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Volumn 26, Issue 9, 2017, Pages 1033-1039

Good practices for real-world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making

(13)  Berger, Marc L a   Sox, Harold b   Willke, Richard J c   Brixner, Diana L d   Eichler, Hans Georg e   Goettsch, Wim f   Madigan, David g   Makady, Amr f   Schneeweiss, Sebastian h   Tarricone, Rosanna i   Wang, Shirley V h   Watkins, John j   Daniel Mullins, C k  


Author keywords

comparative effectiveness; decision making; guidelines; pharmacoepidemiology; real world data; treatment effectiveness

Indexed keywords

ARTICLE; COMPARATIVE EFFECTIVENESS; CONSENSUS; DATA ANALYSIS; MEDICAL DECISION MAKING; PEER REVIEW; PRACTICE GUIDELINE; PRIORITY JOURNAL; ADVISORY COMMITTEE; COMPARATIVE STUDY; DECISION MAKING; HEALTH CARE DELIVERY; HEALTH CARE ORGANIZATION; HUMAN; INTERNATIONAL COOPERATION; PHARMACOECONOMICS; PHARMACOEPIDEMIOLOGY; PROCEDURES; PROSPECTIVE STUDY; RANDOMIZED CONTROLLED TRIAL (TOPIC); RETROSPECTIVE STUDY; STANDARDS; STATISTICS; TREATMENT OUTCOME;

EID: 85029457249     PISSN: 10538569     EISSN: 10991557     Source Type: Journal    
DOI: 10.1002/pds.4297     Document Type: Article
Times cited : (239)

