Improving public health by improving clinical trial guidelines and their application

European Heart Journal

Volumn 38, Issue 21, 2017, Pages 1632-1637

  Landray, Martin J ^a   Bax, Jeroen J ^b   Alliot, Laurence ^c   Buyse, Marc ^d,e   Cohen, Adam ^f   Collins, Rory ^a   Hindricks, Gerhard ^g   James, Stefan K ^h   Lane, Sile ⁱ   Maggioni, Aldo P ^j   Meeker O'Connell, Ann ^k   Olsson, Gunnar ^l   Pocock, Stuart J ^m   Rawlins, Michael ⁿ   Sellors, Jonathan ^o   Shinagawa, Kaori ^p   Sipido, Karin R ^q   Smeeth, Liam ^m   Stephens, Richard ^r   Stewart, Murray W ^s   Stough, Wendy Gattis ^t   Sweeney, Fergus ^u   Van De Werf, Frans ^v   Woods, Kerrie ^w   Casadei, Barbara ^a   more..

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^c INSTITUT DE RECHERCHES INTERNATIONALES SERVIER   (France)

^d INTERNATIONAL DRUG DEVELOPMENT INSTITUTE   (Belgium)

^e HASSELT UNIVERSITY   (Belgium)

^f CENTRE FOR HUMAN DRUG RESEARCH   (Netherlands)

^g UNIVERSITY OF LEIPZIG   (Germany)

^h UPPSALA UNIVERSITY   (Sweden)

ⁱ Sense About Science USA   (United Kingdom)

^j ANMCO RESEARCH CENTER   (Italy)

^k JOHNSON AND JOHNSON   (United States)

^l Board Member (Advisory) of European Society of Cardiology   (Sweden)

^m LONDON SCHOOL OF HYGIENE AND TROPICAL MEDICINE   (United Kingdom)

ⁿ MEDICINES AND HEALTHCARE PRODUCTS REGULATORY AGENCY   (United Kingdom)

^o LLP   (United Kingdom)

^p PHARMACEUTICALS AND MEDICAL DEVICES AGENCY   (Japan)

^q UNIVERSITY OF LEUVEN   (Belgium)

^r INSTITUTE OF CANCER RESEARCH   (United Kingdom)

^s Campbell University College of Pharmacy and Health Sciences ^*  (United States)

^t CAMPBELL UNIVERSITY   (United States)

^u EUROPEAN MEDICINES AGENCY   (United Kingdom)

^v UNIVERSITY HOSPITALS LEUVEN   (Belgium)

^w NATIONAL HEALTH SERVICE   (United Kingdom)

more..

Author keywords

Cardiovascular diseases; Clinical trials; Pragmatic clinical trials; Randomized controlled trials

Indexed keywords

CARDIOVASCULAR DISEASE; HUMAN; INFORMED CONSENT; MEDICAL ETHICS; MORBIDITY; PATIENT CARE; PATIENT SAFETY; PRACTICE GUIDELINE; PRIORITY JOURNAL; PUBLIC HEALTH PROBLEM; QUALITY CONTROL; REVIEW; RISK FACTOR; CARDIOLOGY; EDUCATION; ETHICS; HEALTH CARE QUALITY; INTERPERSONAL COMMUNICATION; MASS COMMUNICATION; PUBLIC HEALTH; RANDOMIZED CONTROLLED TRIAL (TOPIC); RISK ASSESSMENT; STANDARDS;

CARDIOLOGY; DIFFUSION OF INNOVATION; DISCLOSURE; HUMANS; INFORMED CONSENT; PATIENT SAFETY; PRACTICE GUIDELINES AS TOPIC; PUBLIC HEALTH; QUALITY ASSURANCE, HEALTH CARE; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RISK ASSESSMENT;

EID: 85020929263     PISSN: 0195668X     EISSN: 15229645     Source Type: Journal    
DOI: 10.1093/eurheartj/ehx086     Document Type: Review

References (69)

