Cargando…

Cost-consequence model comparing eltrombopag and romiplostim in pediatric patients with chronic immune thrombocytopenia

BACKGROUND: Immune thrombocytopenia (ITP) is an auto-immune disorder characterized by enhanced platelet destruction and, subsequently, the potential for increased bleeding. Thrombopoietin receptor (TPO-R) agonists have recently emerged as promising therapies for ITP patients who are refractory to ot...

Descripción completa

Detalles Bibliográficos
Autores principales: Tremblay, Gabriel, Dolph, Mike, Bhor, Menaka, Said, Qayyim, Roy, Anuja, Elliott, Brian, Briggs, Andrew
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove Medical Press 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6223346/
https://www.ncbi.nlm.nih.gov/pubmed/30464564
http://dx.doi.org/10.2147/CEOR.S177338
_version_ 1783369387108990976
author Tremblay, Gabriel
Dolph, Mike
Bhor, Menaka
Said, Qayyim
Roy, Anuja
Elliott, Brian
Briggs, Andrew
author_facet Tremblay, Gabriel
Dolph, Mike
Bhor, Menaka
Said, Qayyim
Roy, Anuja
Elliott, Brian
Briggs, Andrew
author_sort Tremblay, Gabriel
collection PubMed
description BACKGROUND: Immune thrombocytopenia (ITP) is an auto-immune disorder characterized by enhanced platelet destruction and, subsequently, the potential for increased bleeding. Thrombopoietin receptor (TPO-R) agonists have recently emerged as promising therapies for ITP patients who are refractory to other treatments. While eltrombopag (EPAG) is the only TPO-R agonist US Food and Drug Administration approved for use in pediatric patients, romiplostin (ROMI) has been used in Phase III clinical studies. METHODS: A cost-consequence model (CCM) was developed to evaluate the costs of EPAG, ROMI, and watch-and-rescue (W&R) in relation to their respective treatment outcomes in previously-treated pediatric chronic ITP (cITP) over a 26-week time horizon. The costs of drugs, administration, routine care, rescue medications, adverse events, and mortality were included. Data on platelet count response rate, bleeding events, and adverse events were derived from all relevant identified Phase III-registered clinical trials, health outcomes were compared via indirect treatment comparison. RESULTS: The overall estimated cost of EPAG per patient was US$66,550, compared to US$101,056 for ROMI and US$32,720 for W&R. EPAG’s lower cost compared to ROMI was largely due to lower drug costs (US$62,202 vs US$84,396), administration costs (US$0 vs US$1,955), and significantly lower costs due to severe bleeding (US$354 vs US$10,191). When assessing cost per severe bleeding event avoided, EPAG was dominant over ROMI (less expensive and more effective). EPAG was again dominant over ROMI when assessing the cost per responder and per bleeding event (any grade). Sensitivity analysis was consistent with the base case findings. CONCLUSION: EPAG was the preferred TPO-R agonist to treat cITP when indirectly compared to ROMI, largely driven by its favorable severe bleeding outcomes and lower drug and administration costs.
format Online
Article
Text
id pubmed-6223346
institution National Center for Biotechnology Information
language English
publishDate 2018
publisher Dove Medical Press
record_format MEDLINE/PubMed
spelling pubmed-62233462018-11-21 Cost-consequence model comparing eltrombopag and romiplostim in pediatric patients with chronic immune thrombocytopenia Tremblay, Gabriel Dolph, Mike Bhor, Menaka Said, Qayyim Roy, Anuja Elliott, Brian Briggs, Andrew Clinicoecon Outcomes Res Original Research BACKGROUND: Immune thrombocytopenia (ITP) is an auto-immune disorder characterized by enhanced platelet destruction and, subsequently, the potential for increased bleeding. Thrombopoietin receptor (TPO-R) agonists have recently emerged as promising therapies for ITP patients who are refractory to other treatments. While eltrombopag (EPAG) is the only TPO-R agonist US Food and Drug Administration approved for use in pediatric