References (52)
  • 1
    • 34748856744 scopus 로고    scopus 로고
    • Using real-world data for coverage and payment decisions: The ISPOR real-world data task force report
    • Garrison LP, Neumann PJ, Erickson P, Marshall D, Mullins CD. Using real-world data for coverage and payment decisions: The ISPOR real-world data task force report. Value Health. 2006;10(5):326-335.
    • (2006) Value Health , vol.10 , Issue.5 , pp. 326-335
    • Garrison, L.P.1    Neumann, P.J.2    Erickson, P.3    Marshall, D.4    Mullins, C.D.5
  • 2
    • 85019120991 scopus 로고    scopus 로고
    • What is real-world data? A review of definitions based on literature and stakeholder interviews
    • Makady A, de Boer A, Hillege H, Klungel O, Goettsch W. What is real-world data? A review of definitions based on literature and stakeholder interviews. Value Health. 2017;20(7):858-865.
    • (2017) Value Health , vol.20 , Issue.7 , pp. 858-865
    • Makady, A.1    de Boer, A.2    Hillege, H.3    Klungel, O.4    Goettsch, W.5
  • 3
    • 85010901246 scopus 로고    scopus 로고
    • GetReal Workpackage. Policies for use of real-world data in health technology assessment (HTA): A comparative study of six HTA agencies
    • Makady A, Ham RT, de Boer A, Hillege H, Klungel O, Goettsch W. GetReal Workpackage. Policies for use of real-world data in health technology assessment (HTA): A comparative study of six HTA agencies. Value Health. 2017;20(4):520-532.
    • (2017) Value Health , vol.20 , Issue.4 , pp. 520-532
    • Makady, A.1    Ham, R.T.2    de Boer, A.3    Hillege, H.4    Klungel, O.5    Goettsch, W.6
  • 4
    • 85002412626 scopus 로고    scopus 로고
    • Real-world evidence—what is it and what can it tell us?
    • Sherman RE, Anderson SA, Dal Pan GJ, et al. Real-world evidence—what is it and what can it tell us? N Engl J Med. 2016;375(23):2293-2297.
    • (2016) N Engl J Med , vol.375 , Issue.23 , pp. 2293-2297
    • Sherman, R.E.1    Anderson, S.A.2    Dal Pan, G.J.3
  • 5
    • 65449152879 scopus 로고    scopus 로고
    • Standardizing quality assessment of observational studies for decision making in health care
    • Brixner DI, Holtorf AP, Neumann PJ, Malone DC, Watkins JB. Standardizing quality assessment of observational studies for decision making in health care. J Manag Care Pharm. 2009;15(3):275-283. Available at:http://www.amcp.org/data/jmcp/275-283.pdf
    • (2009) J Manag Care Pharm , vol.15 , Issue.3 , pp. 275-283
    • Brixner, D.I.1    Holtorf, A.P.2    Neumann, P.J.3    Malone, D.C.4    Watkins, J.B.5
  • 7
    • 84907279562 scopus 로고    scopus 로고
    • Observational studies: Getting clear about transparency
    • Accessed December 10, 2015
    • The PLOS Medicine Editors. Observational studies: Getting clear about transparency. PLoS Med. 2014;11(8):e1001711. https://doi.org/10.1371/journal.pmed.1001711guidances/ucm243537.pdf. Accessed December 10, 2015
    • (2014) PLoS Med , vol.11 , Issue.8
  • 8
    • 85021854013 scopus 로고    scopus 로고
    • Using certification to promote uptake of real-world evidence by payers
    • Segal JB, Kallich JD, Oppenheim ER, et al. Using certification to promote uptake of real-world evidence by payers. J Manag Care Pharm. 2016;22(3):191-196.
    • (2016) J Manag Care Pharm , vol.22 , Issue.3 , pp. 191-196
    • Segal, J.B.1    Kallich, J.D.2    Oppenheim, E.R.3
  • 9
    • 85029422845 scopus 로고    scopus 로고
    • (accessed July 6, 2017)
    • International Committee of Medical Journal Editors. http://www.icmje.org/recommendations/browse/publishing-and-editorial-issues/clinical-trial-registration.html (accessed July 6, 2017)
    • International Committee of Medical Journal Editors
  • 10
    • 0005248386 scopus 로고    scopus 로고
    • (accessed July 6, 2017)
    • US Food and Drug Administration. https://clinicaltrials.gov/ct2/manage-recs/fdaaa (accessed July 6, 2017)
    • US Food and Drug Administration
  • 11
    • 0037357132 scopus 로고    scopus 로고
    • A checklist for retroactive database studies—report of the ISPOR task force on retrospective databases
    • Motheral B, Brooks J, Clark MA, et al. A checklist for retroactive database studies—report of the ISPOR task force on retrospective databases. Value Health. 2003;6(2):90-97.
    • (2003) Value Health , vol.6 , Issue.2 , pp. 90-97
    • Motheral, B.1    Brooks, J.2    Clark, M.A.3
  • 12
    • 70350776531 scopus 로고    scopus 로고
