House E&C Unveils Long-Awaited UFA Reauthorization Legislative Package
  • The bill would update PDUFA VII, GDUFA III, BsUFA III, and MDUFA V to continue authorization of these user fee programs.
  • The bill outlines a sizable increase in estimated fees for FY 2023 compared to the first year of the last UFA reauthorization.
  • The Senate is expected to release its draft bill for the UFAs within the coming weeks.
Today, the House Committee on Energy and Commerce unveiled a long-anticipated legislative package (section-by-section) to reauthorize the Food and Drug Administration’s (FDA) user fee agreements (UFA). The Food and Drug Amendments Act of 2022 would update the Prescription Drug User Fee Act (PDUFA VII), Generic Drug User Fee Amendments (GDUFA III), Biosimilar User Fee Act (BsUFA III), and the Medical Device User Fee Amendments (MDUFA V) to continue authorization of these user fee programs. The bill outlines a sizable increase in estimated fees for fiscal year (FY) 2023 compared to the first year of the last UFA reauthorization in 2018. This high-level summary, based on materials publicly released by the Energy and Commerce Committee earlier today, will be followed by a deeper analysis by Thorn Run Partners as this language is further considered by Congress. 

  • Background. The UFAs are reauthorized every five years and represent an agreement between lawmakers, manufacturers, patients, and industry stakeholders to strike a balance between efficient product approval processes and garnering fees to operationalize these goals. The FDA relies on user fees to supplement its appropriated funds. Authorizing language for the UFAs outlines total revenue for the first year of each program and details how the FDA will use the fees — generally during the premarket approval process. The letters linked above elaborate upon the FDA’s commitments to industry groups. 

In addition to the UFAs, the draft bill includes several provisions aimed at increasing diversity in clinical trials. These efforts are focused on requirements and incentives for applicants to focus their trials on diverse patient populations. The bill also contains provisions related to generic drugs in an effort to improve transparency and access to these medications. Research, development, and supply chain initiatives are also included in the bill and are intended to improve FDA inspections — especially in light of the COVID-19 pandemic — and prioritize innovative treatments and technologies. Lastly, the draft legislation focuses more generally on transparency and program integrity as these themes relate to regulatory improvements.  

  • What’s Next? The Senate is expected to release its draft bill for the UFAs within the coming weeks, at which point the two chambers will work to reconcile differences. The current five-year authorization ends on September 30, 2022, and acts as a deadline for the current UFA negotiations.  

MDUFA — Under the MDUFA V, the Energy and Commerce Committee legislation intends to: (1) expedite device development; (2) ensure safety and efficacy; and (3) require higher fees if FDA meets its review goal timelines. The bill notes that the total revenue for FY 2023 will be $312,606,000. If passed, the MDUFA reauthorization would take effect on the later of: (1) October 1, 2022, or (2) on the date of enactment with the exception of provisions related to fees for certain submissions received on or after October 1, 2022, regardless of the date of enactment. The provisions of this reauthorization would no longer be effective on or after October 1, 2027, and the reporting requirements would no longer be effective on or after January 31, 2028. Additionally, the sunset dates from the previous MDUFA reauthorization would be repealed on October 1, 2022. 

PDUFA — The PDUFA agreement would increase the annual base revenue for FY 2023 to $1,151,522,958, representing an increase of over $272 million from 2018, the first year of PDUFA VI. If passed, the PDUFA reauthorization would take effect on the later of: (1) October 1, 2022, or (2) on the date of enactment with the exception of provisions related to fees assessed for all human drug applications submitted on or after October 1, 2022, regardless of the date of enactment. The provisions of this reauthorization would no longer be effective on or after October 1, 2027, and the reporting requirements would no longer be effective on or after January 31, 2028. Additionally, the sunset dates from the previous PDUFA reauthorization would be repealed on October 1, 2022. 

