[0] https://blogs.sciencemag.org/pipeline/archives/2021/06/08/th...
[1] https://blogs.sciencemag.org/pipeline/archives/2021/06/24/op...
[0] https://blogs.sciencemag.org/pipeline/archives/2021/06/08/th...
[1] https://blogs.sciencemag.org/pipeline/archives/2021/06/24/op...
From January 2020 (17 months before aduhelm approved):
https://www.npr.org/sections/health-shots/2020/01/14/7962270...
FDA Approves Drugs Faster Than Ever But Relies On Weaker Evidence, Researchers Find
> Almost half of recent new drug approvals were based on only one pivotal clinical trial instead of the two or more that used to be the norm, according to the study published Tuesday in JAMA, the journal of the American Medical Association. And the reliance on surrogate measures — stand-ins for presumed patient benefits — has increased. In the case of cancer drugs, a surrogate measure could be shrinkage of tumors instead of improvements in survival after treatment.
> …Drugmakers also began paying the FDA fees to fund the review process after AIDS activists protested the agency's sluggishness in the 1980s… "There is some concern about the incentives that this is created within the FDA," Darrow says. "And whether it has created a culture in the FDA where the primary client is no longer viewed as the patient, but as the industry."
["surrogate measures" is a complaint with aduhelm approval, when they say there was no "evidence showing the drug slowed the disease", they mean the approval was based only on surrogate measures.]
Surrogate measures are a trade off that we as a society have decided to make to reduce regulatory burden and accelerate brining new therapies to market. Maybe you disagree with it, but someone with a terrible disease waiting on a new life saving treatment might see the issue differently. In some cases it is clear that the surrogate is a good endpoint. In others maybe less so. Regulators have the tough job of balancing the risks, benefits, and science when making the call on whether to accept one.
Generally FDA is doing many things to streamline and accelerate the regulatory process. This is good. We want them to innovate and take advantage of new scientific knowledge and technological breakthroughs.
Finally I would say that the real precedent for this was the decision for the Duchenne Muscular Dystrophy drug (also happened on Woodcock’s watch). I kind of had this sinking feeling this was about to happen when I saw that she was acting commissioner. Say what you will about Trump, but Gottleib was a damn good appointment - at least until he got fired for going after vaping. Azar was a pussy and basically an empty suit.
I am curious. How much of the society was involved in making this decision. We can all agree that there is enormous financial pressure from the industry to make such decisions go in its favour, and virtually none to go in favor of the public.
So isn't there a chance that eventually such trade offs will go in a direction that is harmful to the public?
>Maybe you disagree with it, but someone with a terrible disease waiting on a new life saving treatment might see the issue differently.
Sure, but we don't do things this way. You can always find someone who benefits from a community wide applied bad idea. Does not justify the bad idea sadly.
This kind of short term, localised thinking might allow the regulatory process to bend heavily under the aforementioned financial pressures, in favour of the industry, and result in great public harm that might go unnoticed for a very long time..
As far as patient advocates, as many pieces point out, one of the historical things was AIDS activism for availability of drugs. I was struck by this from a recent piece about ACT UP, from one of the activists at that time:
«Today, Franke-Ruta is a journalist, and she spoke to Schulman about the wider implications of some of act up’s success. “I don’t think that we realized at the time that this was part of the broader gutting of the FDA that we’ve seen since; that there was a lot of political agendas that we just happened to be in sync,” she said. After President Donald Trump touted the promise of hydroxychloroquine as a treatment for covid-19, the F.D.A. issued an emergency-use authorization for the drug, which was shown to be useless for the purpose. (The agency withdrew the authorization three months later.) “Sometimes it seems like it’s gone too far in the other direction,” Franke-Ruta went on. “But there’s a really strong pharmaceutical lobby against the FDA as well that I don’t think we were aware of.”»
And from the article's author, editorializing a bit:
«In retrospect, one can ask whether act up’s victories on the research front pushed the F.D.A. too far. The drugs-into-bodies approach to fast-tracking—the use of “surrogate endpoints” (like T-cell counts or viral load), for example, rather than clinical benefits in actual people—can be valuable, especially for patients who are facing death and have no good alternatives. This approach at least offers desperate patients a chance, while allowing scientists to gather meaningful data. But, today, a number of drugs, for everything from asthma to periodontitis, have won approval before benefits in human patients were established, and critics argue that drug approval is too often based solely on benefits shown in biomarkers rather than in bodies. “Right to try” legislation, meanwhile, enables the sale of drug candidates without even involving the F.D.A. When restrictions are weakened, experimental drugs—many of which end up proving useless or worse—become harder to distinguish from effective medicine.»
https://www.newyorker.com/magazine/2021/06/14/how-act-up-cha...
I disagree. If you have media in your team, this is easy, given some time. Particularly in current times when you have a treasure trove of possible distractions that you can make the media throw at a gullible public.
If one looks closely, then I think CDC/FDA etc have lost credibility a long time back. The real events portrayed in documentaries like "What lies upstream" and movies like "Dark Waters" should be enough to reject these agencies outright.
What is enormously surprising to me is that, not only we consider them very credible, but are also willing to consider their word as unfailable and final in many cases.
Even more sad thing is that the power of CDC/FDA extends even to many developing countries, so this affects much more than their home country.
It's just a column.
But that's not to say the reactions from journalists weren't critical. Adam from Stat had some choice words about the decision.
The point is, it doesn't seem like a useful distinction.
If the other drug researchers are sticking necks out to say this is bullshit, it's pretty like that ... this is bullshit. The situation he describes that all the other companies had already failed to their useless drugs approved. Now that one useless drug slips through, all the others are going try and do that.