OBJECTIVES: Tumour-agnostic therapies, a new class of medicines that target a genomic alteration within a tumour regardless of where it is in the body, face significant methodological and policy challenges related to evidence development and acceptance, value assessment and reimbursement pathways, and diagnostic infrastructure availability. To better understand the challenges and opportunities in various countries, we performed an in-dept analysis of (adaptive) pathways for tumour-agnostic therapies.

METHODS: We reviewed relevant peer-reviewed and grey literature including research papers, white papers, methodological / working papers and regulatory and HTA assessments up to June 2020, and performed eleven semi-structured interviews with experts in regulatory affairs, market access, health technology assessment (HTA), pharmaceutical and health policy from six countries (Canada, Germany, France, Italy, Spain, and UK).

RESULTS: Tumour-agnostic therapies have been able to gain accelerated access via adaptive regulatory pathways, yet existing HTA processes provide little flexibility to deal with the specific challenges surrounding these innovative therapies. Positive reimbursement recommendations are scarce and usually limited to specific indications. Challenges in developing comparative evidence are considered the main hurdle, followed by the prognostic value of the genomic alteration defining the tumour-agnostic approach, knowledge on natural history of identified patients’ populations, and availability of diagnostic infrastructure and cost attribution of that to a single tumour-agnostic therapy. Potential solutions prioritised by experts varied widely yet included better alignment of evidence sources, endpoint requirements, acceptability of post-authorisation data collection models and innovative payment models.

CONCLUSIONS: Despite challenges for tumour-agnostic treatments being similar in various countries, solutions will likely have to be diverse and include methodological advancements as well as policy-makers’ willingness to adopt new approaches.