Prices are US wholesale acquisition cost (WAC) from FDA approval announcements and manufacturer press releases. Net prices after rebates are typically 20-25% lower. Lifetime costs from peer-reviewed literature:
* Hemophilia B lifetime: $20-23M, midpoint $21.5M used in chart (PubMed PMID 33591884, adult payer perspective)
* SMA/Spinraza: $750K first year, $375K/yr ongoing. $4.7M figure reflects ~11 years of treatment (BMC Cost Effectiveness and Resource Allocation, 2020)
* Sickle cell: $1.6-4.2M depending on severity (Blood Advances, ASH 2022)
Lifetime comparison bars only shown where solid peer-reviewed data exists. For Lenmeldy (MLD) and Elevidys (Duchenne), I couldn’t find reliable lifetime cost estimates in the literature, so those only show the gene therapy price.
Tool: HTML/CSS, screenshotted at 3x
Meet-me-behind-bins on
I’ve read that there’s still a bit of an issue with the uptake and prescription of a lot of these drugs. It seems the economic argument is undeniable and yet they’re not being widely rolled out? I wonder if there’s lobbying and public relations campaigns from pharmaceutical companies that produce prophylaxis treatments that are wildly profitable? I get the district feeling that the last thing a lot of pharmaceutical companies want is to cure diseases. Why have a $4 million one off when you can have a $25 million lifetime customer?
ItsChappyUT on
Medication names are out of control.
ObjectiveAside3266 on
I would love to see a full cost calculation without marketing and profit for those with the assumption that 70% of applicable cases get treatment over a period of 15 years (to recover research)
![[OC] The world's most expensive single-dose medications vs. the lifetime cost of treating the same condition without them](https://www.byteseu.com/wp-content/uploads/2026/04/ac7xa9934xvg1-734x1536.png "[OC] The world’s most expensive single-dose medications vs. the lifetime cost of treating the same condition without them")
4 Comments
Prices are US wholesale acquisition cost (WAC) from FDA approval announcements and manufacturer press releases. Net prices after rebates are typically 20-25% lower. Lifetime costs from peer-reviewed literature:
* Hemophilia B lifetime: $20-23M, midpoint $21.5M used in chart (PubMed PMID 33591884, adult payer perspective)
* SMA/Spinraza: $750K first year, $375K/yr ongoing. $4.7M figure reflects ~11 years of treatment (BMC Cost Effectiveness and Resource Allocation, 2020)
* Sickle cell: $1.6-4.2M depending on severity (Blood Advances, ASH 2022)
Lifetime comparison bars only shown where solid peer-reviewed data exists. For Lenmeldy (MLD) and Elevidys (Duchenne), I couldn’t find reliable lifetime cost estimates in the literature, so those only show the gene therapy price.
Tool: HTML/CSS, screenshotted at 3x
I’ve read that there’s still a bit of an issue with the uptake and prescription of a lot of these drugs. It seems the economic argument is undeniable and yet they’re not being widely rolled out? I wonder if there’s lobbying and public relations campaigns from pharmaceutical companies that produce prophylaxis treatments that are wildly profitable? I get the district feeling that the last thing a lot of pharmaceutical companies want is to cure diseases. Why have a $4 million one off when you can have a $25 million lifetime customer?
Medication names are out of control.
I would love to see a full cost calculation without marketing and profit for those with the assumption that 70% of applicable cases get treatment over a period of 15 years (to recover research)