Evidence map›Paper›PMID 42440217›Full record

ArticlePharmacoEconomics2026

Optimisation or Restriction? Unpacking the Logic Behind NICE Decisions.

Nadine Henderson, Brittany Darrow, Phill O'Neill, Martina Garau

Abstract read
In one paragraph

Article in PharmacoEconomics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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0citing papers in PubMed
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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.

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3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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.

Nadine HendersonOffice of Health Economics, London, UK. nhenderson@ohe.org.ORCID http://orcid.org/0000-0002-0981-9376
Brittany DarrowOffice of Health Economics, London, UK.ORCID http://orcid.org/0009-0009-6232-7168
Phill O'NeillOffice of Health Economics, London, UK.ORCID http://orcid.org/0000-0002-9016-402X
Martina GarauOffice of Health Economics, London, UK.ORCID http://orcid.org/0000-0002-0762-4743

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThe National Institute for Health and Care Excellence (NICE) regularly issues optimised technology appraisal (TA) recommendations, whereby medicines are recommended for a subset of their licensed population. These decisions play a growing role in shaping patient access, yet the underlying drivers and implications of optimisation remain poorly understood.

methodsWe conducted a mixed‑methods analysis of all optimised NICE TAs published in 2023 and 2024. Fifty‑six appraisals met our inclusion criteria for qualitative assessment of committee documentation, while 30 provided sufficient data to estimate the degree of patient access using the established M‑score method. Three case studies were developed to illustrate how optimisation rationales translate into real‑world implications for patients.

resultsOf 131 positive decisions in 2023-2024, 50% were optimised. Qualitative thematic analysis identified four interacting drivers of optimisation: (1) match to clinical trial data; (2) match to NHS clinical practice; (3) match to previous NICE guidance; and (4) subgroup only cost effective. Quantitatively, the mean M‑score across optimised decisions was 31%, indicating that, on average, only one‑third of the licensed patient population receives access. Most optimised decisions (77%) granted access to fewer than half of eligible patients. The case studies illustrate how optimisation decisions translate into meaningful consequences for patients, often limiting access to effective treatments and determining the point at which they can receive care.

conclusionsOptimised decisions constitute a substantial proportion of NICE's positive recommendations and often result in significant restrictions relative to marketing authorisations. Understanding the multifactorial drivers of optimisation is essential for interpreting access and equity implications for patients.

Indexed as

Decision MakingHealth Services AccessibilityTechnology Assessment, BiomedicalCost-Benefit AnalysisHumansState MedicineUnited Kingdom

Identifiers

PMID42440217
PMCPMC13615048

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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.