In new guidance FDA says it may approve devices for use by certain subpopulations only

Clearly, the incorporation of patient preferences into decision-making is important to the FDA.  The Agency codified and formalized the use of patient preference information in a just-released draft guidance on the topic, and if the Maestro approval is any indication, the additional data should help speed up approval times.  After all, the Maestro did not meet its weight-loss endpoint, which usually spells rejection.

For example, the FDA says patient-preference information could someday result in approval among a subset of the relevant population, such as those under a certain age, who may have a higher risk tolerance than their older counterparts.  (Of course, this could also result in narrower approval instead of a new one.)  “It may be appropriate to approve a PMA, approve an HDE application, or grant a de novo request for use of a device by a subset of the population for which an indication is requested when valid scientific evidence shows that the probable benefit of a device outweighs probable risks of the device for that subset. In making such a determination, FDA would consider patient preference information along with the totality of evidence from clinical and nonclinical testing,” the document says.

The FDA recommends the surveys contain quantitative information to identify which outcomes, endpoints or attributes matter most to patients and which factors affect their risk tolerance and perspective on benefit, as well as qualitative information “to provide estimates of how much different outcomes of features matter to patients and the tradeoffs that patients state they are willing to make among them.”  It also listed recommendations of best practices for survey design, such as the use of representative samples, capturing demographic data about the respondents for analysis of results by subpopulation, and design to minimize cognitive bias.

The FDA recommends avoiding fractions and vague terms like “low” or “high” risk.  “If possible, describe uncertainty in both positive and negative frames (e.g., 20% chance of adverse events or 80% chance of no adverse events) to avoid cognitive bias,” the guidance states.

Finally, the FDA encourages uses of patient preference information throughout a product’s life cycle. For example preclinical test methods, such as the study of a “natural clinical trial,” could potentially spare patients from actual clinical trials and speed up the approval process.

The guidance was developed at the behest of the Food and Drug Administration Safety and Innovation Act, which directs the agency to “develop and implement strategies to solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussions.”

It reflects a general desire of patient advocates and the industry itself to include patients’ voices in the device development and commercialization process, for they are less likely to comment on policies like this one than lawyers, company representatives, insurance companies and other institutional stakeholders.

REFERENCE:  Fierce Medical Devices; 14 MAY 2015; Varun Saxena

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