1. Calvo G, McMurray JJ, Granger CB, Alonso-Garcia A, Armstrong P, Flather M, Gomez-Outes A, Pocock S, Stockbridge N, Svensson A, Van de Werf F. Large streamlined trials in cardiovascular disease. Eur Heart J 2014;35:544-548.
2. European Commission. Assessment of the functioning of the "Clinical Trials Directive" 2001/20EC public consultation paper. http://ec.europa.eu/health/files/clinicaltrials/docs/2009-10-09-public-consultation-paper.pdf (25 May 2016).
3. Jackson N, Atar D, Borentain M, Breithardt G, van Eickels M, Endres M, Fraass U, Friede T, Hannachi H, Janmohamed S, Kreuzer J, Landray M, Lautsch D, Le Floch C, Mol P, Naci H, Samani N, Svensson A, Thorstensen C, Tijssen J, Vandzhura V, Zalewski A, Kirchhof P. Improving clinical trials for cardiovascular diseases: A position paper from the Cardiovascular Roundtable of the European Society of Cardiology. Eur Heart J 2016;37:747-754.
4. Komajda M, Coats A, Cowie MR, Jackson N, Svensson A, Vardas P. Championing cardiovascular health innovation in Europe. Eur Heart J 2013;34:2630-2635.
5. Roe MT, Mahaffey KW, Ezekowitz JA, Alexander JH, Goodman SG, Hernandez A, Temple T, Berdan L, Califf RM, Harrington RA, Peterson ED, Armstrong PW. The future of cardiovascular clinical research in North America and beyondaddressing challenges and leveraging opportunities through unique academic and grassroots collaborations. Am Heart J 2015;169:743-750.
6. Mentz RJ, Hernandez AF, Berdan LG, Rorick T, Obrien EC, Ibarra JC, Curtis LH, Peterson ED. Good clinical practice guidance and pragmatic clinical trials: balancing the best of both worlds. Circulation 2016;133:872-880.
7. The Academy of Medical Sciences. A new pathway for the regulation and governance of health research. London: The Academy of Medical Sciences, 2011.
8. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). ICH Harmonised Tripartite Guideline: Guideline for good clinical practice (E6 [R1]). http://www. ich.org/fileadmin/Public-Web-Site/ICH-Products/Guidelines/Efficacy/E6/E6-R1-Guideline.pdf (21 October 2015).
9. European Parliament and Council. Regulation (EU) No 536/2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC. http://ec.europa.eu/health/files/eudralex/vol-1/reg-2014-536/reg-2014-536-en. pdf (25 May 2016).
10. Bill & Melinda Gates Foundation. Clinical studies and regulated research assurances. https://www.google.co.uk/url?sa=t&rct=j&q=&esrc=s&source=web&cd= 1&ved=0ahUKEwippdSz3fjQAhWM6iYKHQHQC1wQFggcMAA&url=https%3A %2F%2Fdocs.gatesfoundation.org%2Fdocuments%2Fregulated-research-module. doc&usg=AFQjCNHLGPCeiRA58aqZRFnAGR-P-3LZuQ&cad=rja (25 May 2016).
11. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use. Final business plan. Addendum for ICH E6: Guideline for Good Clinical Practice. http://www.ich.org/fileadmin/Public-Web-Site/ICH-Products/Guidelines/Efficacy/E6/E6-R2-Business-Plan-July-2014.pdf (22 March 2016).
12. Moretrials: The public campaign for more, better, randomised trials. www.more trials.net (22 March 2016).
13. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). ICH Harmonised Guideline: Integrated Addendum to ICD E6(R1): Guideline for good clinical practice E6 (R2). http://www.ich.org/fileadmin/Public-Web-Site/ICH-Products/Guidelines/Efficacy/E6/E6-R2-Step-4.pdf (22 November 2016).
14. Updated open letter to EMA & ICH: From 5 research organisations and an international consortium of 119 health researchers in 22 countries. http://moretrials. net/the-problem/(25 May 2016).
15. Browne LH, Graham PH. Good intentions and ICH-GCP: Trial conduct training needs to go beyond the ICH-GCP document and include the intention-To-Treat principle. Clin Trials 2014;11:629-634.
16. Grimes DA, Hubacher D, Nanda K, Schulz KF, Moher D, Altman DG. The Good Clinical Practice guideline: A bronze standard for clinical research. Lancet 2005;366:172-174.
