Central Reading of Endoscopy Endpoints in Inflammatory Bowel Disease Trials

Inflammatory Bowel Diseases

Volumn 21, Issue 10, 2015, Pages 2475-2482

  Gottlieb, Klaus ^a   Travis, Simon ^b   Feagan, Brian ^c   Hussain, Fez ^a   Sandborn, William J ^d   Rutgeerts, Paul ^e  

^a IQVIA   (United States)

^b OXFORD UNIVERSITY HOSPITALS NHS FOUNDATION TRUST   (United Kingdom)

^c ROBARTS RESEARCH INSTITUTE   (Canada)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e UNIVERSITY OF LEUVEN   (Belgium)

Author keywords

central reading; clinical trials; Crohn's disease; endoscopy; endpoints; ulcerative colitis

Indexed keywords

ALGORITHM; CENTRAL READING OF ENDOSCOPY; CLINICAL TRIAL (TOPIC); CONSENSUS; ENDOSCOPY; FOOD AND DRUG ADMINISTRATION; HUMAN; INFLAMMATORY BOWEL DISEASE; MEDICAL SOCIETY; NOMENCLATURE; NULL HYPOTHESIS; PLACEBO EFFECT; PRACTICE GUIDELINE; PRIORITY JOURNAL; REVIEW; SAMPLE SIZE; STANDARDIZATION; STATISTICAL BIAS; STUDY DESIGN; VIDEORECORDING; BIOASSAY; COMPUTER ASSISTED DIAGNOSIS; GASTROINTESTINAL ENDOSCOPY; OBSERVER VARIATION; PATHOLOGY; PROCEDURES; STANDARDS;

CLINICAL TRIALS AS TOPIC; ENDOSCOPY, GASTROINTESTINAL; ENDPOINT DETERMINATION; HUMANS; IMAGE INTERPRETATION, COMPUTER-ASSISTED; INFLAMMATORY BOWEL DISEASES; OBSERVER VARIATION; PRACTICE GUIDELINES AS TOPIC;

EID: 84942258792     PISSN: 10780998     EISSN: 15364844     Source Type: Journal    
DOI: 10.1097/MIB.0000000000000470     Document Type: Review

References (39)

