FDA guidance – patient-centered oncology research

Looking Forward: FDA’s Core Patient-Reported Outcomes in Cancer Clinical Trials Final Guidance for Industry

Kelly Dumais, Ph.D. – Director, eCOA Science and Consulting at Clario

*Updated on June 12, 2025 to reflect the time passed since the FDA released the guidance.

The evolving regulatory landscape of oncology clinical trials has just added another guidance to its docket. *In October 2024, the FDA released the final guidance for industry on “Core Patient-Reported Outcomes in Cancer Clinical Trials“¹, issued by the Oncology Center of Excellence (OCE), Center for Biologics Evaluation and Research (CBER), and Center for Drug Evaluation and Research (CDER).

The FDA originally released the draft version of this guidance in June 2021, which was a big step forward in the acknowledgement of the importance of patient-centered research within the oncology field. There have been many initiatives coming out of FDA’s OCE, whose mission is to “achieve patient-centered regulatory decision-making.” Annual public workshops on clinical outcome assessments (COAs) in cancer clinical trials, patient-focused programs such as Project Patient Voice and Project Community, are a few of these initiatives that have provided best practices on collecting and disseminating oncology patient-reported outcome (PRO) data.

This new final guidance is very similar to the draft guidance with no significant updates. The FDA has provided recommendations on how to best incorporate PRO measurements into oncology clinical trials in efforts to correct the historical “heterogeneity in PRO assessment strategies [that] has lessened the regulatory utility of PRO data from cancer trials.¹” It provides guidance on PRO strategy, trial design considerations, and labeling considerations, including recommended procedures when seeking PRO-based labeling.

In line with recommendations for a core set of PROs described previously in the literature,² the new FDA Guidance recommends collecting PROs that map to these core concepts:

1. Disease-related symptoms
2. Symptomatic adverse events
3. Overall side effect impact summary measure
4. Physical function
5. Role function

The guidance includes examples of PROs for all categories, and states that additional PROs outside of these core categories may be beneficial (e.g., measuring cognitive function in neuro-oncology). Importantly however, the FDA acknowledges the need to minimize patient burden as much as possible, by “focusing on the most meaningful and measurable outcomes,” and selecting only “the most important and/or high frequency Adverse Events (AEs) to reduce question burden.¹” The guidance also states that the use of electronic PROs (ePROs) may be a useful method for reducing patient burden by allowing for at-home completion.  Other trial design considerations include putting procedures in place to mitigate missing data, including “training for investigators and patients, a completion monitoring strategy (e.g., reminders to patients), and obtaining PRO data from patients at time of early withdrawal.”

Frequency of PRO administration was another important topic, which aligns with recommendations discussed at the 5th annual FDA-ASCO Public Workshop on COAs in cancer clinical trials. The frequency of PRO assessments should be thoughtfully considered to be sure that the patient symptomatology is being adequately captured and represented. For example, assessment frequency should take into account the administration schedule of the treatment and the administration type (e.g., intravenous or oral), as these will have a significant impact on when symptomatic AEs may occur. Importantly, while recommending to have more frequent assessments within the first few treatment cycles, the FDA keeps with their theme of considering patient burden by suggesting that, when appropriate, different assessment frequencies can be selected for each PRO in order to reduce response burden.

With more people living with cancer now than ever before, it is imperative to truly listen to the patient voice and measure what is most important to patients as we seek to deliver new treatment options. By providing guidance on what core PRO categories to include, considerations for instrument selection for each category, trial design considerations, and PRO labeling considerations, this new draft guidance is a significant step forward in our ability to collect consistent PRO concepts in order to better evaluate and compare data across clinical trials.

Find out how Clario can support your decentralized ePRO capture in oncology clinical trials – visit our eCOA in oncology page or contact us at clario.com/contact

References:

¹ FDA Guidance for Industry: Core Patient-Reported Outcomes in Cancer Clinical Trials. October 2024. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/core-patient-reported-outcomes-cancer-clinical-trials

² Kluetz PG, Slagle A, Papadopoulos E, et al., 2016, Focusing on Core Patient-Reported Outcomes in Cancer Clinical Trials: Symptomatic Adverse Events, Physical Function, and Disease-Related Symptoms, Clin Can Res, Apr 1;22(7):1553-8