Global Genes Guide to Starting a Nonprofit Patient Advocacy Organization

This Global Genes Quick Guide is a resource for advocates focused on pressing topics causing challenges in their lives and communities. 

Launching a nonprofit patient advocacy organization is both a bold step and a deeply meaningful commitment. For many founders, it begins with a personal journey—an urgent need to create change for patients, families, and communities impacted by rare disease. While the passion and vision are essential, transforming that passion into a sustainable, impactful organization requires planning, clarity, and careful execution.

This Quick Guide is designed to help you take the first steps with confidence. It outlines foundational considerations to ensure your organization is not only launched but also positioned to thrive, grow, and make a lasting difference.

Before You Begin: Key Questions to Clarify Your Vision

• Define your focus: Identify the specific rare disease(s) or community your organization will serve.
• Craft your mission: Write a concise, compelling mission statement that communicates your purpose and goals.
• Know your audience: Determine who you aim to serve—patients, families, caregivers, researchers, or all of the above.
• Choose your priorities: Decide if your efforts will center on patient support, research funding, awareness and education, policy and legislative advocacy, or a combination of these.
  

“Before launching a new patient advocacy group, ask yourself—are you filling a gap or duplicating a mission? Sometimes, the most powerful move isn’t starting something new, but strengthening what already exists.”   

– Cheryl Petruk, Patient Advocate & Founder, Heal Canada

Begin by “making a list of all the components and executing them one by one until before you know it, the whole system is there, up and running. The biggest inspiration is all around you. When starting a PAG I recommend connecting with existing ones and leaning on the very welcoming and supportive community “
– Zoe English Manville, Rare mom; singer of Portugal The Man, founder of Cure DHDDS USA

Step 1. Clarify Your Vision and Purpose

• Define your focus: Identify the rare disease(s) or community your organization will serve.
• Craft your mission: Write a concise, compelling mission statement that communicates purpose and goals.
• Know your audience: Decide whether you will serve patients, caregivers, families, clinicians, or researchers.
• Set priorities: Choose your focus areas—patient support, education, research funding, awareness, policy advocacy, or a combination.

“The first thing I’d suggest is to sketch out a draft mission statement of exactly what you want to do. What specific work do you want to offer to your disease space. And then look around. Are there existing organizations within your disease space or a complimentary one that maybe doing similar efforts? Can the work you want to get done…fit within their mission/ organization? Instead of starting a new organization…can you first sit with those leaders and see if collaborative work is possible?  With the best of intentions, many launch an organization without realizing it is running an actual business. Having strategic, collaborative conversations may get the work done that is needed, without the overhead.”

 – Amy Brin, Amy Brin Consulting, LLC

After you have identified a need for your rare disease and clarified which aspects you will focus on, you may decide that starting a nonprofit can be the way to address this need. Before you do, this 16 page toolkit, So You Think You Want to Start a Nonprofit?, from Global Genes prompts you to consider:

• Is the need being met by an existing foundation? Could you instead join their efforts?

 “You need to start with clarifying your purpose and defining your mission. As a small organization, you can’t do everything. So even if you have multiple goals in your mission, you need to know what your top priority is.  Is your goal to support patients, build community, raise money for a cause, develop or accelerate therapies, connect people with care or support services?

For example, when we started the STXBP1 Foundation, we knew that Fund and Drive research to accelerate discovery of a cure was the highest priority and everything else was supportive of that priority. “

– Charlene Son Rigby, CEO of Global Genes, Co-founder of STXBP1

Step 2. Establish Legal and Organizational Foundations

• Choose a legal structure: In the U.S., most advocacy organizations register as 501(c)(3) nonprofits. Explore what structure makes sense for your country or region.
• File governing documents: Draft bylaws, articles of incorporation, and register with your state or national authority.
• Build a board of directors: Recruit diverse, committed individuals who bring expertise in governance, fundraising, and advocacy.
• Establish accountability: Create clear policies for transparency, financial oversight, and compliance.

