Overview
Title
An Act To amend the Federal Food, Drug, and Cosmetic Act with respect to molecularly targeted pediatric cancer investigations, and for other purposes.
ELI5 AI
H.R. 3433, or the "Give Kids a Chance Act of 2024," is a plan to make medicines for kids with cancer better by changing some rules. It wants to make sure more research is done on kids' cancer drugs, changes some rules about rare disease drugs, and adds a new office to help with this work.
Summary AI
H.R. 3433, known as the "Give Kids a Chance Act of 2024," aims to enhance pediatric cancer research and drug development by amending the Federal Food, Drug, and Cosmetic Act. The bill expands the responsibilities of the Food and Drug Administration (FDA) regarding pediatric cancer drugs, ensures completion of pediatric study requirements, extends the authority to issue priority review vouchers for rare pediatric diseases, and introduces limitations on orphan drug exclusivity. It also establishes an Abraham Accords Office within the FDA and introduces registration fees for the Organ Procurement and Transplantation Network.
Published
Keywords AI
Sources
Bill Statistics
Size
Language
Complexity
AnalysisAI
The proposed "Give Kids a Chance Act of 2024" is a multifaceted bill aimed at enhancing pediatric healthcare by amending the Federal Food, Drug, and Cosmetic Act. It introduces measures to advance research on pediatric drug uses, particularly emphasizing molecularly targeted cancer drugs for children. The Act also includes provisions for ensuring the completion of pediatric studies, extending incentives for rare pediatric disease treatments, modifying orphan drug rules, and establishing new organizational initiatives within the Food and Drug Administration (FDA). Additionally, the bill introduces a fee structure for organ transplantation networks.
General Summary
At its core, the "Give Kids a Chance Act of 2024" seeks to refine existing frameworks for drug research and development concerning pediatric applications. The bill emphasizes the importance of pediatric studies, delineates procedures for handling non-compliance with study requirements, and extends priority review vouchers to incentivize the development of treatments for rare pediatric diseases. It also takes steps to regulate the exclusivity of orphan drugs, aiming to ensure that such protections do not hinder patient access. Furthermore, the Act proposes the creation of the Abraham Accords Office within the FDA to foster international cooperation and proposes changes to the fee structures governing organ transplantation networks.
Significant Issues
Several notable issues are embedded within the bill, which warrant careful consideration:
Abraham Accords Office: The establishment of this office within the FDA appears atypical, potentially expanding the agency's role beyond its traditional scope. Concerns about the cost-effectiveness and relevance of this initiative underscore the questions regarding its alignment with the FDA's primary mission.
Priority Review Vouchers: The extension of voucher authority raises concerns about increased spending and potential market distortions favoring larger pharmaceutical companies, potentially impacting smaller entities trying to develop treatments for rare pediatric diseases.
Pediatric Study Requirements: The lack of clarity around "due diligence" in completing pediatric studies could lead to inconsistent enforcement, delaying important research and potentially affecting pediatric patients' access to necessary treatments.
Orphan Drug Exclusivity: Changes in defining "approved use or indication" might create ambiguities, impacting access to orphan drugs by allowing loopholes that could be exploited, particularly affecting patients in need of these critical medications.
Organ Transplantation Fees: Without clear definitions for "registration fees," there is a risk of variable interpretations that could unduly burden transplant candidates and their families, potentially leading to excessive costs without commensurate transparency.
Complexity and Scope of the Legislation: The bill's language is complex and may be difficult for those without specific legal expertise, such as general public and stakeholders, to grasp fully, leading to potential misunderstandings or misinterpretations of the new requirements.
Impact on the Public and Stakeholders
Broadly, the bill carries mixed implications worth considering:
Public and Patients: By prioritizing pediatric research and incentivizing the development of rare disease treatments, the bill has the potential to significantly improve healthcare outcomes for pediatric patients. However, the potential ambiguity in regulations and enforcement could delay benefits and access for some patients.
Healthcare Professionals and Researchers: Increased clarity and guidance can streamline research processes, but the complexity of new requirements might burden some professionals, necessitating additional training and resources to comply with the updated regulations.
Pharmaceutical Industry: The industry might encounter increased impetus to pursue pediatric studies and rare disease treatments, but smaller companies might struggle against larger firms due to competitive advantages in garnering priority review vouchers.
Regulatory Bodies: Agencies such as the FDA may face increased responsibilities and administrative burdens, particularly with the creation of the Abraham Accords Office, possibly necessitating reallocations of resources or manpower to effectively implement the bill's wide-ranging provisions.
Ultimately, while the "Give Kids a Chance Act of 2024" aims to foster advancements in pediatric healthcare, its execution will require careful balancing to ensure its provisions translate into tangible benefits without unintentionally disadvantaging key stakeholders or the public.
Issues
The establishment of the Abraham Accords Office within the Food and Drug Administration in Section 201 appears unusual and potentially outside the typical scope of the FDA's responsibilities, which could imply unnecessary or misdirected spending.
