What patients have to say may influence how the FDA reviews new medical technologies, and how IDNs acquire them
Clinical evidence and documented patient outcomes may represent the Holy Grail for developers – and buyers – of new medical technologies. But in an age of patient-centered medicine, is it any wonder the patient’s voice is getting louder in the development and acceptance of new technologies?
Since 2012, the U.S. Food and Drug Administration has urged manufacturers and developers of medical technology to factor in patients’ perceptions and tolerance of the risks and benefits of new technology. Earlier this year, the agency issued proposed guidelines to help manufacturers collect and measure patients’ perceptions in a meaningful, usable way.
The guidance document (“Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications”), which was issued in May 2015, makes it clear that submission of patient preference information is voluntary on the part of medical device manufacturers and developers. That said, the agency clearly believes considering patient perception is critical in the pre-market (and post market) phase of the product lifecycle.
Assessing patient preferences
“Talking about assessing patient preferences isn’t necessarily new,” says Stephanie Christopher, program manager, patient centered benefit-risk assessment and clinical trials innovation and reform, Medical Device Innovation Consortium, St. Louis Park, Minn. “But using [patient preferences] in a regulatory context is fairly new.”
MDIC is a non-profit public-private partnership that operates in partnership with the FDA, medical device industry and non-profits to improve the medical technology regulatory environment. With a steering committee of member organization and FDA representatives, patient advocates, and other experts, MDIC published its “Framework for Patient-Centered Benefit-Risk Assessment” around the same time that the FDA released its guidance document.
For years, manufacturers – through their marketing departments – have solicited comments and preferences from physicians and other users about their products, such as the ease of use, Christopher points out. What’s new is trying to determine how patient perspectives can be gathered, analyzed and quantified, and thus offer scientifically valid information for regulatory reviewers, she says.
Risk/benefit
The FDA won’t approve a device that isn’t safe or doesn’t work, regardless of patient perspectives, says Christopher. But patient-preference information gives “additional information on risks that patients might be willing to tolerate in exchange for benefits,” she says. “Prior to this move to include patient preference, the benefit-risk assessment was done from the perspective of physicians, clinicians and reviewers. The FDA guidance is saying, ‘The safety and efficacy doesn’t change; but when we’re doing benefit-risk assessments, we want to understand the patient’s perspective on possible benefits and risks of the devices we review.’”
Such input is valuable. “Patients understand the reality of living with a disease,” says Christopher. “They understand their symptoms and how they impact their life, and they understand what it would mean to have some alleviation of symptoms or a disease state, and what risk they might be willing to tolerate for that.”
The MDIC Framework points to an FDA study in the area of obesity, where the FDA’s Center for Devices and Radiological Health had undertaken a patient preference study with obese patients who were asked about several different attributes of potential weight-loss medical devices. The patient preference study led to the development of a decision aid tool available to reviewers who were reviewing new obesity medical devices. The January 2015 marketing clearance for the Maestro Rechargeable System (EnteroMedics, St. Paul, Minn.) for certain obese adults, which targets the nerve pathway between the brain and the stomach that controls feelings of hunger and fullness, was based in part on patient preference information from the CDRH study.
Future
“The field of patient preference is still relatively nascent,” says Christopher. “One area for future research – which we talk about in our Framework document – is looking at what patients would prefer before they have experience with a particular technology, and seeing if those preferences are consistent with choices made by patients who have experience with that technology.
“We want to engage patients. But this is also an access issue. We are showing that there are patients who want access to new technologies, and who understand the risks and benefits. We’ll see where it is applied.”
The FDA guidance document, “Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications” can be accessed at http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM446680.pdf?source=govdelivery&utm_medium=email&utm_source=govdelive
The MDIC “Framework for Patient-Centered Benefit-Risk Assessment” can be accessed at http://mdic.org/pcbr/?source=govdelivery&utm_medium=email&utm_source=govdelivery
When patient input has less value
Analyzing patients’ perceptions of medical devices in the pre- and post-development phases of the product lifecycle isn’t always a useful exercise, points out the Medical Device Innovation Consortium in its “Framework for Patient-Centered Benefit-Risk Assessment.” Some examples:
- When the patient is not a major decision-maker or stakeholder. An example might be surgical tools, because the surgeon is the one who determines how the device is to be used.
- When the disease state, technology, study design, and clinical inputs are generally understood by both sponsors and FDA staff, and there is significant regulatory precedent for approval.
- When the benefits of the technology are so high and the risks so low that approval is highly likely.
- When the treatment is clearly superior to existing therapies such that there exist no benefit-risk tradeoffs compared to alternative therapies.
- When the treatment addresses an unmet medical need with poor outcomes such that the risks of the treatment will not be greater than the risks of the untreated disease.