Cost-effectiveness of alpha-1 antitrypsin replacement therapy in treatment of congenital chronic obstructive pulmonary disease

• Published in: American journal of public health (1971) Vol. 81; no. 4; pp. 427 - 433
• Main Authors: Hay, J W, Robin, E D
• Format: Journal Article
• Language: English
• Published: Washington, DC Am Public Health Assoc 01-04-1991; American Public Health Association
• Subjects: Adult; alpha 1-Antitrypsin - genetics; alpha 1-Antitrypsin - therapeutic use; alpha 1-Antitrypsin Deficiency; Biological and medical sciences; Cardiovascular disease; Chronic obstructive pulmonary disease; Congenital diseases; Cost analysis; Cost-Benefit Analysis; Drugs; Female; Health care expenditures; Humans; Life Expectancy; Lung Diseases, Obstructive - congenital; Lung Diseases, Obstructive - drug therapy; Lung Diseases, Obstructive - economics; Lung Diseases, Obstructive - mortality; Male; Medical research; Medical sciences; Outcome and Process Assessment (Health Care); Pharmacology. Drug treatments; Public health; Respiratory system; Smoking; Value of Life; Human; Replacement therapy; Congenital; Respiratory disease; United States; Deficiency; Proteinase; Enzyme inhibitor; North America; Epidemiology; α1-Antitrypsin; Chemotherapy; Chronic; Health economy; America; Obstructive pulmonary disease; Public health; Cost efficiency analysis
• ISSN: 0090-0036; 1541-0048
• DOI: 10.2105/AJPH.81.4.427

Alpha 1-antitrypsin (AAT) replacement therapy is an expensive intervention ($20,000-$30,000 per patient annually) which may slow or arrest the progression of chronic obstructive pulmonary disease (COPD) in AAT-deficient patients. While FDA-approved, therapy efficacy is unknown. The costs and benefits of AAT replacement therapy were evaluated for patients with congenital COPD.
Epidemiological and disease cost data were taken from published sources. A discrete-time model of disease stage probability transition was developed to calculate the present-value expected cost of disease treatment, under a range of possible therapy efficacy and other parameter values.
At an efficacy of 70 percent, the cost per life year saved with AAT replacement therapy would be between $28,000 and $72,000 (1990 US dollars), depending on patient age, sex, and smoking status. At 30 percent efficacy, the cost per life year saved range would be between $50,000 and $128,000. A controlled efficacy study would cost $53 million or less, if the true efficacy were 50 percent or better.
With efficacy of 30 percent or higher, therapy cost-effectiveness would be comparable to other widely used medical interventions. The economic assessment methodology was used to evaluate both the therapeutic innovation and the value of additional clinical research.