Industry seeks clean user fee reauthorization as lawmakers eye riders

Regulatory NewsRegulatory News | 18 March 2022 |  By 

Every five years, the reauthorization of the US Food and Drug Administration’s (FDA) user fee programs offers an opportunity for lawmakers to pin other policy initiatives, or riders, to the must-pass legislation. This user fee reauthorization cycle is no exception.
During a House Energy and Commerce health subcommittee hearing on Thursday, lawmakers presented 22 bills covering a wide array of issues related to the regulation or development of prescription drugs and biologics, generics and biosimilars. Other bills targeted specific disease areas, clinical trial diversity, advanced manufacturing and the creation of the Advanced Research Project Agency-Health (ARPA-H) agency.
The hearing featured testimony from industry, patient and physician groups, and while user fees were not the explicit topic for discussion, multiple lawmakers acknowledged that the bills could hitch a ride on the user fee package that must be passed by the end of the fiscal year. Last month, the subcommittee officially launched the reauthorization process in Congress for FDA’s human drugs and biosimilars user fee programs. (RELATED: User fee reauthorization process kicks off in Congress, Regulatory Focus 3 February 2022)
The Medical Device User Fee Amendments (MDUFA V) program was left out of the hearing as FDA and industry missed the statutory deadline to transmit the negotiated agreement to Congress. The MDUFA V agreement was delivered to Congress last week. (RELATED: MDUFA V: We have a deal, Regulatory Focus 8 March 2022)
Representatives of the Pharmaceutical Research and Manufacturers of America (PhRMA), the Biotechnology Innovation Organization (BIO) and the Association for Accessible Medicines (AAM) all urged Congress to reauthorize FDA’s user fee programs before they expire and insisted that the bills be passed free of riders or policy proposals that would sap FDA resources derived from the user fee deals.
“We would be concerned with any policy proposals or legislative riders that would undermine the negotiated user fee agreements and threaten timely passage,” said Lucy Vereshchagina, vice president of science and regulatory advocacy at PhRMA.
David Gaugh, senior vice president for sciences and regulatory affairs at AAM, said the group and its Biosimilar Council “strongly support timely Congressional reauthorization of the user fee agreements.” He added that the Generic Drug User Fee Amendments (GDUFA III) and the Biosimilar User Fee Amendments (BsUFA III) “are the culmination of months of negotiation, have been subject to public review and comment and represent a careful balance between all stakeholders. The commitment letters were carefully negotiated to balance the program enhancements and the resources required to be provided to the FDA.”
“AAM would have concern about adding policies into the reauthorization package that would require additional [full-time equivalents] FTEs to implement, if the package does not also include corresponding appropriations,” he said.
Subcommittee Chairwoman Anna Eshoo (D-CA) opened the hearing by welcoming the “22 mostly bipartisan bills to speed the discovery of more cures, improve patient representation in clinical trials and enhance the FDA’s ability to fulfill its vital mission of ensuring the safety, efficacy and quality of America’s drug supply.” She also championed her bill to establish ARPA-H, which received $1 billion in funding under the omnibus spending bill that was signed into law this week and highlighted several bills that would promote greater diversity in clinical trials. All of the industry witnesses present at the hearing supported the creation of ARPA-H.
The highly anticipated Cures 2.0 Act was also up for debate at the hearing, and other bills, such as the Verifying Accurate Leading-edge IVCT Development (VALID) Act, which were not discussed at Thursday’s hearing could wind up as riders to the user fee package. (RELATED: Long-awaited Cures 2.0 bill unveiled, Regulatory Focus 16 November 2021)
Accelerated approval reform
Both Committee Chairman Frank Pallone (D-NJ) and Ranking Member Cathy McMorris Rodgers (R-WA) proposed bills that would make changes to FDA’s accelerated approval program. While Pallone’s bill would rein in accelerated approvals, McMorris Rodgers’ stressed that the pathway should not be restricted to encourage medical innovation in areas of serious unmet medical need. (RELATED: Legislation would set expiration for FDA’s accelerated approvals, Regulatory Focus 9 March 2022)

