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
Abstract 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...
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| Format: | Article |
| Language: | English |
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BMC
2022-08-01
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| Series: | BMC Medical Research Methodology |
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| Online Access: | https://doi.org/10.1186/s12874-022-01659-w |
| _version_ | 1798037954842591232 |
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| author | Rong Chen Dmitry Gultyaev Johanna Lister Rong Han Nan Hu Jean Malacan Alexander Solms Parth Vashi Jamie O’Hara Shanlian Hu |
| author_facet | Rong Chen Dmitry Gultyaev Johanna Lister Rong Han Nan Hu Jean Malacan Alexander Solms Parth Vashi Jamie O’Hara Shanlian Hu |
| author_sort | Rong Chen |
| collection | DOAJ |
| description | Abstract 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. |
| first_indexed | 2024-04-11T21:33:37Z |
| format | Article |
| id | doaj.art-07dc3095af494e4fb75c8fa461ae1d26 |
| institution | Directory Open Access Journal |
| issn | 1471-2288 |
| language | English |
| last_indexed | 2024-04-11T21:33:37Z |
| publishDate | 2022-08-01 |
| publisher | BMC |
| record_format | Article |
| series | BMC Medical Research Methodology |
| spelling | doaj.art-07dc3095af494e4fb75c8fa461ae1d262022-12-22T04:01:50ZengBMCBMC Medical Research Methodology1471-22882022-08-012211910.1186/s12874-022-01659-wPharmacokinetic 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 settingRong Chen0Dmitry Gultyaev1Johanna Lister2Rong Han3Nan Hu4Jean Malacan5Alexander Solms6Parth Vashi7Jamie O’Hara8Shanlian Hu9Hematology Department, Chengdu Third People’s HospitalCertara USA, IncFormerly Certara USA, IncMedical Affairs, Pharmaceuticals, Bayer Healthcare Company. LtdMedical Affairs, Pharmaceuticals, Bayer Healthcare Company. LtdGlobal Market Access Hematology, Bayer Consumer Care AGClinical Pharmacometrics, Bayer AGFormerly at US Data Generation and Observational Studies, Bayer CorporationHCD EconomicsSchool of Public Health, Fudan UniversityAbstract 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.https://doi.org/10.1186/s12874-022-01659-wHemophilia AFactor VIII productsPharmacokineticChinaEconomic modelBAY 81–8973 |
| spellingShingle | Rong Chen Dmitry Gultyaev Johanna Lister Rong Han Nan Hu Jean Malacan Alexander Solms Parth Vashi Jamie O’Hara Shanlian Hu 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 BMC Medical Research Methodology Hemophilia A Factor VIII products Pharmacokinetic China Economic model BAY 81–8973 |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| topic | Hemophilia A Factor VIII products Pharmacokinetic China Economic model BAY 81–8973 |
| url | https://doi.org/10.1186/s12874-022-01659-w |
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