FDA-ASCO Public Workshop: 2019 Clinical Outcome Assessments in Cancer Clinical Trials
Co-Sponsor: American Society of Clinical Oncology (ASCO)
- U.S. Food and Drug Administration
Paul Kluetz, MD
Associate Director of Patient Outcomes, Oncology Center of Excellence, FDA
- American Society of Clinical Oncology
Heidi Klepin, MD, MS
Chair, Cancer Research Committee, ASCO
Stacy W. Gray, MD
Cancer Research Committee, ASCO
Date: July 12, 2019
Time: 8:15 am to 4:30 pm
Food and Drug Administration White Oak Campus
Great Room, Building 31, Room 1503 (B and C)
10903 New Hampshire Avenue
Silver Spring, MD 20993
Malignant hematology and oncology (cancer) is an active area of drug development where patient-centered measures of symptoms and function can add important complementary data to inform the risks and benefits of new therapies. Clinical Outcome Assessments (COA) are trial measures that quantify how a patient feels or functions, and patient-reported outcome (PRO) assessments are one common form of COA used in cancer trials. PRO is defined as a measurement based on a report that comes directly from the patient about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else.
Past FDA workshops have identified disease symptoms, symptomatic adverse events and physical function as core outcomes of interest for FDA product review and have reviewed what characteristics are necessary for a PRO tool to be considered fit for purpose for regulatory decision making. The international community is also interested in identifying core clinical outcome sets that can be used to facilitate registries, pragmatic trials and other data sources that can be used to inform international regulatory, payer, provider and patient decision making. As one key functional domain of health-related quality of life, physical function, is a well-defined clinical outcome that is important across cancer types and can be measured by both existing PRO tools as well as emerging wearable technologies.
While physical function holds promise to be one important component of a core outcome set, a lack of clear research objective has resulted in heterogeneity in measurement tools, assessment frequencies, endpoint definitions and analytic approaches. The purpose of this workshop is to provide an open public forum for a broad representation of stakeholders to review current opportunities for clinical outcome measurement, explore a systematic approach to developing standard research objectives for physical function and generalize this approach to other outcomes and the broader patient-focused drug development effort.
- Provide a forum for academia, industry, international regulatory, HTA bodies, payers, and patient groups to discuss measurement of clinical outcomes
- Explore the use of ePRO in learning healthcare systems
- Review the ICH-E9-R1 structured “estimand” framework as a guide to link research objectives to suitable trial design, COA tools and endpoints to maximize the utility and interpretability of physical function assessments
- Obtain feedback on several cases of physical function research objectives and their impact on trial design and endpoint construction
- Generalize learnings to the broader FDA patient-focused drug development effort
FDA will provide a free-of-charge, live webcast of this workshop. Further information regarding the webcast, including the web link for the webcast, will be made available closer to the workshop date.
FDA plans to post archived webcasts after the meeting, however, in cases where transmission was not successful, archived webcasts will not be available.
Sherwin Sapasap, MS, MBA, MHA
Oncology Center of Excellence
Office of Medical Products and Tobacco
Office of the Commissioner
Program Coordinator, Research & Analysis
Center for Research & Analytics (CENTRA)
American Society of Clinical Oncology