Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials: a start in setting international standards

Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) consortium

doi:10.1016/S1470-2045(16)30510-1

Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials: a start in setting international standards

Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) consortium

Research output: Contribution to journal › Review article › peer-review

100 Scopus citations

Abstract

Measures of health-related quality of life (HRQOL) and other patient-reported outcomes generate important data in cancer randomised trials to assist in assessing the risks and benefits of cancer therapies and fostering patient-centred cancer care. However, the various ways these measures are analysed and interpreted make it difficult to compare results across trials, and hinders the application of research findings to inform publications, product labelling, clinical guidelines, and health policy. To address these problems, the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) initiative has been established. This consortium, directed by the European Organisation for Research and Treatment of Cancer (EORTC), was convened to provide recommendations on how to standardise the analysis of HRQOL and other patient-reported outcomes data in cancer randomised trials. This Personal View discusses the reasons why this project was initiated, the rationale for the planned work, and the expected benefits to cancer research, patient and provider decision making, care delivery, and policy making.

Original language	English (US)
Pages (from-to)	e510-e514
Journal	The Lancet Oncology
Volume	17
Issue number	11
DOIs	https://doi.org/10.1016/S1470-2045(16)30510-1
State	Published - Nov 1 2016

ASJC Scopus subject areas

Oncology

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10.1016/S1470-2045(16)30510-1

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Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials : a start in setting international standards. / Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) consortium.

In: The Lancet Oncology, Vol. 17, No. 11, 01.11.2016, p. e510-e514.

Research output: Contribution to journal › Review article › peer-review

Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) consortium 2016, 'Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials: a start in setting international standards', The Lancet Oncology, vol. 17, no. 11, pp. e510-e514. https://doi.org/10.1016/S1470-2045(16)30510-1

Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) consortium. Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials: a start in setting international standards. The Lancet Oncology. 2016 Nov 1;17(11):e510-e514. https://doi.org/10.1016/S1470-2045(16)30510-1

@article{d38bfc85b3aa4d36a57020922ac079b9,

title = "Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials: a start in setting international standards",

abstract = "Measures of health-related quality of life (HRQOL) and other patient-reported outcomes generate important data in cancer randomised trials to assist in assessing the risks and benefits of cancer therapies and fostering patient-centred cancer care. However, the various ways these measures are analysed and interpreted make it difficult to compare results across trials, and hinders the application of research findings to inform publications, product labelling, clinical guidelines, and health policy. To address these problems, the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) initiative has been established. This consortium, directed by the European Organisation for Research and Treatment of Cancer (EORTC), was convened to provide recommendations on how to standardise the analysis of HRQOL and other patient-reported outcomes data in cancer randomised trials. This Personal View discusses the reasons why this project was initiated, the rationale for the planned work, and the expected benefits to cancer research, patient and provider decision making, care delivery, and policy making.",

author = "{Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) consortium} and Andrew Bottomley and Madeline Pe and Jeff Sloan and Ethan Basch and Franck Bonnetain and Melanie Calvert and Alicyn Campbell and Charles Cleeland and Kim Cocks and Laurence Collette and Dueck, {Amylou C.} and Nancy Devlin and Flechtner, {Hans Henning} and Carolyn Gotay and Eva Greimel and Ingolf Griebsch and Mogens Groenvold and Hamel, {Jean Francois} and Madeleine King and Kluetz, {Paul G.} and Michael Koller and Malone, {Daniel C.} and Francesca Martinelli and Mitchell, {Sandra A.} and Moinpour, {Carol M.} and Jammbe Musoro and Daniel O'Connor and Kathy Oliver and Elisabeth Piault-Louis and Martine Piccart and Pimentel, {Francisco L.} and Chantal Quinten and Reijneveld, {Jaap C.} and Christoph Sch{\"u}rmann and Smith, {Ashley Wilder} and Soltys, {Katherine M.} and Taphoorn, {Martin J.B.} and Galina Velikova and Corneel Coens",

