Because of the rarity, heterogeneity and the complex patient management associated with rare conditions, creating and executing an orphan drug development program presents some of the highest hurdles in medicine. Worldwide Clinical Trials approaches rare diseases with therapeutic acumen and operational sophistication. We understand the science, medicine, potential regulatory constraints, and the operational hindrances.

Worldwide has participated in the design, execution, and/or analysis of >50 studies in orphan diseases. Therapies have included small-molecule enzyme inhibitors, immunotherapies (monoclonal antibodies), hormone therapy, and enzyme replacement therapy.

Global reach is essential to assure access to patients, a process which can be elusive. Protocols must account for highly diverse clinical manifestations and disease progression, effectively introduce dose-ranging solutions and create design structures which are compatible with the demands placed upon families who must present patients to specialized centers for evaluations.

With our practical experience, and knowledge of methodologically rigorous options, we can propose study designs and data-analysis strategies suited to the challenges of orphan diseases.