    • Good research practices for comparative effectiveness research: Defining, reporting and interpreting nonrandomized studies of treatment effects using secondary data sources: the ISPOR good research practices for retrospective database analysis task force report—part I
    • Berger ML, Mamdani M, Atkins D, Johnson ML. Good research practices for comparative effectiveness research: Defining, reporting and interpreting nonrandomized studies of treatment effects using secondary data sources: the ISPOR good research practices for retrospective database analysis task force report—part I. Value Health. 2009;12(8):1044-1052.
    • (2009) Value Health , vol.12 , Issue.8 , pp. 1044-1052
    • Berger, M.L.1    Mamdani, M.2    Atkins, D.3    Johnson, M.L.4
  • 13
    • 70350780206 scopus 로고    scopus 로고
    • Good research practices for comparative effectiveness research: Approaches to mitigate bias and confounding in the design of non-randomized studies of treatment effects using secondary data sources: The ISPOR good research practices for retrospective database analysis task force—part II
    • Cox E, Martin BC, Van Staa T, Garbe E, Siebert U, Johnson ML. Good research practices for comparative effectiveness research: Approaches to mitigate bias and confounding in the design of non-randomized studies of treatment effects using secondary data sources: The ISPOR good research practices for retrospective database analysis task force—part II. Value Health. 2009;12(8):1053-1061.
    • (2009) Value Health , vol.12 , Issue.8 , pp. 1053-1061
    • Cox, E.1    Martin, B.C.2    Van Staa, T.3    Garbe, E.4    Siebert, U.5    Johnson, M.L.6
  • 14
    • 70350776529 scopus 로고    scopus 로고
    • Good research practices for comparative effectiveness research: analytic methods to improve causal inference from nonrandomized studies of treatment effects using secondary data sources: The ISPOR good research practices for retrospective database analysis task force report—part III
    • Johnson ML, Crown W, Martin BC, Dartmouth CR, Siebert U, Normand SL. Good research practices for comparative effectiveness research: analytic methods to improve causal inference from nonrandomized studies of treatment effects using secondary data sources: The ISPOR good research practices for retrospective database analysis task force report—part III. Value Health. 2009;12:1062-1073.
    • (2009) Value Health , vol.12 , pp. 1062-1073
    • Johnson, M.L.1    Crown, W.2    Martin, B.C.3    Dartmouth, C.R.4    Siebert, U.5    Normand, S.L.6
  • 15
    • 84858645799 scopus 로고    scopus 로고
    • Prospective observational studies to assess comparative effectiveness: the ISPOR good research practices task force report
    • Berger ML, Dreyer N, Anderson F, Towse A, Sedrakyan A, Normand SL. Prospective observational studies to assess comparative effectiveness: the ISPOR good research practices task force report. Value Health. 2012;15:217-230.
    • (2012) Value Health , vol.15 , pp. 217-230
    • Berger, M.L.1    Dreyer, N.2    Anderson, F.3    Towse, A.4    Sedrakyan, A.5    Normand, S.L.6
  • 16
    • 84881669041 scopus 로고    scopus 로고
    • Performance-based risk-sharing arrangements—good practices for design, implementation, and evaluation: report of the ISPOR good practices for performance-based risk-sharing arrangements task force
    • Garrison LP, Towse A, Briggs A, et al. Performance-based risk-sharing arrangements—good practices for design, implementation, and evaluation: report of the ISPOR good practices for performance-based risk-sharing arrangements task force, Value Health. 2013;16:703–709.
    • (2013) Value Health , vol.16 , pp. 703-709
    • Garrison, L.P.1    Towse, A.2    Briggs, A.3
  • 17
    • 84896367143 scopus 로고    scopus 로고
    • A questionnaire to assess the relevance and credibility of observational studies to inform healthcare decision making: an ISPOR-AMCP-NPC good practice task force report
    • Berger M, Martin B, Husereau D, et al. A questionnaire to assess the relevance and credibility of observational studies to inform healthcare decision making: an ISPOR-AMCP-NPC good practice task force report. Value Health. 2014;17(2):143-156.
    • (2014) Value Health , vol.17 , Issue.2 , pp. 143-156
    • Berger, M.1    Martin, B.2    Husereau, D.3
  • 18
    • 84953837049 scopus 로고    scopus 로고
    • International Society for Pharmacoepidemiology. Guidelines for good pharmacoepidemiology practices (GPP)
    • Public Policy Committee. International Society for Pharmacoepidemiology. Guidelines for good pharmacoepidemiology practices (GPP). Pharmacoepidemiol Drug Saf. 2016;25:2-10.
