By Ed Silverman, STAT News
May 17, 2016
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, said.
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. […]
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