The importance of patient-reported outcomes in clinical trials and strategies for future optimization

References

• Guidance for Industry: patient-reported outcome measures: use in medical product development to support labelling claimsFood and Drug Administration2009 Available from: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdfAccessed January 10, 2015
   Google Scholar
• RevickiDAOsobaDFaircloughDRecommendations on health-related quality of life research to support labeling and promotional claims in the United StatesQual Life Res20009888790011284208
   PubMed Web of Science ®Google Scholar
• AuHJRingashJBrundageMAdded value of health-related quality of life measurement in cancer clinical trials: the experience of the NCIC CTGExpert Rev Pharmacoecon Outcomes Res201010211912820384559
   PubMed Web of Science ®Google Scholar
• Organisation for Economic Co-operation and Development (OECD)Strengthening the international comparison of health system performance through patient-reported indicatorsRecommendations to OECD Ministers of Health from the High Level Reflection Group on the Future of Health Statistics2017 Available from: https://www.oecd.org/els/health-systems/Recommendations-from-high-level-reflection-group-on-the-future-of-health-statistics.pdfAccessed May 07, 2017
   Google Scholar
• ScogginsJFPatrickDLThe use of patient-reported outcomes instruments in registered clinical trials: evidence from ClinicalTrials.govContemp Clin Trials200930428929219275948
   PubMed Web of Science ®Google Scholar
• VodickaEKimKDevineEBInclusion of patient-reported outcome measures in registered clinical trials: evidence from ClinicalTrials.gov (2007-2013)Contemp Clin Trials2015431925896116
   PubMed Web of Science ®Google Scholar
• Mercieca-BebberRWilliamsDTaitMATrials with patient-reported outcomes registered on the Australian New Zealand Clinical Trials Registry (ANZCTR)Qual Life Res201827102581259129915979
   PubMed Web of Science ®Google Scholar
• Food and Drug AdministrationValue and Use of Patient-Reported Outcomes (PROs) in Assessing Effects of Medical Devices CDRH Strategic PrioritiesCDRH Strategic Priorities 2016–20172017 Available from: https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDRH/CDRHVisionandMission/UCM588576.pdfAccessed February 22, 2018
   Google Scholar
• KluetzPGO’ConnorDJSoltysKIncorporating the patient experience into regulatory decision making in the USA, Europe, and CanadaLancet Oncol2018195e267e27429726391
   PubMed Web of Science ®Google Scholar
• SchnipperLEDavidsonNEWollinsDSUpdating the American Society of Clinical Oncology Value Framework: revisions and reflections in response to comments receivedJ Clin Oncol201634242925293427247218
   PubMed Web of Science ®Google Scholar
• ChernyNISullivanRDafniUA standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS)Ann Oncol20152681547157326026162
   PubMed Web of Science ®Google Scholar
• Cancer AustraliaSupport for Cancer Clinical Trials2013 Available from: http://canceraustralia.gov.au/research-data/support-clinical-trialsAccessed November 22, 2017
   Google Scholar
• European Medicines AgencyAppendix 2 to the guideline on the evaluation of anticancer medicinal products in man: the use of patient-reported outcome (PRO) measures in oncology studiesLondon, UK2016 Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Other/2016/04/WC500205159.pdfAccessed December 05, 2017
   Google Scholar
• duBois AQuinnMThigpenTconsensus statements on the management of ovarian cancer: final document of the 3rd International Gynecologic Cancer Intergroup Ovarian Cancer Consensus Conference (GCIG OCCC 2004)Ann Oncol200416viii7viii12
   Web of Science ®Google Scholar
• Food and Drug AdministrationOvarian Cancer Endpoints Workshop April 26, 2006, Meeting Summary2006 Available from: http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/Developmen-tResources/CancerDrugs/ucm094598.pdfaccessed 09 August 2016 https://www.fda.gov/downloads/AboutFDA/CentersOffices/CDER/ucm120657.pdfAccessed 14 August 2017.v
   Google Scholar
• FriedlanderMTrimbleETinkerAClinical trials in recurrent ovarian cancerInt J Gynecol Cancer201121477177521543939
   PubMed Web of Science ®Google Scholar
