Perspectives on Patient-Reported Outcome Data After Treatment Discontinuation in Cancer Clinical Trials

Patient-reported outcome (PRO) data are critical in understanding treatments from the patient perspective in cancer clinical trials. The potential benefits and methodological approaches to the collection of PRO data after treatment discontinuation (eg, because of progressive disease or unacceptable drug toxicity) are less clear. The purpose of this article is to describe the Food and Drug Administration’s Oncology Center of Excellence and the Critical Path Institute cosponsored 2-hour virtual roundtable, held in 2020, to discuss this specific issue. We summarize key points from this discussion with 16 stakeholders representing academia, clinical practice, patients, international regulatory agencies, health technology assessment bodies/payers, industry, and PRO instrument development. Stakeholders recognized that any PRO data collection after treatment discontinuation should have clearly defined objectives to ensure that data can be analyzed and reported. Data collection after discontinuation without a justification for its use wastes patients’ time and effort and is unethical. •All roundtable participants agreed that it is important to understand long-term effects, from the patients’ perspective, of treatments under investigation in clinical trials, which includes the period after the study treatment has ended. This information is of critical importance for regulatory, payer, clinical, and patient decision making.•To ensure the most effective use of patient-reported outcome (PRO) data collected in the period after study treatment has ended, clinical trial sponsors need to set a research question before designing the collection of the PRO data needed to answer that research question.•The patient advocates who attended this roundtable agreed that patients will complete PRO measures in this period if it is made clear why their input is valuable.