Next User Fee Cycle Should Focus On Policy Improvements, Gottlieb Says
Executive Summary
US FDA commissioner wants to focus on new policies that will improve innovation and move beyond incremental process improvements.
Rather than looking for incremental process improvements every five years during the user fee cycle, the US FDA and industry should put more of their focus on new policies aimed at fostering innovation, Commissioner Scott Gottlieb says.
"That is a different mindset in how you think about how to use user fees." Gottlieb said during Informa's FDA/CMS Summit last month. "We haven't done that as much to date."
Historically, the commissioner said, industry has sought improvements to user fees that bring more predictability, such as an extra meeting in certain phases of development or guidance on specific issues.
"I think that what we did in the last round of user fees and what we should think about doing going forward is to think less about how to make discreet process improvements in the regulatory process itself – and I'm not saying those aren't important – but I think the process itself is very efficient, and has gotten a lot better overtime," Gottlieb said.
"I think we need to think about how to invent new policies that will improve the overall productivity of innovation and drug development."
Mid Cycle Policy Legislation
While Gottlieb was speaking specifically about the five-year user fee cycle, the potential remains for major policy development to take place in between the previous 2017 renewal and the upcoming 2022 renewal.
This happened several times in the most recent user fee cycles. For instance, the Biologics Price Competition and Innovation Act – which established the biosimilar review pathway at FDA – was passed in 2010 as part of the Affordable Care Act, in between the 2007 and 2012 user fee reauthorizations.
Two major policy developments took place in between the 2012 and 2017 reauthorizations. The Drug Supply Chain Security Act (DSCSA) was signed into law by then President Obama in 2013, which outlines steps to build an interoperable system to track and trace certain prescription drugs as they are distributed in the United States. (Also see "Track And Trace Deadline Could Slip Again After Worries Over Inferencing" - Pink Sheet, 24 Aug, 2017.)
And just before leaving office, Obama signed the 21st Century Cures Act into law. (Also see "The Evolution Of 21st Century Cures Legislation" - Pink Sheet, 29 Nov, 2016.)
Future mid-cycle legislation might focus on increasing FDA's seizure authority as part of the agency's oversight of international mail facilities (IMFs) to crackdown on illicit synthetic drugs. Gottlieb has suggested to Congress that this an area where existing statutes might be hindering their agency's ability to combat the opioid epidemic. (Also see " Addiction Recovery Medications' 'Stigma' Needs To Be Eliminated, Gottlieb Says" - Pink Sheet, 25 Oct, 2017.)
More Equitable Support Across The Agency
One ongoing debate surrounding FDA has been determining the optimal percentage for which the agency is funded by user fees.
The issue grabbed the spotlight amid Congress' development of the Food and Drug Administration Reauthorization Act of 2017 (FDARA), as the Trump administration has shared its desire that industry fund 100% of FDA premarket reviews. (Also see "White House Slams Generic Exclusivity Provisions In User Fee Bill" - Pink Sheet, 14 Jul, 2017.)
Gottlieb, however, contended that the best percentage of the agency funded by user fees is not the right discussion to be having. "I think that the issue isn't necessarily what percentage is optimal from my standpoint, but to actively try to make sure that the distribution of the support from user fees is equitable across the agency," he said.
"We do have that to some degree where it is harder to hire in certain parts of the agency, where certain parts of the agency are more heavily supported by budget authority that is more subject to the appropriations cycle that doesn't have the same protections and triggers that the parts of the agency funded by user fees have. And that's where I am trying to focus some attention to try to bring a little bit more balance to the support across the agency," Gottlieb added, pointing to the Center for Veterinary Medicine as an example of a center with potentially fewer opportunities to bring in top level talent.
A Harder Job
Gottlieb argued that the challenges of managing FDA are only increasing. "I always have this debate with Mark McClellan," who served as Commissioner towards the beginning of the George W. Bush administration. Gottlieb was a senior advisor to McClellan during that time, and has tried to convince his former boss "that it’s a lot harder now to be commissioner than when he did the job. And he denies it, so we have this debate back and forth. But my view of the agency now is that the scope of it has gotten a lot bigger than it was in the past. And just with the fact that you now have the tobacco center, and you have many more authorities on the food side and many more responsibilities."
Even in areas of existing authorities, "we’re seeing some natural evolution occur from the scope of the agency, owing to the expansion of technology." For example, the Center for Biologics Evaluation and Research has changed considerably since Gottlieb was last at the agency, when it focused largely on blood products and blood components, to now, when it is assessing gene therapy and other emerging technologies. "Now that’s a pretty robust program. And you see that across the whole agency, areas where there’s been natural expansions because of the expansion of technology," Gottlieb said.
"The other thing that’s changed a lot, and it’s hard to measure how much this really does change the flow at a regulatory agency, is just the 24/7 news cycle, and the fact that we’re forced to respond to things much more quickly," he said. "When you’re a regulatory agency that has to speak with precision and do things very carefully, to respond in that kind of a cycle is challenging. ... That has changed the pace of the front office quite a bit."