2nd Patient Recruitment in Rare Disease Trials

The rare disease trial landscape stands at a pivotal tipping point. While unprecedented advances in trial design, digital tools, and regulatory flexibility are accelerating innovation like never before, the reality for patients remains stark: over 90% of rare diseases still lack an FDA-approved treatment. For many, clinical trials represent the only path to care, yet access remains uneven. Geographic isolation, diagnostic delays, and socioeconomic barriers continue to exclude many patients, especially from underserved communities, from participating in research.

Add to this the small, dispersed patient populations, high protocol complexity, and a shortage of disease-specific expertise, and it's clear that traditional recruitment approaches no longer suffice.

Now in its second year, the Patient Recruitment for Rare Disease Trials Summit stands as the premier forum dedicated to closing this critical gap. Designed for leaders in Patient Recruitment, Clinical Operations, Patient Advocacy, and Medical Affairs this summit unites innovators reshaping rare disease trial access across the entire patient journey. Over three focused days, you'll explore cutting-edge strategies spanning early patient identification to decentralized, patient-centric trial models that support participants from diagnosis through to long-term retention. Now is the time to accelerate creative recruitment strategies, deepen early patient engagement, and harness innovative technologies to ensure no rare disease patient is left behind in the race for transformative therapies.

Speakers: Diego Cortina, Director, Global Pipeline and Value Evidence Strategy, Biogen, LaShell Robinson, Executive Director, Head Global Feasibility and Trial Equity, Takeda, Melissa Cabral, Associate Director, Patient Engagement, Bristol Myers Squibb, Nanci Eannucci, Senior Director, Site Engagement, Teva Pharmaceuticals, Ravi Pathak, Medical Director, Takeda, Susan Symington, Regional Director, Medical Science Liaison, Genentech, Amanda Sowinski, Associate Director, Patient Professional Advocacy and Global Pompe Lead, Amicus Therapeutics, Anna White, Director, Medical Affairs, Calliditas Therapeutics, Brittany Davis, Senior Director, Patient Advocacy and Insights, Avalyn Pharmaceuticals, Charles Shiner, Associate Vice President, Gene Therapy Marketing, Rocket Pharmaceuticals, Christine Lacaria, Senior Manager, Patient Recruitment, Sarepta Therapeutics, Courtney Klein, Patient Recruitment and Engagement Lead, Alexion, Deborah Micklos, Senior Director, Clinical Operations, Inventiva, Fatima Scipione, Vice President, Global Patient Affairs, Blueprint Medicines, Jessica Shull, Clinical Science Liaison and Director of Digital Health, Vicore Pharma, Matt Hay, U.S. Director of Advocacy for Metabolics - Neurofibromatosis (NF) and Hypophosphatasia (HPP), Alexion Pharmaceuticals, Rinaldo Dorman, Associate Director, Clinical Procurement, Sarepta Therapeutics, Seth Hall, Medical Science Liaison, Liquidia Corporation, Wendy Borsari, Associate Director, Patient Advocacy, Tenaya Therapeutics, Isabelle Lousada, Chief Executive Officer and Co-Founder, The LAM Foundation, Kirsti Mitchell, Founder and Chief Executive Officer, Health Equity Outcomes, Sabina Schmidt, Goldstein-Becerra, Executive Director, International Cancer Advocacy Network (ICAN), Tricha Shivas, Chief of Staff and Strategy, Foundation for Sarcoidosis Research, Dennis Akkaya, Chief Commercial Officer, myTomorrows

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Brochure: https://go.evvnt.com/3123656-3?pid=6581