A study published in Cancer found that many patients (>80%) who enroll in early-stage clinical trials (phase I) are motivated by the potential clinical benefit, with approximately half expecting tumor shrinkage and approximately one-tenth anticipating a cure. However, more realistic response rates in phase I trials range from 4 to 20 percent, which "exemplifies the challenges faced by patients and health-care professionals during their interactions [with] phase I studies," the authors wrote.
Saoirse O. Dolly MBBS, PhD, from the Department of Medicine at the Royal Marsden National Health Service Foundation Trust in London, and authors conducted this single-center, prospective, quantitative study of newly referred adult patients considering their first participation in a phase I clinical trials. Patients completed questionnaires about the trial prior to (99%, n=396) and after (76%, n=301) their doctor visits.
When patients were asked about the potential personal benefit prior to the consultation, 43 percent predicted tumor shrinkage initially, while after the consultation this increased to 47 percent, with 14 percent of patients expecting a cure. The authors noted that this percentage is much higher than what is realistic for early stage studies and for conditions that have a median survival of six months.
Sources: Dolly SO, Kalaitzaki E, Puglisi M, et al. A study of motivations and expectations of patients seen in phase 1 oncology clinics. Cancer. 2016 September 26. [Epub ahead of print]; Reuters, September 26, 2016.