Evaluating the Generalisability of Trial Results: Introducing a Centre- and Trial-Level Generalisability Index

PharmacoEconomics

Volumn 33, Issue 11, 2015, Pages 1195-1214

  Gheorghe, Adrian ^a,b   Roberts, Tracy ^a   Hemming, Karla ^a   Calvert, Melanie ^a  

^a UNIVERSITY OF BIRMINGHAM   (United Kingdom)

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

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; CLINICAL EFFECTIVENESS; CLINICAL PRACTICE; COST EFFECTIVENESS ANALYSIS; EXTERNAL VALIDITY; GENERALIZABILITY INDEX; HEALTH CARE COST; HUMAN; PRIORITY JOURNAL; PROBABILITY; QUALITY ADJUSTED LIFE YEAR; RANDOMIZED CONTROLLED TRIAL (TOPIC); SENSITIVITY ANALYSIS; SIMULATION; STATISTICAL ANALYSIS; COST BENEFIT ANALYSIS; ECONOMICS; EPIDEMIOLOGY; HEALTH CARE FACILITY; PHASE 3 CLINICAL TRIAL (TOPIC); PROCEDURES; STANDARDS; THEORETICAL MODEL; TREATMENT OUTCOME;

CLINICAL TRIALS, PHASE III AS TOPIC; COST-BENEFIT ANALYSIS; HEALTH FACILITIES; HUMANS; MODELS, THEORETICAL; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SAMPLING STUDIES; TREATMENT OUTCOME;

EID: 84944513497     PISSN: 11707690     EISSN: 11792027     Source Type: Journal    
DOI: 10.1007/s40273-015-0298-3     Document Type: Article

References (47)

