The FDA Can Harm Patients

If a pharmaceutical company's drug harms or kills a patient, that's front-page news. But that's not the only way that patients can be harmed. A patient who isn't given a helpful medicine is also harmed, but unlike with the first example, this story doesn't make it onto the front page or any other page of the newspaper for that matter, because the victim and the harm caused are invisible. Who was the victim? We usually don't know. What drug would have helped them and how much? We usually don't know.

The 19th century French economist Frederic Bastiat taught economists to study both what is seen and what is unseen. (Read What Is Seen and What Is Not Seen.) For instance, if a broken window is replaced, the natural tendency is to focus on the benefit to the economy--and the glazier--of the payment for the window (the seen) while overlooking the negative result of the building's or car's owner not spending that same amount of money on something else (the unseen).

The "seen" in the pharmaceutical example is a specific drug harming a specific person. The "unseen" is a drug not helping a person. This unseen situation often results from a drug not being developed, perhaps because a company didn't see the economic payback, or because the drug was explicitly rejected by the FDA.

Sometimes, however, the unseen can be observed by careful observation. Consider two young boys with the same deadly disease-- Duchenne muscular dystrophy. Max Leclaire, 10, is currently in a clinical trial for a new drug that has miraculously reversed some of his debilitating symptoms. His brother Austin, 13, tried to enter the clinical trial but didn't meet the criteria.

Sarepta Therapeutics is developing eteplirsen. For the last year Max has received a once-weekly IV infusion in a double-blind clinical trial. After just four months, Max's parents realized that their son was getting the active drug because it has not only slowed the progression of the disease but has dramatically improved his symptoms. They just wish their second boy, who has steadily worsened, could have the same chance.

The juxtaposition of the two boys' conditions allows us to say with some confidence that Austin is being harmed by not receiving eteplirsen. Why won't Sarepta give eteplirsen to Austin? Essentially, the FDA won't allow it. According to the FDA, the proof of eteplirsen's efficacy and safety hasn't been established. Would Austin's parents, given everything they know about eteplirsen, willingly give it to Austin today? Definitely.

By being forced to follow the FDA's assessment of what an acceptable risk is (for all drugs, not just those in clinical trials) instead of his parents' own, Austin's situation gives us a rare opportunity to shine a light on a normally unseen problem and to see just how the FDA can harm patients.


Your blog is so off it is really irresponsible. Let me point out a couple of points you are completely wrong on:

1. "Why won't Sarepta give eteplirsen to Austin? Essentially, the FDA won't allow it."

That is not true because you already stated: "His brother Austin, 13, tried to enter the clinical trial but didn't meet the criteria." So, it was Sarepta who didn't allow Austin to enroll the trail, why? Because they didn't think the drug would help people who don't meet inclusion criteria.

2. "The juxtaposition of the two boys' conditions allows us to say with some confidence that Austin is being harmed by not receiving eteplirsen."

Confidence? What confidence? Sarepta already thought the drug wouldn't help people who couldn't walk which was the case with Austin, why would you have confidence the drug would work for Austin? Have you looked at the trial result released? Two patients who met inclustion criteria progressed rapidly after 24 weeks of treatment Serapta didn't include them in analysis.

I find the case is interesting, but it is not fair to FDA. Without FDA guardianship, we would see more "unseen" harm. In this regards, it is still the equation of risk/benefit that matters.

In addition, from this anecdotal case (a pair of brothers of DMD), it is hard to tell if SRPT's eteplirsen works or not.

From the fact that US FDA is most respected in the world, we can safely assume that FDA is doing its job fairly well.

Just like because democracy has its cost, we can not say China has a better legal, political and economical system than US. In fact, US has its superiority over China in almost every aspects.

The author is 100% right. True drug companies have inclusion/exclusion criteria but that is solely because of the FDA and the (in my opinion) unscientific manner the FDA looks at data. I personally know of drugs (approved for more common diseases) that can help those with rare diseases but the drug companies (BP) won't run clinical trials for these diseases because 1) need a way to determine sub-type to see who it will really help first and 2) those with the rarer disease have other comorbidities that will cause their "clean" drug to look as though has a lot of AE & SE which really doesn't. SO they don't want to "damage" the drug in eyes of FDA.

FDA needs to stop averaging results v. placebo. Many drugs work/cure some, do nothing for others, and should not be scrapped. I know they are trying to change this. Not soon enough.

Frist of all I think I need to say that my comments are based on first hand involvement in clinical trials and following eteplirsen and avi/sarepta carefully. First of all the inclusion / exclusion criteria for this trial was set by sarepta to include only boy who could still walk but exclude boy who walked to fast or could not walk anymore this was done because of the belief that the six minute walk test was the most acceptable test to show functional gain to the FDA and that functional gain would be very important even with the primary end point being the production of dystrophin. The 24 week data set showed that all of the 30 milligram boys are producing dystrophin.The drug was expected to slow the progression not be a cure. It is not that the drug can not help people in wheelchairs it was that sarepta believed it would be hard to show slowed progression/functional gain to the FDA. As for the two boys that had rapid progression this happened in the first 12 weeks of the trial. It was shown in the 12 week data set that dystrophin was not produced in the first 12 weeks and maybe sometime between 16 and 24 weeks before you may see dystrophin and without dystrophin you will not have drug related gains. So it is safe to say that max is making dystrophin and austin is not and that the trial was setup to pass the FDA as fast as possible. Also they have not had any safety related events with this drug. So in this case it would seem that the FDA was the deciding factor in excluding boys in wheelchairs and has caused harm.

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