Introduction

What is Advocacy?

Anyone can be an advocate. Advocates are people who share their stories about how a system is affecting them personally and urge decision makers to take action. Your representatives in state government or in the U.S. Congress can vote to support legislation that expedites diagnosis, access to treatment, and help with treatment costs, just to name a few areas. You do not need special skills to be an advocate. You only need to contact your elected representatives. Sharing your story with legislators can have a huge impact on rare disease awareness. It can help spark the development of new policies and provide greater access to care that can promote a better life for you, your loved ones, other rare disease patients, and their caregivers. The goal of this toolkit is to provide information and resources to help you build confidence with advocacy, understand what is decided by states and what is decided by the federal government, and identify ways that you can raise awareness for your rare disease.

This toolkit focuses on the following topics:

1. How advocacy works
2. How to craft a compelling narrative
3. Differences between advocacy at state and federal levels
4. Advocating at the state level
5. Advocating at the federal level
6. Generating awareness for your cause
7. Health policy and regulations

How Does Advocacy Work?

Lawmakers like to hear from their constituents. They serve you. It’s their job to represent the interests of people in their district. Your representatives may not know about your rare disease or challenges that affect people with rare disease. And they won’t know about how rare disease affects you unless you tell them. Lawmakers are more likely to support legislation that receives support from their constituents. Advocacy is letting your representatives know which legislation you would like them to support—or to vote against. It’s as important to defeat legislation that reduces funding for research or imposes restrictions on healthcare coverage as it is to promote the passage of legislation that addresses challenges related to rare disease. It’s a good idea to do some preparation before you reach out to your representatives. You do not need to know everything about rare disease, research, or funding. While each advocate’s journey is different, there are several common steps to beginning advocacy (Figure 1).

Fact Finding

Before you begin to craft your story, you may need to do some research on your representatives or rare disease. This will ensure that you’re contacting the right person and providing accurate information. You may also discover information that inspires you to join a group or focus your advocacy efforts.

Who Represents You?

You probably know the names of your congressperson who represents your neighborhood in the House of Representatives, and most people know the names of the two senators who represent their state. You can find your congressperson and senators on the websites for the House of Representatives and Senate.

There are some healthcare items, such as Medicaid, that are decided at the state level so you may need to reach out to your elected representatives in your state’s legislative body. Open States helps people to participate in legislation being considered by state governments. Its database is a quick way to find your elected officials in your state’s legislature.

It’s good to note your representatives’ phone numbers in Washington, D.C. or the state capitol, as well as at their local office. Each representative has an office in the district that he or she represents. It’s often easier to schedule a meeting with a representative in their district office, as representatives spend portions of the year in their local offices, not in Washington, D.C. or the state capitol. Most elected officials send newsletters to their constituents. You may already get this newsletter in the mail. You can also sign up on the representative’s website to be added to their email list for press releases and other information.

Before you reach out to your representative, it may be useful to know his or her record on healthcare issues. Open States allows you to search for recently passed laws in your state so you can find out how representatives voted. Pending bills are also shown. Rare Disease Legislative Advocates, a program of the non-profit EveryLife Foundation for Rare Diseases, provides legislative scorecards on members of Congress.

What’s in a Title?

Legislators are referred to by a number of titles. Some of the commonly used titles:

• Lawmaker
• Representative
• Elected official
• Legislator
• Policymaker
• Member (federal)
• Congressman (federal)
• Congresswoman (federal)
• Congressperson (federal)

It can also be good to know if your legislators serve on healthcare-focused committees. At the federal level, most representatives and senators include a list of committees on which they serve. The Senate has the Committee on Health, Education, Labor, and Pensions. In the House of Representatives, the Energy and Commerce Committee oversees healthcare and health insurance. In addition, several other committees–Appropriations Committee, Veterans’ Affairs Committee, and the Ways and Means Committee–have health subcommittees.

As with any new relationship, building rapport is important. You may want to spend time looking at your representative’s website, social media accounts, and record on legislation. Rapport stems from common areas: education, hobbies, sports teams, and charitable affiliations, among others. Rapport can also arise when you thank your representative for supporting specific legislation or serving on a committee.

Knowing details about your representative is not a prerequisite for advocacy. However, building rapport can help the representative remember you and want to be your champion. Keep an open mind if you discover something unfavorable or not relevant to your cause. Your goal is to influence your representative to support policy and legislation that positively affects people with rare disease.

