Valuing Health Gain from Composite Response Endpoints for Multisystem Diseases

MASTERPLANS Consortium

doi:10.1016/j.jval.2022.07.001

Valuing Health Gain from Composite Response Endpoints for Multisystem Diseases

MASTERPLANS Consortium

Research output: Contribution to journal › Article › peer-review

Abstract

Objectives: This study aimed to demonstrate how to estimate the value of health gain after patients with a multisystem disease achieve a condition-specific composite response endpoint. Methods: Data from patients treated in routine practice with an exemplar multisystem disease (systemic lupus erythematosus) were extracted from a national register (British Isles Lupus Assessment Group Biologics Register). Two bespoke composite response endpoints (Major Clinical Response and Improvement) were developed in advance of this study. Difference-in-differences regression compared health utility values (3-level version of EQ-5D; UK tariff) over 6 months for responders and nonresponders. Bootstrapped regression estimated the incremental quality-adjusted life-years (QALYs), probability of QALY gain after achieving the response criteria, and population monetary benefit of response. Results: Within the sample (n = 171), 18.2% achieved Major Clinical Response and 49.1% achieved Improvement at 6 months. Incremental health utility values were 0.0923 for Major Clinical Response and 0.0454 for Improvement. Expected incremental QALY gain at 6 months was 0.020 for Major Clinical Response and 0.012 for Improvement. Probability of QALY gain after achieving the response criteria was 77.6% for Major Clinical Response and 72.7% for Improvement. Population monetary benefit of response was £1 106 458 for Major Clinical Response and £649 134 for Improvement. Conclusions: Bespoke composite response endpoints are becoming more common to measure treatment response for multisystem diseases in trials and observational studies. Health technology assessment agencies face a growing challenge to establish whether these endpoints correspond with improved health gain. Health utility values can generate this evidence to enhance the usefulness of composite response endpoints for health technology assessment, decision making, and economic evaluation.

Original language	English
Pages (from-to)	115-122
Number of pages	8
Journal	Value in Health
Volume	26
Issue number	1
Early online date	23 Aug 2022
DOIs	https://doi.org/10.1016/j.jval.2022.07.001
Publication status	Published - 31 Jan 2023

Bibliographical note

Funding Information:
Funding/Support: This research was funded by the Medical Research Council, United Kingdom as part of the MAximizing Sle ThERapeutic PotentiaL by Application of Novel and Stratified approaches (MASTERPLANS) Consortium (MR/M01665X/1) and part funded by a grant from LUPUS UK. BILAG-BR has been funded by unrestricted educational donations from Roche, GSK, and LUPUS UK.

Funding Information:
Previous studies have compared responders and nonresponders with existing composite response endpoints for SLE with other health-related quality of life instruments. Furie et al39 estimated that a greater percentage of patients who achieved the BICLA composite response endpoint at 52 weeks, than nonresponding patients, experienced at least a 3-point improvement in the Short-Form 36 (SF-36) Physical Component Summary score (57.9% vs 12.8%) and at least a 4.6-point improvement in the SF-36 Mental Component Summary score (42.6% vs 12.3%) as part of a post hoc analysis of the pooled anifrolumab phase III trial data. Similarly, Furie et al40 used pooled 52-week observations from the belimumab in subjects with systemic lupus erythematosus (BLISS)-52 and BLISS-76 trial data for belimumab and found that patients who achieved the SRI composite response endpoint had a greater improvement in the SF-36 Physical Component Summary (4.9 vs 2.6) and SF-36 Mental Component Summary (4.4 vs 1.7) than nonresponding patients. If there is a positive correlation between the SF-36 and EQ-5D-3L in people with SLE,41 these findings could suggest that existing endpoints may also improve EQ-5D scores. Direct comparative evidence of how existing composite response endpoints perform according to instruments preferred by HTA agencies will be valuable evidence to support future decision making.Funding/Support: This research was funded by the Medical Research Council, United Kingdom as part of the MAximizing Sle ThERapeutic PotentiaL by Application of Novel and Stratified approaches (MASTERPLANS) Consortium (MR/M01665X/1) and part funded by a grant from LUPUS UK. BILAG-BR has been funded by unrestricted educational donations from Roche, GSK, and LUPUS UK.

Funding Information:
Conflict of Interest Disclosures: Dr Gavan reported receiving grants from the Medical Research Council and Lupus UK during the conduct of the study and personal fees from UCB outside the submitted work. Dr Bruce reports receiving grants from the Medical Research Council and the National Institute for Health and Care Research during the conduct of the study, grants and speaker fees from GSK, consultancy and speaker fees from Astra Zeneca, and consultancy fees from UCB, BMS, Eli Lilly, and Aurinia outside the submitted work. Dr Payne reports receiving personal fees from UCB outside the submitted work. No other disclosures were reported.

