The House version of the user fee reauthorization package, the Food and Drug Amendments of 2022 (FDA2022), has advanced through the House’s Energy and Commerce Committee and is now set for consideration by the full House of Representatives.
Intro: Congressional reauthorization of FDA’s user fee programs is in full swing.
- The House version of the user fee package was introduced in early May. This legislation, known as the Food and Drug Amendments of 2022 (FDA2022), reauthorizes the user fee programs (PDUFA, MDUFA, GDUFA, BsUFA) and also includes several policy riders related to research diversity, generic and biosimilar development, program transparency and the supply chain. The bill was marked up in the House Energy and Commerce (E&C) subcommittee on May 11th, at which time the bill was amended in several places – including in the provisions on diversity in research, REMS, orphan drug designations, and other changes. At the time, Members voted unanimously to advance the bill to the full E&C Committee. [ Read our analysis of the original bill text here.]
- Today, the bill was advanced unanimously out of E&C. The bill was once again marked up, with lawmakers incorporating several changes to the legislative text (see below). As with the subcommittee markup, lawmakers amended the text of the bill in the nature of a substitute (AINS), which allows them to replace the text of the bill in full, effectively scrapping the previous version and imposing a new text (while maintaining the bill’s status and number). This means that the text of the legislation that was introduced differs from the version that was discharged to the full E&C.
- [Read the text of the bill as introduced here, and the sub-health AINS here and the bill as it currently stands here (plus this amendment to the AINS, described below).]
- The Senate have also previewed their package. This legislative package, known as the FDA Safety and Landmark Advancements Act (FDASLA) of 2022, would also reauthorize the user fee programs and includes several policy riders. However, while the House version includes a high number of smaller, individual policies, the Senate package instead prioritized fewer riders that are more high-level reforms. These include overhauls of the regulatory systems for diagnostic products, dietary supplements and cosmetics. The Senate version was published by Health, Education, Labor and Pensions (HELP) Committee leadership as a discussion draft, on which they are accepting feedback through May 22.
- What’s next? House leadership has indicated that their goal is to get a final version of the package to the President’s desk before the August recess.
The AINS advanced out of E&C made minor modifications to FDA2022.
- The new version makes only targeted changes to the FDA2022 package. The significant majority of the bill remains the same. However, the AINS did make technical correction to the bill text (e.g., clarifying statutory citations) as well as some policy updates. See below for a recap of the changes.
- What was not included: As AgencyIQ previously noted, there was some interest from E&C lawmakers to include provisions in the package related to medical device remanufacturing. However, it appears that lawmakers could not reach a consensus between the health subcommittee and full E&C markups. For now, remanufacturing policies are not contained within either of the user fee packages.
- What did change: Section 501 on diversity action plans. The updated text, as amended by the AINS, now clarifies that the Diversity Plan requirement is in effect for any phase 3 study or other pivotal study “other than bioavailability or bioequivalence studies”. Further, while the last update to the section under the previous AINS clarified that products being made available under the expanded access program could be waived by FDA, the new text clarifies that all expanded access products are formally exempt from the diversity plan requirement without seeking a waiver from FDA. In effect, “no diversity action plan shall be required for a submission described in section 561” (i.e., expanded access) – notably, this applies to both drugs and devices.
- For diversity action plans for devices, the text of the legislation now clarifies when the requirement applies. First, all devices subject to an Investigational Device Exemption (IDE) submission “shall submit” a diversity action plan, while devices that are not required to submit an IDE could still be subject to a diversity action plan. In those cases, the diversity action plan “shall be submitted to the Secretary” in the 510(k) pre-market notification, De Novo request or Pre-Market Approval (PMA) submission.
- Section 601: Sameness for generic drug submissions. The AINS clarified the scope of the feedback that the FDA can provide on qualitative and quantitative sameness for generic product sponsors. Under the updated text, lawmakers clarify that the sponsor of a generic drug can request information about the drug “for which the Secretary has specified in regulation… or recommended in applicable guidance, certain qualitative or quantitative criteria with respect to an inactive ingredient.” In effect, the sameness conversations that regulators can have with generic product sponsors will apply to inactive ingredients in the drug.
- Clarity on the applicability of the Accelerated Approval provisions (section 804). FDA 2022 includes a reworked version of E&C Chairman Pallone’s (D-NJ) Accelerated Approval Integrity Act, which would enhance FDA’s authority to compel and oversee post-approval studies. The updated text of the legislation now clarifies the policy’s applicability for “any application pending” at the time of the bill’s (potential) passage, noting that approval would not necessarily need to be delayed. Effectively, sponsors seeking accelerated approval would be able to make “any applicable changes to the product labeling required to comply [with the new provisions] by supplement after the approval of such application.” As Rep. Buschon (R-IN) noted, the update was intended to ensure that sponsors “won’t have to worry about the FDA changing the rules… in the middle of the game.”
- Another small change to the Accelerated Approval provision. Under this provision, sponsors would need to provide updates on their post-approval studies at a regular cadence. The newest text of FDA2022 removes the phrase “agreed upon” in reference to enrollment targets, progress reports or reports on milestones “as required by the Secretary.” This could potentially give the FDA more flexibility to request updates from sponsors on post-approval studies.
Lawmakers also added a new section in Title VII and two new sections in Title VIII.
- Section 713, adopted as an amendment, focuses on pediatric cancer drug research. This provision is based on the standalone Give Kids a Chance Act, and was introduced by that bill’s original sponsor Rep. Butterfield (R-NC). This would build upon authorities passed under FDA Reauthorization Act (FDARA), known as the Research to Accelerate Cures and Equity (RACE) for Children Act, that required sponsors to assess efficacy of adult cancer treatments in children regardless of the indication and subjected designated orphan cancer drugs to pediatric study requirements under PREA. The Give Kids a Chance Act would further enhance pediatric drug research by authorizing the FDA to request a combination pediatric cancer trial (i.e., require a sponsor to study an investigational product in combination with another product, in certain circumstances). FDA would be tasked with issuing draft guidance on implementing this provision within 6 months – and then finalizing that guidance within a year.
- Section 806 is about De Novo requests for EUA-authorized tests with a home-use indication. The new section 806 of FDA 2022 would clarify that sponsors of devices authorized under EUAs that have been “deemed” to be available for home use under CLIA (i.e., meeting the description in section 353(d)(3) of the PHS Act) may request to keep that designation when submitting their De Novo request. This would allow “a single submission” for a De Novo request for an at-home test, which would include “sufficient information to enable the Secretary to determine whether such laboratory examination or procedure satisfies the criteria to be categorized” as a home test, as described in the PHS Act.
- Section 815 is about workforce improvements. This new section would expand the enhanced hiring authority originally granted under the 21st Century Cures Act in 2016 to not just “medical products” but to “products regulated by the Food and Drug Administration” more generally. As lawmakers at the markup explained, this provision is intended to help shore up FDA staff in key areas related to food safety, including the oversight of infant formula. Further, the provision would require FDA to “develop and begin implementation of” a strategic plan to improve their workforce, including hiring goals, performance measures and planned updates tot eh strategy, within one year. Further, the agency would be tasked with reporting to Congress on their progress.