Members of the U.S. House of Representatives were patting themselves on the back after an overwhelming bipartisan vote for the 21st-Century Cures Act, a bill that promises to accelerate the approval process for new drugs and treatments.
By a vote of 344–77, the House approved a relaxation of restrictions on FDA regulators, enabling speeding up of clinical trials, including allowing the use of data from case studies, patients’ experiences, other countries and medical journals. Medical technologies that qualify as “breakthroughs” will be put on a fast track for approval.
Some $8.75 billion in additional funding for research at the National Institutes of Health over the next five years, and $550 million more for the Food and Drug Administration over the same period, will finance the ambitious streamlining. The Senate will be taking up the issue in the fall.
Maryland Rep. Steny Hoyer, the No. 2 Democrat in the House, hailed it as “the most significant investment in biomedical research in more than a decade.”
“It showed we could come together,” Rep. Fred Upton (R-Mich.), chairman of the House Energy and Commerce Committee and a co-author of the bill, said after the vote.
But the back-patting was not universal. Critics of the legislation thought the bipartisan unity was misdirected, both in terms of public health and federal financing.
Conservatives objected to provisions that would automatically send billions to the NIH and FDA, on top of — not part of — overall congressional spending on domestic programs. They were not mollified by the proposal that most of the money would come from selling $7 billion worth of oil from the Strategic Petroleum Reserve, the government’s emergency stockpile. Only $2.5 billion would derive from cutbacks in federal Medicaid reimbursements to states.
The consumer group Public Citizen charged that it offered a gaudy package of “perks to the pharmaceutical and medical device industries” and called it “an assault on patient safety and science.”
Dr. Aaron Kesselheim, a Harvard University medical school professor, said it would “unnecessarily allow drugs and devices to be approved based on very limited data and unnecessarily accelerate products to market before we know enough about their safety and effectiveness.”
The underlying notion that current FDA approval processes are too slow was a “false assumption,” said Kesselheim.
But for patients whose lives are at stake, the 8–12 years that it now takes for an experimental drug to get from the laboratory to the pharmacy is too slow, and they would no doubt welcome a significant acceleration of the process, no matter what the professor says.
Of course, desperation is not an argument, and impatience alone is not a justification for change. Then, too, one must always be leery of politicians declaring that “now patients have hope,” as some were doing. If they could garner votes with snake oil cures there are those who would do that too.
But the Cures Act isn’t a snake oil cure. It promises no phony remedies or quickie approvals, but a reasonable streamlining of the regulation of a rapidly changing industry.
No one is suggesting that the meticulous, multi-phase clinical trial regime supervised by the FDA should be done away with or drastically shortened. But the need to upgrade the long-stagnant funding of American medical research and to streamline the approval process for innovative therapies is real.
Nor are the supporters of the bill limited to politicians and drug lobbyists. There are medical professionals in favor of it, too.
“Thousands of chronic and deadly diseases, for which we have no treatments or cures, cost billions of dollars and claim millions of lives every year,” said Rep. Andy Harris, a Baltimore Republican and Johns Hopkins-trained anesthesiologist who wrote much of the bill. “This bill is our chance to invest in promising breakthroughs and real cures.”
It could just be that, in this case, the emotions of patients and the interests of biomedical research institutions really have come together, along with Democratic and Republican congressmen. It may just be that in the case of the 21st-Century Cures Act, faster really is better.
In the interests of patient protection, though, a middle way might be found that would satisfy those who are warning that the bill trades off responsible science for speed.
A special class of tentative approval could be created for breakthrough therapies that will have come along the new, fast track. Mandatory disclosure of the accelerated trial history would enable doctors and patients to then make an informed decision as to whether the added risk is acceptable.
It’s not clear yet what the Senate has in mind. Its version of the bill hasn’t been written yet. But it’s expected that it will move through the upper chamber and arrive on President Barack Obama’s desk for signing before the end of the year.
That’s pretty fast, and that’s not bad.