Evidence map›Paper›PMID 38791830›Full record

ArticleInternational journal of environmental research and public health2024

Fair Funding Decisions: Consistency of the Time Horizons Used in the Calculation of Quality-Adjusted Life Years for Therapies for Very Rare Diseases by the National Institute for Health and Care Excellence in England.

Jasmin Barman-Aksözen, Nicole Hentschel, Mårten Pettersson, Eva Schupp, Francesca Granata, Cornelia Dechant, Mehmet Hakan Aksözen, Rocco Falchetto

Abstract read
In one paragraph

Article in International journal of environmental research and public health, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. 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

8 authors.

Jasmin Barman-AksözenInternational Porphyria Patient Network (IPPN), Hegarstrasse 3, 8032 Zurich, Switzerland.ORCID 0000-0001-7272-8824
Nicole HentschelIndependent Researcher, Hegarstrasse 3, 8032 Zurich, Switzerland.
Mårten PetterssonInternational Porphyria Patient Network (IPPN), Hegarstrasse 3, 8032 Zurich, Switzerland.ORCID 0000-0001-8091-5101
Eva SchuppInternational Porphyria Patient Network (IPPN), Hegarstrasse 3, 8032 Zurich, Switzerland.
Francesca GranataInternational Porphyria Patient Network (IPPN), Hegarstrasse 3, 8032 Zurich, Switzerland.ORCID 0000-0002-1115-3374
Cornelia DechantInternational Porphyria Patient Network (IPPN), Hegarstrasse 3, 8032 Zurich, Switzerland.ORCID 0000-0003-1442-3824
Mehmet Hakan AksözenInternational Porphyria Patient Network (IPPN), Hegarstrasse 3, 8032 Zurich, Switzerland.ORCID 0000-0002-9434-6023
Rocco FalchettoInternational Porphyria Patient Network (IPPN), Hegarstrasse 3, 8032 Zurich, Switzerland.ORCID 0000-0003-3524-2141

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The National Institute for Health and Care Excellence (NICE) in England uses quality-adjusted life years (QALYs) to assess the cost-effectiveness of treatments. A QALY is a measure that combines the size of the clinical benefit of a treatment with the time the patient benefits from it, i.e., the time horizon. We wanted to know how consistently QALY gains are calculated at NICE. Therefore, we have analysed information on the time horizons used for the QALY calculations of the concluded evaluations conducted under the Highly Specialised Technologies programme for treatments of very rare diseases at NICE. For treatments with final guidance published by December 2023 (

Indexed as

Cost-Benefit AnalysisQuality-Adjusted Life YearsRare DiseasesDecision MakingEnglandHumansTime Factorserythropoietic protoporphyriahealth economicsHighly Specialised Technologies programmeNational Institute for Health and Care Excellencequality-adjusted life yearsrare disease

Identifiers

PMID38791830
PMCPMC11121024

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.