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Yearly Archives: 2015

November 30, 2015 no comments

DMD Drugs: An Existential Threat To The FDA?

Last week saw the advisory committee meeting for drisapersen, the last of three new candidate treatments for Duchenne Muscular Dystrophy (DMD) to begin the process for approval in 2015.   The outcome is clearly important for patients with DMD and their families. Equally, it is important to the companies who have developed these drugs, and their investors. What may be less obvious is that the outcome could be just as critical for the FDA itself.

The mission of drug regulators is pretty simple (at least in theory): to protect the public from quack medicines that are either unsafe or simply don’t work. In practice, it gets a little more complicated because drugs need to be approved before enough patients have been exposed to be absolutely certain about either safety or efficacy. The regulator, therefore, has to make a call as to whether the available data passes the threshold for approval. In addition, the degree of side-effects that are tolerable vary according to the severity of the disease being treated and the degree of efficacy that is observed. As a result, there are no a priori certainties when it comes to the drug approval process.


But until relatively recently, there was an assumption that the mission of the regulators aligned them with the patients they were created to protect. The battle they were adjudicating lay between companies who would benefit from selling a drug even it didn’t work very well and the patients who would be harmed by ineffective or unsafe medicines.


Today, though, that balance seems to have shifted. And the seismic change has come from “activist” patient groups, who seem more concerned that regulators are denying them access to potential medicines than that companies may be trying to polish up ineffective agents for the purpose of profit. Listing to the DMD advisory committee sessions, one could be forgiven for thinking that the battle lay between patients and the white-knight companies on the one side and the beastly regulator on the other.

Such a position seems all the more ironic, given that it comes at a time when the trust of the wider public in pharmaceutical companies has never been lower. The public at large (except, it seems, those with a serious medical condition and their families) view pharmaceutical companies as capitalist profit machines exploiting the monopolies created by both patents and regulators to charge unjustifiable prices for often marginally effective potions and pills – a position that has more than a grain of truth to it, as DrugBaron has discussed many times previously.


Patients and their families, though, clearly have their priorities skewed in a different direction – again, …


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