Incorporating patient-reported outcomes (PROs) into clinical trials can help sponsors better understand patients’ symptoms and how a therapy will affect their quality of life, and these insights can be particularly valuable in oncology research. However, there has been a lack of guidance to help sponsors ensure consistency and quality when incorporating PROs into cancer drug applications. That’s changed with the recent publication of a new draft guidance from the U.S Food and Drug Administration’s (FDA) Oncology Center of Excellence (OCE), Core Patient-Reported Outcomes in Cancer Clinical Trials.

To ensure reporting consistency, the OCE has been working with patients and outcomes research experts to determine which outcomes to measure, how frequently, and with which tools. The scope of this guidance is specifically for registration trials for anti-cancer therapies intended to demonstrate an effect on survival, tumor response, or delay in progression. The draft guidance recommends collecting and analyzing the following core PROs:

• Disease-related symptoms
• Symptomatic adverse events
• Overall side effect impact measurement summary measure
• Physical function
• Role function

The guidance recommends that sponsors consider additional PROs important to patients based on the specific trial — for instance, swallowing function would be of interest to patients with esophageal cancer. The guidance also addresses how to incorporate PRO assessments into the clinical trial, including considerations for assessment frequency, methods, and documentation. The guidance suggests that in some cases, PROs can be used to support labeling claims; inclusion of PRO data in the product label depends on the adequacy of the design and conduct of the trial, strengths and limitations of the instrument, and the quality of the data submitted. Companies considering this should consult with the FDA when selecting the PRO instrument for a particular clinical trial.

The new draft guidance reflects a larger trend in the use of non-traditional data sources in clinical trials, fueled in part by the rise of decentralized clinical trials. The patient is now a major stakeholder in terms of contributing to the overall data strategy. As patients become more remote, their contributions — whether from traditional PROs or electronic patient-reported outcomes (ePROs) such as wearables and other remote devices — are now relevant pieces of data.

With this new FDA guidance, oncology sponsors can be more confident than ever when it comes to incorporating patient-reported outcomes and other innovative solutions into their clinical development strategy. Successful implementation of these approaches requires careful consideration of the regulatory guidance, processes, and technologies necessary to ensure data quality and manage risk. Learn about our comprehensive regulatory consulting services and full-service strategic and tactical product development expertise in all phases of drug development.