OBJECTIVES : Sickle cell disease (SCD) is a progressive, debilitating genetic disease causing a wide range of acute events and chronic complications. Development of potentially curative, one-time treatments is changing the treatment paradigm. A modeled economic evaluation of such treatments may require a different approach than is used for currently available therapies. The objective of this research was to develop key criteria for an economic model for a potentially curative one-time treatment for SCD and to evaluate selected published models against these criteria.

METHODS : A comprehensive review of published studies of the burden of SCD and effects of treatment was undertaken. Based on the findings, key criteria were developed, which were then vetted by clinical experts. Twelve published models in SCD were identified; 3 pertained to a potentially curative one-time treatment (stem cell transplant). Evaluations of the economic models against the criteria were conducted.

RESULTS : Key criteria included the ability of the model structure and approach to capture the following:

• Heterogeneity in the SCD population leading to differences in disease progression

• Treatment options changing over a patient’s lifetime

• A comprehensive set of future acute SCD-related events and chronic complications, which a curative treatment would avoid

• The occurrence of time-dependent events and their influence on future outcomes

No published economic model in SCD met all of the criteria. Most models considered only a subset of patients with no consideration of population heterogeneity, a limited time horizon with no consideration of changes in treatment or progression trajectories over a lifetime, and/or only a subset of acute events or chronic complications.

CONCLUSIONS : Economic models estimating the value of potentially curative one-time treatments in SCD may require an approach distinct from previous approaches, such as a comprehensive patient-level simulation.