Research
Getting Involved in Research
Resources to Advance Your Research
Research is the key to unlocking new possibilities for rare disease treatments and cures, and patient advocates are taking a more active role in advancing therapies. Whether you’re just beginning your journey or are already well-versed in the complexities of research, these resources will help you navigate the vital areas of research, from discovery to clinical trials and regulatory engagement, and support you in advancing research and treatments for your community.
Explore Our Research Resources:
- Discovery: Lay the foundation for innovative research with essential tools and strategies.
- Preclinical: Understand the steps before clinical trials, from animal models to safety assessments.
- Clinical Research / Clinical Trials: Access resources to design, manage, and implement clinical trials that drive real-world change.
- FDA / Regulatory Engagement: Navigate the complexities of regulatory processes and engage with the FDA effectively.
- Data Collection, Registries, & Natural History Studies: Learn how to collect valuable data and create registries that can shape the future of rare disease treatments.
- Tools to Use Throughout the Research Process: Discover resources to streamline and support research at every phase.
Together, we can accelerate progress and drive hope for the rare disease community.
Overview Resources
- How Are New Drugs & Therapies Developed?
- RARE Research Roadmap
- Create Your Strategy Using the RARE Research Roadmap
- Toolkit for Patient-Foused Therapy Development
- Sharing Patient Voices Throughout the Process
Discovery Resources
- Thinking Like a Researcher: Disease Biology 101
- Small Molecules, Drug Repurposing and Biologics
- Drug Repurposing Guidebook
- Gene Replacement & Gene Editing Therapies
- RNA Therapies: ASOs, mRNAs, siRNAs
- Accelerating Rare Disease Drug Discovery
Preclinical Resources
- Pre-Clinical Research: Why and When to Invest
- Understanding Translational Research Tools
- Biomarkers and Outcome Measures
- Overcoming the Valley of Death in Research
Clinical Research / Clinical Trials Resources
- From Molecules to Medicine: Clinical Research
- Becoming Clinical Trial Ready
- Rethinking Clinical Trials: What’s Doable? What’s Approvable?
- Participating in a Clinical Trial
FDA / Regulatory Engagement Resources
- 7 Ways to Engage the FDA
- Interacting with the FDA: PFDD and Listening Sessions
- FDA Critical Path Innovation Meetings
- FDA Guidance Documents
- Guide to Patient Involvement in Rare Disease Therapy Development
Data Collection, Registries, & Natural History Studies
- The Critical Need for Patient-Led Data Initiatives
- Data DIY: Becoming A Data-Centric Community
- FDA Guidance on Registries
- FDA Guidance on Natural History Studies
- Enabling Cell & Gene Therapies Through Non-Proprietary Data Collection
Tools to Use Throughout the Research Process
- Research Partnership Maturity Model
- Template Agreements
- Think Like a Start Up: Grow your Patient Advocacy Group
- What Will $100K Buy You? Emerging Commercial and Non-Profit Financing Models
- Innovative Funding Strategies
- Patient Engagement Resources
- Patient Compensation Tools & Fair Market Value Calculator
Need More Help?
Global Genes is here to help answer your questions and guide you through your research journey.
Email us at [email protected]
This page is part of the Global Genes 2025 Quick Guide Series, our way to address common needs identified and requested by the rare community. Each Quick Guide 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
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