Keeping Score In Drug Development And Innovation Game? Look To US FDA As The Referee – Califf
Executive Summary
“It's really the elected officials who are making the final determination on what happens. They're really the owners of the teams, you guys in the industry are playing the game we’re refereeing,” FDA Commissioner Robert Califf says at Consumer Healthcare Products Association conference.
The US Food and Drug Administration’s work, says Commissioner Robert Califf, is “refereeing” the game regulated industries are playing according to the rules set by Congress, the owners of federal regulations.
“The more I look at it, the FDA is really a group of referees. We don't write the rulebooks, and it's a fascinating part of what we do that Congress basically writes a rule book and puts in laws,” Califf said on 17 September.
“In the end, Congress has to pass a law or not. And during that final passage, it's really the elected officials who are making the final determination on what happens. They're really the owners of the teams, you guys in the industry are playing the game we’re refereeing,” he added in a pre-recorded presentation to open the Consumer Healthcare Products Association’s regulatory conference in North Bethesda, MD.
robert califf: "The drug development process, driven by the laws and practices developed mostly in the United States, is one of the most elegant aspects of science."
Source: FDA
The commissioner in his second stint at the FDA helm sees the agency’s refereeing factoring prominently in doing work supported by user fees.
“We can justify the business sense what it costs to do the refereeing that's needed and to do it in a way that is unbiased, and that gets substantiated by the law, and then you play the game, and we do the refereeing, at least that's the way I see.”
Califf, speaking with CHPA president and CEO Scott Melville, also noted that the best is yet to come for the FDA’s Over-the-Counter Monograph Drug User Fee program.
Dubbed OMUFA, is up for its first five-year re-authorization after being established in 2020 in legislation to overhaul the FDA’s OTC monograph program Congress included in the Coronavirus Aid, Relief, and Economic Security (CARES) Act. The program, with current legislative authority expiring at the end of fiscal year 2025, includes annual facility registrations and fees for OTC monograph order requests, or proposals for monograph changes. (Also see "US FDA Asks For Comments On Reauthorizing OMUFA, A Program Still New To OTC Industry" - HBW Insight, 29 Aug, 2023.)
“If you look at all the various user fee programs as they go through iterations, the business markets worked out after a few iterations,” Califf said.
“And then you get to the question, what can we do together to create the best products? And the products that have the most new things that people need, in a way which also ensures the safety? That's a very creative enterprise, and I think that's one of the reasons why the American system is the envy of most of the rest of world, as I've learned again on some world travels and conversations I've had over the last two years. It's not perfect. American system can always be better, but I think it's, I think it's a real envy of the rest of the world.”
‘Buffer’ Between
Congress And Staff
The agency is on a direct line of contact with its owners, Congress, as referee in the game of compliance with regulations lawmakers establish played by businesses in industries subject to its oversight.
In addition to overseeing the work of FDA executives, Califf’s daily work frequently includes taking calls from Capitol Hill.
“You have the unknowns coming from our elected friends and in many ways, I think the job of the FDA commissioner is to provide a buffer between civil service, which has to make decisions every day without bias because of the extreme conditions and agreements that civil servants have about financial interests, and politicians who are elected and have often had no inhibitions about calling and demanding certain things or asking questions about things that really they can't know about,” Califf said.
“I'm often fielding unexpected calls from someone who has an interest in something , someone that has a political agenda, whatever. Often with good results. Sometimes I really have to play the buffer role. And it's a 24-by-7 job. I used to run an intensive care unit, so I actually get a lot more sleep than I used to. But you just can't really stop because things come up all around the clock.”
Not that other FDA work supported by direct funding alone without user fees shouldn’t get as much attention, Califf emphasized.
“I think we need to pay a lot more attention to those areas because they're really important to public health … There are other parts, with most products on the market, where it's a big world out there, and we've got to figure out how to do that part better.”
ACNU Latest
In ‘Most Elegant’ Science
As far as the FDA’s progress toward publishing an “additional conditions for nonprescription use” final rule, Califf said refereeing for that game hasn’t determined a final score. He framed the development of applications for OTC switches within the overall drug development playing field with the agency the referee.
“The drug development process, driven by the laws and practices developed mostly in the United States, is one of the most elegant aspects of science ... as far as I'm concerned, because every drug has risks, and the name of the game is that you've got to produce empirical evidence, and benefits outweigh the risks. That is hard stuff,” Califf said.
“People are coming to recognize everything has some risk. I'm not talking about totally free of risk, but now in the switch, you got to demonstrate empirically that what you're proposing actually does work. That is, a consumer can walk into the store and figure out what they're buying and take it in a way that where that risk/benefit calculation still comes out in favor of benefit, and that is daunting,” he added.
“Doing empirical work is not child's play. This is serious effort that's needed, but I think it's very important, and we want to be able to assure the public that when they buy a product like this that has been through the proper evaluation. I say it's a lot to be gained if you want to get into this, but you got to do the work.”
The FDA’s latest semi-annual regulatory agenda of non-binding target dates published in July sets an October goal for an ACNU switch final rule; more than 180 comments have been submitted to the regulatory docket for the proposal, FDA-2021-N-0862. (Also see "ACNU Switch And IND Exemption Final Rules Targeted For October In FDA Regulatory Goals" - HBW Insight, 25 Aug, 2024.)
The FDA says an ACNU approval will be needed when a firm convinces the agency that labeling on a package isn’t sufficient to ensure a consumer can appropriately self-select and use a drug as a nonprescription product; consumers could access additional information needed for self-selection online or through other digital connections.
The CHPA isn’t “the only ones who gave comments, as you might imagine,” Califf said. “We're really not allowed to talk about what we're doing with those comments until we get through the process, but it's a significant priority for us.”