Keywords

composite response endpoint
health state utility
monetary benefit
multisystem disease
systemic lupus erythematous

ASJC Scopus subject areas

Health Policy
Public Health, Environmental and Occupational Health

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1016/j.jval.2022.07.001Licence: CC BY

Cite this

@article{57128745fa474a9a951472cda7d09a96,

title = "Valuing Health Gain from Composite Response Endpoints for Multisystem Diseases",

abstract = "Objectives: This study aimed to demonstrate how to estimate the value of health gain after patients with a multisystem disease achieve a condition-specific composite response endpoint. Methods: Data from patients treated in routine practice with an exemplar multisystem disease (systemic lupus erythematosus) were extracted from a national register (British Isles Lupus Assessment Group Biologics Register). Two bespoke composite response endpoints (Major Clinical Response and Improvement) were developed in advance of this study. Difference-in-differences regression compared health utility values (3-level version of EQ-5D; UK tariff) over 6 months for responders and nonresponders. Bootstrapped regression estimated the incremental quality-adjusted life-years (QALYs), probability of QALY gain after achieving the response criteria, and population monetary benefit of response. Results: Within the sample (n = 171), 18.2% achieved Major Clinical Response and 49.1% achieved Improvement at 6 months. Incremental health utility values were 0.0923 for Major Clinical Response and 0.0454 for Improvement. Expected incremental QALY gain at 6 months was 0.020 for Major Clinical Response and 0.012 for Improvement. Probability of QALY gain after achieving the response criteria was 77.6% for Major Clinical Response and 72.7% for Improvement. Population monetary benefit of response was £1 106 458 for Major Clinical Response and £649 134 for Improvement. Conclusions: Bespoke composite response endpoints are becoming more common to measure treatment response for multisystem diseases in trials and observational studies. Health technology assessment agencies face a growing challenge to establish whether these endpoints correspond with improved health gain. Health utility values can generate this evidence to enhance the usefulness of composite response endpoints for health technology assessment, decision making, and economic evaluation.",

keywords = "composite response endpoint, health state utility, monetary benefit, multisystem disease, systemic lupus erythematous",

author = "{MASTERPLANS Consortium} and Gavan, {Sean P.} and Ian Bruce and Katherine Payne and Ian Bruce and Mark Lunt and Niels Peek and Nophar Geifman and Sean Gavan and Gillian Armitt and Patrick Doherty and Jennifer Prattley and Narges Azadbakht and Angela Papazian and {Le Sueur}, Helen and Carmen Farrelly and Clare Richardson and Zunnaira Shabbir and Lauren Hewitt and Neil McHugh and Caroline Gordon and John Reynolds and Stephen Young and David Jayne and Vern Farewell and Li Su and Matthew Pickering and Elizabeth Lightstone and Alyssa Gilmore and Marina Botto and Timothy Vyse and Morris, {David Lester} and David D'Cruz and Edward Vital and Miriam Wittmann and Paul Emery and Michael Beresford and Christian Hedrich and Angela Midgley and Jenna Gritzfeld and Michael Ehrenstein and David Isenberg and Mariea Parvaz and Jane Dunnage and Jane Batchelor and Elaine Holland and Pauline Upsall and Hazem Youssef and Liza McCann and Rapti Mediwake and Anurag Bharadwaj",

note = "Funding Information: Funding/Support: This research was funded by the Medical Research Council, United Kingdom as part of the MAximizing Sle ThERapeutic PotentiaL by Application of Novel and Stratified approaches (MASTERPLANS) Consortium (MR/M01665X/1) and part funded by a grant from LUPUS UK. BILAG-BR has been funded by unrestricted educational donations from Roche, GSK, and LUPUS UK. Funding Information: Previous studies have compared responders and nonresponders with existing composite response endpoints for SLE with other health-related quality of life instruments. Furie et al39 estimated that a greater percentage of patients who achieved the BICLA composite response endpoint at 52 weeks, than nonresponding patients, experienced at least a 3-point improvement in the Short-Form 36 (SF-36) Physical Component Summary score (57.9% vs 12.8%) and at least a 4.6-point improvement in the SF-36 Mental Component Summary score (42.6% vs 12.3%) as part of a post hoc analysis of the pooled anifrolumab phase III trial data. Similarly, Furie et al40 used pooled 52-week observations from the belimumab in subjects with systemic lupus erythematosus (BLISS)-52 and BLISS-76 trial data for belimumab and found that patients who achieved the SRI composite response endpoint had a greater improvement in the SF-36 Physical Component Summary (4.9 vs 2.6) and SF-36 Mental Component Summary (4.4 vs 1.7) than nonresponding patients. If there is a positive correlation between the SF-36 and EQ-5D-3L in people with SLE,41 these findings could suggest that existing endpoints may also improve EQ-5D scores. Direct comparative evidence of how existing composite response endpoints perform according to instruments preferred by HTA agencies will be valuable evidence to support future decision making.Funding/Support: This research was funded by the Medical Research Council, United Kingdom as part of the MAximizing Sle ThERapeutic PotentiaL by Application of Novel and Stratified approaches (MASTERPLANS) Consortium (MR/M01665X/1) and part funded by a grant from LUPUS UK. BILAG-BR has been funded by unrestricted educational donations from Roche, GSK, and LUPUS UK. Funding Information: Conflict of Interest Disclosures: Dr Gavan reported receiving grants from the Medical Research Council and Lupus UK during the conduct of the study and personal fees from UCB outside the submitted work. Dr Bruce reports receiving grants from the Medical Research Council and the National Institute for Health and Care Research during the conduct of the study, grants and speaker fees from GSK, consultancy and speaker fees from Astra Zeneca, and consultancy fees from UCB, BMS, Eli Lilly, and Aurinia outside the submitted work. Dr Payne reports receiving personal fees from UCB outside the submitted work. No other disclosures were reported. ",