GDUFA — Under the legislation, the estimated revenue from all the fees established under the GDUFA proposal would amount to $582.5 million for FY 2023. The provisions under this act would become effective on October 1, 2022, unless the bill is passed after that date. The provisions of this reauthorization would no longer be effective on or after October 1, 2027, and the reporting requirements would no longer be effective on or after January 31, 2028. Additionally, the sunset dates from the previous GDUFA reauthorization would be repealed on October 1, 2022. 

BsUFA — In FY 2023, the estimated revenue from the collection of user fees for biosimilar biological products under this proposed legislation is expected to be equal to the sum of the annual base revenue for the FY, or $43,376,922 for FY 2023, the amount equal to the inflation adjustment for the FY, the amount equal to the capacity planning adjustment, amount equal to the operating reserve adjustment, an additional $4,428,886 for FY 2023, and an additional $320,569 for FY 2024. Additionally, the provisions under this section would extend the due date of the initial biosimilar biological product development fee due date by two additional days. If passed, the BSUFA reauthorization would take effect on the later of: (1) October 1, 2022, or (2) on the date of enactment except for provisions related to fees assessed for all biosimilar biological product applications received on or after October 1, 2022, regardless of the date of enactment.  

Clinical Trial Diversity 
  • Diversity Action Plans — The bill would require sponsors of clinical trials and studies to submit premarket reports of diversity action plans and would require the FDA to issue or update draft guidance on the reporting of the action plans. The bill would authorize the FDA to evaluate whether regulations should be issued, or identify whether additional authorities are needed, to collect data after approval in the event sponsors do not meet the enrollment goals included in the diversity action plans. 

  • Meetings and Workshops to Enhance Diversity — The proposed legislation would authorize the FDA to convene one or more public workshops to solicit input from stakeholders on increasing the enrollment of historically underrepresented populations in clinical trials no later than September 30, 2023. Additionally, under this bill, the FDA would have to convene a public meeting to discuss recommendations provided during the COVID-19 public health emergency to mitigate the disruption of clinical trials. The legislation would also require the FDA to submit to Congress, and publish on the public website of FDA, an annual report that summarizes information related to such diversity action plans.  

  • Decentralized Clinical Trials — The bill would require the FDA to issue draft guidance that addresses decentralized clinical trials regarding the participation of a diverse clinical population when appropriate. 

Generic Drug Competition 
  • Transparency — This legislation, as drafted, would require the FDA to provide sponsors of generic drugs information on differences in ingredients between the comparable reference listed drug and their generic drug to facilitate generic drug development and review. The FDA would also be required to issue guidance to explain how generic drugs are determined to be qualitatively or quantitatively the same as the listed drug. 

  • Access to Affordable Medicines — Even if a generic drug's proposed labeling differs from that of the brand drug, this bill would allow that generic drug to be approved if the differences are limited to changes approved by the FDA that are made within 90 days of when the application is otherwise eligible for approval.

Research & Development  
  • Emerging Technology Program — The bill would authorize the FDA to make grants to carry out an Emerging Technology Program for industry representatives and academics to meet with FDA officials to support the adoption of innovative approaches to drug design. The proposal would also authorize $20 million each year for FY 2023 through FY 2027 to implement the program. 

  • Rare Diseases and Conditions — This proposal would require the FDA to study processes for evaluating drugs for rare diseases in the U.S. and European Union and convene at least one public meeting to solicit stakeholder feedback on ways in which to improve engagement with rare disease condition patients and stakeholders. HHS would also be required to submit a report to Congress on certain FDA activities surrounding rare diseases within a specified time frame.  

  • Advanced Manufacturing Technologies Designation Pilot Program — The bill stipulates that the FDA would be required to: (1) implement a pilot program to designate an advanced manufacturing technology, which would sunset in 2029; and (2) hold a public meeting, issue guidance, and transmit a report to Congress on the pilot program. Under the proposal, a method for manufacturing that receives such a designation would qualify for expedited application development and review.  

  • Cell and Gene Therapies — Under the bill, the FDA would be required to hold a public workshop regarding best practices for obtaining the scientific data necessary to facilitate the development of cell and gene therapies. 

