The election of a new President always sparks discussions around the new policies that the new leader will pursue. Never has such speculation been more rampant than now with the ascendency of President-Elect Donald Trump. This has been especially true in healthcare, where Mr. Trump, who has long railed against the Affordable Care Act (ACA)--“Obamacare”--has pledged that he’ll have Congress repeal it if elected. He appears now to be modifying this stance somewhat, but how far he goes to deconstruct the ACA is foremost in the minds of many.
He has been a bit more opaque on how he would handle the FDA. Certainly, he is clear about his disdain for big government and the need for less bureaucracy. Last September, he took the FDA to task with respect to their regulations for the food industry:
The FDA Food Police, which dictate how the federal government expects farmers to produce fruits and vegetables, even dictates the nutritional value of dog food. The rules govern the soil farmers use, farm and food production hygiene, food packaging, food temperature and even what animals can roam which fields and when. It also greatly increased inspections of food "facilities" and levies new taxes to pay for this inspection overkill.
This statement set off enormous criticism and the Trump campaign seems to have backed off this publicly. But this is likely to resurface in the coming months. Mr. Trump is no fan of abundant regulations as he believes that they hamper the U.S. economy. If he feels this way about food, what will he do with the FDA’s oversight of the drug industry? In his recently announced First 100 Days Action Plan, he said that:
Reforms will also include cutting the red tape at the FDA; there are over 4,000 drugs awaiting approval, and we especially want to speed the approval of life-saving medications.
The 4,000 number is a bit jarring. My guess is that this includes not just new chemical entities, but also new uses of already approved drugs as well as new applications for the approval of generic drugs. Will Trump direct the FDA to lower the bar when it comes to drug approvals? Actually, some people think that the FDA has already started down this path with the approval of Sarepta’s Duchenne muscular dystrophy drug, eteplirsen, a drug with very limited extant clinical data in its New Drug Application (NDA). Will the eteplirsen example become the norm rather than exception?
I don’t think so. My guess is that once Mr. Trump and his advisors begin peeling the FDA “onion,” he’ll recognize that the FDA does afford protection for the general public. But here are some steps that I think he will take.
- The bar will be lowered for the approval of generic drugs. The backlog at the FDA is pretty large for this drug category, and the lack of generic alternatives for drugs like Daraprim has resulted in extensive price gouging. Mr. Trump can score big points by telling the American public that such a step will foster competition and drive down drug costs, both of which are consistent with his campaign messages.
- The eteplirsen example will, in fact, become more common for orphan drugs where there is no other alternative for patients. Again, patient advocates for rare diseases will be very supportive of this.
- Restrictions will be eased on the off-label use of already-approved drugs. While FDA final approval will be ultimately required, the publication of clinical trials showing the benefit of the drug in a new indication will be sufficient to allow companies to discuss the new use of such a drug with physicians and other healthcare professionals.
There will be pushback against such changes not just from regulators but also from payers who normally want absolute proof of a drug’s efficacy before providing reimbursement. The FDA will also justifiably raise concerns about exposing the American people to unnecessary risks. After all, this is the agency whose role model is Dr. Frances Kelsey, whose diligence as an FDA medical reviewer protected U.S. babies from the horrors of thalidomide in 1960. But the Republicans swept this year’s elections and they believe in smaller government and less regulation. The FDA won’t be immune to this.
