Pharmacokinetic parameter driven outcomes model predicts a reduction in bleeding events associated with BAY 81-8973 versus antihemophilic factor (recombinant) plasma/albumin-free method in a Chinese healthcare setting.
BAY 81–8973
China
Economic model
Factor VIII products
Hemophilia A
Pharmacokinetic
rAHF-PFM
Journal
BMC medical research methodology
ISSN: 1471-2288
Titre abrégé: BMC Med Res Methodol
Pays: England
ID NLM: 100968545
Informations de publication
Date de publication:
05 08 2022
05 08 2022
Historique:
received:
10
12
2021
accepted:
23
05
2022
entrez:
5
8
2022
pubmed:
6
8
2022
medline:
10
8
2022
Statut:
epublish
Résumé
Long-term prophylactic therapy is considered the standard of care for hemophilia A patients. This study models the long-term clinical and cost outcomes of two factor VIII (FVIII) products using a pharmacokinetic (PK) simulation model in a Chinese population. Head-to-head PK profile data of BAY 81-8973 (KOVALTRY®) and antihemophilic factor (recombinant) plasma/albumin-free method (rAHF-PFM, ADVATE®) were applied to a two-state (alive and dead) Markov model to simulate blood FVIII concentrations at a steady state in prophylactically-treated patients with hemophilia A. Worsening of the Pettersson score was simulated and decline was associated with the probability of having orthopaedic surgery. The only difference between the compounds was FVIII concentration at a given time; each subject was treated with 25 IU/kg every 3 days. The model used a lifetime horizon, with cycle lengths of 1 year. Cumulative bleeding events, joint bleeding events, and major bleeding events were reduced by 19.3% for BAY 81-8973 compared to rAHF-PFM. Hospitalizations and hospitalization days were also reduced by 19.3% for BAY 81-8973 compared to rAHF-PFM. BAY 81-8973 resulted in both cost savings and a gain in quality adjusted life years (QALYs) compared to rAHF-PFM. Based on modeled head-to-head comparisons, differences in PK-properties between BAY 81-8973 and rAHF-PFM result in a reduced number of bleeding events, leading to reduced costs and increased quality of life for BAY 81-8973. These results should be used to inform clinical practice in China when caring for patients with severe hemophilia A.
Sections du résumé
BACKGROUND
Long-term prophylactic therapy is considered the standard of care for hemophilia A patients. This study models the long-term clinical and cost outcomes of two factor VIII (FVIII) products using a pharmacokinetic (PK) simulation model in a Chinese population.
METHODS
Head-to-head PK profile data of BAY 81-8973 (KOVALTRY®) and antihemophilic factor (recombinant) plasma/albumin-free method (rAHF-PFM, ADVATE®) were applied to a two-state (alive and dead) Markov model to simulate blood FVIII concentrations at a steady state in prophylactically-treated patients with hemophilia A. Worsening of the Pettersson score was simulated and decline was associated with the probability of having orthopaedic surgery. The only difference between the compounds was FVIII concentration at a given time; each subject was treated with 25 IU/kg every 3 days. The model used a lifetime horizon, with cycle lengths of 1 year.
RESULTS
Cumulative bleeding events, joint bleeding events, and major bleeding events were reduced by 19.3% for BAY 81-8973 compared to rAHF-PFM. Hospitalizations and hospitalization days were also reduced by 19.3% for BAY 81-8973 compared to rAHF-PFM. BAY 81-8973 resulted in both cost savings and a gain in quality adjusted life years (QALYs) compared to rAHF-PFM.
CONCLUSION
Based on modeled head-to-head comparisons, differences in PK-properties between BAY 81-8973 and rAHF-PFM result in a reduced number of bleeding events, leading to reduced costs and increased quality of life for BAY 81-8973. These results should be used to inform clinical practice in China when caring for patients with severe hemophilia A.
Identifiants
pubmed: 35931967
doi: 10.1186/s12874-022-01659-w
pii: 10.1186/s12874-022-01659-w
pmc: PMC9356410
doi:
Substances chimiques
Recombinant Proteins
0
Serum Albumin
0
F8 protein, human
839MOZ74GK
Factor VIII
9001-27-8
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
215Informations de copyright
© 2022. The Author(s).
Références
Value Health. 2017 Sep;20(8):1074-1082
pubmed: 28964439
J Manag Care Spec Pharm. 2021 May;27(5):667-673
pubmed: 33908280
Clin Pharmacokinet. 2017 Sep;56(9):1045-1055
pubmed: 28005225
Acta Paediatr Scand. 1981 Jul;70(4):565-70
pubmed: 6797235
Haematologica. 2020 May;105(5):1443-1453
pubmed: 31371418
Haemophilia. 2017 Jan;23(1):11-24
pubmed: 27633342
Acta Radiol. 2002 Sep;43(5):528-32
pubmed: 12423465
Zhonghua Xue Ye Xue Za Zhi. 2020 Apr 14;41(4):265-271
pubmed: 32295333
Southeast Asian J Trop Med Public Health. 2014 Mar;45(2):455-66
pubmed: 24968688
Curr Med Res Opin. 2008 Mar;24(3):753-68
pubmed: 18234151
Haemophilia. 2021 Mar;27(2):e287-e290
pubmed: 32997865
Haemophilia. 2020 Jul;26(4):584-590
pubmed: 32432832
Haematologica. 2011 May;96(5):738-43
pubmed: 21273268
Haemophilia. 2020 Aug;26 Suppl 6:1-158
pubmed: 32744769
Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):595-604
pubmed: 29222309
Zhonghua Xue Ye Xue Za Zhi. 2017 May 14;38(5):364-370
pubmed: 28565732
Haemophilia. 2016 Sep;22(5):706-12
pubmed: 27339736
Haemophilia. 2009 Jul;15(4):888-93
pubmed: 19473415
Cochrane Database Syst Rev. 2011 Sep 07;(9):CD003429
pubmed: 21901684
Haemophilia. 2020 Jan;26(1):129-135
pubmed: 31657079
Pharmacoeconomics. 2002;20(11):759-74
pubmed: 12201795
Haemophilia. 2016 Nov;22(6):833-840
pubmed: 27785891
Haemophilia. 2019 May;25(3):e215-e218
pubmed: 30866068
Haemophilia. 2017 Jan;23(1):89-97
pubmed: 27599642
Haemophilia. 2015 Nov;21(6):766-71
pubmed: 25952661