An Advocate's Guide to childhood cancer & the government

National Cancer Institute: The NCI is one of 27 institutes that comprise the National Institutes of Health (NIH), the primary Federal agency - within the Department of Health and Human Services- that conducts that supports medical research. The NCI was established by Congress in 1937 to support and coordinate extramural and intramural research projects, as well as collect and disseminate information on cancer. 

  • The Precision Medicine Initiative was first announced by President Obama in January 2015 at his State of the Union address. Precision medicine – the potential to deliver the right treatment at the right time, every time – aligns with our mission of achieving less toxic and more effective therapies for children with cancer. The first goal of the PMI is to create a one-million-member strong research cohort, including children, within 3-4 years. 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 Therapeutically Applicable Research to Generate Effective Treatments (TARGET Initiative) programs seeks to identify and validate therapeutic targets for childhood cancers using the latest genetic and genomic tools, which are the same ones used to develop targeted drugs for adult cancers. The TARGET Initiative is currently focused on identifying therapeutic targets in Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML), Neuroblastoma, Osteosarcoma and Wilms Tumors. A number of factors were considered when selecting these cancers, including prevalance among children, inadequacy of current treatment options, and availability of appropriate human tissue collections. 
  • The Pediatric Preclinical Testing Program (PPTP) is supported by the NCI through a contract with the Research Institute at Nationwide Children's Hospital in Columbus, Ohio. The PPTP provides a system for prioritizing drugs with potential to treat pediatric cancers. It involves multiple pediatric research sites that have expertise in specific childhood cancers. PPTP scientists develop preclinical models that express genes similar to those expressed by human cancers. These models are used to determine which drugs that have been developed for adult cancers may be effective in treat

Advocacy Opportunities: 

  • Director's Consumer Liaison Group (DCLG): Chartered in 1997, this federal advisory committee was designed to include advocates in NCI deliberations. The Office of Advocacy Relations (OAR), part of the Director's office, oversees links with cancer advocates and organizations, and including the DCLG. The DCLG is a committee of select consumer cancer advocates and presents an opportunity for discussion of cancer community concerns and NCI program and policy objectives. It also is a chance for advocates to help identify others to serve on NCI program and policy committees. The DCLG offers an opportunity for childhood cancer survivors and family members to express their concerns and learn from NCI’s administrative and scientific leadership.
  • NCI Advisory Boards represent additional opportunities for patient advocates to become involved in NCI strategy and planning. Advisory slots are filled by Presidential appointment and by the NCI Director. The President’s Cancer Panel includes an advocate/survivor as does the National Cancer Advisory Board, the Board of Scientific Advisors, and Board of Scientific Counselors. Though these opportunities are few, advisory boards, including the DCLG, are a chance for cancer patient advocates, survivors and family members to work with NCI at the highest levels to bring community concerns and solutions to NCI’s work.

Food and Drug Administration: The FDA is responsible for protecting the public health by assuring the safety, efficacy, and security of human and veterinary drugs, biological products, medical devices, among other products. 

  • The Center for Drug Evaluation and Research (CDER) evaluates new drugs before they can be sold. The center doesn't actually test drugs itself, although it does conduct limited research in the areas of drug quality, safety and effectiveness standards.
  • FDA has tools to aid in the testing of drugs for use in children and labeling for pediatric use. The Best Pharmaceuticals for Children Act (BPCA) provides incentives for companies to test drugs in pediatric populations voluntarily in the form of 6 months of additional marketing exlusivity and patent protection. Asa a complement to the incentives offered by BPCA, the Pediatric Research Equity Act (PREA) requires pharmaceutical companies to test new drugs in a pediatric populations when the drug is likely to be used in children.

Advocacy Opportunities:

FDA offers two principal ways in which cancer advocates can represent patient and family perspectives in the agency: on advisory committees and in the drug development process itself. The Office of Health and Constituent Affairs and its Cancer Liaison Program oversee these efforts.

  • The most salient advisory opportunity for childhood cancer advocacy is the Pediatric Subcommittee of the Oncologic Drugs Advisory Committee (Ped ODAC), within the Cancer Patient Representative Program. ODAC responsibilities typically include data review of agent for an adult anticancer indication. The Ped ODAC, however, is charged additionally with advising FDA about “new and emerging therapeutic alternatives available to treat pediatric cancer,” and recommending to FDA strategies for ensuring that children with cancer have timely and more consistent access to the most promising new cancer therapies. The Best Pharmaceuticals for Children Act codifies this subcommittee and further requires that its membership include at least two representatives of the “pediatric cancer patient and patient-family community.”
  • Advocates’ also participate in the drug development process itself. As part of the Cancer Drug Development Patient Consultant Program, “patient consultants” work with FDA staff during the pre-approval stage of new oncology agents. OSHI selects advocates, who are then trained by FDA staff on the drug review process, conflicts of interest, and confidentiality obligations of the advocates. These patient consultants bring cancer patient perspectives to FDA’s early discussions with companies of the design, analysis and implementation of clinical trials to evaluate safety and efficacy of a therapeutic agent. Patient consultants can be especially valuable in interpreting the real world meaning of surrogate endpoints to patients and families as well as offering insights about patients’ and families’ experiences in a trial. While most of the oncology agents under review are for adult indications, this is a unique opportunity for childhood cancer advocates to ensure that pediatric agents, when they do come to FDA for approval, are evaluated with appropriate patient/family input.

Centers for Disease Control: An agency within the Department of Health and Human Services, the CDC's mission is "to collaborate to create the expertise, information, and tools that people and communities need to protect their health - through health promotion, prevention of disease, injury and disability, and preparedness for new health threats."

In the realm of cancer, CDC works with national cancer organizations, state health agencies and other relevant groups to develop, implement, and promote effective strategies for preventing and controlling cancer.

CDC has a National Program of Cancer Registries and provides data on cancer incidences and death.

Congressional Committees

  • The Senate Committee on Health, Education, Labor & Pensions (HELP) has jurisdiction over most of the agencies, institutes and programs of the Department of Health and Human Services, including the FDA, CDC, NIH and others. The Committee also oversees public health and health insurance statutes. The Senators who are members of the Committee have great influence over the legislation affecting health care policy as they have opportunities to question witnesses at committee hearings on related topics and to amend legislation that is considered by the committee in its areas of jurisdiction.
  • The Senate Appropriations Committee has jurisdiction over the funding provided to government agencies, including health care agencies such as NIH and FDA. Specifically, the Subcommittee on Labor, Health and Human Services (HHS) and Education holds hearings on and drafts appropriations legislation affecting agencies within the Department of HHS.
  • The House Committee on Energy and Commerce has jurisdiction generally over health and health facilities, biomedical research and development and public health. The representatives assigned to this committee have great influence over health-related legislation.
  • The House Appropriations Committee has a Subcommittee on Labor, HHS, and Education, which has jurisdiction over funding of key government agencies involving in health care, research and drug development.

The House also has a Childhood Cancer Caucus, with members who have an interest in pediatric cancer. While the caucus does not have specific legislative authority, it is able to serve as a clearinghouse for information on pediatric oncology issues and can use its collective power to influence policy.