2017 Policy Agenda

The 2017 Children’s Cause Policy Agenda was released in January 2017. In December, we updated the agenda to reflect progress and accomplishments from the year.

These year-end updates are provided in all caps.

The Children’s Cause for Cancer Advocacy (CCCA) is the leading national advocacy organization working to achieve access to more effective and less harmful pediatric cancer therapies; to expand resources for research and specialized care; and to address the unique needs and challenges of childhood cancer survivors and their families.  CCCA leads efforts to ensure that the needs and perspectives of children with cancer are integrated into the highest deliberations on health care and cancer policy at the Federal level.

Each year, approximately 13,500 children under the age of 15 are diagnosed with cancer - that's more than one classroom of children each day.  While the survival rates for some childhood cancers have improved significantly, there are still several cancers for which mortality remains high. And, despite our progress, cancer remains the number one cause of death by disease in our children. 

The 2017 policy agenda for CCCA - released in January 2017 - addresses the most significant barriers to care along the continuum of the childhood cancer journey:  access to care, the availability of treatments, and the post-treatment survivorship landscape.



New Therapies

In the past 20 years, only two new drugs have been approved for any childhood cancer.  The small population of children with cancer provides little market incentive for the biopharmaceutical industry to develop new pediatric oncology drugs.  Children with cancer are currently treated with drugs that were developed several decades ago for adults and research documents that they are very damaging to their physical and intellectual development. Childhood cancer remains the leading cause of disease-related death in our children, and new and better therapies are sorely needed for children battling cancer.

YEAR-END UPDATE: There were two groundbreaking precision medicine treatment advances impacting kids with cancer this year:

-  In May, the FDA approved Keytruda, the first-ever cancer therapy based on a patient’s specific genetic traits, rather than cancer type. This approval includes children with the identified genetic trait whose cancer has progressed and who have no alternative treatment options. This was a huge leap forward for the field of precision medicine.

-  In August, the FDA approved Kymriah, a type of immunotherapy called CAR T-cell therapy for children and young adults with B-cell ALL that had relapsed or resisted treatment.  T-cell therapy uses genetically modified cells to attack cancer cells.


YEAR END UPDATE: After years of effort, we achieved passage of the Research to Accelerate Cures and Equity for Children (RACE) Act, important legislation affecting kids and families with cancer that was signed into law in August. Passage of the RACE Act was a landmark achievement long-sought by Children's Cause and made possible by community collaboration, intensive stakeholder negotiations with Congressional champions, and all of you -- grassroots advocates speaking up to make your voices heard.

The RACE Act was passed as part of a larger FDA user fees bill, the FDA Reauthorization Act (FDARA). FDARA is a five-year reauthorization of user fee programs, which fund the agency’s drug and medical device reviews.

This legislation calls for patients to be part of the discussion with FDA about what drugs should be developed for kids. Children’s Cause will continue to be at the table speaking up for our kids, in 2018 and beyond.

Congress has acknowledged obstacles to the appropriate use of drugs in children by enacting the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), two laws that provide requirements and incentives for testing certain drugs in children.  Together, BPCA and PREA create a “carrot and stick” approach to yield important new safety and dosing information for products used to treat children.

The BPCA, originally established in 1997, created a pediatric exclusivity incentive.  Under BPCA, sponsors receive six months of additional market exclusivity as an incentive to conduct studies of drug and biologicals for use in children. 

PREA was enacted in 2003, and was designed to work in conjunction with BPCA.  PREA requires that pharmaceutical manufacturers conduct pediatric studies of certain drug and biologicals before they are marketed, unless a waiver or deferral is granted by the FDA. Under PREA, pediatric studies are required only for drugs that are developed for diseases that occur in both adults and children. Common adult cancers (e.g. breast, colon, lung and prostate cancer) do not occur in children, so virtually all oncology drugs approved for adults are waived of pediatric study requirements.  In addition, PREA specifies that studies are not required for drugs used to treat rare or orphan diseases, therefore excluding application to all forms of childhood cancer.  

While BPCA and PREA have yielded important new safety and labeling information for other children’s diseases, the laws have had a very modest impact on childhood cancer.

Pediatric Cancer Public-Private Drug Development

YEAR-END UPDATE: Throughout 2017, Children’s Cause has been working with stakeholders to evaluate a variety of business structures and methodologies that would generate capital support and deliver a pipeline of potential treatments for pediatric cancer clinical trials.

