Designing "real-World" trials to meet the needs of health policy makers at marketing authorization

Journal of Clinical Epidemiology

Volumn 64, Issue 7, 2011, Pages 711-717

  Calvert, Melanie ^a   Wood, John ^b   Freemantle, Nick ^a  

^a UNIVERSITY OF BIRMINGHAM   (United Kingdom)

^b UNIVERSITY OF EAST ANGLIA   (United Kingdom)

Author keywords

Adherence; Policy; Real world; Reimbursement; Safety; Trial

Indexed keywords

ALTERNATIVE MEDICINE; CARDIAC RESYNCHRONIZATION THERAPY; CASE REPORT; CLINICAL ASSESSMENT; CLINICAL DECISION MAKING; CLINICAL EFFECTIVENESS; CLINICAL TRIAL (TOPIC); CORONARY CARE UNIT; COST EFFECTIVENESS ANALYSIS; EPIDEMIOLOGICAL DATA; HEALTH CARE COST; HEALTH CARE POLICY; HEALTH CARE UTILIZATION; HEALTH ECONOMICS; HEALTH INSURANCE; HEART FAILURE; HUMAN; INTENSIVE CARE; MARKETING; OUTCOME ASSESSMENT; PATIENT COMPLIANCE; PATIENT PREFERENCE; PHARMACEUTICAL CARE; PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; QUALITY OF LIFE; REIMBURSEMENT; REVIEW; SAFETY; SIMULATION;

EID: 79956203991     PISSN: 08954356     EISSN: 18785921     Source Type: Journal    
DOI: 10.1016/j.jclinepi.2010.12.010     Document Type: Note

References (39)

