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Explore Patient-Centric Approaches to Trial Design, Recruitment and Access to Enhanced Outcomes

December 3-4, 2019
  • Arlington, VA

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, researchers and patients to discuss common obstacles in clinical development, exchange best practices and case study learnings and explore opportunities to implement cost saving measures. 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.

Optimize Clinical Collaborations to
Improve Outcomes and Access:

  • Identify opportunities to de-risk clinical development through new technologies and advanced analytics
  • Evaluate opportunities to utilize registry data in pre-clinical and early clinical studies
  • Hear about opportunities to incorporate patient preference data into clinical trial design and determination of endpoints
  • Overcome challenges in patient identification and recruitment
  • Delve into partnerships and collaborations to advance and accelerate clinical pathways
  • Explore the NCATS toolkit for patient-focused therapy development
  • Discuss opportunities to employ innovative trial designs to provide robust data in small patient populations
  • Plan for reimbursement and commercial success in early development