WASHINGTON — She’s been attacked by parents of dying children, vilified by consumer groups, and grilled by Congress, but over the past 30 years, Dr. Janet Woodcock has risen to become the top drug cop at the Food and Drug Administration — and managed to stay there.

“She sticks to her guns,” said Marc Boutin, chief executive officer of the National Health Council, a coalition of patient advocates, pharmaceutical companies, and others. “She’s tough as nails.”

That may be an understatement.

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Woodcock, who has pushed the FDA to consider the drug industry a partner and not an adversary, won a huge and consequential power struggle this week when she successfully pressed the agency to approve a controversial new drug for treatment of Duchenne muscular dystrophy — even though there is scant evidence it works. There is no other treatment for the rare disease, which causes muscular degeneration and, often, early death.

Patients, their parents, their congressmen, and drug maker Sarepta Therapeutics all lobbied the FDA hard for approval, writing heartbreaking letters and flooding public hearings to beg for access to the treatment.

But after parsing the data, the FDA’s scientific experts said there was no proof the drug, known as eteplirsen, actually worked. Dr. Ellis Unger, who works under Woodcock, went so far as to call it  “essentially a scientifically elegant placebo.”

He and others in the agency argued strenuously that the FDA should not give into public pressure to approve the drug. He even filed a formal complaint against Woodcock, accusing her of deviating from the typical drug approval process to advocate for eteplirsen.

Woodcock did not waver.

She overruled the staff — and then won over her boss, FDA Commissioner Dr. Robert Califf.

The power struggle has already rippled through the bureaucracy: Ronald Farkas, another scientist who worked under Woodcock and who wanted the FDA to reject the drug, recently left the agency. Reached at his new post at Parexel International, a science consulting firm, Farkas declined to comment.

Those who know Woodcock well say the episode perfectly showcased her style: equal parts confidence, determination, and steadiness under pressure.

“She’s one of the best witnesses the FDA can send to Capitol Hill because she knows far more than the people asking the questions and she’s definitely not afraid,” said Ira Loss, a Washington policy analyst who has tracked the FDA for 40 years. “The people in Congress, both sides, respect her.”

The episode also reflects the FDA’s transformation from a pure regulator to an industry partner — a makeover that Woodcock has driven. The shift has drawn much opposition from public health advocates and consumer groups, some of whom accuse Woodcock of being too cozy with the drug industry. But it’s drawn cheers from patient advocacy organizations and pharmaceutical companies who want to move new products to market faster.

Boutin, of the National Health Council, recalls talking to Woodcock about 10 years ago to enlist her support for giving the patient viewpoint more weight when the agency considered drug approvals. That was a big step for regulators who had long focused narrowly on the science and the data, not emotional testimony or anecdotal case studies.

“I took them to task,” he recalled, “and said while the benefit-risk determination should certainly be informed by the science, it is ultimately a judgment, and has to reflect the end user.”

Woodcock listened. And the agency changed.

One small but telling victory: “They used to talk about ‘risk and benefit,’” Boutin said, “and now they talk about ‘benefit and risk.’ That came from the patient community.”

In the case of Sarepta, he said, “We saw Janet step up and say, ‘You know the safety of this drug is very well established and the patient community says there’s a benefit.'” Other scientists at the FDA were dubious of that benefit, saying Sarepta’s tiny 12-patient clinical trial had not proved it.

Woodcock made clear even as early as last spring, when the FDA’s expert panel recommended rejecting the drug, that she had heard the patients’ pleas and considered them important. “It’s possible to reach different conclusions based on the data presented today,” she said during a public hearing in April. “Failing to approve a drug that actually works in devastating diseases — these consequences are extreme.”

But some see a risk that the FDA is downplaying serious scientific scrutiny.

Diana Zuckerman, president of the National Center for Health Research, said it’s not enough for the FDA to conclude that the Duchenne muscular dystrophy drug is safe. If it doesn’t work, why should patients and insurers pay an estimated $300,000 a year for it?

“The bigger risk is not the safety of this particular drug,” Zuckerman said. “The bigger risk for this drug is that the patients will end up not being helped, and in some cases, a family will go broke paying for a drug that doesn’t work. And other families will feel terrible because they can’t afford it.”

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Woodcock graduated from Bucknell University, received her medical degree from Northwestern, and did further training and held teaching appointments at Pennsylvania State University and the University of California, San Francisco. The FDA declined to give her age or other biographical details.

Woodcock has done two stints as director of the FDA’s Center for Drug Evaluation and Research, which covers all drug approvals and the thorny area of post-market surveillance — monitoring side effects and other concerns that crop up after a drug has been approved. She served as director from 1994 to 2005, then worked in several other FDA jobs, and returned for a second round in 2007.

