Senate HELP Introduces FDA User Fee Legislation  
  • The package would update PDUFA, GDUFA, BsUFA , and MDUFA V to reauthorize these FDA user fee programs.
  • Funding levels for the user fee agreements mirror those unveiled in the House companion bill as well as the discussion draft released by the Senate earlier this month.
  • Notably, the bill includes new provisions related to the FDA’s accelerated approval process, therapeutic equivalence, and OTC hearing aids.  
Today, the Senate Committee on Health, Education, Labor and Pensions (HELP) Committee unveiled legislative text (section-by-section) to reauthorize the Food and Drug Administration (FDA) user fee agreements (UFA). The Food and Drug Administration Safety and Landmark Advancements 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 reauthorize these user fee programs. The bill outlines a sizable increase in estimated fees for fiscal year (FY) 2023 in comparison to the first year of the most recent UFA reauthorization in 2018.  

  • 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 their 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.  

Varying from the discussion draft (press release) offered by the Committee earlier this month, the legislation released today includes additional provisions related to: (1) the accelerated approval process; (2) therapeutic equivalence; (3) over-the-counter (OTC) hearing aids; and (4) FDA domestic and foreign inspections. Specifically, such additions would allow for the use of real-world evidence (RWE) in certain post-approval studies, provide guidelines for an expedited procedure for withdrawal of an accelerated approval product, and require the Department of Health and Human Services (HHS) to issue guidance on the use of novel endpoints and clinical trial designs in accelerated approval, among other items. Additionally, the proposed legislation would require the FDA to make timely therapeutic evaluations for follow-on drugs approved through the 505(b)(2) pathway that have similar formulations as other approved products. 

In addition to the UFAs, the bill includes several provisions aimed at amending the Federal Food, Drug, and Cosmetic (FD&C) Act to include new provisions for cosmetic and dietary supplement product oversight, as well as to create a new risk-based framework for lab-developed testing regulation. Additionally, the bill contains provisions to address: (1) competition in the prescription drug market; (2) safe disposal of opioids; (3) medical product development data; (4) FDA hiring authorities authorized by the 21st Century Cures Act (Cures); and (5) FDA transparency and accountability for the collection of user fees. The package also includes several provisions related to drug and biologic products, additional program reauthorizations to be extended through 2027, and modernizations to cosmetic regulations.  

  • What’s Next? The current five-year authorization for the UFAs ends on September 30, 2022, which acts as a deadline for current UFA negotiations. As both the House and Senate have formally released their draft legislation for these provisions, the two chambers will work to reconcile differences as committee hearings out of both chambers take place to consider the specifics of the text. Furthermore, additional changes to the text are expected to be made through a managers' amendment ahead of the Senate HELP Committee’s markup of the bill — rumored to be scheduled for June 8, 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, effective October 1, 2022. These guidelines match the legislation introduced in the House earlier this month. 

MDUFA — With respect to the MDUFA V provision, the HELP 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 also would update the definition of “process for the review of device applications” to include de novo classification requests. The bill updates the target base fee amounts for each year: the FY2023 base is $130,184,348, the FY2024 base is increased to $183,280,756, ending at $213,687,660 in FY2027. 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, effective 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, effective October 1, 2022. These guidelines match the legislation introduced in the House earlier this month. 

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. The bill would also establish a minimum operating reserve of carryover user fees for the process for the review of biosimilar biological product applications and would decrease the fees if FDA has carryover balances in excess of 33 weeks for FY 2023, 27 weeks for FY 2024, and 21 weeks for FY 2025 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.

Drugs and Biological Products 
  • Alternatives to Animal Testing — The bill would clarify that drug application sponsors can use alternative testing methods to animal testing for the effectiveness of human drugs. It would also permit sponsors of biosimilar applications to use alternative testing methods to animal studies to demonstrate biosimilarity to a reference product. 

  • Disposal of Opioids — The proposed legislation would allow FDA to require that opioids be dispensed to patients with safe, in-home disposal systems. The bill would also permit the dispensing of such disposal systems to patients.  

  • Clarifications to Exclusivity Provisions — The bill would clarify that FDA can approve interchangeable biosimilar biologic products while the first product has pending exclusivity. 