Get Information on Your Rare Disease

Your representatives may not know about your rare disease and how many people it affects. Because of this, you might want to include a short description of the rare disease and its impact on daily life in your communications with representatives.

If there are no treatments for your rare disease, you might want to advocate for increased funding for the National Institutes of Health (NIH), which is the federal agency charged with medical research.

The National Organization for Rare Disorders (NORD) provides information on more than 1,300 rare diseases. For most conditions, the summaries include a description, symptoms, causes, the number of people with the rare disease, diagnosis, and treatment options. Nonprofit groups and associations that belong to NORD for each rare disease are also shown. Rare disease advocacy organizations often provide information on pending legislation. Some rare disease groups have advocacy networks, which connect people in rare disease and encourage people to contact their representatives en masse in order to have a greater impact.

Jenn McNary on Advocacy and Research

“My first son was diagnosed with Duchenne Muscular Dystrophy just after his third birthday. I was 21 and had two sons at that point and they were diagnosed one after the other with Duchenne. My first entrance into advocacy was actually looking for a diagnosis. I knew something was wrong with my son, but the pediatrician didn’t agree with me. The first time I found myself heavily advocating, I was saying, ‘there’s something wrong with my son. We need to do something.’ I basically forced them to do more testing. I didn’t start out getting an honorary degree in health economics. I started off with getting some help with diagnostics so that’s what I researched. And then when my son got into a clinical trial, I learned how clinical trials work. When I wanted access for my other son who wasn’t in a clinical trial, that’s when I learned how regulatory process and early approvals worked. After the drug was approved, I learned about access and reimbursement because Medicaid can deny coverage for an FDA approved drug. It’s important to have targeted goals. I think new advocates should focus on bite-sized chunks of information that they need, not researching and learning everything at once. Really focus on what is right now. Do you need science? Do you need to know about access to medication? Let that guide your research and learning.”

Craft a Compelling Narrative

A narrative is a story. Sharing a story helps people process and remember information. Stories that focus on one or two main points are easier for a listener to follow and understand. Remember writing about themes of a story in high school? Developing your narrative for advocacy can be similar if you focus on one of the “themes” of rare disease (Figure 2). Lawmakers are familiar with these elements, too, as they probably have introduced, seen, or voted on legislation in one of these areas. The best stories are personal, engage the listener, and are delivered with passion and conviction. Specific examples are more memorable than general statements. For instance, it may be more effective to say, “it took me 9 months to get a diagnosis” than “I had to wait a long time for diagnosis.” Talking about the emotional or financial burden of a rare disease can also resonate with a lawmaker. You want your representative to emotionally connect with your story because that can convince him or her to sponsor or support legislation that can improve your life. People you are meeting with may not have a science or medical background, or know about the condition you are representing. You can use relatively simple, conversational terms to describe the nature or symptoms of the condition.

What’s Your Intention?

Your intention—the reason you are contacting your legislator—directs the story. In certain situations, the intention (the “ask”) is clear. It’s common for people to reach out to their representatives to ask the lawmaker to support or oppose a pending piece of legislation. Staffers at the representative’s office actually count the number of people who support or oppose bills to gauge constituent sentiment. If you’re not contacting your representative about a particular bill, you might want to link your narrative to the “ask” you’re making. Do you want your lawmaker to fund research? If so, you might want to point out that there are no, or few, treatments for your rare disease. Are you asking your representative to expand healthcare coverage? You could point out what your insurance doesn’t cover. Does your medication have a prohibitive cost? Do you see a need for more home healthcare funding? Any of these could be part of the “ask,” and thus, warrant a personal detail about the impact on you. Sometimes the intention is not tied to a request for the legislator to take a specific action. You may want to raise awareness about your rare disease or how rare disease affects 1 in 10 Americans. This, too, is an important part of advocacy and will direct how you craft your narrative.

PRO TIP: GETTING STARTED WITH ADVOCACY

Opportunities to advocate abound. This can be daunting if you’re a first-time advocate. Long-time advocates recommend:

• Focus your attention one or two areas that you’re passionate about.
• Set a goal. Include a timeframe in that goal.
• Look for patient advocacy groups for your rare disease. Take advantage of advocacy tools that have already been built and are available for free.
• Look for advocacy groups for your area of interest, such as treatment costs or access to care.
• Remember: You don’t have to know everything about a rare disease, insurance, science, or regulations. You learn things that you need to know as you advocate.
• Meet others with rare disease by attending conferences, webinars, and other events. You can gain important information about rare disease, diagnosis, treatments, health policy, insurance, and other areas.
• Think of advocacy as a marathon, not a sprint. Advocacy takes time. Pace yourself!

