We are excited to announce the launch of a new series of Quick Guides for the rare disease community. Over the course of the year, this initiative will address some of the most pressing challenges faced by those affected by rare disease to
- Empower individuals affected by rare or undiagnosed diseases with knowledge and actionable next steps to help navigate the complexities of a rare disease diagnosis, treatment options, and emotional wellbeing
- Equip patient advocacy organizations with critical capacity building resources to better serve their communities
Though there are more than 10,000 different rare diseases, many of the overarching needs of the community are the same. Based on feedback from our 800+ member Global Advocacy Alliance, frequently-asked questions to RARE Concierge, and community partners, we’re simplifying the search for actionable information to address these critical topics. These Guides include important insights, and best practices from the community – Global Genes, other leading organizations, subject matter experts and community members – in a variety of formats including videos, podcasts, toolkits, checklists, patient stories, and more.
This series will address:
Medical Gaslighting
When a healthcare provider dismisses, minimizes, or invalidates a person’s symptoms, concerns, or experiences, it can lead to misdiagnosis, delayed treatment, or psychological distress. This is what we mean by Medical Gaslighting, an all too common issue for rare disease patients and caregivers.
This Quick Guide brings powerful stories from the rare community to light, equips you to recognize the signs of medical gaslighting, and offers actionable strategies to reclaim control of your healthcare. You’ll find practical tips to advocate for your needs, and empower yourself to be the strongest voice in your care journey ensuring your symptoms and experience are heard and valued.
Next Generation Drug Development Resources
A roadmap to guide your efforts as you partner with research. Advancing rare disease research is essential for creating meaningful change in the lives of patients and families, in our community. This collection of tools, guides, and expertise will help you navigate the research process. Explore areas of research, from discovery to clinical trials and regulatory engagement, and gain access to the resources that will support you in advancing rare disease research and treatments.
Guide to Interacting with FDA Through PFDD and Listening Sessions
Patient-Focused Drug Development (PFDD) and Listening Sessions provide an opportunity for patients, caregivers, and advocates to share their perspectives directly with the FDA.
This guide offers a roadmap for your patient community to successfully plan and execute Patient-Focused Drug Development (PFDD) or Listening Sessions with the FDA. Learn how to craft discussion guides, prepare educational materials, and coordinate with the FDA for improved collaboration. With actionable steps and strategic insights, this resource equips organizers to elevate patient voices and drive meaningful contributions.
Guide to Fundraising
Successful fundraising is essential for every nonprofit, and one of the biggest challenges. This Quick Guide provides clear, practical steps to help you secure funding, including:
- Grant Writing Best Practices
- Proven Fundraising Strategies
- Innovative Fundraising Ideas
Use this guide to refine and recharge your fundraising efforts, secure more grants, and build stronger donor relationships.
Strategies for Improving Cooperation Across PAGs
Collaboration within specific disease communities and the broader rare disease ecosystem is critical for our collective success. However, when multiple patient advocacy groups (PAGs) exist within the same disease space, challenges can arise. Ideally, these organizations work together seamlessly, but what happens when they do not?
This Quick Guide provides practical strategies to foster collaboration, improve communication, and resolve conflicts, enabling all parties to work towards shared goals.
How to Start a Rare Disease Patient Advocacy Organization
Starting a nonprofit can feel overwhelming. Which forms do you need? Who should be on your team? What should you prioritize first? What should you look for in your board members? This Quick Guide provides essential resources to support you at every step—practical tools, checklists, and mentorship opportunities—empowering you to take the lead in advocating for your rare disease community.
The Importance of Getting a Genetic Diagnosis
We can advocate more effectively and receive better medical care with an accurate diagnosis. However, obtaining one isn’t always easy. Unlock the path to targeted advocacy and better medical care through the power of a precise diagnosis! This Quick Guide demystifies the genetic testing process, transforming confusion into clarity. Dive into actionable next steps and empowering insights designed to help you take control of your family’s health. With this guide, you’re equipped to navigate testing confidently, champion your needs effectively, and pave the way for informed decisions that prioritize your loved ones’ well-being.
Knowing the Rights of Your RARE Child, (or Yourself) – A Checklist
Knowing your rights or those of your rare child (or yourself!) is the first step toward effective advocacy. This guide outlines what to ask for and what you’re entitled to.
Experienced advocates share insights on navigating complex insurance policies, educational requirements, access laws, and strategies for advocating effectively for loved ones.
Top Leadership Concerns for PAGs, And How to Address Them
Harnessing the wisdom of our 800+ member Global Advocacy Alliance, this Quick Guide will address some of the most common leadership pain points for rare disease PAG leaders. Get practical next steps and advice from seasoned leaders and mentors for navigating and improving each situation.
Don’t forget to check out the three Quick Guides released in 2024!
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.
If you would like to support this series contact [email protected]
This series is supported in part by: Amicus, Beam Therapeutics, Biocryst, BridgeBio, Chiesi, Mallinckrodt Pharmaceuticals, Merck, and Parexel
Stay Connected
Sign up for updates straight to your inbox.