Good Clinical Practice Guidance and Pragmatic Clinical Trials : Balancing the Best of Both Worlds

Circulation

Volumn 133, Issue 9, 2016, Pages 872-880

  Mentz, Robert J ^a,b   Hernandez, Adrian F ^a,b   Berdan, Lisa G ^a   Rorick, Tyrus ^a   O'Brien, Emily C ^a,b   Ibarra, Jenny C ^a   Curtis, Lesley H ^a   Peterson, Eric D ^a,b  

^a DUKE CLINICAL RESEARCH INSTITUTE   (United States)

^b DUKE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Clinical protocols; clinical trial; ethics; pragmatic clinical trial

Indexed keywords

ARTICLE; AUTHORITY; CLINICAL DECISION MAKING; CLINICAL TRIAL (TOPIC); COST; ELECTRONIC MEDICAL RECORD; ETHICS; GOOD CLINICAL PRACTICE; HUMAN; INTERNAL CONSISTENCY; MULTICENTER STUDY (TOPIC); PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); REGISTER; STANDARD; TENSION; METHODOLOGY; PRACTICE GUIDELINE; PROCEDURES; STANDARDS;

CLINICAL TRIALS AS TOPIC; HUMANS; PRACTICE GUIDELINES AS TOPIC; RESEARCH DESIGN;

EID: 84959476816     PISSN: 00097322     EISSN: 15244539     Source Type: Journal    
DOI: 10.1161/CIRCULATIONAHA.115.019902     Document Type: Article

References (64)

1. Sackett DL, Rosenberg WM, Gray JA, Haynes RB, Richardson WS. Evidence based medicine: what it is and what it isn't. BMJ. 1996;312:71-72.
2. ACCF/AHA Task Force on Practice Guidelines. Methodology Manual and Policies From the ACCF/AHA Task Force on Practice Guidelines. 2010. http://assets.cardiosource.com/Methodology-Manual-for-ACC-AHA-Writing-Committees.pdf Accessed May 12, 2015.
3. Collins R, MacMahon S. Reliable assessment of the effects of treatment on mortality and major morbidity, I: clinical trials. Lancet. 2001;357:373-380. doi: 10.1016/S0140-6736(00)03651-5.
4. International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use. ICH Harmonized Tripartite Guideline. Guideline for Good Clinical Practice E6(R1). 1996. http://www.ich.org/fileadmin/Public-Web-Site/ICH-Products/Guidelines/Efficacy/E6/E6-R1-Guideline.pdf Accessed May 12, 2015.
5. Devine S, Dagher RN, Weiss KD, Santana VM. Good clinical practice and the conduct of clinical studies in pediatric oncology. Pediatr Clin North Am. 2008;55:187-209, xi. doi: 10.1016/j.pcl.2007.10.008.
6. 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. doi: 10.1056/NEJMsb1300760.
7. Califf RM. Clinical trials bureaucracy: unintended consequences of well-intentioned policy. Clin Trials. 2006;3:496-502. doi: 10.1177/1740774506073173.
8. McMahon AD, Conway DI, Macdonald TM, McInnes GT. The unintended consequences of clinical trials regulations. PLoS Med. 2009;3:e1000131. doi: 10.1371/journal.pmed.1000131.
9. Frankovich J, Longhurst CA, Sutherland SM. Evidence-based medicine in the EMR era. N Engl J Med. 2011;365:1758-1759. doi: 10.1056/NEJMp1108726.
10. Collins FS, Hudson KL, Briggs JP, Lauer MS. PCORnet: turning a dream into reality. J Am Med Inform Assoc. 2014;21:576-577. doi: 10.1136/amiajnl-2014-002864.
11. Randomised trial of intravenous atenolol among 16 027 cases of suspected acute myocardial infarction: ISIS-1. First International Study of Infarct Survival Collaborative Group. Lancet. 1986;2:57-66.
12. Yusuf S. Randomized clinical trials: slow death by a thousand unnecessary policies CMAJ. 2004;171:889-892; discussion 892. doi: 10.1503/cmaj.1040884.
13. Butler J, Fonarow GC, O'Connor C, Adams K, Bonow RO, Cody RJ, Collins SP, Dunnmon P, Dinh W, Fiuzat M, Georgiopoulou VV, Grant S, Kim SY, Kupfer S, Lefkowitz M, Mentz RJ, Misselwitz F, Pitt B, Roessig L, Schelbert E, Shah M, Solomon S, Stockbridge N, Yancy C, Gheorghiade M. Improving cardiovascular clinical trials conduct in the United States: recommendation from clinicians, researchers, sponsors, and regulators. Am Heart J. 2015;169:305-314. doi: 10.1016/j.ahj.2014.12.001.
14. Glickman SW, McHutchison JG, Peterson ED, Cairns CB, Harrington RA, Califf RM, Schulman KA. Ethical and scientific implications of the globalization of clinical research. N Engl J Med. 2009;360:816-823. doi: 10.1056/NEJMsb0803929.
15. Getz KA, Campo RA, Kaitin KI. Variability in protocol design complexity by phase and therapeutic area. Drug Inform J. 2011;45:413-420.
16. Getz KA, Stergiopoulos S, Marlborough M, Whitehill J, Curran M, Kaitin KI. Quantifying the magnitude and cost of collecting extraneous protocol data. Am J Ther. 2015;22:117-124. doi: 10.1097/MJT.0b013e31826fc4aa.
17. Martin SS, Ou FS, Newby LK, Sutton V, Adams P, Felker GM, Wang TY. Patient-and trial-specific barriers to participation in cardiovascular randomized clinical trials. J Am Coll Cardiol. 2013;61:762-769. doi: 10.1016/j.jacc.2012.10.046.
18. Butler J, Tahhan AS, Georgiopoulou VV, Kelkar A, Lee M, Khan B, Peterson E, Fonarow GC, Kalogeropoulos AP, Gheorghiade M. Trends in characteristics of cardiovascular clinical trials 2001-2012. Am Heart J. 2015;170:263-272.e2.
19. Butler J, Subacius H, Vaduganathan M, Fonarow GC, Ambrosy AP, Konstam MA, Maggioni A, Mentz RJ, Swedberg K, Zannad F, Gheorghiade M; EVEREST Investigators. Relationship between clinical trial site enrollment with participant characteristics, protocol completion, and outcomes: insights from the EVEREST (Efficacy of Vasopressin Antagonism in Heart Failure: Outcome Study with Tolvaptan) trial. J Am Coll Cardiol. 2013;61:571-579. doi: 10.1016/j.jacc.2012.10.025.
20. Lovato LC, Hill K, Hertert S, Hunninghake DB, Probstfield JL. Recruitment for controlled clinical trials: literature summary and annotated bibliography. Control Clin Trials. 1997;18:328-352.
21. Bailey W, Cruickshank C, Sharma N. Make your move: taking clinical trials to the best location. https://www.atkearney.com/health/ideas-insights/article/-/asset-publisher/LCcgOeS4t85g/content/make-your-move/10192. Accessed October 7, 2015.
22. World Medical Association. Declaration of Helsinki serves as guide to physicians. JAMA. 1964;189:33-34.
