{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,12]],"date-time":"2026-09-12T08:36:28Z","timestamp":1789202188502,"version":"build-2803163510"},"reference-count":237,"publisher":"National Institute for Health and Care Research","issue":"60","license":[{"start":{"date-parts":[[2019,10,1]],"date-time":"2019-10-01T00:00:00Z","timestamp":1569888000000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/www.nationalarchives.gov.uk\/doc\/non-commercial-government-licence\/non-commercial-government-licence.htm"}],"funder":[{"DOI":"10.13039\/501100000664","name":"Health Technology Assessment programme","doi-asserted-by":"publisher","award":["NIHR127268"],"award-info":[{"award-number":["NIHR127268"]}],"id":[{"id":"10.13039\/501100000664","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"Medical Research Council","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Health Technol Assess"],"abstract":"\n Background<\/jats:title>\n The randomised controlled trial is widely considered to be the gold standard study for comparing the effectiveness of health interventions. Central to its design is a calculation of the number of participants needed (the sample size) for the trial. The sample size is typically calculated by specifying the magnitude of the difference in the primary outcome between the intervention effects for the population of interest. This difference is called the \u2018target difference\u2019 and should be appropriate for the principal estimand of interest and determined by the primary aim of the study. The target difference between treatments should be considered realistic and\/or important by one or more key stakeholder groups.<\/jats:p>\n <\/jats:sec>\n \n Objective<\/jats:title>\n The objective of the report is to provide practical help on the choice of target difference used in the sample size calculation for a randomised controlled trial for researchers and funder representatives.<\/jats:p>\n <\/jats:sec>\n \n Methods<\/jats:title>\n The Difference ELicitation in TriAls2<\/jats:sup> (DELTA2<\/jats:sup>) recommendations and advice were developed through a five-stage process, which included two literature reviews of existing funder guidance and recent methodological literature; a Delphi process to engage with a wider group of stakeholders; a 2-day workshop; and finalising the core document.<\/jats:p>\n <\/jats:sec>\n \n Results<\/jats:title>\n Advice is provided for definitive trials (Phase III\/IV studies). Methods for choosing the target difference are reviewed. To aid those new to the topic, and to encourage better practice, 10 recommendations are made regarding choosing the target difference and undertaking a sample size calculation. Recommended reporting items for trial proposal, protocols and results papers under the conventional approach are also provided. Case studies reflecting different trial designs and covering different conditions are provided. Alternative trial designs and methods for choosing the sample size are also briefly considered.<\/jats:p>\n <\/jats:sec>\n \n Conclusions<\/jats:title>\n Choosing an appropriate sample size is crucial if a study is to inform clinical practice. The number of patients recruited into the trial needs to be sufficient to answer the objectives; however, the number should not be higher than necessary to avoid unnecessary burden on patients and wasting precious resources. The choice of the target difference is a key part of this process under the conventional approach to sample size calculations. This document provides advice and recommendations to improve practice and reporting regarding this aspect of trial design. Future work could extend the work to address other less common approaches to the sample size calculations, particularly in terms of appropriate reporting items.<\/jats:p>\n <\/jats:sec>\n \n Funding<\/jats:title>\n Funded by the Medical Research Council (MRC) UK and the National Institute for Health Research as part of the MRC\u2013National Institute for Health Research Methodology Research programme.<\/jats:p>\n <\/jats:sec>","DOI":"10.3310\/hta23600","type":"journal-article","created":{"date-parts":[[2019,10,29]],"date-time":"2019-10-29T13:45:16Z","timestamp":1572356716000},"page":"1-88","update-policy":"https:\/\/doi.org\/10.3310\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":26,"title":["Practical help for specifying the target difference in sample size calculations for RCTs: the DELTA2 five-stage study, including a workshop"],"prefix":"10.3310","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4156-6989","authenticated-orcid":true,"given":"Jonathan A","family":"Cook","sequence":"first","affiliation":[{"name":"Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9917-7636","authenticated-orcid":true,"given":"Steven A","family":"Julious","sequence":"additional","affiliation":[{"name":"Medical Statistics Group, School of Health and Related Research, University of Sheffield, Sheffield, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"William","family":"Sones","sequence":"additional","affiliation":[{"name":"Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lisa V","family":"Hampson","sequence":"additional","affiliation":[{"name":"Statistical Methodology and Consulting, Novartis Pharma AG, Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Hewitt","sequence":"additional","affiliation":[{"name":"York Trials Unit, Department of Health Sciences, University of York, York, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesse A","family":"Berlin","sequence":"additional","affiliation":[{"name":"Johnson & Johnson, Titusville, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deborah","family":"Ashby","sequence":"additional","affiliation":[{"name":"Imperial Clinical Trials Unit, Imperial College London, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Emsley","sequence":"additional","affiliation":[{"name":"Department of Biostatistics and Health Informatics, Institute of Psychiatry, Psychology and Neuroscience, King\u2019s College London, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3389-2485","authenticated-orcid":true,"given":"Dean A","family":"Fergusson","sequence":"additional","affiliation":[{"name":"Clinical Epidemiology Program, Ottawa Hospital Research Institute, Ottawa, ON, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephen J","family":"Walters","sequence":"additional","affiliation":[{"name":"Medical Statistics Group, School of Health and Related Research, University of Sheffield, Sheffield, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edward CF","family":"Wilson","sequence":"additional","affiliation":[{"name":"Cambridge Centre for Health Services Research, Cambridge Clinical Trials Unit University of Cambridge, Cambridge, UK"},{"name":"Health Economics Group, Norwich Medical School, University of East Anglia, Norwich, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Graeme","family":"MacLennan","sequence":"additional","affiliation":[{"name":"Centre for Healthcare Randomised Trials, University of Aberdeen, Aberdeen, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nigel","family":"Stallard","sequence":"additional","affiliation":[{"name":"Warwick Medical School, Statistics and Epidemiology, University of Warwick, Coventry, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6275-3807","authenticated-orcid":true,"given":"Joanne C","family":"Rothwell","sequence":"additional","affiliation":[{"name":"Medical Statistics Group, School of Health and Related Research, University of Sheffield, Sheffield, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martin","family":"Bland","sequence":"additional","affiliation":[{"name":"Department of Health Sciences, University of York, York, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Louise","family":"Brown","sequence":"additional","affiliation":[{"name":"MRC Clinical Trials Unit, Institute of Clinical Trials and Methodology, University College London, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig R","family":"Ramsay","sequence":"additional","affiliation":[{"name":"Health Services Research Unit, University of Aberdeen, Aberdeen, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew","family":"Cook","sequence":"additional","affiliation":[{"name":"Wessex Institute, University of Southampton, Southampton, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3652-9662","authenticated-orcid":true,"given":"David","family":"Armstrong","sequence":"additional","affiliation":[{"name":"School of Population Health and Environmental Sciences, King\u2019s College London, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Douglas","family":"Altman","sequence":"additional","affiliation":[{"name":"Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luke D","family":"Vale","sequence":"additional","affiliation":[{"name":"Health Economics Group, Institute of Health & Society, Newcastle University, Newcastle upon Tyne, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"6221","published-online":{"date-parts":[[2019,10]]},"reference":[{"key":"key20191028131707088_ref1-bib1","volume-title":"Sample Sizes for Clinical Trials","author":"Julious","year":"2010"},{"key":"key20191028131707088_ref1-bib2","doi-asserted-by":"publisher","first-page":"75","DOI":"10.1002\/pst.1718","article-title":"Practical guide to sample size calculations: superiority trials","volume":"15","author":"Flight","year":"2016","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib3","doi-asserted-by":"publisher","first-page":"80","DOI":"10.1002\/pst.1716","article-title":"Practical guide to sample size calculations: non-inferiority and equivalence trials","volume":"15","author":"Flight","year":"2016","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib4","doi-asserted-by":"publisher","first-page":"448","DOI":"10.1002\/pst.517","article-title":"The ABC of non-inferiority margin setting from indirect comparisons","volume":"10","author":"Julious","year":"2011","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib5","doi-asserted-by":"publisher","first-page":"12","DOI":"10.1002\/bimj.200410085","article-title":"Choice of delta: requirements and reality \u2013 results of a systematic review","volume":"47","author":"Lange","year":"2005","journal-title":"Biom