Posted on 12/23/2012 9:17:26 PM PST by Seizethecarp
A drug for chronic fatigue syndrome that spent decades in clinical development and won fervent patient support has been turned down for approval by a committee of advisers to the US Food and Drug Administration (FDA) who voted 9–4 against it. The drug, named Ampligen (rintatolimod), has not been shown to be effective or safe, the committee determined on 20 December.
“Key deficiencies included inadequate evidence of effectiveness or safety, inadequacy of drug–drug interaction studies, lack of carcinogenicity assessment, lack of anti-drug antibody determination, and inadequate analytical methods and drug product specifications,” the agency wrote in one of several sections listing problems associated with the application.
FDA representatives also expressed concern over inconsistencies in the data and statistical analyses. An agency representative says of the data: “It raises our eyebrows and makes us wonder what else is in the database that we’re not seeing.”
Alaine Perry, who served as a patient representative on the advisory committee, acknowledged that the Ampligen data are slim, but noted that CFS symptoms can be so severe that some patients would willingly take on a significant mortality risk for the promise of even minor relief of their symptoms. “A very small improvement in a disease like this is life altering,” she said. There are no other approved treatments specifically for CFS.
(Excerpt) Read more at blogs.nature.com ...
There were some patients paying $25,000 per year for twice weekly infusions of this drug who testified to the committee that Ampligen helped them greatly, but this could not be widely replicated for the broader population of ME/CFS patients in the small studies that were done.
Ping.
Those are some pretty large deficiencies. That paragraph suggests that large-scale clinical studies were not done, but they were trying to push the drug through to approval anyway? Gee...
They thought approval was certain when the control group made it through an entire hour of PBS broadcasting without falling asleep.
I agree with your assessment.
The problem seems to be that some people with CFS are helped by Ampligen, but they don’t yet know why they are while most others are not. That is not a strong enough result for an FDA approval, but Ampligen did get 4 votes out of 15, so it wasn’t a total washout.
Some studies indicate that there are as many as five sub-categories of illness lumped together as CFS and some might be more responsive to Ampligen than others, but the studies haven’t been done. At $25,000 per year for the drug it takes big bucks to fund a study big enough to persuade the FDA, if successful.
What, does the FDA demand less expenditure on studies if the marketer of the drug only plans to charge a low price for it?
(That’s what your comment sounds like.)
My Mom was afflicted by CFS this past year and we all thought she was a goner as she was sleeping 20 hrs/day. Her recovery was remarkable but took 3 months of Famvir before she started to perk-up.
Syndromes are called that due to no one specific etiology. Treatment for "syndromes" are more difficult to assess for efficacy due to the multiple etiologies. For CFS the antibodies to the viruses are all over the place and symptoms can improve with titers remaining high.
Not aware of the clinical studies with Ampligen but it is hard to imagine how they did not collect the data needed for FDA review.
Funding is an issue for all “orphan” diseases and syndromes.
The FDA committee makes a “cost versus benefit” decision implicitly and/or explicitly with all of its decisions.
AIDS activists, notably the recently deceased activist Spencer Cox, made their name by beating up FDA to waive the normal rules and approve half-baked drugs given the high death rates back in the 1980S.
CFS activists have been trying the same argument with the FDA based on the severe debilitation of CFS, in some cases leading to suicide. “A marginal drug is better than no drug” is the argument.
If FDA approved Ampligen, the hard-pressed US health “insurance” system, especially under Obamacare, might be required to reimburse the $25,000/year Ampligan treatment, as it has with expensive MS infusion drugs, for example. Cost IS a consideration in drug approvals.
In the case of the MS infusion drugs, the benefits were much clearer and the patients that would benefit could be identified with greater accuracy than CFS (no accuracy actually, in CFS it seems). So Ampligen failed both the cost and benefit sides of the cost/benefit analysis, except for the four members of the panel who still thought it did!
Why this intriguing if ill-proven medication would need that sort of expense to crank out of the labs, when it had been discovered in the sixties, is unknown to me. Not all orphan drugs do. The manufacturer is going to want something to recoup their troubles, of course. Whether that part spells the lion’s share of the quoted $25 grand, I do not know. That doesn’t necessarily excuse a poor test, because issues such as the majority of the FDA commissioners alleged are not esoteric things to anyone versed in the science.
I’ve long had a quasi-libertarian view on medicaments of any sort. There could be two classes of medications, that which has been officially blessed by the bureaucrats, and that which has not been. If I were king, the second class could still be administered by doctors, as long as they participated in a sufficiently large liability insurance plan against harm caused by what they administered. Market economics, rather than politics, would then dictate what other drugs would be tolerated in the society.
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