21st Century Cures Drug Bill Triggers a DC Dust-Up Over Relaxed Development Regs

John Carroll, Fierce Biotech: July 10, 2015

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. […]

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.

“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.

Read the full story here.