Dear HNers,<p>You may have read this article:<p>https://www.cnn.com/2025/02/17/health/zolgensma-sma-gene-therapy-drug-pricing-propublica/index.html<p>To over-simplify the article, it's about a non-profit that financed pre-clinical studies on a genetic disease that strikes infants: SMA.<p>https://en.wikipedia.org/wiki/Spinal_muscular_atrophy<p>Once the pre-clinical studies were finished, contacts with the non-profit were much less frequent, and in the end, the drug was approved by the FDA. The price is more than $2 million per dose.<p>This drug, Zolgensma, involves administering an AAV9 virus capsid containing an SMN1 transgene. Today, commercial companies have mastered this technology, are very efficient, and can produce it at little cost.<p>I wonder if there could be a collaborative effort to lower the price of such drugs. One way would be to find alternate providers but a main roadblock is the intellectual property.<p>The first patents on this drug will expire in a few years (they were written in the mid-2000).<p>I know it's possible to make three kinds of attacks on PI:<p>- One would be to find an alternative PI not related to the original patents, but it requires pre-clinical studies. The competition of the patent holder would be pleased to finance such pre-clinical studies.<p>Another option is to find prior art to invalidate the patent. This seems weird, as there should have been at least prior art research before accepting a patent, yet it's feasible. I had the honor of participating in one.<p>- Then it's possible to evade a PI because the system of claims has a weak point. For example, let's say the administration of the drug is part of the claims, and that several claims depend on that particular. Then it would be trivial to create another patent with the same efficacy but with another administration route. Indeed this is only an example to make this last point clear, I don't claim it has some validity.<p>I assume there is no need to sue the patent holder as the mere existence of such a website would lower prices.<p>What do you think of the idea of creating a sort of website where a challenge will be presented: Find a strategy to enable lowering the price on this or that drug, and contributors could discuss and propose solutions. It would be a sort of GitHub project.<p>Thanks for your comments!
The cost isn't justified by the cost of producing the dose, it's justified by the cost of testing a drug like that divided by the small number of recipients. Other gene therapies for rare diseases are priced accordingly, insurance companies will pay because they a worth a lot.
If you can successfully challenge pharma patents you can get rich by shorting the stock. A hedge fund manager named Kyle Bass tried this with a couple dozen drugs to varying degrees of success. But also, a patent being invalidated doesn't mean prices come down immediately. ANDAs still take 3-4 years to get approved on average.