J"},{"key":"key20191028131707088_ref1-bib6","volume-title":"Guideline on the Choice of Non-Inferiority Margin","author":"Committee for Medicinal Products for Human Use","year":"2005"},{"key":"key20191028131707088_ref1-bib7","doi-asserted-by":"crossref","first-page":"2","DOI":"10.1016\/S0197-2456(01)00196-9","article-title":"Choosing an equivalence limit for noninferiority and equivalence trials","volume":"23","author":"Weins","year":"2002","journal-title":"Control Clin Trials"},{"key":"key20191028131707088_ref1-bib8","doi-asserted-by":"publisher","first-page":"36","DOI":"10.1136\/bmj.313.7048.36","article-title":"Trials to assess equivalence: the importance of rigorous methods","volume":"313","author":"Jones","year":"1996","journal-title":"BMJ"},{"key":"key20191028131707088_ref1-bib9","doi-asserted-by":"publisher","first-page":"542","DOI":"10.1186\/s13063-018-2887-x","article-title":"Choosing the target difference (\u2018effect size\u2019) for a randomised controlled trial \u2013 the development of the DELTA2 guidance","volume":"19","author":"Sones","year":"2018","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib10","first-page":"1905","article-title":"Harmonised tripartite guideline ICH. Statistical principles for clinical trials. International Conference on Harmonisation E9 expert working group","volume":"18","author":"International Council for Harmonisation","year":"1999","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib11","volume-title":"Bayesian Approaches to Clinical Trials and Health-Care Evaluation","author":"Spiegelhalter","year":"2004","edition":"1st"},{"key":"key20191028131707088_ref1-bib12","doi-asserted-by":"publisher","first-page":"305","DOI":"10.1002\/pst.232","article-title":"Sample size and the probability of a successful trial","volume":"5","author":"Chuang-Stein","year":"2006","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib13","doi-asserted-by":"publisher","first-page":"b1732","DOI":"10.1136\/bmj.b1732","article-title":"Reporting of sample size calculation in randomised controlled trials: review","volume":"338","author":"Charles","year":"2009","journal-title":"BMJ"},{"issue":"28","key":"key20191028131707088_ref1-bib14","doi-asserted-by":"publisher","DOI":"10.3310\/hta18280","article-title":"Assessing methods to specify the targeted difference for a randomised controlled trial \u2013 DELTA (Difference ELicitation in TriAls) review","volume":"18","author":"Cook","year":"2014","journal-title":"Health Technol Assess"},{"key":"key20191028131707088_ref1-bib15","doi-asserted-by":"publisher","first-page":"e1001645","DOI":"10.1371\/journal.pmed.1001645","article-title":"Methods for specifying the target difference in a randomised controlled trial: the Difference ELicitation in TriAls (DELTA) systematic review","volume":"11","author":"Hislop","year":"2014","journal-title":"PLOS Med"},{"key":"key20191028131707088_ref1-bib16","doi-asserted-by":"publisher","first-page":"300","DOI":"10.1177\/1740774514521907","article-title":"Use of methods for specifying the target difference in randomised controlled trial sample size calculations: two surveys of trialists\u2019 practice","volume":"11","author":"Cook","year":"2014","journal-title":"Clin Trials"},{"key":"key20191028131707088_ref1-bib17","doi-asserted-by":"publisher","first-page":"407","DOI":"10.1016\/0197-2456(89)90005-6","article-title":"Measurement of health status. Ascertaining the minimal clinically important difference","volume":"10","author":"Jaeschke","year":"1989","journal-title":"Control Clin Trials"},{"key":"key20191028131707088_ref1-bib18","doi-asserted-by":"publisher","first-page":"419","DOI":"10.2165\/00019053-200018050-00001","article-title":"The concept of clinically meaningful difference in health-related quality-of-life research. How meaningful is it?","volume":"18","author":"Hays","year":"2000","journal-title":"PharmacoEconomics"},{"key":"key20191028131707088_ref1-bib19","first-page":"1197","article-title":"How well is the clinical importance of study results reported? An assessment of randomized controlled trials","volume":"165","author":"Chan","year":"2001","journal-title":"CMAJ"},{"key":"key20191028131707088_ref1-bib20","doi-asserted-by":"publisher","first-page":"12","DOI":"10.1186\/s13063-014-0526-8","article-title":"Specifying the target difference in the primary outcome for a randomised controlled trial: guidance for researchers","volume":"16","author":"Cook","year":"2015","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib21","doi-asserted-by":"publisher","first-page":"11","DOI":"10.1186\/1471-2288-10-11","article-title":"Association between framing of the research question using the PICOT format and reporting quality of randomized controlled trials","volume":"10","author":"Rios","year":"2010","journal-title":"BMC Med Res Methodol"},{"key":"key20191028131707088_ref1-bib22","doi-asserted-by":"publisher","first-page":"5","DOI":"10.1002\/sim.7033","article-title":"Estimands in clinical trials \u2013 broadening the perspective","volume":"36","author":"Akacha","year":"2017","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib23","volume-title":"ICH E9 (R1) Addendum on Estimands and Sensitivity Analysis in Clinical Trials to the Guideline on Statistical Principles for Clinical Trials","author":"Committee for Human Medicinal Products","year":"2017"},{"key":"key20191028131707088_ref1-bib24","doi-asserted-by":"publisher","first-page":"12","DOI":"10.1002\/pst.1791","article-title":"Disentangling estimands and the intention-to-treat principle","volume":"16","author":"Leuchs","year":"2017","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib25","doi-asserted-by":"publisher","first-page":"6","DOI":"10.1002\/pst.1745","article-title":"Estimands: discussion points from the PSI estimands and sensitivity expert group","volume":"16","author":"Phillips","year":"2017","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib26","doi-asserted-by":"publisher","first-page":"456","DOI":"10.1002\/pst.1536","article-title":"A structured approach to choosing estimands and estimators in longitudinal clinical trials","volume":"11","author":"Mallinckrodt","year":"2012","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib27","doi-asserted-by":"publisher","first-page":"104","DOI":"10.1186\/1471-2288-13-104","article-title":"An audit of sample sizes for pilot and feasibility trials being undertaken in the United Kingdom registered in the United Kingdom Clinical Research Network database","volume":"13","author":"Billingham","year":"2013","journal-title":"BMC Med Res Methodol"},{"key":"key20191028131707088_ref1-bib28","volume-title":"Involve 2017","author":"National Institute for Health Research"},{"key":"key20191028131707088_ref1-bib29","volume-title":"Public Involvement 2017","author":"National Institute for Health and Care Excellence"},{"key":"key20191028131707088_ref1-bib30","volume-title":"WMA Declaration of Helsinki \u2013 Ethical Principles for Medical Research Involving Human Subjects","author":"World Medical Association","year":"2013"},{"key":"key20191028131707088_ref1-bib31","doi-asserted-by":"publisher","first-page":"804","DOI":"10.1016\/S0140-6736(97)02290-3","article-title":"Why \u2018underpowered\u2019 trials are not necessarily unethical","volume":"350","author":"Edwards","year":"1997","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib32","doi-asserted-by":"publisher","first-page":"1348","DOI":"10.1016\/S0140-6736(05)61034-3","article-title":"Sample size calculations in randomised trials: mandatory and mystical","volume":"365","author":"Schulz","year":"2005","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib33","doi-asserted-by":"publisher","first-page":"93","DOI":"10.2307\/2983471","article-title":"Multiplicity considerations in the design and analysis of clinical trials","volume":"159","author":"Cook","year":"1996","journal-title":"J R Stat Soc"},{"key":"key20191028131707088_ref1-bib34","doi-asserted-by":"publisher","first-page":"1591","DOI":"10.1016\/S0140-6736(05)66461-6","article-title":"Multiplicity in randomised trials I: endpoints and treatments","volume":"365","author":"Schulz","year":"2005","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib35","doi-asserted-by":"publisher","first-page":"701","DOI":"10.1001\/archopht.120.6.701","article-title":"The Ocular Hypertension Treatment Study: a randomized trial determines that topical ocular hypotensive medication delays or prevents the onset of primary open-angle glaucoma","volume":"120","author":"Kass","year":"2002","journal-title":"Arch Ophthalmol"},{"issue":"29","key":"key20191028131707088_ref1-bib36","doi-asserted-by":"publisher","DOI":"10.3310\/hta16290","article-title":"Surveillance for ocular hypertension: an evidence synthesis and economic evaluation","volume":"16","author":"Burr","year":"2012","journal-title":"Health Technol Assess"},{"key":"key20191028131707088_ref1-bib37","doi-asserted-by":"publisher","first-page":"118","DOI":"10.1002\/pst.505","article-title":"A convenient formula for sample size calculations in clinical trials with multiple co-primary continuous endpoints","volume":"11","author":"Sugimoto","year":"2012","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib38","doi-asserted-by":"crossref","DOI":"10.1002\/9780470840481","volume-title":"Quality of Life Outcomes in Clinical Trials and Health-Care Evaluation: A Practical Guide to Analysis and Interpretation","author":"Walters","year":"2009"},{"key":"key20191028131707088_ref1-bib39","doi-asserted-by":"publisher","first-page":"26","DOI":"10.1186\/1477-7525-2-26","article-title":"Sample size and power estimation for studies with health related quality of life outcomes: a comparison of four methods using the SF-36","volume":"2","author":"Walters","year":"2004","journal-title":"Health Qual Life Outcomes"},{"key":"key20191028131707088_ref1-bib40","doi-asserted-by":"publisher","first-page":"3189","DOI":"10.1002\/sim.3603","article-title":"Measurement in clinical trials: a neglected issue for statisticians?","volume":"28","author":"Senn","year":"2009","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib41","doi-asserted-by":"publisher","first-page":"3220","DOI":"10.1002\/sim.3658","article-title":"Commentary on \u2018Measurement in clinical trials: a neglected issue for statisticians?