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How to Deal with the Inevitable: Generating Real-World Data and Using Real-World Evidence for HTA Purposes – From Theory to Action

Part of: Special Collection - Policy Forums

Published online by Cambridge University Press:  14 June 2019

Wija Oortwijn ,
Laura Sampietro-Colom  and
Rebecca Trowman
Wija Oortwijn*
Affiliation:
Radboud University Medical Center, Department for Health Evidence, Nijmegen, The Netherlands
Laura Sampietro-Colom
Affiliation:
Hospital Clinic de Barcelona, Barcelona, Spain
Rebecca Trowman
Affiliation:
Health Technology Assessment international, Edmonton, Alberta, Canada
*
Author for correspondence: Wija Oortwijn, Email: w.oortwijn@radboudumc.nl
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Abstract

Objectives

Real-world evidence (RWE), derived from real-world data (RWD), is already used, to some extent, for health technology assessment (HTA) purposes. With the increased availability of RWD, there is potential for more widespread use but also challenges ensuring reliable RWE for HTA. Opportunities to overcome key challenges, identified at a scoping meeting, were discussed during the 2019 HTA international (HTAi) Global Policy Forum (GPF).

Methods

Reflection of discussions using Design Thinking (an interactive process aimed to solve complex problems) between seventy-three representatives from not-for-profit, for-profit organizations, and HTAi leadership. The discussions were informed by a background paper, and presentations from three invited keynote speakers and eleven GPF members.

Results

Several options were listed for addressing the identified key challenges: quality and acceptability, governance and accountability, transferability, and informing decision making. The GPF emphasized that the HTA community should first understand what questions could be answered with RWE. Additionally, more clarity on methods, standards, streamlining RWD collection, data sharing across jurisdictions, replication of RWD, and expert analysis were mentioned as important priorities.

Conclusions

The HTA community is currently standing at a cross-road as it is not yet fully equipped to address these key challenges. It is, therefore, time for action. The community should start aligning on what is the best source of evidence according to purpose and how the data should be collected to create reliable evidence. It should also initiate the development of actions and guidance to properly develop and manage RWD/RWE to inform decision making across the technology lifecycle.

Keywords

health technology assessmentreal-world datareal-world evidencechallengesopportunities
Type
Policy
Information
International Journal of Technology Assessment in Health Care , Volume 35 , Issue 4 , 2019 , pp. 346 - 350
Copyright
Copyright © Cambridge University Press 2019 

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Footnotes

We thank all Global Policy Forum Organizing Committee members (in alphabetical order) for steering our work: Barbara Calvert, Abbott; Joseph Cook, Pfizer Inc.; Tammy Clifford, Canadian Agency for Drugs and Technologies in Health (CADTH), Canada; Wim Goettsch, National Health Care Institute (ZIN), The Netherlands; Elizabeth Cobbs, Merck; Andrew Mitchell, Australian Government Department of Health, Australia; and Sean Tunis, Centre for Medical Technology Policy (CMTP), US. We also thank the HTAi Global Policy Forum attendees for sharing their thoughts and experiences during the HTAi Global Policy Forum 2019 (see Supplementary Table 1) and those GPF members who provided valuable comments and suggestions on an earlier version of this paper.

