National public health policy under the leadership of Department of Health and Human Services Secretary Robert F. Kennedy Jr. made no shortage of headlines in 2025. As a short list, RFK made controversial statements about vaccines, vowed (but failed) to find the root cause of autism, initiated efforts to ban some food dyes, cut funding, cut employment, misused AI, and tried to eliminate medical scientists from publishing in select academic journals based on their funding. Less importantly, he also wears jeans when he works out.
In contrast, the Food and Drug Administration, easily the most powerful sub-agency overseen by the HHS and RFK, has had a quiet 2025. It began the process of banning red dye #3 in food products, reassessed some safety standards for cosmetics, and implemented a rule now requiring complete safety disclosures in drug advertisements.
Most of the FDA’s planned major reforms are still underway. Currently, the agency is opening pathways to more quickly approve biosimilars, generic versions of complex drugs, and a new approval process for “gene-editing” treatments. Perhaps its most ambitious deregulatory effort is to approve new drugs in 1-2 months using a “plausible mechanism pathway” coupled with AI as part of a National Priority Voucher Program.
Slashing approval steps and bureaucratic oversights should bring drugs to the market and to patients faster. But these programs also require government employees to select drugs and their producers for swifter approval and special consideration. That’s grounds for rent-seeking. Record-breaking lobbying totals from the pharmaceutical industry in 2025 signal this is a genuine concern.
Will the FDA’s recent actions lead to genuine deregulation, or will they succumb to regulatory capture? A recent petition might tell us.
Pyruvate Dehydrogenase Complex Deficiency (PDCD) is a rare genetic condition that causes excessive lactic acid buildup because the body is unable to convert carbohydrates into energy, often resulting in extreme fatigue and neurological issues. Some PDCD develops in adulthood. The condition is considerably worse when it emerges in utero, leaving many infants and children with debilitating metabolic and neurological conditions.
Tragically, the condition is often fatal, and treatments only exist for the symptoms. But there may be an exception.
Sodium Dichloroacetate (usually shortened to DCA) has unique properties that help absorb carbohydrates and reduce lactic acid buildup. As highlighted in peer-reviewed research, DCA can prevent fetal lactic acid buildup—which can literally save lives. Although relatively few studies have investigated DCA and PDCD, dozens of parent testimonies provide eye-opening evidence.
Research and testimonies aside, DCA is currently classified as an “investigational compound,” existing in a peculiar regulatory gray area within the FDA’s framework.
PCDC is rare enough (affecting about 1 in 40,000 births) that garnering enough participants for small-scale clinical trials is challenging and highly time-consuming. Without these clinical trials, it is nearly impossible to receive orphan drug status (which ironically exists to help drugs used to treat rare disorders receive approval). Unless PCDC passes its phase 1 trials, it is not eligible for right-to-try access.
The FDA is no stranger to DCA, its situation, or the promise it provides. After receiving a new drug application in December 2024, the agency granted it priority consideration. In September 2025, DCA received a formal rejection and was asked to provide additional data and to conduct new clinical trials. DCA’s producer requested a meeting to discuss potential pathways forward. Their request was answered with a meeting. An accompanying petition signed by more than 80 established medical researchers and physicians surely helped.
If new and/ or accelerated pathways to drug approval can’t find room for DCA and what it represents, what exactly are these pathways for?
For the first time since the COVID-19 pandemic, the FDA finds itself under pressure from the medical field and the broader public to deregulate. It has taken several initial steps in this direction, but now faces its first real test of committing to genuine deregulation as a way to help patients. Let’s see if it passes.