A substantive modification. v3 changes the bill's short title to include "Mikaela Naylon" and revises the proposed allocation for the Medicare Improvement Fund, reducing the amount compared to v2. Other provisions, including those related to pediatric cancer drug investigations and orphan drug exclusivity, remain unchanged.
Mikaela Naylon Give Kids a Chance Act
- Sponsor
- Rep. McCaul, Michael T. [R-TX-10]
- Committees
- Energy and Commerce Committee (primary)
- Last action
- Dec 2, 2025
Bottom line
This bill introduces a range of reforms aimed at accelerating pediatric drug development, particularly for cancer, while also addressing broader healthcare policy areas from organ transplantation to international FDA operations and generic drug transparency.
What it actually does
This bill amends the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act to enhance pediatric cancer drug development, ensure completion of pediatric study requirements, extend rare pediatric disease priority review vouchers, and modify orphan drug exclusivity. It also includes provisions related to the Organ Procurement and Transplantation Network, establishes an Abraham Accords Office within the FDA, increases transparency in generic drug applications, and adjusts the Medicare Improvement Fund.
Proponents argue
Supporters argue that the bill will significantly improve the development of life-saving drugs for children with cancer by requiring more targeted pediatric investigations and ensuring accountability for study completion. They also contend that extending priority review vouchers will continue to incentivize treatments for rare pediatric diseases, and that reforms to organ procurement and generic drug transparency will enhance public health and efficiency.
Opponents contend
Critics might argue that some provisions, such as the changes to orphan drug exclusivity, could inadvertently reduce incentives for developing drugs for rare diseases by limiting market protection. Concerns could also be raised about the administrative burden of new FDA offices or reporting requirements, and the potential for the Medicare Improvement Fund adjustment to be used for unrelated purposes.
The bill is moderately complex due to its technical nature and multiple distinct policy areas, requiring careful reading to understand its full implications, especially given the lack of external analysis provided.
Sec. 2
Molecularly Targeted Pediatric Cancer Investigations
This section amends the Federal Food, Drug, and Cosmetic Act to require molecularly targeted pediatric cancer investigations for certain new drugs and biological products. It specifies that these investigations can involve the new product alone or in combination with existing drugs that are standard of care for pediatric cancer or approved for adult cancer and target a relevant molecular pathway. The investigations must yield clinically meaningful pediatric study data for dosing, safety, and preliminary efficacy.
Supporters argue
Supporters argue that this provision is crucial for accelerating the development of targeted therapies for children with cancer, ensuring that new drugs are studied in pediatric populations early in their development, rather than relying on extrapolation from adult data.
Critics contend
Some in the pharmaceutical industry might argue that these requirements add significant costs and complexity to drug development, potentially delaying market entry or disincentivizing research into certain molecular targets if the pediatric market is perceived as too small.
Tradeoffs
The provision balances the urgent need for pediatric cancer treatments with the financial and logistical challenges faced by drug developers in conducting additional studies.
Sec. 3
Ensuring Completion of Pediatric Study Requirements
This section amends the Federal Food, Drug, and Cosmetic Act to strengthen enforcement for failures to complete required pediatric studies. It clarifies that the FDA can take enforcement actions under section 303 (including civil penalties) if a person fails to meet pediatric study requirements, but only if they demonstrate a lack of due diligence. A new process is established where the Secretary must issue a noncompliance letter, provide a 45-day response period, and then determine if due diligence was lacking before taking action. It also limits enforcement actions for drugs no longer marketed.
Sec. 5
Extension of Rare Pediatric Disease Priority Review Vouchers
This section extends the authority for the FDA to issue priority review vouchers (PRVs) for drugs that treat or prevent rare pediatric diseases. The sunset date for this program is moved from December 20, 2024, to September 30, 2029. It also clarifies that the user fee for using a PRV is due upon submission of the human drug application for which the voucher is used, rather than at the time of the voucher award.
Sec. 6
Limitations on Exclusive Approval or Licensure of Orphan Drugs
This section amends the Federal Food, Drug, and Cosmetic Act to refine the 7-year market exclusivity granted to orphan drugs. Previously, exclusivity applied to the 'same disease or condition.' This amendment specifies that exclusivity now applies to the 'same approved use or indication within such rare disease or condition.' This means a new drug for the same rare disease but a different approved use or indication could potentially gain approval and market access sooner. These changes apply retroactively to any drug designated as an orphan drug, regardless of its designation or approval date.
Sec. 8
Organ Procurement and Transplantation Network Reforms
This section amends the Public Health Service Act to update and expand the requirements for the Organ Procurement and Transplantation Network (OPTN). It encourages the integration of electronic health records (EHR) systems among hospitals, organ procurement organizations, and transplant centers, including automated referrals and remote electronic access to donor records, while maintaining privacy. It also suggests establishing a public dashboard for transplant statistics. Furthermore, it authorizes the Secretary to collect registration fees from OPTN members for each transplant candidate placed on the waiting list, with these fees supporting OPTN operations. This fee collection authority sunsets after 3 years.
