Evidence map›Paper›PMID 29214389›Full record

ArticlePharmacoEconomics2018

Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies.

Amr Makady, Ard van Veelen, Páll Jonsson, Owen Moseley, Anne D'Andon, Anthonius de Boer, Hans Hillege, Olaf Klungel, Wim Goettsch

Abstract readComparative Study
In one paragraph

Article in PharmacoEconomics, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 50 papers, 5 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
50citing papers in PubMed, 5 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

50 citing papers in PubMed, 5 syntheses or guidelines pooled it.

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  3. Real-world data: a comprehensive literature review on the barriers, challenges, and opportunities associated with their inclusion in the health technology assessment process.Journal of pharmacy & pharmaceutical sciences : a publication of the Canadian Society for Pharmaceutical Sciences, Societe canadienne des sciences pharmaceutiques · 2024
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  4. Real-world efficacy of treatment with benralizumab, dupilumab, mepolizumab and reslizumab for severe asthma: A systematic review and meta-analysis.Clinical and experimental allergy : journal of the British Society for Allergy and Clinical Immunology · 2022
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Amr MakadyThe National Healthcare Institute (ZIN), Eekholt 4, 1112 XH, Diemen, The Netherlands. amakady@zinl.nl.ORCID 0000-0002-5260-1946
Ard van VeelenDivision of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Universiteitsweg 99, 3584 CE, Utrecht, The Netherlands.
Páll JonssonThe National Institute for Health and Care Excellence (NICE), Level 1A, City Tower, Piccadilly Plaza, Manchester, M1 4BT, UK.
Owen MoseleyThe Scottish Medicines Consortium (SMC), Healthcare Improvement Scotland (HIS), Delta House (8th floor), 50 West Nile Street, Glasgow, G1 2NP, Scotland, UK.
Anne D'AndonLa Haute Autorité de Santé (HAS), 5 Avenue du Stade de France, Saint-Denis La Plaine Cedex, 93218, Paris, France.
Anthonius de BoerDivision of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Universiteitsweg 99, 3584 CE, Utrecht, The Netherlands.
Hans HillegeDepartment of Epidemiology, University Medical Centre Groningen, Broerstraat 5, 9712 CP, Groningen, The Netherlands.
Olaf KlungelDivision of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Universiteitsweg 99, 3584 CE, Utrecht, The Netherlands.
Wim GoettschThe National Healthcare Institute (ZIN), Eekholt 4, 1112 XH, Diemen, The Netherlands.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundReimbursement 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.

methodsHTA 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.

resultsOverall, 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.

conclusionIn 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.

Indexed as

Data AnalysisCost-Benefit AnalysisEuropeHumansTechnology Assessment, Biomedical

Identifiers

PMID29214389
PMCPMC5834594

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.