How to Organize Your Story

Some people like to share their story orally, others prefer to write letters or emails. Regardless of the medium for communicating your story, you should always be brief, clear, and polite. You should include basic information, such as your name, your address or city, your organization if you’re calling as part of a legislative action of a rare disease group, and why you’re contacting the elected official.

The hook, line, and sinker method is an easy way to organize your story:

The “hook” is your opening statement. Your salutation and hook statement may be: “Dear Representative ________, a rare disease is impacting my child’s development.”

The “line” gives the details of the hook: These details are the name of your disease and other related info. For instance, “Galactosemia almost killed my daughter at birth. She follows a strict diet and has had developmental delays.”

The “sinker” is asking the representative to help you with your specific advocacy goals, such as, “Please support additional funding for the National Institutes of Health to encourage research in galactosemia and other rare diseases. The Orphan Products Grant program at the FDA also needs additional funding to support clinical trials of treatments for rare disease.”

If you would like to receive a response, make sure that you include that in your message, email, or letter, along with your contact information. You should also prepare short and longer versions of your story. For instance, when leaving a voicemail for a representative, you should try to keep the message to about 30 seconds. If you get a staffer on the phone or meet your representative in person, you’ll be able to share more parts of your story.

Similarly, an initial email or one encouraging a lawmaker to vote for a specific bill does not need to be lengthy. Brevity is appreciated by legislators and their staff. Longer emails or letters are appropriate if your story illustrates a particular rare disease challenge, such as difficulty in diagnosis or hurdles for accessing treatments. Other ways to share your story are discussed in “Generating Awareness for Your Cause” section later in this toolkit.

Start Your Advocacy!

Once you’ve done your research, identified your intention (and “ask”), and crafted your message, you’re ready to contact your representative. You may feel nervous. Remember that they work for you!

Make Contact

Many first-time advocates practice before calling their representatives. Even experienced public speakers practice what they’re going to say because it helps them feel more confident. You might want to contact your representative’s local district office, which can be less nerve wracking and provide a more relaxed interaction. Sending emails is less intimidating for some people. Remember to proofread your email for any typos before you hit send.

Follow Up!

It’s important to keep in touch with your representatives. It only takes a few minutes to make a call or send an email. If you asked for a response and didn’t get one, wait a couple of weeks and then reach out again. If you get a legislative win, it’s appropriate to send your lawmaker a note of congratulations.

In addition, if your representative serves on a relevant committee, you can sign up for alerts for committee meetings or hearings to remind you to contact your representative. Some advocates find it helpful to create a calendar of upcoming committee meetings or events. Others check in with their elected officials once every few months. Some send holiday cards. Do whatever works for you. Unless you’ve bonded with your representative, don’t send home-baked goods or birthday bouquets.

Meet Your Representative

Legislators’ calendars are often full of committee meetings, learning about issues, drafting bills, floor debates and votes, and other legislative activities. Many constituents also may be clamoring for in-person meetings. For these reasons, a Town Hall in your local area may be a great way for you to meet with your representative for the first time. Contact your representative’s offices and find out when the next Town Hall is scheduled. Ask a question during the Town Hall or introduce yourself after the event has concluded.

You can arrange a meeting with your representative without having been to a Town Hall. Representatives primarily work in their state capitol offices or on Capitol Hill in Washington, D.C. During session breaks they are often “in-district,” which means they are home in the jurisdiction they represent as a legislator. Contact their capitol or local office to see if you can get an appointment with them the next time they are home.

Legislators usually have staff members who work in different policy areas: healthcare, economics, workforce development, and other specializations. They have expertise in these areas and are generally the legislator’s go-to resource for policy recommendations and related information. Legislative aides can be a great resource for information and for building rapport with your representative. When you first reach out to a representative’s office, you may be connected with the healthcare staff member. That person may also be able to help you set up a meeting with your representative.

