PIH70 FORMATIVE RESEARCH TO DEVELOP A CLINICAL TRIAL DECISION AID THAT INTEGRATES QUANTITATIVE PATIENT PREFERENCE DATA

Abstract

Stated-preference methods have been used in patient-focused drug development to inform the drug development process. Few studies have utilized preference methods to facilitate individual-level decision making. This study employed formative research in the design of a decision aid that integrates a quantitative preference method to support deliberation and values clarification. Best Worst Scaling (BWS) and threshold technique were potential preference approaches. The decision aid is customizable to any pediatric clinical trial. We conducted 20 in-depth cognitive interviews with parents of children with rare disease (n=11) and professionals who educate/consent for trials (n=9). Researchers employed a ‘think aloud’ approach and used targeted prompts to obtain feedback on decision aid approach, educational content, preference elicitation activity, and output. In addition, an Advisory Committee (comprising parents and professionals) provided input during all stages of tool development, including choice of preference methodology (threshold technique for risk/side-effect acceptance and/or BWS for protocol-related burdens and trial design). Advisors did not support the face validity of employing the threshold technique in the tool. In cognitive interviews an alternative direct estimation approach was approved as meaningful to clarify risk acceptance. Participants endorsed content as patient-friendly, important, and easily customizable. The BWS prioritization and associated output required additional instruction but were thereafter found to support education and values clarification by most participants. Participants anticipated the tool would facilitate shared decision making. Formative research resulted in a customizable decision aid comprising educational content, direct estimation of risk acceptability, BWS prioritization of protocol and burden attributes, and a customized output. The decision aid and integrated preference activity were endorsed as supporting deliberation and values clarification. Future research will assess the utility of the decision aid in improving informed choice.