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.