17. International Council for Harmonisation. ICH Reflection on "GCP Renovation": Modernization of ICH E8 and Subsequent Renovation of ICH E6. http://www.ich. org/fileadmin/Public-Web-Site/ICH-Products/GCP-Renovation/ICH-Reflection-paper-GCP-Renovation-Jan-2017-Final.pdf (24 February 2017).
18. Kramer JM, Smith PB, Califf RM. Impediments to clinical research in the United States. Clin Pharmacol Ther 2012;91:535-541.
19. Morrison BW, Cochran CJ, White JG, Harley J, Kleppinger CF, Liu A, Mitchel JT, Nickerson DF, Zacharias CR, Kramer JM, Neaton JD. Monitoring the quality of conduct of clinical trials: A survey of current practices. Clin Trials 2011;8:342-349.
20. Eisenstein EL, Collins R, Cracknell BS, Podesta O, Reid ED, Sandercock P, Shakhov Y, Terrin ML, Sellers MA, Califf RM, Granger CB, Diaz R. Sensible approaches for reducing clinical trial costs. Clin Trials 2008;5:75-84.
21. Andersen JR, Byrjalsen I, Bihlet A, Kalakou F, Hoeck HC, Hansen G, Hansen HB, Karsdal MA, Riis BJ. Impact of source data verification on data quality in clinical trials: An empirical post hoc analysis of three phase 3 randomized clinical trials. Br J Clin Pharmacol 2015;79:660-668.
22. Clinical Trials Transformation Initiative. CTTI recommendations: Effective and efficient monitoring as a component of quality assurance in the conduct of clinical trials. http://www.ctti-clinicaltrials.org/files/Monitoring/Monitoring-Recommendations. pdf (18 December 2015).
23. Landray MJ, Grandinetti C, Kramer JM, Morrison BW, Ball L, Sherman RE. Clinical trials: rethinking how we ensure quality. Drug Inf J 2012;46:657-660.
24. Reith C, Landray M, Devereaux PJ, Bosch J, Granger CB, Baigent C, Califf RM, Collins R, Yusuf S. Randomized clinical trials-removing unnecessary obstacles. N Engl J Med 2013;369:1061-1065.
25. Tudur SC, Stocken DD, Dunn J, Cox T, Ghaneh P, Cunningham D, Neoptolemos JP. The value of source data verification in a cancer clinical trial. PLoS One 2012;7:e51623.
26. Venet D, Doffagne E, Burzykowski T, Beckers F, Tellier Y, Genevois-Marlin E, Becker U, Bee V, Wilson V, Legrand C, Buyse M. A statistical approach to central monitoring of data quality in clinical trials. Clin Trials 2012;9:705-713.
27. Buyse M, George SL, Evans S, Geller NL, Ranstam J, Scherrer B, Lesaffre E, Murray G, Edler L, Hutton J, Colton T, Lachenbruch P, Verma BL. The role of biostatistics in the prevention, detection and treatment of fraud in clinical trials. Stat Med 1999;18:3435-3451.
28. Clinical Trials Transformation Initiative. Quality objectives of monitoring: Workstream 2 final report. http://www.ctti-clinicaltrials.org/files/documents/MonitoringWS2FinalReport.pdf (25 May 2016).
29. European Medicines Agency. Reflection paper on risk based quality management in clinical trials. http://www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/2013/11/WC500155491.pdf (23 October 2015).
30. Medical Research Council, Department of Health, and Medicines and Healthcare Products Regulatory Agency Joint Project. Risk-Adapted approaches to the management of clinical trials of investigational medicinal products. http://webarchive. nationalarchives.gov.uk/20141205150130/http://www.mhra.gov.uk/home/groups/l-ctu/documents/websiteresources/con111784.pdf (23 October 2015).
31. U.S. Department of Health and Human Services and Food and Drug Administration. Guidance for Industry. Oversight of Clinical Investigations-A Risk-Based Approach to Monitoring. http://www.fda.gov/downloads/Drugs/. . ./Guidances/UCM269919.pdf (23 October 2015).
32. Jarow JP, Casak S, Chuk M, Ehrlich LA, Khozin S. The majority of expedited investigational new drug safety reports are uninformative. Clin Cancer Res 2016;22:2111-2113.
33. U.S. Department of Health and Human Services and Food and Drug Administration. Investigational new drug safety reporting requirements for human drug and biological products and safety reporting requirements for bioavailability and bioequivalence studies in humans, 21 CFR Parts 312 and 320. https://www.gpo. gov/fdsys/pkg/FR-2010-09-29/pdf/2010-24296.pdf (25 May 2016).
