March 2025
The ASPHO Advocacy Brief
Welcome to the Advocacy Brief, a quarterly offering from the American Society of Pediatric Hematology/Oncology. The goal of this newsletter is to inform members about legislative and regulatory issues impacting the profession of pediatric hematology/oncology and the patients we serve. Visit ASPHO’s Health Policy & Advocacy webpage for updates on sign-on letters, legislative efforts, and more information to support pediatric hematology/oncology advocacy.
A Brief Message
As a new administration moved into power on January 20, 2025, we have seen a flurry of executive activity that can have a great impact on the work of ASPHO members and your patients. As these actions continue to come at a rapid pace they cause confusion that can impact the most vulnerable pediatric hematology/oncology patients and their families. Through all of this instability, we want our members to know that ASPHO remains committed to our advocacy priorities. We continue to monitor and take action with our coalition partners to ensure the voices of PHO physicians are heard and have an impact.
At the end of each featured article in this month’s Advocacy Brief, you will find opportunities to take action with your members of Congress. We ask that if you have not already done so, you use your voice to make sure policy makers know the real-world impact of the actions they are taking.
As things move forward, ASPHO will continue to sift through the noise and keep you informed on issues of importance. We would also invite you to join us at the Business Meeting during conference in Louisville, as we will be providing you all with an update on advocacy efforts at that time.
*Send your health policy and advocacy news to ASPHO This email address is being protected from spambots. You need JavaScript enabled to view it. .
ASPHO Bill Tracker
Visit ASPHO’s Legislative Tracking Chart to see a list of bills that ASPHO is monitoring.
Special Feature Articles
Children’s Oncology Group Provides Value to Pediatric Cancer Research
By David Dickens, MD FAAP, Clinical Professor and Director, Pediatric Hematology, Oncology, and Bone Marrow Transplantation, University of Iowa Dance Marathon Endowed Chair in Clinical and Translational Research
Every other day, a child in Iowa is diagnosed with cancer. Despite their diverse demographic and socioeconomic backgrounds, they all share the same fundamental wish: to survive. Ideally, they would receive the best available treatments close to home, while experiencing no short- or long-term side effects. As aspirational as this goal may seem, it is what patients hope for and what their providers strive toward. However, the reality remains stark—we continue diagnosing incurable diseases while permanently harming many of those we can cure. The only way to bridge this gap is through research.
A clear example of research-driven progress is the development of blinatumomab for B-cell acute lymphoblastic leukemia (B-ALL). In 2019, the Children’s Oncology Group (COG) launched a phase 3, randomized controlled trial, incorporating blinatumomab into a standard chemotherapy regimen—one refined through decades of prior research. Over five years, this trial enrolled more than 4,000 patients and was closed early due to an interim analysis demonstrating that adding blinatumomab improved three-year event-free survival rates by nearly 10% (87.9% to 96%).
The University of Iowa enrolled 23 patients in that trial. Because of this study, one of them—who otherwise might not have survived—will now be cured. If this trial had been conducted solely in Iowa, it would have taken over 200 years to reach the same conclusion. This is just one life saved through a collaborative, nationwide effort—and many more will follow. It is a testament to the power of cooperative research groups like COG, which have been driving pediatric cancer advancements for decades.
Founded in 2000 through the merger of earlier pediatric cancer research groups, the Children’s Oncology Group is the largest organization in the world dedicated exclusively to childhood and adolescent cancer research. Supported by the National Cancer Institute (NCI), COG unites more than 200 hospitals and institutions across North America, Australia, New Zealand, and parts of Europe. Its mission is to design and oversee clinical trials that ensure new treatments are both effective and safe. Beyond research, COG provides crucial educational resources for families navigating childhood cancer, offering guidance on treatment options, survivorship, and late effects of therapy.
The impact of publicly funded research is undeniable. A recent study on NCI-sponsored phase 3 clinical trials in adult cancer found that most led to new standards of care, generating over 14 million additional life years at an average cost of just $320 per life-year gained. While a similar study on COG trials has yet to be conducted, given the younger average age of pediatric cancer patients and the high cure rates achieved through research, the return on investment is likely even greater.
Yet, for many childhood cancers, cure rates remain unacceptably low, and new treatments are urgently needed. Because pediatric cancers are rare, progress depends on collaboration, which in turn requires sustained investment. Without ongoing national commitment to rigorous, cooperative clinical trials, progress will stall, and children will continue to die—or survive with debilitating long-term effects from their treatment.
At this critical juncture, funding for pediatric cancer research—and pediatric care more broadly—is at risk, and critical funding for research continues to be in jeopardy. Pediatric oncologists have long been fierce advocates for their patients within their institutions and communities. Now, it is imperative to extend that advocacy to the broader field of pediatric oncology.
