Results from a recent FDA survey that explored how much risk patients would tolerate to lose weight through a device-based procedure provide insights that can be adapted to benefit-risk analyses for other medical treatments, CDRH regulators say.

“We wanted to see if it was feasible to elicit patient preferences, and more than that, to assess quantitative preferences and to use that in our review process,” said Telba Irony, chief of the general surgical devices branch in CDRH’s Office of Surveillance and Biometrics, who developed the pilot study with the aid of consultants at RTI Health Solutions. Irony described the results of the study at a patient preference initiative workshop Sept. 18-19 at FDA headquarters in Silver Spring, Md.

“The key factor is patients’ tolerance for risk. These devices aren’t used by my reviewers, they’re used by patients,” CDRH Director Jeffrey Shuren said.

FDA also plans to establish a patient advisory panel, CDRH Director Jeffrey Shuren announced at the recent workshop. Shuren told “The Gray Sheet” that the panel could provide input on patient preferences and agency management of risk/benefit analyses for devices. “In the course of next year, we will flesh out the scope of the group’s charter,” he said.

He added that the patient preference initiative is currently limited to gaining patient input for agency pre-market decision making. For the pilot study, he said, FDA was keen to discover what patients wanted out of their weight-loss procedures.

“The key factor is patients’ tolerance for risk. These devices aren’t used by my reviewers, they’re used by patients,” Shuren said.

Reed Johnson, a fellow with RTI Health Solutions, noted that people are willing to accept varying degrees of risk in exchange for good outcomes. But because “both doctors and patient organizations are reluctant to put a number on patient acceptance of risk,” a better way to extract that data is to survey patients themselves.

The effort is a continuation of FDA’s Patient Preference Initiative, announced in 2012 as a strategy to quantitatively measure patient opinion and use it to help inform benefit-risk decisions in developing and approving devices. (See Also see "FDA Proposes Sliding-Scale Paradigm For Obesity Device Trials" - Medtech Insight, 21 May, 2012..)

Obese Individuals Surveyed

CDRH designed a survey of 650 obese subjects recruited from a population of more than 11,600 individuals. (See Also see "FDA Takes Initial Steps To Quantify Patient Opinion" - Medtech Insight, 28 May, 2012..) The survey was administered online, with households lacking internet coverage given free notebooks and internet services.

The participants were asked a series of discrete-choice questions on which of two hypothetical weight loss device treatments they would prefer, given the likelihood of various side effects and outcomes with each device. Among the choices were different types of weight-loss operations, including endoscopic, laparoscopic or open surgery procedures.

Gastric bands – including Allergan Inc.’s Lap-Band and Johnson & Johnson/Ethicon Endo-Surgery Inc.’s Realize – are currently the only approved obesity-control devices in the U.S., but the agency anticipates many more applications for surgically and endoscopically-placed obesity-control devices in the near future. For example, GI Dynamics Inc. is putting its endoscopically-placed EndoBarrier obesity control and diabetes device through clinical trials in the U.S. with the goal of eventually winning FDA pre-market approval. (See Also see "GI Dynamics Goes Down Under To Fund First Endoscopic Treatment For Diabetes" - Medtech Insight, 29 July, 2013..)

FDA also surveyed patients on their tolerance for dietary restrictions; their expectations for average weight loss (from 5 percent to 30 percent); duration of the weight loss; tolerance for side effects; and acceptance of mortality risk from different procedures in exchange for the benefits of weight loss.

The study included a subset of patients who had already undergone either gastric bypass or banding surgery.

For example, one of the survey questions asked which of two devices “is better for people like you,” with one of the devices described as providing an average of 30 pounds of weight loss lasting five years but posing a 10 percent risk of death, and the other described as offering 60 pounds of weight loss lasting one year and posing a 1 percent risk of death.

One key finding was that an average respondent weighing 243 lbs. and standing 5 feet, 10 inches tall would tolerate a 0.5% risk of death in order to achieve a 24 percent weight loss lasting two years, a 20 weight loss lasting 18 months, or an 18 percent weight loss lasting five years.

Key Findings From Obesity Patient Study

In an FDA survey of 650 obese subjects, the average respondent (243 pounds and 5’ 10”) said that to accept a device associated with a mortality risk of 0.5 percent, they would expect to lose at least:

24 percent of body weight, for 24 months;
22 percent of body weight, for 36 months;
20 percent of body weight, for 18 months; or
18 percent of body weight, for 60 months.

Source: “Patient Preferences Initiative Survey for Weight-Loss Devices,” presented Sept. 18, 2013, by Telba Irony, General and Surgical Devices Branch chief, Office of Surveillance and Biometrics, CDRH.

Patient Reps, Industry, Physicians Weigh In

Representatives of patient organizations at the meeting said patients have clear ideas about their expectations for devices and the government’s role in approving them. “The study design is going to be key, and there is so much patients can tell you about endpoints” for a clinical trial of a device, said Bray Patrick-Lake, director of stakeholder engagement for the Clinical Trials Transformation Initiative.

Wendy Selig, president and CEO of the Melanoma Research Alliance, commented, “I don’t think the patient really cares which technology or test is used. What they want to know is, can they count on the results?” She added that an important consideration for patients is how much the use or implantation of a device will affect their everyday activities.

Sally Okun, vice president of advocacy for PatientsLikeMe, suggested that in designing patient surveys, FDA rely on registries such as PatientLikeMe’s, where researchers and FDA reviewers can query patient-members who live with and are being treated for one or more of 2,000 different diseases. PatientsLikeMe is an online network of 220,000 people with fibromyalgia, multiple sclerosis, major depressive disorder, type 2 diabetes, epilepsy and other disorders who can be asked about the effectiveness of various therapies, side effects, the reasons they began or ended treatments and other patient-related concerns.

Industry and physician representatives at the meeting focused on what FDA should do with the risk-benefit input obtained from patients when reviewing individual devices.

“In addition to looking at the average patient, I think you should look at the distribution of patients,” said Scott Braithwaite, a medical doctor and president of the Society of Medical Decision Making.

Jack Lasersohn, general partner at the venture capital firm Vertical Group, agreed, saying FDA should not examine the average patient’s preferences in approving devices, but instead look at the spectrum of patient responses.

“If we choose the average patient, we deny opportunities” to use a device, but if FDA instead approves a device based “on the response of the most [risk] tolerant patient,” then those who are less tolerant of a device could always turn down the opportunity to use it or have it implanted.

Kate Ryan, senior program coordinator with the National Women’s Health Network, said what is most important is the way information about device risks is conveyed to patients. “Providing that context of harm is most important in patient communications,” she said. Ryan presented the example of female obesity patients who may not understand that an obesity device such as a gastric band doesn’t last forever, or may have to be replaced and explanted because of problems or wear 10 years down the road.

The National Women’s Health Network has a clearinghouse where “we’ve had women who have received the lap-band and had a problem where it needed to be removed, and insurance won’t cover that,” Ryan explained. “If a woman is struggling with serious financial problems and insurance doesn’t cover the re-operation because it’s an elective surgery to begin with, that’s a problem.”

Ryan asked CDRH regulators if they took such considerations into account in the recent survey.

Irony explained that the pilot study was too small to cover all types of situations. “Each patient gets tired of answering questions. They answer eight questions, and you can’t go more than that,” she said.

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