Propensity Score Weighting Using Overlap Weights: A New Method
Applied to Regorafenib Clinical Data and a Cost-Effectiveness
Analysis

J 2019

Propensity Score Weighting Using Overlap Weights: A New Method Applied to Regorafenib Clinical Data and a Cost-Effectiveness Analysis

MLCOCH, Tomas, Tereza HRNCIAROVA, Jan TUZIL, Jakub ZADAK, Marisca MARIAN et. al.

Základní údaje

Originální název

Propensity Score Weighting Using Overlap Weights: A New Method Applied to Regorafenib Clinical Data and a Cost-Effectiveness Analysis

Autoři

MLCOCH, Tomas (203 Česká republika, garant), Tereza HRNCIAROVA (203 Česká republika), Jan TUZIL (203 Česká republika), Jakub ZADAK (203 Česká republika), Marisca MARIAN (756 Švýcarsko) a Tomáš DOLEŽAL (203 Česká republika, domácí)

Vydání

Value in health, NEW YORK, ELSEVIER SCIENCE INC, 2019, 1098-3015

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30104 Pharmacology and pharmacy

Stát vydavatele

Spojené státy

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 4.748

Kód RIV

RIV/00216224:14110/19:00112595

Organizační jednotka

Lékařská fakulta

DOI

http://dx.doi.org/10.1016/j.jval.2019.06.010

UT WoS

000500754600004

Klíčová slova anglicky

colorectal cancer; cost-effectiveness analysis; overlap weights; propensity score; propensity score weighting via overlap weights; regorafenib

Štítky

14110516, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 20. 1. 2020 08:20, Mgr. Tereza Miškechová

Anotace

V originále

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.

Citovat

MLCOCH, Tomas, Tereza HRNCIAROVA, Jan TUZIL, Jakub ZADAK, Marisca MARIAN a Tomáš DOLEŽAL. Propensity Score Weighting Using Overlap Weights: A New Method Applied to Regorafenib Clinical Data and a Cost-Effectiveness Analysis. Value in health. NEW YORK: ELSEVIER SCIENCE INC, 2019, roč. 22, č. 12, s. 1370-1377. ISSN 1098-3015. Dostupné z: https://dx.doi.org/10.1016/j.jval.2019.06.010.

@article{1606856,
   author = {Mlcoch, Tomas and Hrnciarova, Tereza and Tuzil, Jan and Zadak, Jakub and Marian, Marisca and Doležal, Tomáš},
   article_location = {NEW YORK},
   article_number = {12},
   doi = {http://dx.doi.org/10.1016/j.jval.2019.06.010},
   keywords = {colorectal cancer; cost-effectiveness analysis; overlap weights; propensity score; propensity score weighting via overlap weights; regorafenib},
   language = {eng},
   issn = {1098-3015},
   journal = {Value in health},
   title = {Propensity Score Weighting Using Overlap Weights: A New Method Applied to Regorafenib Clinical Data and a Cost-Effectiveness Analysis},
   url = {http://dx.doi.org/10.1016/j.jval.2019.06.010},
   volume = {22},
   year = {2019}
}

TY  - JOUR
ID  - 1606856
AU  - Mlcoch, Tomas - Hrnciarova, Tereza - Tuzil, Jan - Zadak, Jakub - Marian, Marisca - Doležal, Tomáš
PY  - 2019
TI  - Propensity Score Weighting Using Overlap Weights: A New Method Applied to Regorafenib Clinical Data and a Cost-Effectiveness Analysis
JF  - Value in health
VL  - 22
IS  - 12
SP  - 1370-1377
EP  - 1370-1377
PB  - ELSEVIER SCIENCE INC
SN  - 10983015
KW  - colorectal cancer
KW  - cost-effectiveness analysis
KW  - overlap weights
KW  - propensity score
KW  - propensity score weighting via overlap weights
KW  - regorafenib
UR  - http://dx.doi.org/10.1016/j.jval.2019.06.010
L2  - http://dx.doi.org/10.1016/j.jval.2019.06.010
N2  - 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.
ER  -

MLCOCH, Tomas, Tereza HRNCIAROVA, Jan TUZIL, Jakub ZADAK, Marisca MARIAN a Tomáš DOLEŽAL. Propensity Score Weighting Using Overlap Weights: A New Method Applied to Regorafenib Clinical Data and a Cost-Effectiveness Analysis. \textit{Value in health}. NEW YORK: ELSEVIER SCIENCE INC, 2019, roč.~22, č.~12, s.~1370-1377. ISSN~1098-3015. Dostupné z: https://dx.doi.org/10.1016/j.jval.2019.06.010.

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