  • Critical Path Public-Private Partnership Program — The Critical Path Public-Private Partnership would be reauthorized, through FY 2027, at $10 million annually. 

  • Orphan Drug Grants —The bill would reauthorize the Orphan Drug Grants through FY 2027 and expand the grants to include new uses for such grants to treat individuals with rare diseases. 

  • Inspection Related Provisions 
  • FDA Inspections — The bill would require a study of when and how the FDA’s inspection tools are used, would make changes to risk-based inspection criteria, would allow the FDA to use certain records to satisfy preapproval or risk-based surveillance inspection requirements, and would require foreign government inspections to meet preapproval inspection standards in the event that FDA has entered into an agreement with that foreign government.  

  • Improvements and Transparency — Under the legislation, the FDA would be required to share, with the public, information related to the inspections of drug and device facilities and specifies the inclusion of certain information within 120 days of the end of each FY.  

  • GAO Report — The legislation would require the Government Accountability Office (GAO) to issue a report on FDA inspections of foreign establishments which manufacture drugs. 

  • Foreign Facilities Pilot Program — Under this proposal, the FDA would be required to commence a pilot program to increase the number of unannounced surveillance inspections of foreign drug establishments and to make information obtained throughout this process publicly available, among other items.  

Transparency & Regulatory Improvements 
  • Biologics License Applications — The bill would require holders of BLAs to conduct a report to confirm that their products listed in the Purple Book are available for sale only one time. These approved holders would also be required to report to the Secretary when withdrawing a product from the market.  

  • Encouraging Blood Donation — This proposal would exempt FDA information collections to solicit patient perspectives during medical product development and to solicit information from blood donors and potential blood donors to inform recommendations from the Paperwork Reduction Act’s requirements.  

  • Regulation of Certain Products as Drugs — This bill would consider contrast agents, radioactive drugs, and over-the-counter (OTC) monograph drugs as drugs and not medical devices. 

  • Accelerated Approval Drug Studies — This proposal would require the FDA to specify conditions for required post-approval studies, by the time the drug is approved, for such approved drugs under accelerated approval. It would also authorize the FDA to require post-approval studies to be underway for such drugs and would also clarify existing authority to withdraw approvals where sponsors fail to conduct studies with due diligence applies to the approved conditions. The use of real-world evidence (RWE) in these studies would be required to be reported on by the FDA to Congress. 

  • Facilitating the Use of RWE — The bill would also require the FDA to issue guidance to address the use of RWE to support drug and device approvals and clearances, including the data collected for Emergency Use Authorizations (EUA) during the COVID-19 public health emergency (PHE).  

  • Medical Device Meetings — This proposal would require the Medical Device Advisory Committee to meet at least once a year upon enactment and through FY 2027. 

  • Ensuring Cybersecurity of Medical Devices — This bill would require cyber device manufacturers to develop processes to ensure their devices are secure, have plans to identify and address cybersecurity vulnerabilities, provide a software bill of materials in their labeling, and submit this information to the FDA in premarket submissions. Notably, the bill defines cyber devices as those that have software, connect to the internet, or otherwise could be vulnerable to threats and these devices must comply with these provisions or else the FDA would deny 510(k) clearance.  

  • Facilitating Exchange of Product Information — The proposed legislation would not consider drugs or devices as misbranded because of the provision of information regarding investigational drugs or devices, as well as the uses to payors, formulary committees, or other entities under certain conditions, specified under the law.  

  • Bans of Devices — This bill would authorize the FDA to ban a particular use of a medical device. It would also ban the use of shock devices intended for self-harming behavior.  

  • Orphan Drugs — Regarding orphan drugs, this bill provides clarity that exclusivity applies only to the specific indication or use approved by the FDA. 

  • Reauthorization of Reporting and Projects — The proposed legislation would reauthorize reporting on pending generic drug applications and device pilot projects, including those to generate safety surveillance data, through 2027.