  • The Purple Book — The bill would require holders of approved biologic license applications to communicate to the FDA: (1) to confirm that their products listed in the Purple Book are still available for sale; or (2) when withdrawing a product from the market. This proposal would also require the FDA to update the Purple Book to reflect changes regarding any modification in the status of a biologic.  

  • Therapeutic Equivalence Evaluations — To allow for more lower-cost drugs to be automatically substituted at the pharmacy counter, the proposed legislation would require the FDA to make timely therapeutic evaluations for follow-on drugs approved through the 505(b)(2) pathway that have similar formulations as other approved products.  

  • Accelerated Approval — This bill would allow for the use of RWE in certain post-approval studies. Under the bill, post-approval studies may be required to be underway prior to approval by the FDA, and conditions for such an approval may be given by the Secretary of HHS. The bill would provide guidelines for an expedited procedure for withdrawal of an accelerated approval product, including: (1) providing the sponsor with due notice and an explanation; (2) an opportunity for a meeting, an opportunity for written appeal, an opportunity for public comment; (3) the publication of a summary of public comments received; and (4) the convening of, and consulting with, an advisory committee at the sponsor’s request. Accelerated approval drug sponsors would be required to submit a report of progress on post-approval studies every 180 days. Additionally, an interagency coordinating council would be established under the bill to ensure the “consistent and appropriate use” of the accelerated approval pathway. Further, the bill would authorize the Rare Disease Endpoint Advancement Pilot program and would require the HHS to issue guidance on the use of novel endpoints and clinical trial designs in accelerated approval, as well as the expedited withdrawal procedures. 

Other Reauthorizations 
  • Orphan Drug Grants — The legislation would reauthorize the FDA’s jurisdiction over orphan drug development grants through 2027. 

  • Critical Path Public-Private Partnership — The bill proposes to reauthorize the Critical Path Public-Private Partnership (P3) — designed to advance innovation and regulatory science through data sharing and partnerships with public and private entities of expertise — at its current funding level through 2027.  

  • Best Pharmaceuticals for Children Program — The bill proposes to reauthorize the Best Pharmaceuticals for Children Act (BPCA), which supports pediatric clinical trials, at current levels through 2027.  

  • Humanitarian Device Exemption Incentive — The package proposes to reauthorize the Humanitarian Device Exemption (HDE) for rare conditions through 2027.  

  • Pediatric Device Consortia Program — This legislation would reauthorize the pediatric device consortia (PDC) program, which provides expert advising to children’s device applicants, through 2027.  

  • Provision Pertaining to Drugs Containing Single Enantiomers — This section would reauthorize exclusivity provisions for drugs containing single enantiomers, until 2027. 

  • Device Inspections — The bill would reauthorize the FDA’s authority over the accreditation of third-party inspections of certain types of devices.  

Enhancing FDA Hiring Authorities 
  • FDA Hiring Authority — This bill would allow the FDA to streamline its hiring process for scientific, technical, and professional positions. 

  • Strategic Workforce Plan and Report — This bill would require the Secretary of HHS to create and implement a strategic workforce plan to improve the recruiting, hiring, and retention processes at the FDA and would require the HHS to submit annual reports of the plan’s status. 

Cosmetics 
  • Modernization of Cosmetics Regulation Act of 2022 — This proposal includes the Modernization of Cosmetics Regulation Act of 2022, which would amend Chapter VI of the FD&C Act to include new provisions for cosmetic products. These amendments would require persons to submit reports and records of adverse events within 15 days after receiving a report, direct the FDA to establish manufacturing practice regulations, and require a cosmetics manufacturer to register its facilities and submit product listings for each product. Additionally, the provision would amend the FD&C Act to ensure adequate safety substantiation for cosmetic products, increase requirements for labeling of cosmetic products, authorize the FDA to keep and protect detailed and extensive product records, and provide the FDA authority to order a mandatory recall of a product if determined to be unsafe. These amendments would also provide exemptions for small businesses, with average annual sales of less than $1 million for the previous three-year period. This provision would also amend the FD&C Act to allow certain products and facilities that are subject to the drug and device chapters of the FD&C Act to be exempt from the requirements under this section’s established Act. Finally, these amendments would preempt state laws with respect only to the policies mentioned previously.  

