AACR Call to Action

Tremendous progress has been made against pediatric cancer over the past several decades, and many achievements are detailed in this inaugural report. Remarkable advances across the continuum of pediatric cancer science and patient care have translated to a measurable impact, with cancer death rates among youth (age 19 and younger) in the United States declining by 24 percent between 2001 and 2021. As a result, today more than 85 percent of children diagnosed with cancer are alive at least 5 years after diagnosis. These scientific achievements are attributable in large part to bipartisan support in Congress, innovative public and private initiatives, and the commitment of patient advocates.

Continued progress requires robust and sustained federal investments, as well as enacting critical legislation to address key challenges. Pediatric cancer patients and their families rely on federally supported health care services and infrastructure. Without federal support, future breakthroughs as well as ongoing care for pediatric cancer patients are at risk. The bottom line is that decisions made today by Congress and the federal government will shape the fight against pediatric cancer for decades to come.

Therefore, we call on all stakeholders to engage with members of Congress and leaders at federal agencies to prioritize pediatric cancer research and patient care. More specifically, AACR recommends the following actions:

• Congress needs to provide robust and sustained funding for the federal agencies and programs that are focused on supporting pediatric cancer research and patient care. To maintain American global leadership in medical research, Congress should provide at least $51.303 billion for the National Institutes of Health (NIH) in fiscal year (FY) 2026. Congress should also provide at least $7.934 billion for the National Cancer Institute (NCI) in FY 2026, while also prioritizing pediatric cancer research.
• Expand access to clinical trials and promising therapies for children and adolescents with cancer. Introducing targeted regulatory incentives, such as tax credits or extended market exclusivity, to encourage greater industry participation in pediatric cancer trials would very likely increase the number of available clinical trials and eventually accelerate drug approvals to benefit pediatric cancer patients. Beyond the amount of ongoing clinical pediatric cancer research, additional barriers to access exist that must be addressed. Most clinical trials are offered only at academic medical centers, which may be logistically or economically difficult for patients and their caregivers to reach. Moreover, patient and provider awareness of the clinical trial landscape also greatly varies. Increased and targeted outreach efforts to rural and socially disadvantaged areas, Medicare reimbursement models that support the costs involved with clinical trial participation, and increased use of decentralized trials will be required to increase the number and diversity of pediatric patients receiving state-of-the-art care in clinical trials.
• Modernize and evaluate current pediatric cancer research programs and policies to better support the discovery and development of treatments as well as to improve patient care. Lawmakers must update the RACE Act and ORPHAN Cures Act to improve those policies and provide additional incentives to companies to develop pediatric oncology drugs and ensure that pediatric studies beyond their initial evaluation are carried out. Renewing and expanding the Rare Pediatric Disease Priority Review Voucher program will accelerate pediatric oncology drug development and increase the number of available treatment options.
• Support efforts that leverage and harmonize all available data to aid pediatric cancer research. The rarity of pediatric cancers is further compounded by the rarity of individual cancer subtypes that fall under the umbrella of pediatric cancer. The NCI Childhood Cancer Data Initiative (CCDI) is essential to maximizing the quantity and quality of all data collected on pediatric cancer to enhance research capabilities and identify and address potential disparities. The Administration’s recent Executive Order from September 30, 2025, to “prioritize the harnessing of American artificial intelligence innovation to unlock cures for pediatric cancer” includes important objectives and proposals that must be fully supported. In addition, continued STAR Act support for the CCDI will be crucial to efforts to create an environment in which pediatric cancer researchers will have enough data available for studies to uncover and understand drivers of disease, disparities, and outcomes. These programs should be further leveraged to develop a national pediatric data infrastructure that allows for improved data collection and sharing, and informs pediatric cancer diagnostic testing.
• Foster global and public–private partnerships to accelerate pediatric cancer research and the development of innovative treatments for pediatric cancer patients. Pediatric cancer research efforts are often fragmented across different institutions and countries, limiting the ability to pool resources, data, and expertise. Clinical trials for especially rare pediatric cancers require international collaboration and support. Streamlining data-sharing through federated databases and harmonizing regulatory processes across the globe will accelerate research and facilitate the conduct of meaningful clinical trials by providing access to statistically powered patient populations. The United States must build on successful models and prioritize global and public–private partnerships to increase survival rates and improve quality of life for pediatric cancer patients.
• Strengthen survivorship and long-term care for pediatric cancer survivors by ensuring comprehensive, accessible, and reimbursable long-term care services. After enduring pediatric cancer treatment, survivors often face a lifetime of potential late-onset effects, including heart disease, second primary cancers, infertility, and cognitive impairments. These long-term health challenges significantly impact their quality of life, educational attainment, vocational opportunities, and financial well-being. Strengthening survivorship research and care services is an investment in the future of these patients and in society as a whole. Such strengthening includes supporting dedicated and comprehensive survivorship programs at treatment centers, streamlining transitions to adult care, and mitigating financial costs. Oncology professional societies (physicians, nurses, and social workers) should develop standards for transitions of survivors from oncology care to primary care and for appropriate follow-up programs for survivors of different cancer types.

Fulfilling the recommendations of this Call to Action will build on past achievements and further accelerate progress against pediatric cancer. Now is the time for a renewed commitment from stakeholders, including the biopharmaceutical industry, academic and medical institutions, patient-centric organizations, and the federal government, to scientific research with the potential to save and improve the lives of millions of children and adolescents who have or have had cancer. Congress holds tremendous power to continue to advance the progress that has already been made and sustain it for future generations. AACR urges everyone to come together at this critical moment in the fight against pediatric cancer to provide hope to children, adolescents, and their families.