The National Cancer Institute (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 and 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.
There are several programs supported by NCI that focus on pediatric oncology. For example, the Childhood Cancer Therapeutically Applicable Research to Generate Effective Treatments program 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 Tumor. A number of factors were considered when selecting these cancers, including prevalence among children, inadequacy of current treatment options, and availability of appropriate human tissue collections.
The Pediatric Preclinical Testing Program is supported by the NCI through a contract with the Research Institute at Nationwide Children’s Hospital in Columbus, Ohio. Dr. Peter Houghton is the principal investigator. 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 treating pediatric cancers.
The Childhood Cancer Survivor Study (CCSS) is funded by a grant from the NCI to St. Jude Children’s Research Hospital. CCSS is a collaborative, multi-institutional project of individuals who survived five or more years after diagnosis of childhood cancer. The study seeks to gain knowledge about the long-term effects of cancer that will help in the design of treatment protocols and intervention strategies that improve survival while minimizing harm to patients. The cohort being studied includes 20,346 childhood cancer survivors diagnosed before age 21 between 1970 and 1986 and approximately 4,000 siblings of survivors, who serve as a control group. The cohort is being expanded to include an additional 14,000 childhood cancer survivors diagnosed before age 21 between 1987 and 1999.
The NCI also provides data through the Surveillance, Epidemiology, and End Results (SEER) Program, including childhood cancer statistics.
There are a range of opportunities for advocates to become involved on advisory committees, in scientific review and in providing feedback on NCI educational materials and communications.
The formation of the Director's Consumer Liaison Group (DCLG), a chartered federal advisory committee created in 1997, 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 includes 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.
OAR oversees CARRA (Consumer Advocates in Research and Related Activities), a program formed in 2001 to regularize cancer advocates’ involvement in the review of extramural scientific grants and NCI cancer communications. CARRA advocates are selected to represent diverse populations so as to lend valid community input to reviews. CARRA’s aim is to be the mechanism through which all NCI lay reviewers are chosen and assigned
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.
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 FDA’s 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 exclusivity and patent protection. As a complement to the incentives offered by BPCA, the Pediatric Research Equity Act (PREA) requires pharmaceutical companies to test new drugs in a pediatric population when the drug is likely to be used in children.
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 Special Health Issues (OHSI) and its Cancer Liaison Program oversee these efforts.
FDA’s Cancer Patient Representative Program regularizes the process of recruitment, assessment and assignment to advisory committees. Patient representatives serve on advisory committees in three cancer relevant divisions: the Center for Drug Evaluation and Research (CDER); the Center for Biologics Evaluation and Research (CBER); and the Center for Devices and Radiological Health (CDRH). These advocates are expected to have direct experience with a particular cancer, access to cancer community concerns through participation in a patient advocacy group and good communication skills. Patient representatives have the authority to vote on most advisory committees.
Perhaps the most salient advisory opportunity for childhood cancer advocacy is the Pediatric Subcommittee of the Oncologic Drugs Advisory Committee (Ped ODAC). 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.
The CDC is one of the agencies within the Department of Health and Human Services (HHS). Its 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. For childhood cancer, the Caroline Pryce Walker Conquer Childhood Cancer Act of 2008 provided funding for a grant via the CDC to “enhance and expand infrastructure to track the epidemiology of pediatric cancer into a comprehensive nationwide registry of actual occurrences of pediatric cancer.”
There are a number of entities in the U.S. Congress that impact cancer care policy.
In the U.S. Senate, the Committee on Health Education Labor and Pensions (HELP) has jurisdiction over most of the agencies, institutes, and programs of the Department of Health and Human Services, including the Food and Drug Administration, the Centers for Disease Control and Prevention, the National Institutes of Health, the Administration on Aging, the Substance Abuse and Mental Health Services Administration, and the Agency for Healthcare Research and Quality. 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 the appropriations legislation affecting agencies within the Department of HHS.
In the U.S. House, the 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.
Like the Senate, the House has an Appropriations Committee with a Subcommittee on Labor, HHS, Education that has jurisdiction over funding of key government agencies involved in health care research and drug development.
The House also has established a Pediatric Cancer Caucus. Members who have an interest in childhood cancer may join this group. 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.
Making your views know to your representatives in Congress is an important way to advocate for policies that help children with cancer. This can be done by calling, writing or visiting a Members’ of Congress or their staff, either in their home state or in Washington, DC. Representatives and Senators are more responsive to constituents than to individuals who do not reside in their district or state.