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Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies
BACKGROUND: Reimbursement decisions are conventionally based on evidence from randomised controlled trials (RCTs), which often have high internal validity but low external validity. Real-world data (RWD) may provide complimentary evidence for relative effectiveness assessments (REAs) and cost-effect...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer International Publishing
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5834594/ https://www.ncbi.nlm.nih.gov/pubmed/29214389 http://dx.doi.org/10.1007/s40273-017-0596-z |
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author | Makady, Amr van Veelen, Ard Jonsson, Páll Moseley, Owen D’Andon, Anne de Boer, Anthonius Hillege, Hans Klungel, Olaf Goettsch, Wim |
author_facet | Makady, Amr van Veelen, Ard Jonsson, Páll Moseley, Owen D’Andon, Anne de Boer, Anthonius Hillege, Hans Klungel, Olaf Goettsch, Wim |
author_sort | Makady, Amr |
collection | PubMed |
description | BACKGROUND: Reimbursement decisions are conventionally based on evidence from randomised controlled trials (RCTs), which often have high internal validity but low external validity. Real-world data (RWD) may provide complimentary evidence for relative effectiveness assessments (REAs) and cost-effectiveness assessments (CEAs). This study examines whether RWD is incorporated in health technology assessment (HTA) of melanoma drugs by European HTA agencies, as well as differences in RWD use between agencies and across time. METHODS: HTA reports published between 1 January 2011 and 31 December 2016 were retrieved from websites of agencies representing five jurisdictions: England [National Institute for Health and Care Excellence (NICE)], Scotland [Scottish Medicines Consortium (SMC)], France [Haute Autorité de santé (HAS)], Germany [Institute for Quality and Efficacy in Healthcare (IQWiG)] and The Netherlands [Zorginstituut Nederland (ZIN)]. A standardized data extraction form was used to extract information on RWD inclusion for both REAs and CEAs. RESULTS: Overall, 52 reports were retrieved, all of which contained REAs; CEAs were present in 25 of the reports. RWD was included in 28 of the 52 REAs (54%), mainly to estimate melanoma prevalence, and in 22 of the 25 (88%) CEAs, mainly to extrapolate long-term effectiveness and/or identify drug-related costs. Differences emerged between agencies regarding RWD use in REAs; the ZIN and IQWiG cited RWD for evidence on prevalence, whereas the NICE, SMC and HAS additionally cited RWD use for drug effectiveness. No visible trend for RWD use in REAs and CEAs over time was observed. CONCLUSION: In general, RWD inclusion was higher in CEAs than REAs, and was mostly used to estimate melanoma prevalence in REAs or to predict long-term effectiveness in CEAs. Differences emerged between agencies’ use of RWD; however, no visible trends for RWD use over time were observed. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s40273-017-0596-z) contains supplementary material, which is available to authorized users. |
format | Online Article Text |
id | pubmed-5834594 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2017 |
publisher | Springer International Publishing |
record_format | MEDLINE/PubMed |
spelling | pubmed-58345942018-03-09 Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies Makady, Amr van Veelen, Ard Jonsson, Páll Moseley, Owen D’Andon, Anne de Boer, Anthonius Hillege, Hans Klungel, Olaf Goettsch, Wim Pharmacoeconomics Original Research Article BACKGROUND: Reimbursement decisions are conventionally based on evidence from randomised controlled trials (RCTs), which often have high internal validity but low external validity. Real-world data (RWD) may provide complimentary evidence for relative effectiveness assessments (REAs) and cost-effectiveness assessments (CEAs). This study examines whether RWD is incorporated in health technology assessment (HTA) of melanoma drugs by European HTA agencies, as well as differences in RWD use between agencies and across time. METHODS: HTA reports published between 1 January 2011 and 31 December 2016 were retrieved from websites of agencies representing five jurisdictions: England [National Institute for Health and Care Excellence (NICE)], Scotland [Scottish Medicines Consortium (SMC)], France [Haute Autorité de santé (HAS)], Germany [Institute for Quality and Efficacy in Healthcare (IQWiG)] and The Netherlands [Zorginstituut Nederland (ZIN)]. A standardized data extraction form was used to extract information on RWD inclusion for both REAs and CEAs. RESULTS: Overall, 52 reports were retrieved, all of which contained REAs; CEAs were present in 25 of the reports. RWD was included in 28 of the 52 REAs (54%), mainly to estimate melanoma prevalence, and in 22 of the 25 (88%) CEAs, mainly to extrapolate long-term effectiveness and/or identify drug-related costs. Differences emerged between agencies regarding RWD use in REAs; the ZIN and IQWiG cited RWD for evidence on prevalence, whereas the NICE, SMC and HAS additionally cited RWD use for drug effectiveness. No visible trend for RWD use in REAs and CEAs over time was observed. CONCLUSION: In general, RWD inclusion was higher in CEAs than REAs, and was mostly used to estimate melanoma prevalence in REAs or to predict long-term effectiveness in CEAs. Differences emerged between agencies’ use of RWD; however, no visible trends for RWD use over time were observed. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s40273-017-0596-z) contains supplementary material, which is available to authorized users. Springer International Publishing 2017-12-06 2018 /pmc/articles/PMC5834594/ /pubmed/29214389 http://dx.doi.org/10.1007/s40273-017-0596-z Text en © The Author(s) 2017 Open AccessThis article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International License (http://creativecommons.org/licenses/by-nc/4.0/), which permits any noncommercial use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. |
spellingShingle | Original Research Article Makady, Amr van Veelen, Ard Jonsson, Páll Moseley, Owen D’Andon, Anne de Boer, Anthonius Hillege, Hans Klungel, Olaf Goettsch, Wim Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies |
title | Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies |
title_full | Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies |
title_fullStr | Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies |
title_full_unstemmed | Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies |
title_short | Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies |
title_sort | using real-world data in health technology assessment (hta) practice: a comparative study of five hta agencies |
topic | Original Research Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5834594/ https://www.ncbi.nlm.nih.gov/pubmed/29214389 http://dx.doi.org/10.1007/s40273-017-0596-z |
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