HARmonized Protocol Template to Enhance Reproducibility of Hypothesis Evaluating Real-World Evidence Studies on Treatment Effects: A Good Practices Report of a Joint ISPE/ISPOR Task Force

被引:35
|
作者
Wang, Shirley, V [1 ]
Pottegard, Anton [2 ]
Crown, William [3 ]
Arlett, Peter [4 ]
Ashcroft, Darren M. [5 ]
Benchimol, Eric, I [6 ,7 ,8 ,9 ]
Berger, Marc L.
Crane, Gracy [10 ]
Goettsch, Wim [11 ,12 ]
Hua, Wei [13 ]
Kabadi, Shaum [14 ]
Kern, David M. [15 ]
Kurz, Xavier [4 ]
Langan, Sinead [16 ]
Nonaka, Takahiro [17 ]
Orsini, Lucinda [18 ]
Perez-Gutthann, Susana [19 ]
Pinheiro, Simone [13 ]
Pratt, Nicole [20 ]
Schneeweiss, Sebastian [1 ]
Toussi, Massoud [21 ]
Williams, Rebecca J.
机构
[1] Harvard Med Sch, Brigham & Womens Hosp, 1620 Tremt St,Suite 303, Boston, MA 02021 USA
[2] Univ Southern Denmark, Odense, Denmark
[3] Brandeis Univ, Waltham, MA USA
[4] European Med Agcy, London, England
[5] Univ Manchester, Sch Hlth Sci, Manchester, Lancs, England
[6] SickKids Res Inst, Child Hlth Evaluat Sci, Toronto, ON, Canada
[7] ICES, Toronto, ON, Canada
[8] Univ Toronto, Hosp Sick Children, Dept Paediat, Toronto, ON, Canada
[9] Univ Toronto, Hosp Sick Children, Inst Hlth Policy Management & Evaluat, Toronto, ON, Canada
[10] Roche Pharmaceut, Camberley, England
[11] Natl Hlth Care Inst, Diemen, Netherlands
[12] Univ Utrecht, Utrecht, Netherlands
[13] US FDA, Silver Spring, MD USA
[14] Sanofi Aventis US LLC, North Potomac, MD USA
[15] Janssen Res & Dev LLC, Philadelphia, PA USA
[16] London Sch Trop Hyg, London, England
[17] Pharmaceut & Med Devices Agcy, Tokyo, Japan
[18] COMPASS Pathways Ltd, Skillman, England
[19] RTI Hlth Solut, Barcelona, Spain
[20] Univ South Australia, Qual Use Med & Pharm Res Ctr, Clin & Hlth Sci, Adelaide, SA, Australia
[21] IQVIA, Courbevoie, France
基金
英国惠康基金; 澳大利亚国家健康与医学研究理事会;
关键词
protocol; real world evidence; reproducibility; transparency; SECONDARY DATA SOURCES; GLUCOSE COTRANSPORTER-2 INHIBITORS; PHARMACOEPIDEMIOLOGY; DATABASE; GUIDELINES; OUTCOMES; BIAS;
D O I
10.1016/j.jval.2022.09.001
中图分类号
F [经济];
学科分类号
02 ;
摘要
Objectives: Ambiguity in communication of key study parameters limits the utility of real-world evidence (RWE) studies in healthcare decision-making. Clear communication about data provenance, design, analysis, and implementation is needed. This would facilitate reproducibility, replication in independent data, and assessment of potential sources of bias. Methods: The International Society for Pharmacoepidemiology (ISPE) and ISPOR-The Professional Society for Health Economics and Outcomes Research (ISPOR) convened a joint task force, including representation from key international stakeholders, to create a harmonized protocol template for RWE studies that evaluate a treatment effect and are intended to inform decision-making. The template builds on existing efforts to improve transparency and incorporates recent insights regarding the level of detail needed to enable RWE study reproducibility. The over-arching principle was to reach for sufficient clarity regarding data, design, analysis, and implementation to achieve 3 main goals. One, to help investigators thoroughly consider, then document their choices and rationale for key study parameters that define the causal question (e.g., target estimand), two, to facilitate decision-making by enabling reviewers to readily assess potential for biases related to these choices, and three, to facilitate reproducibility. Strategies to Disseminate and Facilitate Use: Recognizing that the impact of this harmonized template relies on uptake, we have outlined a plan to introduce and pilot the template with key international stakeholders over the next 2 years. Conclusion: The HARmonized Protocol Template to Enhance Reproducibility (HARPER) helps to create a shared understanding of intended scientific decisions through a common text, tabular and visual structure. The template provides a set of core recommendations for clear and reproducible RWE study protocols and is intended to be used as a backbone throughout the research process from developing a valid study protocol, to registration, through implementation and reporting on those implementation decisions.
