Clinical Trials to Optimize Outcomes in Early Breast Cancer: FDA Public Workshop October 29, 2018

Workshop | In Person

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• Date: October 29, 2018
• Time: 4:30 a.m. - 12:15 p.m. ET

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Organized by the:
U.S. Food & Drug Administration (FDA) Oncology Center of Excellence (OCE)
in collaboration with
the UCSF-Stanford Centers of Excellence in Regulatory Science and Innovation (CERSI)

•  #OptimizeEarlyBC

Co-Chairs:

• Tatiana Prowell, MD
• Suparna Wedam, MD

Date: October 29, 2018

Time: 7:45 am to 4:15 pm

Location:

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

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

Background:

In 2018, many patients with early breast cancer will be cured of their disease.  While advances in genomic testing have enabled us to identify some patients with favorable prognoses who may be spared the toxicity of unnecessary treatment, it is likely that we continue to overtreat many patients, and we remain unable to identify patients at high risk of serious long-term toxicities of adjuvant therapy. De-escalation trials are therefore appropriate to change practice, but are often challenging to design and conduct. Simultaneously, there remains a high level of unmet need in patients who lack targeted therapy options, such as those with triple-negative breast cancer, and those whose disease is hormone receptor or HER2 positive but resistant to therapy. Some of these patients may be identified by the nature or extent of residual disease following neoadjuvant systemic therapy and others identified only by late relapses following adjuvant therapy. Targeted escalation trials conducted in the neoadjuvant setting provide the opportunity to identify patients at increased risk of breast cancer recurrence and test novel agents to improve clinical outcomes. The focus of this workshop is to gather stakeholders interested in early breast cancer to discuss strategies for successful escalation and de-escalation of therapy to optimize patient outcomes.

Workshop Objectives:

• Provide a forum for open discussion between all stakeholders on strategies to optimize outcomes in early breast cancer
• Discuss trials of de-escalation and targeted escalation of systemic therapy in early breast cancer
• Discuss assessment of response in neoadjuvant trials in early breast cancer with a focus on endpoints that may support regulatory approval
• Discuss feasibility challenges of conducting modern early breast cancer clinical trials and the need for robust evidence generation to satisfy regulatory/payer requirements

Webcast Information:

FDA will provide a free-of-charge, live webcast of this workshop.

• Overview and Session 1: https://collaboration.fda.gov/p88p3r6sgr6/           
• Session 2: https://collaboration.fda.gov/p3i73eq26em/
• Session 3: https://collaboration.fda.gov/p1jawodz0zj/
• Session 4: https://collaboration.fda.gov/p4b9hoszk1v/

Meeting Information:

• Agenda 

Speakers

Opening Remarks

• History and Rationale for Escalation and De-escalation of Therapy in Early Breast Cancer
  Eric Winer, MD
  Dana-Farber Cancer Institute

SESSION 1: De-escalation of Therapy in Early Breast Cancer
Moderator: Suparna Wedam, MD (U.S. Food and Drug Administration)

• Use of the Neoadjuvant Setting to De-Escalate Therapy
  Douglas Yee, MD
  University of Minnesota
• De-Escalation of Therapy in Early Breast Cancer: Lessons for our Field from the TAILORx and MINDACT Experience
  Joseph Sparano, MD
  Albert Einstein College of Medicine
• Toxicity as a Rationale for De-Escalation of Therapy in Early Breast Cancer: Cardio-Oncology as an Example
  Laleh Amiri-Kordestani, MD
  U.S. Food and Drug Administration, Center for Drug Evaluation and Research, Office of New Drugs
  Office of Hematology and Oncology Products

SESSION 2: Targeted Escalation of Therapy in Early Breast Cancer
Moderator: Julia Beaver, MD (U.S. Food and Drug Administration)

• Escalation of Therapy in Patients at Risk of Late Recurrence: Lessons Learned from SOFT/TEXT
  Gini Fleming, MD
  University of Chicago Medicine
• Trial Designs and Statistical Models for Compound Endpoints (Efficacy and Toxicity) in Early Breast Cancer
  Donald Berry, Ph.D
  MD Anderson Cancer Center
• Second Line Therapy in the Neoadjuvant Setting: Rationale and Pragmatic Considerations for Trial Design (Presentation not available)
  Angela De'Michele, MD, MSCE
  University of Pennsylvania
• Addressing Unmet Need in an Era of Diminishing Returns: HER2+ Early Breast Cancer as an Example
  Antonio Wolff, MD
  Johns Hopkins University
• The Inflection Point in Early Breast Cancer: Refining Drug Development to Optimize Individual Outcomes
  Laura Esserman, MD, MBA
  University of California, San Francisco

SESSION 3: Defining Success in Early Breast Cancer: Potential of Pathological Endpoints to Support Regulatory Approval
Moderator: Tatiana Prowell, MD (U.S. Food and Drug Administration)

• Regulatory Perspective on Use of Pathological Complete Response for Accelerated Approval in 2018
  Jennifer Gao, MD
  U.S. Food and Drug Administration, Center for Drug Evaluation and Research, Office of New Drugs
  Office of Hematology and Oncology Products
• European Perspective on Use of Neoadjuvant Setting for Breast Cancer Drug Approval
  Jonas Bergh, MD, PhD., FRCP (London, UK)
  Karolinska Institutet
• Refined Pathological Endpoints for Assessment of Response to Neoadjuvant Therapy
  W. Fraser Symmans, MD
  MD Anderson Cancer Center
• Assessment of Response to Neoadjuvant Therapy in ER+ Breast Cancer
  Ingrid Mayer, MD, MSCI
  Vanderbilt University

SESSION 4: The Need for Robust Evidence Generation & Feasibility Challenges
Moderator: Hope Rugo, MD (University of California, San Francisco)

• Who Will Lead the Effort to De-Escalate Therapy in Early Breast Cancer?
  Larissa Korde, MD, MPH
  National Cancer Institute (NCI)/Cancer Therapy Evaluation Program (CTEP)
• Need for Evidence Generation in Early Breast Cancer: CMS Perspective (Presentation not available)
  Centers for Medicare and Medicaid Services, Coverage Analysis Group
• Payer Role in Advancing Precision Medicine (Presentation not available)
  Marcus Thygeson, MD, MPH
  Bind Health

Workshop Summary

• Tatiana Prowell, MD
  U.S. Food and Drug Administration, Center for Drug Evaluation and Research, Office of New Drugs
  Office of Hematology and Oncology Products
• Suparna Wedam, MD (FDA)
  U.S. Food and Drug Administration, Center for Drug Evaluation and Research, Office of New Drugs
  Office of Hematology and Oncology Products
   

Contact:

Sherwin Sapasap, MS, MBA, MHA
Center for Drug Evaluation and Research
Office of New Drugs
Office of Hematology and Oncology Products
Sherwin.Sapasap@fda.hhs.gov