1. Black N. Why we need observational studies to evaluate the effectiveness of health care. BMJ. 1996;312(7040):1215–8.
2. Rothwell PM. External validity of randomised controlled trials: “to whom do the results of this trial apply?”. Lancet. 2005;365(9453):82–93.
3. Silverman SL. From randomized controlled trials to observational studies. Am J Med. 2009;122(2):114–20.
4. Kravitz RL, Duan N, Braslow J. Evidence-based medicine, heterogeneity of treatment effects, and the trouble with averages. Milbank Q. 2004;82(4):661–87.
5. Kraemer H, Frank E, Kupfer D. Moderators of treatment outcomes: clinical, research, and policy importance. JAMA. 2006;296(10):1286–9.
6. O’Brien BJ. A tale of two (or more) cities: geographic transferability of pharmacoeconomic data. Am J Manage Care. 1997;3:S33–9.
7. Drummond MF, et al. Issues in the cross-national assessment of health technology. Int J Technol Assess Health Care. 1992;8(4):670–82.
8. Moore DA, et al. How generalizable are the results of large randomized controlled trials of antiretroviral therapy? HIV Med. 2000;1(3):149–54.
9. Hoel AW, et al. Under-representation of women and ethnic minorities in vascular surgery randomized controlled trials. J Vasc Surg. 2009;50(2):349–54.
10. Kalata P, et al. Differences between clinical trial participants and patients in a population-based registry: the German Rectal Cancer Study vs. the Rostock Cancer Registry. Dis Colon Rectum. 2009;52(3):425–37.
11. Maasland L, et al. Patients enrolled in large randomized clinical trials of antiplatelet treatment for prevention after transient ischemic attack or ischemic stroke are not representative of patients in clinical practice: the Netherlands Stroke Survey. Stroke. 2009;40(8):2662–8.
12. Costa DJ, et al. How representative are clinical study patients with allergic rhinitis in primary care? J Allergy Clin Immunol. 2011;127(4):920–6.
13. Falagas ME, et al. Patients included in randomised controlled trials do not represent those seen in clinical practice: focus on antimicrobial agents. Int J Antimicrob Agents. 2010;36(1):1–13.
14. Bartlett C, et al. The causes and effects of socio-demographic exclusions from clinical trials. Health Technol Assess. 2005;9(38):1–152.
15. Halm EA, Lee C, Chassin MR. Is volume related to outcome in health care? a systematic review and methodologic critique of the literature. Ann Intern Med. 2002;137(6):511–20.
16. Devereaux PJ, et al. Need for expertise based randomised controlled trials. BMJ. 2005;330(7482):88.
17. Gheorghe A, et al. Centre selection for clinical trials and the generalisability of results: a mixed methods study. PLoS One. 2013;8(2):e56560.
18. Gheorghe A, et al. Rational centre selection for RCTs with a parallel economic evaluation: the next step towards increased generalisability? Health Economics. 2014;24(4):498–504.
19. Sculpher MJ, et al. Generalisability in economic evaluation studies in healthcare: a review and case studies. Health Technol Assess. 2004;8(49):iii–iv, 1–192.
20. Goeree R, et al. Transferability of economic evaluations: approaches and factors to consider when using results from one geographic area for another. Curr Med Res Opin. 2007;23(4):671–82.
21. Wei JW, et al. Variations and determinants of hospital costs for acute stroke in China. PLoS One. 2010;5(9):e13041.
22. Saleh SS, Racz M, Hannan E. The effect of preoperative and hospital characteristics on costs for coronary artery bypass graft. Ann Surg. 2009;249(2):335–41.
23. Becker ER. National trends and determinants of hospitalization costs and lengths-of-stay for uterine fibroids procedures. J Health Care Finance. 2007;33(3):1–16.
24. Lee KH, Yang SB, Choi M. The association between hospital ownership and technical efficiency in a managed care environment. J Med Syst. 2009;33(4):307–15.
25. Chaikledkaew U, et al. Factors affecting health-care costs and hospitalizations among diabetic patients in Thai public hospitals. Value Health. 2008;11(Suppl 1):S69–74.
26. McCollam PL, Lage MJ, Bala M. A comparison of total hospital costs for percutaneous coronary intervention patients receiving abciximab versus tirofiban. Catheter Cardiovasc Interv. 2001;54(2):152–7.
27. Stock GN, McDermott C. Operational and contextual drivers of hospital costs. J Health Organ Manage. 2011;25(2):142–58.
28. Kristensen T, et al. Cost variation in diabetes care delivered in English hospitals. Diabet Med. 2010;27(8):949–57.
29. Laudicella M, Olsen KR, Street A. Examining cost variation across hospital departments: a two-stage multi-level approach using patient-level data. Soc Sci Med. 2010;71(10):1872–81.
30. Daidone S, D’Amico F. Technical efficiency, specialization and ownership form: evidences from a pooling of Italian hospitals. J Product Anal. 2009;32(3):203–16.
31. Gutacker N, et al. Truly inefficient or providing better quality of care? Analysing the relationship between risk-adjusted hospital costs and patients’ health outcomes. Health Econ. 2013;22(8):931–47.
32. Department of Health. PbR and the market forces factor (MFF) in 2013-14. 2013. Available at: https://www.gov.uk/government/uploads/system/uploads/attachment_data/file/141395/PbR-and-the-MFF-in-2013-14.pdf. Accessed 11 Jul 2013.
33. Department of Health. NHS reference costs 2011–2012. 2012. Available at: https://www.gov.uk/government/uploads/system/uploads/attachment_data/file/127112/2011-12-reference-costs-publication.pdf. Accessed 11 Jul 2013.
34. Pinkney TD, et al. Impact of wound edge protection devices on surgical site infection after laparotomy: multicentre randomised controlled trial (ROSSINI Trial). BMJ. 2013;347:f4305.
35. Efron B, Tibshirani RJ. An introduction to the bootstrap. London: Chapman and Hall; 1993.
36. National Institute for Health and Care Excellence. Guide to the methods of technology appraisal 2013. London: National Institute for Health and Care Excellence; 2013.
37. Efron B. Better bootstrap confidence intervals. J Am Stat Assoc. 1987;82(397):171–85.
38. Briggs A, Wonderling DE, Mooney CZ. Pulling cost-effectiveness analysis up by its bootstraps: a non-parametric approach to confidence interval estimation. Health Econ. 1997;6(4):327–40.
39. R Development Core Team. R: a language and environment for statistical computing. Vienna: R Foundation for Statistical Computing; 2013.
40. Gillam SJ, Siriwardena AN, Steel N. Pay-for-performance in the United Kingdom: impact of the quality and outcomes framework: a systematic review. Ann Fam Med. 2012;10(5):461–8.
41. Majumdar SR, et al. Better outcomes for patients treated at hospitals that participate in clinical trials. Arch Intern Med. 2008;168(6):657–62.
42. Rochon J, du Bois A, Lange T. Mediation analysis of the relationship between institutional research activity and patient survival. BMC Med Res Methodol. 2014;14(1):9.
43. Burton A, et al. The design of simulation studies in medical statistics. Stat Med. 2006;25(24):4279–92.
44. Deeks JJ, et al. Evaluating non-randomised intervention studies. Health Technol Assess. 2003;7(27):iii–x, 1–173.
45. Gomes M, et al. Methods for covariate adjustment in cost-effectiveness analysis that use cluster randomised trials. Health Econ. 2012;21(9):1101–18.
46. McCarron CE, et al. Bayesian hierarchical models combining different study types and adjusting for covariate imbalances: a simulation study to assess model performance. PLoS One. 2011;6(10):e25635.
47. Health & Social Care Information Centre. Hospital episode statistics, admitted patient care–England 2011–12. (2013). http://www.hscic.gov.uk/searchcatalogue?q=title%3A%22Hospital+Episode+Statistics%2C+Admitted+patient+care+-+England%22&area=&size=10&sort=Relevance. Accessed 29 May 2013.