Evidence-based sizing of non-inferiority trials using decision models

Background There are significant challenges to the successful conduct of non-inferiority trials because they require large numbers to demonstrate that an alternative intervention is “not too much worse” than the standard. In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity. Methods We use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention ($ p_{S} $) and systematically varying the incidence of the outcome in the alternative intervention ($ p_{A} $). The non-inferiority margin, $ p_{A} $ – $ p_{S} $ = δ, is reached when the lower event rate of the standard therapy counterbalances the higher event rate but improved morbidity burden of the alternative. We consider the appropriate non-inferiority margin as the tipping point at which the quality-adjusted life-years saved in the two arms are equal. Results Using the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies. Conclusions The example demonstrates that applying our proposed method appears feasible in real-world settings and offers the benefits of more explicit and rigorous quantification of the various considerations relevant for determining a non-inferiority margin and associated trial sample size. Ausführliche Beschreibung

Gespeichert in:

Autor*in:	Lansdorp-Vogelaar, Iris [verfasserIn] Jagsi, Reshma Jayasekera, Jinani Stout, Natasha K. Mitchell, Sandra A. Feuer, Eric J.

Format:	E-Artikel
Sprache:	Englisch

Erschienen:	2019

Schlagwörter:	Non-inferiority trial Sample size Power calculation Quality-adjusted life years Non-inferiority margin

Anmerkung:	© The Author(s). 2019

Übergeordnetes Werk:	Enthalten in: BMC medical research methodology - London : BioMed Central, 2001, 19(2019), 1 vom: 07. Jan.
Übergeordnetes Werk:	volume:19 ; year:2019 ; number:1 ; day:07 ; month:01

