Keri J.S. Brady, PHD MPH; John Devin Peipert, PhD; Thomas M. Atkinson, PhD; Cecilia Pompili, PhD; Monica Pinto, MD; James W. Shaw, PhD PharmD MPH; and Jessica Roydhouse, PhD
Recently, the United States Food and Drug Administration (FDA), a major regulatory agency, released for industry that proposed a core set of patient-reported outcomes (PROs) for collection in registrational cancer trials (https://www.fda.gov/media/149994/download). This important new document provides information on regulatory thinking about PROs and is likely to influence decisions and considerations by researchers in industry and academia. The International Society for Quality of Life Research’s (ISOQOL’s) Standards and Best Practices Committee aimed to provide a perspective on this guidance (https://doi.org/10.1007/s11136-023-03396-z). The goal of this perspective was to discuss the positive aspects of the guidance and areas in which additional work or challenges remain. This perspective is aimed at people who are likely users of the draft guidance, including PRO researchers working in regulatory agencies, industry, or academic settings.
According to the draft guidance, core PROs are disease-related symptoms, symptomatic side effects, overall side effects summary measures, physical function, and role function. One very positive aspect of the guidance is how it shows a major regulatory body’s continued commitment to the inclusion of PROs and the patient voice as part of developing new treatments. The guidance also gives its end-users a framework for which PROs should be, at a minimum, assessed in cancer trials. This minimum expectation can help make comparisons across future trials easier.
Including PROs that relate to how patients perceive the side effect of treatments, which can be called treatment tolerability, was also positive. It is still not clear how treatment tolerability will be used in evaluating the benefits and risks of new treatments. Making this clearer would help in designing future trials. In general, most analyses of treatment tolerability so far have been descriptive. One area for future research is if new treatments will be compared in terms of their patient-reported treatment tolerability. This might require the development of new methods to determine clinically meaningful changes in treatment tolerability.
Another important question that should be addressed relates to the choice of symptomatic side effects. Cancer treatments can generate many symptomatic side effects. Discussing how to select side effect measures that are comprehensive but not overly burdensome to patients is an important next step.
A topic for future discussion is about the role that PROs, including PROs about side effects, will have in early phase trials. PROs are not always part of early phase trials but can contribute information when they are included. Collaboration between regulators, patients and researchers can help move this forward.
Finally, once PROs are part of trials, it is important to share the results. It was helpful that the guidance provided some information about how PROs can be included in the labels of new treatments. However, sometimes PROs are not included in these labels. More discussion of how PRO results can be shared is needed, so that they can be used for treatment decisions and help the broader community.
In the end, it was very exciting to see this draft guidance. We hope that use of this draft guidance to help design future trials, efforts to address the challenges, and additional work can lead to new treatments that are evaluated with patients at the center.
This newsletter editorial represents the views of the author and does not necessarily reflect the views of ISOQOL.
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