One of the most notable accomplishments of the first Trump Administration was its willingness to take on the status quo of the drug approval process, which had been beset by a dysfunctional bureaucracy that dragged out the pace for approving drugs and felt no compunction toward getting potentially lifesaving treatments to those out of options.
President Trump’s FDA appointees in his first term fully appreciated the importance of incentivizing investment and risk-taking while removing needless barriers to innovation and growth. It is a perspective that is especially important in the biotech industry--the first Trump Administration brought the epochal weight-loss drug Wegovy to the market and drove the implementation of so-called Right to Try rules, which made it easier for patients who had exhausted currently approved treatment methods to obtain cutting-edge drugs currently undergoing testing. However, the FDA under his second term has all but abandoned any sense of urgency to help suffering patients that governed his first term.
It appears that the reason for this policy change is Vinay Prasad, the left-wing Trump critic whom FDA Commissioner Marty Makary installed as head of the Center for Biologics Evaluation and Research, which oversees much of the approval process for new drugs.
Prasad recently left the FDA after he received a flurry of criticism from the Wall Street Journal, as well as Trump gadfly and confidante Laura Loomer, after he withdrew approval for the gene therapy treatment Elevidys, despite it being one of the few treatments available for Duchenne Muscular Dystrophy, a severe genetic disorder.
The outrage from the patient community when the FDA recalled Elevidys was precisely because those suffering from Duchenne MD had nothing else available to forestall the crippling disease and were perfectly willing to accept the risks entailed.
But this constitutes just one example of the agency’s increasing reluctance to allow patients with few other options to have access to life saving drugs.
For instance, since Makary’s confirmation the FDA’s rate of drug application rejections has significantly increased and made manufacturing issues a primary basis for the issuance of Complete Response Letters, or CRLs--the FDA’s letter of rejection to biotech companies for drugs. Prior to Makary’s appointment, the FDA issued about four CRLs a month, but since then the pace of drug rejections has tripled.
Many of the CRLs have blocked drugs for gene therapies: For instance, one of the last CRLs issued blocked patient access to a new cancer drug from Replimune, which treats advanced melanoma.
Many of the CRLs since 2020 have cited manufacturing or quality deficiencies, which constitute process issues rather than concerns about the safety or effectiveness of the therapies themselves. Just last month, it was revealed that Makary pushed the agency to delay approval of a new drug from Kalvista. Senior agency officials described his baldly political interference in a scientific determination as unprecedented.
While patients’ groups celebrated when the FDA reinstated Elevidys and Prasad resigned from his position after public outrage over the decision, it is unclear that Makary has learned any lesson from the experience. He has indicated that Prasad will still have some sort of role within the FDA and has even said he’d like to reinstate him.
A truism in politics often uttered in Washington D.C. is that personnel is policy. For the Trump Administration, its personnel decisions in the FDA are hard to distinguish from what a second Biden Administration would be doing.