Links:	Volltext

DOI / URN:	10.1186/s12874-018-0643-2

Katalog-ID:	SPR027375854

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520			\|a Background There are significant challenges to the successful conduct of non-inferiority trials because they require large numbers to demonstrate that an alternative intervention is “not too much worse” than the standard. In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity. Methods We use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention ($ p_{S} $) and systematically varying the incidence of the outcome in the alternative intervention ($ p_{A} $). The non-inferiority margin, $ p_{A} $ – $ p_{S} $ = δ, is reached when the lower event rate of the standard therapy counterbalances the higher event rate but improved morbidity burden of the alternative. We consider the appropriate non-inferiority margin as the tipping point at which the quality-adjusted life-years saved in the two arms are equal. Results Using the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies. Conclusions The example demonstrates that applying our proposed method appears feasible in real-world settings and offers the benefits of more explicit and rigorous quantification of the various considerations relevant for determining a non-inferiority margin and associated trial sample size.
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allfields	10.1186/s12874-018-0643-2 doi (DE-627)SPR027375854 (SPR)s12874-018-0643-2-e DE-627 ger DE-627 rakwb eng Lansdorp-Vogelaar, Iris verfasserin aut Evidence-based sizing of non-inferiority trials using decision models 2019 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © The Author(s). 2019 Background There are significant challenges to the successful conduct of non-inferiority trials because they require large numbers to demonstrate that an alternative intervention is “not too much worse” than the standard. In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity. Methods We use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention ($ p_{S} $) and systematically varying the incidence of the outcome in the alternative intervention ($ p_{A} $). The non-inferiority margin, $ p_{A} $ – $ p_{S} $ = δ, is reached when the lower event rate of the standard therapy counterbalances the higher event rate but improved morbidity burden of the alternative. We consider the appropriate non-inferiority margin as the tipping point at which the quality-adjusted life-years saved in the two arms are equal. Results Using the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies. Conclusions The example demonstrates that applying our proposed method appears feasible in real-world settings and offers the benefits of more explicit and rigorous quantification of the various considerations relevant for determining a non-inferiority margin and associated trial sample size. Non-inferiority trial (dpeaa)DE-He213 Sample size (dpeaa)DE-He213 Power calculation (dpeaa)DE-He213 Quality-adjusted life years (dpeaa)DE-He213 Non-inferiority margin (dpeaa)DE-He213 Jagsi, Reshma aut Jayasekera, Jinani aut Stout, Natasha K. aut Mitchell, Sandra A. aut Feuer, Eric J. (orcid)0000-0003-4842-7751 aut Enthalten in BMC medical research methodology London : BioMed Central, 2001 19(2019), 1 vom: 07. Jan. (DE-627)326643818 (DE-600)2041362-2 1471-2288 nnns volume:19 year:2019 number:1 day:07 month:01 https://dx.doi.org/10.1186/s12874-018-0643-2 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_702 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2031 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2061 GBV_ILN_2111 GBV_ILN_2113 GBV_ILN_2190 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 19 2019 1 07 01
spelling	10.1186/s12874-018-0643-2 doi (DE-627)SPR027375854 (SPR)s12874-018-0643-2-e DE-627 ger DE-627 rakwb eng Lansdorp-Vogelaar, Iris verfasserin aut Evidence-based sizing of non-inferiority trials using decision models 2019 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © The Author(s). 2019 Background There are significant challenges to the successful conduct of non-inferiority trials because they require large numbers to demonstrate that an alternative intervention is “not too much worse” than the standard. In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity. Methods We use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention ($ p_{S} $) and systematically varying the incidence of the outcome in the alternative intervention ($ p_{A} $). The non-inferiority margin, $ p_{A} $ – $ p_{S} $ = δ, is reached when the lower event rate of the standard therapy counterbalances the higher event rate but improved morbidity burden of the alternative. We consider the appropriate non-inferiority margin as the tipping point at which the quality-adjusted life-years saved in the two arms are equal. Results Using the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies. Conclusions The example demonstrates that applying our proposed method appears feasible in real-world settings and offers the benefits of more explicit and rigorous quantification of the various considerations relevant for determining a non-inferiority margin and associated trial sample size. Non-inferiority trial (dpeaa)DE-He213 Sample size (dpeaa)DE-He213 Power calculation (dpeaa)DE-He213 Quality-adjusted life years (dpeaa)DE-He213 Non-inferiority margin (dpeaa)DE-He213 Jagsi, Reshma aut Jayasekera, Jinani aut Stout, Natasha K. aut Mitchell, Sandra A. aut Feuer, Eric J. (orcid)0000-0003-4842-7751 aut Enthalten in BMC medical research methodology London : BioMed Central, 2001 19(2019), 1 vom: 07. Jan. (DE-627)326643818 (DE-600)2041362-2 1471-2288 nnns volume:19 year:2019 number:1 day:07 month:01 https://dx.doi.org/10.1186/s12874-018-0643-2 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_702 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2031 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2061 GBV_ILN_2111 GBV_ILN_2113 GBV_ILN_2190 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 19 2019 1 07 01
