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

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 recommend...

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Autores principales: Johnson, Kate M., Jiao, Boshen, Bender, M. A., Ramsey, Scott D., Devine, Beth, Basu, Anirban
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9049306/
https://www.ncbi.nlm.nih.gov/pubmed/35482721
http://dx.doi.org/10.1371/journal.pone.0267448
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author Johnson, Kate M.
Jiao, Boshen
Bender, M. A.
Ramsey, Scott D.
Devine, Beth
Basu, Anirban
author_facet Johnson, Kate M.
Jiao, Boshen
Bender, M. A.
Ramsey, Scott D.
Devine, Beth
Basu, Anirban
author_sort Johnson, Kate M.
collection PubMed
description 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 identified disease attributes in Medicaid and Medicare claims databases, and identified relevant outcomes following the 2(nd) Panel in CEA. We determined an appropriate modeling technique and relevant data sources for parameterizing the model. RESULTS: The conceptual model structure included four dimensions of disease: chronic pain, acute events, chronic conditions, and treatment complications, spanning 26 disease attributes with significant impacts on health-related quality of life and resource. We modeled chronic pain separately to reflect its importance to patients and interaction with all other disease attributes. We identified additional data sources for health state utilities and non-medical costs and benefits of SCD. We will use a microsimulation model with age- and sex-specific transitions between health states predicted by patient demographic characteristics and disease history. CONCLUSION: Developing the model structure through an explicit process of model conceptualization can increase the transparency and accuracy of results. We will populate the conceptual model with the data sources described and evaluate the cost-effectiveness of curative therapies.
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spelling pubmed-90493062022-04-29 Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease Johnson, Kate M. Jiao, Boshen Bender, M. A. Ramsey, Scott D. Devine, Beth Basu, Anirban PLoS One Study Protocol 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 identified disease attributes in Medicaid and Medicare claims databases, and identified relevant outcomes following the 2(nd) Panel in CEA. We determined an appropriate modeling technique and relevant data sources for parameterizing the model. RESULTS: The conceptual model structure included four dimensions of disease: chronic pain, acute events, chronic conditions, and treatment complications, spanning 26 disease attributes with significant impacts on health-related quality of life and resource. We modeled chronic pain separately to reflect its importance to patients and interaction with all other disease attributes. We identified additional data sources for health state utilities and non-medical costs and benefits of SCD. We will use a microsimulation model with age- and sex-specific transitions between health states predicted by patient demographic characteristics and disease history. CONCLUSION: Developing the model structure through an explicit process of model conceptualization can increase the transparency and accuracy of results. We will populate the conceptual model with the data sources described and evaluate the cost-effectiveness of curative therapies. Public Library of Science 2022-04-28 /pmc/articles/PMC9049306/ /pubmed/35482721 http://dx.doi.org/10.1371/journal.pone.0267448 Text en © 2022 Johnson et al https://creativecommons.org/licenses/by/4.0/This is an open access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/) , which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
spellingShingle Study Protocol
Johnson, Kate M.
Jiao, Boshen
Bender, M. A.
Ramsey, Scott D.
Devine, Beth
Basu, Anirban
Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease
title Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease
title_full Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease
title_fullStr Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease
title_full_unstemmed Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease
title_short Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease
title_sort development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease
topic Study Protocol
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9049306/
https://www.ncbi.nlm.nih.gov/pubmed/35482721
http://dx.doi.org/10.1371/journal.pone.0267448
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