Presentation (CTI)

OBJECTIVES: Double-blind placebo-controlled clinical trials have been a “gold standard” design to determine study drug efficacy, especially when no alternative treatment is available. However, patients entering clinical trials may prefer active treatment and view the chance of being randomized to placebo negatively, potentially contributing to slowed recruitment and eventual attrition. A targeted literature review was conducted to characterize the patient perspective of placebo use in clinical trials with a goal of providing drug developers with patient-centered suggestions for recruiting and retaining participants in placebo-controlled trials.
METHODS: Embase, Medline, and Google Scholar were searched from database inception to November 2024 for English-language qualitative studies (interviews, focus groups, or surveys) or literature reviews describing patient perspectives, views, and attitudes of the use of placebos in placebo-controlled trials. Abstracts were excluded if they reported on open-label placebo use in clinical trials or placebo use in clinical practice.
RESULTS: Of the 49 abstracts identified, 15 qualitative research studies and 5 literature reviews met inclusion criteria and were included for full-text examination. The literature review highlighted the limited qualitative research of patient perceptions of placebo use in clinical trials; existing research presents varied views of placebo use related to the prevalence of the disease or condition, severity of symptoms, and overall understanding of placebos.
Recommendations for drug developers include recruitment materials with layperson definitions of placebo, presence of a standard of care treatment arm when available, access to the study drug post-trial, qualitative interviews to understand the study experience, and a debriefing on randomization arm at end of trial.
CONCLUSIONS: The patient view of the use of placebos in clinical trials is varied and complex. Drug developers would benefit from an appreciation of patients’ views of placebos within the context of use and incorporation of patient-centered approaches to attenuate participation barriers related to placebo randomization.