The Food and Drug Administration has a approved a new gene therapy from Novartis that rings in at a whopping $2.125 Million for a one-time treatment making it "the most expensive drug on the market."
The drug Zolgensma is the second gene therapy approved for treatment of a genetic disease and consists of an infusion of genetically modified viruses carrying healthy copies of a defective gene that causes spinal muscular atrophy (SMA), which can be fatal within a year or two of birth. The first, approved in 2017 for treatment of a genetic form of blindness, was priced at a comparatively modest $425,000 per eye.
These backbreaking (eye watering?) prices come with their share of controversy
"We have been slowly subjected to price increases the same way the frog in the boiling water is slowly boiled to death," [Peter Bach, MD, MAPP Director of the Memorial Sloan Kettering's Center for Health Policy and Outcomes says.]
Insurers are expected to cover the cost. The company says payment plans will be available.
AveXis president Lennon acknowledges the numbers might seem shocking. But he argues the drug is easily worth it. The only existing treatment for spinal muscular atrophy, a drug called Spinraza, costs hundreds of thousands of dollars a year. Zolgensma hopefully will be a one-time, life-saving treatment.
"We're talking about a lifetime of benefit being condensed down into a one-time treatment," Lennon says. "We're not used to thinking about this that way. We're used to a system of a chronic medication where we spread things out over years if not decades."
The drug is currently in production and will be available for use "shortly"
Side Note: The fable of the frog being slowly boiled alive is doubly inaccurate. Frogs will progressively become more active attempting to escape slowly heating water and if able will exit the 'pot' long before it comes to the point they are in danger. Additionally a frog dropped into boiling water would likely be unable to hop out in time to save itself.
(Score: 1) by khallow on Tuesday May 28 2019, @12:33PM (3 children)
Well, that would be the problem right there, not the hedonism.
(Score: 2) by JoeMerchant on Tuesday May 28 2019, @01:51PM (2 children)
IMO, IP/patent protection worked reasonably well for the cotton gin and maybe the light bulb, but centuries of development - optimizing exploitation of the laws, society, and human nature, have brought about these special cases where the exploitation is orders of magnitude above anything that could be called reasonable. And, sooner or later, these types of legal abuses should be addressed with the creation new (and/or repeal of the dysfunctional) legislation which allows the exploitation to thrive - because human nature and society are unlikely to address the problem on their own.
🌻🌻 [google.com]
(Score: 1) by khallow on Tuesday May 28 2019, @03:23PM (1 child)
Why hasn't it happened already? Because the medical R&D system is barely functioning with vast sums burned for meager results. Trashing IP protection would be another nail in the coffin since it offers a weak guarantee of profit for the immense gambles that are played.
(Score: 2) by JoeMerchant on Tuesday May 28 2019, @05:49PM
The US medical system is so far out of whack there's no way it's going to get fixed without a lot of pain.
Now, to "fix" it, first you have to define what you want a medical system to do. If that includes providing medical care to people in an efficient manner, effectively treating common diseases for (something approaching) the lowest total cost - that's why I would call the current system broken.
Out of control Pharma profits are just one part of the puzzle. IP protection can be a useful component in the R&D framework, but it's also an easy poster child for abusive pricing policies.
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