Patient empowerment is manifesting itself in many forms across the healthcare landscape, and the influence of patient preferences on medtech approvals is growing.
As a primary stakeholder – the end user – for medical devices, the patient is playing an increasingly significant role in FDA approvals. Over the past several years, the FDA has clarified its position on utilizing patient inputs in its decision-making processes, and it has demonstrated that it’s generally open to both qualitative and quantitative patient input. The agency began formalizing its process for gathering patient input in 2012 and since then has held public workshops and established the Patient Engagement Advisory Committee to help represent patients’ viewpoints and facilitate collaboration. The FDA has taken an encouraging stance and has recognized that the comparisons—and even the definitions—of risks and benefits may be quite different when viewed through the lens of patients and their caregivers rather than healthcare professionals and other stakeholders.
In fact, the FDA recently published a guidance document with a mouthful of a title on this topic: “Patient Preference Information – Voluntary Submission, Review in Premarket Approval Applications, Humanitarian Device Exemption Applications, and De Novo Requests, and Inclusion in Decision Summaries and Device Labeling: Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders.” The agency is quick to mention that this guidance is non-binding and not legally enforceable. It’s meant to share the agency’s thinking on the topic.
What the FDA is trying to accomplish with this document is to encourage medtech companies to consider the ways in which they can successfully utilize patient input to design their products, influence clinical trial set-ups, and monitor patients’ ongoing usage habits and preferences over time. And this guidance document focuses on a particular type of input: patient preference information (PPI), which is most useful when multiple treatment options exist without a clearly dominant option, or when there are multiple segments of patients with heterogeneous views of risks and benefits, especially if those views may differ from those of healthcare professionals.
Medtech manufacturers aren’t required to submit PPI during the market approval processes, although they are encouraged to do so to help provide context about the patients’ points of view. How might the PPI be taken into account? The document provides a few examples:
- An implanted knee device that shows a small overall benefit to the broad population (of those studied) but that shows greater benefit among those with the highest pain and functional limitation might receive FDA approval for just that highest-acuity segment. The FDA might require a post-approval study.
- An implantable wrinkle-removing device that only has temporary aesthetic effects might not have received FDA approval due to its high-risk profile, although if PPI indicates that a significant patient population prefers even the temporary aesthetic effects over its risks, then the product might, in fact, be approved, and the indication label may reflect appropriate language about those temporary effects.
- An aesthetic device that reduces patient recovery time relative to currently marketed products but also has a somewhat higher adverse event rate due to durability issues may give the FDA pause. If strong PPI indicates patients placing a high value on the aesthetic outcome relative to the complication risks, then the FDA may consider that. The agency may not approve the device, but may instead suggest that the manufacturer modify the design to improve the device’s durability and resubmit the application.
- A pediatric device that scales down a prosthesis to fit children’s bodies and shows a clinical benefit but induces some surgical risk may leave the agency with a difficult benefit/risk trade-off. However, if the manufacturer conducts a PPI study that shows strong support for the benefits over the risks from parents, the agency may be swayed to approve the device. The FDA might require special labeling to ensure that parents understand the risks and can make an informed decision.
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