EUnetHTA Joint Action 3 WP4 Rapid assessment of other technologies using the HTA Core Model® for Rapid Relative Effectiveness Assessment STOOL DNA TESTING FOR EARLY DETECTION OF COLORECTAL CANCER Project ID: OTJA10 Version 1.4, 29 July 2019 This report is part of the project/joint action ‘724130/EUnetHTA JA3’, which has received funding from the European Union’s Health Programme (2014-2020) Dec2015 ©EUnetHTA, 2015. Reproduction is authorised provided EUnetHTA is explicitly acknowledged 1 Stool DNA testing for early detection of colorectal cancer DOCUMENT HISTORY AND CONTRIBUTORS Version Date Description V1.0 14/03/19 First draft. V1.1 01/04/19 Input from co-authors has been processed. V1.2 24/05/19 Input from dedicated reviewers has been processed. V1.3 05/07/19 Input from external experts and manufacturer(s) has been processed. V1.4 29/07/19 Input from medical editor has been processed. Disclaimer The assessment represents a consolidated view of the EUnetHTA assessment team members and is in no case the official opinion of the participating institutions or individuals. EUnetHTA Joint Action 3 is supported by a grant from the European Commission. The sole respon- sibility for the content of this document lies with the authors and neither the European Commission nor EUnetHTA are responsible for any use that may be made of the information contained therein. Assessment team Author(s) Austrian Public Health Institute (GOEG) Co-Author(s) National Institute of Public Health (NIJZ), Agency for Medicinal Products and Medical Devices of the Republic of Slovenia (JAZMP), Private University of Health Sciences, Medical Informatics and Technology (UMIT) Dedicated National Institute for Health and Care Excellence (NICE), National Agency for Reviewer(s) Regional Health Services (AGENAS), Social & Health Services and Labour Market (DEFACTUM), Basque Office for HTA (Osteba) Further contributors Five individual patients were interviewed during the assessment. This input was used as additional information for assessing the relevance of potential ethical and social aspects and for answering research questions related to patient aspects. Consultation of the draft Rapid Assessment External experts Gerfried Lexer, Isabel Idigoras Rubio, Eunate Arana-Arri, Fidencio Bao Manufacturer(s) PharmGenomics (Germany), Exact Sciences (USA) (factual accuracy check) Medical editor Morgan Lyons (Compuscript Ltd Publishing Services) Patient(s)/ patient Individual patients in Austria organisation(s)/ citizens Version 1.4, 29 July 2019 EUnetHTA Joint Action 3 WP4 2 Stool DNA testing for early detection of colorectal cancer Conflict of interest All authors, co-authors, dedicated reviewers, external experts and patients involved in the produc- tion of this assessment have declared that they have no conflicts of interest in relation to the tech- nology and comparator assessed according to the EUnetHTA declaration of interest and confiden- tiality undertaking (DOICU) statement form. How to cite this assessment Please cite this assessment as follows: Stürzlinger H, Conrads-Frank A, Eisenmann A, Ivansits S, Jahn B, Janzic A, Jelenc M, Kostnapfel T, Mencej Bedrac S, Mühlberger N, Rochau U, Siebert U, Schnell-Inderst P, Sroczynski G: Stool DNA testing for early detection of colorectal cancer. Joint Assessment. Vienna: EUnetHTA; 2019. Report No.: OTJA10. This document is available on the website of EUnetHTA. Version 1.4, 29 July 2019 EUnetHTA Joint Action 3 WP4 3 Stool DNA testing for early detection of colorectal cancer TABLE OF CONTENTS DOCUMENT HISTORY AND CONTRIBUTORS ............................................................................ 2 TABLE OF CONTENTS ................................................................................................................... 4 LIST OF TABLES AND FIGURES .................................................................................................. 5 LIST OF ABBREVIATIONS ............................................................................................................. 8 SUMMARY OF RELATIVE EFFECTIVENESS OF STOOL DNA TESTING FOR EARLY DETECTION OF COLORECTAL CANCER ............................................................................ 11 SCOPE ....................................................................................................................................... 11 INTRODUCTION ........................................................................................................................... 11 METHODS .................................................................................................................................. 12 RESULTS.................................................................................................................................... 13 DISCUSSION ............................................................................................................................... 19 CONCLUSION .............................................................................................................................. 20 1 SCOPE ..................................................................................................................................... 22 2 METHODS AND EVIDENCE INCLUDED ............................................................................... 25 2.1 ASSESSMENT TEAM ............................................................................................................ 25 2.2 SOURCE OF ASSESSMENT ELEMENTS .................................................................................. 25 2.3 SEARCH ............................................................................................................................. 25 2.4 STUDY SELECTION .............................................................................................................. 26 2.5 DATA EXTRACTION AND ANALYSES ...................................................................................... 27 2.6 QUALITY RATING ................................................................................................................ 28 2.7 PATIENT INVOLVEMENT ....................................................................................................... 28 2.8 DESCRIPTION OF THE EVIDENCE USED ................................................................................. 28 2.9 METHODS OF THE BENEFIT–HARM MODELING ....................................................................... 33 2.10 DEVIATIONS FROM PROJECT PLAN ....................................................................................... 37 3 DESCRIPTION AND TECHNICAL CHARACTERISTICS OF TECHNOLOGY (TEC) ........... 38 3.1 RESEARCH QUESTIONS ....................................................................................................... 38 3.2 RESULTS ........................................................................................................................... 38 4 HEALTH PROBLEM AND CURRENT USE OF THE TECHNOLOGY (CUR) ....................... 44 4.1 RESEARCH QUESTIONS ....................................................................................................... 44 4.2 RESULTS ........................................................................................................................... 44 5 CLINICAL EFFECTIVENESS (EFF) ....................................................................................... 67 5.1 RESEARCH QUESTIONS ....................................................................................................... 67 5.2 RESULTS ........................................................................................................................... 67 6 SAFETY (SAF) ........................................................................................................................ 83 6.1 RESEARCH QUESTIONS ....................................................................................................... 83 6.2 RESULTS ........................................................................................................................... 83 7 BENEFIT–HARM ANALYSIS .................................................................................................. 85 8 POTENTIAL ETHICAL, ORGANISATIONAL, PATIENT AND SOCIAL, AND LEGAL ASPECTS (ETH, ORG, SOC, LEG) ........................................................................... 93 8.1 RESEARCH QUESTIONS ....................................................................................................... 93 8.2 RESULTS ........................................................................................................................... 93 9 PATIENT INVOLVEMENT ....................................................................................................... 94 10 DISCUSSION ........................................................................................................................... 95 11 CONCLUSION ......................................................................................................................... 99 12 REFERENCES ....................................................................................................................... 101 Version 1.4, 29 July 2019 EUnetHTA Joint Action 3 WP4 4 Stool DNA testing for early detection of colorectal cancer APPENDIX 1: METHODS AND DESCRIPTION OF THE EVIDENCE USED ............................ 108 DOCUMENTATION OF THE SEARCH STRATEGIES ......................................................................... 108 DESCRIPTION OF THE EVIDENCE