September kicked off with a day of recognition and awareness for Duchenne Muscular Dystrophy (DMD), a disorder that impacts up to 1 in every 6,000 people born unable to produce the protein dystrophin for themselves. Muscle mass declines over time and becomes increasingly fragile, presenting all manner of challenges for patients and families. 

Biotech innovation continues to surge forward, unlocking therapies for different ailments that we once thought might never be attainable. Recent breakthroughs include Elevidys, a Duchenne muscular dystrophy treatment that targets the underlying genetic cause of DMD. 

Additionally, CRISPR-based treatments are actively rewriting the script for sickle cell disease, and Roctavian and Hemgenix are offering hope to hemophilia A and B patients, who would otherwise have to endure lifelong scheduled blood infusions. 

These therapies are modern miracles that extend the quality of life for countless patients and restore some hope for the future. 

But then there’s the FDA, which continues to deal with fallout from its high-profile and tumultuous leadership changes. With the recent departure and then rehiring of the FDA’s Center for Biologics Evaluation and Research (CBER) Director, Dr. Vinay Prasad, many wonder if politics is taking precedence over streamlining medical innovation. 

After being ousted by President Trump, despite objections from HHS Director Robert F. Kennedy Jr. and FDA Commissioner Marty Makary, Prasad is back to his old post atop CBER.  

During his original three-month tenure, Prasad presided over several controversial decisions, including the rejection of promising breakthrough treatments from Replimune and Ultragenyx, which left patients and drug manufacturers stuck in limbo for an already drawn-out process. 

The recent rejection of Ultragenyx’s UX111 treatment for Sanfilippo Syndrome Type A was particularly troublesome, namely because the FDA cited concerns about the manufacturing sites and not the treatment itself as cause to slow the drug’s approval. 

While these concerns are easily addressable and can be resolved quickly, the company still has to resubmit its Biologics License Application (BLA) to the FDA, where it is expected to take up to six months for the agency to review again. 

Patients living with this rare disease will not have access to this treatment until at least 2026, if ever. That’s the cost of delays and regulatory sandbagging. 

The Accelerated Approval Program is supposed to offer a glimmer of hope to people with these ailments and streamline ways to ‘treat serious conditions and fill an unmet medical need based on a surrogate endpoint.’ Unfortunately, Prasad is a known critic of accelerated approvals and the FDA’s role in the process. 

In 1850, political theorist and economist Frederic Bastiat wrote in "What Is Seen and What Is Not Seen" about the unknowable costs of government getting it wrong on market interventions and regulation. It’s also known as ‘dynamic opportunity cost.’ The downstream effect of FDA’s inconsistencies aligns with the principle that roadblocks disincentivize investment in next-generation therapies. Potential breakthroughs never move from the whiteboard to the lab, and never reach patients in need. 

It can cost well over $1 billion to bring new therapies to market, with large amounts of a company’s money being spent on research and development for clinical trials, all predicated on firms knowing what the FDA process will be like. The costs are not contained to the biotech world or even families; there’s also the fact that China is nipping at the heels of the U.S. in medical technology. It’s better for Americans to have a system that can set the pace in the global race for cures, rather than looking to Beijing for new treatments. 

The FDA’s disarray has left Duchenne patients, Sanfilippo patients, and countless others stuck in the waiting game. Whether it’s under Prasad’s leadership or someone else's, FDA should be prioritizing patients’ needs by establishing clear timelines for review of new treatments, setting predictable standards, and embracing accelerated pathways whenever appropriate. 

Elizabeth Hicks is the U.S. Policy Analyst with Consumer Choice Center.

Image: FDA