Benefit in phase 1 oncology trials: therapeutic misconception or reasonable treatment option?
- 1 December 2008
- journal article
- Published by SAGE Publications in Clinical Trials
- Vol. 5 (6), 617-623
- https://doi.org/10.1177/1740774508097576
Abstract
Novel treatments for cancer are tested initially in phase 1 trials enrolling patients with advanced disease who have exhausted standard treatment options. Although these trials are designed to evaluate safety and to define dosing for future efficacy trials, most patients volunteer with the hope of obtaining medical benefit. Do phase 1 oncology trials promote a `therapeutic misconception' among eligible patients about the personal meaning of trial participation, or do they offer them a reasonable prospect of direct medical benefit as compared with available alternatives? Recent evidence on outcomes of phase 1 oncology trials is examined systematically, with the aim of accurately assessing the prospect of direct medical benefit for participants and drawing implications for informed consent. We argue that, in view of important uncertainties, aggregate data from phase 1 trials relating to the surrogate outcomes of tumor shrinkage and stable disease do not permit any definitive estimate of a `clinical benefit rate.' Nevertheless, these trials do offer participants a prospect of direct medical benefit. As a result, accurately informed patients may reasonably decide to enroll in phase 1 oncology trials in hopes of obtaining benefit, after considering the anticipated risks and available clinical alternatives. Motivation to enroll in these studies to receive personal benefit does not, in itself, compromise informed consent.Keywords
This publication has 27 references indexed in Scilit:
- Clinical outcome and prognostic factors for patients treated within the context of a phase I study: the Royal Marsden Hospital experienceBritish Journal of Cancer, 2008
- Further evidence of clinical benefit associated with participation in phase I oncology trialsBritish Journal of Cancer, 2008
- Ethical, Scientific, and Regulatory Perspectives Regarding the Use of Placebos in Cancer Clinical TrialsJournal of Clinical Oncology, 2008
- Treatment outcome and survival in participants of phase I oncology trials carried out from 2003 to 2006 at Institut Gustave RoussyAnnals Of Oncology, 2007
- Risks and benefits associated with novel phase 1 oncology trial designsCancer, 2007
- Rethinking Risk-Benefit Assessment for Phase I Cancer TrialsJournal of Clinical Oncology, 2006
- Risks and Benefits of Phase 1 Oncology Trials, 1991 through 2002New England Journal of Medicine, 2005
- Risks and Benefits of Phase 1 Oncology Trials, RevisitedNew England Journal of Medicine, 2005
- Ethics of Phase 1 Oncology StudiesJAMA, 2003
- What Makes Clinical Research Ethical?JAMA, 2000