Accelerating cancer treatment optimisation: A multistakeholder roadmap from the Cancer Medicines Forum

Medicines are often tested in highly controlled environments during clinical trials aimed at rapid development and thorough assessment of their benefits and risks, with key efficacy and safety aspects evaluated for regulatory approval. However, important questions regarding the optimisation of medicines' use in clinical practice may remain unanswered at the time of marketing authorisation, notably those related to the optimal dose, duration, schedule, sequence, and combination of treatments. This knowledge gap is particularly critical in oncology, where patient quality of life must be prioritised, and there is the pressing need to understand the most efficient use of costly innovative therapies in the context of rising cancer incidence and prevalence. Addressing these challenges requires multi-stakeholder collaboration. Organisations within the Cancer Medicines Forum (CMF), co-chaired by the European Organisation for Research and Treatment of Cancer and the European Medicines Agency, have advocated for policy actions promoting treatment optimisation research within the European Union. To advance this objective, the CMF convened a multidisciplinary stakeholder workshop, offering a platform for collaborative dialogue by bringing together experts from governmental bodies, regulatory agencies, health technology assessment bodies, patient organisations, academia, learned societies, industry, public health organisations, clinicians, and investigators. This workshop aimed to examine existing barriers and explore strategies to integrate treatment optimisation into the current cancer clinical research framework, ultimately providing key recommendations, summarised in this review, to systematically embed treatment optimisation in oncology research.