On September 30 the U.S. Food and Drug Administration (FDA) approved, Relyvrio, a drug to slow the progression of ALS, also known as Lou Gehrig’s disease. The approval was controversial. The FDA usually requires at least two clinical trials but will on occasion accept one trial if the trial was robust and the data provides persuasive evidence the drug works. The advisory panel (whose votes do not count towards approval) voted 6 to 4 against recommending the drug for approval, saying the data was not conclusive and a bigger study is needed. The federal agency convinced the panel to change its recommendation saying flexibility is needed for dread diseases such as ALS:
In announcing the approval, the FDA acknowledged there were “limitations” to the data that created uncertainty about its effectiveness. But it concluded the evidence was sufficient given the “serious and life-threatening nature of ALS and the substantial unmet need” for treatments.
The price for a drug nobody is sure works? It’s $158,000 for the first year, rising to $163,000 annual for every year thereafter.
The price drew criticism from some advocates. A few weeks ago, the Institute for Clinical and Economic Review, a nonprofit group that analyzes evidence on the effectiveness and value of drugs, said a fair price would be $9,100 to $30,600 annually.
Earlier in 2022 the FDA approved another controversial drug. Aduhelm, is a monoclonal antibody to treat early-stage Alzheimer’s. Many experts on the advisory panel also thought it had scant evidence that it actually works. As in the case of Relyvrio, the evidence for Aduhelm was lacking but the alternative therapies were few so the FDA bowed to pressure from disease advocates and approved the $56,000 a year drug anyway.
If the aforementioned drugs sound expensive, you haven’t seen anything yet. A few days ago Zynteglo, a new drug for a life-shortening blood disorder called beta thalassemia was approved. The firm immediately set the price at $2.8 million for the one-time treatment. Another drug called Zolgensma has a one-time cost of $2.1 million, according to GoodRX. GoodRx tracks drugs prices across thousands of pharmacies and facilitates discounted medications for both insured and uninsured patients. The following examples are also from GoodRx.
According to GoodRx, there are numerous drugs in the $1 Million Club. Zokinvy costs $1,073,760, but that’s the cost every year. It’s used to treat Hutchinson-Gilford progeria syndrome, a rare genetic disease that causes premature aging. Danyelza costs $1,011,882. It is considered a breakthrough therapy for neuroblastoma in the bone or bone marrow of pediatric and adult patients. Funny, the work “breakthrough” is not often applied to these hyper-expensive therapies.
Other drugs that didn’t make the $1 Million Club:
Kimmtrak costs $975,520. It’s a drug to treat a form of eye cancer. It’s taken intravenously, as are most of those that follow.
Myalept costs $77,496 for a monthly supply, or $929.951 a year.
Myalept is one of the only drugs on this list that is dispensed at the pharmacy. It is used to treat leptin deficiency in patients with generalized lipodystrophy, which causes abnormal fat distribution in the body.
Luxturna costs $850,000.
Luxturna is a gene therapy that treats an inherited form of retinal dystrophy, a condition that causes vision loss and often even complete blindness.
Folotyn costs $842,585.
Folotyn is approved to treat peripheral T-cell lymphoma, a rare blood cancer that can cause death. A healthcare professional must administer Folotyn, and people are typically given 135 vials of the drug annually.
Brineura costs $755,898.
Brineura is a first-in-class medication that treats a specific form of Batten disease called late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Specifically, Brineura helps treat the slow loss of walking ability in pediatric patients 3 years and older.
Blincyto costs $754,720.
The ninth-most expensive drug, Blincyto, has an annual list price of $754,720. Manufactured by Amgen, Blincyto is used to treat a rare form of acute lymphoblastic leukemia, a cancer of the blood and bone marrow.
Ravicti costs $695,970.
Used to treat urea cycle disorders, Ravicti has an annual list price of $695,970. Urea cycle disorders are genetic conditions that result in high levels of ammonia in the blood. If left untreated, they can lead to confusion, coma, or even death.
If some of these drug prices seem expensive consider most of them require infusion in a doctor’s office or a hospital. If done in a hospital, the cost will be much, much higher.
Why are these drugs so expensive? There are several reasons. 1) The FDA has created so many barriers to entry that getting a drug through the approval process is a multibillion endeavor. Thus, there is limited competition. 2) Insurance. That fact that government, insurers and employers are on the hook for all of these treatment costs means drug manufacturers have no incentive to compete for consumers on the basis of price. 3) Obamacare. Obamacare banned both annual caps and lifetime caps on benefits. Prior to Obamacare drug makers had little incentive to price drugs exceptionally high because insurers had limits on how much they were required to pay in benefits. Now the sky is the limit. Also, medical care has turned from a gold rush into highway robbery.