I have had the privilege of working alongside some of the most dedicated individuals through organizations such as the American Society of Pediatric Hematology/Oncology, the American Academy of Pediatrics, the American Society of Clinical Oncology, and the Alliance for Childhood Cancer. These groups, and many others, provide avenues for advocacy at both state and federal levels, from emails and phone calls to in-person meetings with legislators. The most powerful messages often emerge when providers and families unite to share their experiences.
For those new to advocacy, the first step is understanding your institution’s guidelines for engagement. While advocacy is time-consuming and does not generate revenue, it is profoundly impactful. Even on its most frustrating days, it is more rewarding than the worst days in the clinic.
I often wonder which of my patients was saved because of this NCI-sponsored COG blinatumomab trial. That thought fuels my drive to advocate for more breakthroughs like it. At the same time, I fear the regret of living in a world where progress in pediatric cancer care stagnates—and knowing I didn’t even try to change it.
Take action today to let your members of Congress know you support ongoing investment into the National Institutes of Health.
AACR Action Alert to Support Medical Research
ASH Action Alert to Support Research Funding
A Threat to Funding, a Threat to Children: A Pediatric Hematologist’s Perspective
By Taizo Nakano, MD, Children’s Hospital Colorado, ASPHO Advocacy Committee Member
As pediatric hematologists, we face an array of life-threatening blood disorders and accept the challenge to cure the incurable. We stand on the shoulders of a molecular renaissance that has helped unlock the secrets of rare disease and has transformed our clinical landscape from one of hopes and dreams to one of evidence-based cure. From congenital dyserythropoietic anemia to hemophagocytic lymphohistiocytosis, rare pediatric blood disorders can be difficult to understand, challenging to study and even harder to pronounce.
With few independent foundations and philanthropic funding programs dedicated to pediatric blood disorders, our field has depended, more than most, on partnership from the nation’s top supporter of innovative and life-saving science, the National Institute of Health (NIH). NIH grant funding is our critical artery that promotes creativity, feeds innovation, and allows our scientists and clinicians to lead the world’s race towards cure. With respect for the critical nature of this funding, we compete for it. We push boundaries for it, we don’t eat or sleep for it, we’ll bleed for it. And when policy threatens the stability of NIH funding and the lives of our pediatric patients, we will vigorously oppose it.
Of course, much of this has already happened and the ground is still shaking. Grant reviews have already been canceled; submissions no longer accepted. Reagents expire, critical cell-lines exhaust and, most importantly, the light within of a generation of young physician scientists is dimming. Still, so long as our cells carry oxygen, we continue to aim to cure the incurable because there are immunocompromised children stuck in hospitals across the country right now waiting for us; parents desperately depending on us. We hope that our effort is strong enough to anastomose political divide, advance our field, and save their lives. We will continue to push boundaries regardless and continue to support national and federal collaboration because, as pediatric hematologists, your children are depending on us.
Take action today to let your members of Congress know you support ongoing investment into the National Institutes of Health.
AACR Action Alert to Support Medical Research
ASH Action Alert to Support Research Funding
ASPHO Participates in the Alliance for Childhood Cancer Action Days 2025
By Zachary D. Prudowsky, MD, ASPHO Advocacy Committee Member and Liaison to the Alliance for Childhood Cancer
The Alliance for Childhood Cancer (Alliance) is a consortium of over 20 professional and philanthropic organizations all with the same collective goal – to improve the lives of children and families affected by childhood cancer through policy. Every year, the Alliance hosts Action Days, an event where volunteers from all over the country converge in Washington D.C. to meet with legislators to advocate for bills and appropriations to advance research and access to care. This year, I represented ASPHO along with Karen Effinger, MD MS, Associate Professor of Pediatrics, Emory University, Co-Director, Survivor Program, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta.
The early months of our new government have been controversial, with the executive branch leading spending cuts that threaten the infrastructure of pediatric cancer care in America. In response, an overwhelming amount of volunteers signed up to join Action Days, forcing online registration to close early and testing the limits of the fire code capacity in the Marriott Metro Center conference hall. Approximately 350 people from 40 states came to D.C., meeting with 222 separate members of Congress and the House of Representatives. Many of the advocates survived childhood cancer and were there to seek better treatments for the next generation, or bereaved parents who lost their child to cancer, approaching Capitol Hill wishing there were better available treatments. All got to share their stories and make an impact.
The agenda for Action Days focused around 3 priorities:
- Co-sponsor childhood cancer legislation: Multiple influential bills on the Alliance’s radar were unfortunately left off of the 2024 year-end budget resolution, and all except for the Gabriella Miller Kids First Research Act (which was passed outside of the budget bill) are still on the table for action this year. The Alliance gave an unofficial nickname to this series of policies, naming it the “Childhood Cancer Package.” Included in this package are the Accelerating Kids Access to Care Act (AKACA), the Give Kids a Chance Act, the Retaining Access and Restoring Exclusivity (RARE) Act, the Creating Hope Reauthorization Act, and the Innovation in Pediatric Drugs Act. The AKACA and Give Kids a Chance Act have been reintroduced in the House and Senate, while the Innovation in Pediatric Drugs Act has been reintroduced in the Senate. ASPHO has endorsed all of these bills.