\u2019","volume":"28","author":"Wittes","year":"2009","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib42","doi-asserted-by":"publisher","first-page":"1099","DOI":"10.1016\/S0140-6736(14)61231-9","article-title":"Integrated collaborative care for comorbid major depression in patients with cancer (SMaRT Oncology-2): a multicentre randomised controlled effectiveness trial","volume":"384","author":"Sharpe","year":"2014","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib43","doi-asserted-by":"publisher","first-page":"670","DOI":"10.1136\/bmj.319.7211.670","article-title":"What is meant by intention to treat analysis? Survey of published randomised controlled trials","volume":"319","author":"Hollis","year":"1999","journal-title":"BMJ"},{"key":"key20191028131707088_ref1-bib44","doi-asserted-by":"publisher","first-page":"4","DOI":"10.1002\/pst.1792","article-title":"Estimands \u2013 new statistical principle or the emperor\u2019s new clothes?","volume":"16","author":"Rosenkranz","year":"2017","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib45","doi-asserted-by":"publisher","first-page":"541","DOI":"10.1016\/j.spinee.2007.01.008","article-title":"Understanding the minimum clinically important difference: a review of concepts and methods","volume":"7","author":"Copay","year":"2007","journal-title":"Spine J"},{"key":"key20191028131707088_ref1-bib46","first-page":"406","article-title":"Minimal clinically important differences: review of methods","volume":"28","author":"Wells","year":"2001","journal-title":"J Rheumatol"},{"key":"key20191028131707088_ref1-bib47","doi-asserted-by":"publisher","first-page":"109","DOI":"10.1097\/00002281-200203000-00006","article-title":"Many faces of the minimal clinically important difference (MICD): a literature review and directions for future research","volume":"14","author":"Beaton","year":"2002","journal-title":"Curr Opin Rheumatol"},{"key":"key20191028131707088_ref1-bib48","doi-asserted-by":"publisher","first-page":"177","DOI":"10.1016\/j.rdc.2018.01.011","article-title":"Minimal Clinically Important Difference: A Review of Outcome Measure Score Interpretation","volume":"44","author":"Engel","year":"2018","journal-title":"Rheum Dis Clin North Am"},{"key":"key20191028131707088_ref1-bib49","doi-asserted-by":"publisher","first-page":"213","DOI":"10.1054\/bjoc.1999.0902","article-title":"Sample size calculation for clinical trials: the impact of clinician beliefs","volume":"82","author":"Fayers","year":"2000","journal-title":"Br J Cancer"},{"key":"key20191028131707088_ref1-bib50","doi-asserted-by":"publisher","first-page":"427","DOI":"10.1093\/ije\/30.3.427","article-title":"Sick individuals and sick populations","volume":"30","author":"Rose","year":"2001","journal-title":"Int J Epidemiol"},{"key":"key20191028131707088_ref1-bib51","doi-asserted-by":"publisher","first-page":"129","DOI":"10.1016\/S0046-8177(71)80023-0","article-title":"Serum lipid precursors of coronary heart disease","volume":"2","author":"Kannel","year":"1971","journal-title":"Hum Pathol"},{"key":"key20191028131707088_ref1-bib52","doi-asserted-by":"publisher","first-page":"371","DOI":"10.4065\/77.4.371","article-title":"Methods to explain the clinical significance of health status measures","volume":"77","author":"Guyatt","year":"2002","journal-title":"Mayo Clin Proc"},{"key":"key20191028131707088_ref1-bib53","doi-asserted-by":"publisher","first-page":"37","DOI":"10.1016\/j.jclinepi.2009.03.011","article-title":"Three ways to quantify uncertainty in individually applied \u2018minimally important change\u2019 values","volume":"63","author":"de Vet","year":"2010","journal-title":"J Clin Epidemiol"},{"key":"key20191028131707088_ref1-bib54","doi-asserted-by":"publisher","first-page":"384","DOI":"10.4065\/77.4.384","article-title":"Group vs individual approaches to understanding the clinical significance of differences or changes in quality of life","volume":"77","author":"Cella","year":"2002","journal-title":"Mayo Proc"},{"issue":"19","key":"key20191028131707088_ref1-bib55","doi-asserted-by":"publisher","DOI":"10.3310\/hta18190","article-title":"A randomised controlled trial of the clinical effectiveness and cost-effectiveness of different knee prostheses: the Knee Arthroplasty Trial (KAT)","volume":"18","author":"Murray","year":"2014","journal-title":"Health Technol Assess"},{"key":"key20191028131707088_ref1-bib56","doi-asserted-by":"publisher","first-page":"73","DOI":"10.1016\/j.jclinepi.2014.08.009","article-title":"Meaningful changes for the Oxford hip and knee scores after joint replacement surgery","volume":"68","author":"Beard","year":"2015","journal-title":"J Clin Epidemiol"},{"key":"key20191028131707088_ref1-bib57","doi-asserted-by":"publisher","first-page":"163","DOI":"10.1002\/pst.334","article-title":"Consultants\u2019 forum: should post hoc sample size calculations be done?","volume":"8","author":"Walters","year":"2009","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib58","doi-asserted-by":"publisher","first-page":"430","DOI":"10.1177\/0962280213476379","article-title":"Estimating effect sizes for health-related quality of life outcomes","volume":"23","author":"Julious","year":"2014","journal-title":"Stat Methods Med Res"},{"key":"key20191028131707088_ref1-bib59","doi-asserted-by":"publisher","first-page":"2593","DOI":"10.1002\/(SICI)1097-0258(19991015)18:19<2593::AID-SIM392>3.0.CO;2-T","article-title":"Examining the minimum important difference","volume":"18","author":"Brant","year":"1999","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib60","doi-asserted-by":"publisher","first-page":"1347","DOI":"10.1161\/STROKEAHA.108.531442","article-title":"Using historical lesion volume data in the design of a new phase II clinical trial in acute stroke","volume":"40","author":"Whitehead","year":"2009","journal-title":"Stroke"},{"key":"key20191028131707088_ref1-bib61","doi-asserted-by":"publisher","first-page":"12","DOI":"10.1037\/0022-006X.59.1.12","article-title":"Clinical significance: a statistical approach to defining meaningful change in psychotherapy research","volume":"59","author":"Jacobson","year":"1991","journal-title":"J Consult Clin Psychol"},{"key":"key20191028131707088_ref1-bib62","doi-asserted-by":"publisher","first-page":"91","DOI":"10.1016\/j.jad.2006.07.001","article-title":"Evaluating the clinical significance of responses by psychiatric inpatients to the mental health subscales of the SF-36","volume":"98","author":"Newnham","year":"2007","journal-title":"J Affect Disord"},{"key":"key20191028131707088_ref1-bib63","doi-asserted-by":"publisher","first-page":"173","DOI":"10.1002\/sim.4780090124","article-title":"Using cost-effectiveness analysis to improve the efficiency of allocating funds to clinical trials","volume":"9","author":"Detsky","year":"1990","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib64","first-page":"517","article-title":"Economics in sample size determination for clinical trials","volume":"88","author":"Torgerson","year":"1995","journal-title":"QJM"},{"key":"key20191028131707088_ref1-bib65","doi-asserted-by":"publisher","first-page":"43","DOI":"10.1177\/1740774512465358","article-title":"Cost-utility analysis conducted alongside randomized controlled trials: are economic end points considered in sample size calculations and does it matter?","volume":"10","author":"Hollingworth","year":"2013","journal-title":"Clin Trials"},{"key":"key20191028131707088_ref1-bib66","doi-asserted-by":"publisher","first-page":"189","DOI":"10.2165\/11585070-000000000-00000","article-title":"Sample size and power for cost-effectiveness analysis (part 1)","volume":"29","author":"Glick","year":"2011","journal-title":"PharmacoEconomics"},{"key":"key20191028131707088_ref1-bib67","doi-asserted-by":"publisher","first-page":"287","DOI":"10.2165\/11585080-000000000-00000","article-title":"Sample size and power for cost-effectiveness analysis (Part 2): the effect of maximum willingness to pay","volume":"29","author":"Glick","year":"2011","journal-title":"PharmacoEconomics"},{"key":"key20191028131707088_ref1-bib68","unstructured":"National Institute for Health and Care Excellence (NICE). NICE Process and Methods Guides. Guide to the Methods of Technology Appraisal. London: NICE; 2013."},{"key":"key20191028131707088_ref1-bib69","doi-asserted-by":"crossref","DOI":"10.1002\/0470033312","volume-title":"Uncertain Judgements: Eliciting Experts\u2019 Probabilities","author":"O\u2019Hagan","year":"2006"},{"key":"key20191028131707088_ref1-bib70","volume-title":"Methods for Eliciting Expert Opinion to Inform Health Technology Assessment","author":"Gosling","year":"2014"},{"key":"key20191028131707088_ref1-bib71","doi-asserted-by":"crossref","DOI":"10.1007\/978-1-4020-5753-3","volume-title":"Using Discrete Choice Experiments to Value Health and Health Care","author":"Ryan","year":"2008"},{"key":"key20191028131707088_ref1-bib72","doi-asserted-by":"publisher","first-page":"667","DOI":"10.1002\/pds.3636","article-title":"Balancing benefit and risk of medicines: a systematic review and classification of available methodologies","volume":"23","author":"Mt-Isa","year":"2014","journal-title":"Pharmacoepidemiol Drug Saf"},{"key":"key20191028131707088_ref1-bib73","doi-asserted-by":"publisher","first-page":"250","DOI":"10.1177\/0272989X05276863","article-title":"Sufficiently important difference: expanding the framework of clinical significance","volume":"25","author":"Barrett","year":"2005","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib74","first-page":"1908","article-title":"Rheumatoid arthritis antirheumatic drug trials. III. Setting the delta for clinical trials of antirheumatic drugs \u2013 results of a consensus development (Delphi) exercise","volume":"18","author":"Bellamy","year":"1991","journal-title":"J Rheumatol"},{"key":"key20191028131707088_ref1-bib75","volume-title":"Software to Support Expert Elicitation. An Exploratory Study of Existing Software Packages","author":"Devilee","year":"2011"},{"key":"key20191028131707088_ref1-bib76","doi-asserted-by":"publisher","first-page":"812","DOI":"10.1002\/gps.2607","article-title":"Determining the minimum clinically important differences for outcomes in the DOMINO trial","volume":"26","author":"Howard","year":"2011","journal-title":"Int