Before your meeting, prepare a list of points you want to make. It can be easy to forget everything you want to say once you get there. Dress appropriately. It’s not a cocktail party or a beach party. You want to look clean and put together. Make sure that you are on time for the appointment. Typically, you will have about 15 minutes to introduce yourself, tell your story, and make your ask. You should be prepared to do most of the talking during the meeting. The critical thing is to connect your ask to your story. Tell the representative how a particular bill, funding, and/or other action you are seeking will impact you, your child, or other people with a rare disease.

You may want to put together a one page summary sheet about your rare disease. The one-page fact sheet should also include your “ask” (what action you would like them to take) and your contact information. You can attach pictures of you or your loved one with a rare disease to the sheet. Give the fact sheet to your representative at the end of the meeting. A fact sheet can make it easier for them to process your information (some people process information by what they hear, others process information by what they read). A typed summary also allows them to refer back to it for your key points and your ask after you leave.

If you take a photo with your representative and have permission to share it on social media, link it to the representative’s Twitter account and Facebook page. Raise awareness for your cause by including a comment about your meeting when you post the photo.

Whether you meet with the representative or a staff member, send a thank you note or email right away. It’s a good idea to send the thank you email in the evening or on weekends when lawmakers and their staff are less busy. When you send a thank you, be sure to include a summary of the meeting, your ask, and any other information you promised to share with them.

PRO TIP: MEETING WITH YOUR LEGISLATOR

If you haven’t met your representative before, experienced advocates have some advice:

• Go to a Town Hall and introduce yourself.
• It can be easier to get an appointment to meet your representative in their local district office than at the state or federal capitol.
• You typically need to make an appointment through the representative’s office.
• If other rare disease advocates will be joining you in the meeting, keep the number small, e.g., 5-6 people. The legislator often will have staff join the meeting, too.
• Expect the unexpected: Your meeting with your legislator may happen as your legislator is walking to another meeting! This is more likely to occur at a state or U.S. Capitol than at a local district office.
• Don’t be disappointed if you meet with legislative staff, not your representative. This is common. It’s important to develop relationships with staffers, too!
• Bring pictures of you and your loved ones.
• Consider bringing a fact sheet about your rare disease and “ask” (if applicable) include your contact information.
• Take a selfie with your legislator and post on social media; tag your representative.
• Write a thank you note or email for the visit even if you meet with a staffer.
• If you are asked a question you do not know the answer to, simply reply “That’s a good question – I’ll research that and get back to you as soon as possible.” Advocates aren’t expected to be legislative experts, and it’s always nice to have a reason to follow up!

Advocating at the State Level

Medicaid and insurance are managed primarily at the state level so rare disease advocates can’t overlook reaching out to state representatives. State governments are structured similarly to the federal government, with an executive branch (governor), legislative branch, and judiciary. The legislature develops and passes bills, which if signed by the governor become law. A department within the executive branch, often called the Department of Health, executes laws passed by the legislature and signed by the governor.

State Legislature Overview

Similarly to Congress, the legislative body for states has two chambers, or “houses.” Nebraska is the exception: its legislative body has only one chamber (is unicameral). The smaller chamber is usually called the Senate. The name of the larger chamber varies by state but is often called the House of Representatives or Assembly. In general, bills are introduced by a representative or senator and then sent to the appropriate committee for deliberation. The name of the committee may vary by state. Once the committee votes to approve the language in the bill, it is sent to the whole chamber for deliberation and voting. If approved, a similar process is repeated in the second chamber of the legislature. If the second chamber makes amendments to the bill, it returns to the first chamber for deliberation and voting. Once the bill has been approved by both chambers, it is sent to the governor to sign or veto (reject).

At each step of the process, advocates have opportunities to shape legislation by interacting with their representative through phone calls, emails, and visits. In fact, advocates can urge their state representatives to sponsor a bill to fund services, address a problem, or change a policy. This can be a long process so don’t expect one phone call or visit to result in your representative drafting a bill. It can take months (or years) of educating your elected official on the need for a proposed change. That’s why building a relationship with your representative is important.

As mentioned earlier, the names of the legislative chambers as well as the names of pertinent committees vary by state. The Internet may be the advocate’s best tool, as it can help you learn about the specific legislative process for your state, the committees which oversee areas of interest, and representatives who serve on those committees. Most state legislatures’ websites include a calendar of scheduled committee meetings, hearings, and voting days for specific bills.

