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changed under President Obama’s first FDA commissioner, Margaret “Peggy” Hamburg, who oversaw new programs to streamline reviews and—of no small importance—helped boost the agency’s budget. (Part of that increase went toward FDA’s new tobacco regulation authority.)
Groups with a mission to speed up life-saving drugs to patients agree. “The FDA is the gold standard in the world in terms of reviews,” says Faster Cures executive director Margaret Anderson. “We’re not hearing anyone interested in lowering the bar.”
Anderson referred to a Faster Cures survey late last year of 150 entities from the biomedical community, including drug companies and patient advocate groups. Everyone who has to work with the agency has suggestions for improvements, many of which the FDA has cautiously begun to explore. But of greater concern, Anderson says, is that the agency be better staffed, fully funded, and that the law to allow it to fund its drug-evaluation work—the Prescription Drug User Fee Act (PDUFA)—be reauthorized this year without delays. Nearly everyone interviewed for this story echoed those same concerns. (Republicans reportedly have set a June PDUFA deadline, but there are reasons to worry about delays.)
Changes under Hamburg and her successor Califf have allowed drugs to come to market with abbreviated testing for efficacy as long as post-approval studies continue to show good results. For example, the successful cancer immunotherapy pembrolizumab (Keytruda), famous for treating President Jimmy Carter’s skin cancer, was first approved in 2014 based on a 173-person trial that compared the drug to historical standards, not to the then-current standard of care. Pembrolizumab has since been approved for certain patients with lung and head-and-neck cancer, as well.
These so-called conditional approvals are not without controversy. Last year, senior FDA officers overruled agency staff to approve eteplirsen (Exondys 51), a drug to treat a subset of patients with the rare genetic disease Duchenne muscular dystrophy. There was no direct evidence of efficacy, but the FDA’s top drug evaluator, Janet Woodcock, gave a green light on Sept. 19 because the data were “reasonably likely to predict clinical benefit” for patients.
Duchenne patient groups, which had exerted intense pressure on politicians and FDA staff, hailed the decision. But others saw it as a slippery slope toward more approvals based on flimsy data, not to mention more encouragement of drug companies to fund patient groups. Consumer watchdog Public Citizen, a critic of the ties between the two, published a report last year on the alignment of pharma funding with patient groups’ lobbying against Medicare changes.
Rare disease patient advocates, working with patients who often don’t have an approved drug for their condition, are very vocal. But what about those who campaign for patients with much more common diseases? Alzheimer’s disease is already a massive health and financial burden and will keep growing as the population ages. Other than a few drugs that temporarily slow cognitive decline for some patients, nothing to treat Alzheimer’s has ever received the FDA’s blessing. The failure rate in clinical trials was nearly 100 percent when this study was published in 2014, and more failures have accrued since then.
But a patient advocate says less regulation wouldn’t necessarily help. “We haven’t had any experience that the FDA is being too tight or over-regulating Alzheimer’s drugs,” says George Vradenburg, chairman of US Against Alzheimer’s. “They’re willing, ready, and able to evaluate drugs that are successful in clinical trials. But they haven’t received any.”
Could the agency make changes to help more Alzheimer’s drugs get into and through clinical trials? The process is daunting because the disease takes so long to advance. Drug makers would love to use a biological signal that shows a drug is working—a short cut, in effect, around long trials that have to show better health outcomes. These signals, known as surrogate endpoints, are often acceptable to FDA reviewers in … Next Page »