What the market will bear

I started college at the dawning of the age of cloning. Recombinant DNA technology represented the transition from descriptive molecular biology to genetic engineering. By the time I started teaching medical students, the genes mutated in terrible diseases like cystic fibrosis, Tay-Sachs, hemophilia and Fragile X were cloned. There was always an obligatory paragraph at the end of the paper to the effect that now the door was open to gene therapy.

For decades, the cloning of such genes made the diagnosis in affected individuals much simpler, freed from pedigree analysis. The bottleneck to gene therapy was not cloning the affected gene, it was delivering a therapeutic gene in useful amounts to the appropriate organ or tissue. 

Since the turn of the century, genuine progress has been made in gene therapies. Unfortunately, this has presented a new barrier to these technology—cost. Pharma companies have priced gene therapies so high that they are out of the reach of middle class and poor patients and their families. The given justification is that Pharma is entitled to recoup the costs of years of development and trialing of the therapies. 

 

Along comes Zolgensma, a transformative therapy for spinal muscular atrophy, a rare, incurable disease that kills its victims by the age of two.

“When Zolgensma hit the market five years later, it was hailed as a miracle drug. Some babies treated with it grew up able to run and play. It helped reduce U.S. death rates from SMA, long the leading genetic cause of infant mortality, by two-thirds.

 

“That leap forward came at a sky-high price: more than $2 million per dose, making Zolgensma then the costliest one-time treatment ever.”

But the developers of Zolgensma are just asking to be reimbursed for their investment costs, right? Uh, no.

“This was a case where the charities and the government did everything to get this thing commercialized, and then it just became an opportunity for a bunch of people to make transformative, generational wealth,” said James Love, director of the public advocacy group Knowledge Ecology International.

This is just capitalism doing what it’s best at. 

 

“Drug companies charge whatever they think they can get away with,” said David Mitchell, the founder of Patients For Affordable Drugs. “And every time the benchmark moves up, they think, ‘Well, we can get away with more.’”

 

“Parents of children with SMA say their concerns about costs pale in comparison to the hope offered by such cutting-edge therapies. “I mean, it’s a child’s life,” said Hailey Weihs, who battled her health plan to get Zolgensma for her daughter. “Anybody would want that for their own child.”

 

“The seven-figure costs of Zolgensma and other gene therapies add to the nation’s ballooning bill for prescription drugs, absorbed by all Americans in the form of rising insurance premiums and taxes for public programs like Medicaid.”

Zolgensma isn’t unique. It belongs to a growing class of therapies that are priced to provide the maximum return for investors. And that’s not all:

“[Novartis] also pocketed yet another taxpayer-funded benefit: a voucher from the Food and Drug Administration redeemable for accelerated review of another drug. Such vouchers — designed to encourage companies to invest in pediatric rare-disease treatments — can be sold, typically bringing prices of around $100 million apiece.”

It's all about greed.

https://www.propublica.org/article/zolgensma-sma-novartis-drug-prices-gene-therapy-avexis?utm_campaign=propublica-sprout&utm_content=1766653386&utm_medium=social&utm_source=facebook&fbclid=IwY2xjawPGZ_lleHRuA2FlbQIxMABicmlkETFxOWFhT2F6S0xWSXE3ZVZRc3J0YwZhcHBfaWQQMjIyMDM5MTc4ODIwMDg5MgABHoi-KEhMqVN_Tsdkx-PAYlKTrHeCjNEgDgN6R0UPY1a3MsKNjotx5WYi0MZN_aem_9Z_f1WJP-obeXAdvQE-pRQ