Some state legislatures’ websites also allow you to search using general terms, such as “healthcare” to find pending or passed legislation so you can see if there are pending bills in your area of interest. (If your state legislature’s website does not accommodate general search terms, try Open State’s database.) You should note the number assigned to the bill, as well as its title. Bills use a combination of letters and numbers as identifiers. For instance, SB187 would refer to a state senate bill. When contacting your representative, you could urge them to vote Yes on SB187.

PRO TIP: HARNESS THE INTERNET

If you don’t know the website for your state’s legislature or departments in the executive branch, let the Internet find it for you. Using an Internet search engine, such as Google, type:

• Your State’s Name + Legislature
• Your State’s Name + Medicaid
• Your State’s Name + Drug Utilization Review Board
• Your State’s Name + Dept Health
• Your State’s Name + Dept Insurance

Medicaid

Medicaid, which is the largest health insurer in the United States, receives funding from both states and the federal government but is managed at the state level. In addition to providing health insurance to low income people, Medicaid provides benefits to some people with a rare disease.

Each state sets the amounts of copayments and deductibles for its Medicaid program. Thus, one state may cover a treatment for a rare disease, while the neighboring state may not. In addition, each state sets its own coverage criteria for reimbursement of treatments through a drug utilization review (DUR) board. DUR boards can require prior authorization for treatments, set stringent criteria for Medicaid reimbursement for a treatment, or require step therapy whereby a patient must “fail” one treatment before he or she is permitted to use another. All these factors can ease the burden of rare disease or create unexpected challenges depending on your state. Most states have a DUR board website that shows DUR board meeting dates. Many provide agendas or background materials for treatments that will be discussed. Some states also have a Pharmacy and Therapeutics (P&T) Committee that sets a Preferred Drug List (PDL).

Both state governments and the federal government contribute to a state’s Medicaid program so the amount of Medicaid funding in your state reflects how much the state contributes to the program. Expanding Medicaid eligibility and state funding is important to rare disease families, as Medicaid can help offset healthcare costs not covered by a private insurer, and because the impact a DUR board decision can have on the life, well-being, and healthcare costs of someone with a rare disease. NORD issues report cards on states’ Medicaid programs to help you identify in which areas you may want to direct your advocacy efforts.

For more information on Medicaid, please see the Global Genes toolkit, Understanding and Navigating Medicaid.

“Making sure that Medicaid is following through and actually covering the treatments that your child needs is really important. This is a big part of where advocacy comes in, because your child is not getting timely access to a treatment. Because of that, families often have to go make the case to Medicaid.” – MAX BRONSTEIN, PRINCIPAL, MGB CONSULTING, FOUNDER, JOURNAL OF SCI

Insurance

Insurance, such as auto, home, and health, is also regulated at the state level. In most states, the Department of Insurance serves to protect the consumer by making sure insurers are solvent (can pay claims) and investigating fraud. Some states provide education on health insurance exchanges or help with appeals for denied medical claims. In addition, a Department of Insurance may have to approve annual rate increases for insurance premiums.

State-Level Initiatives for Rare Disease Advocates

In addition to Medicaid and insurance, there are several areas at the state level which could benefit from rare disease advocacy:

Newborn Screening

The federal government has a Recommended Uniform Screening Panel (RUSP) for all newborns. RUSP currently includes 35 rare core conditions and 26 rare secondary conditions. However, states are not required to screen for all the conditions in the federal RUSP. EveryLife Foundation for Rare Diseases has an online map that shows what is included in states’ newborn screening panels. Contact your state representatives if your state doesn’t screen for all rare conditions included in RUSP.

Early Intervention Programs

States also have early intervention programs for children from infancy to the age of three who have mental or physical developmental delays. These programs are called different names and managed by different departments. The Centers for Disease Control and Prevention (CDC) provides contact information for each state’s program. Early intervention programs can provide additional support to rare disease families and are funded by state governments.

Rare Disease Advisory Councils

North Carolina was the first state to establish a Rare Disease Advisory Council (RDAC) in 2015 to improve the lives of people with rare disease. Sixteen states have established RDACs, which raise awareness for rare disease and can be instrumental in educating lawmakers and others in state government. RDACs are created by bills approved by the state legislature and government. That means that an RDAC receives funding by the state. The structure and location of the RDAC are specified in the legislation. Some states house the RDAC within their Department of Health, others have chosen a university for the RDAC location. An RDAC can bring expertise from a variety of stakeholders, such as legislators, researchers, healthcare providers, patients, and caregivers, among others.