Woodcock’s name has been floated several times for the commissioner’s post, but has drawn opposition from consumer groups and some congressional quarters.

She has testified countless times before Congress. And she’s been put on the spot time and again. She had to parry criticism from Congress that the FDA moved too slowly to mandate labels on certain antidepressants warning of the risk of suicide among children. She also had to stand up to criticism that the FDA moved too slowly on Vioxx, a painkiller which led to heart attacks and strokes in adults. Manufacturer Merck ultimately took it off the market.

“She has been in this position two decades now,” said Dr. Michael Carome, who runs Public Citizen’s Health Research Group, who often goes head-to-head with Woodcock. “Her tenure has been a period in which the relationship between the agency and the regulated pharmaceutical industry has evolved to one where even the agency describes it as a ‘partnership.’”

Carome and Zuckerman both criticized Woodcock’s handling of Avandia, the diabetes drug that caused serious heart problems and resulted in a spate of lawsuits against GlaxoSmithKline, the manufacturer.

Stephen Northrup, a partner in health care policy at lobbying firm Rampy Northrup, said Woodcock is widely considered a good manager and someone Congress can work with.

He was working as a staffer in the Senate during the Vioxx controversy — and he recalls that Woodcock was willing to admit mistakes.

“I found them to be responsive on Vioxx and what it suggested about their shortcomings,” Northrup said. “They were open to working with us. They didn’t dig in their heels.”

Zuckerman says part of Woodcock’s power comes from the fact that as a career public servant she has outlasted so many commissioners, who are political appointees.

“Somebody new comes in, they have an enormous responsibility but they don’t know how to do the job, so they have to rely on the center directors,” Zuckerman said. “Even the ones who have doubts about her have relied on her. She’s been there a long time. She knows how the agency works.”

The Sarepta drug may be Woodcock’s most controversial decision. And it could end up shaping her legacy.

“If the drug is effective and the kids do well and the price is affordable, she’ll get the credit,” Zuckerman said. “And if not, she’ll get the blame for any problems with this drug and with the precedent it sets resulting in other questionable drug approvals.”

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  • If Dr Woodcock worries about the company’s stockprice (as per FDA documents), she should also realize that instead of spending 300k per year on a drug with “no established efficacy” 3 patients with cancer could instead have access to a drug that could save their life that would otherwise be denied to them. Additionally, with CDC data pointing to 4200 DMD patients in the US, ie. 550 for exon51, of which 275 walking, of which some 200 in clinical trials, the AA gives the company a strong incentive to pursue the few patients left for commercial revenue rather than confirmatory trials which it will likely postpone as long as possible, and this will make it extremely difficult for good science from other companies to enroll patients into trials with a working drug. In my view this is a sad outcome for patients giving them false hope and taking money out of families and insurers – if, that’s an if, insurers will pay for something that doesnt work. The FDA made a decision that lowers standards, scientific rigor, and provides the wrong incentives for an industry with overpaid management (CEO got 4.4m USD in comp 2015 and probably a lot more in 2016).

  • I think Dr Woodcock made the right call in this case. The accelerated approvals process was put into place so that precisely for approval of drugs based preliminary evidence against a surrogate endpoint. The law allows for some these drugs to later be proven ineffective based on more rigorous trials. To say that it’s Dr Woodcock fault if that happens is part of a culture of blame in DC that hurts patients.

    That drugs approved under accelerated approval process are invalidated so infrequently is an argument that the FDA actually might have been too conservative with accelerated approvals in the past. After all, what is the purpose of an “accelerated” approvals process that is 100% accurate? The understanding here is that we are trading off time for certainty. If there is no trade off, then why have the “slow” process at all?

  • Woodcock made the right decision here given small but measurable improvements on a fatal condition affecting children and a safety record for the drug. Congress needs to reboot the device side of FDA to elevate patient protection over industry protectionism. CDRH has overridden its advisory committees to mis-classify devices as safe when patients are evidently harmed by medical and dental devices. Unlike drugs that can be stopped at any time, device removal and replacement is costly and invasive, not always covered by insurance (especially dental materials causing harm) or with significant co-pays and deductibles, and systemic inflammatory reactions may continue. Congress needs to fix CDRH now.

  • Dr Woodcock is implementing accelerated approval as congress intended – a mechanism for gathering definitive information about a drug that is reasonably likely to provide clinical benefit while all elligible patients gain access. This is not a full approval – it is a conditional approval with post-marketing requirements that will most definitely be fulfilled. Dr Woodcock has charted the only ethical and scientifically responsible path forward possible for Eteplirsen.

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