      Qualitative research identified guidelines for reimbursement submission from each national reimbursement body. 1–5 orphan product appraisals per country were also reviewed to gain insight into the application of guidelines. Findings are based only on review of the guidelines and selected appraisals.


      The markets studied do not have reimbursement processes specifically for orphan products, however special considerations exist. For example, while direct comparator trials are the preferred sources of clinical evidence for non-orphan products, in certain instances reimbursement of orphan products has been granted based on placebo-controlled trials accompanied by indirect treatment comparisons. In England, exemptions are made only for orphan products which extend survival in patients with short life expectancies. In Scotland, 62% of decisions on orphan indications have been negative, mostly due to a lack of robust economic evidence. In Italy, access to orphan products is encouraged and 94% of those launched are reimbursed: a lower benefit-risk ratio and evidence outside randomized controlled trials is accepted for orphan products. In France, orphan products benefit from fast-track procedures; however no special evidence considerations are acknowledged. Under AM-NutzenV in Germany, the medical benefit of orphan products is considered confirmed through the marketing authorisation; thus orphan submissions require a limited submission document. Whilst no national practices for orphan products exist in Spain, some regions implement risk-sharing agreements.


      Whilst reimbursement bodies recognize data limitations and provide special considerations for orphan products, processes are not explicit and requirements vary, leading to uncertainty in the reimbursement evidence requirements. It is therefore important to engage with appropriate reimbursement bodies to fully determine their specific requirements and modify one's submission to demonstrate an orphan product's ability to meet payers' needs.