- July 24, 2020
User fee goals, policy review
Despite an “incredible surge in volume” of applications and constraints on the Agency’s ability to conduct inspections, Hahn said that FDA, “has maintained the same pace of meeting its goals on applications for medical products for the last six months that is has maintained in recent years.” He added, “we (FDA) are on target to meet our user fee goals for drugs this year by taking action on at least 90% of brand, generic and biosimilar drug applications, even during the pandemic,” Hahn said.
Despite Hahn’s assurances, FDA has acknowledged the possibility that it will miss some of its user fee program goals in its recent guidance on formal meetings and applications. Hahn also reiterated that FDA is reviewing its response to the pandemic, “to identify and address potential organizational and programmatic changes that should be implemented without delay.”
“A major focus of this effort will be to identify what regulatory policies should be continued and accelerated,” Hahn said, referring to Executive Order 13924, which instructs federal agencies to extend regulatory flexibilities granted amid the pandemic to promote economic recovery.
Responding to a question from Ranking Member Greg Walden (R-OR) on where Agencies could use additional funding, Hahn said that FDA needs to improve its ability to gather and use real world evidence, especially in a rapidly evolving public health emergency. “We have learned that we need to collect real world evidence in real-time during an emergency…to inform decisions and how we could change them moving forward, so your support for real world evidence generation would be incredibly helpful,” Hahn said.
Development, approvals and testing
Hahn told members of the committee he could not give a timeline for the approval or Emergency Use Authorization (EUA) of a vaccine or additional therapeutics for COVID-19, but emphasized that “data and science will dictate” when that happens. He added that FDA will use every authority and regulatory flexibility at its disposal to approve products once data is available.
On whether a vaccine could be authorized under an EUA, Anthony Fauci, MD, Director of the National Institute for Allergy and Infectious Diseases, said he “would be very disappointed if we jumped to a conclusion before we knew that a vaccine was truly safe and truly effective because I wouldn’t want the perpetual ambiguity of not knowing whether or not it is truly safe and truly effective.”
Hahn added that FDA is working to accelerate the development of vaccines by providing technical assistance on clinical trial design and endpoints to sponsors, but said much of the acceleration will come from “taking financial risk around the development process … not cutting corners with respect to the assessment of safety and effectiveness.”
As for an EUA or approval for a vaccine, “I cannot prejudge when that will happen,” Hahn said.
Later in the hearing, Rep. Debbie Dingell (D-MI) asked Hahn to commit to receiving data from Phase 3 studies before approving a vaccine for COVID-19, citing concerns over safety if trials are rushed or underpowered. “We are working on, right now, guidance for sponsors and developers of vaccines to exactly address the question that you’re asking,” Hahn said, and assured Dingell that FDA would wait for the data it needs to make a determination about safety and efficacy. Similarly, Hahn said he cannot estimate when the agency might issue EUAs for additional therapeutics, after its EUAs for hydroxychloroquine/chloroquine and remdesivir. “I anticipate that we will receive data regarding several therapies in the future, [convalescent] plasma being one of them … potentially also with some anti-inflammatory agents, as well as monoclonal antibodies,” Hahn said.
Hahn noted that FDA now has safety data from more than 20,000 patients who received convalescent plasma under an expanded access treatment protocol and that the agency’s preliminary assessment of the therapy’s efficacy is “quite encouraging.”
In response to questioning from Rep. Anna Eshoo (D-CA) on reports of political interference in the Agency’s decision making around hydroxychloroquine, Hahn committed to report any perceived political pressure to the committee and stated that “I have not felt political pressure nor has the FDA to make any decision in any specific direction.”
Responding to questions about President Donald Trump’s recent statements about directing officials to slow down coronavirus testing in the US, Hahn, Fauci and Center for Disease Control and Prevention Director Robert Redfield, MD, all denied being instructed to impede testing efforts. “None of us have ever been told to slow down on testing, that just is a fact,” Fauci said. Fauci and the other administration officials agreed that more testing is necessary to combat the outbreak.
Redfield also noted that the CDC requested an EUA for a combined COVID-19 and influenza test last week, which he said will be useful when the flu season begins in the fall.
REFERENCE: RAPS Regulatory Focus; 23 JUN 2020; Michael Mezher