EORTC and EMA, jointly with international stakeholders, have published a multistakeholder roadmap to accelerate treatment optimisation in oncology

EORTC, together with the European Medicines Agency (EMA), and in collaboration with key stakeholders have published a multistakeholder roadmap outlining strategic actions to advance treatment optimisation research in oncology. The recommendations were developed within the Cancer Medicines Forum (CMF)¹ and are presented in a new article titled “Accelerating cancer treatment optimisation: a multistakeholder roadmap from the Cancer Medicines Forum” published in the Journal of Cancer Policy.

Treatment optimisation research focuses on determining how anticancer therapies can be used most effectively in clinical practice, addressing questions related to dose, duration, schedule, sequence, and combinations. While current regulatory trials support marketing authorisation, many of these questions remain unanswered at approval, creating challenges for clinicians, patients, and healthcare systems.

The roadmap is based on a multidisciplinary workshop held in April 2024 within the CMF, which brought together multiple international stakeholders, including regulators, health technology assessment bodies, clinicians, patient representatives, academic researchers, industry, public health organisations. The discussions align with ongoing EORTC initiatives to support treatment optimisation, including a recent event held in the European Parliament focused on the challenges and opportunities in this area. Participants identified key barriers, including limited incentives, funding constraints, regulatory hurdles, and challenges in implementing optimisation findings, and proposed coordinated solutions.

“Recognising treatment optimisation as a public health priority is essential to ensuring that cancer therapies are used as effectively and sustainably as possible,” said Dr Denis Lacombe, EORTC CEO. “Strengthening collaboration and creating the right conditions for pragmatic clinical trials will be key to making this happen.”

The roadmap also emphasises the importance of aligning evidence requirements across regulators and HTA bodies to ensure that optimisation results can inform regulatory approvals, clinical practice and reimbursement decisions.

EORTC remains committed to supporting independent, practice-informing research and to strengthening evidence generation throughout the lifecycle of cancer medicines.

¹The Cancer Medicines Forum is co-chaired by EORTC and EMA and brings together key organisations across the oncology ecosystem.