Authors' objectives:

To analyze the development, provision, and payment in the United States for alglucerase (Ceredase), an important, but extremely expensive new therapy for Gaucher disease.

Authors' results and conclusions: Alglucerase (Ceredase) is a treatment for Gaucher disease, a relatively rare, inherited disorder in which the body lacks an enzyme to process fats. These fats accumulate in the liver, spleen, and bones sometimes causing severe symptoms and even death. Like many new therapies for rare conditions, alglucerase benefited greatly from Federal subsidies including significant discovery and development research at the U. S. National Institutes of Health, as well as the market exclusivity provisions first adopted in the Orphan Drug Act of 1983 (Public Law 97-414). These subsidies on top of an insurance system that usually must pay for new pharmaceutical therapies without regard to price make the development of life-saving, but extremely expensive drugs possible.

Authors' recommendations: This case study illustrates the role that both the Federal government and private sector can have in making new therapies available to patients, and lays out the tradeoffs that can exist between developing new medical technologies and controlling health care costs.

Authors' methods: Review

Project Status: Completed

URL for project: http://www.wws.princeton.edu:80/~ota/ns20/pubs_f.html

Year Published: 1992

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: United States

Organisation Name: U. S. Congress. Office of Technology Assessment

Copyright: Office of Technology Assessment