The Food and Drug Administration is supposed to be the global gold standard for drug approvals, but the start of 2025 has drawn outsized concern from various corners in the healthcare industry. From staffing issues, including the scandalous resignation and rehiring of a key agency official, to increases in delays and denials of new drug applications, the FDA’s direction is unnerving.
The most recent drama involved the high-profile resignation of Dr. Vinay Prasad, the director of the FDA’s Center for Biologics Evaluation and Research, who left amid controversy and the objections of Health and Human Services Secretary Robert F. Kennedy Jr. and FDA Commissioner Marty Makary.
Infighting within Trump-world kept Prasad out for two weeks before Makary brought Prasad back to manage biologics and vaccines.
While administration enforcers were concerned more with political loyalty tests, advocates and medical providers maintained that Prasad’s leadership was chaotic for patients and innovators. Anyone relying on new treatments for FDA approval knows that this can be a life-or-death matter.
Surely, Makary wouldn’t bring him back if he didn’t have full confidence in Prasad’s ability to lead CBER, right?
That’s where uncertainty comes in. Interestingly, Prasad’s rehiring has coincided with increased delays, denials and regulatory confusion regarding how companies are expected to bring cures to market.
Developing breakthroughs in biotech is far from easy and never cheap. It can cost more than $1 billion to bring treatments to market, with exorbitant amounts of a company’s money being spent on research and development, plus drawn-out clinical trials, in hopes of getting the green light from the FDA for a treatment to reach patients.
It’s a dramatic difference when the FDA functions as a red light, rather than a green one. Instead of fast-tracking approvals of new and effective therapies, the agency seems more motivated to dole out Complete Response Letters (CRLs) to stop innovation in its tracks.
It’s another area where the Trump administration has adopted or drifted into the mindset of Europe’s precautionary principle on new technology — a risk-averse posture that serves to freeze the continent’s homegrown tech firms.
Look at Replimune’s RP1, a breakthrough immunotherapy treatment for advanced melanoma, which had its accelerated approval application rejected by the FDA last month. Despite positive results in clinical trials, the agency refused to approve the therapy due to “heterogeneity of the patient population,” although the FDA approved a drug 18 months earlier for the same population with nearly identical results.
Whose job is it to help find reasonable answers for the 8,400 advanced melanoma patients who will succumb this year to the tragic disease?
Take Deramiocel as another example. As Capricor Therapeutics fights to advance this therapy for “Duchenne muscular dystrophy,” a debilitating rare disease with limited treatment options, they’ve been hit with the same roadblocks.
After receiving a CRL from the agency, the company was able to secure a rare Type A meeting, which is often requested when regulatory obstacles need to be addressed and resolved.
The Type A meeting with Capricor and FDA demonstrates a positive step toward transparency, but the meeting minutes have yet to be released. This has left patients and stakeholders understandably concerned that the FDA is more interested in going through the motions to provide the illusion of transparency rather than finding actionable solutions to all the delays.
These inconsistencies threaten the entire biotech ecosystem as the FDA continually moves the goal posts. Prasad talks a big game on cutting red tape and consistently does the opposite.
For now, the United States remains the world’s leader in biotech innovation, but repeated turns away from convention by the FDA seriously risk ceding that ground to China.
Commissioner Makary’s stated commitment to releasing CRLs in real-time could seriously help. However, everyone loses if transparency is just a PR checkbox and not part of a broader strategy to provide regulatory speed and clarity.
