Federal and Private Roles in the Development and Provision of Alglucerase Therapy for Gaucher Disease (Part 3 of 4)

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.