Thread by @awgaffney

This article argues that the FDA's approval of Biogen's new expensive Alzheimer's drug could "mark a seismic change in how Medicare thinks about covering new drugs of marginal effectiveness."

But that's not what I want to see come out of this.

Thread.

theatlantic.com/ideas/archive/…

If the FDA approves a drug, and insurers (like Medicare) deny it for whatever reason, the public will feel that a useful therapeutic is being denied to them. Rich people, of course, will still be able to purchase the drug.

Now you may say: OK, but you said this new Alzheimer's drug may be useless, so what's the problem? Well, there are many.

First, desperate families/patients who do whatever they can to purchase it out-of-pocket may be harmed by the ineffective drug.

washingtonpost.com/outlook/2021/0…

Second, even if Medicare or Medicaid claim that they are declining coverage due to lack of evidence & not cost, the public will see this is as rationing - after all, the FDA itself said the drug works! This undermines solidarity -the wealthy get drugs that others cannot

Third, there will also be very expensive drugs that have, say, some very modest but not non-existent benefit. If public payers begin to decline coverage on basis of cost, perceptions of rationing of useful therapies will indeed be well grounded!

This is why it should be the FDA, and not payers, that make determinations of efficacy.

Our focus must be on INCREASING the regulatory standards used by the FDA for drug approval. Yet these standards have been progressively WEAKENED over past 20 years. What does that involve?

1. Drugs need to improve hard clinical outcomes, not just "surrogate" outcomes (e.g. lab tests) that mean nothing to patients on their own.
2. We need to vastly reduce the use of the various expedited approval pathways that require less rigorous evidence.

3. New drugs should be tried against appropriate comparators, and generally should have to show superiority over existing agents
4. End pharmaceutical company funding of the FDA ("user fees" started in the 90's)
5. Optimally, we should be moving to publicly-funded clinical trials

Approved drugs should then be covered by payers & Medicare (optimally, by Medicare for All system😉), which should use various policy levers (negotiations etc.) to regulate prices to ensure they are affordable for society-at-large. The drugs should then be made free to patients

But apart from all that, the problem here is that FDA standards have become increasingly weaker over time. We need to move towards greater rigor, yet recent actions - including Obama's 21st Century Cure's Act - have taken us in the opposite direction.

newrepublic.com/article/139328…