FDA approves cure for sickle cell disease

[mollyanna58]

Enormously expensive, but hopefully that will change in time.

www.msn.com/en-us/health/other/fda-approves-cure-for-sickle-cell-disease-the-first-treatment-to-use-crispr/ar-AA1ld7zF?ocid=entnewsntp&cvid=ef872af3c64646e5bdf4b83825ac30ae&ei=22


The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the majority of whom are Black.



The therapy, called Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine to be approved in the United States that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in chemistry in 2020.

[chiver78]

this is fantastic news. I read an expanded article in last Sunday's paper about this (Vertex is based in downtown Boston) which interviewed a young woman that was part of the clinical trials. it's likely behind a paywall online, but i can post a linking anyone's interested.

[Spellbound454]

Wow thats terrific, bit expensive at the moment but now that they know there is a treatment which works, other people will get in on the act.

Its a horrible painful disease which effects a lot of people.

[djAdvocate]

i am not seeing where the cost is listed.

what is it?

[mollyanna58]

Dec 9, 2023 22:45:52 GMT -5 djAdvocate said:

i am not seeing where the cost is listed.

what is it?

$2.2million per patient.

[chiver78]

here's that link I mentioned. please lmk if it's behind a paywall. I can post it to PM for anyone that's interested if need be. 

link

[djAdvocate]

Dec 10, 2023 9:43:07 GMT -5 mollyanna58 said:

Dec 9, 2023 22:45:52 GMT -5 djAdvocate said:

i am not seeing where the cost is listed.

what is it?

$2.2million per patient.

um...what?  holy crap!

[mollyanna58]

New study reveals that people with sickle cell disease spend roughly $1.7 million on total lifetime medical costs

(WASHINGTON, May 16, 2022) – In a study published today in Blood Advances, researchers found that privately insured individuals with sickle cell disease (SCD) spend approximately $1.7 million on disease-related medical expenses over their lifetime. These findings highlight the enormous economic burden affecting individuals living with SCD and raise questions about how medical advancements, like gene therapy, may one day factor into the cost of care.

www.hematology.org/newsroom/press-releases/2022/the-cost-of-living-with-sickle-cell-disease#:~:text=%E2%80%9COur%20findings%20suggest%20that%20the%20lifetime%20out-of-pocket%20cost,%28CHOICE%29%20Institute%2C%20Department%20of%20Pharmacy%2C%20University%20of%20Washington.


So, $2.2million vs $1.7million, as well as loss of productivity and other costs relating to long term medical conditions.

[jerseygirl]

Exactly I worked on orphan drugs and usually price was set by taking into consideration the medical costs of the disease. If treatment decreased the medical costs then price of drug reflected the decreased costs.
Also worked on orphan drug that treated a condition that caused huge amounts for hospitalization but also impacted ability to work. Cost of drug took all of that into consideration for pricing. Company worked with charity for the condition and helped pay most of drug costs for patients. Drug Changed their lives