In recent days, it seems like the din of voices arguing that the FDA should approve pharmaceuticals more speedily and on less evidence has grown louder. It is a central theme of the 21st Century Cures Act, which the House may vote on today and which I seemingly will never finish blogging about (most recent post here, with links to previous ones). It is the premise that underlies other legislation recently introduced into Congress. And it was the topic of a Wall Street Journal opinion piece just last week. In the view of these critics, sure, the FDA has some role to play in ensuring safety and some basic level of effectiveness. But the current standard for demonstrating effectiveness is, in their view, much too strict. Instead, we ought to approve drugs more quickly and allow insurance companies and physicians to decide which products have enough supporting evidence to merit reimbursement.
Here’s the problem: that is not the way we’ve set up the system. FDA approval is often linked to insurance coverage. Medicaid must cover essentially all FDA-approved drugs, and Medicare similarly has limited ability to decline to cover FDA-approved drugs. Even private insurers are generally required to cover at least some prescription drugs, although in some cases this may be on a more limited basis. Take Exondys, a drug that recently won accelerated approval from the FDA for the treatment of a small number of patients with Duchenne Muscular Dystrophy (I’ve blogged about Exondys here). Because Exondys was approved based on a surrogate endpoint and not actual evidence of clinical improvement (Exondys’ label actually says that “[a] clinical benefit of Exondys 51 has not been established”), it would seem to be a poster child for these arguments about the FDA. Allow insurers to cover it or not as they choose, since we don’t yet know if it works. Yet many insurers are legally required to pay the $300,000 a year on average the company is charging for the drug.
I’ll put it another way. If we lower the FDA’s approval standards and do nothing to coverage requirements, we will all almost certainly end up paying more money for drugs that don’t work. The pronoun “we” here is important: because an enormous amount of these expenditures will come through Medicare and Medicaid, which are funded by all of us as taxpayers, it costs all of us financially when ineffective or unsafe drugs are approved by the FDA. Many people who argue for a decrease in FDA standards also believe that we spend too much through Medicare and Medicaid, yet they don’t seem to put these two pieces of the argument together.