1. National Institute for Clinical Excellence. 2009. About NICE. Available at http://www.nice.org.uk/aboutnice/. Accessed February 17, 2009.
2. Australian Government Department of Health and Ageing. Pharmaceutical Benefits Advisory Committee. 2009. Available at http://www.health.gov.au/pbs. [accessed 08/02/2011].
3. Institute for Quality and Efficiency in Health Care. Available at http://www.iqwig.de/index.2.en.html. Accessed February 17, 2009.
4. S.R. Tunis, D.B. Stryer, and C.M. Clancy Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy JAMA 290 2003 1624 1632 (Pubitemid 37430387)
5. M. Flather, N. Delahunty, and J. Collinson Generalizing results of randomized trials to clinical practice: reliability and cautions Clin Trials 3 2006 508 512
6. D. Schwartz, and J. Lellouch Explanatory and pragmatic attitudes in therapeutical trials J Chronic Dis 20 1967 637
7. D. Rennie, and H.S. Luft Pharmacoeconomic analyses: making them transparent, making them credible JAMA 283 2000 2158 2160 (Pubitemid 30241314)
8. A. O'Sullivan, D. Thompson, and M. Drummond Collection of health-economic data alongside clinical trials: is there a future for piggyback evaluations? Value Health 8 2005 67 79 (Pubitemid 40293643)
9. N.R. Powe, and R.I. Griffiths The clinical-economic trial: promise, problems, and challenges Control Clin Trials 16 6 1995 377 394 (Pubitemid 26035807)
10. M.J. Calvert, N. Freemantle, G. Yao, J.G. Cleland, L. Billingham, and J.C. Daubert Cost-effectiveness of cardiac resynchronization therapy: results from the CARE-HF trial Eur Heart J 26 2005 2681 2688 (Pubitemid 41746691)
11. J.G.F. Cleland, J.C. Daubert, E. Erdmann, N. Freemantle, D. Gras, and L. Kappenberger The CARE-HF study (CArdiac REsynchronisation in Heart Failure study): rationale, design and end-points Eur J Heart Fail 3 2001 481 489 (Pubitemid 32776875)
12. FDA. Guidance for Industry. Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Available at http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/ Guidances/UCM193282.pdf. [accessed 08/02/2011].
13. International Society for Pharmacoeconomics and Outcomes Research Pharmacoeconomic guidelines around the world Available at http://www.ispor.org/ PEguidelines/index.asp Accessed February 17, 2009
14. J. Brazier, J. Roberts, and M. Deverill The estimation of a preference-based measure of health from the SF-36 J Health Econ 21 2002 271 292 (Pubitemid 34143147)
15. Kind P. The EuroQol Instrument: an index of health-related quality of life. Quality of life and pharmacoeconomics in clinical trials (2nd ed.) 1996:191-201.
16. National Institute for Clinical Excellence. Guidance for Manufacturers and Sponsors. 2001:1-30.
17. ®): concepts, measurement properties and applications Health Qual Life Outcomes 1 1 2003 54
18. X. Badia, M. Roset, M. Herdman, and P. Kind A comparison of United Kingdom and Spanish general population time trade-off values for EQ-5D health states Med Decis Making 21 2001 7 16 (Pubitemid 32245026)
19. K. Noyes, A.W. Dick, and R.G. Holloway The implications of using US-specific EQ-5D preference weights for cost-effectiveness evaluation Med Decis Making 27 2007 327 334 (Pubitemid 46801750)
20. B. O'Brien Economic evaluation of pharmaceuticals: Frankenstein's monster or vampire of trials? Med Care 34 1996 DS99 DS108
21. Preference Collaborative Review Group Patients' preferences within randomised trials: systematic review and patient level meta-analysis BMJ 337 2008 a1864
22. M. King, I. Nazareth, F. Lampe, P. Bower, M. Chandler, and M. Morou Impact of participant and physician intervention preferences on randomized trials: a systematic review JAMA 293 2005 1089 1099 (Pubitemid 40300283)
23. E.S. Petherick, S. O'Meara, K. Spilsbury, C.P. Iglesias, E.A. Nelson, and D.J. Torgerson Patient acceptability of larval therapy for leg ulcer treatment: a randomised survey to inform the sample size calculation of a randomised trial BMC Med Res Methodol 6 2006 43
24. S. Del Prato, L. Blonde, L. Martinez, B. Göke, V. Woo, and A. Millward The effect of the availability of inhaled insulin on glycaemic control in patients with type 2 diabetes failing on oral therapy: the evaluation of ExuberaR as a therapeutic option on insulin initiation and improvement in glycaemic control in clinical practice (EXPERIENCE) trial Diabet Med 25 2008 662 670 (Pubitemid 351782825)
25. N. Freemantle, T. Strack Real World Trialists Will availability of inhaled human insulin (Exubera(R)) improve management of type 2 diabetes? The design of the Real World trial Trials 7 1 2006 25
26. R.B. Haynes, E. Ackloo, N. Sahota, H.P. McDonald, and X. Yao Interventions for enhancing medication adherence Cochrane Database Syst Rev Issue 2 2008 CD000011
27. M.Z. Vitolins, C.S. Rand, S.R. Rapp, P.M. Ribisl, and M.A. Sevick Measuring adherence to behavioral and medical interventions Control Clin Trials 21 2000 S188 S194
28. D.M. Waterhouse, K.A. Calzone, C. Mele, and D.E. Brenner Adherence to oral tamoxifen: a comparison of patient self-report, pill counts, and microelectronic monitoring J Clin Oncol 11 1993 1189 1197 (Pubitemid 23163362)
29. C. Parker, Z. Chen, M. Price, R. Gross, J.P. Metlay, and J.D. Christie Adherence to warfarin assessed by electronic pill caps, clinician assessment, and patient reports: results from the IN-RANGE study J Gen Intern Med 22 2007 1254 1259 (Pubitemid 47247293)
30. J.A. Berlin, S.C. Glasser, and S.S. Ellenberg Adverse event detection in drug development: recommendations and obligations beyond phase 3 Am J Public Health 98 2008 1366 1371
31. N. Freemantle, and A. Irs Observational evidence for determining drug safety BMJ 336 2008 627 628 (Pubitemid 351481223)
32. B.M. Kuehn Institute of Medicine outlines priorities for comparative effectiveness research JAMA 302 2009 936 937
33. R. Baltussen, R. Leidl, and A. Ament Real World designs in economic evaluation: bridging the gap between clinical research and policy-making Pharmacoeconomics 16 5 1999 449 458 (Pubitemid 29530149)
34. G. Yao, N. Freemantle, M.J. Calvert, S. Bryan, J.C. Daubert, and J.G.F. Cleland The long-term cost-effectiveness of cardiac resynchronization therapy with or without an implantable cardioverter-defibrillator Eur Heart J 28 2007 42 51 (Pubitemid 46134956)
35. A.H. Miners, M. Garau, D. Fidan, and A.J. Fischer Comparing estimates of cost effectiveness submitted to the National Institute for Clinical Excellence (NICE) by different organisations: retrospective study BMJ 330 2005 65
36. M. Drummond, M. Barbieri, J. Cook, H.A. Glick, J. Lis, and F. Malik Transferability of economic evaluations across jurisdictions: report of an ISPOR Good Research Practices Task Force Value Health 12 2009 409 418
37. G.A. Akerlof The market for "lemons": quality uncertainty and the market mechanism Q J Econ 84 1970 488 500
38. D. Kendler, M. McClung, N. Freemantle, M. Lillestol, A. Moffett, and J. Borenstein Adherence, preference, and satisfaction of postmenopausal women taking denosumab or alendronate Osteoporos Int 2010 1 11
39. M. Zwarenstein, S. Treweek, J.J. Gagnier, D.G. Altman, S. Tunis, and B. Haynes Improving the reporting of pragmatic trials: an extension of the CONSORT statement BMJ 337 2008 a2390