Understanding the Policy Landscape
The Congressional Session
Ever hear a bill referred to by a number like “H.R. 1731” or “S. 652” and wonder what it really means or why it disappears after a while? That’s where understanding which Congress we’re in comes in handy.
Congress is numbered in two-year sessions. Each session begins in January of odd-numbered years. For example:
The 118th Congress ran from January 2023 to January 2025.
The 119th Congress runs from January 2025 to January 2027.
This number is used to track everything from bills to committee hearings to member directories.
If you’re trying to look up a bill or advocacy campaign, knowing the session number helps you:
Find the right version of a bill (many are reintroduced each session).
Understand the timeline for when a bill can be passed.
Search official sources, like Congress.gov, more efficiently.
Example: If you supported a bill during the 118th Congress, but it didn’t pass, and it was reintroduced in the 119th Congress, the bill number might change, even if the bill is nearly identical.
Advocacy Tip:
When speaking with legislators or doing research, ask or check:
What Congress are we in?
Has this bill been reintroduced?
What’s the new bill number, and who’s sponsoring it?
Federal vs State Policy
Federal
Federal Policy affects the entire country. It includes nationwide programs like the Orphan Drug Act, NIH research funding, Medicaid and Medicare rules, and FDA approvals. These policies are typically created by Congress (House of Representatives and Senate) and carried out by federal agencies.
State
State Policy controls issues that vary by location, like Medicaid eligibility, newborn screening panels, rare disease advisory councils, and public health services. Each state has its own legislature and regulatory agencies that make decisions that may help or hinder rare disease access.
Key Differences
Bill Numbers: Federal bills begin with “H.R.” (House) or “S.” (Senate). State bills have their own formats (varies by state).
Decision-Makers:
Federal: U.S. Representatives and Senators
State: State Representatives and State Senators
Federal advocacy impacts national change; state advocacy can be faster and more personal, and often affects your immediate access to care.
Who Do You Talk to About a Bill?
When you’re advocating for a piece of legislation, knowing who to talk to and when is just as important as what you’re advocating for. In the U.S. Congress, bills are introduced in two separate chambers: the House of Representatives and the Senate. Each version of a bill has to be passed by both chambers before it becomes law. That means there are two sides to almost every federal bill: a House bill and a Senate bill.
Here’s how to know who to bring your advocacy to:
If you’re supporting a Senate bill (it’ll start with “S.” followed by a number like S.123), reach out to your U.S. Senators. Each state has two Senators, and they vote on Senate versions of legislation.
If you’re supporting a House bill (it’ll start with “H.R.” like H.R.456), contact your U.S. House Representative. They represent your specific congressional district.
Tip: If you’re advocating to a member of Congress and only a Senate or House version exists so far, your job may be to ask them to sponsor or co-sponsor the companion bill in their chamber.
For example: “Senator X is sponsoring S.123 to improve rare disease coverage. Would you be willing to introduce or co-sponsor the House companion version when it becomes available?”
Be Strategic
Advocating for the wrong bill to the wrong person doesn’t mean your efforts are wasted, but it can delay progress. When you know whether you’re talking to a Senator or Representative and match it with the right version of the bill, you give them a clear and actionable next step.
Also, some state-level bills can have confusingly similar names or numbers as federal ones, so always double-check whether you’re advocating at the federal or state level.
Major Players
When Congress passes a law, the process doesn’t end there. A wide network of stakeholders, ranging from federal and state agencies to advocacy organizations, works to turn those policies into tangible outcomes for the rare disease community.
Think of Congress as the architect that designs the blueprint, and the diverse players at every level as the builders, advocates, and enforcers who bring it to life.
Federal agencies implement, regulate, and oversee programs, ensuring that laws are carried out as intended. State-level players, including governors, legislators, and health departments, focus on adapting federal policies to the local context and managing state-specific programs.
Advocacy organizations and patient-led nonprofits amplify the voices of those affected by rare diseases, advocate for additional legislative measures, and hold lawmakers and agencies accountable for making meaningful progress. Together, these stakeholders form a collaborative network that drives policy forward, working at every level to ensure laws translate into real-world impact.
Federal
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Members of Congress (Senators (2 per state) and Representatives (based on population)) are the elected officials who write, sponsor, and vote on legislation at the federal level. They play a critical role not only in creating laws that affect rare disease funding, access, research, and equity, but also in carrying them out through ongoing engagement. After a bill becomes law, members continue to shape its impact by conducting oversight, advocating for funding through the appropriations process, and responding to constituent concerns. They can introduce follow-up legislation, request agency reports, and participate in caucuses or committees focused on rare disease. Their active involvement helps ensure that policies are implemented as intended and that any gaps or challenges are addressed.