Sec. 9
Establishment of Abraham Accords Office within FDA
This section mandates the establishment of an Abraham Accords Office within the Food and Drug Administration (FDA), to be headed by a director. The Secretary of Health and Human Services, in consultation with Abraham Accords countries and U.S. diplomatic personnel, must select a location in an Abraham Accords country and establish the office within two years. The office's duties include providing technical assistance to regulatory partners in Abraham Accords countries on strengthening regulatory oversight and converging requirements for regulated products, and facilitating interactions between the FDA and interested parties in these countries.
Sec. 10
Increasing Transparency in Generic Drug Applications
This section amends the Federal Food, Drug, and Cosmetic Act to increase transparency regarding inactive ingredients in generic drug applications. Upon request, the FDA must inform generic drug applicants whether their proposed drug is qualitatively and quantitatively the same as the listed (brand-name) drug. If not, the FDA must disclose which ingredients cause the difference and the amount of deviation. This disclosure is authorized by law but does not otherwise affect trade secret status. Once the FDA determines a generic drug is qualitatively and quantitatively the same, it cannot change this determination unless the listed drug's formulation changed due to safety/effectiveness or an error is identified.
Sec. 11
Medicare Improvement Fund Adjustment
This section amends the Social Security Act to increase the amount available in the Medicare Improvement Fund (MIF). The fund's balance is increased from $1,403,000,000 to $2,622,000,000. The MIF is used by the Secretary of Health and Human Services to make improvements to the Medicare program.
The amendments made by Section 2 (molecularly targeted pediatric cancer investigations) apply with respect to applications submitted on or after the date that is 3 years after the date of enactment of this Act.
Sec. 2(c)
Why it matters:This is standard legislative practice to provide industry with a transition period to adapt to new regulatory requirements, rather than an attempt to obscure the provision.
Case for: Providing a 3-year lead time allows pharmaceutical companies sufficient time to adjust their research and development pipelines, clinical trial designs, and regulatory submission strategies to comply with the new requirements, minimizing disruption.
Case against: Critics might argue that a 3-year delay postpones the benefits of these crucial pediatric cancer drug investigations for children who need treatments sooner, potentially delaying access to life-saving therapies.
Estimated impact: Delays the full implementation and impact of the new pediatric cancer drug investigation requirements by three years post-enactment.
The amendments made by Section 6 (limitations on exclusive approval or licensure of orphan drugs) apply with respect to any drug designated under section 526 of the Federal Food, Drug, and Cosmetic Act, regardless of the date on which the drug was so designated, and regardless of the date on which the drug was approved or licensed.
Sec. 6(b)
Why it matters:This is a critical detail that determines the scope and immediate impact of the policy change, and its retroactive nature could be easily overlooked if not specifically sought out.
Case for: Proponents argue that applying these changes retroactively ensures that the intended policy goal of narrowing exclusivity to specific indications is immediately effective across the entire orphan drug landscape, preventing existing drugs from maintaining overly broad monopolies.
Case against: Opponents, particularly pharmaceutical companies with existing orphan drug approvals, would argue that retroactive application changes the rules mid-game, undermining previous investment decisions made under different exclusivity expectations and potentially creating an unstable regulatory environment.
Estimated impact: Significantly alters the market exclusivity landscape for all currently designated and approved orphan drugs, not just future ones.
The authority to collect registration fees for the Organ Procurement and Transplantation Network (OPTN) shall expire on the date that is 3 years after the date of enactment of the Mikaela Naylon Give Kids a Chance Act.
Sec. 8(d)(6)
Why it matters:Sunset clauses are common legislative tools to ensure periodic review of new authorities or programs, but can be overlooked in the details of a larger bill.
Case for: A sunset clause ensures that the fee collection mechanism is reviewed after a trial period, allowing Congress to assess its effectiveness and impact before deciding on its continuation, promoting accountability.
Case against: Critics might argue that a 3-year sunset period is too short to establish a stable funding mechanism for the OPTN, potentially creating uncertainty and hindering long-term planning for critical infrastructure improvements.
Estimated impact: Limits the fee collection authority to a 3-year period, requiring future legislative action for its continuation.
The disclosures authorized under clauses (i) and (ii) (regarding inactive ingredients in generic drugs) are disclosures authorized by law, including for purposes of section 1905 of title 18, United States Code. This subparagraph shall not otherwise be construed to authorize the disclosure of nonpublic qualitative or quantitative information about the ingredients in a listed drug, or to affect the status, if any, of such information as trade secret or confidential commercial information for purposes of section 301(j) of this Act, section 552 of title 5, United States Code, or section 1905 of title 18, United States Code.
Sec. 10(a)(H)(v)
Why it matters:This language is crucial for balancing transparency with intellectual property protection, and its precise wording is designed to navigate complex legal precedents regarding trade secrets and authorized disclosures.
Case for: Proponents argue that this language provides a critical legal safeguard, ensuring that while specific inactive ingredient information is disclosed to facilitate generic drug development, it does not broadly undermine trade secret protections for innovator drug companies.
Case against: Some critics might argue that even with this carve-out, any disclosure of proprietary formulation information, however limited, could set a precedent that erodes intellectual property rights or creates uncertainty for innovator companies.
Estimated impact: Clarifies the legal boundaries of information disclosure for generic drug applications, aiming to prevent unintended broader impacts on trade secret law.