The drug industry blames the Food and Drug Administration for driving up the research and development costs of new drugs, stifling innovation, and interfering with the sainted mandate to bring cures to suffering medical patients.
The nation's public research agencies, especially the National Institutes of Health, complain they're starved for money.
Put those together, and you get the elements of what drug industry watchdog Ed Silverman calls a "grand bargain": Congress will step up funding for the NIH in return for a loosening of regulatory standards at the FDA. Silverman thinks this is an offer the American public should refuse. He's right.
Nevertheless, a deal is in the making on Capitol Hill. Last month, Senate Health Committee Chairman Lamar Alexander (R-Tenn.) announced "progress" on stepping up funding for NIH initiatives on cancer research and other important goals. He tied that funding implicitly to passage of a version of a 2015 House bill that would provide $8.8 billion for the NIH but also loosened the FDA's reins on the drug industry.
But as many experts have observed since the House passage, increasing funds for the NIH is a good idea. Loosening the FDA's regulatory authority is a terrible idea. Tying the two together is even worse.
Let's take the easy part first. Properly funding the NIH is crucial to the nation's scientific future. This month, the agency's director, Francis Collins, noted that after a period of increasing resources though 2003, the NIH budget went stagnant. Among other problems, it was sliced by $1.5 billion in mid-2013 by the sequester, "from which we have really not quite recovered," he said. NIH grant applications are at their lowest rate of approval in history, he said, with only 16% obtaining funding.
So, yes, the NIH needs more money. But holding the FDA hostage won't help anyone.
The House bill, which was dubbed the "21st Century Cures Act," turned the clock back on numerous standards applied by the FDA to trials of new drugs. Several of these amounted to what Daniel Carpenter, an FDA expert at Harvard, termed "the 19th century frauds act."
The bill would allow "evidence from clinical experience" to be used to justify approval of a new use for an existing drug. This means, essentially, treating anecdotes from doctors as clinical evidence. That's dangerous because such "evidence" easily can be cherry-picked, misunderstood or misleading; it's no substitute for randomized clinical trials, the gold standard in drug testing.
"A homeopath would love this provision, and, I’m sure, so would drug companies," David Gorski, an oncologist and prominent debunker of pseudoscience and medical nostrums, wrote last year. "Why bother with the time, bother, and expense of those pesky clinical trials to get your drug approved for additional indications, when you can rely on clinical experiences?... Indeed, the one thing this provision most definitely does not do is to speed effective treatments to patients. Rather, it smacks of being a payoff to pharmaceutical companies."
The measure also would ease standards for introducing new antibiotics, even if they haven't been tested on humans, as long as they're directed at "serious or life-threatening infections ... in patients with unmet medical needs." That's too loose a threshold to be applied to a category of drugs that already has led to proliferating drug-resistant infections. Doctors already are too free in handing out antibiotics; taking the leash off is likely to cause more problems down the road.
The measure also narrows the mandate that human subjects give their "informed consent" to participating in drug trials, especially in studies that pose "no more than minimal risk.”
That's "a major departure from current human subject protections," an alarmed Jerry Avorn and Aaron S. Kesselheim of Brigham and Women's Hospital and Harvard Medical School observed in the New England Journal of Medicine last year. "It is not clear who gets to determine whether a given trial of a new drug poses 'minimal risk,'" they wrote.
The biggest problem with the "Cures" act is its premise that the FDA is the bottleneck in drug R&D; if anything, the agency may be too accommodating. Avorn and Kesselheim reported that one-third of new drugs "are currently approved on the basis of a single pivotal trial" and "more than two thirds of new drugs are approved on the basis of studies lasting six months or less — a potential problem for medications designed to be taken for a lifetime. Last year, the FDA approved 89% of applications for new drug uses, according to three experts writing at Health Affairs; that made the agency less a bottleneck than a rubber stamp.
The risk from easing the FDA's regulatory standards will be borne not only by patients but also by the drug industry and medical profession. The "Cures" act would mean "not just going back to an era that was less safe, but an era that was less credible," Carpenter says.
The credibility of drugmakers and doctors is on the line when medicines are prescribed without adequate testing. That's what happened with Merck's painkiller and arthritis drug Vioxx, which the FDA approved in 1999 and was pulled off the market in 2004 after it was shown to raise the risk of heart attacks. By then, according to research in the British medical journal Lancet, 88,000 Americans had heart attacks from taking Vioxx, 38,000 of them fatally. The Vioxx fiasco cast a pall over drug sales generally for more than a year.
No evidence exists that overall medical innovation has been hampered by the FDA. Nor is there evidence that "it leads to higher drug prices or is costing lives," Carpenter says.
The evidence that the FDA saves lives is much stronger. One needs only to think back to what may be its finest moment, when FDA official Frances Kelsey fought in 1960 against allowing thalidomide into the U.S., despite off-label claims that it could treat morning sickness and was as safe as aspirin. "The original indication for thalidomide was based on exactly the anecdotal evidence this bill means to bring back," Carpenter says. Instead, the drug was found to cause severe birth defects in the children of women who had taken it during pregnancy.
Memories are short enough on Capitol Hill for America's narrowly averted thalidomide disaster, and even Vioxx, to be forgotten. But no one should want to relive that era.
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