[skadamou]

This is an excellent response and you clearly understand the issue well. I'm curious though, what do you think about the long term effects of continuing to practice gene therapy? Sure, the cost for treating the first few patients will be exorbitant but wouldn't we expect researchers to find ways to bring costs down over time? How will we know if this is even possible if we abandon gene therapy at such an early stage? It might not make market sense to produce these therapies right now but I think there is a strong argument for governments to fund this type of research.

[JunkDNA]

There’s a bigger issue than gene therapy. Precision therapies that work really well for a very small number of people are going to become increasingly common (whether gene based or otherwise). We are seeing this in cancer right now where there are finally true cures emerging. It’s clear current pricing models are not going to work in a world where you need lots of different therapies to treat small clusters of patients.

Outside of cancer (the only real place where there is a broad clinical application of precision medicine at scale) there’s an estimated 25-30 million people in the US with a rare disease (a disease that affects less than 200,000 people) [1]. There are an estimated 7,000 different rare diseases. That makes rare diseasss more common than cancer in the us which is at about 15 million people [2].

So what if we end up with cancer itself being subdivided into thousands of small diseases (something that is already well underway) in addition to all these “rare” diseases? The current model of developing therapies is just not going to cut it. We need new development methods that are not so cost-intensive and we need new payment models that create enough incentive that individuals and companies take enough risk to identify therapies. It’s a ferociously hard problem.

[1] https://rarediseases.info.nih.gov/diseases/pages/31/faqs-abo...

[2] https://seer.cancer.gov/statfacts/html/all.html

[rayiner]

I don't know enough about gene therapy really say what could be improved. If there are common aspects of the process that could be "factored out" I think it would make sense for governments to research that and help streamline approval that way. But that's looking at it from a programming point of view--I don't know enough about the biology to know whether that makes sense.

[maxander]

The inherent cost of manufacturing the drug is relatively negligible. The problem is the cost of development and deployment, and the tiny customer (patient) population to spread that cost around to.

A gene therapy for a more prevalent disorder would not have the same problems, or at least not as badly.

[ekianjo]

No, its only negligible for small molecules. Making proteins/biologics is much more expensive, and this is where many new drugs are headed. Plus the investment required to design the process and build facilities is far from negligible either.

[maxander]

Relatively negligible. I don’t know what the precise price point for biologics are, but equivalent techniques are routinely used in research; a bit of Googling found a lab services page pricing a an AAV (the kind of vector used in Glybera) batch at the neighborhood of $1k [1]. Would be much more for a medical-grade preparation, I’m sure, but not an appreciable fraction of a million.

[1] https://sites.duke.edu/dvvc/services/adeno-associated-vector...