Lawmakers in the House easily passed the 21st Century Cures Act today, a big step toward once again shaking up the legal framework built to guide drug development in the U.S. while significantly boosting the amount of funding that flows to the NIH.
As for the $9 billion being earmarked for the NIH--which comes along with more funding for the FDA--after years of research budget stagnation, there's virtually no debate. The bill's authors have found a way to pay for that by reining in some spending to Medicare prescription drug plans and selling off some of the crude oil in the country's stockpile, and Democrats are being won over by the notion of increased federal spending for more precise medicines. Research groups in the industry and academia are pushing this hard, and finding open arms in the capitol.
The political trip wire in the bill centers on changes to the way drugs and devices are developed and approved. Among the provisions in the bill, regulators would be given the authority to approve new antibiotics based on preclinical testing combined with small human trials. Biomarkers and other so-called surrogate endpoints could be used more easily to approve new drugs, offering developers a shorter clinical path to developing a drug. And the bill strips away some requirements for reporting physician payments from pharma companies, if they're associated with education--a very broad categorization.
BIO, of course, is giving its full-throated support to the bill, alongside the rest of the industry groups operating in Washington. The use of more surrogate endpoints is also routinely endorsed by R&D executives in biopharma. Consumer advocates, though, see the new development regulations as a recipe for unleashing drugs and devices that would later prove dangerous and unhelpful.
"The irony is calling this 21st Century Cures, when they're talking about standards that were left behind in the 20th century, because they were found to not be good," Diana Zuckerman, president of the National Center for Health Research, tells the Washington Post.
But there's also been notable opposition from some well-known experts in the field who aren't in the least bit happy about a switch in rules that would devalue the current gold standard for clinical development, which relies on randomized, well-controlled drug studies. Harvard's Jerry Avorn, for example, has a problem with "shorter or smaller clinical trials" for devices as well as new criteria for relying on "evidence from clinical experience," including "observational studies, registries, and therapeutic use" that could be used to allow for new uses of approved drugs. "Although such data can provide important information about drug utilization and safety once a medication is in use, there is considerable evidence that these approaches are not as rigorous or valid as randomized trials in assessing efficacy," Avorn writes.
Avorn disagrees with the whole premise of the bill, which is that there's a distinct need for accelerating the approval process. Currently, a third of all new drugs are approved based on a single pivotal study, with a median 760 patients per trial. Two-thirds of all drugs are approved based on trials lasting less than 6 months--including therapies intended to be taken for a lifetime. And the FDA is completing its evaluations generally inside a 6- to 10-month time frame--which is pretty impressive.
"In our rush to find new effective treatments, we should not harm our patients with ineffective toxic ones," writes JAMA editor Rita Redberg. That's the line that marks the boundary between supporters and opponents of the bill.
Perhaps most amazing of all is that Congress, which has been hopelessly gridlocked over the issues, has reached a point where there could be enough bipartisan support to get a bill like this passed. But if a bright spotlight of public attention is focused on controversial elements of the bill, large numbers of backers won't find it hard to switch sides in the final days of debate over the issues.