The FDA Empire Strikes Back
The bureaucracy is talking down a drug the agency approved.
Photo: Getty Images/iStockphoto
Dec. 22, 2016 7:04 p.m. ET
37 COMMENTS
This year produced no shortage of surprises, and a rare happy one was Food and Drug Administration approval of a muscular dystrophy drug that had been singled out for destruction by agency staffers. But the bureaucracy is now mounting a misinformation campaign, and as a result insurers are denying coverage to some patients.
In September the FDA approved eteplirsen, now marketed as Exondys 51. The treatment slows the decline in patients with Duchenne muscular dystrophy, and 10 of 12 boys in a trial still walk after four years of treatment. Janet Woodcock, who runs FDA’s drug evaluation center, approved the therapy after a brawl with reviewers, who demanded a placebo trial that would be unethical and impossible to fulfill for a rare pediatric disease.
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But at the same time the agency released some 100 pages of documents trashing the drug’s data, in what looks like a case of bureaucratic revenge to hang approval solely on Dr. Woodcock. Then FDA wrote a label that says clinical benefit “has not been established,” no doubt to scare off insurance companies and doctors. The label omits that the drug was approved because of an effect that is “reasonably likely” to predict clinical benefit.
A member of the committee that advised FDA on Exondys 51, Aaron Kesselheim, lamented FDA’s approval decision in the Journal of the American Medical Association. Dr. Kesselheim has no known experience treating Duchenne patients, and it shows: He says the treatment targets a nonsense mutation, which it does not. The treatment is for a different mutation known as a deletion. He also calls the disease “usually” fatal when the death rate is 100%.
More astounding is that John Jenkins, director of the office of new drugs, called out the drug’s developer in October at a conference: “path taken by Sarepta NOT a good model for other development programs,” according to his Power Point slides on FDA’s website. One slide calls this merely his opinion, but the presentation bears the agency logo. FDA declined to comment, though it recently announced internally that Dr. Jenkins would retire.
All of this is spreading confusion about an approved therapy. Anthem has declined to cover the treatment, calling it “investigational.” (That’s false.) Aetna will require boys to start the drug before age 14, among other stipulations, and insurers like Humana are limiting the treatment to boys who can walk. The companies’ explanation is that there’s no proof of clinical benefit or improved health outcomes.
But the walking test is a bogus measure. Dr. Woodcock approved the drug because it produced dystrophin, a missing protein. Performance on a walking test had nothing to do with it, though the trial boys did walk nearly two football fields farther than those in a control group. The drug can slow the disease of boys who no longer walk, even if it’s more effective the earlier it’s started. Sarepta must have more detailed data on how the drug helps preserve pulmonary function, and the company should let it rip.
That might help twins Jack and Nolan Willis, the two patients who lost the ability to walk during Sarepta’s trial. The boys have continued infusions every week for more than five years, which allowed Sarepta to collect data on boys whose condition had progressed further. The Willis twins testified at an April FDA meeting that treatment had helped them retain abilities like picking up books and brushing their teeth. Their hearts and lungs are also performing better than expected.
Get this: The twins have so far been denied coverage for a drug they’ve spent their lives trying to get approved. An initial denial letter we’ve reviewed calls the drug “not medically necessary,” which must be news to the boys and their family. Excellus, a BlueCross BlueShield affiliate in New York, is also only covering patients who can walk, according to its policy online. The company declined to comment on specific cases.
The cost of the drug is high: Price is based on weight and can run $300,000 a year. Yet fewer than 2,000 patients in the U.S. are amenable, which means insurers won’t have an influx of beneficiaries. Care for Duchenne patients in their late teens or early 20s is already expensive, from hospitalizations to cardiac medication, so insurers will pay in any event.
Insurers are usually hypersensitive to avoid public shaming campaigns, but here they don’t seem too concerned. That’s probably because the reporters who cover FDA have taken the side of the reviewers and insurers. Thus one of the grim ironies of this story: The men who in the 1980s beat down the doors at FDA to demand approval for AIDS treatments are rightly regarded as heroes, yet these boys and their families have been treated as desperate losers.
Still, the genesis of these mixed messages is FDA, which should stop undercutting a drug the agency has approved. But that will require changing the bureaucracy’s toxic culture of political control and contempt for private innovation, and Donald Trump’s FDA commissioner should make that his first priority.
