- A Record Price: The gene therapy Zolgensma helped children born with a fatal disease, spinal muscular atrophy, grow up to run and play. But the cost was stunning: $2 million per dose.
- Cashing In: While taxpayers and small charities funded the drug’s early development, executives, venture-capital backers and a pharma giant have reaped the profits.
- Priced Out: The drug’s cost adds to the nation’s ballooning bill for prescription drugs and puts Zolgensma out of reach for kids in many low- and middle-income countries.
Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma had ended and the forces that turned it into the world’s most expensive drug had taken over.
It was May 2014. He and his wife were sitting in the cafeteria at Nationwide Children’s Hospital in Columbus, Ohio.
Elsewhere in the hospital, an infant — patient No. 1 in a landmark clinical trial — was receiving an IV infusion that, if it worked, would fix the genetic mutation that caused spinal muscular atrophy, a rare, incurable […]
This article is a great illustration of an essentially dysfunctional process. The United States has been governed so long under the “privatize the profits, and socialize the costs” scam of “Private/Public” partnerships that we have lost our way. The American taxpayers have been funding basic research for decades through grants and research studies, but the fruits of these investments are not seen by the public. Instead the research university patents the results of government funded research, under the cloak of a fraudulent “not for profit” status, and the pharmaceutical companies do the same commandeering public research for private gain. It is time for the taxpayer to realize the fruits of their own investment, and demand a share in the outcomes through lower drug and therapeutic prices.