In a quest to bring new medical products to Americans, Congress is considering a grand bargain.
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.
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.
“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.”
“I call it the 19th Century Fraud Act.”
Daniel Carpenter, Harvard University
The final details of the Senate bill remain uncertain, but some proposals are also prompting objections. As an example, one suggestion for speeding approvals of medical devices is to label certain products as “breakthroughs,” a designation that would offer an expedited review pathway. To qualify as a breakthrough, though, a company need only argue its product is either a significant advance over existing devices or is in the best interest of patients.
The use of the word “or” is problematic, because it creates wiggle room for unproven claims. “It’s very vague and only encourages companies to seek breakthrough status,” said Diana Zuckerman, who heads the National Center for Health Research, a nonprofit think tank.
There are other portions of the House and Senate proposals that merit criticism, but one doesn’t need to be a Washington insider to have concerns.
A new poll from STAT and the Harvard T.H. Chan School of Public Health found that 58 percent of Americans oppose changing safety and effectiveness standards to allow for faster approvals of new medicines, and half of the public also objects to regulatory changes to accelerate approvals of new medical devices.
Dr. Robert Califf, the FDA commissioner, may not have participated in the survey, but he expressed similar sentiments earlier this month. In a speech at an industry conference, he said that, if the legislation is not “carefully crafted, [it] could pose significant risks for FDA and American patients.”
“Innovative therapies are not helpful to patients if they don’t work, or worse, cause harm,” Califf told the gathering.
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.
There is no question that more research funding is necessary and that finding legitimate ways to get medicines to patients faster is crucial. But Congress ought to separate the debate over research funding from the rest of the legislation. Loosening regulatory standards would only create problems for which real cures will be needed.