• StuartGCKitchenerHBaconM2010 Gynecologic Cancer InterGroup (GCIG) consensus statement on clinical trials in ovarian cancer: report from the Fourth Ovarian Cancer Consensus ConferenceInt J Gynecol Cancer201121475075521543936
   PubMed Web of Science ®Google Scholar
• Centres for Medicare and Medicaid ServicesMEDCAC Meeting 3/22/2017 – Health Outcomes in Heart Failure Treatment Technology Studies2017 Available from: https://www.cms.gov/medicare-coverage-database/details/medcac-meeting-details.aspx?MEDCACId=73Accessed October101017
   Google Scholar
• SchorrAPatient-reported outcomes and where patient advocacy groups can helpChange Together2017 Available from: https://www.chan-getogether.com/general/patient-reported-outcomes-patient-advocacy-groups-can-helpAugust 08, 2017
   Google Scholar
• WilsonRPatient led PROMs must take centre stage in cancer researchRes Involv Engagem201847
   Google Scholar
• TannockIFOsobaDStocklerMRChemotherapy with mitoxantrone plus prednisone or prednisone alone for symptomatic hormone-resistant prostate cancer: a Canadian randomized trial with palliative end pointsJ Clin Oncol1996146175617648656243
   PubMed Web of Science ®Google Scholar
• OsobaDTannockIFErnstDSNevilleAJHealth-related quality of life in men with metastatic prostate cancer treated with prednisone alone or mitoxantrone and prednisoneJ Clin Oncol19991761654165410561201
   PubMed Web of Science ®Google Scholar
• MesaRAGotlibJGuptaVEffect of ruxolitinib therapy on myelofibrosis-related symptoms and other patient-reported outcomes in COMFORT-I: a randomized, double-blind, placebo-controlled trialJ Clin Oncol201331101285129223423753
   PubMed Web of Science ®Google Scholar
• Food and Drug Administration: JAKAFI® (ruxolitinib) tablets for oral use2017
   Google Scholar
• GnanasakthyADemuroCClarkMPatient-reported outcomes labeling for products approved by the Office of Hematology and Oncology Products of the US Food and Drug Administration (2010-2014)J Clin Oncol201634161928193427069082
   PubMed Web of Science ®Google Scholar
• GnanasakthyAMordinMEvansEDowardLDemuroCA review of patient-reported outcome labeling in the United States (2011-2015)Value Health201720342042928292487
   PubMed Web of Science ®Google Scholar
• GnanasakthyAMordinMClarkMA review of patient-reported outcome labels in the United States: 2006 to 2010Value Health201215343744222583453
   PubMed Web of Science ®Google Scholar
• KyteDDuffyHFletcherBSystematic evaluation of the patient-reported outcome (PRO) content of clinical trial protocolsPLoS One2014910e11022925333349
   PubMed Web of Science ®Google Scholar
• CalvertMKyteDMercieca-BebberRGuidelines for inclusion of patient-reported outcomes in clinical trial protocols: The SPIRIT-PRO ExtensionJAMA2018319548349429411037
   PubMed Web of Science ®Google Scholar
• Mercieca-BebberRFriedlanderMKokPSThe patient-reported outcome content of international ovarian cancer randomised controlled trial protocolsQual Life Res201625102457246527294435
   PubMed Web of Science ®Google Scholar
• AhmedKKyteDKeeleyTSystematic evaluation of patient-reported outcome (PRO) protocol content and reporting in UK cancer clinical trials: the EPiC study protocolBMJ Open201669e012863
   Web of Science ®Google Scholar
• ChanAWTetzlaffJMAltmanDGSPIRIT 2013 statement: defining standard protocol items for clinical trialsAnn Intern Med2013158320020132207
   Web of Science ®Google Scholar
• ChanAWTetzlaffJMGøtzschePCSPIRIT 2013 explanation and elaboration: guidance for protocols of clinical trialsBMJ20133462013
   Web of Science ®Google Scholar
• EQUATOR NetworkEnhancing the QUAlity and Transparency Of health Research Available from: https://www.equator-network.orgAccessed July 12, 2017
   Google Scholar
• Mercieca-BebberRLPerrecaAKingMPatient-reported outcomes in head and neck and thyroid cancer randomised controlled trials: a systematic review of completeness of reporting and impact on interpretationEur J Cancer20165614416126851406
   PubMed Web of Science ®Google Scholar
• KluetzPGSlagleAPapadopoulosEJFocusing on core patient-reported outcomes in cancer clinical trials: symptomatic adverse events, physical function, and disease-related symptomsClin Cancer Res20162271553155826758559
   PubMed Web of Science ®Google Scholar
