This led me to wonder how expensive the IVs are to justify $3.5M per dose (and I'm guessing it's not a cure, just a "treatment") and how long this drug lasts. Found this -...
Currently, patients receive frequent, expensive IVs of a protein that helps blood clot and prevent bleeding.
This led me to wonder how expensive the IVs are to justify $3.5M per dose (and I'm guessing it's not a cure, just a "treatment") and how long this drug lasts. Found this - https://www.cslbehring.com/newsroom/2022/fda-hemgenix - which says:
reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion.
So "years" but not an actual number of years expected. That link later mentions study results at the 24 month point, but nothing past that. The prescribing information packet has levels to look for at a 2 year follow up appointment, but nothing after that. Just mentions of 5 year follow ups in the study to examine any adverse reactions, but not levels. So it's pretty much $3.5M every two years, although the data they used for FDA approval was only for efficacy up to 18 months.
Drugmaker CSL Behring, based in Pennsylvania, announced the $3.5 million price tag shortly after the FDA approval, saying its drug would ultimately reduce health care costs because patients would have fewer bleeding incidents and need fewer clotting treatments.
Ah, so not based on the development costs, risk, or anything like that. Just "eh, if it works perfectly for the patient it'll be a few bucks cheaper than the current treatment based on our inflated estimates of treatment and hospital visits for the worst cases."
Like most medicines in the U.S., most of the cost of the new treatment will be paid by insurers, not patients, including private plans and government programs.
CBS needs to do a little fact checking on how insurers get their money...
Hemophilia B is a rare, lifelong bleeding disorder caused by a single gene defect, resulting in insufficient production of factor IX, a protein primarily produced by the liver that helps blood clots form.
This led me to wonder how expensive the IVs are to justify $3.5M per dose (and I'm guessing it's not a cure, just a "treatment") and how long this drug lasts. Found this - https://www.cslbehring.com/newsroom/2022/fda-hemgenix - which says:
So "years" but not an actual number of years expected. That link later mentions study results at the 24 month point, but nothing past that. The prescribing information packet has levels to look for at a 2 year follow up appointment, but nothing after that. Just mentions of 5 year follow ups in the study to examine any adverse reactions, but not levels. So it's pretty much $3.5M every two years, although the data they used for FDA approval was only for efficacy up to 18 months.
Ah, so not based on the development costs, risk, or anything like that. Just "eh, if it works perfectly for the patient it'll be a few bucks cheaper than the current treatment based on our inflated estimates of treatment and hospital visits for the worst cases."
CBS needs to do a little fact checking on how insurers get their money...
Come on CRISPR (pie in the sky, I know)!