ABSTRACT
Objectives: Post-COVID, healthcare systems are balancing the need to improve health outcomes with expenditure. Healthcare and payer policy reforms have been approved or are being discussed in major European markets: France, Germany, Italy, Spain and England. This study outlines the evolving access landscape and potential implications on orphan drug (OD) access. Method(s): For each market, qualitative and quantitative analyses were performed on information collected via targeted literature review of publicly available sources: national authority websites, peer-reviewed articles, and grey literature. Findings were segmented into policy scope, policy occurrence likelihood and OD access impact. Policy scope comprised 4 categories: access, pricing, assessing methodology and wider healthcare policy. Occurrence likelihood was subdivided into occurred, expected, and discussed reforms. The OD access impact (positive/negative) was categorised as high, intermediate, and low. Analysing the relationship between likelihood of occurrence and impact to OD access pinpointed reforms most likely to change the OD payer landscape. Result(s): This research identified 29 reforms: 8 in England, 7 in Italy, 6 in France, 4 in Spain and Germany. Our analysis framework identified highly significant reforms: 3 in France and Germany, 2 in Italy, and 1 in England and Spain. England and Italy have the highest number of reforms encouraging OD launch. France showed a relative increase in attractiveness, while Spain lagged. Draft legislation in Germany appeared the most punitive for OD access to patients. Conclusion(s): Overall, attractiveness for OD launch is increasing in Europe. Due to the size of market potential, cost containment measures in Germany may hinder European launch strategy and mitigate the positive impact of increased OD patient access in other markets. Monitoring of upcoming changes alongside working closely with physician and patient advocacy groups to maintain OD funding priorities will ensure rare disease patients are not left behind and can continue to access therapies. Copyright © 2022