Regulatory Affairs Professionals Society – With an executive order (EO) and comments from President Donald Trump yesterday calling for a massive overhaul of US Food and Drug Administration (FDA) regulations, legal experts and former FDA officials are offering some practical and creative ways the agency could clean house.
And before this dive into what FDA might or even could do following the signing of this EO, it is important to remember a constant refrain from all experts Focus spoke with: FDA codifies statutes passed by Congress and many deal with processes that make clarifications for industry.
Erika Lietzan, an associate professor of law at the University of Missouri School of Law and former associate general counsel of industry group PhRMA, told Focus via email: “The EO does not – could not – change the agency’s mandate from Congress. In addition, I don’t see why the agency’s interpretations of the statute or its regulatory policy would change. If the agency had to remove its NDA [new drug application] regulations, for instance, that doesn’t change the fact that under the FDCA [Federal Food, Drug, and Cosmetic Act] a new drug cannot be introduced into interstate commerce without an approved new drug application that is held to the statutory safety and effectiveness standard.”
And Lietzan does think there are regulations FDA could remove with little impact on industry or public health.
“I think there are probably quite a few regulations on the books that could be removed,” she said. “21 CFR Part 99, for instance, contains 15 regulations, and the statutory authority for the regulations sunsetted. As explained on page 6 of this draft guidance, those regulations are no longer applicable. So that’s 15 right off the bat. I think there are quite a few others that are dated, no longer used, and so forth. If the EO applies to guidance documents, the same thing is probably true; I suspect there are numerous old guidance documents that have not been (but should be) formally withdrawn.”
But from what President Trump said Tuesday to pharmaceutical CEOs, his plan for FDA does, at least at face value, seem to be about more than just cleaning up outdated regulations.
Aaron Kesselheim, an associate professor of medicine at Harvard Medical School, told Focus: "I think it depends which regulations and guidances we're talking about eliminating. If there are vestigial guidances that are on the books but have been rendered moot or superseded, then removing them would not have much impact. But from what President Trump said, it doesn't sound like he means those."
Others also think FDA already does a decent job of cleaning up, at least outdated guidance documents, though the agency could take a creative approach to rescinding regulations.
Bob Pollock, former acting deputy director of the Office of Generic Drugs at FDA, told Focus to “remember that each year the FDA removes or withdraws outdated guidance documents - very seldom do they withdrawal regulations ... Most of the regulations don’t really deal with safety or effectiveness but deal with process. And they simply codify statutes passed by the Congress. So it is Congress that would need to stop passing statutory bills or rescind some laws to help out the agency meet this ridiculous target.”
Erick Turner, former FDA reviewer of psychotropic drugs from 1998 to 2001, told Focus FDA could rescind regulations that require drug safety or efficacy problems to be redacted from public view. Or FDA could rescind regulations on its clinical hold tribunals.
“If you are concerned about the safety of a first-in-human clinical trial and your review group puts the IND [investigational new drug] on clinical hold, you have to go before a tribunal and defend your decision,” Turner said. “The holds tribunals can only discourage clinical holds, which are there to protect patients.”
As for the EO’s wider implications, Lietzan said she expects it could hamper industry’s understanding of FDA’s interpretations of laws set by Congress, which will likely be key to industry understanding how FDA will apply provisions from the recently passed 21st Century Cures Act.
“So I think the real issue would be lack of certainty for regulated industry and lack of transparency to the public,” she explained. “For instance, the agency has been slow to issue guidance to implement the biosimilar statute (though several things have come out in recent months), and in my own view aspects of it would benefit from rulemaking. Obviously FDA will continue to hold biosimilar biologics to high standards, will continue to provide biosimilar sponsors advice in meetings, and will continue to approve biosimilars when it is confident they satisfy the statutory standard.
"The lack of regulations (and guidance, if this also precludes guidance) will simply mean that industry has to deduce the agency’s policies and views by studying its case-by-case decisions – its approval decisions, its enforcement actions, and so forth," she added. "Also, the underlying interpretations and policy calls will not benefit from the input of a wider stakeholder group.”
But the “one out, two in” plan does not seem to make sense in the long term as FDA and other agencies would eventually run out of regulations to cut.
“Obviously, the math is what it is; if the rule is 2-for-1 indefinitely, eventually we end up with zero. But the EO instruction seems to be labeled just for fiscal year 2017. I suspect that FDA can clean up for a year or two, and the overall apparatus will be leaner and cleaner as a result,” Lietzan said.