$2.8m gene therapy treatment is America's most expensive drug ever
- Reference: 1660864557
- News link: https://www.theregister.co.uk/2022/08/18/28m_gene_therapy_treatment/
- Source link:
Zynteglo is a treatment designed to tackle the rare condition known as transfusion-dependent beta-thalassemia (TDBT), a blood disorder that reduces hemoglobin and red blood cell counts in the bloodstream, leading to reduced oxygen delivery. Each dose is tailored to an individual's genetic profile for maximum effectiveness.
Transfusion-dependent patients have to undergo red blood cell transfusions as frequently as every two weeks. According to Bluebird, clinical trials of Zynteglo resulted in 89 percent of patients achieving "transfusion independence," defined as not needing a transfusion for at least 12 months.
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Of Zynteglo's price, Bluebird said $2.8 million is actually a deal, as "the lifetime cost of medical care for a patient with transfusion-dependent beta-thalassemia can reach up to $6.4 million."
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According to the [4]FDA's statement on Zynteglo's approval, the medication regulator fast-tracked the drug through a pediatric disease voucher. TDBT is an inherited disease that [5]significantly lowers life expectancy , with the average US patient suffering from the condition dying at age 37. Bluebird estimates between 1,300 and 1,500 people in the US suffer from the disease.
TDBT is caused by genetic mutations, which gene therapies are designed to target, repair or supercede, and return cells to normal working order. Zynteglo [6]works by adding functional, modified copies of the gene that causes TDBT to a patient's blood, a process that Bluebird calls "one time," but said can still take months to administer.
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Bluebird said the drug will be available beginning in the fourth quarter of 2022.
The most expensive drug ever
At $2.8 million for a treatment, Zynteglo dethroned the previously [8]most-expensive drug , another gene therapy product called Zolgensma priced at $2.1 million.
Gene therapy treatments can benefit from machine-learning algorithms tuned for [9]drug discovery , just like other medicines can benefit from some AI.
Researchers at MIT have used machine learning for [10]just such a task , discovering a way to improve on a gene therapy treatment for Duchenne muscular dystrophy (DMD). Current gene therapy treatments for the rare genetic condition require heavy doses of drugs, as the molecule developed for the treatment has difficulty entering cells.
As with other drug development using AI, researchers could propose a wide range of new peptides for the DMD drug. Instead of having to synthesize and test each one, AI simulates the process to find the best potential treatments.
The MIT experimenters say a new peptide discovered with the aid of machine learning boosted delivery of DMD drugs 50 fold.
While the MIT researchers are hesitant to predict their work's wider efficacy, the team did say that such drug discovery can increase medicine efficiency, reducing dosage volume and side effects.
In addition, the MIT team said treatments could become less costly. Much of the cost of drugs, especially highly-specialized ones like Zynteglo, comes from [11]drug development , a good portion of which involves the sort of discovery that MIT's system could automate away.
[12]According to Reuters , gene therapies are often curative and priced steeply, making them a difficult sell for insurance companies. In the case of Zolgensma, produced by Texas-based Novartis Gene Therapies, the drug was [13]too pricey for many insurers , leaving patients without access to the medication.
Bluebird, which said it set the price of Zynteglo so high "in recognition of its robust and sustained clinical benefit," appears to at least be aware of possible blowback from putting a multi-million dollar price tag on a potentially life-saving drug, coming out early with [14]plans to help finance the treatment.
According to Bluebird, the company is working with commercial and government payers to establish plans for an upfront payment with an outcomes-based agreement that will see companies refunded 80 percent of the cost of therapy if the treatment doesn't work. So that's all right, then.
Bluebird said it's in "late-stage negotiations with leading commercial payers" that could make it available on "dozens of plans." The company said it's also working with state Medicaid agencies, which represent 80 percent of publicly-insured TDBT patients, to ensure they have access as well.
