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As mentioned in the article, a prominent cost-effectiveness research group, ICER, suggests that the treatment may be cost effective at prices of up to $1.5M, bu
by aaavl2821 8y ago
As mentioned in the article, a prominent cost-effectiveness research group, ICER, suggests that the treatment may be cost effective at prices of up to $1.5M, but with a different set of the assumptions, the cost-effectiveness threshold could be as low as $310K.
Other studies suggest CAR-T drugs are cost-effective at current prices, but they are not widely covered by payers. In these cases, it is less clear whether drug companies or payers are to blame for access to treatment
The article also does not mention what the gene therapy in question actually does. In small studies, it has essentially cured a disease that would otherwise kill babies before they are two. It's a pretty amazing product. And there are many companies hot on their heels, some of which are developing small molecule drugs -- much cheaper to develop and manufacture than gene therapy -- that actually seem to be potentially nearly as effective
The detailed ICER review is a good read if you're looking to understand how drugs are valued: https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Final_Evidence_Report_040319.pdf https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_...
- celinehh94 8y agoQuick interesting note on this front - ICER is a US non-profit, but has basically zero influence in the actual pricing of drugs. It's actually illegal to value drugs this way in the US
- celinehh94 8y agoSorry - should say use the valuation of the drug to determine patient access to it in the US. AKA, ICER could determine that drug 1 is massively un-cost-effective in comparison to drug 2, but you cannot restrict access to drug 1 due to this information. Most other countries tie drug price somewhat to health gain delivered by said drug.
- refurb 8y agoI wouldn't say it has zero influence. Their assessments definitely impact the public perception of pricing in the US, so companies pay attention. But yes, it's not like NICE in the UK where the decisions have a direct impact on how much insurers pay for drugs.