Innovative oncology trial designs: Time to act – A review with recommendations of the Cancer Drug Development Forum

Abstract

Introduction

Clinical oncology trial design constantly adapts and evolves to meet the needs of all stakeholders, from patients to regulators. This evolution has however also led to an increase in the complexity of clinical development, and investigators are expected to invest more time and money in planning and running their studies and overall clinical and regulatory pathway.

Methods

Here we review recent innovations in trial designs, study endpoints, and relevant regulatory guidelines, before making recommendations to enhance the clinical trial ecosystem in the EU.

Results

Innovative clinical study designs that go beyond traditional randomised, double-blinded, placebo-controlled trials often promise greater flexibility and efficiency in the conduct of oncology studies, but adoption has been slow. Furthermore, the increased complexity associated with innovative trials means that coordination between all relevant stakeholders is essential to every phase of clinical development. Despite recent advances, there is a risk that Europe is becoming seen as a less attractive location for clinical trials, and this article outlines initiatives and possible steps to improve patient access to medicines and processes across Europe.

Policy summary

Innovative trial designs promise more efficient, flexible, and inclusive clinical trials, but more needs to be done to encourage their adoption if the advantages outweigh the limitations. Partnership and coordination between all stakeholders, from patients to regulators, and at all phases, is more important than ever. Finally, effective action is needed to make Europe a more attractive destination for clinical research and to improve access to innovative medicines for patients.