Co-Sponsor: American Society of Clinical Oncology (ASCO)
- U.S. Food and Drug Administration
Paul Kluetz, MD
Deputy Director, 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: 9:00 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.
Meeting Goal: To explore the use of physical function as an outcome measure and advance the standardization of data collection, COA tools, endpoints, analysis, and visualization of physical function in oncology for regulatory decision-making.
- Exploring measurement of physical function to inform cancer drug development (PDF - 853 KB)
- COA-CCT Session IIIUsing a standardized estimandframework for medical product review and labeling: a case study (PDF - 652 KB)
- Summary of the Day-Seeing the Forest Through the Trees (PDF - 265 KB)
- Efforts to Advance Core Clinical Outcomes and Standard Analyses in Oncology (PDF - 137 KB)
- Clevenger, KC Integrating Patient Reported Outcomes (PRO) into Practice (PDF - 1.14MB)
- Srivastava, P. Real World Data from Real World Patients (PDF - 635.54KB)
- King-Kallimanis, B. Systematically defining research objectives and framing questions using the estimand framework (PDF - 1.07MB)
- Mullin, T. FDA Patient-Focused Drug Development and why we are interested in Standard Core COAs (PDF -241.15KB)
FDA archived webcast of this meeting is available as follows: