$2.125 million per dose : The Most Expensive Drug in the World

Since retiring I have not read much of the medical literature. However, one publication I still subscribe to is The Medical Letter on Drugs and Therapeutics. It is a twice monthly newsletter that as you would suspect reviews recently released medications and occasionally reviews the treatment of a particular illness.

The July 29, 2019, issue features Zolgensma – One-Time Gene Therapy for Spinal Muscular Atrophy. It describes a revolutionary gene therapy for the treatment of children less than two years old with this debilitating and often fatal illness. It involves the infusion of a manufactured gene carried by a virus into nerve cells, resulting in the synthesis of the missing protein needed for neuro-muscular function. It appears to be one of the first of many “gene therapies,” that are in the pipeline. The FDA estimates that 10 to 20 of these remarkable therapies will be approved annually by 2025.

The Medical Letter said that Zolgensma is approved for intravenous infusion for one dose in children less than two years old. The cost is $2.125 million. That amount jumped out at me and led to a search for more information. The website medicalxpress.com on August 8 reported that approval of Zolgensma was based on manipulated data. However, the FDA and Novartis, the company selling Zolgensma, agreed that the therapy was safe and effective and should stay on the market.

I was curious to find the source of funding for the development of Zolgensma. The website keionline.org reports that “The NIH RePORTer database lists nearly a half billion in grants, when the search term “Spinal Muscular Atrophy” is used…” Almost $150 million in NIH grants have been awarded to three United States investigators. I was surprised to see that one of the investigators is Jerry Mendell, a neurologist at Ohio State University, who was one of my classmates at UT Southwestern Medical School.  From www.latestly.com: Jerry Mendell, a doctor involved in the trial said, “The level of efficacy, delivered as a single, one-time therapy, is truly remarkable and provides a level of unprecedented hope for families.”

Still my interpretation of that information is that charitable donations and NIH grants have funded the research. So why $2.125 million for a single treatment?

The industry argument is that it takes billions to shepherd a drug through the FDA to get approval. They also remind critics that the cost of unsuccessful drugs must be borne by those that do reach the marketplace. But still in my reading I found no one who suggested that much of that cost is borne by charitable donations and taxpayers. Am I mistaken? If I am not what is the return on our investment as taxpayers?

Another suggestion that has not gained much traction is a one-time prize for drug development. The price could be on a cost plus basis, assuming that the true costs of development and approval could be fairly arrived at. Then a prize of say $2 billion would be awarded by the FDA (and lesser amounts could be awarded by other governments) and then the drug would be sold at an affordable price.

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Drugs, Choice, and the “Free Market”

I went to see my allergist yesterday. One of the treatments she prescribed was Dymista. It is a nasal spray containing an antihistamine (Astelin) and a corticosteroid (Fluticasone). I have been using these separately with some success but the combination simplifies their use. She gave me a couple of samples that will last me 2 to 3 weeks. She mentioned that my insurance might not pay for it. However, since I generally buy my own medication I was not too concerned about insurance. That is until I looked up the price from the wholesaler that I use and found that Dymista is $175 for 30 doses. If used twice a day, like she prescribed, that is a little over two weeks of medication.

I am all for modern medicine and I think many of the drugs we take have improved health and prolonged life but I for one think Big Pharma has taken advantage of us. And the government has been impotent in curbing their rampant greed.

If you bought Dymista at Walmart, which is the cheapest price I found on GoodRx.com, it would cost $188. Its ingredients Astelin ($30) and Fluticasone($15) cost about $45 for the same amount.

Mylan Pharmaceuticals is charging four times as much to combine the two drugs into one formulation. It is much like charging $3 dollars for a cup of coffee and a dollar for cream and then charging $17 for coffee with cream.

This is an example of the “free market” that conservatives think solve all economic ills. If regulators would get out of the way and just let the market decide we would all be better off.

What never seems to come up is why regulations are necessary in the first place. The term “government overreach” is a favorite term of conservatives but what they fail to understand and acknowledge is that the overreach began with players in the market, pushing the legal and moral limits for power and profit. Regulations are a reaction to wrongdoing in the marketplace to try to serve the “common good.”

Until we as a society decide that pharmaceutical price gouging cannot be tolerated the Mylan Pharmaceutical’ s of the world will continue to manipulate and take advantage of consumers for their bottom line benefit.

In a review of William Cavanaugh’s book Being Consumed: Economics and Christian Desire, Alex Abecina says, “Cavanaugh contends that the so-called ‘free-market’, having been detached from objectively good ends, actually leaves consumers vulnerable to economic enslavement under an advertising saturated society, controlled and surveyed by a small handful of transnational corporations. This small but powerful sector possesses the research and marketing ‘know-how’ to produce artificially created consumer desires that are capable of dictating our choices for us.”

Freedom in a “free market” involves freedom to make informed choices, taking into account ethical concerns of fairness and the common good. Absent those parameters the “free market” is just about greed.

I went to jail Saturday

I went to Jail Saturday

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Last Supper Mural in Beto Chapel  by Mario Ayala painted on concrete block 1998

Actually I went to the Kairos community Christmas party in the Chapel of the George Beto Penitentiary Unit of the Texas Department of Criminal Justice near Palestine, TX.

Cherry and I worked several Kairos weekends until a few years ago but always try to make it back for the Christmas.

Kairos (Greek for God’s special time) needs to be experienced to fully understand. It is a 3 day Christian retreat in the prison that demonstrates to society’s forgotten that God loves them. It is one of the most powerful emotional experiences imaginable. And for my agnostic and atheist friends, I would challenge you to experience it before you knock it.

Briefly, sixteen whites, sixteen blacks, and sixteen Hispanics are brought together, hostile and suspicious. They only come because they have been told, correctly, that the food is great – Barbecue, lasagna, taco salad, fried chicken, salad, ice cream, cake. But through talks, singing, and forgiveness exercises over the three days a transformation takes place that is unique.  Suffice it to say, lives are changed. Not perfectly and not 100% but a sense of purpose and community is formed that replaces isolation, anger, depression, and gangs. Many experience love and acceptance for the first time in their lives.

So I arose at 5 AM Saturday and drove the 120 miles to Tennessee Colony, TX. Seventy five of us entered the Beto chapel around eight and were soon joined by over 180 of our “brothers in white.” All wore beltless button-less uniforms with a nametag clipped to the front pocket. Clean cut, no facial hair. Many were tattooed from the top of their heads to their fingertips. As I wrote in First You Pay:

Time and how to fill it is the second problem facing the incarcerated—staying safe and alive being the first. Many pass the time drawing on paper or on each other.

 But what I heard from several of my “brothers in white” at Beto astounded me. There is an epidemic going on there and, as I found after an internet search, in the free world as well. AND I HAD NEVER HEARD OF IT! Here is one brief story.

He takes a single puff of potpourri, called K2 in here. He immediately sheds his clothes and races out of his cell, one of one hundred and seventy on this wing. He wrestles the keys and pepper spray from an officer and puts the officer’s hat on his head. He streaks down the wing, three floors of cells on either side. He pushes another guard aside and climbs to the second tier of cells. As officers close in, he leaps across the hall to the opposite row of cells, dangling from the railing on the second level. He is sprayed with pepper spray but it has no effect. He begins using the pepper spray on the officers who must retreat until the K2 wears off. He eventually drops to the floor and is subdued and led away to administrative segregation.

Scenarios like this are repeated daily at Beto. For the last two to three months smoking K2 has caused psychotic breaks, violence, bizarre behavior such as jumping off elevated wings, and a few deaths. It is known by other names K2…Spice…Potpourri…fake weed…Yucatan fire…Skunk…Moon Rocks…Synthetic Marijuana…and its cousin Bath Salts.  You can look it up on the internet. 

In a perverse way that behavior has something in common with Kairos.

Andrew Weil in his 1972 book The Natural Mind popularized the human need to alter consciousness. From childhood dreams or spinning around until dizzy to Walter Mitty fantasies to alcohol and drugs – all have one thing in common – to alter our reality or consciousness. And I would add religious ecstasy to the list.

Walker Percy spent his literary career writing about existentialism and the human need to get out of “ordinariness” and find meaning. Borrowing from Kierkegaard he basically asked us in his novels to come out of our ordinary lives and recognize the Wonder and Uniqueness around us – without drugs. The hypnotist Ted Barber told me that he thought that most people needed to be brought out of hypnosis, rather than induced into it. Look at the person next to you at the next stoplight and you’ll see what he means (unless they have a phone in their ear!). Not present!

K2 and Kairos both offer an emotional high that takes us out of the ordinary to alter our consciousness. Certainly one is dangerous and the other can be positively (OK or negatively) life transforming but both begin with an alternate focus of reality.

So it seems to me that altering our perceptions or consciousness is part of our human condition or as BF Skinner said:

The object of life is to gratify yourself without getting arrested.