Description

The drug development and approval process is time-consuming, risky, and potentially costly enough to preclude the development and marketing of treatments for rare disorders. The case of alglucerase, an expensive new therapy for the uncommon inherited disorder called Gaucher disease, illustrates how the Federal Government can help manufacturers overcome critical scientific, financial, and regulatory barriers to the development of such treatments. This paper analyzes public and private investments in the research and development (R&D) of alglucerase. It also examines uncertainty surrounding the appropriate dosing of the drug and the cost implications of alglucerase therapy for patients, their insurers, and the Federal Government.

Gaucher disease is caused by deficient activity of an enzyme necessary to break down glycolipids, substances produced by white blood cells. The accumulation of these glycolipids in the spleen, liver, bone marrow, and other organs can lead to abdominal and bone pain, anemia, and other severe manifestations of disease. Gaucher disease causes significant disability and can be fatal. Between 2,100 and 11,000 people in the United States are believed to have symptoms severe enough to warrant medical intervention.