The bureaucracy is talking down a drug the agency approved.
Photo: Getty Images/iStockphoto
Dec. 22, 2016 7:04 p.m. ET
37 COMMENTS
This year produced no shortage of surprises, and a rare happy one was Food and Drug Administration approval of a muscular dystrophy drug that had been singled out for destruction by agency staffers. But the bureaucracy is now mounting a misinformation campaign, and as a result insurers are denying coverage to some patients.
In September the FDA approved eteplirsen, now marketed as Exondys 51. The treatment slows the decline in patients with Duchenne muscular dystrophy, and 10 of 12 boys in a trial still walk after four years of treatment. Janet Woodcock, who runs FDA’s drug evaluation center, approved the therapy after a brawl with reviewers, who demanded a placebo trial that would be unethical and impossible to fulfill for a rare pediatric disease.
***
But at the same time the agency released some 100 pages of documents trashing the drug’s data, in what looks like a case of bureaucratic revenge to hang approval solely on Dr. Woodcock. Then FDA wrote a label that says clinical benefit “has not been established,” no doubt to scare off insurance companies and doctors. The label omits that the drug was approved because of an effect that is “reasonably likely” to predict clinical benefit.
A member of the committee that advised FDA on Exondys 51, Aaron Kesselheim, lamented FDA’s approval decision in the Journal of the American Medical Association. Dr. Kesselheim has no known experience treating Duchenne patients, and it shows: He says the treatment targets a nonsense mutation, which it does not. The treatment is for a different mutation known as a deletion. He also calls the disease “usually” fatal when the death rate is 100%.
More astounding is that John Jenkins, director of the office of new drugs, called out the drug’s developer in October at a conference: “path taken by Sarepta NOT a good model for other development programs,” according to his Power Point slides on FDA’s website. One slide calls this merely his opinion, but the presentation bears the agency logo. FDA declined to comment, though it recently announced internally that Dr. Jenkins would retire.
All of this is spreading confusion about an approved therapy. Anthem has declined to cover the treatment, calling it “investigational.” (That’s false.) Aetna will require boys to start the drug before age 14, among other stipulations, and insurers like Humana are limiting the treatment to boys who can walk. The companies’ explanation is that there’s no proof of clinical benefit or improved health outcomes.
But the walking test is a bogus measure. Dr. Woodcock approved the drug because it produced dystrophin, a missing protein. Performance on a walking test had nothing to do with it, though the trial boys did walk nearly two football fields farther than those in a control group. The drug can slow the disease of boys who no longer walk, even if it’s more effective the earlier it’s started. Sarepta must have more detailed data on how the drug helps preserve pulmonary function, and the company should let it rip.
That might help twins Jack and Nolan Willis, the two patients who lost the ability to walk during Sarepta’s trial. The boys have continued infusions every week for more than five years, which allowed Sarepta to collect data on boys whose condition had progressed further. The Willis twins testified at an April FDA meeting that treatment had helped them retain abilities like picking up books and brushing their teeth. Their hearts and lungs are also performing better than expected.
Get this: The twins have so far been denied coverage for a drug they’ve spent their lives trying to get approved. An initial denial letter we’ve reviewed calls the drug “not medically necessary,” which must be news to the boys and their family. Excellus, a BlueCross BlueShield affiliate in New York, is also only covering patients who can walk, according to its policy online. The company declined to comment on specific cases.
The cost of the drug is high: Price is based on weight and can run $300,000 a year. Yet fewer than 2,000 patients in the U.S. are amenable, which means insurers won’t have an influx of beneficiaries. Care for Duchenne patients in their late teens or early 20s is already expensive, from hospitalizations to cardiac medication, so insurers will pay in any event.
Insurers are usually hypersensitive to avoid public shaming campaigns, but here they don’t seem too concerned. That’s probably because the reporters who cover FDA have taken the side of the reviewers and insurers. Thus one of the grim ironies of this story: The men who in the 1980s beat down the doors at FDA to demand approval for AIDS treatments are rightly regarded as heroes, yet these boys and their families have been treated as desperate losers.
Still, the genesis of these mixed messages is FDA, which should stop undercutting a drug the agency has approved. But that will require changing the bureaucracy’s toxic culture of political control and contempt for private innovation, and Donald Trump’s FDA commissioner should make that his first priority.
