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Adaptive phase 2/3 designs for oncology drug development - Time to hedge

Cong Chen1, Eric H Rubin2

  • 1Biostatistics and Research Decision Sciences, Merck & Co., Inc., Rahway, NJ 07065, USA.

Contemporary Clinical Trials
|December 12, 2022
PubMed

Insights

Oncology drug development often skips Phase 2 trials. Adaptive Phase 2/3 designs can prevent costly Phase 3 failures and optimize drug development pathways.

Area of Science:

  • Oncology
  • Clinical Trial Design
  • Drug Development

Background:

  • Randomized-controlled Phase 2 trials are frequently omitted in oncology drug development.
  • The current practice involves proceeding directly to Phase 3 trials after Phase 1 signal detection.
  • This speed-focused approach is becoming unsustainable due to improvements in the standard of care.

Purpose of the Study:

  • To advocate for the adoption of adaptive 2-in-1 Phase 2/3 designs in oncology.
  • To highlight the risks associated with skipping Phase 2 trials and proceeding directly to Phase 3.
  • To propose a more efficient and robust clinical trial strategy.

Main Methods:

  • Discussion of current oncology drug development paradigms.
  • Analysis of the limitations of traditional Phase 1 to Phase 3 progression.
  • Proposal of adaptive Phase 2/3 designs as a state-of-the-art alternative.

Main Results:

  • Skipping Phase 2 trials increases the risk of expensive Phase 3 trial failures.
  • Adaptive designs offer a more flexible and efficient approach to clinical trials.
  • Optimizing study pathways can lead to more successful drug development.

Conclusions:

  • The omission of Phase 2 trials in oncology is a risky and unsustainable practice.
  • Implementing adaptive Phase 2/3 designs can mitigate risks and improve success rates.
  • Adopting advanced trial designs is crucial for modern oncology drug development.
Keywords:
Adaptive designImmunotherapyOncology

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