On October 27, 2016, Children’s Cause for Cancer Advocacy (CCCA), the Pharmaceutical Research and Manufacturers of America (PhRMA), and the Biotechnology Innovation Organization (BIO) convened a diverse group of key stakeholders and experts in pediatric oncology drug development to discuss scientific and operational challenges of developing new drugs for children with cancer and to explore potential approaches to solving them. The workshop brought together pediatric oncologists and other clinicians from leading medical centers and children’s hospitals in the U.S., Europe, and Canada, academicians, patient and provider advocacy groups, nonprofit consortia and cooperative groups, senior officials at the Food and Drug Administration (FDA) and National Cancer Institute (NCI)/National Institutes of Health (NIH), and representatives of 15 biopharmaceutical research companies.

Workshop participants agreed that the next step should be the creation of a pre-competitive public-private partnership that will explore potential approaches to solving the identified challenges. Throughout 2017 Children’s Cause will be working with stakeholders to evaluate a variety of business structures and methodologies that would generate capital support and deliver a pipeline of potential treatments for pediatric cancer clinical trials.

NCI Funding & Initiatives

Funding for the National Institutes of Health (NIH) for FY 2016 was increased by some six (6) percent or $32 billion, a much-needed boost after a dozen years of flat funding and 2013’s sequestration cuts.  The National Cancer Institute received $5.2 billion of that allocation, amounting to a 5.3 percent increase.

For 2017, as a result of the 21st Century Cures Act of 2016, $872 million was provided – above the 2016 levels – to boost medical research and drug approval efforts, including $352 million for the Cancer “Moonshot” and Precision Medicine initiatives.  The promise of those funds in future years, however, will dependent on additional congressional action.

YEAR-END UPDATE:  In a huge show of Congressional support for cancer research, lawmakers added $2 billion to the NIH budget for the remainder of FY2017 in a spring spending bill.

While NCI currently spends only about four (4) percent of funds on pediatric cancer, advances in treatment are very dependent on NCI-funded clinical trials and increases in funding will expand opportunities for additional trials.  Furthermore, additional funding will support the NCI’s Pediatric MATCH clinical trial program, testing molecularly targeted therapies on children with solid tumors who have exhausted other treatment options.

Anticipating Congressional consideration of the fiscal year 2018 budget, Children’s Cause urges the administration and Congress to build on the momentum of 21st Century Cures and deliver on the promise of the Cancer “Moonshot” initiative by providing NIH and the NCI with increases of $2.2 billion (7%) and $680 million (13%), respectively.

YEAR-END UPDATE: The Administration has called for FY2018 cuts of $7.2 billion to the National Institutes of Health, but lawmakers on both sides of the aisle – plus public health advocates like the Children’s Cause - have pushed back strongly. Congress has passed temporary spending measures to keep the government open in the final months of 2017 but remains in budget negotiations for a full FY2018 bill, as of mid-December.  Complicating matters is the need to raise the non-defense discretionary budget caps imposed by the Budget Control Act in order to fund the NIH through the next fiscal year, an issue we’ve spoken up about to Congressional leaders.


By 2020, there will be at least 500,000 childhood cancer survivors in the United States, many of whom will experience serious long-term and late effects on their health and well-being. Two-thirds of these individuals are likely to experience at least one late effect of treatment, and almost one-fourth will face a late effect that is serious or life-threatening.  Childhood cancer survivors may face a range of late effects, including second cancers, heart and lung damage, osteoporosis, financial pressures, psychosocial issues, employment and fertility problems.

Unfortunately, the health care delivery system is not currently equipped to address these issues.  Among medical professionals, primary care providers are often unfamiliar with the problems faced by childhood cancer survivors, and the system lacks a cadre of experts in care delivery. Survivorship care availability and quality varies greatly by region and there is a lack of a standardized and extended benefit plans that account for key transition issues (pediatrics to adult care, oncology to primary care).   Insurance coverage, where it exists for childhood cancer survivors, is inconsistent.  And both survivors and health care professionals need more education about emerging evidence of risk to survivors.


YEAR-END UPDATE: The year kicked off with the re-introduction of the Childhood Cancer STAR Act -- legislation that now has support from over 75% of the United States House of Representatives! We continue to seek additional support from the Senate but we have high hopes that we will see this legislation advance through Congress early in 2018.

The bipartisan Childhood Cancer Survivorship, Treatment, Access & Research Act of 2017 includes provisions the Children’s Cause has championed for years, expanding opportunities for childhood cancer research, improving efforts to identify and track childhood cancer incidences, enhancing the quality of life for childhood cancer survivors, and ensuring publicly accessible expanded access policies that provide hope for patients who run out of options.  

The bill would address the challenges faced by childhood cancer survivors and this legislation would enhance research on the late effects of pediatric cancer, including a study on insurance coverage and payment of care for childhood cancer; improve collaboration among providers so that doctors are better able to care for this population as they age; and establish a new pilot program to explore innovative models of care for childhood cancer survivors.  The Children’s Cause has long championed legislation to address childhood cancer survivorship.


