OBJECTIVES: To review health technology assessment (HTA) decisions for rare cancer therapies and rationale for these decisions across EU5 countries.

METHODS: We used the Global Market Access Solutions (GMAS) database to assess HTA decisions for seven rare cancers across EU5 countries. Additional information was extracted from individual HTA reports. Decisions were categorised as recommended, restricted, or rejected. Clinical and economic factors influencing decisions were analysed.

RESULTS: In total,

CONCLUSIONS: Manufacturers developing new therapies in rare cancers should consider providing robust clinical data in HTA submissions. Provisions are made by HTA agencies for early access and managed entry programmes, which allow for some uncertainty when unmet need is high and few or no alternative therapies are available. However, robust economic analysis that adjusts for uncertainty and incorporates appropriate cost and utility values could facilitate reimbursement, particularly in markets driven by cost-effectiveness.