Patient Engagement
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.
Global Genes provides hope for the more than 400 million people affected by rare disease around the globe. We fulfill our mission by helping patients find and build communities, gain access to information and resources, connect to researchers, clinicians, industry, government, and other stakeholders, share data and experiences, stand up, stand out, and become effective advocates on their own behalf.
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.
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.
Supports for Rare Disease Patient Advocacy Groups
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.
Guidelines for Patient Engagement in Research
EveryLife Foundation: Guide to Patient Involvement in Rare disease Therapy Development
For all stakeholders (i.e. Patient Advocacy Groups and sponsors)
Clinical Trials Transformation Initiative (CTTI): Patient Group Engagement
For all stakeholders (i.e. PAGs and sponsors)
NIH National Center for Advancing Translational Sciences: Toolkit for Patient-Focused Therapy Development (NCATS Toolkit)
For new PAGs with limited resources and experience, interested in research
Guidance for PAG Development and Growth
NORD: Mentorship for growing a patient organization/nonprofit
For existing organizations (platinum member is already engaged in research; gold member is not engaged in research)
Global Genes: Guide on Starting a Non-Profit
For new/interested rare disease advocates
Rare Advocacy Movement: Roadmap for Equity Pledge
For any PAG interested in working toward diverse, equitable, and inclusive programming
PAG Leadership Development
FasterCures Milken Institute: LeaderLink Program (builds leadership skills and network through mentorship, capstone project, and virtual collaboration)
For new leaders of nonprofit organization with interest in research (new leadership within 3 years)
YARR Leadership Academy: (series of online classes)
For young adults in the rare community who are interested in future leadership roles
Registry/Data Infrastructure and/or Health Record Collection
RARE-X: Data storage, sharing, standardization, and analysis
For any PAG interested in collecting data
Simons Searchlight: Data storage, sharing, standardization, and analysis
For any PAG interested in collecting data; any patient interested in contributing personal data
AllStripes: Health record collection and storage infrastructure (individual patients can contribute their own data to database)
For any PAG interested in collecting data; any patient interested in contributing personal data
Ciitizen: Health record collection and storage infrastructure (individual patients can contribute their own data to database)
For any PAG interested in collecting data; any patient interested in contributing personal data
Guidance on Data Collection
Global Genes: Data DIY (guidance for collecting and working with data)
For any nonprofit leader interested in collecting data
Prometheus IQVIA: Registry Platform & Consulting Service
For existing/mature PAG interested in creating a registry/considering therapeutic development
Research Networks and Research Funding
FasterCures Milken Institute: The Research Acceleration and Innovation Network (TRAIN)
For mature/advanced organizations with paid staff, budget for research, and research initiatives
NIH Rare Diseases Clinical Research Network: Coalition of Patient Advocacy Groups (CPAG)
For mature organizations with an interest in research
Chan Zuckerberg Initiative (CZI): Rare As One Project (provides research grants and collaborative network)
For existing nonprofit PAGs with less than US$2 million budget