When recruiting board members, it is important to gather varied talents, expertise and backgrounds. Ask yourself if collectively your new board will include skills such as Legal, Marketing, Finance, Public Relations, and Philanthropy. Each board member brings unique abilities and perspectives. A nonprofit’s Board of Directors meets as often as once a month or as little as once every quarter.

“As a new nonprofit, if you have developed a ‘working board’ (meaning one that doesn’t just advise but also helps with tactical execution), it should meet once a month by phone for 1 hour. You can develop board work groups around focus areas that can also meet in between the meetings.  Often this could be Finance, Board Development, Fundraising, and other program specific work. During the board meetings members are asked to share their progress, garner feedback and determine any action items or next steps. Another thought to consider is hosting an annual in-person ‘board retreat’ to bring all of the board members together for a half or full day,, this is especially productive when there is an opportunity to work on strategy for the org.  Most seasoned board members know how to run these, and your board/chairs can help develop and lead these important sessions “ 
— Nicole Boice, Chief Mission Officer & Founder, Global Genes

Step 3. Build Your Team and Network

• Start small but strategic: Volunteers, part-time staff, or advisors can help you get off the ground.
• Engage patients and families: Center the voices of those living with the disease in everything you do.
• Connect with partners: Collaborate with clinicians, researchers, other nonprofits, and umbrella groups like Global Genes.
• Form an advisory council: Bring in subject matter experts to guide your efforts.
  

“Make sure your PAG has a clear mission and vision, so everyone knows what you’re working towards. Choose trustees or core team members who have time and energy to support the group – there’s a lot to do as it grows.Make sure you are connected both with the families affected by the condition and the scientists studying it.  You can also find researchers working on your condition by reading the relevant scientific papers online. In the UK, you can sign up with Beacon, which offers guidance on setting up a rare disease charity and can match you with a mentor.”
– Mel Dixon, Rare mom, founder of Cure DHDDS

Step 4. Create a Sustainable Funding Model

• Develop a fundraising plan: Mix individual donations, grants, events, corporate sponsorships, and online campaigns.
• Communicate impact: Share real stories and data to demonstrate why funding your work matters.
• Start small, grow steadily: Focus on achievable goals in your first year while building credibility for larger opportunities.
• Consider fiscal sponsorship: Partner with an established nonprofit to access funding sooner while you set up your own infrastructure.

“One (fundraising tactic) that I think is really simple and anyone can do it, is just have some sort of physical shirt or a car magnet or a yard sign or something like that. For me, I created a very simple sign that had a QR code that goes to our GoFundMe. And I get donations all the time because of them.”
– Brittany Markham, Rare mom, ASMD, “Please Save Damian” Campaign

In this clip from the film, Rare Humans: Turning Hope into Action, Jennifer Sills breaks down why and how you need to start a patient advocacy group if one doesn’t already exist to address your rare disease. Jennifer is mom to Jules who has Okur-Chung, and is the President and Founder CSNK2A1 Foundation. 

Step 5. Deliver Programs and Measure Impact

• Start with pilot projects: Begin with manageable initiatives (support groups, webinars, small grants, or awareness campaigns).
• Track outcomes: Collect both quantitative and qualitative data (number of families served, testimonials, policy wins).
• Adapt and improve: Listen to your community and refine based on feedback.
• Celebrate early wins: Recognition builds momentum, credibility, and motivation.

“All around you, there are people who’ve done this before–they started their own patient advocacy group from scratch. Talk to them. Get on calls with them, take notes, copy their homework, and do what they did. They’re years ahead of you and if they’re going to be generous with their time to show you the way, don’t feel the least bit bad about copying them. That’s the whole point of these connections.”
– Bo Bigelow , founder of the Foundation for Hao-Fountain Syndrome, co-founder The Disorder Channel. 

Step 6. Grow for Long-Term Sustainability

• Invest in leadership: Provide training for staff and advocates in governance, fundraising, and research readiness.
• Diversify funding streams: Reduce reliance on a single donor or grant source.
• Engage in coalition building: Join alliances, networks, and collaborations to amplify impact.
• Plan for the future: Create a strategic plan with 3–5 year goals, aligned with your mission and capacity.

