If only we would make tough choices. The system – at least for the past years- is arranged to punt decisions down the line. Where the choices are the same, anyway- just more expensive.
It’s why we have the BRAC (Defense Base Closure and Realignment) system. No one wants to be responsible for cutting the Pentagon’s budget, even knowing there’s plenty of waste. So, we appoint a commission to remove bases that no longer serve their mission or are inadequately provisioned (and too expensive to update or redundant)- and then lobby to have some of those decisions rescinded.
The same thing happens with drug approvals. You may think that the only consideration for drug approvals are that they are safe and effective. But, there are other considerations.
Like, what happens when a drug (or therapy) is developed that will only treat 500, 1000, or 10000 people. How do you determine that it is effective? If there are only 500 people with the disease, do you test the drug on every one of them- because normal drug tests require at least that many people to be tested. And, then, how many of them must be cured or relieved of symptoms?
And, what happens when a drug is being developed for this small population? Once a drug is approved by the FDA, Medicaid must pay for the coverage. Regardless of its price.
Therein lies the problem with these orphan drugs. Orphan drugs receive special consideration- as stated above. Normally, phase III clinical trials require at least 1000 test subjects. Not so for orphan drugs. Orphan drugs also have special tax incentives, longer patent life (to preclude competitors), etc. And, many of the orphan drugs are just s-l-i-g-h-t-l-y different than their sister formulations that treat other conditions. But, their prices- often sky high.
These high-priced drugs are not “officially” a problem, because they are only used for a few patients, so the overall cost is not too high for society to pick up. But, as I’ve written before, many of these orphan drugs are immediately used for other diseases- for which testing and approval was not granted- because the FDA can’t stop physicians from employing them for off-label uses. (That practice would officially means that the FDA is regulating the practice of medicine; it is not so permitted.)
That’s a real problem- and certainly time for it to be “officially” recognized. Within 5 years or so, our prescription drug spending on orphan drugs will have risen from about 5.1% to 15.9% of the total drug expenditures- and that total spending has grown from about $ 140 billion to $ 300 billion. (If you are weak in math, that means that the orphan drug market will have grown from $ 7 billion to $ 48 billion!)
That’s exactly what just happened with a new drug that was approved by FDA. Procysbi and Cystagon are both the drug cysteamine. As you can see from the diagram, they are even listed together on medical information databases.
Cystagon has been around for a while, marketed by Mylan Pharmaceuticals. It was designed to treat cysteine excretion disorders (such as cystinosis and cystinuria), by breaking (cleaving is the term) the disulfide bond with cysteine, allowing for its excretion. It is also used in radiation sickness. Without the therapy, cysteine accumulates in the kidneys and eyes- and, if you live long enough- the thyroid gland, your muscles, and eventually other organs in the body.
There are side-effects with Cystagon. You might guess from how I described the drugs action above that one may have bad breath and/or body odor problems- the rotten egg smell- due to the sulfide reactions. One may also feel nauseous or have abdominal problems, while taking this drug every six hours. And, the drug is not very cheap, costing some $ 8000 a year for the patients who need it.
But, there is this fairly rare inherited disease- nephropathic cystinosis. Born with it, and you are untreated, your kidneys are gone before adolescence; kidney transplants don’t even guarantee you a life beyond your 30th birthday. The population- about 500 in the US and maybe 3000 in the world.
So, Raptor Pharmaceuticals has reformulated the drug. It coated it, so it will traverse further through the digestive system, where it is then released (enteric-coating), precluding that rotten egg smell as a side effect. It will also only need to be taken every 12 hours, instead of the 6 for the uncoated version. Oh, and the annual costs for therapy? About $ 250K.
If you think that is worth an extra $ 242K a year, that’s great. But, maybe we need to change the rules for Medicaid- so that is won’t pay for orphan drugs that have nearly equivalent (since the side-effects are different) alternatives. And, save society some money.
And, before you scream at me that I am only talking about $ 100 million a year (and that ain’t chicken feed, folks), let me also remind you that this drug is used for Batten’s disease (about the same prevalence for cystinosis) and Huntingdon’s disease (about 30,000)- which means those expenditures approach $ 200 million to $ 1 billion…
Roy, there is something wrong when you coat a pill and charge big bucks for doing that. I am also disturbed about the costs to the US people, but I think when a person gets one of those diseases, we should not withhold medication because they can’t afford it. Tough decisions.
My big complaint is about the orphan drug act (ODA). I understand that it is difficult to entice folks to design drugs for a small population, but we need to modify the ODA to insure that pricing is reasonable and that the number of dosages sold a year are limited (to stop off-label use that is among the most profitable for orphan drugs).
I believe that generic drugs are the future. I hope that it will decrease the price of such drugs!
I am not so sure. Why would I develop a new drug and not make money from that effort, Muriel? There may be more appropriate methodology to price drugs, but it you only allow generic drugs, then you better impress upon your governments that research dollars are going to have to be a very large component of their budgets.
Roy
Why can’t our government see any of these things instead of spending all their focus on messing with the social security system to find the money the government needs? That fix might just go along way to fixing the problems in Medicare. I am sure there are many other orphan drugs with the same drain on the money in medicare.
Yes, these orphan drugs tend to be very high prices. As I wrote to Ann (Mullen), we need to change the system to render it more equitable, William.
Thanks for the visit and the comment.
I never understood what measures are taken to test drugs and what increases their costs to such dramatic heights. It’s a cruel thing to know that a drug exists that can help extend your life, yet you can’t have it.
Well, there are costs to be considered. But, I am not sure that the system (orphan drugs) works to a patient’s advantage, Suerae.
Ok, I am tired and I’m not real sharp on stuff like this but if I understand you Cystagon went from 8K a year to 242K a year simply due coating for minor side effects? That is, at least to me, absurd. Also a little stumped on the comment regarding doctors using drugs for purposes other than their approved purpose. Interesting….
Cytagon decided that their coated drug was worth 242 K a year, yes, Alessa. But, i will grant them it’s not just for minor side effects, in that the drug now has a slow release capability. But, I don’t think that merits a 30X increase in cost for the patient.