Cystic Fibrosis Gene Correction Drug Approved by the FDA 264
tguyton writes "The good news: the FDA just approved the distribution of the first drug to treat the underlying cause of Cystic Fibrosis, called Kalydeco by Vertex Pharmaceuticals. The bad news: this drug will only affect 4% of patients with the disease in the U.S. From the article: '[Affected patients] with the so-called G551D mutation have a defective protein that fails to balance the flow of chloride and water across the cell wall, leading to the buildup of internal mucus. The vast majority of cystic fibrosis patients have a different genetic defect, in which the protein does not reach the cell wall. Vertex is developing another drug to try and address that problem. Study data for that drug is expected later this year.' Hopefully the research involved will be applicable to finding treatments for other genetic diseases."
Further bad news: "...executives said Kalydeco would cost $294,000 for a year's supply, placing it among the most expensive prescription drugs sold in the U.S."
Cell MEMBRANE (Score:5, Informative)
Good lord, we are animals not plants. There is no such thing as a "cell wall" in our cells! Call it what it is: the cell membrane.
Pedantic? Yes, but the definitions are precise and are intended to be used precisely. Journalism like this makes me want to gouge my eyes out; a single high-school biology class teaches cell wall vs. cell membrane!
Same thing as VX-770 and Ivacaftor (Score:5, Informative)
Just so no one gets confused, this molecule goes by 3 common names VX-770 and Ivacaftor and Kalydeco
http://en.wikipedia.org/wiki/Kalydeco [wikipedia.org]
There are not three separate drugs for the same problem etc.
Re:That's not such bad news (Score:5, Informative)
http://www.forbes.com/2010/02/19/expensive-drugs-cost-business-healthcare-rare-diseases.html [forbes.com]
Alexion Pharmaceutical's Soliris, at $409,500 a year, is the world's single most expensive drug. This monoclonal antibody drug treats a rare disorder in which the immune system destroys red blood cells at night. The disorder, paroxysymal nocturnal hemoglobinuria (PNH), hits 8,000 Americans.
Elaprase ($375,000 per year) treats an ultra-rare metabolic disorder called Hunter's syndrome. Just 500 Americans suffer from the disease, which causes infections, breathing problems and brain damage.
Naglazyme from BioMarin Pharmaceuticals treats another rare metabolic disorder and costs $365,000 a year, according to investment bank Robert W. Baird. Viropharma predicts that sales of its Cinryze, a treatment to prevent a dangerous swelling of the face, will increase from $95 million last year to $350 million several years from now. The drug costs an estimated $350,000 a year.
May be free in California (Score:5, Informative)
From my understanding, those suffering from CF in California generally have their bills picked up by the state since no insurance plan could ever afford to treat CF patients. I believe this is under the state's Genetically Handicapped Persons Program [ca.gov].
But not having CF myself, I'm certainly no expert on the cost of care for it...
Re:So, treating 4000 people (Score:5, Informative)
From a radio report, apparently they will also provide it free for uninsured people who earn less than $150k, so they'll be treating more than they'll be getting paid for.
Re:Exon Skipping? (Score:5, Informative)
Re:Nonetheless a good day (Score:4, Informative)
As I noted above, the cost is only for those with insurance - they'll treat the uninsured free if they make less than $150k. So while it might cause insurance premiums to rise slightly, it shouldn't bankrupt anyone.
Re:Cost (Score:5, Informative)
Re:Cost (Score:5, Informative)
Re:Nonetheless a good day (Score:4, Informative)
Re:Ethical? (Score:4, Informative)
This is why Congress passed the Patient Protection and Affordable Care Act in 2010. This law "[p]rohibits individual and group health plans from placing lifetime limits on the dollar value of coverage, rescinding coverage except in cases of fraud, and from denying children coverage based on pre-existing medical conditions or from including pre-existing condition exclusions for children. Restricts annual limits on the dollar value of coverage (and eliminates annual limits in 2014)[.]" It also "[c]reates a temporary program to provide health coverage to individuals with pre-existing medical conditions who have been uninsured for at least six months." The guaranteed availability of insurance provision will be implemented in 2014.
You should check to make sure that your insurer is not imposing an annual limit lower than what is allowed. As of 2014 they won't be allowed to have one at all.
Check it out. [kff.org]
Re:So, treating 4000 people (Score:5, Informative)
Re:So, treating 4000 people (Score:4, Informative)
We spend ~$30billion a year on research in the U.S. on the NIH, so a partial solution is already in place.
The other thing to keep in mind is this drug is only highly priced for the next 20 years. After that the generic versions will be cheap, so future patients will benefit hugely. That's the beauty of the patent system. It hasn't been outrageously extended to hell like the copyright system has.
It's worth pointing out that part of the calculus that goes into pricing a drug has to do with the fact that drugs rarely enjoy all 20 years of patent protection, due to the fact that the invention of the drug usually occurs in the R&D phase which predates the clinical trials, approvals, and manufacturing scale-up. The average effective patent life (i.e. the period during which a drug is actually for sale) is 7 to 12 years, so prices tweaked to compensate. The flip side is that it really discourages treatments for diseases that affect very small portions of the population,since you cannot count on recouping costs over long periods of time to compensate for the small patient pool. This is partially addressed by the Orphan Drug Act, but more often than not this is where charities funding disease-specific research really play a crucial role.