Series: Pragmatic trials and real world evidence: Paper 1. Introduction

Journal of Clinical Epidemiology

Volumn 88, Issue , 2017, Pages 7-13

  Zuidgeest, Mira G P ^a   Goetz, Iris ^b   Groenwold, Rolf H H ^a   Irving, Elaine ^c   van Thiel, Ghislaine J M W ^a   Grobbee, Diederick E ^a  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^b ELI LILLY AND COMPANY   (United States)

^c GLAXOSMITHKLINE   (United Kingdom)

Author keywords

Generalizability; Pragmatic trial; Real world evidence; Routine clinical practice; Trial conduct; Trial design

Indexed keywords

PLACEBO;

ARTICLE; CLINICAL EFFECTIVENESS; CLINICAL PRACTICE; EVIDENCE BASED PRACTICE; HEALTH CARE POLICY; HEALTH CARE SYSTEM; HUMAN; INFORMATION PROCESSING; MEDICAL CARE; OUTCOME ASSESSMENT; POPULATION; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SOCIAL MARKETING; STUDY DESIGN; TREATMENT RESPONSE; EPIDEMIOLOGY; OBSERVATIONAL STUDY; PATIENT SELECTION; PROCEDURES;

DATA COLLECTION; EPIDEMIOLOGIC RESEARCH DESIGN; HUMANS; OBSERVATIONAL STUDIES AS TOPIC; PATIENT SELECTION; RANDOMIZED CONTROLLED TRIALS AS TOPIC;

EID: 85021105573     PISSN: 08954356     EISSN: 18785921     Source Type: Journal    
DOI: 10.1016/j.jclinepi.2016.12.023     Document Type: Article

References (45)