J Geriatr Psychiatry"},{"key":"key20191028131707088_ref1-bib77","doi-asserted-by":"publisher","first-page":"e0120981","DOI":"10.1371\/journal.pone.0120981","article-title":"Elicitation of expert prior opinion: application to the MYPAN trial in childhood polyarteritis nodosa","volume":"10","author":"Hampson","year":"2015","journal-title":"PLOS ONE"},{"key":"key20191028131707088_ref1-bib78","doi-asserted-by":"publisher","first-page":"89","DOI":"10.1186\/1471-2288-11-89","article-title":"Using e-mail recruitment and an online questionnaire to establish effect size: a worked example","volume":"11","author":"Kirkby","year":"2011","journal-title":"BMC Med Res Methodol"},{"key":"key20191028131707088_ref1-bib79","doi-asserted-by":"publisher","first-page":"375","DOI":"10.1016\/S0140-6736(01)05558-1","article-title":"Monitoring of large randomised clinical trials: a new approach with Bayesian methods","volume":"358","author":"Parmar","year":"2001","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib80","doi-asserted-by":"publisher","first-page":"1297","DOI":"10.1002\/sim.4780131304","article-title":"The CHART trials: Bayesian design and monitoring in practice. CHART Steering Committee","volume":"13","author":"Parmar","year":"1994","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib81","doi-asserted-by":"publisher","first-page":"581","DOI":"10.1002\/sim.694","article-title":"Quantifying and documenting prior beliefs in clinical trials","volume":"20","author":"Chaloner","year":"2001","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib82","doi-asserted-by":"publisher","first-page":"4186","DOI":"10.1002\/sim.6225","article-title":"Bayesian methods for the design and interpretation of clinical trials in very rare diseases","volume":"33","author":"Hampson","year":"2014","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib83","doi-asserted-by":"publisher","first-page":"68","DOI":"10.1177\/0272989X09340583","article-title":"Sample size in obesity trials: patient perspective versus current practice","volume":"30","author":"Allison","year":"2010","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib84","doi-asserted-by":"publisher","first-page":"67","DOI":"10.1186\/1471-2288-10-67","article-title":"What is a pilot or feasibility study? A review of current practice and editorial policy","volume":"10","author":"Arain","year":"2010","journal-title":"BMC Med Res Methodol"},{"key":"key20191028131707088_ref1-bib85","doi-asserted-by":"publisher","first-page":"484","DOI":"10.1001\/archpsyc.63.5.484","article-title":"Caution regarding the use of pilot studies to guide power calculations for study proposals","volume":"63","author":"Kraemer","year":"2006","journal-title":"Arch Gen Psychiatry"},{"key":"key20191028131707088_ref1-bib86","doi-asserted-by":"publisher","first-page":"197","DOI":"10.1016\/j.jclinepi.2012.09.002","article-title":"Sample size calculations for pilot randomized trials: a confidence interval\u2009approach","volume":"66","author":"Cocks","year":"2013","journal-title":"J Clin Epidemiol"},{"key":"key20191028131707088_ref1-bib87","doi-asserted-by":"publisher","first-page":"264","DOI":"10.1186\/1745-6215-15-264","article-title":"Sample size requirements to estimate key design parameters from external pilot randomised controlled trials: a simulation study","volume":"15","author":"Teare","year":"2014","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib88","doi-asserted-by":"publisher","first-page":"1057","DOI":"10.1177\/0962280215588241","article-title":"Estimating the sample size for a pilot randomised trial to minimise the overall trial sample size for the external pilot and main trial for a continuous outcome variable","volume":"25","author":"Whitehead","year":"2016","journal-title":"Stat Methods Med Res"},{"key":"key20191028131707088_ref1-bib89","doi-asserted-by":"publisher","first-page":"g4606","DOI":"10.1136\/bmj.g4606","article-title":"Predicting risk of upper gastrointestinal bleed and intracranial bleed with anticoagulants: cohort study to derive and validate the QBleed scores","volume":"349","author":"Hippisley-Cox","year":"2014","journal-title":"BMJ"},{"key":"key20191028131707088_ref1-bib90","doi-asserted-by":"publisher","first-page":"20","DOI":"10.1016\/S0140-6736(10)61045-8","article-title":"Clinical trials should begin and end with systematic reviews of relevant evidence: 12 years and waiting","volume":"376","author":"Clarke","year":"2010","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib91","doi-asserted-by":"publisher","first-page":"29","DOI":"10.1186\/1471-2288-9-29","article-title":"Evidence synthesis as the key to more coherent and efficient research","volume":"9","author":"Sutton","year":"2009","journal-title":"BMC Med Res Methodol"},{"key":"key20191028131707088_ref1-bib92","doi-asserted-by":"publisher","first-page":"625","DOI":"10.1002\/sim.2934","article-title":"Recent developments in meta-analysis","volume":"27","author":"Sutton","year":"2008","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib93","doi-asserted-by":"publisher","first-page":"358","DOI":"10.1001\/jama.288.3.358","article-title":"The continuing unethical conduct of underpowered clinical trials","volume":"288","author":"Halpern","year":"2002","journal-title":"JAMA"},{"key":"key20191028131707088_ref1-bib94","volume-title":"Statistical Power Analysis for the Behavioral Sciences","author":"Cohen","year":"1977","edition":"Revised edn"},{"key":"key20191028131707088_ref1-bib95","volume-title":"Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0","author":"Higgins","year":"2011"},{"key":"key20191028131707088_ref1-bib96","doi-asserted-by":"publisher","first-page":"3127","DOI":"10.1002\/1097-0258(20001130)19:22<3127::AID-SIM784>3.0.CO;2-M","article-title":"A simple method for converting an odds ratio to effect size for use in meta-analysis","volume":"19","author":"Chinn","year":"2000","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib97","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.MR000009.pub4","article-title":"Outcomes of patients who participate in randomized controlled trials compared to similar patients receiving similar interventions who do not participate","volume":"3","author":"Vist","year":"2008","journal-title":"Cochrane Database Syst Rev"},{"key":"key20191028131707088_ref1-bib98","doi-asserted-by":"publisher","first-page":"200","DOI":"10.7326\/0003-4819-121-3-199408010-00008","article-title":"The use of predicted confidence intervals when planning experiments and the misuse of power when interpreting results","volume":"121","author":"Goodman","year":"1994","journal-title":"Ann Intern Med"},{"key":"key20191028131707088_ref1-bib99","doi-asserted-by":"publisher","first-page":"1657","DOI":"10.1016\/S0140-6736(05)66516-6","article-title":"Multiplicity in randomised trials II: subgroup and interim analyses","volume":"365","author":"Schulz","year":"2005","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib100","doi-asserted-by":"publisher","first-page":"328","DOI":"10.1016\/S0140-6736(11)60751-4","article-title":"Urinary incontinence in men after formal one-to-one pelvic-floor muscle training following radical prostatectomy or transurethral resection of the prostate (MAPS): two parallel randomised controlled trials","volume":"378","author":"Glazener","year":"2011","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib101","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD001843.pub3","article-title":"Conservative management for postprostatectomy urinary incontinence","volume":"2","author":"Hunter","year":"2007","journal-title":"Cochrane Database Syst Rev"},{"key":"key20191028131707088_ref1-bib102","doi-asserted-by":"publisher","first-page":"1586","DOI":"10.1167\/iovs.10-6287","article-title":"Internal limiting membrane peeling versus no peeling for idiopathic full-thickness macular hole: a pragmatic randomized controlled trial","volume":"52","author":"Lois","year":"2011","journal-title":"Invest Ophthalmol Vis Sci"},{"key":"key20191028131707088_ref1-bib103","doi-asserted-by":"publisher","first-page":"741","DOI":"10.1016\/S0161-6420(13)38009-9","article-title":"Early Treatment Diabetic Retinopathy Study design and baseline patient characteristics. ETDRS report number 7","volume":"98","author":"Early Treatment Diabetic Retinopathy Study (ETDRS) Research Group","year":"1991","journal-title":"Ophthalmology"},{"key":"key20191028131707088_ref1-bib104","doi-asserted-by":"publisher","first-page":"1939","DOI":"10.1016\/S0161-6420(00)00331-6","article-title":"Macular hole surgery with and without internal limiting membrane peeling","volume":"107","author":"Brooks","year":"2000","journal-title":"Ophthalmology"},{"key":"key20191028131707088_ref1-bib105","doi-asserted-by":"publisher","first-page":"932","DOI":"10.1016\/S0161-6420(99)00512-6","article-title":"Effect of autologous platelet concentrate in surgery for idiopathic macular hole: results of a multicenter, double-masked, randomized trial. Platelets in Macular Hole Surgery Group","volume":"106","author":"Paques","year":"1999","journal-title":"Ophthalmology"},{"key":"key20191028131707088_ref1-bib106","doi-asserted-by":"publisher","first-page":"7","DOI":"10.1186\/1745-6215-7-7","article-title":"Protocol for the Arterial Revascularisation Trial (ART). A randomised trial to compare survival following bilateral versus single internal mammary grafting in coronary revascularisation [ISRCTN46552265].","volume":"7","author":"Taggart","year":"2006","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib107","doi-asserted-by":"publisher","first-page":"870","DOI":"10.1016\/S0140-6736(01)06069-X","article-title":"Effect of arterial revascularisation on survival: a systematic review of studies comparing bilateral and single internal mammary arteries","volume":"358","author":"Taggart","year":"2001","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib108","doi-asserted-by":"publisher","first-page":"c332","DOI":"10.1136\/bmj.c332","article-title":"CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials","volume":"340","author":"Schulz","year":"2010","journal-title":"BMJ"},{"key":"key20191028131707088_ref1-bib109","doi-asserted-by":"publisher","first-page":"200","DOI":"10.7326\/0003-4819-158-3-201302050-00583","article-title":"SPIRIT 