year = "2023",

month = jan,

day = "31",

doi = "10.1016/j.jval.2022.07.001",

language = "English",

volume = "26",

pages = "115--122",

journal = "Value in Health",

issn = "1098-3015",

publisher = "Elsevier",

number = "1",

}

TY - JOUR

T1 - Valuing Health Gain from Composite Response Endpoints for Multisystem Diseases

AU - MASTERPLANS Consortium

AU - Gavan, Sean P.

AU - Bruce, Ian

AU - Payne, Katherine

AU - Bruce, Ian

AU - Lunt, Mark

AU - Peek, Niels

AU - Geifman, Nophar

AU - Gavan, Sean

AU - Armitt, Gillian

AU - Doherty, Patrick

AU - Prattley, Jennifer

AU - Azadbakht, Narges

AU - Papazian, Angela

AU - Le Sueur, Helen

AU - Farrelly, Carmen

AU - Richardson, Clare

AU - Shabbir, Zunnaira

AU - Hewitt, Lauren

AU - McHugh, Neil

AU - Gordon, Caroline

AU - Reynolds, John

AU - Young, Stephen

AU - Jayne, David

AU - Farewell, Vern

AU - Su, Li

AU - Pickering, Matthew

AU - Lightstone, Elizabeth

AU - Gilmore, Alyssa

AU - Botto, Marina

AU - Vyse, Timothy

AU - Morris, David Lester

AU - D'Cruz, David

AU - Vital, Edward

AU - Wittmann, Miriam

AU - Emery, Paul

AU - Beresford, Michael

AU - Hedrich, Christian

AU - Midgley, Angela

AU - Gritzfeld, Jenna

AU - Ehrenstein, Michael

AU - Isenberg, David

AU - Parvaz, Mariea

AU - Dunnage, Jane

AU - Batchelor, Jane

AU - Holland, Elaine

AU - Upsall, Pauline

AU - Youssef, Hazem

AU - McCann, Liza

AU - Mediwake, Rapti

AU - Bharadwaj, Anurag

N1 - Funding Information: Funding/Support: This research was funded by the Medical Research Council, United Kingdom as part of the MAximizing Sle ThERapeutic PotentiaL by Application of Novel and Stratified approaches (MASTERPLANS) Consortium (MR/M01665X/1) and part funded by a grant from LUPUS UK. BILAG-BR has been funded by unrestricted educational donations from Roche, GSK, and LUPUS UK. Funding Information: Previous studies have compared responders and nonresponders with existing composite response endpoints for SLE with other health-related quality of life instruments. Furie et al39 estimated that a greater percentage of patients who achieved the BICLA composite response endpoint at 52 weeks, than nonresponding patients, experienced at least a 3-point improvement in the Short-Form 36 (SF-36) Physical Component Summary score (57.9% vs 12.8%) and at least a 4.6-point improvement in the SF-36 Mental Component Summary score (42.6% vs 12.3%) as part of a post hoc analysis of the pooled anifrolumab phase III trial data. Similarly, Furie et al40 used pooled 52-week observations from the belimumab in subjects with systemic lupus erythematosus (BLISS)-52 and BLISS-76 trial data for belimumab and found that patients who achieved the SRI composite response endpoint had a greater improvement in the SF-36 Physical Component Summary (4.9 vs 2.6) and SF-36 Mental Component Summary (4.4 vs 1.7) than nonresponding patients. If there is a positive correlation between the SF-36 and EQ-5D-3L in people with SLE,41 these findings could suggest that existing endpoints may also improve EQ-5D scores. Direct comparative evidence of how existing composite response endpoints perform according to instruments preferred by HTA agencies will be valuable evidence to support future decision making.Funding/Support: This research was funded by the Medical Research Council, United Kingdom as part of the MAximizing Sle ThERapeutic PotentiaL by Application of Novel and Stratified approaches (MASTERPLANS) Consortium (MR/M01665X/1) and part funded by a grant from LUPUS UK. BILAG-BR has been funded by unrestricted educational donations from Roche, GSK, and LUPUS UK. Funding Information: Conflict of Interest Disclosures: Dr Gavan reported receiving grants from the Medical Research Council and Lupus UK during the conduct of the study and personal fees from UCB outside the submitted work. Dr Bruce reports receiving grants from the Medical Research Council and the National Institute for Health and Care Research during the conduct of the study, grants and speaker fees from GSK, consultancy and speaker fees from Astra Zeneca, and consultancy fees from UCB, BMS, Eli Lilly, and Aurinia outside the submitted work. Dr Payne reports receiving personal fees from UCB outside the submitted work. No other disclosures were reported.

