This week came more ominous signs that the removal of independent oversight at the Food and Drug Administration is producing predictable effects for investors and the companies they fund — and devastating ones for the patients those same investors have learned to treat as props.
UniQure announced Wednesday that the agency will now consider an accelerated approval for its Huntington’s disease gene therapy that had been blocked by prior leadership who took evidence seriously. The debilitating neuro-degenerative disease afflicts about 40,000 patients in the U.S. desperate for any treatment, and there are no current therapies that slow progression — which is precisely why those patients deserve the highest evidentiary standard, not the lowest. Rushing an unproven therapy through on weak evidence would be a betrayal of those patients, not a favor to them. The people most exploited by shortcuts in evidence are the dying, because they will accept anything if you call it hope.
Dismissed Commissioner Marty Makary and his biologics chief Vinay Prasad insisted on rigorous standards for approving the treatment. A three-year study showed it slowed progression by 75% compared to an external control group — external-control comparisons, historically the kind of design that has overstated treatment effects in past trials. Dr. Prasad demanded the kind of trial that separates real medicine from expensive hope: a randomized controlled trial with a placebo group undergoing sham brain surgery, the gold-standard method for distinguishing genuine benefit from wishful thinking when the procedure itself involves drilling into a patient’s skull.
The editorial page calls that demand “unethical.” We call it doing the job. A placebo arm with sham brain surgery is how you separate signal from noise when the stakes are a lifetime of degeneration. The discomfort of that design is the whole point — it is why the result means something. The job of a regulator is not to give patients what they want. It is to give them what works. Such a trial is the bare minimum of scientific rigor before exposing thousands of patients to an irreversible brain intervention.
When desperate patients, whose hopes had been raised by the advocacy push for approval, protested, a spokesperson for the agency accurately called them “the swamp.” A senior agency official who refused to be identified publicly — understandably, given the industry retaliation that can follow when a regulator demands proof — accused UniQure of pushing “distorted” and “manipulated” clinical data for a “failed” therapy. Dr. Makary stated in a TV interview that the treatment showed “no benefit” based on the clinical evidence.
Such independent judgment, evidentiary discipline, and refusal to be bullied by industry are exactly why the White House this spring gave Drs. Makary and Prasad the boot. Their habit of holding the line chilled the investment returns and deal flow the industry craves as it faces competition from China. Their insistence on scientific proof made investors uncomfortable — and investor comfort, not patient safety, is the currency this administration trades in. Acting Commissioner Kyle Diamantas so far looks to be doing a commendable job restoring the conditions that make that comfort possible while dismantling every protection the departed leadership erected.
He met this month with rare disease groups — the fox sitting down with the chickens, announced as reassurance. On Thursday the FDA celebrated a return to industry comfort by convening an outside advisory committee to review Moderna’s mRNA flu vaccine, which was 27% more effective than a standard flu vaccine in a large clinical trial. Dr. Prasad had overruled career staff by refusing to rubber-stamp Moderna’s application — which is, again, the job. He had properly insisted on adequate evidence by refusing to review the application until the data met the bar.
The advisory committee unanimously recommended approval. FDA advisory committees draw heavily from the same academic centres that receive industry research funding, and unanimity on a commercial vaccine application is the expected outcome, not a surprise. The mRNA platform could theoretically enable vaccines to better match flu strains in circulation, though that promise remains exactly that — a promise, not a demonstrated reality. The shots may produce a more durable immune response, but that too must be proven, not assumed. They could be available this fall if the agency grants approval soon, and investors will celebrate another quarter of deal flow and pipeline announcements.
The editorial page uses patients with a fatal disease as a prop for an investor-friendly agenda and calls it compassion. The people dismantling evidence requirements are not saving 40,000 Huntington’s patients. They are removing the only protection those patients have against expensive treatments that do not work. The patients deserve better than being a talking point for people who have decided that evidence is optional when the pipeline looks thin — and better than an agency that has decided the dying are an acceptable cost of keeping biotech share prices aloft.
