-
Je něco špatně v tomto záznamu ?
Propensity Score Weighting Using Overlap Weights: A New Method Applied to Regorafenib Clinical Data and a Cost-Effectiveness Analysis
T. Mlcoch, T. Hrnciarova, J. Tuzil, J. Zadak, M. Marian, T. Dolezal,
Jazyk angličtina Země Spojené státy americké
Typ dokumentu hodnotící studie, časopisecké články, práce podpořená grantem
- MeSH
- analýza nákladů a výnosů MeSH
- doba přežití bez progrese choroby MeSH
- fenylmočovinové sloučeniny ekonomika terapeutické užití MeSH
- klinické zkoušky, fáze III jako téma MeSH
- kolorektální nádory farmakoterapie ekonomika mortalita MeSH
- lidé středního věku MeSH
- lidé MeSH
- přežití bez známek nemoci MeSH
- pyridiny ekonomika terapeutické užití MeSH
- randomizované kontrolované studie jako téma MeSH
- registrace MeSH
- tendenční skóre * MeSH
- Check Tag
- lidé středního věku MeSH
- lidé MeSH
- mužské pohlaví MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- hodnotící studie MeSH
- práce podpořená grantem MeSH
BACKGROUND: In situations of markedly different population characteristics and weak population overlap, inverse propensity score (PS) weights suffer from extreme values. The new propensity score weighting method using overlap weights (PSOW) overcomes this limitation by estimating the overlap population at the point of highest mutual overlap, thus may be preferred to other balancing methods (trimming, target, or inverse weights) in some situations. OBJECTIVES: To evaluate the performance of PSOW with regorafenib effectiveness data from previously treated patients with metastatic colorectal cancer based on the Czech national registry data (regorafenib) and a global phase 3 randomized clinical trial (RCT) (placebo). The second goal was to assess the cost-effectiveness of regorafenib versus placebo. METHODS: Individual data on progression-free survival (PFS)/overall survival (OS) were balanced via PSOW for age, sex, Eastern Cooperative Oncology Group performance status, number of treatment lines, metastatic colorectal cancer location, KRAS mutation, and time from metastases estimated using logistic regression. The weighted Kaplan-Meier PFS/OS curves were used in a 3-state partitioned survival model. The R code is provided. RESULTS: In comparison with target or inverse PS weights, PSOW showed remarkable performance measured by effective sample size and PS weight distribution or extreme weights despite the weak overlap between the registry and RCT. In the registry or RCT cohort, regorafenib provided better survival compared with the RCT. The new PSOW hazard ratio for OS was 0.53 (RCT: 0.79), which is conservative compared with inverse or target weights with a hazard ratio of 0.44 and 0.27, respectively. CONCLUSION: This is the first use of PSOW for clinical data and cost-effectiveness analysis. It is promising in cases of weak or small population overlap and makes pharmacoeconomic modeling, in such cases, feasible.
1st Faculty of Medicine Charles University Prague Czech Republic
Bayer Pharmaceuticals Basel Switzerland
Faculty of Medicine Masaryk University Department of Pharmacology Brno Czech Republic
Citace poskytuje Crossref.org
- 000
- 00000naa a2200000 a 4500
- 001
- bmc20023454
- 003
- CZ-PrNML
- 005
- 20201214130100.0
- 007
- ta
- 008
- 201125s2019 xxu f 000 0|eng||
- 009
- AR
- 024 7_
- $a 10.1016/j.jval.2019.06.010 $2 doi
- 035 __
- $a (PubMed)31806193
- 040 __
- $a ABA008 $b cze $d ABA008 $e AACR2
- 041 0_
- $a eng
- 044 __
- $a xxu
- 100 1_
- $a Mlcoch, Tomas $u Value Outcomes, Prague, Czech Republic. Electronic address: tomas.mlcoch@valueoutcomes.cz.