note = "Funding Information: The objective of the SISAQOL initiative is to produce a suite of tools, guidance, and international consensus standards for the analysis of HRQOL and other patient-reported outcome data from clinical trials. We also aim to provide template macros to be used in a number of commonplace missing data settings and illustrative macros to address these requirements. We expect that having freely available guidelines and tools to facilitate their implementation will result in more reliable findings and faster dissemination of data. These findings will stem from a higher quality use of statistical methods and an improved interpretability of data because of greater familiarity with standardised reporting. We are aware that standardising statistical analyses for HRQOL and other patient-reported outcome data in cancer clinical trials is an ambitious goal. However, the need for such standards has become prominent given the expanding interest in HRQOL and other patient-reported outcome endpoints. Trials cost substantial time, money, and effort. Moreover, study participants, in the interest of improving their situations and helping others, voluntarily give up their time to complete measures for these trials. Therefore, the data we gather must be exploited to the full, with statistical analyses done in the most rigorous and standardised way, and with results that clearly highlight clinical benefits and not just statistical significance. 9 HRQOL and other findings also have a large potential effect on treatment benefit assessments, and, as resources to cover costs of cancer care become more scarce and treatment costs increase, it is imperative that these findings are based on valid and reliable statistical methods. For these reasons, choosing methods that reflect the best possible available evidence and the expertise of a diverse group of stakeholders is crucial. Members of the SISAQOL initiative have a shared interest in addressing this gap by working together to articulate a set of standards, best practices, and tools for the analysis and interpretation of HRQOL and other patient-reported outcome endpoints in cancer clinical trials. For more on the SISAQOL consortium , please visit http://www.eortc.org/sisaqol for more details Contributors The manuscript was conceptualised with the attendees of the SISAQOL kick-off meeting in Brussels, Belgium, on Jan 26, 2016. All authors reviewed and contributed to revisions of the article. All authors approved the final draft of the manuscript. Declaration of interests AB reports grants from Boehringer Ingelheim, grants from EORTC cancer research fund during the conduct of the study; grants from Merck outside the submitted work; and is a member of the EORTC Quality of Life Group executive committee. FB reports grants, personal fees, and non-financial support from Ipsen, Amgen, Janssen, Novartis and Roche, personal fees from Amgen, Merck Serono, Ipsen, Bayer, BMS, Nestle, Integragen, Chugai, and Janssen outside the submitted work. MC reports other conflicts from International Society for Quality of Research, personal fees from Astellas Pharma, and Ferring Pharma outside the submitted work; and MC is Director of the Centre for Patient Reported Outcomes Research at the University of Birmingham and is involved in a number of local and international initiatives to promote best practice for patient-reported outcomes in trials including the development of a SPIRIT-PRO Extension and CONSORT-PRO. AC reports that she is an employee of Genentech. KC reports a grant from EORTC Quality of Life Group and personal fees from Amgen, Endomag, AstraZeneca, and Bristol-Myers Squibb outside the submitted work. ND reports that the Office of Health Economics receives funding from the Association of the British Pharmaceutical Industry. ND is a member of the EuroQoL Group, the European-based international group that developed the EQ-5D. ND is also a member of the Board of Directors of ISPOR. IG is an employee of Boehringer Ingelheim, Germany. Boehringer Ingelheim provided an unrestricted educational fund to initiate this work. MG reports that he is past chair of the EORTC Quality of Life Group, has received grants from this group, and is Board member of EORTC and International Society of Quality of Life Research. MK reports grants from EORTC Quality of Life Group and personal fees from Mundipharma outside the submitted work. KO reports grants for the International Brain Tumour Alliance from Roche, GSK, Lilly, Novocure, Novartis, Celldex, MagForce, Accuray, Photonamic, Medac, to-BBB, Apogenix, Pfizer, AbbVie, Northwest Biotherapeutics, VBL Therapeutics, The Sontag Foundation, The Brain Tumour Network, and the Ivy Foundation, all of which are outside the submitted work. KO has a consultancy company that reports honoraria and personal fees from GSK, Lilly, Novartis, Bayer, Bristol-Myers Squibb, and Sarcoma Patients EuroNet Association, all of which are outside the submitted work. MJBT reports personal fees from Hoffman-La Roche outside the submitted work. GV reports that she is the past chair of the EORTC Quality of Life group and past president of the International Society for Quality of Research. All other authors declare no competing interests. Acknowledgments EORTC received an unrestricted education grant from Boehringer Ingelheim GmbH to initiate this work and additional financial support was provided by the EORTC Cancer Research Fund and Fonds Cancer (FOCA) from Belgium. Andrew Bottomley confirms final responsibility for the decision to submit for publication. This publication reflects the views of the individual authors and should not be construed to represent official views or policies of the US Food and Drug Administration, US National Cancer Institute, Medicines and Healthcare products Regulatory Agency, or Health Canada. Publisher Copyright: {\textcopyright} 2016 Elsevier Ltd",