Example: A senator who co-sponsored a rare disease bill may later work to secure CDC funding to implement a new data collection initiative authorized by the law or hold a congressional hearing if rollout delays occur.
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A bipartisan group in Congress that champions rare disease issues. While it doesn’t pass laws directly, its members bring attention to rare disease priorities and often co-sponsor legislation that advocates promote.
Example: After rare disease advocates raise concerns about insurance barriers to clinical trials, members of the Caucus may host a bipartisan briefing, introduce legislation to fix coverage gaps, and push relevant agencies to prioritize access and equity in rare disease care.
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The U.S. Department of Health and Human Services (HHS) is a critical agency in carrying out policies created by Congress, overseeing and coordinating a wide array of agencies (CMS, FDA, NIH, CDC, HRSA), programs, and services aimed at improving the health and well-being of Americans, including those impacted by rare diseases. As the cabinet-level department responsible for health-related matters, HHS sets the broad policy direction for public health, medical research, and services.
Example: When Congress passes legislation like the 21st Century Cures Act, which includes provisions for increasing funding for rare disease research, HHS is tasked with executing these provisions. It directs its agencies, such as the National Institutes of Health (NIH), to allocate funding to research centers, establish new initiatives, and guide the development of clinical trial networks. HHS also facilitates coordination among its divisions, including CDC and HRSA, ensuring that policies on drug development, patient access to treatments, and data sharing are carried out effectively. Additionally, HHS manages national strategies, administers grants, and ensures that the goals of policies, such as improving access to healthcare, advancing research, and addressing health disparities, are achieved.
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CMS administers health insurance programs for millions of Americans. If Congress changes Medicaid policy, CMS works with each state to apply those changes, affecting what services patients can access and how much they cost. CMS administers major public health insurance programs (Medicare, Medicaid, and CHIP). It plays a crucial role in deciding what treatments are covered and reimbursed, directly impacting rare disease patients’ access to care.
Example: When Congress authorizes expanded Medicaid benefits for rare disease patients, CMS issues guidance to state Medicaid directors on how to implement the changes, such as covering new genetic testing panels or reimbursing out-of-state specialists for ultra-rare conditions.
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Once Congress passes legislation like the Orphan Drug Act, the FDA plays a critical role in turning policy into practice. It regulates drugs, biologics, and medical devices, and is responsible for reviewing and approving new rare disease therapies. The FDA sets the guidelines that researchers and companies must follow, oversees clinical trial safety, and ensures that treatments are safe and effective before they reach patients. Its regulatory decisions and enforcement of key rules directly shape how quickly new therapies become available, making it a central force in bringing innovation from lab to life.
Example: Following the Orphan Drug Act, the FDA created the Office of Orphan Products Development to incentivize rare disease drug development by offering tax credits, fee waivers, and market exclusivity to pharmaceutical companies. The FDA reviews applications under these incentives and approves therapies deemed safe and effective.
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The NIH is the nation’s medical research agency, responsible for funding and conducting biomedical research that advances our understanding of health and disease. When Congress allocates research funding, the NIH distributes it to scientists, academic institutions, and research centers, including those focused on rare diseases. Within the NIH, the National Center for Advancing Translational Sciences (NCATS) and the Office of Rare Diseases Research (ORDR) lead efforts specifically targeting rare disease research. They support key initiatives like the Rare Diseases Clinical Research Network (RDCRN), which facilitates natural history studies and clinical trials essential to developing new treatments and understanding the patient journey.
Example: After Congress allocates rare disease research funding, NIH may direct resources through NCATS and its Office of Rare Diseases Research to expand the Rare Diseases Clinical Research Network (RDCRN), supporting natural history studies and clinical trials for dozens of rare conditions.
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Once Congress enacts legislation related to public health, data collection, or early detection, such as initiatives for newborn screening or rare disease surveillance, the CDC plays a vital role in implementing those policies. As the nation’s lead public health agency, the CDC collects and analyzes health data, monitors disease trends, and develops national guidelines for prevention and early diagnosis. For rare diseases, it supports states in standardizing newborn screening programs, tracks disease prevalence, and works to identify gaps in diagnosis and care. The CDC’s work helps shape public health strategies, guide funding decisions, and provide the evidence base that policymakers and health systems rely on to respond effectively to rare disease challenges.