2013 statement: defining standard protocol items for clinical trials","volume":"158","author":"Chan","year":"2013","journal-title":"Ann Intern Med"},{"key":"key20191028131707088_ref1-bib110","article-title":"Optimal Pathway for TreatIng neurOpathic paiN in Diabetes Mellitus (OPTION-DM) Trial","author":"Tesfaye","year":"2019","journal-title":"Health Technol Assess"},{"key":"key20191028131707088_ref1-bib111","doi-asserted-by":"publisher","first-page":"238","DOI":"10.1186\/1745-6215-15-238","article-title":"Use of drug therapy in the management of symptomatic ureteric stones in hospitalized adults (SUSPEND), a multicentre, placebo-controlled, randomized trial of a calcium-channel blocker (nifedipine) and an \u03b1-blocker (tamsulosin): study protocol for a randomized controlled trial","volume":"15","author":"McClinton","year":"2014","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib112","doi-asserted-by":"publisher","first-page":"230","DOI":"10.1186\/s13063-019-3318-3","article-title":"Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme)","volume":"20","author":"Blackshaw","year":"2019","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib113","doi-asserted-by":"publisher","first-page":"246","DOI":"10.1186\/s13063-019-3314-7","article-title":"Clarithromycin and endoscopic sinus surgery for adults with chronic rhinosinusitis with and without nasal polyps: study protocol for the MACRO randomised controlled trial","volume":"20","author":"Philpott","year":"2019","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib114","doi-asserted-by":"publisher","first-page":"e1002115","DOI":"10.1371\/journal.pmed.1002115","article-title":"An audit and feedback intervention for reducing antibiotic prescribing in general dental practice: the RAPiD Cluster Randomised Controlled Trial","volume":"13","author":"Elouafkaoui","year":"2016","journal-title":"PLOS Med"},{"key":"key20191028131707088_ref1-bib115","doi-asserted-by":"publisher","first-page":"f232","DOI":"10.1136\/bmj.f232","article-title":"Treatment for acute anterior cruciate ligament tear: five year outcome of randomised trial","volume":"346","author":"Frobell","year":"2013","journal-title":"BMJ"},{"key":"key20191028131707088_ref1-bib116","doi-asserted-by":"publisher","first-page":"331","DOI":"10.1056\/NEJMoa0907797","article-title":"A randomized trial of treatment for acute anterior cruciate ligament tears","volume":"363","author":"Frobell","year":"2010","journal-title":"N Engl J Med"},{"key":"key20191028131707088_ref1-bib117","volume-title":"The 2012 User\u2019s Guide to: Knee injury and Osteoarthritis Outcome Score KOOS","author":"Roos","year":"2012"},{"key":"key20191028131707088_ref1-bib118","doi-asserted-by":"publisher","first-page":"64","DOI":"10.1186\/1477-7525-1-64","article-title":"The Knee injury and Osteoarthritis Outcome Score (KOOS): from joint injury to osteoarthritis","volume":"1","author":"Roos","year":"2003","journal-title":"Health Qual Life Outcomes"},{"key":"key20191028131707088_ref1-bib119","doi-asserted-by":"publisher","first-page":"208","DOI":"10.1002\/acr.20632","article-title":"Measures of knee function: International Knee Documentation Committee (IKDC) Subjective Knee Evaluation Form, Knee Injury and Osteoarthritis Outcome Score (KOOS), Knee Injury and Osteoarthritis Outcome Score Physical Function Short Form (KOOS-PS), Knee Outcome Survey Activities of Daily Living Scale (KOS-ADL), Lysholm Knee Scoring Scale, Oxford Knee Score (OKS), Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), Activity Rating Scale (ARS), and Tegner Activity Score (TAS)","volume":"63","author":"Collins","year":"2011","journal-title":"Arthritis Care Res"},{"key":"key20191028131707088_ref1-bib120","doi-asserted-by":"publisher","first-page":"159","DOI":"10.1177\/1536867X1401400111","article-title":"Sample size and power calculations for trials and quasi-experimental studies with clustering","volume":"14","author":"Batistatou","year":"2014","journal-title":"Stata J"},{"key":"key20191028131707088_ref1-bib121","doi-asserted-by":"publisher","first-page":"2","DOI":"10.1186\/1745-6215-13-2","article-title":"Clustering in surgical trials \u2013 database of intracluster correlations","volume":"13","author":"Cook","year":"2012","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib122","doi-asserted-by":"publisher","first-page":"1324","DOI":"10.1056\/NEJMoa042580","article-title":"Morphine, gabapentin, or their combination for neuropathic pain","volume":"352","author":"Gilron","year":"2005","journal-title":"N Engl J Med"},{"key":"key20191028131707088_ref1-bib123","doi-asserted-by":"publisher","first-page":"105","DOI":"10.1016\/j.jpain.2007.09.005","article-title":"Interpreting the clinical importance of treatment outcomes in chronic pain clinical trials: IMMPACT recommendations","volume":"9","author":"Dworkin","year":"2008","journal-title":"J Pain"},{"key":"key20191028131707088_ref1-bib124","doi-asserted-by":"publisher","first-page":"1921","DOI":"10.1002\/sim.1783","article-title":"Sample sizes for clinical trials with normal data","volume":"23","author":"Julious","year":"2004","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib125","doi-asserted-by":"publisher","first-page":"1171","DOI":"10.1016\/S0140-6736(06)69474-9","article-title":"Medical therapy to facilitate urinary stone passage: a meta-analysis","volume":"368","author":"Hollingsworth","year":"2006","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib126","doi-asserted-by":"publisher","first-page":"552","DOI":"10.1016\/j.annemergmed.2007.05.015","article-title":"A systematic review of medical therapy to facilitate passage of ureteral calculi","volume":"50","author":"Singh","year":"2007","journal-title":"Ann Emerg Med"},{"key":"key20191028131707088_ref1-bib127","first-page":"A9","article-title":"Survey of the effect of tamsulosin and nifedipine on facilitating juxtavesical ureteral stone passage","volume":"19","author":"Taghavi","year":"2005","journal-title":"J Endourol"},{"key":"key20191028131707088_ref1-bib128","doi-asserted-by":"publisher","first-page":"568","DOI":"10.1097\/01.ju.0000132390.61756.ff","article-title":"Nifedipine versus tamsulosin for the management of lower ureteral stones","volume":"172","author":"Porpiglia","year":"2004","journal-title":"J Urol"},{"key":"key20191028131707088_ref1-bib129","doi-asserted-by":"publisher","first-page":"167","DOI":"10.1097\/01.ju.0000161600.54732.86","article-title":"Randomized trial of the efficacy of tamsulosin, nifedipine and phloroglucinol in medical expulsive therapy for distal ureteral calculi","volume":"174","author":"Dellabella","year":"2005","journal-title":"J Urol"},{"key":"key20191028131707088_ref1-bib130","doi-asserted-by":"publisher","first-page":"525","DOI":"10.1002\/alr.21315","article-title":"The fate of chronic rhinosinusitis sufferers after maximal medical therapy","volume":"4","author":"Baguley","year":"2014","journal-title":"Int Forum Allergy Rhinol"},{"key":"key20191028131707088_ref1-bib131","doi-asserted-by":"publisher","first-page":"e8","DOI":"10.2500\/ar.2012.3.0027","article-title":"Efficacy of medical therapy in treatment of chronic rhinosinusitis","volume":"3","author":"Young","year":"2012","journal-title":"Allergy Rhinol"},{"key":"key20191028131707088_ref1-bib132","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD006991.pub2","article-title":"Surgical versus medical interventions for chronic rhinosinusitis with nasal polyps","volume":"12","author":"Rimmer","year":"2014","journal-title":"Cochrane Database Syst Rev"},{"key":"key20191028131707088_ref1-bib133","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD006990.pub2","article-title":"Surgical interventions for chronic rhinosinusitis with nasal polyps","volume":"11","author":"Sharma","year":"2014","journal-title":"Cochrane Database Syst Rev"},{"key":"key20191028131707088_ref1-bib134","doi-asserted-by":"publisher","first-page":"447","DOI":"10.1111\/j.1749-4486.2009.01995.x","article-title":"Psychometric validity of the 22-item Sinonasal Outcome Test","volume":"34","author":"Hopkins","year":"2009","journal-title":"Clin Otolaryngol"},{"key":"key20191028131707088_ref1-bib135","doi-asserted-by":"publisher","first-page":"582","DOI":"10.1097\/01.MLR.0000062554.74615.4C","article-title":"Interpretation of changes in health-related quality of life: the remarkable universality of half a standard deviation","volume":"41","author":"Norman","year":"2003","journal-title":"Med Care"},{"key":"key20191028131707088_ref1-bib136","doi-asserted-by":"publisher","first-page":"581","DOI":"10.1586\/14737167.4.5.581","article-title":"The truly remarkable universality of half a standard deviation: confirmation through another look","volume":"4","author":"Norman","year":"2004","journal-title":"Expert Rev Pharmacoecon Outcomes Res"},{"key":"key20191028131707088_ref1-bib137","doi-asserted-by":"publisher","first-page":"600","DOI":"10.3109\/02770903.2014.995308","article-title":"Managing chronic rhinosinusitis and respiratory disease: a qualitative study of triggers and interactions","volume":"52","author":"Erskine","year":"2015","journal-title":"J Asthma"},{"key":"key20191028131707088_ref1-bib138","doi-asserted-by":"publisher","first-page":"125","DOI":"10.1177\/0194599814541627a300","article-title":"Preliminary findings: the feasibility study for a randomized controlled trial of clarithromycin in chronic rhinosinusitis","volume":"151","author":"Bewick","year":"2014","journal-title":"Otolaryngol Head Neck Surg Endosc"},{"key":"key20191028131707088_ref1-bib139","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD000259.pub3","article-title":"Audit and feedback: effects on professional practice and healthcare outcomes","volume":"6","author":"Ivers","year":"2012","journal-title":"Cochrane Database Syst Rev"},{"key":"key20191028131707088_ref1-bib140","doi-asserted-by":"publisher","first-page":"1234","DOI":"10.1016\/j.jclinepi.2007.02.006","article-title":"A simple sample size formula for analysis of covariance in randomized clinical trials","volume":"60","author":"Borm","year":"2007","journal-title":"J Clin Epidemiol"},{"key":"key20191028131707088_ref1-bib141","doi-asserted-by":"publisher","first-page":"50","DOI":"10.1186\/1748-5908-9-50","article-title":"Evaluating