One of the latest fiascos is about Huntington's Disease. If you're an Olde Folkie, you surely know of Arlo Guthrie and "Alice's Restaurant" who was exempted from Vietnam because of the littering offense told in the song. Bet he didn't know that would happen. His father Woody, had (what was then called) Huntington's Chorea. Woody died in 1967. It is progressive and fatal and is yet without therapy for the disease directly (some symptom therapies exist). Two of Woody's daughters died of it. His mother died of it, from whom he inherited the disease.
It is classified as a Rare Disease, and FDA, at least titularly, has recently loosened the requirements on therapies for Rare Disease. uniQure is a drug company in the Netherlands and previously in Lexington, MA which has been developing a gene therapy. They recently published the results of a trial of the therapy, and claimed that the therapy reduced disease progression by 75% after 3 years.
Enthusiasm reigned and the stock price soared. Then FDA went on the warpath against both the therapy and the company. Now, the WSJ has a report, anonymously sourced, that FDA calls uniQure liars and the therapy worthless. Being the WSJ, one must be subscribed to read it up.
Federal Health Officials Attack Rare-Disease Drug, Say Company LiedFortunately, Fierce Biotech has a report which covers the main points of the battle betwixt uniQure and FDA.
The sticking point is simple: uniQure claims it has, in writing, approval for the trial while this FDA source claims that it never gave its approval and requires a (double?) blind, placebo controlled study for approval. Even before the recent 'loosening' of Rare Disease study requirements, given the small patient population typical of Rare Disease, running such trials is difficult; small patient pool and reluctance to take the chance of NOT getting the therapy. Moreover, in fatal diseases with no other therapy, is it ethical to demand standard structure trials? Mayhaps not.
The Fierce Biotech report mentions other Rare Disease
"Moreover, FDA, as a general rule, never makes such assurances. FDA will always say, 'Well, we have to see the data when we get it.'"And that's, at best, a half truth. There are discussions with FDA about trial design as a matter of course. FDA has a separate, formal approach, called a Special Protocol Assessment. It's a semi-fixed agreement on the conduct of a trial, however FDA has been known to toss out an SPA if they change their mind.
As I was typing this out, the wonderful Adam Feuerstein made (a free!!) account of the fiasco available at STAT. Enjoy.
