FDA is showing that it takes patient input and preferences seriously, by giving the medical device industry new draft guidance on the role of patient preference information in the review process and the product development process. The draft guidance, "Patient Preference Information—Submission, Review in PMAs, HDE Applications, and De Novo Requests, and Inclusion in Device Labeling," outlines best practices for patient preference studies and data collection, and how best to incorporate the information in labeling.
This isn't the first time FDA has shown interest in patient preference information. In 2012 FDA published guidance on the patient perspectives that may be considered by agency reviewers, including patient tolerance for risk and perspective on benefit. This new draft guidance sticks to considering patient risk tolerance and benefit perspective, and emphasizes that submitting such information is voluntary for device sponsors. FDA believes such information could be useful because it helps reviewers understand how patients prioritize certain risks and benefits and how widespread these views are among the patient group. In addition, the guidance states that devices may be better suited than drugs for patient preference information "because the mechanism of action for devices is often well-characterized and fairly localized," unlike drugs.
FDA notes that inclusion of patient preference information may be particularly helpful in "preference-sensitive" diseases, where the device under review would compete with other possible therapies and there is no clear best treatment.
The draft guidance points out that patient preference information differs from patient-reported outcomes by showing "how much the patient values one outcome when facing a trade-off with other potential therapies." Patient preferences may be quantified using a "stated preference" method that gives patients various choices, or a "revealed-preference" method that determines patient preference using the patient's real treatment decisions.
FDA recommends that sponsors consider the following criteria when designing patient preference studies:
- Representativeness of the sample and generalizability of results
- Capturing heterogeneity of patients’ preferences
- Established good research practices by recognized professional organizations
- Patient centeredness
- Effective communication of benefit, harm, uncertainty, and risk
- Minimal cognitive bias
- Logical soundness
- Robustness of analysis of results
- Study conduct
- Comprehension by study participants
The draft guidance states that FDA may use patient preference information to set conditions of approval in situations where use is warranted only in a subset of the patient population. FDA also delves further into how the patient preference information should be conveyed to physicians and patients on device labeling, and gives hypothetical examples of how FDA may consider patient preference information.
Earlier this year, FDA, which has a Patient Preference Initiative, highlighted a patient preference study agency scientists conducted on weight loss devices. EnteroMedics' Maestro Rechargeable System was approved soon after that study's publication, the first obesity device to receive FDA clearance since 2007. Additionally, the Medical Device Innovation Consortium, a partnership between FDA, NIH, CMS, and industry, is trying to improve the approach toward "Patient Centered Benefit-Risk Assessment."
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