Alagille Syndrome
Industry News

Action in Advocacy: A 3 Minute Guide

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Written by Roberta Smith, Alagille Syndrome Alliance President


Part of the Alagille Syndrome Alliance Advocacy Education Series

 Understanding advocacy and the importance of that role in the lives of those living with a rare disease can help you define what types of efforts may suit you, your lifestyle, and your capacity. Advocacy can include sharing your story, fundraising, raising awareness, supporting science, donating to the cause, hosting or attending meaningful events, connecting with others in the community, pushing for legislation and policy changes, and volunteering your time. Rare Disease advocacy led by patient groups, communities, individuals, and families has led to remarkable outcomes like new treatments and therapies, new laws and policies that make living with a rare disease easier, improved medical care, advanced science and research, and much more. Individuals, families, and caretakers have an extraordinary perspective that pharma, biotech, medical professionals, lawmakers, and scientists and researchers are hungry for. This is the patient voice.

As we advance toward Rare Disease Day, February 28th, I’m eager to encourage rare disease families to advocate on some scale. I will explain why every effort must be thoughtfully focused, with a specific call to action that leads the receiver of that advocacy to a tangible place where more information, education, and a way to participate is easily accessible. Advocating makes a much more impactful punch when it is focused and structured. Don’t worry, this doesn’t have to be time-consuming. With a little guidance, you can engage in advocacy with confidence.

Here are 3 specific actions rare disease families can take to advocate on behalf of themselves or others living or dealing with a rare disease to make progress toward addressing challenges, raising awareness, funding science and research, and educating the world around them.

Tell Your Story
Telling your story is the most powerful tool in your advocacy toolbox. If you are interested in sharing your story, consider drafting a long version and pocket version. Consider also identifying your WHY, refining your elevator pitch, and staying laser-focused on your goals. Always lead the reader/listener to the central hub of information and advocacy for your disease state whether it be a patient advocacy nonprofit, a hospital system, or science research center. Tell your story in several ways throughout the year, including across your personal social media network, as a highlight in partnership with your rare disease organization, with the local paper or news team, or at local, state, or national events where appropriate. Before telling your story, understand your goals which will serve as a guide for the content, length, audience, call to action, and the message you send. Always end with a call to action, be specific, and connect your audience with that central hub.

Engage with Local, State, and Federal Elected Government Officials
Elected officials have the power to address health disparities and needed changes to the healthcare system. They support policies that can directly affect rare disease families, caregivers, and providers and can change long-term outcomes. Get started by understanding the challenges your rare disease community faces. This might include the diagnostic odyssey, lack of available treatments, heavy burden of disease, little to no current science or science funding available, and healthcare policies that make dealing with a rare disease day to day more difficult. These officials need to hear from you. If you need help finding your elected representative, here is a great place to start – ​​https://everylifefoundation.org/rare-advocates.

Educate and Raise Awareness
Pair up with your disease-specific rare disease advocacy organization (PAG – Patient Advocacy Group) to understand what effort is already being made to educate, to whom and how. Raising awareness and educating go hand-in-hand. You want to work smarter, not harder so utilize already created tools and resources. When educating, provide a medical overview of the disease and your personal story. Your disease PAG can help provide you with resources to share. Education needs are vast including medical students, physicians, pharma and biotech, disease-related corporations and businesses, community in-person and online, and students of all ages.

Remember these guiding principles with every advocacy effort:

  • RESEARCH what is currently being done, has already been done, what has worked and why, and what resources and tools are available. Re-inventing the wheel can lead to wasted time or unintentionally stepping on current efforts. Start with your PAG.
  • UNDERSTAND your “WHY” – Why are you advocating? Why are you motivated and determined? Why should people care? Why is gaining help and support for the cause so important?
  • ASSESS your audience and IDENTIFY the goal of your message
  • Always LEAD the receiver to a tangible place to gain more knowledge or information – the advocacy organization, hospital system, or research center. Telling your story with the local news station is great, but if you don’t tell them who is leading the charge to support science, disease education, family programming, and building community then the effort can fall flat.
  • Clearly state your CALL TO ACTION: Do you want them to donate to the cause, join the effort, volunteer, spread the word, make connections?

Be specific, focused, and educated because “if not us, then who?”