allfields_unstemmed	10.1186/s12874-018-0643-2 doi (DE-627)SPR027375854 (SPR)s12874-018-0643-2-e DE-627 ger DE-627 rakwb eng Lansdorp-Vogelaar, Iris verfasserin aut Evidence-based sizing of non-inferiority trials using decision models 2019 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © The Author(s). 2019 Background There are significant challenges to the successful conduct of non-inferiority trials because they require large numbers to demonstrate that an alternative intervention is “not too much worse” than the standard. In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity. Methods We use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention ($ p_{S} $) and systematically varying the incidence of the outcome in the alternative intervention ($ p_{A} $). The non-inferiority margin, $ p_{A} $ – $ p_{S} $ = δ, is reached when the lower event rate of the standard therapy counterbalances the higher event rate but improved morbidity burden of the alternative. We consider the appropriate non-inferiority margin as the tipping point at which the quality-adjusted life-years saved in the two arms are equal. Results Using the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies. Conclusions The example demonstrates that applying our proposed method appears feasible in real-world settings and offers the benefits of more explicit and rigorous quantification of the various considerations relevant for determining a non-inferiority margin and associated trial sample size. Non-inferiority trial (dpeaa)DE-He213 Sample size (dpeaa)DE-He213 Power calculation (dpeaa)DE-He213 Quality-adjusted life years (dpeaa)DE-He213 Non-inferiority margin (dpeaa)DE-He213 Jagsi, Reshma aut Jayasekera, Jinani aut Stout, Natasha K. aut Mitchell, Sandra A. aut Feuer, Eric J. (orcid)0000-0003-4842-7751 aut Enthalten in BMC medical research methodology London : BioMed Central, 2001 19(2019), 1 vom: 07. Jan. (DE-627)326643818 (DE-600)2041362-2 1471-2288 nnns volume:19 year:2019 number:1 day:07 month:01 https://dx.doi.org/10.1186/s12874-018-0643-2 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_702 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2031 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2061 GBV_ILN_2111 GBV_ILN_2113 GBV_ILN_2190 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 19 2019 1 07 01
allfieldsGer	10.1186/s12874-018-0643-2 doi (DE-627)SPR027375854 (SPR)s12874-018-0643-2-e DE-627 ger DE-627 rakwb eng Lansdorp-Vogelaar, Iris verfasserin aut Evidence-based sizing of non-inferiority trials using decision models 2019 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © The Author(s). 2019 Background There are significant challenges to the successful conduct of non-inferiority trials because they require large numbers to demonstrate that an alternative intervention is “not too much worse” than the standard. In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity. Methods We use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention ($ p_{S} $) and systematically varying the incidence of the outcome in the alternative intervention ($ p_{A} $). The non-inferiority margin, $ p_{A} $ – $ p_{S} $ = δ, is reached when the lower event rate of the standard therapy counterbalances the higher event rate but improved morbidity burden of the alternative. We consider the appropriate non-inferiority margin as the tipping point at which the quality-adjusted life-years saved in the two arms are equal. Results Using the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies. Conclusions The example demonstrates that applying our proposed method appears feasible in real-world settings and offers the benefits of more explicit and rigorous quantification of the various considerations relevant for determining a non-inferiority margin and associated trial sample size. Non-inferiority trial (dpeaa)DE-He213 Sample size (dpeaa)DE-He213 Power calculation (dpeaa)DE-He213 Quality-adjusted life years (dpeaa)DE-He213 Non-inferiority margin (dpeaa)DE-He213 Jagsi, Reshma aut Jayasekera, Jinani aut Stout, Natasha K. aut Mitchell, Sandra A. aut Feuer, Eric J. (orcid)0000-0003-4842-7751 aut Enthalten in BMC medical research methodology London : BioMed Central, 2001 19(2019), 1 vom: 07. Jan. (DE-627)326643818 (DE-600)2041362-2 1471-2288 nnns volume:19 year:2019 number:1 day:07 month:01 https://dx.doi.org/10.1186/s12874-018-0643-2 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_702 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2031 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2061 GBV_ILN_2111 GBV_ILN_2113 GBV_ILN_2190 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 19 2019 1 07 01
allfieldsSound	10.1186/s12874-018-0643-2 doi (DE-627)SPR027375854 (SPR)s12874-018-0643-2-e DE-627 ger DE-627 rakwb eng Lansdorp-Vogelaar, Iris verfasserin aut Evidence-based sizing of non-inferiority trials using decision models 2019 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © The Author(s). 2019 Background There are significant challenges to the successful conduct of non-inferiority trials because they require large numbers to demonstrate that an alternative intervention is “not too much worse” than the standard. In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity. Methods We use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention ($ p_{S} $) and systematically varying the incidence of the outcome in the alternative intervention ($ p_{A} $). The non-inferiority margin, $ p_{A} $ – $ p_{S} $ = δ, is reached when the lower event rate of the standard therapy counterbalances the higher event rate but improved morbidity burden of the alternative. We consider the appropriate non-inferiority margin as the tipping point at which the quality-adjusted life-years saved in the two arms are equal. Results Using the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies. Conclusions The example demonstrates that applying our proposed method appears feasible in real-world settings and offers the benefits of more explicit and rigorous quantification of the various considerations relevant for determining a non-inferiority margin and associated trial sample size. Non-inferiority trial (dpeaa)DE-He213 Sample size (dpeaa)DE-He213 Power calculation (dpeaa)DE-He213 Quality-adjusted life years (dpeaa)DE-He213 Non-inferiority margin (dpeaa)DE-He213 Jagsi, Reshma aut Jayasekera, Jinani aut Stout, Natasha K. aut Mitchell, Sandra A. aut Feuer, Eric J. (orcid)0000-0003-4842-7751 aut Enthalten in BMC medical research methodology London : BioMed Central, 2001 19(2019), 1 vom: 07. Jan. (DE-627)326643818 (DE-600)2041362-2 1471-2288 nnns volume:19 year:2019 number:1 day:07 month:01 https://dx.doi.org/10.1186/s12874-018-0643-2 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_702 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2031 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2061 GBV_ILN_2111 GBV_ILN_2113 GBV_ILN_2190 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 19 2019 1 07 01
language	English
source	Enthalten in BMC medical research methodology 19(2019), 1 vom: 07. Jan. volume:19 year:2019 number:1 day:07 month:01
sourceStr	Enthalten in BMC medical research methodology 19(2019), 1 vom: 07. Jan. volume:19 year:2019 number:1 day:07 month:01