• SnyderCFWatsonMEJacksonJDPatient-reported outcome instrument selection: designing a measurement strategyValue Health200710Suppl 2S76S8517995477
   PubMed Web of Science ®Google Scholar
• LuckettTKingMTChoosing patient-reported outcome measures for cancer clinical research – practical principles and an algorithm to assist non-specialist researchersEur J Cancer201046183149315720869232
   PubMed Web of Science ®Google Scholar
• KyteDIvesJDraperHKeeleyTCalvertMInconsistencies in quality of life data collection in clinical trials: a potential source of bias? Interviews with research nurses and trialistsPLoS One2013810e7662524124580
   PubMed Web of Science ®Google Scholar
• Mercieca-BebberRCalvertMKyteDStocklerMKingMTThe administration of patient-reported outcome questionnaires in cancer trials: Interviews with trial coordinators regarding their roles, experiences, challenges and trainingContemp Clin Trials Commun20189233229696221
   PubMed Web of Science ®Google Scholar
• International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH): Integrated Addendum to ICH E6 (R1): Guideline for Good Clinical Practice E6 (R2), (ed 4)2016 Available from: https://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E6/E6_R2__Step_4_2016_1109.pdfAccessed February 02, 2017
   Google Scholar
• RutherfordCCostaDMercieca-BebberRMode of administration does not cause bias in patient-reported outcome results: a meta-analysisQual Life Res201625355957426334842
   PubMed Web of Science ®Google Scholar
• YoungTde HaesDCurranDGuidelines for assessing Quality of Life in EORTC clinical trialsBrusselsEORTC Quality of Life Group2002
   Google Scholar
• QOL OfficeChecklist of instructions for the administration of Patient Reported Outcome Measures2015 Available from: http://www.pocog.org.au/docview.aspx?id=355Accessed February 02, 2017
   Google Scholar
• HansenLKMoinpourCMErmeteRBLessons learned: enhancing nurse contributions to cancer clinical trials in SWOGSemin Oncol Nurs201430263124559777
   PubMedGoogle Scholar
• LarkinMELorenziGMBaylessMEvolution of the study coordinator role: the 28-year experience in Diabetes control and complications trial/epidemiology of Diabetes interventions and complications (DCCT/EDIC)Clin Trials20129441842522729476
   PubMed Web of Science ®Google Scholar
• FaircloughDLPetersonHFCellaDBonomiPComparison of several model-based methods for analysing incomplete quality of life data in cancer clinical trialsStat Med1998175–77817969549823
   PubMed Web of Science ®Google Scholar
• FaircloughDLPetersonHFChangVWhy are missing quality of life data a problem in clinical trials of cancer therapy?Stat Med1998175–76676779549815
   PubMed Web of Science ®Google Scholar
• BellMLFieroMHortonNJHsuCHHandling missing data in RCTs; a review of the top medical journalsBMC Med Res Methodol20141411825407057
   PubMed Web of Science ®Google Scholar
• FieldingSMaclennanGCookJARamsayCRA review of RCTs in four medical journals to assess the use of imputation to overcome missing data in quality of life outcomesTrials200895118694492
   PubMed Web of Science ®Google Scholar
• FieldingSOgbuaguASivasubramaniamSMaclennanGRamsayCRReporting and dealing with missing quality of life data in RCTs: has the picture changed in the last decade?Qual Life Res201625122977298327650288
   PubMed Web of Science ®Google Scholar
• BellMLFaircloughDLPractical and statistical issues in missing data for longitudinal patient-reported outcomesStat Methods Med Res201423544045923427225
   PubMed Web of Science ®Google Scholar
• BellMLOlivierJKingMTScientific rigour in psycho-oncology trials: why and how to avoid common statistical errorsPsychooncology201322349950522315186
   PubMed Web of Science ®Google Scholar
• CurranDBacchiMSchmitzSFMolenberghsGSylvesterRJIdentifying the types of missingness in quality of life data from clinical trialsStat Med1998175–77397569549820
   PubMed Web of Science ®Google Scholar
• Mercieca-BebberRPalmerMJBrundageMDesign, implementation and reporting strategies to reduce the instance and impact of missing patient-reported outcome (PRO) data: a systematic reviewBMJ Open201666e010938
   PubMed Web of Science ®Google Scholar
• Mercieca-BebberRPalmerMJBrundageMDesign, implementation and reporting strategies to reduce the instance and impact of missing patient-reported outcome (PRO) data: a systematic reviewBMJ Open201666e010938
