When Americans imagine the future, it always has a lot to do with flying cars, robot housekeepers, and just about anything else first seen on TVs, “The Jetsons”. When it came to healthcare, the Jetson family offered an early glimpse into telemedicine and robotic pills that, when taken, could diagnose just about any internal ailment, but they didn’t ever go so far as suggesting a future without the 7-day prescription pillbox.
One of the latest medical breakthroughs is a “one-and-done” gene-editing therapy that slashes LDL cholesterol (the bad cholesterol) by at least 69 percent, something that could transform treatment for millions living with heart disease.
It is, in fact, a one-dose treatment that frees patients from a lifetime of daily pills. Could Jane Jetson even imagine?
It’s the kind of innovation that you’d expect policymakers and regulators to race to get into the hands of patients, but given the present situation at the FDA, that’s far from guaranteed. The key sub-agency responsible for evaluating gene-editing therapies, the FDA’s Center for Biologics Evaluation and Research (CBER), is now led by Dr. Vinay Prasad, a man who has spent much of his career criticizing the accelerated approval pathways that helped bring us mRNA vaccines, CAR-T cancer treatments, and gene therapies like Verve’s. One of the agency's top regulators on gene therapies, Nicole Verdun, was shown the door this week.
Dr. Prasad stated recently that he doesn’t plan to revisit past approvals, but what he does next could make or break the future of biopharmaceutical innovation in the United States.
Someone in the U.S. dies from cardiovascular disease every 33 seconds, so when advocates like myself say that the stakes are high, it’s hardly an exaggeration. Traditional cholesterol drugs work fine, but they require lifelong discipline and don’t address the root genetic causes for many high-risk patients.
These individuals can maintain a healthy diet and regular physical exercise, but a suboptimal genetic predisposition for high LDL cholesterol creates a situation where the uphill battle has no real end in sight. If there’s an alternative, that choice is worth having.
An effective gene-editing treatment can dramatically improve lives by permanently deactivating the gene that drives LDL cholesterol to abnormally high levels. The Jetsons had some cool telemedicine technology, but this is next level.
Not only are health outcomes likely to improve, but out-of-pocket costs would drop, and patients would spend less time in doctors' offices and pharmacies. It’s the kind of outcome most pharmaceutical ads are always projecting – the patient living life, not managing medications.
The problem here is that the FDA is struggling. DOGE cuts led to mass layoffs across federal health agencies, with the FDA itself losing roughly 3,500 employees. This has resulted in a regulatory traffic jam where meetings with drug manufacturers are canceled, clinical trial feedback is delayed, and borderline radio silence is the norm from agency staff.
There is a point at which efficiency in the form of personnel cuts becomes total stagnation.
Under the direction of Health and Human Services Secretary Robert F. Kennedy Jr., the trick is balancing his skepticism of vaccines and emerging biotech innovations with a quick, predictable approval process.
Grinding everything to a halt doesn’t just threaten patients who are waiting on solutions for their ailments; it also disincentivizes medical innovation of any kind by the companies that invest capital in those breakthroughs. This is why accelerated approvals are so crucial for both parties.
America is still the global engine for medical technologies, but it’s beginning to lose altitude. If we want to maintain our top status, then we need an FDA that supports innovation as a matter of principle and leadership that understands the value of a transparent process. As it stands today, FDA approval has become a black box.
Breakthrough medicines should reach the market as quickly and safely as possible. Red tape and bureaucracy are the reason we don’t have the Jetsons’ flying cars, and we shouldn’t settle for a future where better cholesterol medication is kept in limbo, too.
