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Why are gene therapies so expensive? Is it simply the need to recoup high R&D costs across a smaller "market" of patients with rare diseases? Or is the marginal per-patient cost actually higher than a regular pharmaceutical?

These new gene-/immuno-therapies tend to be:

1) much more sophisticated/cutting edge in terms of R&D,

2) the R&D is all of a new kind, so there has been little prior amortization of resources from prior successes.

3) _curative_, so from a purely market perspective a XX% discount on a [cost/year * lifetime] relative to a small-molecule drug that needs to be taken regularly, forever, is in these very high ranges, and still economically appealing from a cold hard actuarial perspective.

The high prices also attract advanced technology development in the area, and I think society wants & demands that rapid technological development of these powerful tools. Imagine trying to pay competitive salaries to a large number of sophisticated genetic engineers and all of their tools and equipment, when there are literally only a five or six products on the market with which to support their efforts.

It is still very unclear how we as a society should be paying for that development. And placing that burden on (those) individuals seems unreasonable.

Avexis, the company that developed this drug before Novartis bought it, spent ~$700M to get it approved. The drug will treat a few hundred patients per year. So they have to charge a high price

zolgensma is the second gene therapy ever approved in the US. The technical risks are very high and making them at scale at a high level of quality required rare and expensive expertisr

i think the article says it s the former. engineering a virus is not that expensive

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