23. U.S. National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research. The Belmont Report: Ethical Principles and Guidelines for the Conduct of Research Involving Human Subjects. Washington, DC: US Department of Health, Education, and Welfare, 1978.
24. US Department of Health and Human Services. Code of Federal Regulations. Title 5, Part B. Public Welfare: Protection of Human Subjects. http://www.hhs.gov/ohrp/humansubjects/guidance/45cfr46.html. Accessed October 7, 2015.
25. World Medical Association Declaration of Helsinki. Recommendations guiding physicians in biomedical research involving human subjects. JAMA. 1997;277:925-926.
26. Sims JM. A brief review of the Belmont report. Dimens Crit Care Nurs. 2010;29:173-174. doi: 10.1097/DCC.0b013e3181de9ec5.
27. 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. doi: 10.1016/S0140-6736(05)66875-4.
28. Schulz KF, Grimes DA. Allocation concealment in randomised trials: defending against deciphering. Lancet. 2002;359:614-618. doi: 10.1016/S0140-6736(02)07750-4.
29. Mills EJ, Ayers D, Chou R, Thorlund K. Are current standards of reporting quality for clinical trials sufficient in addressing important sources of bias Contemp Clin Trials. 2015;45(pt A):2-7. doi: 10.1016/j.cct.2015.07.019.
30. US Department of Health and Human Services. Food and Drug Administration. Guidance for Industry: Oversight of Clinical Investigations-A Risk-Based Approach to Monitoring. www.fda.gov/downloads/Drugs/Guidances/UCM269919.pdf Accessed August 17, 2015.
31. Fordyce CB, Roe MT, Ahmad T, Libby P, Borer JS, Hiatt WR, Bristow MR, Packer M, Wasserman SM, Braunstein N, Pitt B, DeMets DL, Cooper-Arnold K, Armstrong PW, Berkowitz SD, Scott R, Prats J, Galis ZS, Stockbridge N, Peterson ED, Califf RM. Cardiovascular drug development: is it dead or just hibernating J Am Coll Cardiol. 2015;65:1567-1582. doi: 10.1016/j.jacc.2015.03.016.
32. 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 beyond-addressing challenges and leveraging opportunities through unique academic and grassroots collaborations. Am Heart J. 2015;169:743-750. doi: 10.1016/j.ahj.2015.03.002.
33. Tunis SR, Stryer DB, Clancy CM. Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy. JAMA. 2003;290:1624-1632. doi: 10.1001/jama.290.12.1624.
34. Thorpe KE, Zwarenstein M, Oxman AD, Treweek S, Furberg CD, Altman DG, Tunis S, Bergel E, Harvey I, Magid DJ, Chalkidou K. A pragmaticexplanatory continuum indicator summary (PRECIS): a tool to help trial designers. J Clin Epidemiol. 2009;62:464-475. doi: 10.1016/j. jclinepi.2008.12.011.
35. Rothwell PM. External validity of randomised controlled trials: "to whom do the results of this trial apply". Lancet. 2005;365:82-93. doi: 10.1016/S0140-6736(04)17670-8.
36. An international randomized trial comparing four thrombolytic strategies for acute myocardial infarction. The GUSTO investigators. N Engl J Med. 1993;329:673-82.
37. Loudon K, Treweek S, Sullivan F, Donnan P, Thorpe KE, Zwarenstein M. The PRECIS-2 tool: designing trials that are fit for purpose. BMJ. 2015;350:h2147.
38. Fröbert O, Lagerqvist B, Olivecrona GK, Omerovic E, Gudnason T, Maeng M, Aasa M, Angerås O, Calais F, Danielewicz M, Erlinge D, Hellsten L, Jensen U, Johansson AC, Kåregren A, Nilsson J, Robertson L, Sandhall L, Sjögren I, Ostlund O, Harnek J, James SK; TASTE Trial. Thrombus aspiration during ST-segment elevation myocardial infarction. N Engl J Med. 2013;369:1587-1597. doi: 10.1056/NEJMoa1308789.
39. Hess CN, Rao SV, Kong DF, Aberle LH, Anstrom KJ, Gibson CM, Gilchrist IC, Jacobs AK, Jolly SS, Mehran R, Messenger JC, Newby LK, Waksman R, Krucoff MW. Embedding a randomized clinical trial into an ongoing registry infrastructure: unique opportunities for efficiency in design of the Study of Access site For Enhancement of Percutaneous Coronary Intervention for Women (SAFE-PCI for Women). Am Heart J. 2013;166:421-428. doi: 10.1016/j.ahj.2013.06.013.
40. Chin CT, Wang TY, Anstrom KJ, Zhu B, Maa JF, Messenger JC, Ryan KA, Davidson-Ray L, Zettler M, Effron MB, Mark DB, Peterson ED. Treatment with adenosine diphosphate receptor inhibitors-longitudinal assessment of treatment patterns and events after acute coronary syndrome (TRANSLATE-ACS) study design: expanding the paradigm of longitudinal observational research. Am Heart J. 2011;162:844-851. doi: 10.1016/j.ahj.2011.08.021.
41. Choudhry NK, Brennan T, Toscano M, Spettell C, Glynn RJ, Rubino M, Schneeweiss S, Brookhart AM, Fernandes J, Mathew S, Christiansen B, Antman EM, Avorn J, Shrank WH. Rationale and design of the Post-MI FREEE trial: a randomized evaluation of first-dollar drug coverage for post-myocardial infarction secondary preventive therapies. Am Heart J. 2008;156:31-36. doi: 10.1016/j.ahj.2008.03.021.
