When deciding whether to approve a medical device, the FDA looks for confirmation that the potential benefits outweigh the potential risks. While simple in concept, questions have swirled around the use of benefit–risk analysis in premarket decision making. To help clarify the thought process that goes into making benefit–risk decisions during the premarket review of certain medical devices, the FDA released final guidance earlier this week, the Association for the Advancement of Medical Instrumentation reports.
“By providing greater clarity about FDA’s decision-making process, we hope to improve the predictability, consistency, and transparency of the review process for applicable devices,” the agency wrote in Factors to Consider When Making Benefit–Risk Determinations in Medical Device Premarket Approval and De Novo Classifications.
According to the FDA, it considers four main factors when assessing a device’s potential benefit:
- The type of benefit
- The magnitude of the benefit
- The probability that a patient will experience the benefit
- How long the benefit can be expected to last
When assessing risk, the FDA evaluates the severity, type, number, and rate of harmful events, the likelihood of a person experiencing a harmful event, how long the adverse consequences last, and the possible effects of a false-positive or false-negative diagnostic result.
In addition, the agency assesses a number of other factors as part of its decision-making process, including patient-centric assessments and patient-reported outcomes, characterization of the disease, the availability of alternative treatments or tests, and risk mitigation strategies.
The FDA also noted that “risk tolerance will vary among patients, and this will affect individual patient decisions as to whether the risks are acceptable in exchange for a probable benefit.” Therefore, the agency said it would take the results of patient preference studies into consideration.
In a companion guidance document released the same day, the agency explained the type of patient preference information (PPI) that it may use for premarket decisions. The main objectives of this guidance, according to the FDA, are to:
- Encourage manufacturers or other stakeholders to submit PPI to help with FDA decision making.
- Describe what constitutes “valid scientific evidence” in relation to patient preference studies.
- Provide recommendations for collecting and submitting PPI to the FDA.
- Discuss the FDA’s inclusion of PPI in its decision summaries and provide recommendations for the inclusion of such information in device labeling.
“By documenting reviewers’ thought processes as part of the administrative record, and in certain cases, the publicly available summary of our decision, sponsors will have a better idea of the basis for FDA’s favorable decisions and gain a greater understanding of what factors were considered as part of an approval or a down-classification decision through the de novo process,” the agency wrote.