   PubMed Web of Science ®Google Scholar
• Mercieca-BebberRFriedlanderMCalvertMA systematic evaluation of compliance and reporting of patient-reported outcome endpoints in ovarian cancer randomised controlled trials: implications for generalisability and clinical practiceJ Patient Rep Outcomes20171529757300
   PubMedGoogle Scholar
• PalmerMJMercieca-BebberRKingMA systematic review and development of a classification framework for factors associated with missing patient-reported outcome dataClin Trials20181519510629124956
   PubMed Web of Science ®Google Scholar
• BernhardJCellaDFCoatesASMissing quality of life data in cancer clinical trials: serious problems and challengesStat Med1998175-75175329549801
   PubMed Web of Science ®Google Scholar
• FieldingSFayersPRamsayCRAnalysing randomised controlled trials with missing data: choice of approach affects conclusionsContemp Clin Trials201233346146922265924
   PubMed Web of Science ®Google Scholar
• PostWJBuijsCStolkRPde VriesEGLe CessieSThe analysis of longitudinal quality of life measures with informative drop-out: a pattern mixture approachQual Life Res201019113714820041307
   PubMed Web of Science ®Google Scholar
• BottomleyAPeMSloanJAnalysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials: a start in setting international standardsLancet Oncol20161711e510e51427769798
   PubMed Web of Science ®Google Scholar
• BrundageMBassBDavidsonJPatterns of reporting health-related quality of life outcomes in randomized clinical trials: implications for clinicians and quality of life researchersQual Life Res201120565366421110123
   PubMed Web of Science ®Google Scholar
• BylickiOGanHKJolyFPoor patient-reported outcomes reporting according to CONSORT guidelines in randomized clinical trials evaluating systemic cancer therapyAnn Oncol201526123123725355720
   PubMed Web of Science ®Google Scholar
• EfficaceFFayersPPusicAQuality of patient-reported outcome reporting across cancer randomized controlled trials according to the CONSORT patient-reported outcome extension: a pooled analysis of 557 trialsCancer2015121183335334226079197
   PubMed Web of Science ®Google Scholar
• EfficaceFJacobsMPusicAPatient-reported outcomes in randomised controlled trials of gynaecological cancers: investigating methodological quality and impact on clinical decision-makingEur J Cancer201450111925194124825114
   PubMed Web of Science ®Google Scholar
• JolyFVardyJPintilieMTannockIFQuality of life and/or symptom control in randomized clinical trials for patients with advanced cancerAnn Oncol200718121935194217698837
   PubMed Web of Science ®Google Scholar
• MackDEWilsonPMSantosEBrooksKStandards of reporting: the use of CONSORT PRO and CERT in individuals living with osteoporosisOsteoporos Int201829230531328971256
   PubMed Web of Science ®Google Scholar
• Mercieca-BebberRRouetteJCalvertMPreliminary evidence on the uptake, use and benefits of the CONSORT-PRO extensionQual Life Res20172661427143728168602
   PubMed Web of Science ®Google Scholar
• SchandelmaierSConenKvon ElmEPlanning and reporting of quality-of-life outcomes in cancer trialsAnn Oncol20152691966197326133966
   PubMed Web of Science ®Google Scholar
• GlasziouPAltmanDGBossuytPReducing waste from incomplete or unusable reports of biomedical researchLancet2014383991326727624411647
   PubMed Web of Science ®Google Scholar
• BrundageMBlazebyJRevickiDPatient-reported outcomes in randomized clinical trials: development of ISOQOL reporting standardsQual Life Res20132261161117522987144
   PubMed Web of Science ®Google Scholar
• CalvertMBlazebyJAltmanDGReporting of patient-reported outcomes in randomized trials: the CONSORT PRO extensionJAMA2013309881482223443445
   PubMed Web of Science ®Google Scholar
• SchulzKFAltmanDGMoherDCONSORT GroupCONSORT 2010 statement: updated guidelines for reporting parallel group randomised trialsBMJ2010340c33220332509
   PubMed Web of Science ®Google Scholar
• CalvertMBrundageMJacobsenPBSchünemannHJEfficaceFThe CONSORT Patient-Reported Outcome (PRO) extension: implications for clinical trials and practiceHealth Qual Life Outcomes20131118424168680
   PubMed Web of Science ®Google Scholar