Obesity Devices Target of FDA ‘Patients’ Feelings’ Survey

Another potential question to panels and others is, she said, “If we decide that patients don’t value benefits which we think are important, how will we use that information?” Irony noted that sometimes the agency learns that its reviewers like to avoid risk, but they forget about the risk of not approving something that could benefit some patients.

The study, to be done in conjunction with RTI International, was announced for public comment in the Federal Register on April 19. It will recruit 450 people to constitute a national cross section of well-informed subjects and ask them what risk they would tolerate for the benefits of obesity-reducing devices. The survey will include 100–150 people who have already undergone weight reduction procedures such as by-pass surgery or banding so that it can capture attitudes both before and after treatment.

Obesity was chosen as the subject, Irony said, because it has a high prevalence disease and treating it could have a big public health impact. In addition there are several devices that will be coming to the market to reduce weight. However, she noted, obesity treatment also offers a number of difficult risk and benefits trade-offs. “It was a challenging problem to pursue, but I think that it is going to be very rewarding,” she said.

Irony’s talk was at the session sponsored by RTI to showcase its work in the arena of patient feelings on risk-benefit. After the public comment period and approval by the Office of Management and Budget, the survey should take only a few weeks to complete, Irony said. She noted that if sponsors or patient advocacy groups do similar national representative studies, the agency would be interested in those results as well. FDA issued guidance to reviewers and industry on risk and benefits, including patient attitudes, on March 28.

Studying risks and benefits will be complex for the obesity study, as well as for other areas, Irony stressed. Questions will include whether a procedure will be performed once or repeatedly, what uncertainties will remain after the clinical trials, what the variations in outcomes might be among surgeons or medical centers, what the severity of the disease is, whether there is alternative treatments, whether the technology might improve with time, and whether patients tolerate more or less risk for conditions they have had for a longer time.

RTI Health Solutions principal economist Reed Johnson, who does research in this area, said that often when FDA advisory committee meetings have their public comment periods, “Typically a number of patients come in and cry.” They cry, he said, either because a loved one was injured by some therapy or because they believe they can benefit from a treatment which they fear the advisory committee will not recommend for approval. Thus physician advisors on the panels evaluate the evidence often for two days, “And then there is this sort of completely uncontrolled emotional input,” he said.

It’s hard to know how the committees are influenced, Johnson said. But if patients are going to have input, there ought to be a way to interpret whether it is representative, how many patients hold a particular view, and what the characteristics are for the patients who hold particular views, he urged. Calling for moving quickly and decisively toward turning patient input into evidence, Johnson said, “I think we need to think of patients as a source of data and not just a source of emotion.”