Archived Events

2023 RARE Advocacy Summit

Each year, Global Genes convenes one of the world’s largest gatherings of rare disease patients, caregivers, advocates, healthcare professionals, researchers, partners, and allies at the RARE Advocacy Summit.

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Connect. Inspire. Learn

The 2023 Summit provided attendees with insights about the latest in rare disease innovations, best practices for advocating on an individual and organizational level, and actionable strategies they could take home and implement immediately to accelerate change. In 2023, the Summit was held in San Diego California where educational sessions and networking opportunities allowed attendees to connect, learn and inspire. Educational sessions were also live-streamed online.

Keep scrolling to watch videos of the sessions.

Watch Keynotes from the 2023 RARE Advocacy Summit

We the Scientists: How Patients Are Driving Research

Hear how a daring team of doctors and parents forged a new path for medicine. Amy Dockser Marcus followed patients, families, caregivers, and patient advocates throughout the process, and wrote wrote a moving narrative of these patient advocates who are revolutionizing the way medical research is conducted.

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Rebel Health: How Rare Disease Communities Lead the Revolution

Susannah Fox, a health and technology strategis, shares how the rare disease community led the way for patient-driven innovations in healthcare, also the topic of her book, Rebel Health: A Field Guide to the Patient-Led Revolution in Medical Care.

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Empowering the Rare Individual Track
at the 2023 RARE Advocacy Summit

Coordinating Complex Care

Learn to navigate the challenges of RARE care, including the essential role of finding a physician who can serve as quarterback, how to improve communication, work across institutions, navigate insurance and sort through the pros and cons of centers of excellence.

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I’m an Expert, Too!

While people with rare diseases may be experts on their condition, doctors may not always recognize this fact. Learn how to talk to doctors, advocate as an expert in your disease, and what to do when health care providers fail to speak in understandable ways.

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Coping with the Challenge of Rare Disease – With or Without a Diagnosis

This panel of rare patients and mental health professionals share practical strategies for managing symptoms, coordinating care across many specialties, prioritizing mental health, and navigating the diagnostic journey.

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Managing Life Without a Diagnosis

This session addresses challenges, questions, and opportunities for those living without a specific diagnosis. Topics include genetic testing basics, research studies, support services, finding community, and developing strategies to empower yourself or a family member in navigating life without a diagnosis.

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Grieving Diagnosis, Lack of Diagnosis and Loss

When the life you’ve anticipated is forever changed by rare disease, there is often an overwhelming sense of loss. This session will discuss managing many forms of grief, learning to live well in your new reality and how to find organizations and resources that can help you cope.

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Activating Advocacy for Kids, Teens and Youth

This panel is for young advocates or those looking to inspire young advocates looking to be more involved in rare disease advocacy. This discussion centers on the value of engaging young people and offers tips on how to host a Hill Day or instill programming for a younger demographic.

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Legislative Action Track
at the 2023 RARE Advocacy Summit

Emerging Rare Policy Issues

The EveryLife Federal & State Policy Team provided an overview on current policy issues that impact the rare disease community.

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The Cost of Delayed Diagnosis:

This panel takes a look at how delayed diagnosis affects healthcare costs in the United States, including a high-level overview of the data and provides the groundwork for what is coming next in the Cost of Delayed Diagnosis in Rare Disease: A Health Economics Study.

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Community and Capacity Building Track
at the 2023 RARE Advocacy Summit

Beginner’s Guide to Community Activation

Understand how to engage your community, welcome the newly diagnosed, bond through local
and online meetups, communicate effectively, and activate people to participate in research and
data collection.

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Why Genetic and Genomic Testing is Key to Increased Diagnosis

Learn about current genomic
testing in clinical and research settings, find out about free programs offered by biopharma
companies that help identify patients, and legislative efforts to add genetic testing to newborn screening panels.

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Developing Sustainable Funding Models for Your Organization

There’s no one-size-fits all solution for finding the funds that will allow your advocacy organization to survive and grow. We’ll consider various models of success including crowdsourcing, events, donor relations and grants.

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Develop a Long-Term Strategy

Gather insights into strategic planning, recruiting active leaders and tracking success. We’ll also
consider how to find and build relationships with other rare disease organizations and assemble
health and medical review boards

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Expanding Your Global Reach

To find larger patient populations and be more inclusive, it is necessary to look beyond your home country. This session will focus on how to increase your reach and navigate international research, regulatory agencies, and healthcare systems.