The extension of the authority to issue priority review vouchers for rare pediatric diseases in Section 104 could lead to increased spending without adequate oversight on its impact on drug development and might create market distortion favoring larger companies.
The lack of clarity on what constitutes 'lack of due diligence' in Section 102 can lead to varied interpretations and potential inconsistencies in enforcing pediatric study requirements, potentially affecting the timeliness and effectiveness of these studies.
Section 105's language changes in defining 'same approved use or indication' could create loopholes or ambiguities in the enforcement of orphan drug exclusivity, potentially affecting patient access to necessary drugs.
The absence of clear definitions and criteria for 'registration fees' in Section 301 could lead to varying interpretations and excessive charges, impacting stakeholders like transplant candidates and their families.
The bill’s language across several sections, including Sections 101 and 102, is quite complex and may be difficult for those without legal expertise to understand, potentially leading to misinterpretation of the new rules and requirements.
The requirement for investigations in Section 101 to include results from previously completed preclinical studies is not clearly defined in terms of scope, which could lead to discrepancies in interpretation and affect research actions.
Sections
Sections are presented as they are annotated in the original legislative text. Any missing headers, numbers, or non-consecutive order is due to the original text.
1. Short title; table of contents Read Opens in new tab
Summary AI
The "Give Kids a Chance Act of 2024" outlines various initiatives to enhance pediatric healthcare, including research into children's use of drugs, ensuring compliance with study requirements, and promoting treatments for rare pediatric diseases by extending priority review benefits. It also highlights cooperation with the Abraham Accords and addresses organ transplantation network registration fees.
101. Research into pediatric uses of drugs; additional authorities of Food and Drug Administration regarding molecularly targeted cancer drugs Read Opens in new tab
Summary AI
The section amends the Federal Food, Drug, and Cosmetic Act to establish new requirements for drug investigations specifically aimed at pediatric cancer, requiring the FDA to ensure these investigations provide meaningful clinical data and specifying conditions under which new drug applications must include these studies. It also mandates the issuance of guidance, applies changes to future applications, and calls for reports to Congress and a study by the GAO on the effectiveness of these requirements.
102. Ensuring completion of pediatric study requirements Read Opens in new tab
Summary AI
The text from the bill amends the Federal Food, Drug, and Cosmetic Act to establish clear guidelines for what happens if companies do not complete necessary pediatric studies for their drugs. It requires that companies receive a warning letter and a chance to respond before any enforcement action is taken, and limits penalties for non-marketed products.
103. FDA report on PREA enforcement Read Opens in new tab
Summary AI
The section amends part of the Food and Drug Administration Safety and Innovation Act to improve the evaluation of compliance with deadlines and add a requirement for listing penalties related to non-compliance with specific drug requirements. It mandates reporting on compliance and includes details about penalties or settlements for each drug.
104. Extension of authority to issue priority review vouchers to encourage treatments for rare pediatric diseases Read Opens in new tab
Summary AI
The bill proposes to extend the authority to issue priority review vouchers for treatments of rare pediatric diseases from September 30, 2024, to September 30, 2029. It also requires a study, conducted by the Government Accountability Office, on the effectiveness of these vouchers in motivating drug development, examining aspects such as the drugs they support, unmet needs addressed, company sizes, and the impact on the FDA's processes.
105. Limitations on exclusive approval or licensure of orphan drugs Read Opens in new tab
Summary AI
The section makes changes to rules about orphan drugs, focusing on how "approved use or indication" is defined when it comes to granting exclusive rights to these drugs. It clarifies that these changes apply to all designated orphan drugs, no matter when they were designated or approved.
106. Program for pediatric studies of drugs Read Opens in new tab
Summary AI
The revised section of the Public Health Service Act allows the Director of the NIH to use up to one percent of the funds allocated for pediatric research from each national research institute and center for the years 2025 to 2027 for research under this section.
201. Establishment of Abraham Accords Office within Food and Drug Administration Read Opens in new tab
Summary AI
The Abraham Accords Office is being established within the Food and Drug Administration to strengthen regulatory partnerships with countries that are part of the Abraham Accords. Its main duties include providing technical assistance, facilitating communications, and improving coordination for overseeing medical products. A report on its progress and the cooperation achieved with these partner countries will be submitted to Congress after three years.
1015. Abraham Accords Office Read Opens in new tab
Summary AI
The U.S. Secretary of Health and Human Services is required to set up an Abraham Accords Office within the Food and Drug Administration. This office will be located in one of the Abraham Accords countries and will work with local governments to enhance regulatory oversight related to medical products and provide support and information on U.S. regulatory pathways to relevant parties in these countries.
301. Registration fees Read Opens in new tab
Summary AI
The section adds a provision allowing the Secretary of Health and Human Services to collect registration fees from members of the Organ Procurement and Transplantation Network for each transplant candidate listed. These fees will support the network's operations, and their collection and distribution will be transparent, with updates posted online. Additionally, a review of the activities funded by these fees will be conducted by the Government Accountability Office two years after this provision's enactment.