Example: Following the passage of federal policies supporting newborn screening expansion, the CDC collaborated with the Health Resources and Services Administration (HRSA) and state programs to enhance data systems, improve test accuracy, and promote uniform screening across the country. This effort helped ensure that babies with rare but treatable conditions, like severe combined immunodeficiency (SCID), could be identified and treated early, dramatically improving health outcomes.
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HRSA often funds newborn screening programs and health equity initiatives. They carry out policies that impact how early certain rare diseases are diagnosed, especially for underserved populations.
Example: After Congress includes funding in the federal budget for expanding newborn screening programs, HRSA may administer those funds through grants to state health departments, ensuring that new screening panels are implemented and public health staff are trained accordingly.
State
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Each state has its own legislative body made up of elected lawmakers who draft, introduce, and vote on policies that directly impact rare disease patients at the local level. These include decisions around Medicaid expansion, newborn screening programs, access to specialists, rare disease advisory councils, and genetic testing mandates. State legislators play a critical role in setting healthcare priorities, and advocates can engage them by sharing personal stories, proposing bill ideas, or providing testimony during committee hearings.
Example: A state legislator may introduce a bill to establish or fund a Rare Disease Advisory Council, expand coverage of genetic testing under Medicaid, or mandate rare disease education for providers.
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Governors set public health priorities for their states by proposing budgets, issuing executive orders, and signing bills into law. They oversee state agencies that carry out these policies and can champion initiatives that expand access to care for rare disease patients. Working closely with governors, state health commissioners, often appointed by the governor or legislature, lead state health departments and are responsible for implementing healthcare programs, enforcing regulations, and directing the allocation of public health resources. Their decisions can significantly impact how rare disease services are prioritized, delivered, and monitored across the state.
Example: A governor might advocate for a statewide newborn screening expansion, while the health commissioner determines how the program is rolled out, including timelines, provider training, and reporting systems.
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Each state administers its own Medicaid program within federal guidelines, determining eligibility, coverage, and reimbursement for healthcare services. Since Medicaid is jointly funded by state and federal governments, your access to care often depends heavily on where you live. For many rare disease patients, especially those who are medically complex or from low-income backgrounds, Medicaid is the primary or only source of insurance. State Medicaid directors have the authority to approve or deny coverage for life-saving treatments, clinical genetic testing, orphan drugs, or visits to rare disease specialists. Their decisions are shaped by state budgets, clinical criteria, and broader health policy goals.
Example: A state Medicaid program may approve a new orphan drug only for patients who meet strict clinical criteria, or deny it altogether due to cost concerns.
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Rare Disease Advisory Councils (RDACs) are formal bodies established at the state level to bring together patients, caregivers, healthcare providers, researchers, and policy experts to advise government leaders on the unique needs of the rare disease community. While their recommendations are not legally binding, they play a crucial role in shaping policy by offering expert, patient-informed insights that might otherwise be overlooked. RDACs help legislators, governors, and health officials understand barriers to care, gaps in provider education, and opportunities to improve diagnosis, treatment access, and coordination of care.
Example: An RDAC may issue a policy report recommending expanded provider education on rare diseases or the creation of specialized care networks across the state.
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Local public health departments are responsible for implementing state and federal health policies at the community level. They manage frontline services such as immunizations, health education, disease surveillance, and in some regions, newborn screening and early diagnosis programs. These agencies play a critical role in connecting rare disease policy to real-world impact by coordinating public awareness campaigns, supporting outreach efforts, and administering grant-funded programs that may include rare disease components. Their work helps ensure that patients and families benefit from larger health initiatives, especially in underserved or rural areas.
Example: A local health department may collaborate with hospitals to promote newborn screening education or operate a mobile clinic offering genetic counseling in remote communities.
Advocacy Organizations & Stakeholders
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The EveryLife Foundation is a policy-driven nonprofit dedicated to advancing science-based legislation that improves the lives of people with rare diseases. Operating at both the federal and state levels, EveryLife empowers patients and families to engage in the legislative process by providing advocacy training, organizing events like Rare Disease Week on Capitol Hill, and facilitating meetings with lawmakers through its Rare Disease Legislative Advocates (RDLA) program. The Foundation leads coalition-building efforts, develops model legislation, and publishes influential reports, such as the National Economic Burden of Rare Disease Study, to drive data-informed policy change.
Example: EveryLife may draft a policy brief on a bill like the STAT Act or BENEFIT Act, then help patient advocates use it in scheduled meetings with their legislators to build support.
