Thread by @JorgeCondeBio

1/ Recently $NVS announced a $2.1m gene therapy, the most expensive medicine in history. Gene therapies are potentially curative. How should we price them? How will we pay for them? Could a cure be considered a pre-existing condition? 🧬 💊
a16z.com/2019/05/30/cur…

2/ Rare disease therapies are expensive because: i) they treat devastating diseases, ii) patient pops are small, so biopharma recoups R&D investment w/ high price, iii) payors tolerate because limited risk of many patients in a given plan + limited # of new therapies to cover.

3/ Individually, rare diseases (by definition) affect few people; collectively, 30m Americans (1 in 10) suffer from one of 7000 rare diseases. There are now lots of gene therapies on the horizon for lots of rare diseases; many >$1m therapies in our future. How will we pay?
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4/ Unlike traditional therapies, gene tx has potential to be one-time cure. So gene tx cost is >$1m upfront vs $10-100k/yr for traditional tx. Even if gene tx price is justified by patient benefit, our system is not well-equipped for upfront investment of >$1m cures.

5/ I chatted w/ @MIT economist @AndrewWLo about his intriguing idea to create “mortgages for medicines” to match the timing of payments to the long-term benefit of therapies (we also chat about drug R&D risk and how markets act like biological systems)
a16z.com/2019/05/30/and…

6/ Assuming your insurance provider makes your gene mortgage payments, what happens when you switch your plan or get a new job? Is your new insurer or employer obligated to continue making those payments? If not, are you liable for that debt?

7/ Gene tx is one-time because the new gene incorporates into and lives in cells, replacing the diseased gene. If you receive gene tx, is your new gene (and it’s associated mortgage cost) a pre-existing condition? If so, Obamacare could require your new insurance to cover it...