Comparative effectiveness research in the United States and primary immunodeficiency diseases

Abstract

The United States enacted legislation in 2010 that will promote the use of comparative effectiveness research in the making of healthcare decisions. Of concern with this relatively new mandate is the possibility of using comparative effectiveness research as a means only to mitigate costs rather than to focus on quality of care. This is of particular concern for patients with rare and chronic conditions, such as primary immunodeficiency diseases. The Immune Deficiency Foundation (IDF) uses their survey research to advocate for the needs of patients with primary immunodeficiency diseases to ensure that their unique medical concerns are not overlooked but instead, integrated within an overall healthcare emphasis on personalized medicine and differences in patient treatment response. IDF research shows the efficacy of treatment with immunoglobulin replacement therapy (IG therapy) for many patients with primary immunodeficiency diseases, that IG therapy is underused in the primary immunodeficient community, and that IG therapies are unique and not interchangeable.