FDA dragging feet while children with muscular dystrophy are denied promising new treatment

If you've ever known a family touched by muscular dystrophy, you know what an agonizing experience it is - for the young patients (usually but not always boys), for their parents, and also for healthy siblings. A genetic condition, MD attacks the muscles in the body to the point where children are usually in wheelchairs before they reach their teens, and they rarely live much past 20. It's horrible. There's no other way to put it. If you're old enough, you might remember seeing some of the kids on the old Jerry Lewis Labor Day Telethons. Many of them accepted their fates with tremendous grace, but the truth is they deserved much, much better.

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And all those times you saw Jerry Lewis standing there talking about progress toward a cure, you probably wondered a time or two: Is there really any such progress? I know I did. But the truth is, yes, there is now a very promising treatment (if not an out-and-out cure) that's making a very big difference for patients who have had the opportunity to use it in clinical trials. It's called eteplirsen, and its manufactured by the drug company Sarepta. While it is only designed to treat patients with a strain of MD known as Duchenne, it's a very promising development that could lead to more development to help other MD patients as well. If, that is, the FDA ever approves it. After five years of successful clinical trials, Sarepta is still getting the door shoved in its face by our fine government regulators, who appear to care nothing for the kids but plenty for the i-dotting and t-crossing of their cherished process. The Wall Street Journal explains:

Sarepta has gone back and forth with the FDA since 2013, and this is somehow considered the expedited track: A 2012 law allows the agency flexibility to accelerate approval in first-in-class drugs for lethal diseases, though the FDA seems to be flouting the spirit of this directive. The agency planned to assemble an advisory committee—which offers recommendations that the FDA typically follows—in January. But the meeting was postponed due to a blizzard in Washington, one that apparently snowed in the FDA for four months. In January the agency issued a harsh report about eteplirsen, haggling over minutiae on the trial’s design and findings, and here’s why: The FDA is religious in trusting only large trials in which half of participants receive a placebo treatment. Yet such trials would prove near impossible since Duchenne patients are so rare.

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And more important, unethical: What parent would sign up a child for years of weekly muscle injections and high-risk biopsies if the cocktail might be saline? FDA acknowledges these concerns and admitted in a Thursday report that the agency previously told Sarepta it would consider the type of study the company performed—only to change its mind. At the FDA, process trumps patients. More than 35 physicians and leading experts, who have seen some 5,000 Duchenne patients and hail from the likes of UCLA and Harvard, offered their opinion in a February letter to the FDA, not that the agency asked. The doctors say the FDA’s work includes “scientifically questionable comparisons” and even errors. The boys are “clearly performing better than our collective clinical experience and the published literature would predict,” and the data show “substantial evidence of efficacy,” wrote the doctors. Accelerated approval and continuing further trials as the 2012 law prescribes, they concluded, “is the most ethical choice.” Adding more weight to the decision is that eteplirsen can only treat a certain mutation of Duchenne, and a no from the FDA would scuttle iterations in the drug-development pipeline that might help more patients. Biotech companies working on treatments for other rare conditions are also watching. If a drug with no safety risks, a four-year record of effectiveness, and a strong legal and ethical basis can’t win approval, what can?

Not only is the FDA's foot-dragging hurting the patients, which is obviously the most important thing, it's also also tanking Sarepta's stock, which fell 44 percent when the FDA released a briefing that trashed eteplirsen. If that's how the FDA is going to treat drug companies who take risks to develop these treatments, you know what's going to happen? No one is going to take the risks. Why would you if all that's going to happen is that you put millions into development only to have regulators refuse to approve your drugs for distribution, while patients wither and die when you could have been helping them? This is what happens when government gets too much power. People who worship process over people, and who are more upset about "drug company profits" than they are about the suffering of children, stop you in your tracks just because they have the power to do so and the arrogance to want to use it. I'm not saying there should be no regulation of drugs and no approval process. I think there should be. But the goal should be to make the drugs legally available quickly after a reasonable testing period to ensure they're safe, not to put drug companies and patients through a protracted hell while bureaucrats wave their clipboards around to show everyone how powerful they are. Stop this crap and approve the drug so these boys will have a fighting chance. And let's elect some new leaders who care about serving people rather than basking in their own power and self-importance. Jerks.

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