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Rare Disease Clinical Development & Access Summit

Arlington, VA, USA

December 3–4, 2019

While there is an increasing interest and awareness in exploring clinical development for rare disease therapies, significant challenges remain in ensuring successful and sustainable clinical trials. CBI’s Rare Disease Clinical Development & Access Summit provides a unique opportunity for industry professionals, researchers and patients to discuss common obstacles in clinical development, exchange best practices and case study learnings and explore opportunities to de-risk drug development for rare disease therapies. Key topics addressed will include patient identification, recruitment and retention, novel study design and approaches, patient preference studies, preclinical collaborations and evolving technologies to enable adaptive trials. Patient-driven progress will also be highlighted through discussions of changes in the drug development paradigm.