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Rare Diseases: Patients, Caregivers and Advocates as Equal Partners in Clinical Trial Process
Session Chair(s)
Barbara Szymaszek
Advocacy, Diversity and Patient Engagement
Bristol-Myers Squibb Company, United States
This forum will discuss ways to enhance protocol design, recruitment and retention strategies for clinical trials in rare/orphan diseases, offering both strategic and tactical approaches. We will examine lessons learned on data from over 50 rare disease studies, underscoring the critical role of referring physicians. We will address operational challenges, strategies and tactics to design and conduct protocols that are practical from the patient and caregiver perspectives, and will share a case study of the impact patient advocacy organizations make in drug development. Finally, the panel will examine the definition of return on investment and other measures of success.
Learning Objective : Identify benefits of collaboration with multiple key stakeholders - patients, caregivers, physicians and advocacy organizations - to shape phase 1-3 programs for rare/orphan diseases; Describe ways to build relationships and incorporate feedback from key stakeholders; Summarize challenges and lessons learned in applying a patient-centered approach.
Speaker(s)
Engaging Patients Every Step of the Way
Patricia Furlong, BSN
Parent Project Muscular Dystrophy, United States
Founding President and Chief Executive Officer
Relationship Building in Rare Diseases: The Critical Role of Referring Physicians
Jaime Cohen
BBK Worldwide, United States
Strategic Consultation & Research