Evidence map›Paper›PMID 41022438›Full record

SynthesisBMJ open2025

Use of real-world data and real-world evidence in NICE (UK) health technology appraisals of new therapeutics in oncology: a systematic review.

Filipa Tunaru, Danielle E Robinson, Amy MacDougall, Lewis Carpenter

Abstract readSystematic Review
In one paragraph

Synthesis in BMJ open, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–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

1 citing paper in PubMed.

  1. Article
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

4 authors.

Filipa TunaruArcturis Data Limited, Oxford, England, UK.
Danielle E RobinsonArcturis Data Limited, Oxford, England, UK Danielle.Robinson@arcturisdata.com.
Amy MacDougallArcturis Data Limited, Oxford, England, UK.
Lewis CarpenterArcturis Data Limited, Oxford, England, UK.ORCID http://orcid.org/0000-0001-6889-7502

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivesTo quantify and describe the use of real-world data (RWD) in National Institute for Health and Care Excellence (NICE) oncology technology appraisal (TA) final appraisal determination documents.

designA systematic literature review was conducted on pharmaceutical NICE oncology TAs published between April 2000 and March 2024 (covering financial years 2000/2001 to 2023/2024 inclusive) extracted on 22 August 2023 (2000/2001 - 2022/2023) and 8 August 2024 (2023/2024). DATA SOURCES: NICE TA final appraisal determination documents. ELIGIBILITY CRITERIA: All pharmaceutical oncology TAs published between April 2000 and March 2024 (financial years 2000/2001 to 2023/2024) that did not go on to be terminated. DATA EXTRACTION AND SYNTHESIS: The data required for eligibility screening was extracted from an Excel file directly from the NICE website, where data related to each TA was extracted using an automated script derived from published sources. TAs were assessed based on prespecified review criteria covering whether an RWD submission was reported by the committee, and if so, which RWD sources were used, alongside the methods reported and any feedback from the committee regarding the use of RWD. Bias was not assessed as part of the study.

resultsOf 310 TAs identified, 135 (48.0%) used RWD. A variety of RWD types were used, mostly from UK or US data sources. 47 TAs (34.8%) leveraged RWD from multiple sources. RWD was mostly used in comparisons of survival (41.5%), to inform utility values (26.7%) and to compare baseline characteristics (19.3%), with matched adjusted indirect comparisons (MAICs) and external control arms (ECAs), seen from 2015 and 2018, respectively. The committee expressed concerns around the RWD presented by the company in 53 TAs (39.2%), the most common being a lack of generalisability to the UK population and/or National Health Service practice and comprehensiveness of the RWD.

conclusionsThis study quantifies the increasing use of diverse RWD sources in NICE oncology TAs, as well as the shift towards more complex methods like MAICs and ECAs. The feedback of the NICE committee highlights key areas of improvement as the generalisability and maturity of the RWD presented.

Indexed as

Antineoplastic AgentsMedical OncologyNeoplasmsTechnology Assessment, BiomedicalCost-Benefit AnalysisHumansUnited KingdomAntineoplastic AgentsElectronic Health RecordsHealth policyONCOLOGYProtocols & guidelinesReviewSystematic Review

Identifiers

PMID41022438
PMCPMC12481278

What OpenQuestion holds

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LicenceCC BY-NC
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Registered trials

None linked

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.