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How to develop treatments for biologically heterogeneous "diseases"

Richard M Simon1

  • 1Biometric Research Branch, Division of Cancer Treatment & Diagnosis, National Cancer Institute, Bethesda, Maryland, USA. rsimon@nih.gov

Clinical Cancer Research : an Official Journal of the American Association for Cancer Research
|June 9, 2012
PubMed

Insights

Standard phase III clinical trial designs fail for targeted cancer therapies in diverse patient groups. A new framework using tumor genomics can improve the design of future phase III trials for molecularly targeted treatments.

Area of Science:

  • Oncology
  • Clinical Trial Design
  • Genomics

Background:

  • The current phase III clinical trial paradigm is inadequate for evaluating molecularly targeted treatments.
  • Patient populations in oncology are often biologically heterogeneous, complicating standard trial designs.

Purpose of the Study:

  • To address the limitations of standard phase III trial designs for molecularly targeted therapies.
  • To propose an alternative framework for phase III trials that incorporates tumor genomics.

Main Methods:

  • Review of existing phase III clinical trial designs.
  • Proposal of a prospective discovery/evaluation framework.

Main Results:

  • The standard phase III design is unsuitable for heterogeneous patient groups receiving targeted treatments.
  • A novel framework is proposed to integrate tumor genomics into phase III trial design.

Conclusions:

  • Alternative clinical trial designs are necessary for molecularly targeted treatments.
  • Integrating tumor genomics into a prospective framework can optimize phase III trial design for precision oncology.

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