The Good, Bad, and the Ugly: FDA Edition

It’s been a wild few weeks at the Food and Drug Administration (FDA). The agency, which spends about $7 billion per year on evaluating new medications, scrutinizing reduced-risk products such as e-cigarettes, and ensuring food safety, has been widely criticized for limiting vaccine availability for millions of Americans. In addition, the FDA drew the ire of thousands of Duchenne muscular dystrophy patients by erratically stopping shipments of a key treatment before suddenly resuming them. But at the same time, the FDA is making strides toward more transparency by giving consumers access to more information on drug denials. It’s hard to keep track of all the goings on at the FDA, but fear not. The Taxpayers Protection Alliance (TPA) has been closely following agency developments, good, bad, and ugly:

The Good

FDA Starts Telling Patients its Reasons for Drug Denials

For decades, the FDA has been notoriously tight-lipped about why it rejects drugs. The FDA has remained all-but-silent even when the agency rejects the advice of its advisory committees and refuses to approve life-saving drugs. Fortunately, this unwarranted secrecy is (seemingly) no more. On July 10, the FDA announced it was embracing “radical transparency” by publishing more than 200 complete response letters (CRLs), which typically specify why medications are rejected. On September 4, the agency announced it’s releasing even more CRLs, including doing it in real time (i.e., right after a drug gets rejected). These letters are already offering a glimpse into the often-bizarre decision-making plaguing America’s drug regulator.

For example, in 2023, the agency rejected a medication called Legubeti (to treat acetaminophen overdoses) because of “an unpleasant odor that may affect the tolerability of oral ingestion.” While the FDA ultimately approved the medication the following year, the odious regulatory delay very likely cost lives. Acetaminophen (e.g., Tylenol) overdoses result in about 500 deaths annually in the U.S., and delays over trivialities are a reckless exercise in regulatory foot-dragging. The well-known diabetes medication metformin remained on the market even though patients complained that it smells like “dead fish.” With that kind of precedent, surely smell and taste shouldn’t be an issue in approving other life-saving drugs.

Hopefully, the FDA continues this increased transparency and tells patients why they can’t have access to new therapies.

The Bad

FDA Claims New Approval Pathway, But It’s a Nothingburger

On September 3, the FDA “introduced the Rare Disease Evidence Principles (RDEP) to provide greater speed and predictability in the review of therapies intended to treat rare diseases with very small patient populations with significant unmet medical need and that are driven by a known genetic defect.” That sounds like it belongs in the “good” section, but the news isn’t as encouraging as it sounds. According to an analysis by Paul Kim, advisor at the National Organization for Rare Disorders, the new RDEP “is all wrapper and no gift.” 

Kim explains, “the webpage simply regurgitates what FDA has historically done for very rare and n-of-1 therapies. Nothing is new in the discussion of confirmatory evidence – external controls, natural history studies, non-clinical models, case reports, patient experience data. FDA’s willingness to accept one trial with confirmatory evidence to approve drugs is well documented – and in statute.”

Meanwhile, the FDA continues to reject promising therapies. It recently rejected a biologics license application for bevacizumab-vikj (ONS-5010) to treat wet age-related macular degeneration, an affliction that impacts 20 million Americans. The medication was rejected despite promising data from clinical trials, which was sufficient for regulators in the EU and the U.K. The FDA should commit to a more flexible and forward-thinking approval process. 

The Ugly

FDA Hampers Vaccine Access for Millions of Americans

Recently, the FDA announced that it has approved the latest iteration of COVID-19 vaccines, but with new restrictions. Approval is now limited to seniors and younger Americans with at least one underlying health condition that increases their risk of severe COVID-19 infection. The FDA is also demanding that every new iteration of a Covid shot “use a true placebo control (salt water) so we will learn the side-effect profile.” These changes will drastically limit patient choice, hamper health, and raise costs for taxpayers.

Because the FDA has limited vaccine approval, millions of Americans now require prescriptions to get boosters—instead of being able to walk into a CVS and Walgreens and get a booster as they choose. While people can disagree whether zero boosters or five is the “right” number to get, that decision should be for patients, not bureaucrats, to make.

These boosters will also be more expensive because of the bolstered testing requirements. Experts such as Northeastern pharmaceutical sciences professor Mansoor Amiji note that “placebo trials can last for months or years” and can therefore be immensely costly. These costs aren’t just a concern for vaccine makers. The history of drug approvals—including vaccines—shows that requirements needlessly imposed by the FDA are ultimately passed along to consumers. It’s no coincidence that drug approvals can cost more than $2 billion per medication, and median list prices for new drugs are high and rising as regulations pile up. Taxpayers also foot this bill through pricey government healthcare programs such as Medicaid, which costs Americans nearly $1 trillion annually. More expensive healthcare directly fuels the $37 trillion national debt.

The FDA’s new vaccine policies will increase healthcare costs and make millions of patients’ lives more difficult.

Conclusion

The FDA has promised Americans a new and better approach to healthcare and drug approvals. So far, its track record is far from promising. TPA will continue to call on the FDA to embrace transparency and improve access to lifesaving treatments.