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Published on: August 16, 2017
General and statistical hierarchy of appropriate biologic endpoints
1Cancer Center Statistics, Mayo Clinic, Rochester, Minnesota 55905, USA. Sargent.Daniel@mayo.edu
Oncology (Williston Park, N.Y.)
|June 16, 2006
Summary
Newer cancer therapies require updated clinical trial designs. Progression-free survival (PFS) is now a key endpoint, balancing overall survival (OS) and response rate (RR) for evaluating drug efficacy.
Area of Science:
- Oncology
- Clinical Trial Design
- Pharmacology
Background:
- Novel chemotherapeutic agents present challenges to traditional oncology clinical trial designs.
- The rise of cytostatic biologic agents necessitates re-evaluating primary endpoints for advanced disease trials.
Purpose of the Study:
- To reassess appropriate primary endpoints for phase II and III oncology trials.
- To establish an endpoint hierarchy for evaluating novel chemotherapeutic agents.
Main Methods:
- Review of recent data and clinical trial outcomes.
- Analysis of efficacy metrics for cytostatic and cytotoxic agents.
Main Results:
- Progression-free survival (PFS) is established as a primary endpoint.
- PFS is positioned hierarchically between overall survival (OS) and response rate (RR).
Conclusions:
- The established endpoint hierarchy supports the assessment of novel anticancer agents.
- Progression-free survival (PFS) is a suitable primary endpoint for evaluating clinical efficacy in advanced oncology trials.
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