Analysis of Reimbursement Outcomes for Orphan Drugs with EMA Exceptional Circumstances Designation in France, Germany, and the UK
Author(s)
Lach K*1;Pisarczyk K1;Beckerman R2, Meletiche D3
1Maple Health Group, LLC, Krakow, Poland, 2Maple Health Group, LLC, New York, NY, USA, 3Maple Health Group, LLC, Cambridge, MA, USA
OBJECTIVES: European Medicines Agency’s (EMA) exceptional circumstances designation (ECD) is granted in case comprehensive efficacy and safety data cannot be collected due to disease rarity or ethical concerns. The objective of this study was to compare reimbursement outcomes for orphan products with and without ECD in France, Germany, and the UK.
METHODS: All EMA-approved orphan products from 2015, with and without ECD, were analyzed for reimbursement outcomes by Haute Autorité de Sante (HAS), Gemeinsamer Bundesausschuss (G-BA), and The National Institute for Health and Care Excellence (NICE) or Scottish Medicines Consortium (SMC). Reimbursement outcomes were stratified by favorable, defined as: at least ASMR IV (HAS), minor added benefit (G-BA), and recommended (NICE/SMC); or unfavorable, defined as: ASMR V (HAS), no added benefit or non-quantifiable benefit (G-BA), and not recommended (NICE/SMC). Odds ratio (OR) of unfavorable reimbursement outcomes was calculated using Fisher's exact test.
RESULTS: A total of 67 orphan products, including nine (13.4%) with ECD were identified with 212 available reimbursement outcomes by HAS, G-BA, and NICE/SMC. 57% of orphan products without ECD were granted a favorable reimbursement outcome compared to 38.5% orphan products with ECD. OR of an unfavorable reimbursement outcome was twice higher for orphan products with ECD than without ECD, although not statistically significant (OR=2.12, p=0.080). However, when analyzing reimbursement outcomes only by HAS, and G-BA, OR of an unfavorable reimbursement outcome was threefold higher and statistically significant (OR=3.17, p=0.033). Small population size and non-comparative design were common factors contributing to unfavorable reimbursement outcomes.
CONCLUSIONS: Our analysis found that orphan products with ECD have much higher odds of receiving unfavorable reimbursement outcomes by HAS and G-BA as compared with orphan products without ECD. Future research should focus on examining whether e.g. real-world evidence generation efforts play a role in maintaining the reimbursement outcome for orphan products with ECD.
METHODS: All EMA-approved orphan products from 2015, with and without ECD, were analyzed for reimbursement outcomes by Haute Autorité de Sante (HAS), Gemeinsamer Bundesausschuss (G-BA), and The National Institute for Health and Care Excellence (NICE) or Scottish Medicines Consortium (SMC). Reimbursement outcomes were stratified by favorable, defined as: at least ASMR IV (HAS), minor added benefit (G-BA), and recommended (NICE/SMC); or unfavorable, defined as: ASMR V (HAS), no added benefit or non-quantifiable benefit (G-BA), and not recommended (NICE/SMC). Odds ratio (OR) of unfavorable reimbursement outcomes was calculated using Fisher's exact test.
RESULTS: A total of 67 orphan products, including nine (13.4%) with ECD were identified with 212 available reimbursement outcomes by HAS, G-BA, and NICE/SMC. 57% of orphan products without ECD were granted a favorable reimbursement outcome compared to 38.5% orphan products with ECD. OR of an unfavorable reimbursement outcome was twice higher for orphan products with ECD than without ECD, although not statistically significant (OR=2.12, p=0.080). However, when analyzing reimbursement outcomes only by HAS, and G-BA, OR of an unfavorable reimbursement outcome was threefold higher and statistically significant (OR=3.17, p=0.033). Small population size and non-comparative design were common factors contributing to unfavorable reimbursement outcomes.
CONCLUSIONS: Our analysis found that orphan products with ECD have much higher odds of receiving unfavorable reimbursement outcomes by HAS and G-BA as compared with orphan products without ECD. Future research should focus on examining whether e.g. real-world evidence generation efforts play a role in maintaining the reimbursement outcome for orphan products with ECD.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Code
RO2
Topic
Health Policy & Regulatory, Health Technology Assessment, Study Approaches
Topic Subcategory
Decision & Deliberative Processes, Literature Review & Synthesis, Reimbursement & Access Policy
Disease
Rare and Orphan Diseases