Designing for Recruitment and Retention in Rare Disease Studies

An area of a clinical study that is gaining momentum is a rare disease. The publisher acknowledges pharma’s growing interest in this space. While this means opportunity for the drug development industry, the unique nature of rare disease studies is burdensome. Greater challenges are baked into rare disease studies compared to typical studies: very small patient pools, specialized protocols, difficult-to-define endpoints, often very sick patients with limited ability or time, and the predicament of high cost versus low volume.

Designing for Recruitment and Retention in Rare Disease Studies report takes a deep dive into two areas impacting the success of these studies: recruitment and retention. Given the importance of each rare disease patient, these activities are time-intensive and resource-heavy. We asked 125 respondents involved in these activities about the challenges they face, methods and support services used, and their perspectives and practices regarding patient-friendly alternatives to the clinical setting.

Sponsors:

• Understand the recruitment and retention challenges facing rare disease studies and how to mitigate them
• Learn the practices and support services being utilized for rare disease studies, plus perspectives on unconventional methods
• Explore various patient-friendly alternatives to the clinical setting and their impact on patient retention

Service Providers:

Understand the pain points associated with recruitment and retention activities in rare disease studies
Gain insight into rare disease study preferences and practices, and use this information to evaluate/market your offerings
Learn the specific support services needed by sponsors to best accommodate patients in rare disease studies

Major Topics:

• Baseline Challenges & Perspectives
• Recruitment Methods
• Retention Practices
• Study Data