S-3302 : Still Just a Bill

The Mikaela Naylon Give Kids a Chance Act of 2025 proposes significant amendments to the Federal Food, Drug, and Cosmetic Act, aiming to enhance the development of drugs for pediatric cancers and rare pediatric diseases.

Key provisions of this bill include:

• Pediatric Cancer Investigations: The Act expands the types of required pediatric cancer investigations for new molecularly targeted cancer drugs. It allows for studies of the drug either alone or in combination with other active ingredients, specifically:
  • An approved generic or biosimilar that is part of the standard of care for a pediatric cancer.
  • An active ingredient of an adult cancer drug, if approved by the same applicant and targeting a molecular pathway relevant to pediatric cancer growth or progression.
  These investigations must generate clinically meaningful pediatric data concerning dosing, safety, and preliminary efficacy. The Food and Drug Administration (FDA) will determine the applicable requirements for each drug application.
• Implementation and Oversight: The bill mandates the FDA to issue draft guidance on these new requirements within one year of enactment and finalize it within another year. It also requires the Secretary of Health and Human Services to report to Congress within six years on the implementation of these amendments. Additionally, the Government Accountability Office (GAO) is directed to study the effectiveness of these new requirements in pediatric cancer drug development within eight years and report its findings to Congress within ten years.
• Rare Pediatric Disease Priority Review Vouchers: The authority for the FDA to issue priority review vouchers, which incentivize the development of drugs for rare pediatric diseases, is extended from December 20, 2024, to September 30, 2030. The bill also clarifies that the user fee for utilizing such a voucher is due upon the submission of the human drug application for which the priority review is requested.
• GAO Study on Voucher Effectiveness: The GAO is tasked with conducting a study within five years on the effectiveness of these rare pediatric disease priority review vouchers. This study will examine various aspects, including the indications of approved drugs, the extent to which unmet needs were addressed, the size of companies involved, voucher transfer values, and the overall impact on drug development and FDA operations.