Drugs

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

Twitter: #OCEOutcomes19

Co-Sponsor: American Society of Clinical Oncology (ASCO)

Co-Chairs:

  • 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

Date: July 12, 2019

Time: 8:15 am to 4:30 pm

Location:

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

Background:

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.

Workshop Objectives:

  • 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

Webcast Information:

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.

Meeting Information:

9:00 am – 9:15 am

Introduction
Welcome and Opening Remarks
Objectives:

  • Provide an overview of meeting goals and review agenda

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

  • 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” setting, carefully defining the research question to develop appropriate endpoints is critical to inform health policy decisions

10:30 am – 11:00 am
Break

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

  • Current landscape and quality of physical function submission information
  • Highlight need for standardization of its estimands for descriptive, physical function and comparative physical function
  • Provide a brief overview of ICHE9 estimand guidelines (1 slide)
  • If we had 12 months of physical function data- what question would each stakeholder ask?
  • Q&A

12:00 pm - 1:00 pm
Lunch

1:00 pm - 3:15 pm
Session III
Efforts to standardize an analysis framework for physical function in medical product review and labeling
Objectives:

  • Describing patient perspective on treatment physical function across cancer trials in the interventional arm
  • Supporting a marketing claim of comparative physical function

3:15 pm - 3:30 pm
Break

3:30 pm - 4:30 pm
Session IV
Summary of the Day- Generalizing physical function learnings
Objectives:

  • Generalize workshop findings across therapeutic areas in broader PFDD FDA initiatives
  • Update on OCE regulatory review work with respect to PRO analysis and review
  • Obtain feedback from industry on where they see clinical outcome efforts going

Contact:

Sherwin Sapasap, MS, MBA, MHA
Oncology Center of Excellence
Office of Medical Products and Tobacco
Office of the Commissioner
Sherwin.Sapasap@fda.hhs.gov

Nicole Jenkins
Program Coordinator, Research & Analysis
Center for Research & Analytics (CENTRA)
American Society of Clinical Oncology
571-483-1346
Nicole.Jenkins@asco.org

Page Last Updated: 02/27/2019
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