34. U.S. Department of Health and Human Services Food and Drug Administration. Guidance for industry and investigators: Safety reporting requirements for INDs and BA/BE studies. http://www.fda.gov/downloads/Drugs/. . ./Guidances/UCM227351.pdf (25 May 2016).
35. U.S. Department of Health and Human Services Food and Drug Administration. Safety assessment for IND safety reporting: Guidance for industry. http://www.fda.gov/down loads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM477584.pdf (25 May 2016).
36. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). ICH Harmonised Tripartite Guideline: Clinical safety data management-definitions and standards for expedited reporting (E2A). http://www.ich.org/fileadmin/Public-Web-Site/ICH-Products/Guidelines/Efficacy/E2A/Step4/E2A-Guideline.pdf (21 October 2015).
37. Kenter MJ, Cohen AF. The return of the prodigal son and the extraordinary development route of antibody TGN1. Br J Clin Pharmacol 2015;79:545-547.
38. U.S. Department of Health and Human Services Food and Drug Administration. FDA Webinar: New draft guidance on safety data collection. http://www.fda.gov/Drugs/ucm296761.htm (25 May 2016).
39. US Department of Health and Human Services Food and Drug Administration. Guidance for clinical investigators, sponsors, and IRBs: Adverse event reporting to IRBs-improving human subject protection. http://www.fda.gov/downloads/RegulatoryInformation/Guidances/UCM126572.pdf (25 May 2016).
40. Glasziou P, Chalmers I, Rawlins M, McCulloch P. When are randomised trials unnecessary? Picking signal from noise. Bmj 2007;334:349-351.
41. Rawlins M. De testimonio: on the evidence for decisions about the use of therapeutic interventions. Lancet 2008;372:2152-2161.
42. U.S. Department of Health and Human Services Food and Drug Administration. Guidance for industry: Determining the extent of safety data collection needed in late stage premarket and postapproval clinical investigations. http://www.fda. gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm291158.pdf (25 May 2016).
43. Archdeacon P, Grandinetti C, Vega JM, Balderson D, Kramer JM. Optimizing expedited safety reporting for drugs and biologics subject to an investigational new drug application. Ther Innov Regul Sci 2013;doi:10.1177/2168479013509382.
44. Swedberg K, Borer JS, Pitt B, Pocock S, Rouleau J. Challenges to data monitoring committees when regulatory authorities intervene. N Engl J Med 2016;374:1584.
45. Frobert O, Lagerqvist B, Olivecrona GK, Omerovic E, Gudnason T, Maeng M, Aasa M, Angeras O, Calais F, Danielewicz M, Erlinge D, Hellsten L, Jensen U, Johansson AC, Karegren A, Nilsson J, Robertson L, Sandhall L, Sjogren I, Ostlund O, Harnek J, James SK. Thrombus aspiration during ST-segment elevation myocardial infarction. N Engl J Med 2013;369:1587-1597.
46. Cowie MR, Blomster JI, Curtis LH, Duclaux S, Ford I, Fritz F, Goldman S, Janmohamed S, Kreuzer J, Leenay M, Michel A, Ong S, Pell JP, Southworth MR, Stough WG, Thoenes M, Zannad F, Zalewski A. Electronic health records to improve patient care and facilitate clinical research. Clin Res Cardiol 2016;doi: 10.1007/s00392-016-1025-6.
47. Hernandez AF, Fleurence RL, Rothman RL. The ADAPTABLE Trial and PCORnet: shining light on a new research paradigm. Ann Intern Med 2015;163:635-636.
48. Vickers AJ, Scardino PT. The clinically-integrated randomized trial: proposed novel method for conducting large trials at low cost. Trials 2009;10:14.
49. Clinical Trials Transformation Initiative. Using mobile technology to facilitate clinical trials. http://www.ctti-clinicaltrials.org/what-we-do/ctti-projects/mobile-clin ical-Trials (25 May 2016).
