H.R. 1262 — Mikaela Naylon Give Kids a Chance Act

This section provides the short title, the “Mikaela Naylon Give Kids a Chance Act,” and sets out the table of contents for the Act’s 11 sections.

This section revises the pediatric study requirements under the Food, Drug, and Cosmetic Act for certain molecularly targeted cancer drugs and biological products. It amends section 505B(a)(3) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(3)) to define a “molecularly targeted pediatric cancer investigation” as an investigation of: (1) the drug or biological product in the application; or (2) the drug or biological product used in combination with either an active ingredient in an approved generic or biosimilar product that the Secretary determines is part of the standard of care for treating a pediatric cancer, or an active ingredient in an approved adult cancer drug held by the same sponsor and directed at a molecular target the Secretary determines is substantially relevant to pediatric cancer growth or progression. It requires such investigations to be designed to yield clinically meaningful pediatric study data using appropriate formulations for each age group, regarding dosing, safety, and preliminary efficacy to inform pediatric labeling. It limits when combination-product investigations may be required to products containing either a single new active ingredient or multiple active ingredients that have not previously been approved as a combination, if each ingredient has been previously approved to treat an adult cancer. It also authorizes the Secretary to require submission of completed preclinical study results at the same time as the initial pediatric study plan and clarifies that the provision does not address inactive ingredients in combination products. This section also requires the Secretary to determine whether the pediatric study requirements in subparagraph (A) or (B) of section 505B(a)(1) apply before the applicant must submit the initial pediatric study plan, and it adds a rule of construction stating that an application subject to one set of requirements is not subject to the other. It makes conforming changes to cross-references in section 505B(a)(3). This section directs the Secretary of Health and Human Services to issue draft guidance within 12 months after enactment and to finalize that guidance within 12 months after the close of the comment period. It applies the amendments to applications submitted under section 505(b) of the Federal Food, Drug, and Cosmetic Act and section 351(a) of the Public Health Service Act on or after the date that is 3 years after enactment. It also requires a report from the Secretary to the House Committee on Energy and Commerce and the Senate Committee on Health, Education, Labor, and Pensions within 6 years after enactment, and directs the Comptroller General to conduct a study within 8 years after enactment on the effectiveness of the pediatric assessment and investigation requirements in section 505B.

This section revises the enforcement framework for pediatric study requirements under section 505B(d) of the Federal Food, Drug, and Cosmetic Act, which governs pediatric studies for certain drugs and biological products. It requires the Food and Drug Administration (FDA) to issue a noncompliance letter before concluding that a sponsor failed to submit or otherwise meet a pediatric study requirement, and it gives the sponsor 45 days to respond in writing. After reviewing that response, FDA must determine whether the sponsor demonstrated a lack of due diligence in satisfying the requirement. The section also changes the consequences for noncompliance by providing that a drug or biological product may be subject to criminal enforcement under 21 U.S.C. § 333 only if the responsible person demonstrated a lack of due diligence, and it bars enforcement actions under section 303 for failures involving a drug or biological product that is no longer marketed. In addition, it makes a conforming amendment to 21 U.S.C. § 333(f)(4)(A) to include section 505B among the provisions covered by that subsection. Finally, it delays application of these enforcement changes until 180 days after enactment.

This section expands the annual Food and Drug Administration report on pediatric study requirements under the Pediatric Research Equity Act (PREA) by requiring the report to include: (1) an evaluation of compliance with deadlines in deferrals and deferral extensions; and (2) a listing of penalties, settlements, or payments under section 303 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 353) for failure to comply with section 505B, including the drug name, sponsor, and amount paid.

This section extends the authority to issue priority review vouchers for rare pediatric disease product applications through September 30, 2029 (from December 20, 2024, unless extended otherwise). It also revises the timing of the priority review user fee so that the fee is due upon submission of the human drug application for which the voucher is used, rather than under the prior timing rules, while other user fees remain due as required by the Secretary or applicable law. In addition, this section directs the Comptroller General of the United States to study the effectiveness of the rare pediatric disease priority review voucher program and to report the findings to the House Committee on Energy and Commerce and the Senate Committee on Health, Education, Labor, and Pensions not later than 5 years after enactment.

This section limits the orphan-drug exclusivity bar under section 527 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360cc) by replacing references to the “same disease or condition” with the “same approved use or indication” for a rare disease or condition. As background, orphan-drug exclusivity generally prevents the Food and Drug Administration from approving or licensing a competing drug for the same rare disease or condition for 7 years after approval or licensure. The section also revises the exception for a drug that is clinically superior to an already approved or licensed orphan drug by tying the exception to the same approved use or indication for which the 7-year period applies, and it clarifies that the clinical superiority determination may relate to the approved use or indication. It further revises the limitation on approval or licensure of a drug that is the same as an already approved or licensed orphan drug by substituting “same use or indication” for “same rare disease or condition.” Finally, it defines “approved use or indication” as the use or indication approved under section 505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355) or licensed under section 351 of the Public Health Service Act (42 U.S.C. 262) for a drug designated under section 526 for a rare disease or condition. The amendments apply to any drug designated under section 526, regardless of the date of designation or the date of approval or licensure.

