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Other paradigms: better treatments are identified by better trials: the value of randomized phase II studies

Manish R Sharma1, Michael L Maitland, Mark J Ratain

  • 1Section of Hematology, University of Chicago Medical Center, Chicago, IL, USA. manish.sharma@uchospitals.edu

Cancer Journal (Sudbury, Mass.)
|October 15, 2009
PubMed

Insights

Randomized phase II trials are crucial for selecting effective anticancer drugs and optimizing subsequent studies. This approach minimizes the risk of advancing ineffective treatments to phase III trials in oncology.

Area of Science:

  • Oncology
  • Clinical Trial Design
  • Pharmacology

Background:

  • The increasing number of molecularly targeted anticancer drugs necessitates a reevaluation of phase II trial methodologies.
  • Advancing drugs to phase III trials without robust evidence of efficacy carries a significant risk of failure.

Purpose of the Study:

  • To advocate for the adoption of randomized phase II trial designs in oncology.
  • To demonstrate the benefits of randomized phase II trials in selecting effective agents and optimizing subsequent trial parameters.
  • To highlight the feasibility of randomized phase II trials when employing sound statistical standards.

Main Methods:

  • Reviewing phase II trial designs used for other chronic and progressive diseases.
  • Analyzing the impact of randomized versus single-arm phase II trials in advanced non-small cell lung cancer.
  • Emphasizing the importance of statistical rigor in randomized phase II trial design.

Main Results:

  • Randomized phase II designs are essential for selecting agents and optimizing subsequent phase III trials.
  • Randomized phase II trials have shown positive impacts in oncology, exemplified by advanced non-small cell lung cancer.
  • Single-arm phase II trials have historically led to negative phase III trial outcomes in the same disease.

Conclusions:

  • Randomized phase II trials are indispensable for efficient drug development in oncology.
  • This design enhances the probability of success in phase III trials by providing better agent selection and optimization.
  • Randomized phase II trials are a feasible and statistically sound approach for modern oncology drug development.

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