Rare Disease State Legislative Caucuses

A legislative caucus is different from a legislative committee. Committees are established components of the legislative process to draft, deliberate, and amend bills. Committees may also hold hearings as part of legislative oversight responsibilities. A caucus is a group of legislators who share an interest, background, or political affiliation. EveryLife Foundation for Rare Diseases encourages advocates to collaborate with their state lawmakers to create a Rare Disease Legislative Caucus. A caucus can raise awareness by hosting briefing meetings on rare disease and helping garner support for legislative efforts. Caucuses are usually cochaired by one member from each of the major political parties (Democrat and Republican). If your state doesn’t have a Rare Disease Legislative Caucus, contact your representative to generate interest.

Advocating at the Federal Level

The Orphan Drug Act of 1983 is an example of the potential change that rare disease advocates can promote at the federal level. This legislation put incentives in place to motivate the pharmaceutical industry to focus their efforts on therapies for smaller disease populations. Prior to the passage of the act, there were only 38 approved orphan drugs. Now there are more than 600 approved orphan drugs and more are in development. Advocates also played a key role in passage of the Patient Protection and Affordable Care Act (Affordable Care Act), enacted in 2010. The Affordable Care Act expanded access to health insurance for millions of Americans, including those with rare disease. It expanded eligibility for Medicaid, banned insurers’ practice of not covering pre-existing conditions, and extended coverage for children to the age of 26.

Federal Legislative Overview: Congress

Congress has two chambers: The House of Representatives and the Senate. Bills can be introduced on either the floor of the House of Representatives or the Senate, after which the bill is sent to the appropriate committee for deliberation and review. Most issues relevant to the rare disease community are under the jurisdiction of the House Energy and Commerce Committee or the Senate Health, Education, Labor, and Pensions Committee. However, some committees have subcommittees which oversee health issues. For instance, the House Appropriations Committee, Veterans Affairs Committee, and Ways and Means Committee have health subcommittees.

The committee or subcommittee may hold hearings, discuss provisions, or add amendments to the bill. The committee can vote to approve the bill for consideration by the full House of Representatives or Senate or defer passing the bill forward. If the committee decides to forward the bill to the full chamber for deliberation, staff members prepare a report that details the bill’s intent, scope, impact of existing laws and programs, position of the executive branch, and views of dissenting members of the chamber. The bill is then placed on the schedule for deliberation and voting by the full chamber.

Following passage by the House or Senate, the bill is passed to the other chamber, where it follows the same route from committee to floor action. The bill may be received, rejected, ignored, or changed. If significant changes or alterations to the bill are introduced by the other chamber, a conference committee is formed to reconcile the differences. If it is unable to reach a compromise, the legislation dies. If they agree, a conference report is prepared with the recommended changes. Both the House and Senate must approve the conference report for the bill to move forward.

After approval by the House and Senate, a bill is sent to the president to sign or veto. If the president approves the legislation, the bill becomes a law. If the president does not take action, the bill can become a law without the president’s signature after 10 days depending on whether Congress is in session or has adjourned. If Congress has adjourned its session during the 10-day period, the bill will not become a law. This is called a pocket veto. The president can also veto the bill. Congress can override a veto if at least two-thirds of the House and Senate vote to do so.

Rare Disease Congressional Caucus

Encourage your representative and senators to join the Rare Disease Congressional Caucus if they are not already members. The caucus allows MOCs to share constituent experiences with rare disease and collaborate on legislation that addresses rare disease challenges. In addition, it is a forum for MOC to receive briefings on rare diseases, diagnostics, and treatments.

Congress and Key Federal Agencies

Congress decides the size of the federal budget and where funds will be directed. Congress also provides oversight of the executive branch, which is important for rare disease families. Through legislation, Congress can affect how key federal agencies operate. This creates many opportunities for advocates to interact with their representatives.

National Institutes of Health (NIH)

NIH is a federal agency within the Department of Health and Human Services (HHS) that oversees scientific research to improve the health and lives of Americans. It promotes research in the causes and treatment of diseases. There are 27 institutes, centers, and offices that operate within the NIH, including the National Human Genome Research Institute, National Center for Advancing Translational Sciences, and NIH Clinical Center, all of which are involved in rare disease. The NIH also sponsors a Rare Disease Day in February of each year to put the spotlight on rare disease, enable people to share their stories, and foster collaboration among stakeholders.

