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The Evolution of Clinical Trials in Metastatic Breast Cancer: Design Features and Endpoints That Matter

Andrew D Seidman1, Julia Maues1, Tiah Tomlin1

  • 1My Style Matters, Inc., Atlanta, GA.

American Society of Clinical Oncology Educational Book. American Society of Clinical Oncology. Annual Meeting
|April 1, 2020
PubMed

Insights

This study reviews how clinical trial endpoints for metastatic breast cancer (MBC) treatments have evolved, focusing on overall survival (OS) and progression-free survival (PFS). It examines physician, regulatory, and patient views to improve trial design for better patient outcomes.

Area of Science:

  • Oncology
  • Clinical Trial Design
  • Regulatory Science

Background:

  • Historically, regulatory approval for metastatic breast cancer (MBC) therapies relied on endpoints like overall survival (OS), time to progression (TTP), or progression-free survival (PFS).
  • The U.S. Food and Drug Administration (FDA) updated its guidance in 2018, acknowledging scientific advancements may introduce new, valid endpoints for oncology product approvals.

Purpose of the Study:

  • To critically analyze the evolution of clinical trial endpoint assessment in MBC therapy.
  • To present diverse stakeholder perspectives (physician, regulatory, patient/advocate) on advancing clinical trials for advanced breast cancer.

Main Methods:

  • Literature review of historical and current FDA guidance on clinical trial endpoints.
  • Synthesis of perspectives from key stakeholders in MBC clinical research.
  • Analysis of the evolution of benefit assessment in clinical trials for systemic therapy in MBC.

Main Results:

  • The assessment of benefit in MBC clinical trials has evolved beyond traditional endpoints like OS and PFS.
  • Recent FDA guidance encourages the exploration of novel endpoints supported by scientific advancements.
  • Understanding stakeholder perspectives is crucial for optimizing clinical trial design, conduct, and reporting.

Conclusions:

  • The evaluation of treatment benefit in metastatic breast cancer clinical trials is dynamic and influenced by scientific progress.
  • A multi-stakeholder approach, incorporating physician, regulatory, and patient insights, is vital for advancing clinical trial methodologies.
  • Optimizing clinical trial design and reporting is essential for improving and extending the lives of women with advanced breast cancer.

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