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


METHODS
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.


RESULTS
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.


CONCLUSIONS
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.
Hay
J W
JW
Hoover Institution, Stanford University, CA 94305-6010.
Robin
E D
ED
D000328:Adult; D003362:Cost-Benefit Analysis; D005260:Female; D006801:Humans; D008017:Life Expectancy; D008173:Lung Diseases, Obstructive / Q000151:congenital / Q000188:drug therapy* / Q000191:economics / Q000401:mortality; D008297:Male; D010043:Outcome and Process Assessment, Health Care; D012907:Smoking; D026712:Value of Life; D000515:alpha 1-Antitrypsin / Q000235:genetics / Q000627:therapeutic use*; D019896:alpha 1-Antitrypsin Deficiency
D000515:alpha 1-Antitrypsin