The Food and Drug Administration may establish an Office of Patient Affairs, the agency announced in a Federal Register notice Monday. Such an office has the potential to elevate patient advocates’ perspective, which some fear is already too influential at an agency where science is supposed to take priority over emotional appeals from the sick or dying.
In recent years, the FDA has held a series of meetings with patient groups seeking their input on aspects of treatment and the risks they are willing to take. The medical innovation bill known as the “21st Century Cures” act (PL 114-255) that Congress cleared in September requires the FDA to consider patient engagement by coming up with a way to collect and measure patient experience data.
Over time, the agency would incorporate the patient data into its benefit and risk assessments when making approval decisions. For example, if one patient population is willing to take greater risks for a treatment that has severe side effects but has positive effects on certain aspects of the disease, it could make approval more likely than for the general population.
The FDA is seeking comments on the new office, which would “be tasked with supporting and coordinating patient engagement activities across medical product centers and other offices that engage with patients and their advocates,” according to the Federal Register notice. Its responsibilities would include being a main FDA liaison for the patient community and maintaining the data systems that would manage the data collected from patients.
Jeff Allen, president of Friends of Cancer Research, welcomed the announcement and said the office would be a good way to make patient engagement consistent across different agency divisions that focus on drugs and devices. He also noted that it would “provide a more streamlined and more efficient structure that can make it more obvious to advocacy organizations or patients individually who have questions for FDA to have a common place to go for answers.”
The announcement will likely be cheered by other patient advocates, whose influence has risen as drug development has increasingly focused on rare diseases. Robert Califf, the former FDA commissioner, discussed their importance at a conference shortly after he stepped down from the post in January. “If I had a disease with an ineffective advocacy group, I’d be worried right now,” he said, according to the biotech news website Xconomy.
That influence was on display last year when the FDA approved a drug to treat Duchenne muscular dystrophy, a rare but fatal condition that affects young boys. The FDA approved the drug, Eteplirsen, even though many of the officials who reviewed its application thought it was ineffective. Janet Woodcock, head of the FDA’s Center for Drug Evaluation and Research, overrode the decision of reviewers. The move was largely seen as a result of pressure from the families of affected children and hundreds of lawmakers who urged the FDA to approve it.
Critics of the FDA’s decision to approve Eteplirsen understand the need to listen to patients but worry about what the new office could mean for future approvals. “While certainly the views of patients are important and should be heard I think it’s important that emotional, anecdotal pleas for faster approval of drugs should never take precedence over the science,” said Michael Carome, director of the health research group at Public Citizen.