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Arguing that such an expensive drug is actually a bargain, which Bluebird has asserted, may not be the best approach. Despite the life-saving nature of Novartis' gene therapy drug, insurers simply wouldn't pay out, forcing the company to negotiate [16]similar discounts to the ones Bluebird is proposing out of the gate, presumably in a bid to avoid such a conversation. ®
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[4] https://www.fda.gov/news-events/press-announcements/fda-approves-first-cell-based-gene-therapy-treat-adult-and-pediatric-patients-beta-thalassemia-who
[5] https://onlinelibrary.wiley.com/doi/10.1111/bjh.17091
[6] https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-fda-approval-zynteglor-first-gene-therapy
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[8] https://www.goodrx.com/healthcare-access/drug-cost-and-savings/most-expensive-drugs-period
[9] https://www.theregister.com/2022/02/24/ai_drug_humans/
[10] https://news.mit.edu/2021/machine-learning-discovers-sequences-boost-drug-delivery-0810
[11] https://sitn.hms.harvard.edu/flash/2020/modern-drug-discovery-why-is-the-drug-development-pipeline-full-of-expensive-failures/
[12] https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-bluebird-bios-gene-therapy-rare-blood-disorder-2022-08-17/
[13] https://www.businessinsider.com/health-insurance-companies-deny-kids-with-sma-gene-therapy-zolgensma-2019-7
[14] https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-us-commercial-infrastructure-enable
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[16] https://www.reuters.com/article/us-novartis-sma-insurers-exclusive/exclusive-novartis-pitches-discounts-on-pricey-gene-therapy-for-deadly-muscle-disorder-idUSKCN1SG22N
[17] https://whitepapers.theregister.com/
Non-affordable for the non-rich
No insurance affordable by someone not independently-rich will pay out for this. As to being cheaper in the long run than paying for transfusions, insurance companies' actuaries may conclude it's cheaper to pay for some tranfusions, knowing some (or many?) transfusion-receiving patients will die sooner than will die later.
Further, most "major medical" health insurance policies pay only 80% of treatment cost. The remaining 20% of that $2.8M, which the patient will pay, is $560,000.00.*
As to "discounts" ... we'll see.
*People living in countries with socialized medicine may possibly be in a better situation.
Re: Non-affordable for the non-rich
N.I.C.E is never going to say yes to this.
Re: Non-affordable for the non-rich
Health insurance plans have a copay like 20% or whatever, but only up to a certain yearly maximum which is usually in the $5000 to $10,000 range. If the therapy was covered by insurance that's what the patient would pay, the insurance would pay the rest. Plans used to have a lifetime maximum benefit limit in the $1 to $2 million range, but the ACA banned those.
I imagine very few insurance companies will approve this treatment. While theoretically paying $2.8 million is better than paying $6.4 million for lifetime treatment there are two issues with that math. One is that the present value of $6.4 million in treatments over someone's entire life is probably much less than $2.8 million, the other is that few people will remain on the same insurance policy their whole life (and they'll definitely be off it when they're eligible for Medicare at age 67 or whatever)
So from the perspective of an insurance company, they would much rather keep paying for transfusions since they will spend less that way unless you get on their insurance with a new job at age 18 and keep working there until you retire.
Countries on socialized medicine may not fare any better. When there are only one or two treatments that cost $2 million and only a few hundred or few thousand people need it, that's one thing. What happens when they have a $1 million treatment to cure diabetes, or that is guaranteed to prevent Alzheimer's so long as you are treated before any symptoms show up and you don't know if you will ever even get it? The sheer number of people who would need/want such treatment will break the bank.
Ethical minefield, and maybe eugenics wasn't so last century?
So never really having heard much about this disease, other than vaguely recollecting thalassemia, I hit da Wiki for a quick overview-
https://en.wikipedia.org/wiki/Beta_thalassemia
Beta thalassemia is a hereditary disease allowing for a preventative treatment by carrier screening and prenatal diagnosis.
So it's avoidable. Assuming you can get that screening. If both genetic donors are screened prior to conception. If screening's done after conception, then there might be an option to avoid future suffering via a termination. If that's available. I think there's been something in the news about that recently.
Or, perhaps we could screen everyone and give them a social credit score based on whether they carry a risk. We could combine that with Covid-style proximity warnings that flash red, if there's a risk of passing that on. Or, because Big Tech has been hoovering people's medical data, they may know you're a risk and decline cover.
Or, perhaps we could use the same techniques to fix the genes of carriers, preventing the problem arising. But if that'd cost $2.8m a shot, it would be a hard sell as preventative medicine. I suspect ethics and politics wrt stem cell research & treatment might also come up. Plus if you can fix 'bad' genes, why not improve 'good' ones?
Or we could look at how much these treatments actually cost, and regulate on a cost+ basis. There are obvious humane considerations around avoiding painful or distressing treatments that may or may not prevent early deaths though.
Beta thalassemia is a hereditary disease allowing for a preventative treatment by carrier screening and prenatal diagnosis.
Apparently, by abortion of that fetus, Some people are not going to do that.
Or use IVF
Screen the fertilized eggs and only implant ones that don't have the disease. If I knew I had that disease in my family history, or had it myself, that's definitely how I would go. I'd bank a bunch of sperm to use for IVF then have a vasectomy to prevent accidental pregnancy.
The ODIN, anyone?
So if this doesn't work then the insurance will only be paying $560,000 for it, and I'll bet that Bluebird Bio will still be making a profit off the failed treatment.
I really hope that things like this in the future can be handled with something like The ODIN kit and public AI.