- Prioritize appropriations to the National Institutes of Health: In previous years, the Alliance has been on Capitol Hill advocating for higher budgeting and appropriations for the National Institutes of Health (NIH). This year, it is dire to ensure federal funding is maintained, as cuts threaten to shrink overall NIH funding and infrastructure, the results of which could have devastating impacts towards pediatric cancer research and access to clinical trials. The Alliance asked Congress for continued and uninterrupted NIH support, and to maintain funding to the Childhood Cancer Survivorship, Treatment, Access, Research (STAR) Act and the Childhood Cancer Data Initiative (CCDI).
- Avoid cuts to Medicaid that would restrict access to childhood cancer care: The House of Representatives passed a budget resolution outlining their priorities for the fiscal year (FY) 2026 budget, just days prior to Action Days. In it were proposed cuts to Medicaid aimed to address “waste, fraud, and abuse.” Alliance advocates reminded members of Congress that 50% of the nation’s children are on Medicaid, and that there is nothing wasteful, fraudulent, or abusive about ensuring access to care for children with cancer.
The Alliance will continue to directly advocate on these important issues. Meanwhile, any and all members of ASPHO are encouraged to contact their Senators and Representatives directly to ensure these efforts continue to be a priority for them to address as soon as possible. Meanwhile, feel free to use these online tools to identify and contact your members of Congress:
Take action today to let your members of Congress know your thoughts on investing in NIH and protecting the Medicaid program.
AACR Action Alert to Support Medical Research
ASH Action Alert to Support Research Funding
ACS-CAN Action Alert on Medicaid Cuts
Legislative/Regulatory News
Continuing Resolution Passes to Avoid Government Shut Down
In order to avoid a government shut down, Congress passed, and President Trump signed, a Continuing Resolution (CR) on March 14. This CR will fund the government through September 30, 2025, which is the end of federal fiscal year (FY) 2025. Funding for the National Cancer Institute (NCI) was maintained at the FY 2024 level. However, there was a $859 million, or 58%, cut to the Congressionally Directed Medical Research Program (CDMRP), which includes the Peer Reviewed Cancer Research Program (PRCRP). ASPHO has consistently supported the PRCRP, urging Congress to maintain funding of the program and asking it to continue including pediatric and AYA cancers, pediatric brain tumors, neuroblastoma, sarcomas, germ cell cancers, blood cancers, lymphoma, and thyroid cancer as eligible funding topics under the program. ASPHO will continue to work with our partners at the Alliance for Childhood Cancer to work to restore funding to this important program.
Continuing Resolution vs. Budget Resolution
The process of passing the CR should not be confused with the recent passage of a budget resolution by both the House and Senate. A budget resolution is a non-binding blueprint that outlines fiscal goals but does not carry the force of law. However, you may have seen news stories with concerns about potential drastic cuts to the Medicaid program. The House budget resolution did contain language indicating they would like to make $880 billion in cuts from programs under the jurisdiction of the House Energy and Commerce Committee, this would include Medicaid. As the administration has claimed they do not wish to cut the Medicare or Social Security programs, this leaves Medicaid as the possible target of these massive cuts.
ASPHO has joined the Alliance for Childhood Cancer in opposing cuts or changes to the Medicaid program that would impede access and threaten needed coverage for children. As such massive potential cuts to Medicaid would have a disproportionate impact on pediatric patients, ASPHO will continue to monitor this important issue and provide members with additional opportunities to take action.
Sickle Cell Disease Comprehensive Care Act Reintroduced
Senator Corey Booker of New Jersey has reintroduced the Sickle Cell Comprehensive Care Act (S.721), which would expand the existing Medicaid Health Home program to allow states to submit a state plan amendment (SPA) to establish a Health Home with sickle cell disease (SCD) as the single qualifying condition for eligibility. ASPHO has previously supported this legislation which would greatly improve the care delivered to Medicaid beneficiaries living with SCD. You can read more about this legislation in our June 2024 Advocacy Brief.
Please take action today and ask your Senators to co-sponsor this important legislation.
For more information on our coalition partners and activities, visit:
American Academy of Pediatrics
Alliance for Childhood Cancer
American Society of Hematology
Sickle Cell Disease Coalition
Please visit our Health Policy & Advocacy website section for past issues of Advocacy Brief and stay up to date by following #ASPHOAdvocates on Facebook and X (formerly Twitter).