About Patient Registries

In 2009, approximately 85,000 patients were treated with immunoglobulin therapy. Diseases requiring immunoglobulin therapy are often described as rare, and although these diseases are clinically described in medical literature, there is no comprehensive and well characterized portrait available of the patient with primary immune deficient and/or immune-mediated neurological disorders.

Patient registries and randomized controlled trials have important and complementary roles in evaluating patient outcomes. Patient registries collect data in a comprehensive manner, and therefore, produce outcome results that can be generalized to a wide range of patients. They also evaluate care as it is actually provided, because care is not assigned, determined, or even recommended by a protocol. As a result, the outcomes reported are more representative of what is achieved in real-world practice.

A patient registry can be a powerful tool to observe the course of disease; to understand variations in treatment and outcomes; to examine factors that influence prognosis and quality of life; to describe care patterns, including appropriateness of care and disparities in the delivery of care; to assess effectiveness; to monitor safety; and, to change behavior through feedback of data.

Benefits of a Registry

• Obtain real-world therapeutic effectiveness and safety data
• Large patient numbers can detect rare adverse events
• Heterogeneity among numerous investigative sites
• Research collaboration with aggregate data reporting to investigators
• Real-world publications/add to the body of knowledge on effectiveness data and outcomes
• Usual diagnostic and follow-up procedures can be used rather than "research" procedures
• Evidence-based medicine for outcomes and reimbursement
• Study subjects are heterogeneous
  • Various treatments
  • Various concomitant medications
  • Various co-morbidities