Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease.

ABSTRACT:

Background

Sickle cell disease (SCD) is a clinically heterogeneous disease with many acute and chronic complications driven by ongoing vaso-occlusion and hemolysis. It causes a disproportionate burden on Black and Hispanic communities. Our objective was to follow the SMDM/ISPOR Task Force recommendations for good practices and create a conceptual model of the progression of SCD under current clinical practice to inform cost-effectiveness analyses (CEA) of promising curative therapies in the pipeline over a lifetime horizon.

Methods

We used consultations with experts, providers, and patients to identify acute events and chronic conditions in the conceptual model. We compared our model structure to previous CEA models of interventions for SCD, assessed the prevalence of the id

SUBMITTER: Johnson KM 

PROVIDER: S-EPMC9049306 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature