A Muscular Dystrophy Study Raises Questions About Scientific Studies

Published September 29th, 2016 by USA Rx
Fact Checked by
Chris Riley

A Muscular Dystrophy Study Raises Questions About Scientific StudiesRecently, the people who suffer from muscular dystrophy, or MD, have received some good news: for the first time, the FDA has approved a new drug that promises to fight the disease directly. However, in the process, FDA officials got into some heated arguments about just how much they can trust the main study that showed what the drug can do.

MD refers to a set of genetic diseases caused by mutated genes. Most of the time MD starts in infancy or during childhood, but sometimes it waits for middle age to strike. Some versions of the disease are relatively mild, while others are eventually life-threatening. In each case, MD prevents the body from producing dystrophin, an important protein that protects your muscles from damage. Without it, victims will eventually lose the ability to walk and may waste away completely.

The drug in question, eteplirsen, promises to help fight Duchenne muscular dystrophy (the most common kind) by producing extra dystrophin. The company that makes the drug, Sarepta Therapeutics, proved it by funding a study that appeared in Annals of Neurology.

However, studies funded by pharmaceutical companies have always been at least a little suspicious. While they have to go through a peer-review process to make sure they’re scientifically sound, there’s always an urge to fudge the numbers in favor of the company that’s footing the bill. This is especially true when the company is your full-time employer, which was true for four of the study’s coauthors.

This potential for bias became a problem for eteplirsen because of a fight between different factions within the FDA over whether the drug was as effective as the study promised. Some said the study was misleading and should be retracted, others said it was perfectly accurate, but at the end of the day it seems like the drug would have been approved either way. As for the Annals of Neurology, according to them they haven’t heard of any scientific evidence that the study was skewed and they refuse to retract it until someone provides some.

So are company-sponsored studies inherently biased? Are they a good way to save taxpayer money that would have been spent on public studies? Is the peer review system trustworthy enough to keep these studies honest for at least a majority of the time? Unfortunately, the jury is still out on these questions.

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