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PP12-89: Reimbursement of Orphan Drugs in EU-5 between 2010-2019: A Landscape Assessment

Poster Presenter

      Tulika Paul

      • Associate Consultant
      • IQVIA


The study aimed to assess the current health technology assessment (HTA) landscape of orphan drugs in EU-5 and compare and identify the key drivers of reimbursement decisions across HTA agencies in these countries. Co-Authors: Suprapta Ghosh; Mou Chatterjee; IQVIA, Gurugram, India


All drugs that were granted orphan designation between 2010-2019 by European Commission (EC) were identified from the European Medicines Agency website. Current HTA decisions for the EC-approved orphan drugs were analyzed using IQVIA™’s proprietary platform HTA Accelerator across EU-5 HTA agencies.


A total of 74 drugs were granted orphan designation for 86 unique orphan conditions, between 2010-2019, by the EC. Among these 74 drugs, 48 received authorization, 8 received conditional authorization, 3 are not authorized yet, 4 were refused authorization and 11 were voluntarily withdrawn by the manufacturer. 73% (n/N=35/48) of the authorized drugs used patient-reported outcomes (PROs), indication-specific surveys and quality of life (QoL) questionnaires as supporting evidence for HTA evaluations. Additionally, data from real-world studies were also used by 58% (n/N=28/48) of the authorized drugs. Among the studied HTA agencies, highest number of assessment reports were published by HAS for 37 authorized orphan drugs, followed by G-BA for 30 drugs and IQWiG for 28, whereas AETSA published the least for only 3 drugs. Among the authorized drugs, Imbruvica had the highest number of HTA submissions (n=34). Majority of submissions with positive recommendation were provided by HAS (n=45), followed by G-BA (n=36) and SMC (n=17). NICE submissions had the highest number of ‘positive recommendation with restrictions’ (n=18), with 'use in specific patient subgroup', 'contingent on risk sharing agreement', and 'treatment sequence' being the common restriction criteria. Positive recommendations were driven by demonstration of ‘significant clinical benefit’, ‘robust economic analysis’, ‘addressal of high unmet need’ or ‘innovative nature of the product’. Highest number of submissions with negative recommendation was provided by SMC (n=20) citing ‘insufficient clinical evidence’ and ‘non-robust economic analysis’. Among all HTA submissions, cost-utility analysis was the most commonly reported economic analysis. To enable favorable patient access, simple discount on list price was commonly recommended by the HTA agencies.


In the last decade, more than half of the drugs designated as “orphan drug” received marketing authorization by EMA indicating focus towards addressing unmet need in orphan disease area. Majority of these drugs received positive reimbursement recommendations across HTA authorities in the EU-5 countries. Orphan diseases are challenging to study majorly because of its low incidence, which results in insufficient or poor-quality clinical evidences that often leads to negative recommendation. Therefore, evidences derived from real-world studies and PROs can support regulatory and HTA assessments, which is evident from the analysis of HTA landscape of the approved orphan drugs in last decade. Furthermore, detailed analysis of the published HTA assessment reports of recently approved orphan drugs helps to identify the key decision drivers of favorable reimbursement decisions in orphan conditions and facilitates patient access to innovative treatment options.