In 1960, Kelsey was hired by the FDA in Washington, D.C. At that time, she “was one of only seven full-time and four young part-time physicians reviewing drugs”[4] for the FDA. One of her first assignments at the FDA was to review an application by Richardson Merrell for the drug thalidomide (under the tradename Kevadon) as a tranquilizer and painkiller with specific indications to prescribe the drug to pregnant women for morning sickness. Even though it had already been approved in Canada and more than 20 European and African countries,[8] she withheld approval for the drug and requested further studies.[3] Despite pressure from thalidomide’s manufacturer, Kelsey persisted in requesting additional information to explain an English study that documented a nervous system side effect.[4]
Kelsey’s insistence that the drug should be fully tested prior to approval was vindicated when the births of deformed infants in Europe were linked to thalidomide ingestion by their mothers during pregnancy.[9] Researchers discovered that the thalidomide crossed the placental barrier and caused serious birth defects.[7] She was hailed on the front page of The Washington Post as a heroine[10] for averting a similar tragedy in the U.S.[11]Morton Mintz, author of The Washington Post article, said “[Kelsey] prevented… the birth of hundreds or indeed thousands of armless and legless children.”[10] Kelsey insisted that her assistants, Oyam Jiro and Lee Geismar, as well as her FDA superiors who backed her strong stance, deserved credit as well. The narrative of Dr. Kelsey’s persistence, however, was used to help pass rigorous drug approval regulation in 1962.[1]
When other countries worldwide were approving this drug for use, the U.S. FDA stood strong, and would not approve.
It is even more awesome, that part of the issue with the drug being released in many other countries, was the lack of thought prescriptions would effect pregnant women, and in the U.S. we had a woman in charge of this drug’s approval, and she held fast against the pressures of the large drug company and her peers.
I wonder if the other countries had a woman working on the approval of that drug?
This is one of the things they teach you when you work at a pharmaceutical company. You may hate the FDA for the scrutiny that they have, but they certainly have done their jobs in the past.
It highlights the main issue with the current state of pharmaceutical companies and drugs for extremely rare diseases, specifically “cures” because they are a one time cost.
1 million dollars for one treatment seems astronomical, but when you compare it to costs of nearly 300,000 a year for the comparable replacement therapy, it pays for itself in less than 5 years.
The rarity of the disease and the singular curative function of the treatment mean the price has to be high to re-coup the costs of the treatment (typically)
Though I have read the Glybera is currently undergoing evaluation by the FDA to be sold in the U.S. I think that most insurers would cover the treatment, even with the expense, a one time cure is cheaper than decades of hospital visits. But it depends entirely on the insurer.
This is an example with the structural defect of relying on insurance to deliver ongoing long term health care. Why? B/C insurance policies are typically one year contracts and an insurance company, under our laws, doesn’t have to offer the same policy year after year. We allow insurers to opt in or out of the ACA exchanges on a year by year basis, and we also allow insurance companies to opt in or out of group health care on a yearly basis. Which means that the equation for an insurer is not one time cure vs decades of hospital visits but instead is “large one time cost versus at most one year of treatments, unless we agree to extend the policy for longer”.
Historically, relying on insurance to deliver health care arose out of the practice of insuring against catastrophic losses, and the weird historical circumstances during WWII. It really makes no sense to rely on an insurance model for ongoing health care, especially for profit insurance.
I agree, I don’t think it would be likely, but it would entirely depend on the insurer. In the U.S. the drug company selling this treatment would likely have deals with the insurer to lower costs etc.
With the advent of gene therapies and other advancements in medicine, I think we will be seeing an increase in drugs that “cure” conditions, so I think this problem will continue to get worse and worse.
I think it will be likely that we will have to have some legislation passed that might protect treatments like this, i.e. forcing insurers to provide the “cure” if it is recommended by doctors. If all insurers are required to follow the same rule, then it shouldn’t be an issue of “the next guy will pick it up”. With diseases this rare a couple 1 million dollar treatments per couple hundred thousand customers it isn’t going to push the math too hard.
The issue will come when we have a Type 2 diabetes gene therapy, or a therapy for controlling cholesterol, as treatments like that will effect a wider swath of patients (though likely that means the one time treatment won’t be 1 million)
The heart-breaking part of this is that we have a cure for a life-threatening (but not immediately deadly) illness, and people can’t get it. That is very wrong.
I feel like the issue of “cure” type treatment is a strong argument for a longer-term more-broad-based approach to health care such as single payer or a very strong public option in a multi-payer system, as opposed to our current fragmented-in-time-and-space contract-based insurance-based health care system.
Even from a business perspective, shutting down because no one was willing to pay $1million is bizarre. The costs (they say research, but most of that was publicly funded - really what the companies paid for the rights) has already been spent. Would it not be better to lower the price to something someone was willing to pay, in order to recoup some of that investment?
Someone made a massive miscalculation about how much to pay for the rights, and now it is both the patients and whoever is paying the cost to treat them without the cure who suffer the consequences.
No, because that does not convey the appropriate message to the market place, of “You want to live? Pay us what we want.”
I will say it again - certain things do not work with capitalism. Much like in Rome, where if you had a building fire, Crassus would show up with his private fire department and then negotiate to buy your home while the fire burned, it is effectively impossible to negotiate (certainly as an individual) when your literal life is the benefit the other side is offering.
We need to recognize that a system that permits companies to hold consumer’s health hostage at the bargaining table is not a system an enlightened society should have.
Yes, that is something that could likely be helped by that, but this drug in question was developed an marketed in areas with strong single payer or state backed healthcare plans, and it still didn’t work.
It does really feel like the problem with this drug is that someone paid way too much for the rights, and they now are pushing the cost up to cover their asses for a bad business decision.
I am in no way saying that capitalism is the right mechanic for health care - I’m saying that eating millions in losses rather than recoup some by selling the drug for less is lousy capitalism.
Both can be true. I’m not 100% certain it is lousy capitalism though - they basically are saying the market’s offer is too low, so we’ll hang on to this and use our exclusivity leverage to try to force a higher price.
I feel like this drug would have been a good case for governments to purchase the IP and then contract a company or two to produce it. Especially if most of the money to develop the drug came from governments in the first place.
For 31 million euros, Chiesi acquired the rights to sell Glybera in Europe,
In April 2017, just two years after it first went on the market, Chiesi announced it was abandoning Glybera. The company allowed the European marketing licence to expire.
Three doses left on the shelf were basically given away.
"Van Deventer says the company never considered lowering the price.
“Why would we? Pricing shouldn’t be a political decision. It should be a rational decision based on merits and values,” he said. “Hundreds of millions of investor money has gone into the company, and if there is no return for those investments, there will be no new drugs because nobody’s going to do that in the future, right?”
I.e. Fuck you, we’ll get the return we want, or no one gets it. I.e. you’re not paying us enough, pay more or get fucked.
The problem is this wasn’t through company funded research. This was university funded research, which was spun off into a separate company to go through the regulatory approvals (which, being gene therapy were understandably strict, and unfortunately expensive). This bag of dicks merely bought the rights after the hard work of creating and testing it was done.
Capitalism does not say price something so high no one buys it, and then quit the market. These decisions are being made because these companies have zero fear for competition. No is really able to come in behind them, pick up the dropped ball and run with it nor did they have a reason or incentive to sell it and walk away so someone else could try. There are other factors that are pushing these companies to make the decisions they do.
Which is the stupid, against their own self-interest capitalism. Or maybe I don’t understand capitalism and am lucky to not be a businessperson, because I am missing the value in spending millions and then quitting without any return.
