Giving Patients a Voice in Regulatory Decisions

Center for Devices and Radiological Health (CDRH), U.S. Food and Drug Administration
A doctor conducts an in-person survey of a patient
As leaders in patient preference research, RTI Health Solutions (RTI-HS) experts made significant contributions this year to expanding the role of the patient perspective in regulatory decisions. Our efforts, in collaboration with the Center for Devices and Radiological Health (CDRH) of the U.S. Food and Drug Administration (FDA) have resulted in the first-ever approval decision by the FDA to consider patient preferences. We also helped develop a framework for how sponsors and regulators might incorporate the patient perspective on benefit and risk in future decision-making regarding medical devices.

First FDA Approval to Consider Evidence on Patient Preferences

In January 2015, for the first time in its history, the FDA considered evidence on patient preferences in its decision to approve a new device to treat obesity, drawing on study data developed by a team of RTI-HS and FDA researchers.

Published in Surgical Endoscopy, the study quantified patients’ preferences regarding health benefits and risks of various obesity treatment devices. The study estimated the maximum mortality risk patients were willing to accept for a certain amount of weight loss and the minimum amount of weight loss sufficient to undergo the risks of a weight loss device.

Researchers surveyed 540 clinically obese adults, gathering scientific data on their preferences to inform clinical trial design for obesity treatment devices. Study data were determined to be sufficient to inform FDA’s regulatory decision-making.

“This obesity preference study allowed us to systematically quantify the tradeoffs patients are willing to make among benefits, harms, and other features of weight-loss devices,” said Juan Marcos Gonzalez, PhD, senior research economist at RTI-HS.

While it is not possible to say how much weight the CDRH gave to the available evidence on patient preferences in this approval, the announcement clarified that this was an example of a decision that benefited from having this evidence at hand.

As the first device approved to treat obesity since 2007, the new device—the Maestro Rechargeable System—offers a new clinical option for patients who have not responded to other treatments.

Recognizing the Importance of Patient Input on Regulatory Decisions

Prior to the obesity preference study, the FDA did not have the scientific data to quantify trade-offs patients were willing to make among the benefits and risks of a weight-loss device. By demonstrating a method for capturing patient sentiment and translating it into a decision-making tool, RTI-HS researchers contributed to an important precedent in regulatory approvals of medical devices.

As noted by the study authors, “Patients have a unique role in deciding what treatments should be available for them, and regulatory agencies should take their preferences into account when making treatment approval decisions.” 

Developing a Framework for the Future

Later in the year RTI-HS joined an effort led by the Medical Device Innovation Consortium (MDIC) to build on the precedent set by this landmark approval decision.

MDIC brought together representatives from government, medical device companies, and patient-centered nonprofits to develop a framework to guide sponsors and regulators in incorporating evidence on patient perspectives about benefits and risks of medical interventions.

Brett Hauber, PhD, an RTI-HS expert in health preference assessment and senior economist, led an interdisciplinary team in creating a catalog of research methods for how to collect and analyze patient preference information—a key component of the framework.

Released in May 2015, this framework addresses the value of including patient benefit-risk considerations in a regulatory submission, discusses when in the product lifecycle such information might be collected, and outlines factors to consider when selecting a research method.