  • Enforcement — This proposal would establish new enforcement provisions that would become effective one year after the enactment of the Modernization of Cosmetics Regulation Act of 2022.  

  • Records Inspection — The bill would also make conforming edits to Section 704 of the FD&C Act to require FDA inspections on records and information, such as safety substantiation information.  

  • Talc Containing Cosmetics — Additionally, the legislation would require the FDA to establish testing methods for detecting asbestos in talc-containing cosmetics.  

  • Funding — This bill would appropriate an unidentified amount for each year from FY 2023 through 2029 toward the provisions under the Modernization of Cosmetics Regulation Act of 2022.  

  • PFAS in Cosmetics — This provision would require FDA to publish a summary of a report on their website within two years of this bill’s passage related to the use of perfluoroalkyl and polyfluoralkyl substances (PFAS) in cosmetics.  

Dietary Supplements 
  • Regulation of Dietary Supplements — This proposal would amend Chapter IV of the FD&C Act to include new provisions related to dietary supplements, specifically focused on the listings of dietary supplemental products.  

  • Dietary Supplement Listing Requirements — The provisions under this section would require dietary supplement manufacturers, packers, and distributors to list those products with the FDA as soon as they are introduced into interstate commerce. This bill would also mandate that certain information, primarily related to dietary supplement labeling requirements, be included within the listings. Multiple dietary supplements with identical formulations — that are different in color, additives, or flavor — would be allowed to be submitted under a single listing. Regarding dietary supplemental products, this proposed legislation would require manufacturers to: (1) notify the FDA within a year of discontinuance of a product; and (2) submit changes to listing information at the time of introduction. The proposed legislation would also require the FDA to provide listing numbers for dietary supplements and create an electronic database for certain listing information. Additionally, this section would authorize the FDA to: (1) obtain the names and addresses of dietary ingredient suppliers; (2) publish final guidance on new dietary ingredient notifications within 18 months after enactment; (3) and direct resources to inspect products and facilities that may present a public health risk. This section would also negate misbranded dietary supplements that have failed to comply with the listing requirements. It also would prohibit the introduction of dietary supplement products that do not meet the definition of such product under the FD&C Act — as well as products that were manufactured by a person who has been debarred — into the interstate commerce. 

In-Vitro Clinical Tests (IVCT) 
  • VALID Act — The package includes the Verifying Accurate Leading-edge IVCT Development (VALID) Act of 2022, which would create a new chapter of the FD&C Act to create a new risk-based framework for lab-developed testing regulation. The bill details requirements for premarket reviews of IVCTs, as well as abbreviated premarket and supplemental application and reviews. These specifications include pathways for testing instruments, as well as IVCT exemptions. Additionally, the bill provides for technology certifications for developers of moderate-risk IVCTs. The language includes mitigation measures for IVCTs with the same intended use, and it would establish a voluntary process by which developers can classify their IVCTs by risk level. Notably, the proposal provides specifications for existing IVCT tests that can be grandfathered into the program. The bill also accounts for breakthrough tests, advisory committees to solicit scientific recommendations, registration requirements, and design and quality testing, among other factors related to the accreditation and regulatory process. 
 
Other Provisions 
  • Facilities Management — The bill would clarify that FDA can use its congressionally appropriated funding for facilities costs can count toward the spending trigger for user fee agreements.  

  • Annual Report on Inspections — This legislation would align the timing of the FDA’s annual reporting requirement on inspections with reporting requirements for the PDUFA user fee program.

  • User Fee Program Transparency and Accountability — This provision would enhance reporting requirements for the user fee programs and requires the FDA to provide regular reporting updates to Congress. 

  • OTC Hearing Aids Final Rule — No later than 30 days after the enactment of this bill, the FDA would be required to issue a final rule to create a new category of over-the-counter hearing aids.  

  • Intra-Agency Coordination and Public Health Assessment — This bill include language from the Manufacturing API, Drugs, and Excipients (MADE) in America Act, which would advance coordination between FDA field investigators and drug shortage staff. Additionally, the FDA would be required to submit specified information on the agency’s domestic and foreign inspections, as well as recognition of foreign government inspections in an annual report. The FDA would also have to include additional information in that annual report regarding the timing of inspections, as well as regulatory and enforcement actions.