By Ayah El-khatib, MD, MS, Member, ASPHO Advocacy Committee

Over the past year, the U.S. FDA has undergone major shifts in its usual regulatory pathways. This shift now emphasizes accelerated drug approvals, streamlined clinical trial requirements, improved domestic manufacturing of products, and expanded use of gene therapies. These changes are highly relevant to the landscape of Pediatric Hematology/Oncology, enabling earlier access to novel therapies for this population. As accelerated regulatory pathways can provide an advantage for our population, they can also introduce new risks of widening disparities in access and outcomes.

The increased acceptance of trial designs that include pediatric patients and accelerated drug support for pediatric patients are changes intended to benefit the pediatric hematology/oncology patients we serve. Our population suffers from rarer diagnoses or smaller patient numbers compared to their adult counterparts, which has historically delayed the therapeutic advances for this vulnerable population. As a result of these regulatory changes, children can gain access to more targeted therapies, immunotherapies, and gene therapies, as well as to phase I-III clinical trials.

Timely legislative efforts also reinforce this momentum, including the Innovation in Pediatric Drugs Act of 2025, which expands the FDA’s authority to ensure timely access to the completion of pediatric studies. It also increases funding for the Best Pharmaceuticals for Children Act NIH Program, which expedites the approval of drugs for pediatric use. In addition, the Mikaela Naylon Give Kids a Chance Act of 2025, which enables targeted therapy trials in pediatric oncology and reauthorizes the Rare Pediatric Disease Priority Review Voucher program, which incentivizes drug development in this population. Both reforms represent an opportunity to accelerate innovation and access to life-saving therapies for our population. Both pieces of legislation recently became law as part of the Consolidated Appropriations Act, 2026.

Despite these advances and increased access, they do not inherently ensure equitable progress and access. Children with no insurance, public insurance, living in underserved communities, and getting care in under-resourced health centers are at higher risk of lower access to newer and novel therapies. Insurance complications, geographic limitations, and health center capacity to provide novel therapies compound these challenges. In addition, clinical trials can be at risk of underrepresentation of minority patients, families with limited English proficiency, and those facing structural barriers to participation, which can threaten the generalizability of study findings and perpetuate inequities and medical mistrust in the outcomes of these trials.

ASPHO is in a unique and advantageous position to ensure that regulatory and legislative improvements translate into equitable benefits for our population. Key priorities should include:

1. Protections for Equitable Access: Advocacy for improved and uniform insurance coverage, trial access, and novel drug therapies.
2. Inclusive Trial Design: Encouraging support for community-driven recruitment strategies, enrolling patients with health-related social needs, and transparent reporting of all demographic factors.
3. Investment for Infrastructure: Federal funding that expands access to early-phase trials and therapies at all pediatric care centers.
4. Regulatory Accountability: Monitoring relevant legislation, such as the Innovation in Pediatric Drugs Act of 2025 and the Mikaela Naylon Give Kids a Chance Act of 2025.
5. Patient and Family Engagement: Inclusion of diverse voices in regulatory and design trial processes.

The current FDA and legislative landscape offer new opportunities to accelerate access, treatment, and cures in rare and potentially devastating diagnoses for kids with oncologic or hematologic disorders. However, without deliberate attention to equity, these advances can potentially worsen existing disparities. ASPHO is committed to ensuring innovation that is matched with improvement in equitable outcomes for all children.

The Alliance for Childhood Cancer (Alliance) is a national advocacy group that supports pediatric cancer research and policies to prevent cancer, improve public education, and the diagnosis, treatment, supportive care and survivorship of children and adolescents with cancer in the United States. The members of the alliance includes 24 organizations that promote pediatric cancer research and policies across a continuum of diseases and patient/family needs. ASPHO, St. Baldrick’s Foundation, Children’s Oncology Group (COG), American Society of Clinical Oncology (ASCO), and the American Academy of Pediatrics (AAP) are just a few of the member organizations.

Despite concerns regarding pediatric cancer research and policies over the last year, there has been some success during this Congressional session. Two key pieces of legislation were passed earlier this year:

• The Mikaela Naylon Give Kids a Chance Act, which will encourage drug development and accelerate pediatric cancer treatments and increase access for treatment of rare diseases. Mikaela Naylon was a young lady with osteosarcoma who passed away last year and advocated for passage of this legislation through her illness. Her parents were present at Alliance Action Days.
• Accelerating Kids’ Access to Care Act, which will address administrative delays and create an alternative pathway for providers to enroll in multiple state Medicaid programs, allowing for patients to access care out of state.

On February 24 – 25, 2026, the Alliance for Childhood Cancer held its annual Action Days event in Washington, DC. During the two-day event, 270 individuals converged at the Westin Hotel in Washington to fight for research funding from the federal government, a topic that has become a flashpoint over the past year. Advocates included patients, survivors, and bereaved families, as well as physicians and other medical professionals and friends and family who braved the storms of the northeast to honor themselves and their family members. On day one, the advocates listened to a description of programs initiated at the National Institutes of Health (NIH), including the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act, Childhood Cancer Data Initiative (CCDI), and the Advanced Research Projects Agency for Health (ARPA-H) which have been transformative to the daily activities of predication oncology programs in North America.

Afterwards, advocates split into their state delegations and practiced sharing their stories in preparation for their meetings with Congressional offices the next day, where they would advocate for continued federal research dollars. While many of us had worked together before, a third of the advocates were attending Action Days for the first time, and their stories were both heartbreaking and inspiring. As happens during this event, one is overcome with respect for those who have undergone a loss of function, development, or life, and who still have the strength to relive their loss for the good of the children yet to treat.

Congressman Michael McCaul closed the activities of the day. He is a passionate advocate for pediatric oncology and the co-founder of the bipartisan Congressional Childhood Cancer Caucus. Representative McCaul, who is retiring this year, was honored by the Alliance for his many years of advocacy for our patients.

On the second day, the advocates converged on Capitol Hill. About 180 meetings occurred in the space of one day, each meeting relaying the successes and tragedies of the advocates and translating those passions into their asks. As a group, the Alliance representatives uniformly thanked members of Congress for the passage of the two laws mentioned above. The formal Action Days asks for this year included funding requests for federal fiscal year (FY) 2027:

• $7.3 billion for the National Cancer Institute (NCI)
• $51.3 billion for the National Institutes of Health (NIH)
• $35 million for FY 2027 for the STAR act, a $5 million increase over previous funding levels. The STAR Act was originally passed in 2018 and has been fully funded since its initial passage.
• $100 million for the Childhood Cancer Data Initiative (CCDI), stable with 2026 funding line.
• $1.5 billion for Advanced Research Projects Agency for Health (ARPA-H).

By the end of the day the advocates were foot sore but accomplished.

ASPHO members always work for our patients – to relieve their pain, improve their quality of life, to offer cure where possible and compassion, respect, equity and justice regardless. We lead that work for children with cancer and blood disorders throughout the world. But for two days, it was inspiring to see our patients and families take the lead in this work. Every day we listen to our families, but rarely is that our only purpose, as we order, coordinate, and plan on their behalf. Simply having a moment to listen to these families’ journeys was inspiring.

The Snowman [excerpt]

For the listener, who listens in the snow,
And, nothing himself, beholds
Nothing that is not there and the nothing that is.

-Wallace Stevens