OBJECTIVES: To analyse timelines and the degree of involvement from the Market Authorisation Holder (MAH) in health technology assessment (HTA) of orphan drugs across European countries.

METHODS: Desktop research was conducted in January 2023. Reported timelines for orphan drug HTA processes were investigated, as well as any involvement from the MAH throughout each process. Involvement from the MAH was termed an ‘action’. The analysis included 10 countries: Belgium, England, France, Ireland, Italy, Germany, the Netherlands, Scotland, Spain, and Switzerland. Direct evidence from HTA body websites and reports, as well as secondary evidence from reviews, journal articles, and expert knowledge were used in the research.

RESULTS: The average duration of the HTA process from submission to reimbursement decision of the selected countries is 193 days. This timeframe excludes pre-submission activities, for which guidance is available in four countries: Belgium, England, Ireland, and Scotland. Germany has the longest HTA process overall (441 days), which can be attributed to price negotiations taking up to 270 days after a primary decision on the clinical data is made (up to 171 days after submission). The country with the shortest HTA process is Spain (90 days); however, in practice, this can take up to 120 days.

Belgium, England, Germany, Ireland, and Scotland required the most actions from the MAH, including pre-submission activities, with each country requiring five actions from the MAH. The Netherlands and Switzerland required the least involvement from the MAH, with two actions required.

CONCLUSIONS: This research highlights the large variations in HTA processes and timelines for orphan drugs across Europe, emphasising the importance of a streamlined market access launch plan. Holistic awareness of HTA processes across Europe allows for adequate allocation of resources throughout market access launch and can help ensure that patients get timely access to medicines in the rare disease space.