References

1.Garrison, L, Neuman, P, Erickson, P, et al. (2007) Using real-world data for coverage and payment decisions: the ISPOR Real-World Data Task Force Report. Value Health 10, 326–335.Google Scholar
2.HTA glossary (2019) http://htaglossary.net/real+world+data and http://htaglossary.net/real+world+evidence.Google Scholar
3.Husereau, D, Henshall, C, Jivraj, J (2014) Adaptive approaches to licensing, health technology assessment, and introduction of drugs and devices. Int J Technol Assess Health Care 30, 241–248.Google Scholar
4.Facey, K, Henshall, C, Sampietro-Colom, L, et al. (2015) Improving the Effectiveness and Efficiency of Evidence Production for Health Technology Assessment. Int J Technol Assess Health Care 31, 201–206.Google Scholar
5.Husereau, D, Henshall, C, Sampietro-Colom, L, et al. (2016) Changing Health Technology Assessment Paradigms? Int J Technol Assess Health Care 32, 191–199.Google Scholar
6.Mundy, L, Trowman, R, Kearney, B (2018) Universal healthcare in Asia: HTA and Real-world data overcoming barriers. DIA Newsletter, January. https://globalforum.diaglobal.org/issue/january-2018/universal-healthcare-in-asia-hta-and-real-world-data-overcoming-barriers/.Google Scholar
7.OECD (2019) Using routinely collected data to inform pharmaceutical policies. Analytical Report for OECD and EU Countries. OECD: Directorate for Employment, Labour and Social Affairs. Health Division, February. http://www.oecd.org/health/health-systems/Using-Routinely-Collected-Data-to-Inform-Pharmaceutical-Policies-Analytical-Report-2019.pdf.Google Scholar
8.Murphy, G, de Léséleuc, L, Kaunelis, D, et al. (2018) Use of real-world evidence in single-drug assessments. Ottawa: CADTH, April 2018. (Environmental scan; no. 74). https://www.cadth.ca/use-real-world-evidence-single-drug-technology-assessment-processes-health-technology-assessment-and.Google Scholar
9.U.S. Department of Health and Human Services, Food and Drug Administration (2017) Use of real-world evidence to support regulatory decision-making for medical devices. Guidance for industry and Food and Drug Administration staff. August 31. https://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm513027.pdf.Google Scholar
10.U.S. Food and Drug Administration (2018) Framework for FDA's Real-World Evidence Program. Silver Spring: FDA; December. https://www.fda.gov/downloads/ScienceResearch/SpecialTopics/RealWorldEvidence/UCM627769.pdf.Google Scholar
11.Plueschke, K, McGettigan, P, Pacurariu, A, et al. (2018) EU-funded initiatives for real world evidence: descriptive analysis of their characteristics and relevance for regulatory decision-making. BMJ Open 8, e021864. doi:10.1136/bmjopen-2018-021864.Google Scholar
12.Makady, A (2018) Real-world evidence for health technology assessment for pharmaceuticals: opportunities and challenges. PhD thesis. Amsterdam: Off Page, May. https://dspace.library.uu.nl/bitstream/handle/1874/364283/Makady.pdf?sequence=1&isAllowed=y.Google Scholar
13.Bowrin, K, Briere, J-P, Levy, P, et al. (2019) Cost-effectiveness analyses using real-world data: an overview of the literature. J Med Econ 28, 1–9.Google Scholar
14.The Academy of Medical Sciences (2018) Next steps for using real world evidence. Summary report of a FORUM follow-up roundtable held on 24 January 2018. https://acmedsci.ac.uk/file-download/7021031.Google Scholar
15.Oortwijn, W, on behalf of the HTAi Global Policy Forum (2018) Real-world evidence in the context of health technology assessment processes - from theory to action. Background Paper. December. https://htai.org/wp-content/uploads/2019/02/HTAiGlobalPolicyForum2019_BackgroundPaper.pdf.Google Scholar
16.Chatham House (2019) The Royal Institute of International Affairs. Chatham House Rule. https://www.chathamhouse.org/about/chatham-house-rule.Google Scholar
17.Interaction Design Foundation (2019) What is design thinking? https://www.interaction-design.org/literature/topics/design-thinking.Google Scholar
18.Karim, S, Booth, CM (2019) Effectiveness in the absence of efficacy: cautionary tales from real-world evidence. J Clin Oncol 37, 1047–1050.Google Scholar
19.Gerstein, HC, McMurray, J, Holman, RR (2019) Real-world studies no substitute for RCTs in establishing efficacy. Lancet 393, 210–211.Google Scholar
20.Berger, M, Sox, H, Wilke, R, et al. (2017) 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. Value Health 20, 1003–1008.Google Scholar
21.Cole, A, Garrison, L, Mestre-Ferrandiz, J, et al. (2015) Data Governance Arrangements for Real-World Evidence. London, UK: Office for Health Economics, 2015. https://www.ohe.org/publications/data-governance-arrangements-real-world-evidence#.Google Scholar
22.Chordoma Foundation (2018) Latest updates March 21. Chordoma Foundation Biobank Collaborates with Children's Hospital of Philadelphia [website]. https://www.chordomafoundation.org/latest-updates/biobank-partner-chop/.Google Scholar
23.Patient-Centered Outcomes Research Institute (PCORI) (2019) PCORnet: The National Patient-Centered Clinical Research Network [website]. https://www.pcori.org/research-results/pcornet-national-patient-centered-clinical-research-network.Google Scholar
24.Pearson, S, Dreitlein, B, Towse, A, et al. (2018) Understanding the context, selecting the standards: a framework to guide the optimal use and real world evidence for coverage decisions. ICER, OHE, March 2018. https://icer-review.org/material/rwe-white-paper-companion/.Google Scholar
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