Executive Summary

Stephen Hahn also said the expedited approval process could be even more efficient.

The rolling application assessment that the US Food and Drug Administration is using for coronavirus therapeutics and vaccines may be among the pandemic-inspired regulatory changes that become standard practice.

FDA Commissioner Stephen Hahn said the agency’s approach to coronavirus therapeutics and vaccines, which included early engagement with sponsors and transparent communication of expectations, should translate post-pandemic for some products. He also said that the process could be made quicker.

“Even if it’s a partial application, particularly about a product that is urgently needed, we should be able to do this rolling review so that we can expedite the process ultimately,” he said on 13 October during the annual meeting of Reagan-Udall Foundation for the FDA.

“We have to figure out how to do that in an efficient way because sometimes that’s not as efficient as it could be, but I know that we can,” Hahn added. “We did it for COVID-19 products at the same time we were doing all of our regular approvals so the agency can do this. We have to think about the best process for that.”

Hahn did not appear to be discussing the well-established rolling review pathway, which is one of the FDA’s many expedited application approval systems. Rolling Review allows new drug application and biologics license application sponsors to submit sections of an application as they are completed. (Also see ““Breakthrough” Designation: FDA Clarifies How To Get It – And How It Can Be Lost” – Pink Sheet, 29 May, 2014.)

FDA officials have offered what has been called “real-time” assessment for many coronavirus therapeutics and vaccines. Agency staff are looking at data as it becomes available, rather than waiting for the entire application package to be completed and submitted, in order to allow for faster decision-making. (Also see “Moderna’s COVID-19 Vaccine To Get Real-Time Data Assessment By US FDA” – Pink Sheet, 27 Jul, 2020.)

Real-time review began in the FDA oncology divisions with a 2018 pilot program that allowed its use for supplements. The program allowed agency staff a jump-start on several assessment activities. (Also see “Real-Time Oncology Review Has Sponsors Rethinking What Data To Share With US FDA And When” – Pink Sheet, 26 Nov, 2018.)

Hahn did not reveal any more details about the idea, which came in response to a question about the pandemic-related regulatory changes that could be appropriate for permanent implementation.

A more specific definition of “urgently needed” product likely will be among the questions to be answered, since that may predict pathway eligibility. A larger discussion about the value of expedited pathways and the impact on the FDA’s workload also could ensue if more products could be eligible for the faster assessments. (Also see “Swift Pace Of Accelerated Approval Submissions Suggests Strong Constituency For US FDA Program” – Pink Sheet, 2 Jul, 2020.)

Clinical Trial Innovations Also Preferred For Permanent Implementation

Hahn also said that another pandemic innovation that could remain is the agency’s incorporation of digital and remote elements of clinical trials.

Principal Deputy Commissioner Amy Abernethy also said that issues like remote evaluation and use of real-world evidence were front of mind at the FDA before the coronavirus outbreak, but the pandemic allowed staff to see them in action.

“What we’ve seen is the need to shift our way of thinking to how do we accommodate for patients who can’t come into the clinic, who ultimately need to continue their participation in clinical trials, may even need to become new participants in clinical trials,” she said. “There needs to be mechanisms to allow that to happen.”

Shortly after the pandemic began, the FDA issued guidance to help sponsors avoid missing data problems and implement virtual visits to continue trials affected by the pandemic. (Also see “US FDA Offers More Advice On Mid-Trial Changes For Pandemic-Impacted Studies” – Pink Sheet, 30 Mar, 2020.)

Abernethy also said that real-world evidence will help the agency answer questions about the natural history of the pandemic and treatment patterns. She said that collaborations like the COVID-19 Evidence Accelerator could become a model for addressing questions about improving management of rare diseases.

The COVID-19 Evidence Accelerator, a partnership between the Friends of Cancer Research and Reagan-Udall Foundation, was created to inform rapid decision-making and clinical trial designs for therapeutics using real-world evidence. (Also see “Real-World Evidence On COVID-19: US FDA Approaching With ‘Sense Of Urgency’” – Pink Sheet, 21 Apr, 2020.)

Pandemic After Action Review Already Under Way

The FDA already has launched an after-action review of its response to the pandemic, which includes considering whether to retain pandemic-related regulatory changes.

Hahn has said stakeholders will be allowed to weigh in on the changes that worked well, along with those that did not. (Also see “BIO 2020 Notebook: FDA’s Hahn On COVID-19 Lessons To Be Learned; NIH’s Fauci On Health Care Disparities; Pfizer’s Young On Forgoing Gov’t Vaccine Funds” – Pink Sheet, 11 Jun, 2020.)

In addition, industry officials want the prescription drug user fee reauthorization to incorporate some lessons learned during the pandemic. (Also see “PDUFA VII Talks Begin With Similar Goals For Industry, US FDA” – Pink Sheet, 4 Oct, 2020.)

Some changes, like faster guidance development, seem likely to be incorporated into agency practice. (Also see “CBER’s ‘Courageous’ Coronavirus Guidance Strategy May Catch-On” – Pink Sheet, 25 Jun, 2020.)

https://pink.pharmaintelligence.informa.com/PS143098/After-Pandemic-US-…