Food and Drug Administration (FDA)

The FDA reviews new drug applications to ensure that a new treatment is safe and effective for use in people. The Orphan Drug Act of 1983 accelerated the regulatory review process for therapies to address rare diseases. However, a majority of rare diseases do not have an FDA-approved treatment. The Office of Orphan Products Development (OOPD) at the FDA reviews applications for orphan drug status and offers grants for the development of drugs and medical devices treating rare diseases. The OOPD has a Rare Pediatric Disease Priority Review Voucher Program that assesses whether a treatment can receive expedited review and is eligible for a voucher that it can use later or sell. This program is scheduled to expire on October 1, 2026.

Centers for Medicare and Medicaid Services (CMS)

Each state has its own Medicaid program where the federal government matches each state’s financial contribution. CMS is the federal agency within HHS that provides oversight to state Medicaid programs. It offers guidance to states on implementing Medicaid programs, approves states’ amendments to program amendments and reimbursement rates, and is involved in Medicaid waiver negotiations, among other areas. Centers for Disease Control and Prevention (CDC) The CDC, another agency within HHS, is known for its role in managing responses to public health crises, such as infectious disease outbreaks. However, the CDC also collects data on different diseases and provides information and fact sheets to the public. In addition, the CDC is building a knowledge base of the impact of genomics on public health through its Public Health Genomics Knowledge Base program and recently added rare diseases to this database.

PRO TIP: JOIN OTHER ADVOCATES

EveryLife Foundation for Rare Disease hosts a Rare Disease Week on Capitol Hill each year. It’s an opportunity to meet other advocates and patient organizations, learn about important issues in rare disease, and jointly meet with lawmakers. Advocates may also gather together to encourage representatives to hold a Congressional briefing on a specific rare disease or a rare disease challenge. Reach out to your representative to request they sponsor a Congressional briefing. You can work with your representative’s office and advocacy groups to determine the agenda and invite speakers to educate lawmakers on what needs to be done to improve the health and lives of people with rare disease. Make sure you invite Representatives and Senators in the Rare Disease Congressional Caucus who serve on relevant Congressional committees!

Generating Awareness for Your Cause

Educating people on rare disease is important because you can call on people to reach out to their representatives when an important piece of legislation is being considered. Lawmakers pay attention to the number of calls or emails they receive on bills and regulations. Thus, generating awareness for your cause amplifies your voice with legislators.

Think Nationally, Act Locally

If your rare disease affects a relatively large number of people, there may be a national advocacy organization already in place. Search the Internet to see if there is a foundation or nonprofit that is focused on your rare disease. You might want to start a local or regional chapter or support group for others with your rare disease.

Educate Your Family, Friends, and Neighbors

Many people do not know about rare diseases or do not follow healthcare policy at the state or federal level. Start small with something that’s manageable. Maybe it’s your story that you tell to family, friends, and people at church. You can create an email list for forwarding information on the rare disease and your story. Post updates on your Facebook page. You can grow your reach to a broader audience as you become comfortable with sharing your story and rare disease news.

Advocates are using a variety of methods to reach a broader audience. You could create a group on social media to post news and events. Some advocates post on Twitter. Others post videos on YouTube and Facebook. Others have created blogs or self-published e-books about their experience. Let your interests and comfort with the written word (or camera) guide you on the best medium for sharing your story and expanding your advocacy.

“Never stress yourself out to the point where it is no longer enjoyable. Take it one step at a time. Remember that you can’t plan everything.” – TRINA MASSEY-DAVIS, SOUTHEASTERN SARCOIDOSIS SUPPORT GROUP

Rally Your Troops for Action!

Once you have established a way to update people on rare diseases (email, social media, etc.), use that platform to rally the group to make calls and send emails to lawmakers when a bill is being deliberated in state government or Congress. Give your troops a form letter or sample script to make it easy for them to advocate on your behalf.

In addition, you can start a petition on Change.org to advocate for action. When you select the Health button, Change.org will notify people signed up for health alerts about your petition. Write an eye-catching title, and in the body of the petition, explain to people why an action needs to be taken. Resistbot is another online platform that allows you to create petitions and send emails to representatives. If you have a sample form letter, you can post the letter to your social media accounts and recommend people use Resistbot to send the letter to their representatives.

