Why would anyone want the FDA to approve drugs without real evidence they work?

The same reasons people want other things they shouldn't. Money and ideology.

Dr. Vinay Prasad is in trouble again.

Prasad is the chief medical and scientific officer for the Food and Drug Administration. He and his boss, Dr. Marty Makary, who runs the FDA, are supposed to make sure new medicines work and are reasonably safe.

But Prasad has a problem. He takes his job too seriously. So he has made enemies on all sides — from profit-hungry drug companies who want him to approve medicines on dubious evidence and from left-leaning scientific reporters who will not forgive him for telling the truth about the dangers of mRNA jabs.

There’s nothing more dangerous than being an honest man in Washington.

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(Standing for the truth - and those who fight for it. With your help.)

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Prasad already got himself fired from the FDA once, last July, for standing up to a biotechnology company called Sarepta. Sarepta makes a therapy that costs up to $1.5 million a year and has never been shown to help patients. (You read that right.)

Ten days later, Prasad was back, after Makary and Robert F. Kennedy Jr. interceded for him with the White House. A cautious man might have learned his lesson and bent to the power of Big Pharma.

Prasad is not a cautious man.

In the last month, he has again infuriated the industry and its water-carriers in the media — not once but twice.

First, in early February, he tried to reject Moderna’s mRNA-based flu vaccine.

To be clear, the Moderna flu jab is junk, at best. I hope to write a longer piece about it shortly. But here’s the key takeaway: in the only large trial where full results are publicly available, it worked no better than a standard shot but had far worse immediate side effects.¹

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(Vinay’s got 99 problems, but telling the truth ain’t one.)

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Still, Prasad made a tactical error.

The politics around the mRNAs make opposing them publicly difficult, even if no one will take Covid shots any more. Trump still wants credit for Warp Speed, and legacy media reporters continue to insist the Covid jabs saved millions of lives.

Making matters even more complicated, Blackstone, the giant private equity company, has invested $750 million in Moderna’s flu shot. Prasad’s decision threatened to sink that entire investment, and Blackstone’s chairman, Stephen A. Schwarzman, is one of Trump’s biggest donors.

Given the stakes, Prasad needed an ironclad reason for saying no to Moderna. But he didn’t reject the shot after the agency reviewed Moderna’s full application. Instead, he said the FDA wouldn’t even review it. The reason he gave was that Moderna hadn’t tested its jab against the right older shot in its big trial.

Prasad is correct, but the decision still seemed technical and punitive. A wave of negative articles in the legacy media outlets that have been calling mRNA a miracle since 2020 followed.

A few days later, the White House forced Prasad and the FDA to back off, and the agency agreed to review the shot.

Prasad would have been better off promising to work with the FDA’s reviewers to consider the shot fully. If he and Makary did decide to reject it after full review, doing so because of its lousy side-effect profile would have been more defensible. It would also have made intuitive sense to anyone who has gotten sick after an mRNA Covid shot, aka almost everyone who has gotten an mRNA Covid shot.

Now the agency has little choice but to approve the jab. And millions of older Americans every year will pay the price in side effects.

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(The wages of spin)

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But Prasad keeps trying.

This week, he caused yet another storm, this time over a treatment for Huntington’s disease, a genetically caused brain disorder that is inevitably fatal.

A little-known biotechnology company called Uniqure has asked the FDA to approve a gene therapy for Huntington’s.

The therapy comes with serious risks, both because it requires neurosurgery so it can be injected deep into the brain and because the genetic changes it causes are permanent. Still, given Huntington’s horrors, many patients would probably accept the risks even though no one believes the treatment is fully curative.

Last September, Uniqure announced that the therapy had slowed the disease over a three-year-period in a tiny group of patients. The news caused its stock to soar five-fold and reporters (including many of the same reporters who praised the mRNA shots) to call the treatment a breakthrough.