PY - 2023/1/31

Y1 - 2023/1/31

N2 - Objectives: This study aimed to demonstrate how to estimate the value of health gain after patients with a multisystem disease achieve a condition-specific composite response endpoint. Methods: Data from patients treated in routine practice with an exemplar multisystem disease (systemic lupus erythematosus) were extracted from a national register (British Isles Lupus Assessment Group Biologics Register). Two bespoke composite response endpoints (Major Clinical Response and Improvement) were developed in advance of this study. Difference-in-differences regression compared health utility values (3-level version of EQ-5D; UK tariff) over 6 months for responders and nonresponders. Bootstrapped regression estimated the incremental quality-adjusted life-years (QALYs), probability of QALY gain after achieving the response criteria, and population monetary benefit of response. Results: Within the sample (n = 171), 18.2% achieved Major Clinical Response and 49.1% achieved Improvement at 6 months. Incremental health utility values were 0.0923 for Major Clinical Response and 0.0454 for Improvement. Expected incremental QALY gain at 6 months was 0.020 for Major Clinical Response and 0.012 for Improvement. Probability of QALY gain after achieving the response criteria was 77.6% for Major Clinical Response and 72.7% for Improvement. Population monetary benefit of response was £1 106 458 for Major Clinical Response and £649 134 for Improvement. Conclusions: Bespoke composite response endpoints are becoming more common to measure treatment response for multisystem diseases in trials and observational studies. Health technology assessment agencies face a growing challenge to establish whether these endpoints correspond with improved health gain. Health utility values can generate this evidence to enhance the usefulness of composite response endpoints for health technology assessment, decision making, and economic evaluation.

AB - Objectives: This study aimed to demonstrate how to estimate the value of health gain after patients with a multisystem disease achieve a condition-specific composite response endpoint. Methods: Data from patients treated in routine practice with an exemplar multisystem disease (systemic lupus erythematosus) were extracted from a national register (British Isles Lupus Assessment Group Biologics Register). Two bespoke composite response endpoints (Major Clinical Response and Improvement) were developed in advance of this study. Difference-in-differences regression compared health utility values (3-level version of EQ-5D; UK tariff) over 6 months for responders and nonresponders. Bootstrapped regression estimated the incremental quality-adjusted life-years (QALYs), probability of QALY gain after achieving the response criteria, and population monetary benefit of response. Results: Within the sample (n = 171), 18.2% achieved Major Clinical Response and 49.1% achieved Improvement at 6 months. Incremental health utility values were 0.0923 for Major Clinical Response and 0.0454 for Improvement. Expected incremental QALY gain at 6 months was 0.020 for Major Clinical Response and 0.012 for Improvement. Probability of QALY gain after achieving the response criteria was 77.6% for Major Clinical Response and 72.7% for Improvement. Population monetary benefit of response was £1 106 458 for Major Clinical Response and £649 134 for Improvement. Conclusions: Bespoke composite response endpoints are becoming more common to measure treatment response for multisystem diseases in trials and observational studies. Health technology assessment agencies face a growing challenge to establish whether these endpoints correspond with improved health gain. Health utility values can generate this evidence to enhance the usefulness of composite response endpoints for health technology assessment, decision making, and economic evaluation.

KW - composite response endpoint

KW - health state utility

KW - monetary benefit

KW - multisystem disease

KW - systemic lupus erythematous

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DO - 10.1016/j.jval.2022.07.001

M3 - Article

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SN - 1098-3015

VL - 26

SP - 115

EP - 122

JO - Value in Health

JF - Value in Health

IS - 1

ER -

Valuing Health Gain from Composite Response Endpoints for Multisystem Diseases

Abstract

Bibliographical note

Keywords

ASJC Scopus subject areas

UN SDGs

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