Fueled by new technologies and a growing commitment to patient-centric trials, patient-reported outcomes (PROs) are becoming increasingly important in the conduct of successful clinical trials. That’s the ground-level assessment of experts at the FDA/CMS Summit held this week in Washington, D.C.
“We’ve been talking [about PRO] for 20 years, but I think technology is pushing [adoption] forward and really making a difference,” said Christine McSherry, executive director of The Jett Foundation.
Patient centricity puts the focus where it should be, said Cynthia Grossman, PhD, an associate director with FasterCures, a center of the Milken Institute. Legacy information created by clinicians is important, “but maybe it’s not the most important voice” when determining trial goals and endpoints, she said.
“We need to listen to the patient” when crafting PRO programs, added Tammy Phinney, vice president for U.S. regulatory sciences at Biogen. Clinicians and others shouldn’t assume they know what’s important to patients, or how patients are impacted during a trial.
Biogen has used PROs extensively in trials involving nearly 20,000 patients related to drugs treating muscular dystrophy, for example. “We’re able to get the clinician information in real time as to how the patient is feeling,” Phinney said. At Biogen, PROs help to track patient pain, fatigue, and whether they can return to work during the trial. “It’s important to see the value” of PROs, she added.
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Phinney recounted an anecdotal example from a Biogen trial. A patient reported that it was very important for them to retain use of their fingers to be able to work their own wheelchair, rather than require assistance. While that kind of desire makes perfect sense, PROs can help to highlight when an endpoint or treatment outcome is more important than study personnel might be able to appreciate. “The importance to patients is not always an outcome,” Phinney said. “It might be a performance measurement.”
McSherry, a former nurse who was active in AIDS and HIV trials, reported that a patient in a trial clarified that it wasn’t the basic ability to walk they valued, as much as it was the ability to perform certain tasks if they were unable to walk. While subtle, that kind of distinction can better help hone a trial to best suit patients.
Author: Michael Causey