Federal Advisory Committees
One way for advocates to get involved with agencies and the regulations they produce is to become part of or present information to Federal Advisory Committees. Advisory committees are responsible for counseling the President and the Executive Branch on a wide range of issues. They, therefore, play an important role in shaping the policies and programs of the federal government. Today, an average of 1,000 advisory committees serve this purpose, providing advocates like you with a plethora of opportunities to further issues important to the PI community.
To ensure that various advisory committees' advice is objective and accessible to the public, the Federal Advisory Committee Act (FACA) was enacted in 1972. Through this act, Congress formally recognized the value of seeking the advice and assistance of citizens and advocates through advisory committees. FACA oversees many committees falling under the Department of Health and Human Services (HHS) umbrella relevant to the PI community, including the Advisory Commission on Childhood Vaccines, the Blood Products Advisory Committee, and the Advisory Committee on Heritable Disorders in Newborns and Children. Tools such as the FACA Database allow advocates such as those in the PI community to influence these committees and the policies they shape.
Federal Agency engagement on issues important to the PI community will most likely require engagement with HHS or one of its centers.
Department of Health and Human Services (HHS)
HHS is responsible for a large volume of proposed rules surrounding public health and healthcare issues and is, therefore, a reliable and consistent outlet for agency advocacy. HHS regularly develops regulations of consequence to the PI community, such as expanding research in therapies and ensuring patient health coverage. HHS has 11 operating divisions, including eight agencies in the U.S. Public Health Service and three human services agencies. These divisions administer a wide variety of health and human services and conduct lifesaving research for the nation, protecting and serving all Americans. Divisions that IDF typically engage with include the CDC, CMS, FDA, HRSA, and the NIH. Each division consists of centers, offices, and institutes that focus on specific functions or areas of oversight. The HHS organizational chart includes links to each agency’s chart showing the breakdown of offices, centers, and institutes. The centers are generally where rules and regulations are developed by staff experts in the particular area of focus. For example, the FDA has nine center-level organizations, including the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER), that focus on policies related to biologics and drug development and approval.
Opportunities for HHS and other agency advocacy generally entail engagement with staff from an agency center. In developing rules and regulations, the center staff will develop drafts shared with the public to allow those who might be affected to comment. All notices of draft and final federal rules are published in the daily federal register available online. This database is searchable. The notices include a summary of the issues, dates of publication, deadlines for opportunities to comment, and instructions on submitting comments electronically or by mail or hand delivery. Submitting public comments is an effective and simple way to engage with large agencies such as HHS and allow the PI community's interests to be represented in regulatory discussions. Since there are innumerable draft rules and regulations published by HHS every week, the best way to identify what rules may affect the PI community is to work with IDF to focus your efforts.
The CDC is the nation’s leading public health agency. CDC focuses on a number of issues including infectious diseases, birth defects and development disabilities and chronic conditions like PI. IDF engages with CDC on multiple issues like public health issues of importance to our community, such as the COVID pandemic, vaccination policy, and more.
CMS oversees the Medicare and Medicaid programs. IDF works very close with CMS on issues pertaining to Medicare beneficiary access to PI therapies, particularly immunoglobulin (Ig) treatments. This includes the ongoing IVIG bundled payment demonstration program and implementation of Medicare’s home infusion services payment.
The FDA protects the public’s health by ensuring the safety, efficacy, and security of human drugs, biological products, and medical devices. Effectively advocating to this federal agency allows those in the PI community to influence FDA regulations and ensure that such regulations are supporting the needs of those with PI.
HRSA runs a number of healthcare programs including the nation’s Newborn Screening program. This issue is very important to IDF as federal recommendations call for the screening of all newborns for SCID. IDF worked to get SCID onto the Recommended Uniform Screening Panel or RUSP and to then work to pass legislation so all states test for SCID.
The NIH is the world’s largest funder of biomedical research. IDF works closely with the NIH, particularly the National Institute of Allergy and Infectious Diseases (NIAID) which leads research on primary immunodeficiencies (PI).
While not all issues addressed by the preceding resources are relevant to the PI community, keeping an eye on the actions of these groups and getting involved in consequential topics is an excellent way to influence the rules and regulations created by different agencies. There are a large number of tools available to advocates, so don’t limit yourself to those explicitly listed here. This is a team effort, and IDF is always here to help navigate agency advocacy, whether it is with local, state, or federal entities. If there is ever a question about your advocacy, don’t hesitate to ask IDF staff for assistance.
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