format_phy_str_mv	Article
institution	findex.gbv.de
topic_facet	Non-inferiority trial Sample size Power calculation Quality-adjusted life years Non-inferiority margin
isfreeaccess_bool	true
container_title	BMC medical research methodology
authorswithroles_txt_mv	Lansdorp-Vogelaar, Iris @@aut@@ Jagsi, Reshma @@aut@@ Jayasekera, Jinani @@aut@@ Stout, Natasha K. @@aut@@ Mitchell, Sandra A. @@aut@@ Feuer, Eric J. @@aut@@
publishDateDaySort_date	2019-01-07T00:00:00Z
hierarchy_top_id	326643818
id	SPR027375854
language_de	englisch
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In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity. Methods We use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention ($ p_{S} $) and systematically varying the incidence of the outcome in the alternative intervention ($ p_{A} $). The non-inferiority margin, $ p_{A} $ – $ p_{S} $ = δ, is reached when the lower event rate of the standard therapy counterbalances the higher event rate but improved morbidity burden of the alternative. We consider the appropriate non-inferiority margin as the tipping point at which the quality-adjusted life-years saved in the two arms are equal. Results Using the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies. 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author	Lansdorp-Vogelaar, Iris
spellingShingle	Lansdorp-Vogelaar, Iris misc Non-inferiority trial misc Sample size misc Power calculation misc Quality-adjusted life years misc Non-inferiority margin Evidence-based sizing of non-inferiority trials using decision models
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topic_title	Evidence-based sizing of non-inferiority trials using decision models Non-inferiority trial (dpeaa)DE-He213 Sample size (dpeaa)DE-He213 Power calculation (dpeaa)DE-He213 Quality-adjusted life years (dpeaa)DE-He213 Non-inferiority margin (dpeaa)DE-He213
topic	misc Non-inferiority trial misc Sample size misc Power calculation misc Quality-adjusted life years misc Non-inferiority margin
topic_unstemmed	misc Non-inferiority trial misc Sample size misc Power calculation misc Quality-adjusted life years misc Non-inferiority margin
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title	Evidence-based sizing of non-inferiority trials using decision models
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title_full	Evidence-based sizing of non-inferiority trials using decision models
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author_browse	Lansdorp-Vogelaar, Iris Jagsi, Reshma Jayasekera, Jinani Stout, Natasha K. Mitchell, Sandra A. Feuer, Eric J.
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title_sort	evidence-based sizing of non-inferiority trials using decision models
title_auth	Evidence-based sizing of non-inferiority trials using decision models
abstract	Background There are significant challenges to the successful conduct of non-inferiority trials because they require large numbers to demonstrate that an alternative intervention is “not too much worse” than the standard. In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity. Methods We use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention ($ p_{S} $) and systematically varying the incidence of the outcome in the alternative intervention ($ p_{A} $). The non-inferiority margin, $ p_{A} $ – $ p_{S} $ = δ, is reached when the lower event rate of the standard therapy counterbalances the higher event rate but improved morbidity burden of the alternative. We consider the appropriate non-inferiority margin as the tipping point at which the quality-adjusted life-years saved in the two arms are equal. Results Using the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies. Conclusions The example demonstrates that applying our proposed method appears feasible in real-world settings and offers the benefits of more explicit and rigorous quantification of the various considerations relevant for determining a non-inferiority margin and associated trial sample size. © The Author(s). 2019
abstractGer	Background There are significant challenges to the successful conduct of non-inferiority trials because they require large numbers to demonstrate that an alternative intervention is “not too much worse” than the standard. In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity. Methods We use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention ($ p_{S} $) and systematically varying the incidence of the outcome in the alternative intervention ($ p_{A} $). The non-inferiority margin, $ p_{A} $ – $ p_{S} $ = δ, is reached when the lower event rate of the standard therapy counterbalances the higher event rate but improved morbidity burden of the alternative. We consider the appropriate non-inferiority margin as the tipping point at which the quality-adjusted life-years saved in the two arms are equal. Results Using the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies. Conclusions The example demonstrates that applying our proposed method appears feasible in real-world settings and offers the benefits of more explicit and rigorous quantification of the various considerations relevant for determining a non-inferiority margin and associated trial sample size. © The Author(s). 2019
abstract_unstemmed	Background There are significant challenges to the successful conduct of non-inferiority trials because they require large numbers to demonstrate that an alternative intervention is “not too much worse” than the standard. In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity. Methods We use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention ($ p_{S} $) and systematically varying the incidence of the outcome in the alternative intervention ($ p_{A} $). The non-inferiority margin, $ p_{A} $ – $ p_{S} $ = δ, is reached when the lower event rate of the standard therapy counterbalances the higher event rate but improved morbidity burden of the alternative. We consider the appropriate non-inferiority margin as the tipping point at which the quality-adjusted life-years saved in the two arms are equal. Results Using the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies. Conclusions The example demonstrates that applying our proposed method appears feasible in real-world settings and offers the benefits of more explicit and rigorous quantification of the various considerations relevant for determining a non-inferiority margin and associated trial sample size. © The Author(s). 2019
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Evidence-based sizing of non-inferiority trials using decision models

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