I am Dr. Laura Gottschalk, speaking on behalf of the National Center for Health Research. I received my Ph.D. from Johns Hopkins School of Medicine. Our research center scrutinizes medical data and provides objective health information to patients and providers. We do not accept funding from pharmaceutical companies, and I have no conflicts of interest.
We agree that FDA should get safe and effective new treatments to patients as quickly as possible, especially for a devastating disease such as Duchenne. We were hoping for persuasive data on eteplirsen. But with only 12 patients, inadequate control groups, and variations in disease progression, approval would only be appropriate if there’s a very clear benefit. Sarepta was warned about this in advance.
Unfortunately, the data do not meet a scientific standard of evidence of effectiveness. While there was an increase in dystrophin positive muscle fibers, the western blot shows a total amount of protein below what is estimated to be clinically significant. And, the 6-minute walk test was fraught with problems.
- After less than half a year, Sarepta eliminated placebo controls for a drug intended for life-long use.
- It became an open-label study, which could influence the walk test results.
- There are problems with the historical controls used, such as evidence that boys in the control group had little incentive to comply with the walk-test, and so some were mislabeled as non-ambulatory.
- Two of the patients did very poorly on the drug. Sarepta assumes that their early loss of ambulation was unrelated to treatment, but this hasn’t been proven.
Any one of these problems undermines the study results. But to have ALL these problems (and others) is simply unacceptable.
U.S. law requires evidence of safety and effectiveness. The burden of proof lies with Sarepta. If this drug actually works, then Sarepta has failed itself, the patients, and their families, by not conducting a better study that could provide convincing evidence showing that it works.
Since 2014, Sarepta has been enrolling patients into a larger study of more than 100 boys, but none of those results were provided to FDA for this meeting. Why not? Even 40 more patients would provide better evidence if the results showed clear benefits. Sarepta should have provided the additional data to FDA to examine and provide to this Advisory Committee. That’s how the process works. This committee should not make a decision based on evidence that has not been vetted by the FDA.
You’re hearing from many patients and family members today who believe in this drug. Your role on the Advisory Committee is to pressure the company to provide scientific evidence BEFORE approval, not to pressure the FDA to ignore the lack of scientific evidence.
Your decision today will send a message about whether scientific standards should matter to the FDA. I am very sorry to say that approval of eteplirsen based on today’s data would set a dangerously low bar for all drugs in the future.
We all want an effective drug for Duchenne. I strongly urge the FDA and Sarepta to work together as quickly as possible to prove whether or not eteplirsen is that drug. Better data are needed to do that, and that’s what is needed to save lives.
Treatments for rare diseases can be proven on small samples, but not based on 12 patients in a poorly designed study with ambiguous results. These boys and their families deserve better.