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Benefit in phase 1 oncology trials: therapeutic misconception or reasonable treatment option?

Franklin G Miller1, Steven Joffe

  • 1Department of Bioethics, Clinical Center, National Institutes of Health, Bethesda, MD 20892-1156, USA. fmiller@nih.gov

Clinical Trials (London, England)
|November 26, 2008
PubMed

Insights

Phase 1 cancer trials test new treatments for safety and dosage. While not guaranteeing a cure, these trials offer patients a reasonable chance of medical benefit, supporting informed consent for participation.

Area of Science:

  • Oncology
  • Clinical Trials
  • Medical Ethics

Background:

  • Phase 1 oncology trials evaluate novel cancer treatments in patients with advanced disease who have exhausted standard options.
  • While designed for safety and dosing, patients often enroll hoping for personal medical benefit.
  • The 'therapeutic misconception' questions whether patients understand the primary research goals versus personal treatment expectations.

Purpose of the Study:

  • To systematically examine evidence on phase 1 oncology trial outcomes.
  • To accurately assess the prospect of direct medical benefit for participants.
  • To draw implications for the process of informed consent in these trials.

Main Methods:

  • Systematic review of recent evidence on outcomes from phase 1 oncology trials.
  • Analysis of surrogate outcomes such as tumor shrinkage and stable disease.
  • Evaluation of the prospect of direct medical benefit in comparison to available alternatives.

Main Results:

  • Aggregate data on surrogate outcomes do not allow a definitive 'clinical benefit rate' estimation.
  • Phase 1 trials do offer participants a prospect of direct medical benefit.
  • Patient motivation for personal benefit does not inherently compromise informed consent.

Conclusions:

  • Accurately informed patients can reasonably choose to enroll in phase 1 oncology trials for potential benefit.
  • Informed consent requires careful consideration of risks, benefits, and alternatives.
  • Understanding the balance between research objectives and personal benefit is crucial for ethical trial participation.

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