Defining the role of pragmatic clinical trials in clinical cancer research: outcomes of a collaborative workshop hosted by the European Organisation for Research and Treatment of Cancer (EORTC)

Brussels, 5 May 2025 – Pragmatic Clinical Trials (PCTs) represent a form of clinical research with high external validity and address important routine clinical questions based on medically relevant patient endpoints. With a largely inclusive target patient population, the questions PCTs are addressing include optimising therapeutic strategies such as dose, schedule, combination and sequence, inclusive of de-escalation trials to address over-utilisation and improve the quality of life of cancer patients.

However, PCTs can encounter challenges in their acceptability and conduct across all stakeholders. Acceptability by patients and doctors, inadequate legal frameworks, regulatory acceptability and challenging designs represent some of the limitations encountered today.

Therefore, EORTC convened a multistakeholder panel involving regulators, health technology assessors, patient advocates, investigators, clinicians, epidemiologists and statisticians to address the opportunities of PCTs for therapeutic progress. The outcomes of the workshop are today published in Lancet Oncology¹.

Professor Winette van der Graaf, EORTC President, reacted: “This workshop has been a game-changer to collectively embrace a revisited and refreshed role for pragmatic trials. PCTs can help doctors to adapt their practice for the optimal use of therapeutic agents and technologies. It conveys also an important dimension for the benefits of patients where patient-centric outcomes do prevail”

Dr Denis Lacombe, EORTC CEO follows with: “PCTs give us the possibility to reach many more patients wherever they are. They represent a form of public health research solution contributing to equal access to practice changing clinical research programmes”.

Fabio Borges, Research Fellow at EORTC emphasises: “PCTs deserve greater attention by policymakers due to their transformative potential for patients and society. An enabling eco-system and innovative funding solutions for PCTs can certainly deliver therapeutic progress for patients and be of major value to healthcare systems.”

EORTC is a unique clinical cancer research organisation that brings together many critical and empowered stakeholders and can set the tone for a new vision of how treatments are developed and made accessible to patients, leaving no one behind.

¹ Borges F et al. (2025). Defining the role of pragmatic clinical trials in cancer clinical research: outcomes of a collaborative workshop hosted by the European Organisation for Research and Treatment of Cancer. https://doi.org/10.1016/S1470-2045(24)00756-3 or https://authors.elsevier.com/c/1l0j85EIIgPf2B