But no one bothered to point out that years earlier, when Uniqure compared its therapy directly to a placebo — and gave the placebo patients a short “sham” surgery so they would not know if they had received therapy or nothing — it failed to work better than the placebo.

Its September announcement didn’t come from a placebo-controlled trial. Instead, Uniqure just compared the course of the disease in patients who’d gotten the therapy to how Huntington’s patients usually do — and said they declined more slowly than expected.

The problem here is obvious: the people willing to try an unproven brain surgery to slow a disease are likely highly motivated to beat it. They may also have better access to care, and the doctors who are encouraging them may believe they are likely to do well.

There is simply no way to measure for those factors, and so comparing the outcomes of the Uniqure patients to a made-up control group doesn’t prove anything. At best, it offers evidence that needs to be confirmed in a clinical trial — but the clinical trial that was run already failed.

(Further, the biology of Huntington’s suggests Uniqure’s treatment may just not work. The therapy may not reduce the protein it is targeting, and even if it does, that reduction may not have the positive effects the company hopes. If you are interested in more details, this article offers an overview.)

So Prasad and the FDA want Uniqure to run another trial. The company is refusing. Doing so would deprive dying patients of hope, its backers say.

But the FDA’s job isn’t to provide hope, particularly when a treatment itself can be dangerous — as the brain surgery required to inject the therapy obviously is. The agency’s job to make sure a treatment offers some benefit and isn’t too risky compared to the disease it is intended to treat.

If the treatment hasn’t been shown to provide any benefit, then the equation is simple: any risk is too much. Even for a terrible disease.

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(They can fire Vinay. They can’t fire me. Not as long as you’re with me.)

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Prasad doesn’t hate patients — or drug companies.

He’s just trying to stick to some minimum standards for drug approval.

1: If companies want the FDA to approve a new treatment where an older one already exists, they should have to show that it works at least as well as the older one and doesn’t have way worse side effects.

And if the older one doesn’t work very well — like the flu shot — they should also have to compare both the new and old treatments to a placebo in a three-armed trial.

2: If companies want the FDA to approve a new treatment where none exists, they should have to run a clinical trial against a placebo — even if the disease they are trying to treat is very serious. Arguably, it’s even more important to run placebo-controlled trials for such diseases, because patients and doctors deserve to know if a treatment works.

The only exception should be if a disease is so vanishingly rare — with a few hundred patients at most every year — that no clinical trial is reasonably possible. Unfortunately, that is not the case for Huntington’s disease, which affects about 40,000 Americans.

It’s easy to understand why drug companies and their investors want low standards for FDA approval.

If Uniqure can get its therapy through, it will be able to charge millions of dollars per-patient for it, and some patients will go for the surgery even if they aren’t sure it works. Moderna’s calculation is even more cynical. It will likely be able to charge high prices for its mRNA flu shot and then offer “rebates” (don’t call them bribes) to pharmacies and pharmacy benefit companies to make patients get them.

But what about the media?

In a simpler, happier time, Prasad could have counted on the investigative reporters who covered the FDA to help him, broadly. But after going all in on mRNA, they’re now stuck defending it, and their lack of skepticism has spread to other novel, expensive, high-risk biotechnologies — especially those with a genetic component.

They’re being used, but they hate Donald Trump too much to care.

1. Last June, Moderna claimed a second large trial showed the shot worked marginally better than the standard shot. The company hasn’t released any details, including on side effects, in the eight months since.

Further, no one knows how well the works against a placebo, or saline, shot, since the FDA didn’t make Moderna test it against placebo in a big trial. Those results might have been even more embarrassing for the company. Standard flu shots barely work, and Moderna’s shot is no more effective, but it likely would have looked even worse compared to a placebo on the safety side.

The details for the company are even worse than this topline description sounds and — as I mentioned above — deserve a fuller explanation. mRNA is a deeply troubled technology and probably should be shelved completely for any vaccines intended for healthy adults, much less children (as opposed to cancer vaccines). More to come.