Host an Awareness Event

You can host an Awareness Day or event for your rare disease. If there is a larger organization focused on your rare disease, you should check to see if they have national initiatives to which you can tie a local event. You can also host an event separate from a larger advocacy organization. Events include block parties, pancake breakfasts, potluck dinners, movie nights, and 5K walk-runs, among others.

PRO TIP: HOSTING A LOCAL EVENT

Trina Massey-Davis joined others in her local Sarcoidosis Support Group to host a 5K walk-run in Savannah, Georgia. Trina offers the following the advice for people interested in hosting a local event:

• Pick a date for your event
• Start preparing for the event 3-6 months before the event
• Check with your city or county to see if you need special permits for the event
• Invite local officials, physicians, and advocacy groups to Save the Date
• Create Save the Date posts on social media
• Hand out flyers at local businesses
• Let local newspapers know about the event
• Prepare opening remarks to welcome people to the event and thank them for supporting your rare disease
• Learn from your experience and decide what you want to improve for the next event

You could also host a booth at a local fair or other community events. Some advocates do presentations at community organizations and clubs, such as the Rotary Club, Lion’s Club, Elks Lodge, Moose Lodge, etc.

Join an Advocacy Network

Just as your personal efforts to grow awareness for your rare disease can amplify your voice, you can join others to amplify their voices. Several patient groups have advocacy networks that you can join. In addition to helping other rare disease advocates, you can learn about proposed state and federal legislation that affects you. Some of the advocacy networks will also provide you with the opportunity to share your own story so you can raise awareness among the broader rare disease community about your condition.

EveryLife Foundation for Rare Diseases and NORD have well-established advocacy networks. EveryLife Foundation for Rare Diseases has created the Rare Disease Legislative Advocates program, which offers action alerts on legislation and provides scorecards on how lawmakers have voted on bills that affect people with rare disease. The NORD Rare Action Network connects rare disease advocates on health policy matters. The Rare Action Network provides action statements on state and federal issues as well as action alerts that specify specific, timely steps you can take to support the rare disease community. In terms of sharing your story with other advocates, Rare Disease Legislative Advocates hosts monthly webinars in which people share their advocacy stories. NORD invites people to write about their rare disease and share their stories online. You can also post about your experience on Patients Rising Stories, which is focused on lifethreatening and chronic diseases.

Health Policy and Regulations

Several issues are perennial topics for rare disease advocates: funding research, expanding newborn screening, supporting clinical trials, expediting diagnosis, improving access to affordable healthcare, improving access to treatments, making treatments affordable, and reducing the financial burden of rare disease.

You may advocate for legislation in one or several of these areas. If a bill is not passed by Congress during its two-year term, the bill will need to be re-introduced in the current Congress. The same principle applies to state legislatures. That is why people with rare disease say that advocacy is a marathon, not a sprint. It takes time to pass legislation, and you may have to re-advocate for legislation with a new Congress.

In addition, health policy and regulations are always evolving, as advocates bring rare disease challenges to the attention of their lawmakers. With cell and gene therapy being developed for rare diseases, you might want to learn about barriers for development and approval for these treatments. You might want to understand how clinical trials for rare disease are designed or how patient registries work. If a treatment has recently received FDA approval, you may want to prepare for a Drug Utilization Review board. You may be motivated to learn about fair drug pricing and health economics. Check out global genes resource hub for more resources on these topics.

Even people whose job is to stay abreast of healthcare news and trends can find it challenging to be up to date on all areas. Choose what interests you or matters to your family. If you’re not certain where you’d like to focus your efforts, signing up with an advocacy network gives you exposure to the current “hot topics.” You can also find position statements and webinars that provide education on various topics within rare disease. Some organizations, such as NORD and Patients Rising, also have podcasts with interviews of experts in different fields or spotlights on a specific rare disease or challenge.

PRO TIP: GETTING YOUR ADVANCED DEGREE IN ADVOCACY

The more you know about health policy and the assessment of treatments’ safety, effectiveness, and costs, the more it can help make you an effective advocate. In fact, you could become the expert that your representatives rely on to get updates on treatments or the trusted voice they listen to when considering the impact of proposed legislation. Patients Rising Now, the advocacy arm of Patients Rising, offers a Master Class on healthcare advocacy. In addition to teaching people “need to know” about health policy, the instructors explain Drug Utilization Review Boards, health economics and the Institute for Clinical and Economic Review (ICER) and FDA Advisory Panels.

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