1. Naci, H., O'Connor, A.B., Assessing comparative effectiveness of new drugs before approval using prospective network meta-analyses. J Clin Epidemiol 66 (2013), 812–816.
2. Droitcour, J., Silberman, G., Chelimsky, E., A new form of meta-analysis for combining results from randomized clinical trials and medical-practice databases. Int J Technol Assess Health Care 9 (1993), 440–449.
3. Kaizar, E.E., Estimating treatment effect via simple cross design synthesis. Stat Med 30 (2011), 2986–3009.
4. Rothwell, P.M., External validity of randomised controlled trials: “to whom do the results of this trial apply?”. Lancet 365 (2005), 82–93.
5. Calvert, M., Wood, J., Freemantle, N., Designing “Real-World” trials to meet the needs of health policy makers at marketing authorization. J Clin Epidemiol 64 (2011), 711–717.
6. Califf, R.M., Sugarman, J., Exploring the ethical and regulatory issues in pragmatic clinical trials. Clin Trials 12 (2015), 436–441.
7. Makady, A., GetReal. Updated glossary of definitions of common terms, 2015 Available at https://www.imi-getreal.eu/Portals/1/Documents/Publications/D1.3%20GetReal%20Glossary%20-%20Update.pdf. Accessed August 9, 2016.
8. Pocock, S.J., Elbourne, D.R., Randomized trials or observational tribulations?. N Engl J Med 342 (2000), 1907–1909.
9. Tunis, S.R., Stryer, D.B., Clancy, C.M., Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy. JAMA 290 (2003), 1624–1632.
10. Zwarenstein, M., Oxman, A., Why are so few randomized trials useful, and what can we do about it?. J Clin Epidemiol 59 (2006), 1125–1126.
11. Freemantle, N., Strack, T., Real-world effectiveness of new medicines should be evaluated by appropriately designed clinical trials. J Clin Epidemiol 63 (2010), 1053–1058.
12. Ware, J.H., Hamel, M.B., Pragmatic trials–guides to better patient care?. N Engl J Med 364 (2011), 1685–1687.
13. Price, D., Hillyer, E.V., van der Molen, T., Efficacy versus effectiveness trials: informing guidelines for asthma management. Curr Opin Allergy Clin Immunol 13 (2013), 50–57.
14. Sackett, D.L., Clinician-trialist rounds: 16. Mind your explanatory and pragmatic attitudes! - part 1: what?. Clin Trials 10 (2013), 495–498.
15. Berger, M.L., Dreyer, N., Anderson, F., Towse, A., Sedrakyan, A., Normand, S.L., Prospective observational studies to assess comparative effectiveness: the ISPOR good research practices task force report. Value Health 15 (2012), 217–230.
16. Hoes, A.W., Grobbee, D.E., Clinical epidemiology. 2nd ed., 2015, Jones & Bartlett Learning, Burlington, MA.
17. Rothman, K.J., Greenland, S., Lash, T.L., Modern Epidemiology. 3rd edn. 2008, Lippincott, Williams & Wilkins, Philadelphia, PA.
18. Anglemyer, A., Horvath, H.T., Bero, L., Healthcare outcomes assessed with observational study designs compared with those assessed in randomized trials. Cochrane Database Syst Rev(4), 2014, MR000034.
19. Hemkens, L.G., Contopoulos-Ioannidis, D.G., Ioannidis, J.P., Agreement of treatment effects for mortality from routinely collected data and subsequent randomized trials: meta-epidemiological survey. BMJ, 352, 2016, i493.
20. Grobbee, D.E., Hoes, A.W., Confounding and indication for treatment in evaluation of drug treatment for hypertension. BMJ 315 (1997), 1151–1154.
21. Sackett, D.L., Straus, S.E., Richardson, W.S., Rosenberg, W., Haynes, B., Evidence-based medicine: how to practice and teach EBM. 2nd ed., 2000, Churchill Livingstone, Edinburgh.
22. Bothwell, L.E., Greene, J.A., Podolsky, S.H., Jones, D.S., Assessing the gold standard–lessons from the history of RCTs. N Engl J Med 374 (2016), 2175–2181.
23. Jones, D.S., Podolsky, S.H., The history and fate of the gold standard. Lancet 385 (2015), 1502–1503.
24. Schwartz, D., Lellouch, J., Explanatory and pragmatic attitudes in therapeutical trials. J Chronic Dis 20 (1967), 637–648.
25. Thorpe, K.E., Zwarenstein, M., Oxman, A.D., Treweek, S., Furberg, C.D., Altman, D.G., et al. A pragmatic-explanatory continuum indicator summary (PRECIS): a tool to help trial designers. J Clin Epidemiol 62 (2009), 464–475.
26. Kane, J.M., Kishimoto, T., Correll, C.U., Assessing the comparative effectiveness of long-acting injectable vs. oral antipsychotic medications in the prevention of relapse provides a case study in comparative effectiveness research in psychiatry. J Clin Epidemiol 66 (2013), S37–S41.
27. Riddle, D.L., Johnson, R.E., Jensen, M.P., Keefe, F.J., Kroenke, K., Bair, M.J., et al. The Pragmatic-Explanatory Continuum Indicator Summary (PRECIS) instrument was useful for refining a randomized trial design: experiences from an investigative team. J Clin Epidemiol 63 (2010), 1271–1275.
28. Bratton, D.J., Nunn, A.J., Alternative approaches to tuberculosis treatment evaluation: the role of pragmatic trials. Int J Tuberc Lung Dis 15 (2011), 440–446.
29. Selby, P., Brosky, G., Oh, P.I., Raymond, V., Ranger, S., How pragmatic or explanatory is the randomized, controlled trial? The application and enhancement of the PRECIS tool to the evaluation of a smoking cessation trial. BMC Med Res Methodol, 12, 2012, 101.
30. Loudon, K., Treweek, S., Sullivan, F., Donnan, P., Thorpe, K.E., Zwarenstein, M., The PRECIS-2 tool: designing trials that are fit for purpose. BMJ, 350, 2015, h2147.
31. Dekkers, O.M., Bossuyt, P.M., Vandenbroucke, J.P., How trial results are intended to be used: is PRECIS-2 a step forward?. J Clin Epidemiol 84 (2017), 25–26.
32. Nordon, C., Karcher, H., Groenwold, R.H., Ankarfeldt, M.Z., Pichler, F., Chevrou-Severac, H., et al. The “Efficacy-Effectiveness gap”: Historical Background and current Conceptualization. Value Health 19 (2016), 75–81.
33. Sedgwick, P., Explanatory trials versus pragmatic trials. BMJ, 349, 2014, g6694.
34. Peto, R., Baigent, C., Trials: the next 50 years. Large scale randomised evidence of moderate benefits. BMJ 317 (1998), 1170–1171.
35. Peto, R., Collins, R., Gray, R., Large-scale randomized evidence: large, simple trials and overviews of trials. J Clin Epidemiol 48 (1995), 23–40.
36. van Staa, T.P., Dyson, L., McCann, G., Padmanabhan, S., Belatri, R., Goldacre, B., et al. The opportunities and challenges of pragmatic point-of-care randomised trials using routinely collected electronic records: evaluations of two exemplar trials. Health Technol Assess 18 (2014), 1–146.
37. New, J.P., Bakerly, N.D., Leather, D., Woodcock, A., Obtaining real-world evidence: the Salford lung study. Thorax 69 (2014), 1152–1154.
38. Segal, J.B., The prospect of early pharmaceutical pragmatic clinical trials: a background paper. 2013, Center for Medical Technology Policy Available at: http://www.cmtpnet.org/docs/resources/Segal-PCT-Background-Paper.pdf. Accessed August 9, 2016.
39. Laken, M.A., Dawson, R., Engelman, O., Lovelace, O., Way, C., Egan, B.M., Comparative effectiveness research in the “real” world: lessons learned in a study of treatment-resistant hypertension. J Am Soc Hypertens 7 (2013), 95–101.
40. Kolitsopoulos, F.M., Strom, B.L., Faich, G., Eng, S.M., Kane, J.M., Reynolds, R.F., Lessons learned in the conduct of a global, large simple trial of treatments indicated for schizophrenia. Contemp Clin Trials 34 (2013), 239–247.
41. Zamorano, J., Erdine, S., Lopez, A.P., Kim, J.H., Al Khadra, A., Westergaard, M., et al. Design and rationale of a real-life study to compare treatment strategies for cardiovascular risk factors: the CRUCIAL study. Postgrad Med 122 (2010), 7–15.
42. Saag, K.G., Mohr, P.E., Esmail, L., Mudano, A.S., Wright, N., Beukelman, T., et al. Improving the efficiency and effectiveness of pragmatic clinical trials in older adults in the United States. Contemp Clin Trials 33 (2012), 1211–1216.
43. Delaney, B.C., Curcin, V., Andreasson, A., Arvanitis, T.N., Bastiaens, H., Corrigan, D., et al. Translational medicine and patient safety in Europe: TRANSFoRm–Architecture for the Learning health system in Europe. Biomed Res Int, 2015, 2015, 961526.
44. Zuidgeest, M.G., Goetz, I., Grobbee, D.E., Consortium WPotG. PRECIS-2 in perspective: what is next for pragmatic trials?. J Clin Epidemiol 84 (2017), 22–24.
45. Nieuwenhuis, J.B., Irving, E., Oude Rengerink, K., Lloyd, E., Goetz, I., Grobbee, D.E., et al. Pragmatic trial design elements showed a different impact on trial interpretation and feasibility than explanatory elements. J Clin Epidemiol 77 (2016), 95–100.