引用
收藏
页码:1663 / 1672
页数:10
相关论文
共 9 条
  • [1] HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real-world evidence studies on treatment effects: A good practices report of a joint ISPE/ISPOR task force
    Wang, Shirley, V
    Pottegard, Anton
    Crown, William
    Arlett, Peter
    Ashcroft, Darren M.
    Benchimol, Eric, I
    Berger, Marc L.
    Crane, Gracy
    Goettsch, Wim
    Hua, Wei
    Kabadi, Shaum
    Kern, David M.
    Kurz, Xavier
    Langan, Sinead
    Nonaka, Takahiro
    Orsini, Lucinda
    Perez-Gutthann, Susana
    Pinheiro, Simone
    Pratt, Nicole
    Schneeweiss, Sebastian
    Toussi, Massoud
    Williams, Rebecca J.
    PHARMACOEPIDEMIOLOGY AND DRUG SAFETY, 2023, 32 (01) : 44 - 55
  • [2] 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
    Berger, Marc L.
    Sox, Harold
    Willke, Richard J.
    Brixner, Diana L.
    Eichler, Hans-Georg
    Goettsch, Wim
    Madigan, David
    Makady, Amr
    Schneeweiss, Sebastian
    Tarricone, Rosanna
    Wang, Shirley V.
    Watkins, John
    Daniel Mullins, C.
    PHARMACOEPIDEMIOLOGY AND DRUG SAFETY, 2017, 26 (09) : 1033 - 1039
  • [3] 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
    Berger, Marc L.
    Sox, Harold
    Willke, Richard J.
    Brixner, Diana L.
    Eichler, Hans-Georg
    Goettsch, Wim
    Madigan, David
    Makady, Amr
    Schneeweiss, Sebastian
    Tarricone, Rosanna
    Wang, Shirley V.
    Watkins, John
    Mullins, C. Daniel
    VALUE IN HEALTH, 2017, 20 (08) : 1003 - 1008
  • [4] The joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making: an interview with Marc Berger
    Berger, Marc L.
    JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH, 2018, 7 (01) : 11 - 13
  • [5] Assessing Real-World Data From Electronic Health Records for Health Technology Assessment: The SUITABILITY Checklist: A Good Practices Report of an ISPOR Task Force
    Fleurence, Rachael L.
    Kent, Seamus
    Adamson, Blythe
    Tcheng, James
    Balicer, Ran
    Ross, Joseph S.
    Haynes, Kevin
    Muller, Patrick
    Campbell, Jon
    Bouee-Benhamiche, Elsa
    Marti, Sebastian Garcia
    Ramsey, Scott
    VALUE IN HEALTH, 2024, 27 (06) : 692 - 701
  • [6] Conducting Indirect-Treatment-Comparison and Network-Meta-Analysis Studies: Report of the ISPOR Task Force on Indirect Treatment Comparisons Good Research Practices: Part 2
    Hoaglin, David C.
    Hawkins, Neil
    Jansen, Jeroen P.
    Scott, David A.
    Itzler, Robbin
    Cappelleri, Joseph C.
    Boersma, Cornelis
    Thompson, David
    Larholt, Kay M.
    Diaz, Mireya
    Barrett, Annabel
    VALUE IN HEALTH, 2011, 14 (04) : 429 - 437
  • [7] 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, Marc L.
    Mamdani, Muhammad
    Atkins, David
    Johnson, Michael L.
    VALUE IN HEALTH, 2009, 12 (08) : 1044 - 1052
  • [8] 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, Michael L.
    Crown, William
    Martin, Bradley C.
    Dormuth, Colin R.
    Siebert, Uwe
    VALUE IN HEALTH, 2009, 12 (08) : 1062 - 1073
  • [9] Good Research Practices for Comparative Effectiveness Research: Approaches to Mitigate Bias and Confounding in the Design of Nonrandomized Studies of Treatment Effects Using Secondary Data Sources: The International Society for Pharmacoeconomics and Outcomes Research Good Research Practices for Retrospective Database Analysis Task Force Report-Part II
    Cox, Emily
    Martin, Bradley C.
    Van Staa, Tjeerd
    Garbe, Edeltraut
    Siebert, Uwe
    Johnson, Michael L.
    VALUE IN HEALTH, 2009, 12 (08) : 1053 - 1061