an audit and feedback intervention for reducing antibiotic prescribing behaviour in general dental practice (the RAPiD trial): a partial factorial cluster randomised trial protocol","volume":"9","author":"Prior","year":"2014","journal-title":"Implement Sci"},{"key":"key20191028131707088_ref1-bib142","doi-asserted-by":"publisher","first-page":"33","DOI":"10.1111\/biom.12251","article-title":"Minimum clinically important difference in medical studies","volume":"71","author":"Hedayat","year":"2015","journal-title":"Biometrics"},{"key":"key20191028131707088_ref1-bib143","doi-asserted-by":"publisher","first-page":"433","DOI":"10.1016\/j.jclinepi.2013.10.009","article-title":"The minimal clinically important difference determined using item response theory models: an attempt to solve the issue of the association with baseline score","volume":"67","author":"Rouquette","year":"2014","journal-title":"J Clin Epidemiol"},{"key":"key20191028131707088_ref1-bib144","doi-asserted-by":"publisher","first-page":"888","DOI":"10.1016\/j.jclinepi.2015.02.017","article-title":"Although not consistently superior, the absolute approach to framing the minimally important difference has advantages over the relative approach","volume":"68","author":"Zhang","year":"2015","journal-title":"J Clin Epidemiol"},{"key":"key20191028131707088_ref1-bib145","doi-asserted-by":"publisher","first-page":"54","DOI":"10.1177\/1740774512467404","article-title":"Determining optimal sample sizes for multistage adaptive randomized clinical trials from an industry perspective using value of information methods","volume":"10","author":"Chen","year":"2013","journal-title":"Clin Trials"},{"key":"key20191028131707088_ref1-bib146","doi-asserted-by":"publisher","first-page":"296","DOI":"10.1177\/0272989X15614814","article-title":"Adjusting estimates of the expected value of information for implementation: theoretical framework and practical application","volume":"36","author":"Andronis","year":"2016","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib147","doi-asserted-by":"publisher","first-page":"1468","DOI":"10.1002\/hec.3103","article-title":"Valuing trial designs from a pharmaceutical perspective using value-based pricing","volume":"24","author":"Breeze","year":"2015","journal-title":"Health Econ"},{"key":"key20191028131707088_ref1-bib148","doi-asserted-by":"publisher","first-page":"E11","DOI":"10.1177\/0272989X12443010","article-title":"Expected net present value of sample information: from burden to investment","volume":"32","author":"Hall","year":"2012","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib149","doi-asserted-by":"publisher","first-page":"584","DOI":"10.1177\/0272989X15578125","article-title":"Computing expected value of partial sample information from probabilistic sensitivity analysis using linear regression metamodeling","volume":"35","author":"Jalal","year":"2015","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib150","doi-asserted-by":"publisher","first-page":"327","DOI":"10.1177\/0272989X13514774","article-title":"Strategies for efficient computation of the expected value of partial perfect information","volume":"34","author":"Madan","year":"2014","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib151","doi-asserted-by":"publisher","first-page":"715","DOI":"10.1080\/10543406.2014.902851","article-title":"An optimization approach to calculating sample sizes with binary responses","volume":"24","author":"Maroufy","year":"2014","journal-title":"J Biopharm Stat"},{"key":"key20191028131707088_ref1-bib152","doi-asserted-by":"publisher","first-page":"853","DOI":"10.1177\/0272989X11399921","article-title":"Addressing adoption and research design decisions simultaneously: the role of value of sample information analysis","volume":"31","author":"McKenna","year":"2011","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib153","doi-asserted-by":"publisher","first-page":"308","DOI":"10.1177\/0272989X15583495","article-title":"An efficient estimator for the expected value of sample information","volume":"36","author":"Menzies","year":"2016","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib154","doi-asserted-by":"publisher","first-page":"877","DOI":"10.1002\/hec.2869","article-title":"Two-level resampling as a novel method for the calculation of the expected value of sample information in economic trials","volume":"22","author":"Sadatsafavi","year":"2013","journal-title":"Health Econ"},{"key":"key20191028131707088_ref1-bib155","doi-asserted-by":"publisher","first-page":"25","DOI":"10.1007\/s40273-012-0008-3","article-title":"A systematic and critical review of the evolving methods and applications of value of information in academia and practice","volume":"31","author":"Steuten","year":"2013","journal-title":"PharmacoEconomics"},{"key":"key20191028131707088_ref1-bib156","doi-asserted-by":"publisher","first-page":"311","DOI":"10.1177\/0272989X13505910","article-title":"Estimating multiparameter partial expected value of perfect information from a probabilistic sensitivity analysis sample: a nonparametric regression approach","volume":"34","author":"Strong","year":"2014","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib157","doi-asserted-by":"publisher","first-page":"352","DOI":"10.1177\/0272989X13501229","article-title":"Expected value of sample information for multi-arm cluster randomized trials with binary outcomes","volume":"34","author":"Welton","year":"2014","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib158","doi-asserted-by":"publisher","first-page":"608","DOI":"10.1177\/0272989X15570113","article-title":"Accounting for heterogeneity in relative treatment effects for use in cost-effectiveness models and value-of-information analyses","volume":"35","author":"Welton","year":"2015","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib159","doi-asserted-by":"publisher","first-page":"447","DOI":"10.2165\/11592250-000000000-00000","article-title":"Value of information and pricing new healthcare interventions","volume":"30","author":"Willan","year":"2012","journal-title":"PharmacoEconomics"},{"key":"key20191028131707088_ref1-bib160","doi-asserted-by":"publisher","first-page":"1183","DOI":"10.1002\/hec.1781","article-title":"Accounting for between-study variation in incremental net benefit in value of information methodology","volume":"21","author":"Willan","year":"2012","journal-title":"Health Econ"},{"key":"key20191028131707088_ref1-bib161","doi-asserted-by":"publisher","first-page":"31","DOI":"10.1186\/1471-2288-12-31","article-title":"Is it possible to estimate the minimal clinically important treatment effect needed to change practice in preterm birth prevention? Results of an obstetrician survey used to support the design of a trial","volume":"12","author":"Ross","year":"2012","journal-title":"BMC Med Res Methodol"},{"key":"key20191028131707088_ref1-bib162","doi-asserted-by":"publisher","first-page":"522","DOI":"10.1177\/1740774513490250","article-title":"Caution regarding the choice of standard deviations to guide sample size calculations in clinical trials","volume":"10","author":"Chen","year":"2013","journal-title":"Clin Trials"},{"key":"key20191028131707088_ref1-bib163","doi-asserted-by":"publisher","first-page":"990","DOI":"10.1177\/1740774513506965","article-title":"An alternative property for evaluating sample size for normal data using preliminary data","volume":"10","author":"Fay","year":"2013","journal-title":"Clin Trials"},{"key":"key20191028131707088_ref1-bib164","doi-asserted-by":"publisher","first-page":"373","DOI":"10.1002\/pst.1521","article-title":"Discounting phase 2 results when planning phase 3 clinical trials","volume":"11","author":"Kirby","year":"2012","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib165","doi-asserted-by":"publisher","first-page":"301","DOI":"10.1016\/j.jclinepi.2011.07.011","article-title":"The size of a pilot study for a clinical trial should be calculated in relation to considerations of precision and efficiency","volume":"65","author":"Sim","year":"2012","journal-title":"J Clin Epidemiol"},{"key":"key20191028131707088_ref1-bib166","doi-asserted-by":"publisher","first-page":"84","DOI":"10.1016\/j.jclinepi.2015.10.017","article-title":"How to communicate effect sizes for continuous outcomes: a review of existing options and introducing a new metric","volume":"72","author":"Valentine","year":"2016","journal-title":"J Clin Epidemiol"},{"key":"key20191028131707088_ref1-bib167","doi-asserted-by":"publisher","first-page":"933","DOI":"10.2165\/11587130-000000000-00000","article-title":"Sample size determination for cost-effectiveness trials","volume":"29","author":"Willan","year":"2011","journal-title":"PharmacoEconomics"},{"key":"key20191028131707088_ref1-bib168","doi-asserted-by":"publisher","first-page":"105","DOI":"10.1007\/s40273-014-0219-x","article-title":"A practical guide to value of information analysis","volume":"33","author":"Wilson","year":"2015","journal-title":"PharmacoEconomics"},{"key":"key20191028131707088_ref1-bib169","doi-asserted-by":"publisher","first-page":"253","DOI":"10.1016\/j.jclinepi.2011.06.018","article-title":"A critical review of methods used to determine the smallest worthwhile effect of interventions for low back pain","volume":"65","author":"Ferreira","year":"2012","journal-title":"J Clin Epidemiol"},{"key":"key20191028131707088_ref1-bib170","doi-asserted-by":"publisher","first-page":"930","DOI":"10.1001\/jamasurg.2016.1640","article-title":"Defining a research agenda for patient-reported outcomes in surgery: using a delphi survey of stakeholders","volume":"151","author":"Pezold","year":"2016","journal-title":"JAMA Surg"},{"key":"key20191028131707088_ref1-bib171","doi-asserted-by":"publisher","first-page":"132","DOI":"10.1186\/1745-6215-13-132","article-title":"Developing core outcome sets for clinical trials: issues to consider","volume":"13","author":"Williamson","year":"2012","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib172","doi-asserted-by":"crossref","DOI":"10.1002\/9780470723586","volume-title":"Statistical Issues in Drug Development","author":"Senn","year":"2007","edition":"2nd"},{"key":"key20191028131707088_ref1-bib173","doi-asserted-by":"publisher","first-page":"3729","DOI":"10.1002\/sim.2074","article-title":"Controversies