Phase 0 clinical trials: point of view in France

摘要

Phase 0 clinical trials are a new concept in the clinical development of drugs, especially for targeted therapies. They bridge the gap between preclinical studies and Phase 1 studies. However, the ethical issues arising from this new step in the development of molecules are very different from those in Phase 1 studies. Several authors have debated these issues but few have focused on the role of patients or tried to include them in this debate. To address this question, we designed a questionnaire that we sent to members of the French Committee for Clinical Research of the League Against Cancer/INCa (Institut National du Cancer). Interestingly, though the majority of respondents agreed with the principle of Phase 0 trials, few believed the burden associated with this kind of clinical trial was acceptable. Thus, before being implemented in France, the profile of patients who should be invited to participate in Phase 0 clinical trials has to be clearly defined. Moreover, patients with no therapeutic alternative should probably not be enrolled unless Phase 0 is designed as a screening tool for personalized treatment programs. Patients and their close relatives should be included in the discussion.