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Clinical trial design for target-based therapy
Elizabeth Fox1, Gregory A Curt, Frank M Balis
1Center for Cancer Research, National Cancer Institute, Bethesda, Maryland, USA. foxb@mail.nih.gov
The Oncologist
|October 29, 2002
Summary
Anticancer drug discovery now uses targeted approaches based on molecular understanding. Clinical trials for these novel drugs require new endpoints beyond traditional toxicity and response measures.
Area of Science:
- Oncology
- Pharmacology
- Molecular Biology
Background:
- Anticancer drug discovery has evolved from random screening to a rational, target-based approach.
- Advances in understanding cancer pathogenesis at the molecular level provide new drug targets.
Purpose of the Study:
- To discuss the necessary changes in clinical trial design for target-based anticancer drugs.
- To highlight the limitations of traditional endpoints for novel therapeutic agents.
Main Methods:
- Review of traditional and proposed clinical trial endpoints for anticancer drugs.
- Discussion of the shift towards mechanistic and molecularly targeted therapies.
Main Results:
- Traditional endpoints like toxicity and response may be insufficient for cytostatic, target-based agents.
- Biological or pharmacokinetic endpoints are proposed for early-phase trials (Phase I and II).
Conclusions:
- Phase III trials will still prioritize measurable clinical benefit.
- Adapting clinical trial designs is crucial for evaluating the utility of new targeted anticancer drugs.