“Starting a PAG is a lot of work! I was warned about this but took it on regardless – but it is a lot of work. Be warned.

That said, if it is something you are passionate about, then you’ve got to do what you’ve got to do! I don’t regret it for a minute, and every day I learn something new. A lot of days we make noticeable progress – which certainly helps with the motivation to keep going!

My biggest piece of advice for anyone considering doing this is to get some really great and supportive folk around you.”
– Jonathan Bracey,  rare disease dad, Founder of MVA Society

Final Thought

Starting a nonprofit patient advocacy organization is a journey that blends heart and strategy. Your vision, coupled with sound planning, can transform the lives of patients and families, accelerate research, and shift what’s possible in rare disease. Global Genes is here as a partner—providing resources, knowledge, and community to help you succeed.

Resources

Starting a Nonprofit: The First Step This 16 page toolkit from Global Gene covers:

• Where to Begin? What Paperwork?
• Fulfilling State and Federal Requirements
• Building a Board of Directors
• Assembling a Staff
• And more

Global Genes Toolkit: Building a Foundation This toolkit covers: 

• Self-check: What Kind of Leader are you?
• Starting a Nonprofit  
• State Laws  
• Mission Statement 
• Getting IRS 501(c)(3) Status  
• Strategic Planning  
• Building a Board  
• Adding Volunteers and Staff

How to Run a Support Group for a Rare Disease

RARE Foundation Tactics: Foundations of Organizational Planning for Nonprofits

This panel discussion from the Global Genes 2021 RARE Patient Advocacy Summit covers governance and core operational components of running a patient advocacy organization, and offers  a checklist of key people, resources, and infrastructure you’ll need.

Keynote: When Leadership is Thrust Upon You
This presentation from the RARE Advocacy Exchange in March 2025, discusses the transition from seeing an unmet need for your rare disease and growing into the leader and the foundation founder that your community needs. 

Global Advocacy Alliance Webinar: Board of Directors 101 – tips for recruiting and managing your board members.

Tip Sheet: Starting a State Based Organization – From Everylife Foundation 

Advice on Starting Nonprofit Groups for Rare and Other Diseases 

A checklist of the vital components for your bylaws. – From Blue Avocado

Foundations Of Rare: Syngap1 – a short film from The Disorder Channel
What if there’s no organization for your rare disease? Here’s how you start your own.

Filing Paperwork

Instructions for filling out the IRS Publication 557, Tax-Exempt Status for Your Organization. 

On the website http://usa.gov  search for “State Filings for Nonprofits” to learn each individual state’s specific requirements for securing nonprofit status.

Set up your website

Creating an Amazing Rare Disease Nonprofit Website

How to Build a Website That Advocates for Rare Diseases

Fundraising

Global Genes Quick Guide to Fundraising
Guide to Creating a Comprehensive Fundraising Strategic Document
Innovative Fundraising RARE Advocacy Exchange 2025 Session 
Funding Sources You May Not Have Heard Of – Global Genes video
Fundraising Do’s And Don’ts –  Global Genes video
Navigating Resources & Grants – Confessions of a Rare Disease Mama Podcast

Podcasts

The 12 Commandments for starting a rare disease patient advocacy group. – Once Upon a Gene podcast
Unraveling Rare Ep12: Forming a rare disease foundation – This short video podcast includes tips for your first steps from two rare PAG founders.

Non-Profit Marketing Strategies for Rare Diseases – Podcast
Trena Myers, CEO of Rare Wish, shares her experience building a successful organization for rare diseases.

NORD webinar: Starting a Not-for-Profit Organization

Global Genes 2025 Quick Guide Series is our way to respond to a common need we have heard for more information in the rare community. Think of them as the answer to one frequently asked question or pain point we have heard from our Rare Concierge, our online and in person communities and our Global Advocacy Alliance.  Each one is complete with links to additional related resources across various media types, both those that are authored by Global Genes and from outside sources as well.

This series is supported in part by: Amgen, Amicus, Beam Therapeutics, Biocryst,  BridgeBio, Chiesi, Mallinckrodt Pharmaceuticals, Merck, and Parexel