For Patient Advocacy Groups
Advocacy Supports
The Rosamund Stone Zander Translational Neuroscience Center provides hope for the more than 400 million people affected by rare disorders around the globe. We fulfill our mission by helping patients and advocacy groups find and build communities, gain access to information and resources, and connect to researchers. The organizations and resources provided below can help support patient advocacy organizations as they further develop their research partnerships, infrastructure, and funding models.
The table below is adapted from our 2023 article, “Emerging roles and opportunities for rare disease patient advocacy groups” in Therapeutic Advances in Rare Disease (Vol. 4: 1–18). You can read the article HERE.
Global Genes
Global Genes is a 501(c)(3) non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally
Combined Brain
Combined Brain is a non-profit consortium led by patient advocacy foundations, working with the clinicians, researchers and pharmaceutical firms that are developing treatments for the disorders they represent.
Rare Epilepsy Network
REN is comprised of mature organizations as well as those newly formed. We are frequently asked for resources for starting a new rare epilepsy organization. There are many resources for starting a nonprofit.
Simons Searchlight
Simons Searchlight is an international research program with the goal of accelerating science and improving lives for people with rare genetic neurodevelopmental disorders.
Guidelines for Patient Engagement in Research
Guide based on FDA recommendations on inclusion of patient experience in rare disorder therapy development.
CTTI
Framework for ensuring patient advocacy group engagement throughout the entire clinical trial process.
NCATS
Toolkit for Patient-Focused Therapy Development (NCATS Toolkit)
Guidance for PAG Development and Growth
Mentorship for growing a patient organization/nonprofit
Detailed toolkit for starting and building a foundation
Service for applying and maintaining 501c3 status
Guidance on Data Collection
Guide for collecting and working with data
PAG Leadership Development
Program to build leadership skills and network through mentorship, capstone project, and virtual collaboration
Online academy for young adults in the rare disease community who are interested in learning more about advocacy, policy making, therapeutic development, and regulatory processes
Research Networks and Research Funding
Network of over 160 foundations taking a “venture philanthropy” approach to funding medical research
NIH Rare Diseases Clinical Research Network (RDCRN)
Network of patient advocacy groups affiliated with the NIH RDCRN promoting collaboration and better access to rare disease research
Chan Zuckerberg Initiative
Funding opportunities for research and community building
Other Supports
NeuroJourney
Tool to support caregivers of children with complex neurological medical needs
The Rare Disease Diversity Coalition
Network dedicated to addressing the extraordinary challenges faced by historically underrepresented rare disease patients as encompassed by social determinants of health (SDOH).
Simons Searchlight Event Planning Toolkit
Event planning toolkit, designed in collaboration with Global Genes, for patient advocacy members.