Analysis of EORTC treatment optimisation studies shows their value to patients and healthcare providers

The current evidence gap between pre- and post-authorisation stages of cancer drug development handicaps effective decision-making by clinicians, payers, and health technology assessment bodies. Treatment optimisation studies help to plug that gap, but are their methodological and organisational features used to their best advantage? This is the question that was addressed in a paper* published today in the European Journal of Cancer.

EORTC research fellow Robbe Saesen and colleagues set out to identify the most important characteristics of treatment optimisation studies (TOSs) performed by the EORTC. “Such studies address research questions that are typically not commercially attractive and that are therefore under-explored in industry-sponsored trials. The EORTC has been spearheading efforts to promote the conduct of TOSs, and the results of the study we undertook provide a model that others can follow in this regard,” he says.

The researchers searched the EORTC clinical trials database for studies on breast, lung, and colorectal cancer that could be classified as TOSs. They then explored the design, organisational aspects, and the scientific impact of the 113 studies that were selected, which were mainly standard-sized, multicentre international trials featuring a relatively simple randomised, open-label design. A typical objective was to compare the efficacy of an investigational drug regime against that of the current standard of care in terms of improving patients’ overall survival. Their scientific significance was found to be substantial, and this was evidenced by the number of citations they received following their publication in high-impact journals.

The results were in line with what stakeholders in European drug development have said that TOSs should resemble and confirmed their demands and expectations. They underscored the important role that not-for-profit research institutions and academic groups play in setting up potentially practice-changing trials whose primary objective is not commercially oriented, as was shown by the large number of EORTC TOSs performed over the years.

“Though it was good to have the quality and utility of these studies confirmed, one major thing that needs to be improved is their quantity. Even though the EORTC has conducted many of them, there are still important questions left unanswered for many anticancer therapies on the market today. A more systematic approach is needed in which these types of studies are carried out routinely. This will require structural funding mechanisms and support from regulators,” Mr Saesen says.

EORTC will follow up this work in May, when the findings will be presented at a multi-stakeholder scientific workshop organised with the European Medicines Agency.  “We hope to discuss the future of European TOSs across Europe,” says Mr Saesen. The investigators are also continuing to look at the impact of the EORTC TOSs included in the study on clinical guidelines in the field of oncology.

The EORTC is a unique organisation with a rich history of conducting academic cancer trials, so it could be difficult to find a similar institution to make the comparison in other disease areas.  “However, we are convinced that there is a need for such studies in other areas and believe other academic research organisations can follow in the EORTC’s footsteps and learn from its experiences,” Mr Saesen concludes.

* https://doi.org/10.1016/j.ejca.2021.04.012