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NORD is a leading umbrella organization that advances rare disease advocacy, education, research, and patient support across the country. It plays a critical role in shaping federal and state policy by tracking legislation, submitting testimony, and promoting access to treatment and protections for rare disease patients. NORD supports the development and sustainability of Rare Disease Advisory Councils (RDACs), offers direct services like patient assistance programs, and manages the IAMRARE registry platform to accelerate research. Additionally, it helps build and maintain rare disease Centers of Excellence across the U.S.
Example: NORD may assist in establishing an RDAC in a state without one, while also providing legislators with research and policy guidance to support new rare disease legislation.
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RDDC is dedicated to eliminating health disparities and advancing equity for historically underserved populations in the rare disease space. The coalition brings together patients, advocates, clinicians, and industry leaders to inform and influence federal legislative and administrative processes. RDDC works closely with members of Congress and federal agencies to support and introduce policies that improve equity in clinical trials, telehealth access, newborn screening, rural healthcare, and rare disease funding. By mobilizing diverse voices and expertise, RDDC plays a key role in shaping an inclusive healthcare landscape.
Example: RDDC championed the HEARD Act and PROTECT Rare Act to ensure diverse patient populations are represented in research and have equitable access to diagnosis, treatment, and participation in clinical trials.
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Global Genes is a global advocacy organization that empowers rare disease communities through education, strategic resources, and capacity-building. While not solely focused on policy, it plays a vital role in helping grassroots leaders, patient advocates, and small nonprofits understand and engage in policy efforts. Through toolkits, summits, and partnerships, Global Genes equips advocates with the knowledge and connections needed to influence change, whether locally, nationally, or globally. It also fosters coalition-building and promotes cross-community collaboration across the rare disease ecosystem.
Example: Global Genes may host a legislative briefing or training session to help advocates learn how to build alliances and effectively support policy change in their states or communities.
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Often founded by patients or caregivers, these grassroots organizations are dedicated to a single rare condition and play a critical role in shaping disease-specific policy. They advocate for targeted legislative and regulatory changes, fund and promote research, support affected families, and work directly with lawmakers to improve access to care, treatment coverage, and awareness. While highly specialized, these groups often collaborate with larger advocacy coalitions to amplify their impact and ensure that the unique needs of their communities are represented in broader policy conversations.
Example: A Duchenne muscular dystrophy foundation may advocate for Medicaid coverage of respiratory support devices, while a histiocytosis organization might push for increased NIH research funding or inclusion in federal data collection programs. Advocacy is based on the specific foundation’s priority initiatives and needs of the patient community.
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While they aren’t government agencies or advocacy groups, biopharmaceutical companies, assistive technology manufacturers, and access and long-term care providers are important players in the rare disease policy ecosystem. They often respond to, influence, and help shape policies that affect innovation, access, affordability, and patient quality of life.
These stakeholders are involved in areas like:
Regulatory policy (working with the FDA on drug/device approvals)
Reimbursement and access policy (collaborating with CMS or private insurers to determine coverage and pricing)
Legislative advocacy (supporting or opposing bills related to drug pricing, incentives, or medical innovation)
Partnerships (funding research, participating in coalitions, and supporting patient-led policy initiatives)
They may also sponsor and attend congressional briefings, submit public comments on proposed regulations, and partner with advocacy groups to advance shared goals.
Example: A biotech company developing a gene therapy for a rare disease may engage with FDA regulators through the accelerated approval pathway, lobby Congress to support tax credits under the Orphan Drug Act, and work with CMS to define how the therapy will be reimbursed under Medicaid. Meanwhile, an assistive device company might advocate at the state level to ensure Medicaid covers essential technologies for patients with rare neuromuscular disorders.
Access Determined by Zip Code
In rare disease, geography is everything. One state might fully fund newborn screening, offer Medicaid expansion, and operate an active Rare Disease Advisory Council. Another state might offer none of these. Even within a single country, the quality and accessibility of rare disease care can differ dramatically depending on where you live.
This is why cross-state and national advocacy is critical and why policies must be equitable.
How You Can Help
Whether you’re a patient, caregiver, researcher, or industry partner, your story and expertise are vital. Advocacy isn’t limited to protest or politics, it’s everything from joining a workgroup to helping draft legislation to showing up for a lobby day.
Ways Stakeholders Can Collaborate Across Policy Levels:
Share your story with legislators (federal and state).
Join a Rare Disease Advisory Council or policy task force.
Help translate data into lived experience.
Partner with nonprofits to create equity-centered policy briefs.
Offer input on regulatory comment periods for new policy changes.
When you understand the rare disease policy map, you realize we’re not in separate lanes, we’re building the road together.