concerning randomization and additivity in clinical trials","volume":"23","author":"Senn","year":"2004","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib174","volume-title":"Sample Size Tables for Clinical Studies","author":"Machin","year":"2009","edition":"3rd"},{"key":"key20191028131707088_ref1-bib175","doi-asserted-by":"publisher","first-page":"250","DOI":"10.1002\/pst.459","article-title":"The role of the minimum clinically important difference and its impact on designing a trial","volume":"10","author":"Chuang-Stein","year":"2011","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib176","doi-asserted-by":"publisher","first-page":"333","DOI":"10.1002\/pst.362","article-title":"Back to basics: explaining sample size in outcome trials, are statisticians doing a thorough job?","volume":"8","author":"Carroll","year":"2009","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib177","doi-asserted-by":"publisher","first-page":"81","DOI":"10.1186\/1745-6215-12-81","article-title":"Designs for clinical trials with time-to-event outcomes based on stopping guidelines for lack of benefit","volume":"12","author":"Royston","year":"2011","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib178","doi-asserted-by":"publisher","first-page":"b3985","DOI":"10.1136\/bmj.b3985","article-title":"The tyranny of power: is there a better way to calculate sample size?","volume":"339","author":"Bland","year":"2009","journal-title":"BMJ"},{"key":"key20191028131707088_ref1-bib179","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1186\/1741-7015-8-17","article-title":"Current sample size conventions: flaws, harms, and alternatives","volume":"8","author":"Bacchetti","year":"2010","journal-title":"BMC Med"},{"key":"key20191028131707088_ref1-bib180","doi-asserted-by":"publisher","first-page":"305","DOI":"10.1038\/d41586-019-00857-9","article-title":"Scientists rise up against statistical significance","volume":"567","author":"Amrhein","year":"2019","journal-title":"Nature"},{"key":"key20191028131707088_ref1-bib181","doi-asserted-by":"publisher","first-page":"52","DOI":"10.1016\/j.jclinepi.2018.08.013","article-title":"Sample size calculations are poorly conducted and reported in many\u2009randomized trials of hip and knee osteoarthritis: results of a systematic review","volume":"104","author":"Copsey","year":"2018","journal-title":"J Clin Epidemiol"},{"key":"key20191028131707088_ref1-bib182","doi-asserted-by":"publisher","first-page":"348","DOI":"10.1002\/bimj.200800266","article-title":"Sample size re-estimation in clinical trials","volume":"51","author":"Proschan","year":"2009","journal-title":"Biom J"},{"key":"key20191028131707088_ref1-bib183","doi-asserted-by":"publisher","first-page":"23","DOI":"10.1002\/sim.2180","article-title":"The reassessment of trial perspectives from interim data \u2013 a critical view","volume":"25","author":"Bauer","year":"2006","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib184","doi-asserted-by":"publisher","first-page":"311","DOI":"10.1002\/pst.467","article-title":"The perils with the misuse of predictive power","volume":"10","author":"Dallow","year":"2011","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib185","doi-asserted-by":"publisher","first-page":"672","DOI":"10.1161\/STROKEAHA.108.532051","article-title":"Are unadjusted analyses of clinical trials inappropriately biased toward the null?","volume":"40","author":"Kent","year":"2009","journal-title":"Stroke"},{"key":"key20191028131707088_ref1-bib186","doi-asserted-by":"publisher","first-page":"68","DOI":"10.1002\/pst.1709","article-title":"Practical guide to sample size calculations: an introduction","volume":"15","author":"Flight","year":"2016","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib187","doi-asserted-by":"publisher","first-page":"781","DOI":"10.1016\/S0140-6736(02)07882-0","article-title":"Sample size slippages in randomised trials: exclusions and the lost and wayward","volume":"359","author":"Schulz","year":"2002","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib188","doi-asserted-by":"publisher","first-page":"244","DOI":"10.1053\/ejso.1998.0635","article-title":"Sample size estimation in phase III cancer clinical trials","volume":"25","author":"Curran","year":"1999","journal-title":"Eur J Surg Oncol"},{"key":"key20191028131707088_ref1-bib189","doi-asserted-by":"publisher","first-page":"2899","DOI":"10.1002\/sim.1294","article-title":"The role of covariates in estimating treatment effects and risk in long-term clinical trials","volume":"21","author":"Ford","year":"2002","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib190","doi-asserted-by":"publisher","first-page":"441","DOI":"10.1016\/j.ejca.2007.10.026","article-title":"A Greenwood formula for standard error of the age-standardised relative survival ratio","volume":"44","author":"Pokhrel","year":"2008","journal-title":"Eur J Cancer"},{"key":"key20191028131707088_ref1-bib191","doi-asserted-by":"publisher","first-page":"314","DOI":"10.1186\/1745-6215-15-314","article-title":"An approach to trial design and analysis in the era of non-proportional hazards of the treatment effect","volume":"15","author":"Royston","year":"2014","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib192","doi-asserted-by":"publisher","first-page":"327","DOI":"10.1191\/0962280205sm406oa","article-title":"Uses and limitations of randomization-based efficacy estimators","volume":"14","author":"White","year":"2005","journal-title":"Stat Methods Med Res"},{"key":"key20191028131707088_ref1-bib193","doi-asserted-by":"publisher","first-page":"39","DOI":"10.1093\/epirev\/24.1.39","article-title":"Sample size calculations for randomized controlled trials","volume":"24","author":"Wittes","year":"2002","journal-title":"Epidemiol Rev"},{"key":"key20191028131707088_ref1-bib194","doi-asserted-by":"publisher","first-page":"237","DOI":"10.1177\/0962280209105014","article-title":"Mediation and moderation of treatment effects in randomised controlled trials of complex interventions","volume":"19","author":"Emsley","year":"2010","journal-title":"Stat Methods Med Res"},{"key":"key20191028131707088_ref1-bib195","doi-asserted-by":"publisher","first-page":"369","DOI":"10.1191\/0962280205sm403oa","article-title":"Estimating treatment effects from randomized clinical trials with noncompliance and loss to follow-up: the role of instrumental variable methods","volume":"14","author":"Dunn","year":"2005","journal-title":"Stat Methods Med Res"},{"issue":"93","key":"key20191028131707088_ref1-bib196","doi-asserted-by":"publisher","DOI":"10.3310\/hta19930","article-title":"Evaluation and validation of social and psychological markers in randomised trials of complex interventions in mental health: a methodological research programme","volume":"19","author":"Dunn","year":"2015","journal-title":"Health Technol Assess"},{"key":"key20191028131707088_ref1-bib197","doi-asserted-by":"publisher","first-page":"149","DOI":"10.1186\/s13063-016-1277-5","article-title":"Sample size calculations in clinical research should also be based on ethical principles","volume":"17","author":"Cesana","year":"2016","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib198","doi-asserted-by":"publisher","first-page":"29","DOI":"10.1186\/s40814-015-0025-z","article-title":"Nail bed INJury Assessment Pilot (NINJA-P) study: should the nail plate be replaced or discarded after nail bed repair in children? Study protocol for a pilot randomised controlled trial","volume":"1","author":"Jain","year":"2015","journal-title":"Pilot Feasibility Stud"},{"key":"key20191028131707088_ref1-bib199","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/1471-2288-10-1","article-title":"A tutorial on pilot studies: the what, why and how","volume":"10","author":"Thabane","year":"2010","journal-title":"BMC Med Res Methodol"},{"key":"key20191028131707088_ref1-bib200","doi-asserted-by":"publisher","first-page":"1933","DOI":"10.1002\/sim.4780141709","article-title":"On the use of a pilot sample for sample size determination","volume":"14","author":"Browne","year":"1995","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib201","doi-asserted-by":"publisher","first-page":"529","DOI":"10.1016\/S0140-6736(14)61184-3","article-title":"Long-term outcomes after stenting versus endarterectomy for treatment of symptomatic carotid stenosis: the International Carotid Stenting Study (ICSS) randomised trial","volume":"385","author":"Bonati","year":"2015","journal-title":"Lancet"},{"key":"key20191028131707088_ref1-bib202","doi-asserted-by":"publisher","first-page":"389","DOI":"10.2307\/2348574","article-title":"An average power criterion for sample size estimation","volume":"43","author":"Gillett","year":"1994","journal-title":"J R Stat Soc Ser D"},{"key":"key20191028131707088_ref1-bib203","doi-asserted-by":"publisher","first-page":"561","DOI":"10.1177\/1740774512453784","article-title":"Some thoughts on sample size: a Bayesian-frequentist hybrid approach","volume":"9","author":"Gordon Lan","year":"2012","journal-title":"Clin Trials"},{"key":"key20191028131707088_ref1-bib204","doi-asserted-by":"publisher","first-page":"5","DOI":"10.1177\/1740774509356002","article-title":"Summarizing historical information on controls in clinical trials","volume":"7","author":"Neuenschwander","year":"2010","journal-title":"Clin Trials"},{"key":"key20191028131707088_ref1-bib205","doi-asserted-by":"publisher","first-page":"1023","DOI":"10.1111\/biom.12242","article-title":"Robust meta-analytic-predictive priors in clinical trials with historical control information","volume":"70","author":"Schmidli","year":"2014","journal-title":"Biometrics"},{"key":"key20191028131707088_ref1-bib206","doi-asserted-by":"publisher","first-page":"346","DOI":"10.1186\/1745-6215-15-346","article-title":"Meta-analysis of randomized phase II trials to inform subsequent phase III decisions","volume":"15","author":"Burke","year":"2014","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib207","doi-asserted-by":"publisher","first-page":"29","DOI":"10.1016\/0197-2456(87)90023-7","article-title":"Projection from previous studies: a Bayesian and frequentist compromise","volume":"8","author":"Brown","year":"1987","journal-title":"Control