OBJECTIVES: With advances in understanding the molecular basis of disease, the number of therapeutics with pan-tumor efficacy in oncology has grown exponentially since 2019. However, despite advances in precision oncology, current regulatory and value assessment frameworks are not optimized for indication-agnostic therapies, with different pricing and reimbursement outcomes across markets. This study aimed to understand the evidence requirements and reimbursement outcomes for these therapies.

METHODS: This analysis explored the evidence requirements and reimbursement outcomes for selected pan-tumor therapies in the US, UK, France, Germany, China, and Japan through a review of regulatory and health-technology assessment (HTA) reports.

RESULTS: Evidence requirements and assessment outcomes vary by geography, with the US more likely to accept pan-tumor data packages than ex-US markets. Despite several European regulatory approvals, the outcomes of national HTA for pan-tumor indications have been suboptimal. Common challenges included a lack of pan-tumor specific clinical guidance, comparator selection (or absence), endpoint selection, and differing willingness-to-pay across tumor types and subpopulations.

CONCLUSIONS: The challenges presented by current evidence standards in the appraisal of pan-tumor products highlight the need for evolution in payer approaches to reimbursement decision-making. Until appraisal methods evolve, manufacturers should consider the trade-offs of launch strategies leveraging a lead indication versus a pan-tumor initial indication. Policymakers need to recognize the evolving treatment landscape and ensure appraisal methods reflect the novel technologies coming to market.