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Working Nicely in the Same Sandbox – How Patient Groups Can Work Together, Not Against Each Other

Three organization leaders explain how how foundations can work together and what you can do as a leader to produce this cohesive relationship in your community.

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Supporting Your Rare Community’s Mental Health

This panel of mental health professionals, industry partners and rare parents will discuss mental health challenges faced by rare families and highlight support programs provided by rare disease advocacy organizations to meet the needs of their communities.

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Financial Support for Your Community Members

Rare disease has a big impact on your financial well-being. This session will highlight resources available to help patients and caregivers with healthcare costs, disability, and tax-advantaged savings accounts for individuals with disabilities known as ABLE accounts.

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Becoming a Research Ready Organization Track
at the 2023 RARE Advocacy Summit

Create Your Strategy: The Rare Research Roadmap

Learn how organizations can create a research strategy and build a toolbox of what researchers will need to progress research, and discover and advance potential therapies

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Developing Scientific Expertise to Drive Research Strategy

Learn how to find and engage scientific advisors and expert researchers, build a scientific advisory board, and recruit a full-time or shared scientific director.

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Using Data Collection to Improve Outcomes

Find out how a well-designed data collection program can help improve clinical care, create disease
progression models, identify mechanistic changes and target therapies and windows of efficacy.

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Becoming Clinical Trial Ready

Make sure you have essential elements in place to allow experimental therapies to advance to
clinical trials. We’ll discuss conducting natural history studies, identifying and validating biomarkers,
determining appropriate endpoints, disease concept models and regulatory strategies.

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Pre-Clinical Research: Why & When to Invest

One of the best ways for patient advocacy organizations to lay a foundation for therapy development is to invest in in-vivo and in-vitro models and tools. This session will explore essential tools for basic research including animal models, induced pluripotent stem cells, and next generation sequencing.

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Crafting a Data Sharing Strategy

Gathering data is not enough. To leverage that work requires tools and technology to share it safely and privately, as well as enable its use with other data sets. This session will discuss data sharing and data federation, how to evaluate options, and the critical responsibilities around data governance.

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Power of Partnering: Children’s Hospital Colorado

Learn about collaborative research models and case studies involving Children’s Hospital Colorado,
including its Neurogenetics Clinic in partnership with STXBP1 Foundation, LC6A1 Connect, Ring14 USA and Project 8p.

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Power of Partnering: Rosamund Stone Zander Translational Research Neuroscience Center

Learn about collaborative research models and case studies involving advocacy groups and Rosamund Stone Zander Translational Research Neuroscience Center at Boston Children’s Hospital.

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RARE Disease Master Class: Science & Tech Innovation Track
at the 2023 RARE Advocacy Summit

Thinking Like a Researcher: Disease Biology 101

This session explains how genes work and what can go wrong – and how a growing toolkit of therapeutic approaches is enabling precision approaches to address the underlying molecular causes of diseases.

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Small Molecules, Drug Repurposing and Biologics

This session will explore how small molecule therapies work, opportunities to repurpose existing approved therapies, and how monoclonal antibodies and enzyme replacement therapies work.

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RNA Therapies: ASOs, mRNAs, siRNAs

RNA therapies can either disrupt the translation of a faulty gene into a pathogenic protein or provide instructions for producing a needed missing protein. This session explores how these therapies work, why delivery is a critical issue, and how to overcome challenges.

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Gene Replacement and Gene Editing Therapies

Gene replacement and gene editing therapies have the potential to provide a one-and-done cure
for genetic diseases. We’ll explore how these therapies work, when these may be appropriate, and barriers that need to be overcome.

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Spinal Muscular Atrophy Case Study

Spinal muscular atrophy (SMA) has benefitted from therapeutic innovations including an antisense
therapy, a gene therapy, and a small molecule therapy. This case study will explore different
therapeutic strategies and their pros and cons in the real world

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How Companies Decide Which Therapies to Pursue

Why do some therapies advance to clinical trials and others are discontinued? What attracts a
company to pursue research on a particular project? Find out the decision factors that companies
use to answer these questions.

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7 Ways to Engage the FDA

Communicating with regulatory authorities can help avoid missteps in the research process that are costly in terms of both time and money. Learn when and how to bring the patient voice into the discussion.

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Looking Back at the 2023 RARE Advocacy Summit

2023 Recap

Check out some stats from our event this year.

See more stats from the 2023 RARE Advocacy Summit