- 245 10
- $a Propensity Score Weighting Using Overlap Weights: A New Method Applied to Regorafenib Clinical Data and a Cost-Effectiveness Analysis / $c T. Mlcoch, T. Hrnciarova, J. Tuzil, J. Zadak, M. Marian, T. Dolezal,
- 520 9_
- $a BACKGROUND: In situations of markedly different population characteristics and weak population overlap, inverse propensity score (PS) weights suffer from extreme values. The new propensity score weighting method using overlap weights (PSOW) overcomes this limitation by estimating the overlap population at the point of highest mutual overlap, thus may be preferred to other balancing methods (trimming, target, or inverse weights) in some situations. OBJECTIVES: To evaluate the performance of PSOW with regorafenib effectiveness data from previously treated patients with metastatic colorectal cancer based on the Czech national registry data (regorafenib) and a global phase 3 randomized clinical trial (RCT) (placebo). The second goal was to assess the cost-effectiveness of regorafenib versus placebo. METHODS: Individual data on progression-free survival (PFS)/overall survival (OS) were balanced via PSOW for age, sex, Eastern Cooperative Oncology Group performance status, number of treatment lines, metastatic colorectal cancer location, KRAS mutation, and time from metastases estimated using logistic regression. The weighted Kaplan-Meier PFS/OS curves were used in a 3-state partitioned survival model. The R code is provided. RESULTS: In comparison with target or inverse PS weights, PSOW showed remarkable performance measured by effective sample size and PS weight distribution or extreme weights despite the weak overlap between the registry and RCT. In the registry or RCT cohort, regorafenib provided better survival compared with the RCT. The new PSOW hazard ratio for OS was 0.53 (RCT: 0.79), which is conservative compared with inverse or target weights with a hazard ratio of 0.44 and 0.27, respectively. CONCLUSION: This is the first use of PSOW for clinical data and cost-effectiveness analysis. It is promising in cases of weak or small population overlap and makes pharmacoeconomic modeling, in such cases, feasible.
- 650 _2
- $a klinické zkoušky, fáze III jako téma $7 D017326
- 650 _2
- $a kolorektální nádory $x farmakoterapie $x ekonomika $x mortalita $7 D015179
- 650 _2
- $a analýza nákladů a výnosů $7 D003362
- 650 _2
- $a přežití bez známek nemoci $7 D018572
- 650 _2
- $a ženské pohlaví $7 D005260
- 650 _2
- $a lidé $7 D006801
- 650 _2
- $a mužské pohlaví $7 D008297
- 650 _2
- $a lidé středního věku $7 D008875
- 650 _2
- $a fenylmočovinové sloučeniny $x ekonomika $x terapeutické užití $7 D010671
- 650 _2
- $a doba přežití bez progrese choroby $7 D000077982
- 650 12
- $a tendenční skóre $7 D057216
- 650 _2
- $a pyridiny $x ekonomika $x terapeutické užití $7 D011725
- 650 _2
- $a randomizované kontrolované studie jako téma $7 D016032
- 650 _2
- $a registrace $7 D012042
- 655 _2
- $a hodnotící studie $7 D023362
- 655 _2
- $a časopisecké články $7 D016428
- 655 _2
- $a práce podpořená grantem $7 D013485
- 700 1_
- $a Hrnciarova, Tereza $u Value Outcomes, Prague, Czech Republic; First Faculty of Medicine, Charles University, Prague, Czech Republic.
- 700 1_
- $a Tuzil, Jan $u Value Outcomes, Prague, Czech Republic; First Faculty of Medicine, Charles University, Prague, Czech Republic.
- 700 1_
- $a Zadak, Jakub $u Bayer, Prague, Czech Republic.
- 700 1_
- $a Marian, Marisca $u Bayer Pharmaceuticals, Basel, Switzerland.
- 700 1_
- $a Dolezal, Tomas $u Value Outcomes, Prague, Czech Republic; Faculty of Medicine, Masaryk University, Department of Pharmacology, Brno, Czech Republic.
- 773 0_
- $w MED00007383 $t Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research $x 1524-4733 $g Roč. 22, č. 12 (2019), s. 1370-1377
- 856 41
- $u https://pubmed.ncbi.nlm.nih.gov/31806193 $y Pubmed
- 910 __
- $a ABA008 $b sig $c sign $y a $z 0
- 990 __
- $a 20201125 $b ABA008
- 991 __
- $a 20201214130059 $b ABA008
- 999 __
- $a ok $b bmc $g 1595773 $s 1114130
- BAS __
- $a 3
- BAS __
- $a PreBMC
- BMC __
- $a 2019 $b 22 $c 12 $d 1370-1377 $e 20190820 $i 1524-4733 $m Value in health $n Value Health $x MED00007383
- LZP __
- $a Pubmed-20201125