    • (2016) Pharmacoepidemiol Drug Saf , vol.25 , pp. 2-10
  • 23
    • 85029457673 scopus 로고    scopus 로고
    • (EU PAS Register)., (Accessed July 13
    • European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP). The European Union electronic register of post-authorisation studies (EU PAS Register). http://www.encepp.eu/encepp_studies/indexRegister.shtml (Accessed July 13, 2017).
    • (2017) The European Union electronic register of post-authorisation studies
  • 24
    • 84982175156 scopus 로고    scopus 로고
    • Pragmatic clinical trials
    • Ford I, Norrie J. Pragmatic clinical trials. NEJM. 2016;375:454-463.
    • (2016) NEJM , vol.375 , pp. 454-463
    • Ford, I.1    Norrie, J.2
  • 25
    • 66249128241 scopus 로고    scopus 로고
    • Economic evaluation for devices: Same or different?
    • Drummond M, Griffin A, Tarricone R. Economic evaluation for devices: Same or different? Value Health. 2009;12(4):402-404.
    • (2009) Value Health , vol.12 , Issue.4 , pp. 402-404
    • Drummond, M.1    Griffin, A.2    Tarricone, R.3
  • 27
    • 84930645309 scopus 로고    scopus 로고
    • The REporting of studies Conducted using Observational Routinely-collected health Data (RECORD) statement
    • Benchimol EI, Smeeth L, Guttmann A, et al., RECORD Working Committee. The REporting of studies Conducted using Observational Routinely-collected health Data (RECORD) statement. PLoS Med. 2015. https://doi.org/10.1371/journal.pmed.1001885
    • (2015) PLoS Med
    • Benchimol, E.I.1    Smeeth, L.2    Guttmann, A.3
  • 28
    • 35649019787 scopus 로고    scopus 로고
    • Strengthening the Reporting of Observational Studies in Epidemiology (STROBE): Explanation and elaboration
    • Vandenbroucke JP, von Elm E, Altman DG, et al. Strengthening the Reporting of Observational Studies in Epidemiology (STROBE): Explanation and elaboration. PLoS Med. 2007;4(10):e297. https://doi.org/10.1371/journal.pmed.0040297
    • (2007) PLoS Med , vol.4 , Issue.10
    • Vandenbroucke, J.P.1    von Elm, E.2    Altman, D.G.3
  • 30
    • 85029442969 scopus 로고    scopus 로고
    • Assessment of medical devices: Challenges and solutions
    • Accessed
    • Tarricone R, Torbica A, Drummond M, Schreyögg J. Assessment of medical devices: Challenges and solutions. Health Econ. 2017;S1:1-152. Accessed 4/29/2017
    • (2017) Health Econ , vol.S1 , pp. 1-152
    • Tarricone, R.1    Torbica, A.2    Drummond, M.3    Schreyögg, J.4
  • 31
    • 85029432740 scopus 로고    scopus 로고
    • internal-validity-non-randomised-studies-nrs-interv
    • http://www.eunethta.eu/outputs/publication-ja2-methodological-guideline- internal-validity-non-randomised-studies-nrs-interv.
  • 32
    • 84927675287 scopus 로고    scopus 로고
    • From adaptive licensing to adaptive pathways: Delivering a flexible life-span approach to bring new drugs to patients
    • Mar;
    • Eichler HG, Baird LG, Barker R, et al. From adaptive licensing to adaptive pathways: Delivering a flexible life-span approach to bring new drugs to patients. Clin Pharmacol Ther. 2015 Mar;97(3):234-246. https://doi.org/10.1002/cpt.59
    • (2015) Clin Pharmacol Ther , vol.97 , Issue.3 , pp. 234-246
    • Eichler, H.G.1    Baird, L.G.2    Barker, R.3
  • 33
    • 85029433105 scopus 로고    scopus 로고
    • https://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm513027.pdf (Accessed 4/29/2017)
  • 35
    • 84901415559 scopus 로고    scopus 로고
    • Distinguishing between exploratory and confirmatory preclinical research will improve translation
    • May;
    • Kimmelman J, Mogil JS, Dirangl U. Distinguishing between exploratory and confirmatory preclinical research will improve translation. PLoS Biol. 2014 May;12(5):e1001863.
    • (2014) PLoS Biol , vol.12 , Issue.5
    • Kimmelman, J.1    Mogil, J.S.2    Dirangl, U.3
  • 36
    • 85029431318 scopus 로고    scopus 로고
    • https://www.congress.gov/bill/114th-congress/house-bill/34 (Accessed 4/29/2017)
  • 37
    • 85029428355 scopus 로고    scopus 로고
    • (Accessed March 21, 2017)
    • HSRPoj. https://wwwcf.nlm.nih.gov/hsr_project/home_proj.cfm#. (Accessed March 21, 2017).
  • 41
    • 84872075614 scopus 로고    scopus 로고
    • SPIRIT 2013 statement: defining standard protocol items for clinical trials
    • Chen AW, Tetzlaff JM, Altman DG, et al. SPIRIT 2013 statement: defining standard protocol items for clinical trials. Ann Intern Med. 2013;158(3):200-207.
    • (2013) Ann Intern Med , vol.158 , Issue.3 , pp. 200-207
