Using a multistakeholder collaboratory and patient surveys to inform the conduct of personalized (N-of-1) trials.

OBJECTIVE

Personalized trials have the potential to improve the precision of treatment selection and foster patient involvement in clinical decision making. Little is known about the attitudes of patients with multimorbidities. To address this, stakeholders designed and conducted a national survey that determined general attitudes and features of personalized trials that may increase their use among patients with multimorbidities in clinical and research practice.

METHOD

A multistakeholder collaboratory of patients, clinicians, scientists, methodologists, statisticians, and research disseminators designed a survey to determine the conditions, symptoms, and design attributes most applicable to personalized trials according to patients. A sample of U.S. patients with two or more prespecified personalized-trial-amenable chronic conditions completed the online survey.

RESULTS

Multimorbid participants (N = 501; M age = 56.1 years) showed that some conditions, symptoms or use cases for personalized trials include pain (57.6%), hypertension (38.8%), diabetes (28.8%), sleep problems (27.4%), and depression (23.0%). Overall, 82.0% of the participants with multimorbidities were interested in participating in personalized trials. The percentage that were interested varied by trial attributes, including physician involvement (86.4%), patient-driven treatment selection (88.0%), clinician blinding (59.2%), placebo treatment options (57.5%), and out-of-pocket costs (41.8%).

CONCLUSION

Participants with multimorbidities identified prevalent use cases that are suited to personalized trials. Participants also identified design features of such trials, including patient-driven treatment selection, active comparators, and nonblinding. This study demonstrates that eliciting input from a collaboratory and patients with multimorbidities can inform research priorities for this rapidly growing patient population and increase adoption by researchers and clinicians alike. (PsycInfo Database Record (c) 2021 APA, all rights reserved).