OBJECTIVES: A growing number of European joint Health Technology Assessment (HTA) initiatives have emerged, driven by an increasing number of high-cost medicines. This research aims to provide an overview of the current cross-country HTA landscape and identify implications for orphan drugs (ODs) undergoing joint HTA.

METHODS: Five multi-country HTA initiatives (EUnetHTA, FINOSE, BeNeLuxA, Valletta Declaration and FAAP) were selected on their level of establishment and focus on joint HTA. Information was retrieved via a targeted literature review of healthcare authority websites, peer-reviewed articles, and grey literature. A qualitative analysis across the initiatives compared the scope, objectives, activities, member countries and number of therapies reviewed. Nusinersen, as the first OD to undergo successful joint BeNeLuxA HTA, was selected to extrapolate key learnings.

RESULTS: EUnetHTA was identified as the largest collaboration, comprising of thirty countries; FINOSE, BeNeLuxA and FAAP were found to be the smallest, comprising five each, while the Valletta Declaration comprises ten. Amongst the groups, the highest number of joint HTAs was found to be conducted by EUnetHTA. The scope of the health technologies targeted by the collaborations differed, however a focus on high-cost therapies was detected.

Nusinersen achieved similar pricing and reimbursement decisions in Belgium and the Netherlands, through BeNeLuxA. The benefit in review speeds varied across markets; the Belgian decision was reached after 13 months (versus 14 months on average for national processes), the Dutch decision was 13 months (versus 9 months). Representatives of BeNeLuxA highlighted Biogen’s willingness to proactively engage with the initiative during the process.

CONCLUSIONS: Joint HTA initiatives vary in size and activity level, and many groups focus on expensive, innovative medicines. These collaborations could result in more efficient HTA and pricing processes, leading to faster than average reimbursement decisions in some countries. Proactive engagement and collaboration by manufacturers with these groups could support faster patient access to ODs.