User fee reauthorization marches on with first Senate hearing

Regulatory NewsRegulatory News | 05 April 2022 |  By 

Following multiple House hearings, the Senate Health, Education, Labor and Pensions committee held its first hearing on Tuesday to reauthorize the US Food and Drug Administration’s (FDA) user fee programs.
The full committee hearing was the first of two for the Senate committee to review FDA’s proposed user fee agreements and featured testimony from industry groups and the Pew Charitable Trusts on the need for timely reauthorization. (RELATED: Industry seeks clean user fee reauthorization as lawmakers eye riders, Regulatory Focus 18 March 2022; Shuren apologizes for MDUFA delay, says FDA will start closing the spigot on new EUAs, Regulatory Focus 30 March 2022)
“It’s no small task ensuring the safety of nearly 80% of our nation’s food supply, inspecting thousands of food, drug and device manufacturing sites each year, and of course, quickly and carefully reviewing the data on new and potentially life-saving medical products. And as we have seen throughout this pandemic, this work is incredibly important, which is why it’s also important we reauthorize the user fee programs, which ensure as FDA gets new drugs or devices to consider for approval … it also gets more resources to support that work,” Committee Chair Patty Murray (D-WA) said in her opening statement.
Murray said it would be “unthinkable” to delay passage of a reauthorization bill and force layoffs at the agency in the wake of the COVID-19 pandemic. She added that it would also be unthinkable to fail to incorporate the lessons learned from the pandemic in the legislation, “Like how we can get tests to families sooner, or how we can avoid political interferences like we saw during the Trump administration, including the reckless push for unproven treatments like hydroxychloroquine.” Murray also noted the need to improve transparency and communication around the timelines for COVID-19 booster shots and vaccines for younger children, which have proven to be points of confusion and frustration for many Americans.
Ranking Member Richard Burr (R-NC) focused his introductory remarks on the significant expansion of all four user fee programs – PDUFA, MDUFA, GDUFA and BsUFA – since their inception a decade or more ago.
“When members of Congress complain about the cost of prescription drugs or devices, we should evaluate all aspects of the pipeline, including the cost of development and regulatory review. To make sure costly and burdensome regulations are not part of the problem,” he said.
“The enormous growth in the oldest of the user fee programs underscores my longstanding concerns. In 1993, FDA collected just over $35 million in annual fees from the prescription drug user fee program. Today, FDA collects over $1 billion in PDUFA user fees annually. Even accounting for inflation, that’s an increase of more than 1,500% since the start of the program. Yet, FDA continues to request more and more resources from industry, even when they don’t meet agreed upon performance goals,” Burr added, noting that FDA missed most of its performance goals for meetings under PDUFA in FY2019 and FY2020.
Burr took the agency to task for seeking funding for hundreds of new full-time equivalent (FTE) staff when it has hundreds of vacancies across its product centers. “In fact, across all four programs, FDA is committing to more than 880 new hires, when the agency has more than 700 vacancies outstanding,” he said.
Burr also decried FDA’s inspectional delays during the COVID-19 pandemic, noting that those challenges have delayed dozens of generic drug applications. He also raised concerns about the agency’s delayed submission of the draft MDUFA V commitment letter. (RELATED: Shuren apologizes for MDUFA delay, says FDA will start closing the spigot on new EUAs, Regulatory Focus 30 March 2022)
Moving to medical devices, Burr noted the massive expansion in revenue proposed under MDUFA V and questioned provisions of the program he saw as ceding ground on some goals outlined under MDUFA IV. “This agreement almost doubles the user fees and includes longer review times for some categories of products. ‘Pay more, get less’ is not exactly a selling point,” Burr said.
He also questioned the need for the total product lifecycle advisory program (TAP), a central component of the MDUFA V draft agreement and stressed the need for prespecified deliverables. “As the author of the breakthrough device pathway, I still can’t figure out whether this new program will offer anything different from FDA’s current activities for breakthrough devices. The new pilot program is set to grow over the five-year cycle, without clear accountability, metrics, or deliverables to help measure the success of the program,” Burr said.
Liz Richardson, director of the Health Care Products Project at the Pew Charitable Trusts, called on the committee to ensure there is not an interruption in FDA’s ability to collect user fees. “No matter how the agency decides to adapt its regulatory approaches, it needs adequate resources to fund its core activities, and this includes user fees,” she said.
She also championed two pieces of proposed legislation as possible riders to the user fee package – the Pioneering Antimicrobial Subscriptions To End Up surging Resistance (PASTEUR) Act and the Verifying Accurate Leading-edge IVCT Development (VALID) Act.
“The [2012] GAIN Act represented an important first step in launching innovator companies devoted to antimicrobial research and development, but more work is needed to ensure that these companies can earn a fair return on investment and continue to innovate. To that end, the PASTEUR Act proposes a unique ‘only pay for success’ pull incentive that will strengthen US preparedness for future pandemics,” she said.
She also said the VALID Act “is of critical importance,” to ensuring effecting oversight of diagnostic tests, regardless of how they are developed. “Current gaps in oversight have allowed tests that are developed and used within the same laboratories, called lab-developed tests, to come to market without FDA approval, even if those tests are otherwise high-risk,” Richardson said.
Cartier Esham, chief scientific officer and executive vice president of emerging companies at the Biotechnology Innovation Organization (BIO) urged the swift reauthorization of the PDUV VII and BsUFA III programs. She praised the meeting enhancement provisions under PDUFA VII, as well as those that would improve the integration of patient perspectives in drug development and review.
Esham also highlighted increased support for FDA’s cell and gene therapy program, improvements in safety monitoring, and advancements in patient-centric evidence collection and analytical approaches, as well as measures to promote the use of advanced manufacturing processes.
David Gaugh, senior vice president, sciences and regulatory affairs, at the Association for Accessible Medicines, called on the committee to reauthorize the GDUFA III and BsUFA III programs “as negotiated and without changes.” He credited the current programs with bringing a record number of generic drugs to market in recent years and the approval of 34 biosimilars.
Gaugh said the GDUFA III agreement would spur the development of more complex generics and hold the FDA to conducting re-inspections of generic facilities within specified timeframes. Plus, he highlighted provisions to speed the review of supplemental biologics license applications (BLAs) for biosimilars and facilitate the development of more interchangeable biosimilars.
Getting to medical devices, Mark Leahy, president and chief executive officer of the Medical Device Manufacturers Association (MDMA) noted the “historic increase in user fee funding” which would enable the agency to hire between 273-387 new FTEs, depending on its performance.
“This represents a historical increase in both overall funds and people, and it is our expectation that this will be the last major investment needed for the MDUFA program, and that moving forward, any necessary increases will be much more modest and targeted,” he said.
Leahy also noted that the performance goals around de novo and premarket approval (PMA) products are expected to improve throughout the five-year course of the program. “
“One goal that was elusive under MDUFA IV was the 510(k) total time to decision goal in FY2022 of 108 days. COVID did impact FDA capacity, including the ability to meet certain MDUFA IV goals. Under MDUFA V, the 510(k) total time to decision goal will improve each year, hopefully achieving 108 days by FY2026,” he said. “Also, for the first time, the agreement incorporates add-on payments that will provide the agency up to $115 million in additional funding above the baseline in the final year of the agreement, if FDA meets modest but important performance goals in the first two years of the agreement.”


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