year = "2016",

month = nov,

day = "1",

doi = "10.1016/S1470-2045(16)30510-1",

language = "English (US)",

volume = "17",

pages = "e510--e514",

journal = "The Lancet Oncology",

issn = "1470-2045",

publisher = "Lancet Publishing Group",

number = "11",

}

TY - JOUR

T1 - Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials

T2 - a start in setting international standards

AU - Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) consortium

AU - Bottomley, Andrew

AU - Pe, Madeline

AU - Sloan, Jeff

AU - Basch, Ethan

AU - Bonnetain, Franck

AU - Calvert, Melanie

AU - Campbell, Alicyn

AU - Cleeland, Charles

AU - Cocks, Kim

AU - Collette, Laurence

AU - Dueck, Amylou C.

AU - Devlin, Nancy

AU - Flechtner, Hans Henning

AU - Gotay, Carolyn

AU - Greimel, Eva

AU - Griebsch, Ingolf

AU - Groenvold, Mogens

AU - Hamel, Jean Francois

AU - King, Madeleine

AU - Kluetz, Paul G.

AU - Koller, Michael

AU - Malone, Daniel C.

AU - Martinelli, Francesca

AU - Mitchell, Sandra A.

AU - Moinpour, Carol M.

AU - Musoro, Jammbe

AU - O'Connor, Daniel

AU - Oliver, Kathy

AU - Piault-Louis, Elisabeth

AU - Piccart, Martine

AU - Pimentel, Francisco L.

AU - Quinten, Chantal

AU - Reijneveld, Jaap C.

AU - Schürmann, Christoph

AU - Smith, Ashley Wilder

AU - Soltys, Katherine M.

AU - Taphoorn, Martin J.B.

