FDA ruins every Neurologists holiday weekend by approving Gene Therapy for the #1 genetic killer of infants

[Jwheel86]

https://www.nbcnews.com/health/health-news/2-1-million-drug-treat-rare-genetic-disease-approved-fda-n1009956

 

Zolgensma is a one time (supposedly) $2.1 million IV infusion for children under 2 years old with Spinal Muscular Atrophy. 

[Spawn_of_Apathy]

that will not be covered by any insurance company. So good luck coming up with $1.2million if your kid has it. 

[Jwheel86]

28 minutes ago, Spawn_of_Apathy said:

that will not be covered by any insurance company. So good luck coming up with $1.2million if your kid has it. 

It's cheaper than it's competitor Spinraza, which costs $125k a dose every 4 months for life, that's after 4 loading doses in the first 4 months. 

[CayceG]

Eugenics didn't go away after the 30s. We just reconfigured medical commerce to support eugenics through medicine costs. 

[TheGreatGamble]

3 hours ago, CayceG said:

Eugenics didn't go away after the 30s. We just reconfigured medical commerce to support eugenics through medicine costs. 

Are you seriously against getting rid of genetic disease? Seems ridiculous to want people to lead worse lives because you are scared of the implications of what else might happen with it. Gene editing will obviously need limitations and laws, but I can't think of a more important science for the future of humanity. 

[TyphoidHater]

He is saying the outrageous costs being charged by insurance companies, and private insurance's reluctance to pay them, is akin to a sort of present day eugenics.

[TheGreatGamble]

New treatments are expensive. If the medical industry didn't profit, there would be no medical industry. This stuff is bound to be prohibitively expensive for many years. Its the nature of medicine. Research needs to paid. These companies don't work for nothing, new treatments cost billions. 

 

 

[Jwheel86]

5 hours ago, TheGreatGamble said:

Are you seriously against getting rid of genetic disease? Seems ridiculous to want people to lead worse lives because you are scared of the implications of what else might happen with it. Gene editing will obviously need limitations and laws, but I can't think of a more important science for the future of humanity. 

 

Minor correction, this is not gene editing. It uses a virus to insert an artificial strand of DNA into your cells (specifically the missing SMN1 gene) which acts independently from your DNA creating RNA and then SMN Protein (which we SMA patients are not getting enough of) for the life of the cell, it does not carry over to new cells when it divides. 

 

The interesting thing isn't the price tag, it's actually significantly cheaper than what we were bracing for ($5m - $8m), it's the pricing model the CEO wants to implement in light of Zolgensma and other one and done treatments. Basically what it seems like he's advocating for is a system where the drug price is based off the cost of an untreated SMA patient and any SMA related care costs would result in Novartis refunding part of the drug cost. Not sure how I feel about that or if it even work since the bulk of SMA care is not covered by private insurance but Medicaid. 

[CayceG]

9 hours ago, TheGreatGamble said:

Are you seriously against getting rid of genetic disease? Seems ridiculous to want people to lead worse lives because you are scared of the implications of what else might happen with it. Gene editing will obviously need limitations and laws, but I can't think of a more important science for the future of humanity. 

 

Hey dork. 

 

I'm saying this is way too fucking expensive for the people who need it to have easy access to it. These drugs being priced this way are depriving people of care and keeping them from living longer, fuller lives, killing them early, and culling the poor sick out of the population. 

 

8 hours ago, TheGreatGamble said:

New treatments are expensive. If the medical industry didn't profit, there would be no medical industry. This stuff is bound to be prohibitively expensive for many years. Its the nature of medicine. Research needs to paid. These companies don't work for nothing, new treatments cost billions. 

 

 

 

"Rich people deserve to be healthy because they have money."

[SimpleG]

https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Final_Evidence_Report_040319.pdf

 

Manufacturers
5. To align reasonably with the benefits for patients and families, the price for Spinraza
should be far lower, and that for Zolgensma should be lower than the hypothetical $4-5
million price the manufacturer has suggested could be justified. To achieve the needed
balance between incentives for innovation and health system affordability, all manufacturers
should exercise their monopoly pricing power responsibly, setting prices that do not exceed a
reasonable cost-effectiveness threshold.

 

 

The price of innovative treatments for SMA should better align with the demonstrated benefits for
patients. The New England CEPAC acknowledge the remarkable effectiveness and many additional
potential benefits and contextual considerations of Spinraza and Zolgensma; nevertheless, the 

panel voted 12-0 that Spinraza represented low long-term value for money due to its high price. It
is possible for a high-cost treatment to demonstrate good cost-effectiveness in a life-threatening
rare condition (e.g., Kymriah, a CAR-T cell therapy for B-cell acute lymphoblastic leukemia). The US
health care system cannot sustain paying prices far above traditional cost-effectiveness levels for
the growing tide of treatments for ultra-rare disorders

[Jwheel86]

43 minutes ago, SimpleG said:

https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Final_Evidence_Report_040319.pdf

 

Manufacturers
5. To align reasonably with the benefits for patients and families, the price for Spinraza
should be far lower, and that for Zolgensma should be lower than the hypothetical $4-5
million price the manufacturer has suggested could be justified. To achieve the needed
balance between incentives for innovation and health system affordability, all manufacturers
should exercise their monopoly pricing power responsibly, setting prices that do not exceed a
reasonable cost-effectiveness threshold.

 

 

The price of innovative treatments for SMA should better align with the demonstrated benefits for
patients. The New England CEPAC acknowledge the remarkable effectiveness and many additional
potential benefits and contextual considerations of Spinraza and Zolgensma; nevertheless, the 

panel voted 12-0 that Spinraza represented low long-term value for money due to its high price. It
is possible for a high-cost treatment to demonstrate good cost-effectiveness in a life-threatening
rare condition (e.g., Kymriah, a CAR-T cell therapy for B-cell acute lymphoblastic leukemia). The US
health care system cannot sustain paying prices far above traditional cost-effectiveness levels for
the growing tide of treatments for ultra-rare disorders

The 7 doses of Spinraza I've gotten (5 paid for by Medicaid at $135k each and 2 by BCBS at $250k each) have cost $1,175,000 since April 2018.