Cost-effectiveness analysis of augmentation therapy for severe α1-antitrypsin deficiency

A Markov-based decision model was created to assess the cost-effectiveness of augmentation therapy (Aug) for severe oil-antitrypsin deficiency, comparing strategies of: (1) no Aug, (2) Aug for life, and (3) Aug until FEV1 is below 35% predicted. A hypothetical cohort of 46-year-old patients with FEV1 49% predicted was followed over time using Monte Carlo simulation across five possible health states: (1) FEV1 50 to 79% predicted, (2) FEV1 35 to 49% predicted, (3) FEV1 below 35% predicted, (4) status-post-lung transplantation, and (5) dead. Treatment for life yielded 7.19 quality-adjusted life-years (QALYs) and cost $895,243. Treating until FEV1 is below 35% predicted cost $511,930 and produced 6.64 QALYs. "No Aug" cost $92,091 with 4.62 QALYs. The incremental cost-effectiveness ratio was $207,841/QALY for Aug until FEV1 is below 35% predicted and $312,511/QALY for the "Aug for life" strategy. In all sensitivity analyses, the incremental cost-effectiveness ratio for Aug for life exceeded $100,000. The cost of Aug needed to be reduced from $54,765 to $4,900 for the "Aug for life" strategy to be considered cost-effective. We conclude that, compared with other conventionally used health interventions, Aug is relatively less cost-effective. These results should encourage the development of more clinically and cost-effective therapies for alpha(1)-antitrypsin deficiency.