    • Chen, A.W.1    Tetzlaff, J.M.2    Altman, D.G.3
  • 42
    • 84881589353 scopus 로고    scopus 로고
    • Evaluating the impact of database heterogeneity on observational studies
    • Madigan D, Ryan P, Schuemie M, et al. Evaluating the impact of database heterogeneity on observational studies. Am J Epidemiol. 2013. https://doi.org/10.1093/aje/kwt010
    • (2013) Am J Epidemiol
    • Madigan, D.1    Ryan, P.2    Schuemie, M.3
  • 43
    • 77952766373 scopus 로고    scopus 로고
    • Orthogonal predictions: Follow-up questions for suggestive data
    • Walker AM. Orthogonal predictions: Follow-up questions for suggestive data. Pharmacoepidemiol Drug Saf. 2010;19(5):529-532.
    • (2010) Pharmacoepidemiol Drug Saf , vol.19 , Issue.5 , pp. 529-532
    • Walker, A.M.1
  • 44
    • 85006791198 scopus 로고    scopus 로고
    • Emerging guidelines for patient engagement in research
    • Kirwan JR, de Wit M, Frank L, et al. Emerging guidelines for patient engagement in research. Value Health. 2017;20(3):481-486.
    • (2017) Value Health , vol.20 , Issue.3 , pp. 481-486
    • Kirwan, J.R.1    de Wit, M.2    Frank, L.3
  • 45
    • 84949526282 scopus 로고    scopus 로고
    • Stakeholder-driven comparative effectiveness research: an update from PCORI
    • Selby JV, Forsydth LP, Sox HC. Stakeholder-driven comparative effectiveness research: an update from PCORI. JAMA. 2015;314:2335-2336.
    • (2015) JAMA , vol.314 , pp. 2335-2336
    • Selby, J.V.1    Forsydth, L.P.2    Sox, H.C.3
  • 46
    • 85018868818 scopus 로고    scopus 로고
    • Patient, caregiver and clinician views on engagement in comparative effectiveness research
    • Feb 8., [Epub ahead of print] PubMed; 28173732
    • Forsythe LP, Frank LB, Workman TA, Hilliard T, Harwell D, Fayish L. Patient, caregiver and clinician views on engagement in comparative effectiveness research. J Comp Eff Res. 2017 Feb 8. https://doi.org/10.2217/cer-2016-0062. [Epub ahead of print] PubMed; 28173732
    • (2017) J Comp Eff Res
    • Forsythe, L.P.1    Frank, L.B.2    Workman, T.A.3    Hilliard, T.4    Harwell, D.5    Fayish, L.6
  • 47
    • 85018910797 scopus 로고    scopus 로고
    • Health researcher views on comparative effectiveness research and research engagement
    • Feb 8., [Epub ahead of print] PubMed PMID
    • Forsythe LP, Frank LB, Workman TA, Borsky A, Hilliard T, Harwell D, Fayish L. Health researcher views on comparative effectiveness research and research engagement. J Comp Eff Res. 2017 Feb 8. https://doi.org/10.2217/cer-2016-0063. [Epub ahead of print] PubMed PMID: 28173710.
    • (2017) J Comp Eff Res
    • Forsythe, L.P.1    Frank, L.B.2    Workman, T.A.3    Borsky, A.4    Hilliard, T.5    Harwell, D.6    Fayish, L.7
  • 48
    • 85018896794 scopus 로고    scopus 로고
    • Patient, caregiver and clinician use of comparative effectiveness research findings in care decisions: Results from a national study
    • Feb 8., [Epub ahead of print] PubMed PMID
    • Frank LB, Forsythe LP, Workman TA, Hilliard T, Lavelle M, Harwell D, Fayish L. Patient, caregiver and clinician use of comparative effectiveness research findings in care decisions: Results from a national study. J Comp Eff Res. 2017 Feb 8. https://doi.org/10.2217/cer-2016-0061. [Epub ahead of print] PubMed PMID: 28173724.
    • (2017) J Comp Eff Res
    • Frank, L.B.1    Forsythe, L.P.2    Workman, T.A.3    Hilliard, T.4    Lavelle, M.5    Harwell, D.6    Fayish, L.7
  • 49
    • 0001155795 scopus 로고
    • Observation and experiment
    • Hill AB. Observation and experiment. N Engl J Med. 1953;248:995-1001.
    • (1953) N Engl J Med , vol.248 , pp. 995-1001
    • Hill, A.B.1
  • 51
    • 33846563409 scopus 로고    scopus 로고
    • Why most published research findings are false
    • e124
    • Loannidis JP. Why most published research findings are false. PLoS Med. 2005;2(8): e124
    • (2005) PLoS Med , vol.2 , Issue.8
    • Loannidis, J.P.1
  • 52
    • 80555145867 scopus 로고    scopus 로고
    • False-positive psychology: Undisclosed flexibility in data collection and analysis allows presenting anything as significant
    • Simmons JP, Nelson LD, Simonsohn U. False-positive psychology: Undisclosed flexibility in data collection and analysis allows presenting anything as significant. Psychol Sci. 2011;22(11):1359-1366.
    • (2011) Psychol Sci , vol.22 , Issue.11 , pp. 1359-1366
    • Simmons, J.P.1    Nelson, L.D.2    Simonsohn, U.3


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