The 21st Century Cures Act: Still alive, and still poised to endanger patients

it was less than a year ago that I described a bill wending its way through Congress called the 21st Century Cures Act “old vinegary wine in a new bottle.” The reason I characterized the bill that way was because it really was nothing new and it rested on a very old fallacy, namely that the only way to speed up medical “innovation” is to weaken the FDA and its standards for drug and medical device approval, which is exactly what the 21st Century Cures Act would do if passed into law. It’s basically the American cousin to the British Saatchi Bill, which in essence proposed to do very similar things, but even more so, so much so that Andy Lewis referred to it as the “quack’s charter.” Fortunately for the British, the version of the bill that ultimately passed bears little resemblance to the original Saatchi bill and lacked the most onerous removals of patient protections. I’m not so optimistic that the American Saatchi bill will so watered down.

It turns out that the 21st Century Cures Act is back in the news again. Having been passed by the House of Representatives last year, the 21st Century Cures Act is now up for consideration in the Senate, as Ed Silverman tells us:

Legislation passed last year by the House would provide billions more dollars for medical research and encourage faster approval of prescription drugs and devices. The Senate, meanwhile, is currently working on a set of companion bills in hopes of crafting a compromise measure.

“If we succeed, this will be the most important bill signed into law this year,” Senate health committee chairman Lamar Alexander, a Tennessee Republican, said at a committee meeting last month.

This sounds promising. After all, adding $9 billion to the National Institutes of Health’s budget over the next five years to underwrite new cures is a good idea. And in an era when desperately ill patients are clamoring for new medicines, giving the Food and Drug Administration extra tools also makes sense.

But there’s a catch. By linking the extra funds to speedier approvals, Congress appears ready to undermine regulatory standards. And this is a misguided notion that, unfortunately, is more likely to help companies than patients.

When I wrote about this misbegotten bill, I pointed out just this aspect of it. What I perhaps didn’t emphasize enough, is that the bill is in essence a “grand bargain,” as Silverman puts it. The bargain is this: Congress will step up funding for the NIH in return for a loosening of regulatory standards at the FDA. Not surprisingly, Silverman thinks that this is a bargain the American people should turn down. Even less surprisingly, I agree even more strongly.

As I pointed out last year, the NIH budget could really use an increase. After the doubling of the budget that occurred between 1998 and 2003 under Presidents Clinton and Bush, the NIH budget has remained more or less flat when adjusted for inflation. In fact, it’s worse than that. As NIH director Francis Collins noted, the NIH budget was cut by $1.5 billion in 2013 during the budget sequester, a blow “”from which we have really not quite recovered,” pointing out that approval rates of NIH grant applications are at their lowest rate in history. That’s because, since 2003, there has been a 20% decline in NIH budget adjusted for inflation. In essence, what the 21st Century Cures Act does is to hold the FDA hostage. In return for changes in the FDA that align with industry desires and those of an ideological bent to believe that the primary, overwhelming reason why the pace of pharmaceutical and device discovery has declined and costs have gone up so much is because of an overweening state, says Lamar Alexander, the Senate sponsor of the 21st Century Cures Act, we’ll fund the FDA to a greater level.

Unfortunately, as I discussed last year, the NIH and by extension the American public don’t get very much for giving up a lot, as this is a bad deal. That’s not to say that there aren’t some good things. For example, the 21st Century Cures Act doesn’t add that much to the NIH budget, a 3% increase per year for three years plus $1.86 billion a year “innovation fund” to support precision medicine initiatives and young scientists. Given that the current budget of the NIH is approximately $32 billion, 3% per year is less than $1 billion a year, which means that nearly two thirds of the increase is spoken for, much of it for “precision medicine.” Having been around, I’m also cynical enough to doubt that the part of the funds allocated to “young investigators” will actually benefit young investigators as much as Mr. Alexander thinks it will. If Mr. Alexander is willing to pump $2.86 billion a year for three years into the NIH, it would do a lot more good if there were no strings attached to the budget increase.

Perhaps the worst part of this mostly execrable bill (I find it hard to call a bill that increases the NIH budget significantly totally execrable) is this:

For instance, the House bill, which is known as the 21st Century Cures Act, would allow the FDA to approve added uses for a drug without relying on a randomized clinical trial — the gold standard for determining whether a medicine can benefit patients and the extent to which there are serious side effects. Instead, the bill would permit the agency to base such decisions on “clinical experience,” a loosely defined term that means, essentially, anecdotal observations from physicians and patients.

