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FDA Public Workshop: 2018 Clinical Outcome Assessments in Cancer Clinical Trials

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


U.S. Food and Drug Administration

  • Paul Kluetz, MD

American Society of Clinical Oncology

  • Heidi Klepin, MD, MS


June 22, 2018


8:15 a.m. to 5:00 p.m.


FDA White Oak Campus
Building 31, Room 1503 - Great Room
10903 New Hampshire Avenue
Silver Spring, MD 20993


The FDA and ASCO expressed a willingness to leverage their combined strengths for the joint development of the substantive actions of this workshop to identify best practices for the collection and analysis of patient-reported outcome (PRO) data in cancer clinical trials. The co-sponsors engaged with leaders in academia, industry, international regulatory and health technology assessment (HTA) bodies, and patient groups. Patient-reported outcome data are commonly integrated into trials, and this workshop evaluated current practices and identified opportunities to advance strategies that can provide rigorous PRO data suitable to support regulatory, payer, clinician and patient decision making.

Patient-focused drug development efforts are underway across therapeutic areas. Malignant hematology and oncology (cancer) is an active area of drug development where patient-centered measures of symptoms and function can add important data to inform the risks and benefits of cancer therapies. Clinical Outcome Assessments (COA) are trial measures that quantify how a patient feels or functions. Patient-reported outcomes (PRO), one form of COA, is 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. Unique issues exist in cancer clinical trials which make collection of patient-reported outcome (PRO) data challenging. This workshop provided an open public forum for a broad representation of stakeholders to (a) explore measurement tools and trial design features to generate patient-focused data from cancer clinical trials and (b) explore efforts to analyze, present and interpret PRO data obtained from cancer clinical trials.

Workshop Objectives:

  • To provide a forum for open discussion between academia, industry, international regulatory, HTA bodies, and patient groups to advance measurement of the patient experience in cancer clinical trials;
  • Seek feedback from stakeholders on current FDA thinking regarding characteristics of PRO measurement tools and trial designs that can be used to support product labeling;
  • Ensure the leading experts in PRO measurement have an opportunity to evaluate international efforts to standardize PRO data analysis from cancer trials;
  • Obtain feedback on current FDA approaches to the analysis and review of PRO data submitted to FDA to support cancer product applications.

Webcast Information: Recordings of the webcast can be found at the following links:

Session 1:
Session 2:
Session 3:
Session 4:

Meeting Materials:

Persons with disabilities having problems accessing the PDF file(s) below may call (301) 796-3634 for assistance.

Final Agenda  (PDF)

Cochairs: Paul Kluetz, MD, and Heidi Klepin, MD, MS

Session 1: What to Measure: PRO Core Concepts in Cancer Trials
Session Chair: Bellinda King-Kallimanis, PhD (No Slides)


Fit for Purpose Instruments
Selena Daniels, PharmD, MS
U.S. Food and Drug Administration

Illustrating the Issue
Lynn Howie, MD
U.S. Food and Drug Administration

Proposed Core PRO Assessment Strategy
Paul Kluetz, MD
U.S. Food and Drug Administration

Panel Discussion

Andrea Ferris (No slides)

Beate Wieseler, Dr. rer. nat.
Institute for Quality and Efficiency in Health Care (IQWiG)

Ethan Basch, MD, MSc
University of North Carolina

Daniel O’Connor, MB, ChB, PhD, MFPM (No slides)
Medicines and Healthcare Products Regulatory Agency (MHRA)

Session 2: How to Measure: Case Based Examples of PRO Strategies
Session Chair: Paul Kluetz, MD

Presentations: Each speaker will build an assessment strategy using existing measurement systems & libraries to assess the core outcomes discussed in Session 1.


Roxanne Jensen, PhD
National Institutes of Health

Aaron Kaat, PhD
Northwestern University

Mogens Groenvold, MD, PhD, DSc
University of Copenhagen and Bispebjerg University Hospital

Panel Discussion

Alicyn Campbell
Genentech, a Member of the Roche Group

Selena Daniels, PharmD, MS (No slides)
U.S. Food and Drug Administration

Session 3: Efforts to Standardize PRO Analysis Methods
Session Chair: Laura Lee Johnson, PhD


SISAQOL – An International Collaboration
Andrew Bottomley, PhD
European Organisation for Research and Treatment of Cancer (EORTC)

A Regulatory Perspective on PRO Research Objectives
Kathy Soltys, MD
Health Canada

Missing Data – Common Issues, Proposed Solutions, and Future Work
Amylou Dueck, PhD
Mayo Clinic

Statistical Methods – Common Challenges and Potential Solutions
Rajeshwari (Raji) Sridhara, PhD
U.S. Food and Drug Administration

Panel Discussion

Beate Wieseler, Dr. rer. nat.
Institute for Quality and Efficiency in Health Care (IQWiG)

Aaron Kaat, PhD (No slides)
Northwestern University

Li Li, PhD (No slides)
Eli Lilly and Company

Session 4: Using PRO Data in Regulatory Review: Current FDA Approaches to Analyze Descriptive Patient Experience Data
Session Chair: Jason Schroeder, PhD


Clarifying Disposition and Completion
Lynn Howie, MD
U.S. Food and Drug Administration

Current FDA Approach
Mallorie Fiero, PhD
U.S. Food and Drug Administration

PRO Data Overview – Presenting the Big Picture
Karon Cook, PhD
Northwestern University

Panel Discussion

Jane Perlmutter, PhD, MBA
Patient Representative

Michael Brundage, MD, MSc, FRCPC
Queen’s University

Josephine Norquist (No slides)

Wrap Up
Paul Kluetz, MD


Valerie Vashio
Center for Drug Evaluation and Research
Office of New Drugs
Office of Hematology and Oncology Products

Janice Kim
Center for Drug Evaluation and Research
Office of New Drugs
Office of Hematology and Oncology Products

Page Last Updated: 09/19/2018
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