[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