Waste Not, Want Not – Making the Most of Small Patient Population Data
_***Recommended for Young Investigators/Researchers**_ **Strategies for Successful Research in Small Patient Populations** * Discuss how to support and engage in research activities and develop interest for small patient population studies **Understanding Measurement Tools to Capture Patient Data in Rare and Ultr…
Advances in Clinical Trial Design & Real World Evidence in Rare Disease
_***Recommended for Young Investigators/Researchers**_ **Addressing the Need for Innovative Clinical Trials to Advance Rare Disease Therapies** * Challenges in traditional double-blind randomized clinical trial design for rare disease studies * Ethics and access to clinical trials in rare disease research **Bas…
Building a Research Strategy Roadmap for Your Organization
_***Recommended for Patients/Advocates**_ **Strategizing for Success: Developing a Research Strategy for Your Foundation:** * Foundational elements of building a research development strategy for patient advocacy groups * Tactics to scale operations and leverage resources for maximum impact * Discuss how to id…
Beyond Publish or Perish – Attracting Funding for Impact
_***Recommended for Young Investigators/Researchers**_ **Opportunities to Attract Funding for Rare Disease Research** * Funding from advocacy organizations and nonprofit organizations * Government/public grants and funding – NIH, DOD, and more * Novel funding sources emerging in rare disease research **Findin…
Welcome, Opening Remarks and Keynote Presentation – Collaborations, Community and Citizen Science
**Keynote Speaker: David Fajgenbaum, MD, MBA, MSc, Co-Founder & President, Castleman Disease Collaborative Network** Explore the evolution of patient-driven progress in research and the critical ways in which patients, advocates, and caregivers are changing the drug development paradigm. From patient engagement in en…
