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  1. News & Events for Human Drugs

FDA-ASCO Public Workshop: 2019 Clinical Outcome Assessments in Cancer Clinical Trials Fourth Annual Workshop

Twitter: #OCEOutcomes19

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

FDA Campus Information, (e.g., local airports, directions, and local hotels)

Registration: https://www.surveymonkey.com/r/2019COA-CCT


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.


Webcast Information:

FDA archived webcast of this meeting is available as follows:
Webcast/Adobe Connect:

Meeting Information:

9:00 am – 9:15 am

Welcome and Opening Remarks

  • Provide an overview of meeting goals.
  • Review agenda


  • Paul Kluetz, MD
  • Heidi Klepin, MD

9:15 am – 10:30 am
Session I
Exploring the value of electronic PRO assessment to facilitate learning healthcare systems


  • Discuss use of ePRO in healthcare systems and potential for clinical care, quality improvement, registries, prospective pragmatic trials, and real-world data
  • Acknowledge the importance of physical function across therapeutic areas
  • Recognize whether obtained in trial, or “real-world” settings, carefully defining the research question to develop appropriate endpoints is critical to inform health policy decisions


  • Ethan Basch, MD, MSc


  • KC Clevenger, PhD, RN, CPNP, CCRP
  • Piyush Srivastava, MD
  • Pall Jonsson, PhD
  • Corrie Painter, PhD
  • David Martin, MD, MPH

10:30 am – 11:00 am

11:00 am-12:00 pm
Session II
Systematically defining research objectives and framing questions using the estimand framework


  • Review the current landscape and quality of physical function submission information
  • Introduce the ICHE9 estimand guideline as a framework to develop rigorous PRO endpoints
  • Explore a case study - If a trial had captured 12 months of physical function data at each cycle of therapy and concluded that “there was no difference between the two treatment arms on physical function”, what conclusion would each stakeholder draw?
  • Q&A


  • Bellinda King-Kallimanis, PhD
  • Laura Lee Johnson, PhD


  • Sigrid Klaar, MD, PhD
  • Jim Shaw, PhD, PharmD, MPH
  • Katherine Szarama, PhD
  • Lori Minasian, MD
  • Jane Perlmutter, PhD, MBA

12:00 pm - 1:00 pm

1:00 pm - 3:15 pm
Session III

Using a standardized estimand framework for medical product review and labeling: a case study


  • Research Objective 1: Supporting a marketing claim of comparative physical function
  • Research Objective 2: Describe on-treatment physical function


  • Mallorie Fiero, PhD
  • Chana Weinstock, MD
  • Madeline Pe, PhD


  • Andrea Ferris, MBA
  • Sigrid Klaar, MD, PhD
  • Alicyn Campbell, MPH
  • Surya Singh, MD
  • David Cella, PhD
  • Kim Cocks, PhD

3:15 pm - 3:30 pm

3:30 pm - 4:30 pm
Session IV

Seeing the Forest Through the Trees – Where do we go from here?


  • Generalize workshop findings across therapeutic areas in broader Patient-Focused Drug Development (PFDD) FDA initiatives
  • Provide an update on Oncology Center of Excellence (OCE) core outcomes, analysis, and review
  • Discuss the role of PRO in cancer trials and real-world settings, and its impact in product development and FDA review


  • Stacy Gray, MD
  • Ethan Basch, MD, MSc


  • Theresa Mullin, PhD
  • Paul Kluetz, MD
  • Amy Abernethy, MD, PhD


Sherwin Sapasap, MS, MBA, MHA
Oncology Center of Excellence
Office of Medical Products and Tobacco
Office of the Commissioner

Nicole Jenkins
Program Coordinator, Research & Analysis
Center for Research & Analytics (CENTRA)
American Society of Clinical Oncology

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