Childhood Cancer STAR Act

The bipartisan Childhood Cancer Survivorship, Treatment, Access & Research (STAR) Act of 2015 is sweeping childhood cancer legislation that includes provisions the Children’s Cause has championed for years and helped strengthen for this new bill.

Specifically, the STAR Act will expand opportunities for childhood cancer research, improve efforts to identify and track childhood cancer incidences, enhance the quality of life for childhood cancer survivors, and ensure publicly accessible expanded access policies that provide hope for patients who have run out of options.

This legislation is the result of many years of hard work from our Congressional champions, our community at large, and this organization. Its proposals are grounded in solid data, smart analysis, and a robust roundtable process that achieved community consensus on top policy priorities.

Children's Cause has actively advocated for cosponsors through the Alliance for Childhood Cancer. As of May 2016, the Childhood Cancer STAR Act has the support of more than HALF of the House of Representatives!

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ACE Kids Act

Children’s Cause for Cancer Advocacy supports S. 298/H.R. 546, The Advancing Care for Exceptional Kids Act of 2015 (The ACE Kids Act). Children with cancer, like children with other medical complexities, often see multiple specialists and a variety of physicians.  There are over 30,000 children with cancer covered by the Medicaid system and the specialized care they require often takes them across state lines. Under the current Medicaid system, parents of children with cancer and other multiple, life threatening disabilities struggle to coordinate the complex, multi-state care of their kids.  Only federal legislation can fix the fragmented system for children with medical complexity.

The ACE Kids Act creates an organized system of health care delivery for this unique population of children to ensure ready access across state lines and to reduce the burden on their families. Nationally designated children’s hospitals networks created under the ACE Kids Act would employ national quality standards and coordinate care, both essential to improving quality and saving money. These networks would include the full range of acute, post-acute and primary care providers, with children’s hospitals as anchors, and a focus on outpatient care to ensure children get the care that they need in the most appropriate settings closest to home, while reducing hospitalizations and emergency room visits.  

We are honored to join the nation’s leading children’s hospitals, along the Children’s Hospital Association, to support this legislation, which is voluntary for states, families, children’s hospitals and other providers.  The ACE Kids Act can deliver savings to Medicaid through increased efficiencies, including reducing hospitalizations and emergency room visits, while providing the array of outpatient and community services and support needed by these children.  

Precision Medicine Initiative / Moonshot Initiative

Precision - or personalized - medicine refers to using a patient's own genomic information to predict, prevent, and treat diseases like cancer with improved accuracy and outcomes. By selecting therapies to target cancer's molecular errors, doctors hope to more safely and effectively deliver the right dose of the right medicine to the right tumor. Precision medicine aligns with our mission of achieving less toxic and more effective therapies for children with cancer. We're seeing early promise from analyzing the genomic profile of children with cancer and tailoring treatment to match the molecular make-up of their tumors.

The Precision Medicine Initiative (PMI) was announced by President Obama in January 2015. In July 2015, Children's Cause was honored to attend a round-table discussion on precision medicine, focused on the FDA's plans to reconfigure its evaluation of new therapies in response to advances in precision medicine. In September, NIH Director Francis Collins launched the initiative's implementation phase with a call to create a one-million-member strong research cohort within 3-4 years. [See: Working Group Report on the Cohort Program - PDF]

A related Cancer Moonshot Initiative was announced in January 2016, to be led by Vice President Joe Biden and a White House Task Force. The White House is requesting nearly $1 billion from Congress over the next two years to support the Moonshot, with pediatric cancer listed as one of eight "cutting edge research opportunities."

The Children's Cause will continue to engage with policymakers and other stakeholders to ensure that children with cancer are at the forefront of this new era of scientific discovery.

RELATED: The NCI's Pediatric MATCH clinical trial program will test molecularly targeted therapies on children with solid tumors who have exhausted other standard treatment options. Enrollment is expected to begin later in 2016. Researchers hope to enroll 300 pediatric patients annually to screen for genetic alterations and, if possible, match those abnormalities with a targeted treatment. Pediatric MATCH will differ from its larger adult counterpart (NCI-MATCH) in several significant ways, including the sequencing of blood samples (in addition to tumor tissue) and offering enhanced screening to family members when underlying germline mutations are found in a child.

BPCA/PREA reauthorization

The Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA) provides critically important information on the safe and effective use of medications in the pediatric population, advancing the health of children. The Children’s Cause supports permanent re-authorization of PREA and BPCA with amendments. 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.

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 can be 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.

Children’s Cause President Susan Weiner co-chairs the Alliance for Childhood Cancer task force on PREA and BPCA and is working with the Alliance on a package of BPCA and PREA recommendations for this year’s reauthorization of the Prescription Drug User Fee Act (PDUFA).

NIH/NCI Funding

To support a renewed commitment to investing in cancer research and genomic advances, NIH funding for Fiscal Year 2016 was increased to $32 billion, a much-needed boost after a dozen years of flat funding and 2013's sequestration cuts. The National Cancer Institute will receive $5.2 billion of that allocation, amounting to a 5.3 percent increase.

Children's Cause will continue to work for budget increases that are robust, sustainable, and predictable to ensure continued progress in medical research.