Should Health-Care Systems Pay for Replacement Therapy in Patients With Alpha1-Antitrypsin Deficiency? A Critical Review and Cost-Effectiveness Analysis

Stephan A. Alkins, MD and Patrick O'Malley, MD, MPH From the Pulmonary and Critical Care Medicine Service (Dr. Alkins) and the Department of Internal Medicine (Dr. O'Malley), Walter Reed Army Medical Center, Washington, DC.


CHEST. 2000;117(3) 

In This Article


The incremental cost in a 70-kg alpha1-AT-deficient subject with severe emphysema (FEV1 < 50% of predicted) receiving weekly replacement therapy at 60 mg/kg is $13,971 per year of life saved. The cost-effectiveness of this therapy is calculated by assuming a 55% 5-year mortality rate reduction without discounting the years of life saved (Table 1 ; see also the Appendix).

Table 1. Incremental Cost Per Year of Life Saved*

Sensitivity analysis highlights the predominant dependence of this analysis on the degree of mortality rate reduction and, to a lesser degree, on the costs of treatment. If the effect size is only 10% (instead of 55% as seen in the NIH Registry data[10] ) and if yearly costs were unchanged ($52,000), then the incremental cost would be $152,941 per year of life saved. Conversely, if the effect size were 70%, then the incremental cost would be $7,330 per year of life saved (Table 1) . If effect size is 55% (as in the NIH data[10] ) but the yearly cost increases by nearly 300% to $150,000, then the incremental cost would only increase to $40,301 (Table 1) .

Cost-effectiveness thresholds are value judgments that are used to define scarce societal resources. These thresholds are arbitrary, however, they are used by health-care systems in resource allocation decision making. The cost-effective thresholds used in this analysis are presented only to give context to our findings. A therapy being considered for its cost-effectiveness can be compared to the annual cost of renal dialysis (a therapy that prolongs life, is accepted by the community, and is reimbursed by Medicare). The cost of renal dialysis per year of life saved is approximately $40,000 to $50,000.[19,20] If cost-effectiveness is defined as a therapy that costs < $40,000 per year of life saved, then the effect size of alpha1-AT replacement therapy would have to be >= 30% (with current therapy cost). The NIH Registry data reported that alpha1-AT replacement therapy reduced the 5-year mortality rate by 55% (33 to 15%). This therapy results in an incremental cost that is cheaper than the $40,000 per year of life saved when the annual cost is approximately < $149,000 (see Example 2 in the Appendix). When years of life saved is discounted by 7% and current therapy costs are used, then the effect size of alpha1-AT replacement therapy would have to be >= 33% (Table 2; see also Appendix) to result in an incremental cost of < $40,000 per year of life saved.


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