AU - Velikova, Galina

AU - Coens, Corneel

N1 - Funding Information: The objective of the SISAQOL initiative is to produce a suite of tools, guidance, and international consensus standards for the analysis of HRQOL and other patient-reported outcome data from clinical trials. We also aim to provide template macros to be used in a number of commonplace missing data settings and illustrative macros to address these requirements. We expect that having freely available guidelines and tools to facilitate their implementation will result in more reliable findings and faster dissemination of data. These findings will stem from a higher quality use of statistical methods and an improved interpretability of data because of greater familiarity with standardised reporting. We are aware that standardising statistical analyses for HRQOL and other patient-reported outcome data in cancer clinical trials is an ambitious goal. However, the need for such standards has become prominent given the expanding interest in HRQOL and other patient-reported outcome endpoints. Trials cost substantial time, money, and effort. Moreover, study participants, in the interest of improving their situations and helping others, voluntarily give up their time to complete measures for these trials. Therefore, the data we gather must be exploited to the full, with statistical analyses done in the most rigorous and standardised way, and with results that clearly highlight clinical benefits and not just statistical significance. 9 HRQOL and other findings also have a large potential effect on treatment benefit assessments, and, as resources to cover costs of cancer care become more scarce and treatment costs increase, it is imperative that these findings are based on valid and reliable statistical methods. For these reasons, choosing methods that reflect the best possible available evidence and the expertise of a diverse group of stakeholders is crucial. Members of the SISAQOL initiative have a shared interest in addressing this gap by working together to articulate a set of standards, best practices, and tools for the analysis and interpretation of HRQOL and other patient-reported outcome endpoints in cancer clinical trials. For more on the SISAQOL consortium , please visit http://www.eortc.org/sisaqol for more details Contributors The manuscript was conceptualised with the attendees of the SISAQOL kick-off meeting in Brussels, Belgium, on Jan 26, 2016. All authors reviewed and contributed to revisions of the article. All authors approved the final draft of the manuscript. Declaration of interests AB reports grants from Boehringer Ingelheim, grants from EORTC cancer research fund during the conduct of the study; grants from Merck outside the submitted work; and is a member of the EORTC Quality of Life Group executive committee. FB reports grants, personal fees, and non-financial support from Ipsen, Amgen, Janssen, Novartis and Roche, personal fees from Amgen, Merck Serono, Ipsen, Bayer, BMS, Nestle, Integragen, Chugai, and Janssen outside the submitted work. MC reports other conflicts from International Society for Quality of Research, personal fees from Astellas Pharma, and Ferring Pharma outside the submitted work; and MC is Director of the Centre for Patient Reported Outcomes Research at the University of Birmingham and is involved in a number of local and international initiatives to promote best practice for patient-reported outcomes in trials including the development of a SPIRIT-PRO Extension and CONSORT-PRO. AC reports that she is an employee of Genentech. KC reports a grant from EORTC Quality of Life Group and personal fees from Amgen, Endomag, AstraZeneca, and Bristol-Myers Squibb outside the submitted work. ND reports that the Office of Health Economics receives funding from the Association of the British Pharmaceutical Industry. ND is a member of the EuroQoL Group, the European-based international group that developed the EQ-5D. ND is also a member of the Board of Directors of ISPOR. IG is an employee of Boehringer Ingelheim, Germany. Boehringer Ingelheim provided an unrestricted educational fund to initiate this work. MG reports that he is past chair of the EORTC Quality of Life Group, has received grants from this group, and is Board member of EORTC and International Society of Quality of Life Research. MK reports grants from EORTC Quality of Life Group and personal fees from Mundipharma outside the submitted work. KO reports grants for the International Brain Tumour Alliance from Roche, GSK, Lilly, Novocure, Novartis, Celldex, MagForce, Accuray, Photonamic, Medac, to-BBB, Apogenix, Pfizer, AbbVie, Northwest Biotherapeutics, VBL Therapeutics, The Sontag Foundation, The Brain Tumour Network, and the Ivy Foundation, all of which are outside the submitted work. KO has a consultancy company that reports honoraria and personal fees from GSK, Lilly, Novartis, Bayer, Bristol-Myers Squibb, and Sarcoma Patients EuroNet Association, all of which are outside the submitted work. MJBT reports personal fees from Hoffman-La Roche outside the submitted work. GV reports that she is the past chair of the EORTC Quality of Life group and past president of the International Society for Quality of Research. All other authors declare no competing interests. Acknowledgments EORTC received an unrestricted education grant from Boehringer Ingelheim GmbH to initiate this work and additional financial support was provided by the EORTC Cancer Research Fund and Fonds Cancer (FOCA) from Belgium. Andrew Bottomley confirms final responsibility for the decision to submit for publication. This publication reflects the views of the individual authors and should not be construed to represent official views or policies of the US Food and Drug Administration, US National Cancer Institute, Medicines and Healthcare products Regulatory Agency, or Health Canada. Publisher Copyright: © 2016 Elsevier Ltd

PY - 2016/11/1

Y1 - 2016/11/1

N2 - Measures of health-related quality of life (HRQOL) and other patient-reported outcomes generate important data in cancer randomised trials to assist in assessing the risks and benefits of cancer therapies and fostering patient-centred cancer care. However, the various ways these measures are analysed and interpreted make it difficult to compare results across trials, and hinders the application of research findings to inform publications, product labelling, clinical guidelines, and health policy. To address these problems, the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) initiative has been established. This consortium, directed by the European Organisation for Research and Treatment of Cancer (EORTC), was convened to provide recommendations on how to standardise the analysis of HRQOL and other patient-reported outcomes data in cancer randomised trials. This Personal View discusses the reasons why this project was initiated, the rationale for the planned work, and the expected benefits to cancer research, patient and provider decision making, care delivery, and policy making.

AB - Measures of health-related quality of life (HRQOL) and other patient-reported outcomes generate important data in cancer randomised trials to assist in assessing the risks and benefits of cancer therapies and fostering patient-centred cancer care. However, the various ways these measures are analysed and interpreted make it difficult to compare results across trials, and hinders the application of research findings to inform publications, product labelling, clinical guidelines, and health policy. To address these problems, the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) initiative has been established. This consortium, directed by the European Organisation for Research and Treatment of Cancer (EORTC), was convened to provide recommendations on how to standardise the analysis of HRQOL and other patient-reported outcomes data in cancer randomised trials. This Personal View discusses the reasons why this project was initiated, the rationale for the planned work, and the expected benefits to cancer research, patient and provider decision making, care delivery, and policy making.

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DO - 10.1016/S1470-2045(16)30510-1

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AN - SCOPUS:84995577351

VL - 17

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JO - The Lancet Oncology

JF - The Lancet Oncology

SN - 1470-2045

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ER -

Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials: a start in setting international standards

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