BIO Comments on Core Patient-Reported Clinical Outcomes in Cancer Clinical Trials
On Thursday, June 10th, 2021, the U.S. Food and Drug Administration (FDA) published a draft guidance, Core Patient-Reported Clinical Outcomes in Cancer Clinical Trials, providing recommendations to sponsors on the collection of a core set of patient-reported clinical outcomes in cancer clinical trials and related considerations for instrument selection and trial design. According to the FDA, the Guidance is intended to facilitate generation of high-quality data on a core set of patient-reported symptom and functional impacts that are important contributors to a patient's health-related quality of life.
On Monday, August 9th, 2021, BIO submitted comments to the FDA on the draft guidance. While BIO does not typically comment on disease-specific guidances, BIO acknowledges that many of the concepts presented in the draft guidance can and should be utilized more broadly.
In the comments submitted, BIO suggests that the Agency provide clarity on FDA-Sponsor meetings regarding the use of PROs; for example, the timing, meeting type, recommended attendees, and necessary information for sponsors to prepare in anticipation of the discussion. Additionally, BIO notes that some of the Agency’s suggested PROs, such as disease-related symptoms and symptomatic adverse events, may appear repetitive and burdensome to patients. From a clinical perspective, BIO recommends a flexible approach in selecting functional scale(s) so that they can be chosen based on the context of a given disease, rather than adding a role function to the core concept. Further, we suggest adding treatment symptoms to the list of core PROs to avoid measurement redundancy across disease symptoms and treatment symptoms. Additionally, we believe it would be helpful if the FDA addressed the topic of PRO data collection and adverse event (AE) reporting in clinical trials and clarified how PRO data enhance safety data. Lastly, BIO states that it would be helpful if the FDA included more pediatric-specific recommendations in the Final Guidance, and if the Agency reiterated the goals of PFDD and confirmed how the core PRO set will inform regulatory decision-making beyond obtaining a label claim in the Final Guidance.