Clin Trials"},{"key":"key20191028131707088_ref1-bib208","doi-asserted-by":"publisher","first-page":"275","DOI":"10.1177\/1740774516628825","article-title":"Selection of the effect size for sample size determination for a continuous response in a superiority clinical trial using a hybrid classical and Bayesian procedure","volume":"13","author":"Ciarleglio","year":"2016","journal-title":"Clin Trials"},{"key":"key20191028131707088_ref1-bib209","doi-asserted-by":"publisher","first-page":"221","DOI":"10.1214\/13-BA809","article-title":"On the limiting behaviour of the \u2018probability of claiming superiority\u2019 in a Bayesian context","volume":"8","author":"Eaton","year":"2013","journal-title":"Bayesian Anal"},{"key":"key20191028131707088_ref1-bib210","doi-asserted-by":"publisher","first-page":"2307","DOI":"10.1002\/sim.3140","article-title":"Bayesian sample size for exploratory clinical trials incorporating historical data","volume":"27","author":"Whitehead","year":"2008","journal-title":"Stat Med"},{"key":"key20191028131707088_ref1-bib211","doi-asserted-by":"publisher","first-page":"209","DOI":"10.1111\/1467-9884.00077","article-title":"Bayesian sample size determination for normal means and differences between normal means","volume":"46","author":"Joseph","year":"1997","journal-title":"J R Stat Soc Ser D"},{"key":"key20191028131707088_ref1-bib212","doi-asserted-by":"publisher","first-page":"183","DOI":"10.1177\/0962280208089298","article-title":"The choice of sample size: a mixed Bayesian\/ frequentist approach","volume":"18","author":"Pezeshk","year":"2009","journal-title":"Stat Methods Med Res"},{"key":"key20191028131707088_ref1-bib213","doi-asserted-by":"publisher","first-page":"609","DOI":"10.1002\/bimj.201500228","article-title":"Determination of the optimal sample size for a clinical trial accounting for the population size","volume":"59","author":"Stallard","year":"2017","journal-title":"Biom J"},{"key":"key20191028131707088_ref1-bib214","doi-asserted-by":"publisher","first-page":"341","DOI":"10.1016\/S0167-6296(98)00039-3","article-title":"The irrelevance of inference: a decision-making approach to the stochastic evaluation of health care technologies","volume":"18","author":"Claxton","year":"1999","journal-title":"J Health Econ"},{"key":"key20191028131707088_ref1-bib215","doi-asserted-by":"publisher","first-page":"282","DOI":"10.1177\/0272989X16635130","article-title":"Expected value of sample information with imperfect implementation: improving practice and reducing uncertainty with appropriate counterfactual consideration","volume":"36","author":"Eckermann","year":"2016","journal-title":"Med Decis Making"},{"key":"key20191028131707088_ref1-bib216","doi-asserted-by":"publisher","first-page":"2543","DOI":"10.1177\/0962280215601137","article-title":"Sample size considerations for split-mouth design","volume":"26","author":"Zhu","year":"2017","journal-title":"Stat Methods Med Res"},{"key":"key20191028131707088_ref1-bib217","doi-asserted-by":"publisher","first-page":"69","DOI":"10.1186\/s12874-016-0176-5","article-title":"Stepped wedge cluster randomised trials: a review of the statistical methodology used and available","volume":"16","author":"Barker","year":"2016","journal-title":"BMC Med Res Methodol"},{"key":"key20191028131707088_ref1-bib218","doi-asserted-by":"publisher","first-page":"b3883","DOI":"10.1136\/bmj.b3883","article-title":"Cervical collar or physiotherapy versus wait and see policy for recent onset cervical radiculopathy: randomised trial","volume":"339","author":"Kuijper","year":"2009","journal-title":"BMJ"},{"key":"key20191028131707088_ref1-bib219","doi-asserted-by":"crossref","DOI":"10.1002\/9780470686164","volume-title":"An Introduction to Statistics in Early Phase Trials","author":"Julious","year":"2010"},{"key":"key20191028131707088_ref1-bib220","doi-asserted-by":"publisher","first-page":"177","DOI":"10.1002\/pst.515","article-title":"Sample sizes for trials involving multiple correlated must-win comparisons","volume":"11","author":"Julious","year":"2012","journal-title":"Pharm Stat"},{"key":"key20191028131707088_ref1-bib221","doi-asserted-by":"publisher","first-page":"579","DOI":"10.1177\/0962280210378943","article-title":"Multiplicity adjustments in trials with two correlated comparisons of interest","volume":"20","author":"Fernandes","year":"2011","journal-title":"Stat Methods Med Res"},{"key":"key20191028131707088_ref1-bib222","doi-asserted-by":"publisher","first-page":"549","DOI":"10.1136\/bmj.316.7130.549","article-title":"Sample size in cluster randomisation","volume":"316","author":"Kerry","year":"1998","journal-title":"BMJ"},{"key":"key20191028131707088_ref1-bib223","volume-title":"Design and Analysis of Cluster Randomization Trials in Health Research","author":"Donner","year":"2000"},{"issue":"5","key":"key20191028131707088_ref1-bib224","doi-asserted-by":"publisher","DOI":"10.3310\/hta3050","article-title":"Methods for evaluating area-wide and organisation-based interventions in health and health care: a systematic review","volume":"3","author":"Ukoumunne","year":"1999","journal-title":"Health Technol Assess"},{"key":"key20191028131707088_ref1-bib225","doi-asserted-by":"publisher","first-page":"99","DOI":"10.1191\/1740774505cn071oa","article-title":"Determinants of the intracluster correlation coefficient in cluster randomized trials: the case of implementation research","volume":"2","author":"Campbell","year":"2005","journal-title":"Clin Trials"},{"key":"key20191028131707088_ref1-bib226","doi-asserted-by":"publisher","first-page":"1292","DOI":"10.1093\/ije\/dyl129","article-title":"Sample size for cluster randomized trials: effect of coefficient of variation of cluster size and analysis method","volume":"35","author":"Eldridge","year":"2006","journal-title":"Int J Epidemiol"},{"key":"key20191028131707088_ref1-bib227","doi-asserted-by":"publisher","first-page":"1051","DOI":"10.1093\/ije\/dyv113","article-title":"Methods for sample size determination in cluster randomized trials","volume":"44","author":"Rutterford","year":"2015","journal-title":"Int J Epidemiol"},{"key":"key20191028131707088_ref1-bib228","doi-asserted-by":"publisher","first-page":"152","DOI":"10.1191\/1740774505cn076oa","article-title":"Design and analysis of clinical trials with clustering effects due to treatment","volume":"2","author":"Roberts","year":"2005","journal-title":"Clin Trials"},{"key":"key20191028131707088_ref1-bib229","doi-asserted-by":"crossref","DOI":"10.1002\/0470854596","volume-title":"Cross-Over Trials in Clinical Research","author":"Senn","year":"2002","edition":"2nd"},{"key":"key20191028131707088_ref1-bib230","doi-asserted-by":"publisher","first-page":"229","DOI":"10.1016\/j.jclinepi.2003.08.009","article-title":"Subgroup analyses in randomized trials: risks of subgroup-specific analyses; power and sample size for the interaction test","volume":"57","author":"Brookes","year":"2004","journal-title":"J Clin Epidemiol"},{"key":"key20191028131707088_ref1-bib231","doi-asserted-by":"publisher","first-page":"80373","DOI":"10.18632\/oncotarget.12124","article-title":"Strategies for power calculations in predictive biomarker studies in survival data","volume":"7","author":"Chen","year":"2016","journal-title":"Oncotarget"},{"key":"key20191028131707088_ref1-bib232","doi-asserted-by":"publisher","first-page":"355","DOI":"10.1016\/S0197-2456(03)00006-0","article-title":"Planning for subgroup analysis: a case study of treatment-marker interaction in metastatic colorectal cancer","volume":"24","author":"G\u00f6nen","year":"2003","journal-title":"Control Clin Trials"},{"key":"key20191028131707088_ref1-bib233","doi-asserted-by":"publisher","first-page":"664","DOI":"10.1016\/j.cct.2013.09.005","article-title":"Sample size and threshold estimation for clinical trials with predictive biomarkers","volume":"36","author":"Mackey","year":"2013","journal-title":"Contemp Clin Trials"},{"key":"key20191028131707088_ref1-bib234","doi-asserted-by":"publisher","first-page":"145","DOI":"10.1186\/1745-6215-13-145","article-title":"Adaptive trial designs: a review of barriers and opportunities","volume":"13","author":"Kairalla","year":"2012","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib235","doi-asserted-by":"crossref","DOI":"10.1007\/978-3-319-32562-0","volume-title":"Group Sequential and Confirmatory Adaptive Designs in Clinical Trials","author":"Wassmer","year":"2016"},{"key":"key20191028131707088_ref1-bib236","doi-asserted-by":"publisher","first-page":"39","DOI":"10.1186\/1745-6215-10-39","article-title":"Issues in applying multi-arm multi-stage methodology to a clinical trial in prostate cancer: the MRC STAMPEDE trial","volume":"10","author":"Sydes","year":"2009","journal-title":"Trials"},{"key":"key20191028131707088_ref1-bib237","doi-asserted-by":"publisher","first-page":"4269","DOI":"10.1002\/sim.5513","article-title":"Optimal design of multi-arm multi-stage trials","volume":"31","author":"Wason","year":"2012","journal-title":"Stat Med"}],"container-title":["Health Technology Assessment"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.journalslibrary.nihr.ac.uk\/publications\/hta23600\/hta23600.xml","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/njl-admin.nihr.ac.uk\/document\/download\/2010496","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.journalslibrary.nihr.ac.uk\/publications\/hta23600\/hta23600.xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/njl-admin.nihr.ac.uk\/document\/download\/2030857","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,15]],"date-time":"2023-08-15T15:56:05Z","timestamp":1692114965000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.journalslibrary.nihr.ac.uk\/hta\/hta23600"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,10]]},"references-count":237,"journal-issue":{"issue":"60","published-online":{"date-parts":[[2019,10]]}},"alternative-id":["10.3310\/hta23600"],"URL":"https:\/\/doi.org\/10.3310\/hta23600","relation":{},"ISSN":["1366-5278","2046-4924"],"issn-type":[{"value":"1366-5278","type":"print"},{"value":"2046-4924","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,10]]},"assertion":[{"value":"This content has been made freely available to all.","name":"free","label":"Free to read"}]}}