How will we pay for the coming generation of potentially curative gene therapies?
By U.S. Senator Bill Cassidy, M.D.
June 12th, 2019
We have arrived at a special moment in health care. Innovative, life-changing gene therapies are here that will cure or ease debilitating diseases. Yet these expensive treatments are entering a market structure that was not built to price them.
Congress will likely need to play a part in developing a new paradigm for financing such treatments. As a senator and a physician, I have been following this issue closely, and see several possible paths forward. There are multiple issues to address.
Regarding the impact upon taxpayers, a 2009 paper by Teresa L. Kauf and colleagues estimated that individuals with sickle cell disease consume almost $1 million worth of health services by age 45. With approximately 70,000 sickle cell disease patients nationwide, a gene therapy priced at $1 million to reflect the avoided cost of future health services would cost $70 billion. Since there would be a moral imperative to treat individuals as soon as possible, this would be incurred over a one- to two-year period. With an estimated 3,000 individuals with sickle cell disease in my home state of Louisiana, the expense would be $3 billion over one to two years. And this is for just a single genetic disease. In short, gene therapy may not be affordable for federal and state government budgets.
With these considerations in mind, I describe five payment approaches that have been proposed. There are certainly others.
Life-changing gene therapies are coming. We must give thought now on how to determine the price of these innovative, new-age treatments and how to finance them to ensure that we realize their full, beneficial potential while also ensuring that society can pay for them.
I do not believe that any single one of these proposed models can do that in all cases, and at least for now we need to explore them all. The tragedy would be if the benefits of innovation were denied because of an inability to afford the innovation. It is an issue which must be addressed.