Federal and private roles in the development and provision of alglucerase therapy for Gaucher disease

Office of Technology Assessment
Record ID 31995000078
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
Year Published: 1992
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: United States
MeSH Terms
  • Drug Costs
  • Drug Industry
  • Gaucher Disease
  • Orphan Drug Production
Organisation Name: U. S. Congress. Office of Technology Assessment
Copyright: Office of Technology Assessment
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