Conducting a research study for an orphan indication and/or rare disease involves unique considerations in addition to the more common challenges of clinical research. Key logistical and operational considerations will be discussed including sharing of specific, relevant examples from different therapeutic areas and clinical settings as well as strategies and techniques to navigate the challenges. The session will also address specific examples of materials, procedures and techniques developed and utilized for protocol/study design development, site identification and support, IRB/EC approval, subject recruitment and retention, subject support, and program timeline and budget management.

Learning Objectives:
Identify unique challenges and risks associated with conducting studies for orphan indications and rare diseases
Recognize the logistical and operational considerations associated with studies for orphan indications and rare diseases.