A new report from the Medical Device Innovation Consortium (MDIC) sets out a preliminary framework for industry and FDA to consider when factoring in patient risk-benefit preferences.

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The document aims to supplement FDA's 2012 guidance on "Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications,” according to MDIC CEO Bill Murray. (See Also see "FDA’s Final Risk-Benefit Guidance Excludes 510(k)s, Adds De Novo Petitions" - Medtech Insight, 2 April, 2012..)

The guidance outlines key factors CDRH considers when evaluating risk-benefits of PMA and de novo applications including patient perspectives. It recommended sponsors talk to FDA early and often about using patient preference information, but it "did not discuss in what situations or how sponsors should collect and present the information," Murray said in an interview with "The Gray Sheet."

The MDIC report, released May 13, attempts to address those issues, outlining in particular how to incorporate patient preferences into device applications. The group also developed a "catalog of methods” that researchers and regulators can use to collect and analyze patient preference information. It was released the same day as an FDA draft guidance on inclusion of patient preference information in certain device submissions and product labeling.

Medical Device Innovation Consortium

MDIC is a public-private partnership that includes participation from FDA and other government agencies, industry, patient advocates and standards organizations to advance medical device regulatory science. Its latest report aligns with FDA's expanded focus on formally incorporating patient input on acceptable risk in exchange for specific benefits as a formal factor in regulatory decisions.

"The report does not suggest a specific finding or recipe for sponsors or FDA," cautioned Murray in an interview with "The Gray Sheet." "New discoveries occur throughout the product lifecycle, and so patient preferences can be and should be considered throughout that process."

A 'Living Document'

MDIC says the report is meant to be a "living document" that will be periodically updated by the organization as more experience is gained in using patient preferences to support product approvals, reimbursement and shared-decision making in general.

But the group says that certain metrics such as validated quality of life measures can be useful for physicians when they talk to patients about treatments and also could signify increased benefit to patients when devices are being considered for approval.

"These types of metrics allow the physician to better quantify the impact of the device on the patient’s well-being and help the patient make a more informed decision," the report states. "Moreover, it may be appropriate to approve a device where only a minority of the intended patient population would accept the risks as weighed against the benefits if the information necessary for patients and health care practitioners to make well-informed decisions is available and can be presented in a manner that can be understood by the practitioners and patients."

The report also states patient-centric assessments should take into account and balance a patient's willingness versus reluctance to use the device. When considering preference-sensitive decisions and situations where a patient's feedback could be used to approve a device, authors of the report say three factors should be considered including the patient's perspective as a stakeholder, risk-benefit tradeoffs inherent to the device or technology and the novelty of the device to regulators.

"While these three groups of factors may not capture all possible considerations about whether or not to collect patient preference information in a specific situation, they do represent a core set of factors that can be considered," the report adds.

"We didn't fully appreciate how little research has been done in this area; first in the regulatory area ... but also patient preferences is a whole untapped area for reimbursement," MDIC President Bill Murray said.

A Whole New World

Murray says that while MDIC knew patient preference was a very new area of regulatory science research, he was surprised by just how little literature was available on the topic.

"We didn't fully appreciate how little research has been done in this area; first in the regulatory area, for sure, but also patient preferences is a whole untapped area for reimbursement that needs to be considered in the future as well," he added.

Murray emphasized their framework is only recommending using patient preference as an additional factor to the risk-benefit analysis FDA already considers and shouldn't be thought of as lowering the bar for product approvals and putting more patients at risk. "We believe that the additional evidence that is provided through patient preferences will be valuable in informing both the sponsors and FDA on factors that could be considered in the clinical trial design and the review process," he added.

When considering how useful patient preferences are to the regulatory process, Murray points to a matrix in the report that balances risk against benefit in determining the value of patient preference information (pictured above).

So far, FDA has emphasized patient-preference factors most formally in the obesity device space. Earlier this year, the agency approved EnteroMedics Inc.’s Maestro neuromodulator to treat obesity despite the device not meeting all endpoints in its pivotal trial. FDA relied in part on a survey that found obese patients were willing to take more risks in exchange for weight loss to support the approval. (See Also see "FDA Weighs Patients' Risk Tolerance in Approving Obesity Device" - Medtech Insight, 14 January, 2015..)

Device Consortium Tries To Shed Light on Patient Risk-Benefit Preferences

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