patients, romiplostin (ROMI) has been used in Phase III clinical studies. METHODS: A cost-consequence model (CCM) was developed to evaluate the costs of EPAG, ROMI, and watch-and-rescue (W&R) in relation to their respective treatment outcomes in previously-treated pediatric chronic ITP (cITP) over a 26-week time horizon. The costs of drugs, administration, routine care, rescue medications, adverse events, and mortality were included. Data on platelet count response rate, bleeding events, and adverse events were derived from all relevant identified Phase III-registered clinical trials, health outcomes were compared via indirect treatment comparison. RESULTS: The overall estimated cost of EPAG per patient was US$66,550, compared to US$101,056 for ROMI and US$32,720 for W&R. EPAG’s lower cost compared to ROMI was largely due to lower drug costs (US$62,202 vs US$84,396), administration costs (US$0 vs US$1,955), and significantly lower costs due to severe bleeding (US$354 vs US$10,191). When assessing cost per severe bleeding event avoided, EPAG was dominant over ROMI (less expensive and more effective). EPAG was again dominant over ROMI when assessing the cost per responder and per bleeding event (any grade). Sensitivity analysis was consistent with the base case findings. CONCLUSION: EPAG was the preferred TPO-R agonist to treat cITP when indirectly compared to ROMI, largely driven by its favorable severe bleeding outcomes and lower drug and administration costs. Dove Medical Press 2018-11-05 /pmc/articles/PMC6223346/ /pubmed/30464564 http://dx.doi.org/10.2147/CEOR.S177338 Text en © 2018 Tremblay et al. This work is published and licensed by Dove Medical Press Limited The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution – Non Commercial (unported, v3.0) License (http://creativecommons.org/licenses/by-nc/3.0/). By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed.
spellingShingle Original Research
Tremblay, Gabriel
Dolph, Mike
Bhor, Menaka
Said, Qayyim
Roy, Anuja
Elliott, Brian
Briggs, Andrew
Cost-consequence model comparing eltrombopag and romiplostim in pediatric patients with chronic immune thrombocytopenia
title Cost-consequence model comparing eltrombopag and romiplostim in pediatric patients with chronic immune thrombocytopenia
title_full Cost-consequence model comparing eltrombopag and romiplostim in pediatric patients with chronic immune thrombocytopenia
title_fullStr Cost-consequence model comparing eltrombopag and romiplostim in pediatric patients with chronic immune thrombocytopenia
title_full_unstemmed Cost-consequence model comparing eltrombopag and romiplostim in pediatric patients with chronic immune thrombocytopenia
title_short Cost-consequence model comparing eltrombopag and romiplostim in pediatric patients with chronic immune thrombocytopenia
title_sort cost-consequence model comparing eltrombopag and romiplostim in pediatric patients with chronic immune thrombocytopenia
topic Original Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6223346/
https://www.ncbi.nlm.nih.gov/pubmed/30464564
http://dx.doi.org/10.2147/CEOR.S177338
work_keys_str_mv AT tremblaygabriel costconsequencemodelcomparingeltrombopagandromiplostiminpediatricpatientswithchronicimmunethrombocytopenia
AT dolphmike costconsequencemodelcomparingeltrombopagandromiplostiminpediatricpatientswithchronicimmunethrombocytopenia
AT bhormenaka costconsequencemodelcomparingeltrombopagandromiplostiminpediatricpatientswithchronicimmunethrombocytopenia
AT saidqayyim costconsequencemodelcomparingeltrombopagandromiplostiminpediatricpatientswithchronicimmunethrombocytopenia
AT royanuja costconsequencemodelcomparingeltrombopagandromiplostiminpediatricpatientswithchronicimmunethrombocytopenia
AT elliottbrian costconsequencemodelcomparingeltrombopagandromiplostiminpediatricpatientswithchronicimmunethrombocytopenia
AT briggsandrew costconsequencemodelcomparingeltrombopagandromiplostiminpediatricpatientswithchronicimmunethrombocytopenia