FDA Releases Framework for Considering Patient Experiences in Its Medical-Device Reviews

For decades, the Food and Drug Administration and the medical device industry have puzzled over how to factor the experiences of patients when making regulatory decisions. This month, they got some answers.

The Medical Device Innovation Consortium, a collaboration of the device industry, the FDA and the National Institutes of Health, released a framework — what it calls an “initial thought piece” — to help guide device makers as they collect patient-preference information and submit the data to the FDA when seeking product approvals. The topic will be on lawmakers’ minds as they work through the so-called 21st Century Cures legislation in the House Energy and Commerce Committee. The draft calls for the agency to incorporate patient input as it weighs new device applications.

“Patient preference is not a requirement for FDA approval,” explained Ross Jaffe of Versant Ventures, a venture capital firm. “The way to think about it is as an enhancement of a submission.”

The FDA also issued much-anticipated guidance outlining the patient-preference information the agency will consider on device applications. The new guidance builds on documents the agency issued in 2012 clarifying how it would consider data on risk tolerance and perceived benefits, as long as the data was validated by the agency.

“We never thought this day would come; that anyone would pay attention to our research,” said John F.P. Bridges, a professor at the Johns Hopkins Bloomberg School of Public Health and longtime researcher of patient perspectives in medicine. “Based on good science and justice, one day preference information will become ubiquitous.”

Medical devices can range from those used by physicians and nurses in clinical settings, including endoscopes and diagnostic tests, to those used by consumers, from insulin pumps to gastric lap bands.

But the research comes from a newly emerging area of regulatory science. The essential challenge lies in determining how to measure patients’ preferences in “preference sensitive” decisions — those in which benefits don’t clearly outweigh risks. The framework provides a catalog of quantitative methods that device makers can use, but is only a start, members of the consortium agree.

“This is going to be fairly overwhelming to the industry,” said Diana Salditt, a regulatory affairs adviser at medical-device giant Medtronic. “There’s a lot of education that needs to be done. I think that people will need to see and maybe even work though some case studies on how this will be applicable.

“We understand how to identify the attributes patients want,” Salditt added. “What we don’t understand as well is how to weigh attributes and measure the trade-offs.”

Still, the industry said it welcomes the agency’s consideration of the additional data. “We’re looking for an avenue to present new information,” said Bennett Levitan, senior director of benefit and risk assessment at Johnson & Johnson. “Compared to the cost of clinical trials, a preference study is not terribly expensive.”

The FDA says it can use the patient- preference data in evaluating device applications and in designing trials. But the process is still challenging and unclear.

“How much benefit do we want to see?” asked Telba Irony, of the agency’s Center for Devices and Radiological Health. “If we don’t approve the technology for the 40 percent [who are more risk tolerant], then nobody has access to it.”

Robert Califf, the FDA’s deputy commissioner for Medical Products and Tobacco, said patient perspectives are “near and dear to his heart,” having used such input in his research from his early days in medicine. Still, he urged the industry to be cautious.

“When we talk about risk and benefits, it’s not so simple,” he said. “We really need to make sure, when we present things to people, we give them a fair picture.”

That is not meant to stifle innovation, he added. “It’s to say: Let’s go do it.”

Theresa Mullin, who oversees the agency’s risk-benefit frameworks, asked the consortium how it might scrutinize biases in patient-preference data, particular coming from industry applicants.

“I think that’s a tough question,” said Brett Hauber, of RTI Health Solutions, a lead author on the framework. “You could manipulate these things.”

But Hauber explained that any peer reviewed data would be held up to scrutiny. “Each group has their own agenda. But each group is accountable to the others.”

The FDA’s Irony said the situation was analogous to the clinical trials process. “FDA tries to scrutinize for bias,” she said. “We’re going to have to go through the same process with this evidence. These preferences will come with uncertainties as well.”

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