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Phase III trials of targeted anticancer therapies: redesigning the concept

Alberto Ocana1, Eitan Amir, Francisco Vera-Badillo

  • 1Authors' Affiliation: Division of Medical Oncology and Hematology, Princess Margaret Cancer Centre and the University of Toronto, Toronto, Canada.

Clinical Cancer Research : an Official Journal of the American Association for Cancer Research
|July 25, 2013
PubMed

Insights

Phase III trials are crucial for drug approval but face challenges like limited benefits and high costs. Adaptive designs in prescreened populations may offer solutions for more efficient clinical trials.

Area of Science:

  • Clinical pharmacology
  • Drug development
  • Biostatistics

Background:

  • Randomized phase III trials are the gold standard for new drug approval.
  • Current phase III trials face limitations including small observed clinical benefits, large sample size requirements, and high costs.
  • Challenges exist in designing trials for drug combinations and biomarker-driven studies.

Purpose of the Study:

  • To review the limitations of current phase III trial designs in drug development.
  • To propose adaptive designs in selected populations as a potential solution.

Main Methods:

  • Review of existing literature and common challenges in phase III trial design.
  • Discussion of the potential benefits of adaptive designs and prescreening.

Main Results:

  • Phase III trials often show limited clinical benefit for new drugs.
  • Identifying rare toxicities and evaluating drug combinations/biomarkers pose design challenges.
  • Adaptive designs in prescreened populations are suggested to mitigate limitations.

Conclusions:

  • Current phase III trial designs have inherent limitations impacting drug development efficiency and cost-effectiveness.
  • Adaptive phase III trial designs in selected, prescreened populations offer a promising approach to address these challenges and improve drug evaluation.

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