1. Fleming TR, Powers JH. Biomarkers and surrogate endpoints in clinical trials. Stat Med. 2012;31:2973-2984.
2. Gottlieb K, Randazzo G, Walsh M. Chapter 11-Prescription Drug Product Submissions. Fundamentals of US Regulatory Affairs [Internet]. 8th ed. Regulatory Affairs Professionals Society; 2013:121-138. Available at: http://www.raps.org/WorkArea/DownloadAsset.aspx?id14;5190. Accessed January 7, 2015.
3. Hyams JS. "GREAT"er than Last Year: GREAT 2 j AGA Washington Insider [Internet]. Available at: http://agapolicyblog.org/2013/11/18/greater-than-last-year-great-2/. Accessed January 7, 2015.
4. European Medicines Agency. Guideline on the Development of New Medicinal Products for the Treatment of Ulcerative Colitis [Internet]. European Medicines Agency; 2008. Report No.: CHMP/EWP/18463/2006. Available at: http://www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/2009/09/WC500003266.pdf. Accessed January 7, 2015.
5. Feagan BG, Sandborn WJ, D'Haens G, et al. The role of centralized reading of endoscopy in a randomized controlled trial of mesalamine for ulcerative colitis. Gastroenterology. 2013;145:149-157.e2.
6. Kobayashi K, Hirai F, Naganuma M, et al. A randomized clinical trial of mesalazine suppository: the usefulness and problems of central review of evaluations of colonic mucosal findings. J Crohns Colitis. 2014. Available at: http://www.sciencedirect.com/science/article/pii/S1873994614001780. Accessed January 7, 2015.
7. Rosner B. Hypothesis Testing: Categorical Data-Estimation of Sample Size and Power for Comparing Two Binomial Proportions. Fundamentals of Biostatistics. Boston, MA: Brooks/Cole, Cengage Learning; 2011.
8. Su C, Lewis JD, Goldberg B, et al. A meta-analysis of the placebo rates of remission and response in clinical trials of active ulcerative colitis. Gastroenterology. 2007;132:516-526.
9. FDA. Guidance for Industry Non-inferiority Clinical Trials. Draft [Internet]. FDA; 2010. Available at: http://www.fda.gov/downloads/Drugs/Guidances/UCM202140.pdf. Accessed January 7, 2015.
10. Lachin JM, Marks JW, Schoenfield LJ, et al. Design and methodological considerations in the National Cooperative Gallstone Study: a multicenter clinical trial. Control Clin Trials. 1981;2:177-229.
11. Walovitch RC, Yao B, Chokron P, et al. Subjective endpoints in clinical trials: the case for blinded independent central review. Open Access J Clin Trials. 2013;5:111.
12. Hata J, Arima H, Zoungas S, et al. Effects of the endpoint adjudication process on the results of a randomised controlled trial: the ADVANCE trial. PLoS One. 2013;8:e55807.
13. Abreu MT, Travis SPL, Cooney RM, et al. Conduct of clinical trials in UC: impact of independent scoring of endoscopic severity on results of a randomised controlled trial. Am J Gastroenterol. 2006;101:S429-S430.
14. Cooney RM, Warren BF, Altman DG, et al. Outcome measurement in clinical trials for Ulcerative Colitis: towards standardisation. Trials. 2007; 8:17.
15. Sandborn WJ, Regula J, Feagan BG, et al. Delayed-release oral mesalamine 4.8 g/day (800-mg tablet) is effective for patients with moderately active ulcerative colitis. Gastroenterology. 2009;137:1934-1943.e1-3.
16. Travis SPL, Schnell D, Krzeski P, et al. Developing an instrument to assess the endoscopic severity of ulcerative colitis: the Ulcerative Colitis Endoscopic Index of Severity (UCEIS). Gut. 2012;61:535-542.
17. Travis SPL, Schnell D, Krzeski P, et al. Reliability and initial validation of the ulcerative colitis endoscopic index of severity. Gastroenterology. 2013;145:987-995.
18. Hébuterne X, Lémann M, Bouhnik Y, et al. Endoscopic improvement of mucosal lesions in patients with moderate to severe ileocolonic Crohn's disease following treatment with certolizumab pegol. Gut. 2013;62:201-208.
19. Rutgeerts P, Van Assche G, Sandborn WJ, et al. Adalimumab induces and maintains mucosal healing in patients with Crohn's disease: data from the EXTEND trial. Gastroenterology. 2012;142:1102-1111.e2.
20. Schroeder KW, Tremaine WJ, Ilstrup DM. Coated oral 5-aminosalicylic acid therapy for mildly to moderately active ulcerative colitis. A randomized study. N Engl J Med. 1987;317:1625-1629.
21. Travis SPL, Higgins PDR, Orchard T, et al. Review article: defining remission in ulcerative colitis. Aliment Pharmacol Ther. 2011;34: 113-124.
22. Feagan BG, Rutgeerts P, Sands BE, et al. Vedolizumab as induction and maintenance therapy for ulcerative colitis. N Engl J Med. 2013; 369:699-710.
23. Vermeire S, O'Byrne S, Keir M, et al. Etrolizumab as induction therapy for ulcerative colitis: a randomised, controlled, phase 2 trial. Lancet. 2014; 384:309-318.
24. Campbell MJ, Julious SA, Altman DG. Estimating sample sizes for binary, ordered categorical, and continuous outcomes in two group comparisons. BMJ. 1995;311:1145-1148.
25. Gottlieb K, Hussain F. Voting for Image Scoring and Assessment (VISA)-theory and application of a 2+1 reader algorithm to improve accuracy of imaging endpoints in clinical trials. BMC Med Imaging. 2015;15:6.
26. FDA. Summary Minutes of the Oncologic Drugs Advisory Committee Meeting July 24, 2012 [Internet]. 2014. Available at: http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/OncologicDrugsAdvisoryCommittee/UCM316323.pdf. Accessed January 7, 2015.
27. The Cancer Letter. FDA to Move Away from Central Radiology to Investigator Review in PFS Endpoint Trials-the Cancer Letter Publications [Internet]. 2012. Available at: http://www.cancerletter.com/articles/20120730. Accessed January 7, 2015.
28. Goldmacher G. Decentralization of Imaging-Benefits and Caveats for Sponsors. Contract Pharma [Internet]. 2012. Available at: http://www. contractpharma.com/issues/2012-11/view-features/decentralization-ofimaging/. Accessed January 7, 2015.
29. Dodd LE, Korn EL, Freidlin B, et al. Blinded independent central review of progression-free survival in phase III clinical trials: important design element or unnecessary expense? J Clin Oncol. 2008;26:3791-3796.
30. FDA. Guidance for Industry Standards for Clinical Trial Imaging Endpoints. Draft. [Internet]. FDA; 2011. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM268555.pdf. Accessed January 7, 2015.
31. FDA. Guidance for Industry Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics [Internet]. 2007. Available at: http://www. fda.gov/downloads/Drugs/./Guidances/ucm071590.pdf. Accessed January 7, 2015.
32. FDA. Guidance for Industry Developing Medical Imaging Drug and Biological Products Part 3: Design, Analysis, and Interpretation of Clinical Studies [Internet]. FDA; 2004. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071604.pdf. Accessed January 7, 2015.
33. European Medicines Agency. Appendix 1 to the Guideline on Clinical Evaluation of Diagnostic Agents (CPMP/EWP/1119/98 REV. 1) on Imaging Agents [Internet]. European Medicines Agency; 2009. Report No.: EMEA/CHMP/EWP/321180/2008. Available at: http://www.ema. europa.eu/docs/en-GB/document-library/Scientific-guideline/2009/09/WC500003581.pdf. Accessed January 7, 2015.
34. Ahmad H, Berzin TM, Yu HJ, et al. Central endoscopy reads in inflammatory bowel disease clinical trials: the role of the imaging core lab. Gastroenterol Rep (Oxf). 2014;2:201-206.
35. Rex DK. Looking over your shoulder during colonoscopy: potential roles for videorecording colonoscopy withdrawals. Gastrointest Endosc. 2012; 75:134-137.
36. Ford R, Schwartz L, Dancey J, et al. Lessons learned from independent central review. Eur J Cancer. 2009;45:268-274.
37. Krzeski P, O'Leary DH, Zabbatino S. Lessons Learned from the Use of Central Endoscopy Review in Inflammatory Bowel Disease Trials [Internet]. Medpace; 2013. Available at: http://www.medpace.com/Offers/GI-Central-Reader/Medpace-GI-Imaging-IBD-White-paper-May2013.pdf. Accessed January 7, 2015.
38. Daperno M, Comberlato M, Bossa F, et al; on behalf of IG-IBD Group. PC.01.8 increasing interobserver agreement on IBD endoscopic scoring systems: results from the IGIBDENDO educational program. Dig Liver Dis. 2014;46:S4.
39. Levesque BG, Sandborn WJ, Ruel J, et al. Converging goals of treatment of inflammatory bowel disease from clinical trials and practice. Gastroenterology. 2015;148:37-51.