This section provides $25 million for each of fiscal years 2026 through 2028 for the program for pediatric studies of drugs, replacing the prior open-ended reference to funding under section 409I(d)(1) of the Public Health Service Act. (Thus, the provision specifies annual appropriations for pediatric drug studies for 3 fiscal years.)

This section revises the Organ Procurement and Transplantation Network (OPTN) requirements under section 372 of the Public Health Service Act (42 U.S.C. 274) by making technical conforming edits to punctuation and formatting in subsection (b)(2), and by expanding the network’s duties to include: (1) encouraging integration of electronic health records systems through application programming interfaces, or successor technologies, among hospitals, organ procurement organizations, and transplant centers, including automated electronic hospital referrals and remote electronic access to potential donors’ hospital records, subject to Health Insurance Portability and Accountability Act of 1996 privacy regulations; and (2) considering whether to establish a dashboard showing the number and types of transplants performed, the number and types of organs that entered the OPTN system and were not transplanted, and other appropriate statistics, updated more frequently than annually. This section also establishes a new registration fee authority for the Secretary of Health and Human Services to collect fees from OPTN members for each transplant candidate placed on the national waiting list. The fees must be used only to support OPTN operations, are authorized to remain available until expended, and may be collected directly by the Secretary or through awards under subsection (b)(1)(A). The section requires that collected amounts be credited as discretionary offsetting collections and made available only to the extent provided in advance in appropriations Acts to distribute the fees among awardees. It further requires the Secretary to post quarterly transparency information on the OPTN website showing the amount collected from each member and the activities supported, directs the Comptroller General to review the fee program and report to Congress within 2 years after enactment, and sunsets the fee-collection authority 3 years after enactment of the Mikaela Naylon Give Kids a Chance Act.

This section establishes, within the Food and Drug Administration (FDA), an Abraham Accords Office headed by a director and requires the Secretary of Health and Human Services, acting through the FDA Commissioner, to select the office’s location in an Abraham Accords country and establish the office not later than 2 years after enactment. As background, the office is intended to support regulatory cooperation with countries that signed the Abraham Accords Declaration by providing technical assistance on regulatory oversight and convergence requirements for regulated products, including good manufacturing practices for medical products, and by facilitating interactions between the FDA and interested parties in those countries, including sharing information on U.S. regulatory pathways and receiving feedback on research, development, and manufacturing activities. The Secretary may assign FDA personnel as necessary and may carry out other functions needed to implement the section, but no action may be taken that is inconsistent with a federal national security recommendation. This section also requires the Secretary of Health and Human Services to submit a report to Congress not later than 3 years after enactment evaluating the office’s progress in helping manufacturers in Abraham Accords countries conform to FDA regulatory requirements, identifying the number of parties for which the office facilitated interactions or feedback, summarizing the technical assistance provided, and recommending ways to increase and improve coordination between the FDA and entities in Abraham Accords countries.

This section revises the Food and Drug Administration’s abbreviated new drug application process under 21 U.S.C. § 355(j)(3) by requiring the Secretary of Health and Human Services, upon request or on the Secretary’s own initiative during review, to tell an applicant whether a proposed generic drug is qualitatively and quantitatively the same as the listed drug. It also requires the Secretary, if the drug is not the same, to disclose the ingredient or ingredients causing the difference and, for any ingredient with a quantitative deviation, the amount of that deviation. The section prohibits the Secretary from changing or rescinding a same-drug determination after an abbreviated application is submitted unless the listed drug formulation has changed and the prior formulation was withdrawn for safety or effectiveness reasons, or the Secretary issues a written determination correcting an error; if the Secretary changes the determination for error, the Secretary must notify the requester and provide a copy of the written determination. The section further provides that these disclosures are authorized by law for purposes of 18 U.S.C. § 1905, while preserving the nonpublic status of ingredient-level qualitative or quantitative information as trade secret or confidential commercial information under 21 U.S.C. § 331(j), 5 U.S.C. § 552, and 18 U.S.C. § 1905. It directs the Secretary to issue draft or updated guidance within 1 year after enactment describing how the agency will determine whether a drug is qualitatively and quantitatively the same as the listed drug, including how pH adjusters are assessed, and requires at least a 60-day public comment period before final guidance is issued within 1 year after the comment period closes. The new requirements apply on the date of enactment, regardless of when the guidance is finalized.

This section provides an additional $1.219 billion for the Medicare Improvement Fund, increasing the amount available for expenditures from the Fund for services furnished during and after fiscal year 2026 to $2.622 billion (from $1.403 billion). The Medicare Improvement Fund is used to make improvements under original Medicare fee-for-service under parts A and B, including adjustments to payments for items and services furnished by providers and suppliers.