Families with children diagnosed with cancer face enormous challenges including significant financial burdens, employment disruptions, insurance restrictions and geographic barriers to accessing appropriate care. 

YEAR-END UPDATE: Among the work we did this year to investigate and highlight problem areas for families of children with cancer:

-  Financial Access Webinar: We held a webinar at the end of April on the topic of treatment-related financial hardships facing families. See blog for highlights.

-  Survey of Patient Needs: We conducted a survey in the spring, with the goal of better understanding the financial hardships and insurance challenges facing childhood cancer families and survivors. See blog.

-  Panel Briefing on Capitol Hill: In partnership with the American Cancer Society Cancer Action Network, we co-hosted and moderated a panel discussion in September on access and treatment issues impacting kids with cancer and survivors.

In 2018, we aim to turn the lessons we've learned into concrete policy solutions.

According to one study, almost one-third of families whose children were being treated for cancer faced food, housing or energy insecurity and a quarter lost more that 40 percent of household income. 

During their child’s treatment, caregivers frequently miss work or must quit or change jobs to accommodate the time requirements and caregivers often report substantial financial burden placed on them by their child’s cancer.[1]

While charity programs or public insurance may provide support for some, even those with employer-based insurance face significant burdens and are at risk of losing that support with a loss of or diminishment of employment.  A loss of health insurance may result in increased direct costs of cancer care as a result of greater out-of-pocket costs, further increasing the family’s financial burden and compromising continuity of care for the patient.

Financial and logistical challenges are increased for those families who live in more remote areas.  Childhood cancers are typically treated at specialized centers in urban areas, which can create substantial challenges for families in remote communities.  Studies have shown that balancing a rigorous treatment schedule with school, work, and family obligations can be financially taxing for families affected by pediatric cancer.

Rural patients with greater distances to treatment, in particular, are more likely to miss school and to fall behind their peers.  In fact, over half of pediatric patients elect to stop attending regular school while undergoing therapy.

Furthermore, the financial stresses do not end with treatment.  Children treated for cancer face unique and increased risks for chronic conditions and socioeconomic challenges.  Yet, almost a third of childhood cancer survivors have foregone needed healthcare because of costs. 

While the Affordable Care Act (ACA) provided notable guarantees for children, including coverage under a parent’s insurance plan up to age 26, the health insurance plans provided in state exchanges leave many families vulnerable to significant out-of-pocket costs.  Essential health benefits, or the baseline of care guaranteed under the plans, also do not include recommended standards of care for childhood cancer survivors.

YEAR-END UPDATE: In national debates on healthcare, we spoke up time and again this year to protect affordable, accessible, high-quality care for children with cancer and survivors. Thanks to strong advocacy like ours, children with pre-existing conditions continue to receive care without discrimination and there are no lifetime caps on a child's coverage.

Medicaid / CHIP

A significant source of support for the treatment of childhood cancer is covered by the Medicaid and/or the Children’s Health Insurance Program (CHIP), which are joint federal-state funded programs.   Funding for CHIP, which has helped cut the child uninsured rate in half, expires in 2017.

Increasingly, under new program flexibility, Medicaid and CHIP enrollees are receiving benefits through managed care arrangements.  While the new regulations set minimum standards, they leave a great deal of flexibility for states.  Since these requirements go into effect as early as July 2017, advocates will need to reach out to their state Medicaid and CHIP agencies to discuss opportunities to ensure that patients have timely and appropriate access to services.

YEAR-END UPDATE: Despite CHIP’s bipartisan support on Capitol Hill, Congress let CHIP expire at the end of September. Public health advocates, including Children’s Cause, rallied public support around CHIP’s renewal and continue to put pressure on Congress to act. The situation is urgent and dire – If Congress doesn’t act quickly to pass a new CHIP renewal, millions of vulnerable children face a loss of critical health insurance.

UPDATE, December 22: CHIP Funding Restored -- For Now. (CCCA Blog)


Children with cancer must often cross state lines to access the best treatment.   Since Medicaid is a state-based program, patients covered by it have been limited to providers within their state.  The Advancing Care for Exceptional Kids (ACE) Act allows for networks of children’s hospitals across state lines and provide for coordination among multiple providers.

YEAR END UPDATE: The ACE Kids Act of 2017 has 21 bipartisan Senate cosponsors.

[1] Geography and the Burden of Care in Pediatric Cancers; Fluchel, Kirchhoff, Bodson, Sweeney, et al, Pediatric Blood Cancer, 2014;61:1918-11924

For additional information on Children’s Cause for Cancer Advocacy and its policy agenda, contact George Dahlman at gdahlman@childrenscause.org – 202-304-1850 or Sue Emmer at emmerconsulting@verizon.net.