Speaker: Patient preferences can aid FDA in advancing new dialysis devices
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A speaker at the virtual Innovations in Dialysis: Expediting Advances Symposium discussed how patient-preference information can be used by the FDA in making regulatory decisions on devices designed for patients with kidney failure.
In her presentation, Jenny Flythe, MD, associate professor of medicine at the University of North Carolina (UNC) School of Medicine and medical director of acute dialysis at UNC hospitals, first distinguished patient-preference information (PPI) from patient-reported outcomes (PROs), defining the latter as any report on the status of a patient’s health condition that comes directly from the patient with no clinician interpretation; in other words, she explained, the PRO instrument is designed to measure a patient’s perception of their health status before, during or after therapy.
On the other hand, she explained, “PPI captures the value that patients place on aspects of a medical device in a way that accounts for differing perspectives on the benefits and risks that come with using that device for treating a condition.”
According to Flythe, while the nephrology community has some experience with PPI studies (four studies, she said, have been conducted in patients with kidney disease), the measure has not been utilized much in the regulatory context for dialysis devices.
“The FDA, particularly the Center for Devices and Radiological Health [CDRH], has had a commitment over last 9 years to incorporating more patient preference science into their regulatory decision making,” she contended, citing the agency’s 2012 benefit-risk guidance which stated that patient-centered metrics are important when assessing which modes of treatment are worth further consideration.
The CDRH then launched a Patient Preference Initiative, in which experts in health economics, social sciences, patient advocacy and the medical device industry convened to discuss methods and tools for measuring treatment preferences, as well as to identify gaps in evidence.
The goal, according to Flythe, was to determine how patient preferences can be “valid and meaningful” in making regulatory decisions.
“One of core concepts the CDRH discovered was that patients vary greatly in the degree to which they will accept risk for a given benefit,” she said. “Not all people are the same. Not all patients are the same. They’re going to have different risk tolerances, and there needs to be a way to measure this.”
Flythe added that a significant component of a patient-preference study is the way preferences can be assessed not only for a patient population overall, but in a way that looks at heterogeneity in preference. As she described, patient preference information can determine “whether there are distinct subgroups whose preferences would lead them to make different decisions.”
She argued that these types of considerations are especially important for patients with kidney failure because the disease is “highly preferent sensitive,” meaning that multiple treatment options exist with none clearly superior to another for all patients; the evidence for one option over another is uncertain; and patients’ views about the benefits and acceptable risks of the treatments vary considerably.
“We all know that in-center hemodialysis is a miserable treatment,” she added. “It’s hard. It’s hard for someone who tolerates it well, but almost unlivable for patients who don’t tolerate it well.”
Therefore, she contended, utilizing PPI can help patients find a treatment that more closely aligns with their goals, whether that be in-center hemodialysis, peritoneal dialysis, home hemodialysis or wearable artificial kidneys that she hopes will come to market soon.
Flythe said there is currently a patient preference project in progress, known as Integrating Patient Preference into Regulatory Decision Making to Advance Innovation in Kidney Disease. The project is run by the Kidney Health Initiative, a public-private partnership between the FDA and the American Society of Nephrology.
“The overall objective is to develop a sustainable strategy for collecting PPI data from a representative sample of dialysis patients and to drive patient-centered innovation in dialysis devices,” she said, adding that the focus is on wearable dialysis devices that may be available in the near future. This, she said, makes these more immediately useful to the FDA.
“Well-designed and conducted patient preference information studies can provide valid and important evidence,” Flythe argued, again noting that the strongest studies are representative of the overall patient population and are also capable of capturing heterogenous preferences.
“An important ingredient is bringing together diverse stakeholders from the beginning,” she concluded. “This makes it more likely your patient preference survey will be a success, which will ultimately lead toward advancing patient centeredness in kidney failure. This is what we are all here to do.”
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Flythe J. Using patient preferences to drive patient-centered innovation in dialysis. Presented at: Innovations in Dialysis: Expediting Advances Symposium; Aug. 31-Sept. 1, 2020 (virtual meeting).
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