50. Staa TP, Goldacre B, Gulliford M, Cassell J, Pirmohamed M, Taweel A, Delaney B, Smeeth L. Pragmatic randomised trials using routine electronic health records: putting them to the test. Bmj 2012;344:e55.
51. van Staa TP, Dyson L, McCann G, Padmanabhan S, Belatri R, Goldacre B, Cassell J, Pirmohamed M, Torgerson D, Ronaldson S, Adamson J, Taweel A, Delaney B, Mahmood S, Baracaia S, Round T, Fox R, Hunter T, Gulliford M, Smeeth L. The opportunities and challenges of pragmatic point-of-care randomised trials using routinely collected electronic records: evaluations of two exemplar trials. Health Technol Assess 2014;18:1-146.
52. Clinical Trials Transformation Initiative. Mobile clinical trials (MCT). https://www. ctti-clinicaltrials.org/programs/mobile-clinical-Trials (6 December 2016).
53. www.clinicalstudydatarequest.com (25 May 2016).
54. Nisen P, Rockhold F. Access to patient-level data from GlaxoSmithKline clinical trials. N Engl J Med 2013;369:475-478.
55. Taichman DB, Backus J, Baethge C, Bauchner H, de Leeuw PW, Drazen JM, Fletcher J, Frizelle FA, Groves T, Haileamlak A, James A, Laine C, Peiperl L, Pinborg A, Sahni P, Wu S. Sharing clinical trial data: A proposal from the International Committee of Medical Journal Editors. The Lancet 2016;387:e9-e11.
56. Devereaux PJ, Guyatt G, Gerstein H, Connolly S, Yusuf S. Toward fairness in data sharing. N Engl J Med 2016;375:405-407.
57. Krumholz HM, Waldstreicher J. The Yale Open Data Access (YODA) Project-A mechanism for data sharing. N Engl J Med 2016;375:403-405.
58. Patel MR, Armstrong PW, Bhatt DL, Braunwald E, Camm AJ, Fox KA, Harrington RA, Hiatt WR, James SK, Kirtane AJ, Leon MB, Lincoff AM, Mahaffey KW, Mauri L, Mehran R, Mehta SR, Montalescot G, Nicholls SJ, Perkovic V, Peterson ED, Pocock SJ, Roe MT, Sabatine MS, Sekeres M, Solomon SD, Steg G, Stone GW, Van de Werf F, Wallentin L, White HD, Gibson M. Sharing data from cardiovascular clinical trials-A proposal. N Engl J Med 2016;375:407-409.
59. Warren E. Strengthening research through data sharing. N Engl J Med 2016;375:401-403.
60. Horton R. Offline: data sharing-why editors may have got it wrong. Lancet 2016;388:1143.
61. Landray MJ, Haynes R, Hopewell JC, Parish S, Aung T, Tomson J, Wallendszus K, Craig M, Jiang L, Collins R, Armitage J. Effects of extended-release niacin with laropiprant in high-risk patients. N Engl J Med 2014;371:203-212.
62. The HPS2-THRIVE Collaborative Group. Supplementary appendix 2: THRIVE adverse events by MedDRA term. http://www.nejm.org/doi/suppl/10.1056/NEJMoa1300955/suppl-file/nejmoa1300955-Appendix2.html (18 December 2015).
63. Califf RM, Platt R. Embedding cardiovascular research into practice. Jama 2013;310:2037-2038.
64. Institute of Medicine. The Learning Healthcare System: Roundtable on Evidence-based Medicine Workshop Summary. Washington DC: National Academies Press; 2007.
65. Bhatt DL, Kandzari DE, Oneill WW, Dagostino R, Flack JM, Katzen BT, Leon MB, Liu M, Mauri L, Negoita M, Cohen SA, Oparil S, Rocha-Singh K, Townsend RR, Bakris GL. A controlled trial of renal denervation for resistant hypertension. N Engl J Med 2014;370:1393-1401.
66. Clinical Trials Transformation Initiative. http://www.ctti-clinicaltrials.org/home (25 May 2016).
67. Echt DS, Liebson PR, Mitchell LB, Peters RW, Obias-Manno D, Barker AH, Arensberg D, Baker A, Friedman L, Greene HL. Mortality and morbidity in patients receiving encainide, flecainide, or placebo. The Cardiac Arrhythmia Suppression Trial. N Engl J Med 1991;324:781-788.
68. U.S. Department of Health and Human Services. Federal policy for the protection of human subjects: proposed rules. http://www.gpo.gov/fdsys/pkg/FR-2015-09-08/pdf/2015-21756.pdf (24 September 2015).
69. U.S. Department of Health and Human Services Food and Drug Administration. Code of Federal Regulations Title 21. www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=312.3 (25 May 2016).