Incorporating patient-preference evidence into regulatory decision making

Abstract

Patient preference evidence was used make regulatory decision making more patient-centered. In addition, we captured the heterogeneity of patient preferences allowing market approval of effective devices for risk tolerant patients. CDRH is using the study tool to define minimum clinical effectiveness to evaluate new weight-loss devices. The methods presented can be applied to a wide variety of medical products. This study supports the ongoing development of a guidance document on incorporating patient preferences into medical-device premarket approval decisions.

Incorporating Quantitative Patient Preference Data into Healthcare Decision Making Processes: Is HTA Falling Behind?

“…A 2015 publication detailed how data from a DCE were directly incorporated into a benefitrisk assessment (BRA) for the CDRH [16]. Since this case study, the CDRH, along with another FDA center, the Center for Biologics Evaluation and Research (CBER), have finalized guidance on incorporating patient preferences into BRAs, acknowledging the merits of DCEs [17].…”

The Ball is in Your Court: Agenda for Research to Advance the Science of Patient Preferences in the Regulatory Review of Medical Devices in the United States

“…Published in 2015, the study by Ho et al quantified benefit-risk preferences for weight-loss devices from among a sample of overweight and obese people in the USA using a discrete-choice experiment [4].…”

“…Although some examples of the application of patient preference data to regulatory decisions exist [4,22,29], many questions remain about how to incorporate patient preference data in regulatory decisions and the standards that should be applied to these data. A number of organizations, including, but not limited to, patient groups, such as the National Health Council and FasterCures, industry associations, such as the Pharmaceutical Research and Manufacturers of America and the Biotechnology Innovation Organization, and public-private partnerships, such as the Medical Device Innovation Consortium in the USA and the Innovative Medicines Initiative in Europe, have suggested or are evaluating frameworks for systematically incorporating patient preference information in regulatory benefit-risk assessments.…”

“…Among these approaches, patient preference studies have taken on an increasingly important role in assessing the benefits and risks of medical devices. Patient preference studies can provide a number of transparent and defensible measures of the patient perspective on treatment benefits, risks, and other characteristics, including an identification of what features of medical device treatments matter to patients, quantitative estimates of how much each feature matters, the tradeoffs patients are willing to make among the treatment features, and an estimate of the proportion of patients who would perceive the benefits of a medical technology to outweigh its risks [3,4]. Therefore, patient preference information can inform medical device development strategy, development planning, regulatory submission, and post-approval assessment.…”

Unmet needs: Research helps regulators do their jobs

“…To provide a quantitative framework for incorporating patient preferences, CDRH reviewers developed a proof-of-concept tool based on quantitative measures of the importance of safety, ef cacy, and other attributes of weight-loss devices for obese patients (24). T e study collected preferences of more than 600 obese respondents and used the data to inform regulatory assessment and approval of the EnteroMedics Maestro Rechargeable System for weight loss.…”