“If we want to realize the promise of precision medicine, such as relying on genetics and proteins to treat diseases early, accelerated approval must be in FDA’s toolkit. I cannot support anything that undermines this important pathway,” she said.
McMorris Rodgers also emphasized the importance of swift passage of a user fee reauthorization package. "FDA’s authority to collect user fees expires 30 September, and without user fees, FDA’s ability to keep pace with innovation for patients will be severely limited. So, continuing this committee’s bipartisan tradition for this process is extremely important. This reauthorization also gives us the opportunity to pursue other bipartisan policies related to the FDA that can improve the review process and ensure new cures receive consistent, timely and thoughtful review,” she said.
Members’ stances on reforming the accelerated approval pathway largely fell along party lines, with Republicans favoring a more hands-off approach and Democrats eyeing reforms that would enable products that fail to confirm clinical benefit to be removed from the market more easily and expeditiously. On Pallone’s bill, Brett Guthrie (R-TX) said that, “Instead of adding more red tape, we should be focused on developing policy solutions that are intended to break down regulatory barriers and promote more collaboration between the regulatory community and the private sector.”
Both PhRMA and BIO also resisted any proposals that would restrict FDA’s accelerated approval pathway beyond what is included in the Prescription Drug User Fee Act (PDUFA VII) agreement.
“The PDUFA VII agreement includes commitments that would further strengthen this pathway by advancing regulatory understanding about what is necessary to support the utilization of a surrogate endpoint as the basis of approval. It includes provision to improve processes to allow for more effective dialogue and design of assessments of PMR needs and study designs and improve the continued evaluation of PMR postapproval to ensure requirements are being met and remain scientifically valid,” said Cartier Esham, executive vice president, emerging companies at BIO.
“[Accelerated approval] is a critical tool for patients and regulators and the industry continues to support the pathway in its current form,” Vereshchagina said.
Jeff Allen, president and CEO of the Friends of Cancer Research, also pushed back on changes that would make the accelerated approval pathway more stringent. “These data indicate that the accelerated approval is working as intended. It has enabled patients with serious diseases to have access to new medicines years earlier. But this pathway can, and should be, improved to maximize the benefits. Key to continued success is early planning when accelerated approval may be used and transparency to robust postapproval evidence generation,” he said.
However, Reshma Ramachandran, chair of the Doctors for America FDA Task Force, urged Congress to reform the pathway. “Nearly half of the 253 accelerated approval drugs approved by the FDA between 1992 and 2020 have not been confirmed to be clinically effective. Just last year, FDA maintained marketing authorization of four cancer drug indications despite their required postapproval studies failing to confirm clinical benefit. Moreover, relying on real-world evidence to confirm clinical benefit has not been shown to work. Of the 50 required confirmatory trials for drugs granted accelerated approval by the FDA between 2009 and 2018, none could be feasibly emulated using available real-world evidence,” she said. “Reforms within the Accelerated Approval Integrity Act offer a crucial opportunity to recenter the expedited review pathway around patients.”
Healthcare economic data and competition
Several bills introduced at the hearing are aimed at facilitating the exchange of healthcare economic information and increasing competition.
Guthrie sponsored a bill intended to facilitate the exchange of healthcare economic and scientific information before a product is approved which garnered support from industry representatives at the hearing, who praised FDA’s 2018 guidance on payor communications.
The bill seeks to address the so-called “valley of death” between when a product is approved and when it is covered by payors. “This should help patients gain access to potentially lifesaving treatments, such as Aduhelm, more quickly by giving the marketplace a chance to price in therapies working toward FDA approval,” Guthrie said. (RELATED: FDA finalizes product communications guidances, Regulatory Focus 12 June 2018)
Vereshchagina said that PhRMA members “find this FDA guidance very helpful and very impactful, and in fact, between 2017 and 2021, the number of publicly announced value-based contracts has more than doubled, so we’re seeing real positive impact of FDA giving very clear guidance on this issue.”
One bill meant to boost generic competition, the Increasing Transparency in Generic Drug Applications Act, would improve “FDA’s communication about the correct portion of ingredients during the application process” and reduce “needless delays” in bringing competition to the market, according to its sponsor Ann Kuster (D-NH).
While another bill that would amend title III of the Public Health Service Act to facilitate swifter biosimilar competition received support in principle from AAM, Gaugh said the group was concerned the bill as written could have “unintended consequences on really opening back up [the Biologics Price Competition and Innovation Act] BPCIA and could lead to some other exclusivity issues.” He said the group would like to work with Congress to address those issues.
Clinical trial diversity
Three bills, the Diversifying Investigations Via Equitable Research Studies for Everyone Trials Act, the Diverse and Equitable Participation in Clinical Trials Act and the Equity in Neuroscience and Alzheimer’s Clinical Trials Act of 2021 aim to increase diversity and equity in clinical research.
“The lack of diversity across clinical trials today and the systemic underrepresentation of certain groups weaken our ability to develop new therapies that could improve on existing treatments,” said Ruben Mesa, executive director of the Mays Cancer Center at UT Health San Antonio MD Anderson, who said he supported the bills targeting increased diversity in clinical trials.
While Vereshchagina said PhRMA supports “increasing diverse enrollment in clinical trials,” she said the group opposes “policies that would create additional mandates would reinforce rather than help overcome known barriers to participation for patients and have serious unintended consequences, including unfeasibly large and long studies, delayed access to medicines, or disincentives for industry to invest in high-risk therapeutic areas.”


© 2022 Regulatory Affairs Professionals Society.

Discover more of what matters to you