42. 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. doi: 10.7326/M15-1460.
43. Simon GE, VonKorff M, Heiligenstein JH, Revicki DA, Grothaus L, Katon W, Wagner EH. Initial antidepressant choice in primary care. Effectiveness and cost of fluoxetine vs tricyclic antidepressants. JAMA. 1996;275:1897-1902.
44. Barry SJ, Dinnett E, Kean S, Gaw A, Ford I. Are routinely collected NHS administrative records suitable for endpoint identification in clinical trials Evidence from the West of Scotland Coronary Prevention Study. PLoS One. 2013;8:e75379. doi: 10.1371/journal.pone.0075379.
45. Ingelsson E, Arnlöv J, Sundström J, Lind L. The validity of a diagnosis of heart failure in a hospital discharge register. Eur J Heart Fail. 2005;7:787-791. doi: 10.1016/j.ejheart.2004.12.007.
46. Bayley KB, Belnap T, Savitz L, Masica AL, Shah N, Fleming NS. Challenges in using electronic health record data for CER: experience of 4 learning organizations and solutions applied. Med Care. 2013;51(8 suppl 3):S80-S86. doi: 10.1097/MLR.0b013e31829b1d48.
47. Botsis T, Hartvigsen G, Chen F, Weng C. Secondary Use of EHR: data quality issues and informatics opportunities. AMIA Jt Summits Transl Sci Proc. 2010;2010:1-5.
48. Pham HH, Schrag D, O'Malley AS, Wu B, Bach PB. Care patterns in Medicare and their implications for pay for performance. N Engl J Med. 2007;356:1130-1139. doi: 10.1056/NEJMsa063979.
49. Bourgeois FC, Olson KL, Mandl KD. Patients treated at multiple acute health care facilities: quantifying information fragmentation. Arch Intern Med. 2010;170:1989-1995. doi: 10.1001/archinternmed.2010.439.
50. Geller NL, Kim DY, Tian X. Smart technology in lung disease clinical trials. Chest. 2016;149:22-26. doi: 10.1378/chest.15-1314.
51. Psaty BM, Breckenridge AM. Mini-sentinel and regulatory science-big data rendered fit and functional. N Engl J Med. 2014;370:2165-2167. doi: 10.1056/NEJMp1401664.
52. Sen Biswas M, Newby LK, Bastian LA, Peterson ED, Sugarman J. Who refuses enrollment in cardiac clinical trials Clin Trials. 2007;4:258-263. doi: 10.1177/1740774507079434.
53. Hasbrouck J, Tindal GA. Oral reading fluency norms: a valuable assessment tool for reading teachers. The Reading Teacher. 2006;59:636-644.
54. McCambridge J, Witton J, Elbourne DR. Systematic review of the Hawthorne effect: new concepts are needed to study research participation effects. J Clin Epidemiol. 2014;67:267-277. doi: 10.1016/j. jclinepi.2013.08.015.
55. Lauer MS, Kiley JP, Mockrin SC, Mensah GA, Hoots WK, Patel Y, Cook NL, Patterson AP, Gibbons GH. National Heart, Lung, and Blood Institute (NHLBI) strategic visioning: setting an agenda together for the NHLBI of 2025. Circulation. 2015;131:1106-1109. doi: 10.1161/CIRCULATIONAHA.115.015712.
56. Lauer MS, D'Agostino RB Sr. The randomized registry trial-the next disruptive technology in clinical research N Engl J Med. 2013;369:1579-1581. doi: 10.1056/NEJMp1310102.
57. Gill D. Re-inventing clinical trials through TransCelerate. Nat Rev Drug Discov. 2014;13:787-788. doi: 10.1038/nrd4437.
58. TransCelerate. Risk-Based Monitoring. http://www.transceleratebiopharmainc. com/assets/risk-based-monitoring/on Accessed August 12, 2015.
59. Tenaerts P, Madre L, Archdeacon P, Califf RM. The Clinical Trials Transformation Initiative: innovation through collaboration. Nat Rev Drug Discov. 2014;13:797-798. doi: 10.1038/nrd4442.
60. CTTI. Quality by Design. http://www.ctti-clinicaltrials.org/files/QbD-QRM/QbDQRM-PrinciplesDocument-DeviceWorkshop-2014-01-16.pdf Accessed August 12, 2015.
61. Mini-Sentinel and Clinical Trials Transformation Initiative. Developing Approaches to Conducting Randomized Trials using the Mini-Sentinel Distributed Database. http://www.mini-sentinel.org/work-products/Statistical-Methods/Mini-Sentinel-Methods-CTTI-Developing-Approaches-to-Conducting-Randomized-Trials-Using-MSDD.pdf Accessed August 12, 2015.
62. Hess CN, Roe MT, Gibson CM, Temple RJ, Pencina MJ, Zarin DA, Anstrom KJ, Alexander JH, Sherman RE, Fiedorek FT, Mahaffey KW, Lee KL, Chow SC, Armstrong PW, Califf RM. Independent data monitoring committees: preparing a path for the future. Am Heart J. 2014;168:135-41.e1. doi: 10.1016/j.ahj.2014.05.003.
63. Rao SV, Califf RM, Kramer JM, Peterson ED, Gross TP, Pepine CJ, Williams DO, Donohoe D, Waksman R, Mehran R, Krucoff MW. Postmarket evaluation of breakthrough technologies. Am Heart J. 2008;156:201-208. doi: 10.1016/j.ahj.2008.01.036.
64. Berger JS, Bairey-Merz CN, Redberg RF, Douglas PS. Improving the quality of care for women with cardiovascular disease: report of a DCRI Think Tank, March 8 to 9, 2007. Am Heart J. 2008;156:816-25, 825.e1.