Evidence map›Paper›PMID 35396306›Full record

ArticleBMJ open2022

Incremental benefits of novel pharmaceuticals in the UK: a cross-sectional analysis of NICE technology appraisals from 2010 to 2020.

Tobias B Polak, David GJ Cucchi, Jonathan J Darrow, Matthijs M Versteegh

Abstract read
In one paragraph

Article in BMJ open, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
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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

4 authors.

Tobias B PolakErasmus School of Health Policy & Management, Erasmus University Rotterdam, , Rotterdam, The Netherlands t.polak@erasmusmc.nl.ORCID 0000-0002-2720-3479
David GJ CucchiDepartment of Haematology, Amsterdam UMC Locatie VUmc, Amsterdam, The Netherlands.ORCID 0000-0001-6706-5464
Jonathan J DarrowDepartment of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts, USA.
Matthijs M VersteeghInstitute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, The Netherlands.ORCID 0000-0003-4804-235X

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivesTo evaluate the incremental value of new drugs across disease areas receiving favourable coverage decisions by the UK's National Institute for Health and Care Excellence (NICE) over the past decade. DESIGN, SETTING, AND

participantsThis cross-sectional study assessed favourable appraisal decisions of drugs between 1 January 2010 and 31 December 2020. Estimates of incremental benefit were extracted from NICE's evidence review groups reports. PRIMARY OUTCOME MEASURE: Incremental benefit of novel drugs relative to the best alternative therapeutic option, expressed in quality-adjusted life-years (QALYs).

results184 appraisals of 129 drugs provided QALYs. The median incremental value was 0.27 QALY (IQR: 0.07-0.73). Benefits varied across drug-indication pairs (range: -0.49 to 5.22 QALY). The highest median benefits were found in haematology (0.70, IQR: 0.55-1.22) and oncology (0.46, IQR: 0.20-0.88), the lowest in ophthalmology (0.09, IQR: 0.04-0.22) and endocrinology (0.02, IQR: 0.01-0.06). Eight appraisals (4.3%) found contributions of more than two QALYs, but one in four (50/184) drug-indication pairs provided less than the equivalent of 1 month in perfect health compared to existing treatments.

conclusionsIn our review period, the median incremental value of novel drugs approved for use within the English National Health System, relative to the best alternative therapeutic option, was equivalent to 3-4 months of life in perfect health, but data were heterogeneous. Objective evaluations of therapeutic value helps patients and physicians to develop reasonable expectations of drugs and delivers insights into disease areas where medicinal therapeutic progress has had the most and least impact.

Indexed as

Biomedical TechnologyTechnology Assessment, BiomedicalCost-Benefit AnalysisCross-Sectional StudiesHumansPharmaceutical PreparationsQuality-Adjusted Life YearsUnited KingdomPharmaceutical Preparationsgeneral medicine (see internal medicine)health policytherapeutics

Identifiers

PMID35396306
PMCPMC8996039

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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.