To be fair, the FDA would not have to consider such information. And the Senate has, so far, not adopted this notion. But this is hardly the sort of rigorous scientific data that should be used to establish whether a drug is safe and effective.

I remember what I said about this at the time about this provision. To paraphrase, I said that homeopaths would love this provision, and so would drug companies. I asked: 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 based on therapeutic use, uncontrolled observational studies, or registries instead?

Why indeed?

As I said before, if I were the CEO of a pharmaceutical or medical device company, I’d love this bill. 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.

I was happy to find someone who is even harsher in his assessment of this bill than I am, namely Harvard University political scientist Daniel Carpenter, who is quoted as saying:

“This is a harsh way of putting it, but this is why I call it the 19th Century Fraud Act,” said Harvard University political scientist Daniel Carpenter, who studies the FDA. “This is a part of the bill that threatens to take us back more than a century.”

Exactly, but it’s even worse than that. The House version of the bill explicitly weakens patient protections in clinical trials. It would accomplish this by creating another category of clinical investigation where it is acceptable to forego informed consent. It accomplishes this by saying informed consent is not necessary when “the proposed clinical testing poses no more than minimal risk to the human subject and includes appropriate safeguards to protect the rights, safety, and welfare of the human subject.” Informed consent is a central tenet of all clinical trials in all countries that do them. This might—might—be defensible if the act defined what constitutes “minimal risk” or states who would determine whether a study is “minimal risk.” A charitable interpretation is that an institutional review board (IRB) would determine this. Even so, it’s a disturbing provision, and the number of studies that would be facilitated by such a change in the requirements for informed consent is likely to be vanishingly small.

The bill goes beyond even this unfortunately. Basically, the 21st Century Cures Act is Christmas in May, with Santa Lamar bestowing all sorts of gifts on the usual suspects. For instance, there’s a provision that sidesteps the Sunshine Act, adding an exemption for reporting medical education payments, which has the effect of increasing the number of things drug companies can pay doctors for without having to report them to the physician payment Sunshine database. One wonders how such a provision contributes to “medical innovation.” Actually, one doesn’t, because this provision contributes nothing. It’s a sop to medical groups who don’t like transparency.

Let’s take the arguments of proponents of the 21st Century Cures Act at face value for a moment. The central assumption behind the bill is that, for greater medical innovation at a lower cost to let the medical advances flow to a grateful populace, the FDA needs to be reined in. Of course, what the best level of regulation is to balance cost and time to approval versus guaranteeing the safest, most efficacious devices and drugs. It’s such a seductive idea, and you don’t have to be a batshit nutty government-hating libertarian to feel the attraction. The idea seems so…logical.

Here’s the problem. The premise is wrong. It turns out that such radical reform involving weakening the FDA is not needed. The FDA already has the tools to do what the 21st Century Cures Act demands without weakening patient protections or scientific rigor. In fact, the FDA, despite being underfunded, is actually pretty efficient at new drug approvals, evaluating nearly all new drug applications within 6 to 10 months, an impressive turnaround for such complex assessments. It’s been pointed out that the FDA actually acts more rapidly than European regulatory agencies. Basically, there is no evidence that the FDA hampers overall medical innovation, nor is there evidence that the FDA’s current requirements lead to higher drug prices or cost lives.

Michael Hiltzik points out that the 21st Century Cures Act is not just bad for patients but bad for industry:

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.

There is no doubt that the NIH is criminally underfunded right now. That is a separate question from what the appropriate level of regulation is for the approval of drugs and devices. Linking the two is nothing more than a cynical ploy to make weakening of the FDA, or at the very least encouraging FDA administration to weaken drug approval standards, palatable. Silverman describes this cynical conflation well:

The Republicans in Congress know what they are doing. By bundling NIH funding with faster FDA approvals, the lawmakers are making it easier to win support from universities and small biotech companies, which rely on government research grants. (In a pointed blog post last fall, University of Pennsylvania President Amy Gutmann wrote that Congress can “change the course of history” by approving the measures.)

They are also appealing to many patients and their families — an increasingly large and vocal group of Americans — who want the FDA to approve more medicines for unmet medical needs.

The 21st Century Cures Act is cynical politics played to increase pharmaceutical company profits. It is not, nor has it ever been, about protecting patients. Although its advocates genuinely believe that its purpose is to bring cures faster to patients who need them, the 21st Century Cures Act will do no such thing, and tying changes in the FDA regulatory framework to increasing NIH funding is the ultimate cynical political ploy to gut the FDA and turn back the clock on drug development at least 50 years.