CMS realizes that some drugs approved through the FDA’s accelerated-approval process won’t prove to be safe and effective in final analysis. It wants to balance its payment for products that might not pan out in confirmatory trials by trying to make firms sprint, rather than crawl, to finalizing such studies. How? Well, we know that CMS agreed to pay for the infamous Alzheimer’s drug, Aduhelm, only for patients participating in the confirmatory trial. But now HHS is floating a potential trial balloon (and not one to be shot down by a sidewinder missile) to help motivate firms to speed those trials to conclusion.
CMS’s idea is to pay less for drug products that are approved under the accelerated process based on surrogate endpoints until efficacy is confirmed in subsequent trials. Probably not a popular idea for the pharmaceutical industry, which has the most to lose. Some drugs approved through the accelerated process have significantly profited firms while lengthy studies are conducted, but with some products the sponsor continues to profit, even after confirmatory studies have not established that they work, during the time that the FDA makes final decisions on what to do with them. The CMS “balloon” may place the risk more on the companies by paying more reasoned prices for treatments while such studies are ongoing, delaying “full price” until they have proven that the products work as intended, thus confirming the surrogate endpoint.
We will have to see how the process plays out or even whether CMS adopts the policy over time, but something does need to be done to make potentially lifesaving drugs more accessible without breaking the bank for the government or the people who try them before full approval is given.
