Evidence map›Paper›PMID 33860928›Full record

SynthesisPharmacoEconomics2021

Guidance for the Harmonisation and Improvement of Economic Evaluations of Personalised Medicine.

Heleen Vellekoop, Simone Huygens, Matthijs Versteegh, László Szilberhorn, Tamás Zelei, Balázs Nagy, Rositsa Koleva-Kolarova, Apostolos Tsiachristas, Sarah Wordsworth, Maureen Rutten-van Mölken and 1 more

Open access · hybridAbstract readSystematic Review
In one paragraph

Synthesis in PharmacoEconomics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers, 6 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
25citing papers in PubMed, 6 pooled it
9.2field-weighted citation impact, top 2% of its field
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

25 citing papers in PubMed, 6 syntheses or guidelines pooled it, 41 citations in OpenAlex.

  1. Pooled it
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  3. Systematic Literature Review of Economic Evaluations of Treatment Alternatives in Chronic Lymphocytic Leukemia.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2023
    Pooled it
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  15. Development of the PICCOTEAM Reference Case for Economic Evaluation of Precision Medicine.International journal of health policy and management · 2025
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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

11 authors at 4 institutions in 3 countries.

Heleen VellekoopInstitute for Medical Technology Assessment, Erasmus University Rotterdam, P.O. Box 1738, 3000 DR, Rotterdam, The Netherlands. vellekoop@imta.eur.nl.ORCID http://orcid.org/0000-0001-5127-240X
Simone HuygensInstitute for Medical Technology Assessment, Erasmus University Rotterdam, P.O. Box 1738, 3000 DR, Rotterdam, The Netherlands.ORCID http://orcid.org/0000-0003-0669-4149
Matthijs VersteeghInstitute for Medical Technology Assessment, Erasmus University Rotterdam, P.O. Box 1738, 3000 DR, Rotterdam, The Netherlands.ORCID http://orcid.org/0000-0003-4804-235X
László SzilberhornSyreon Research Institute, Budapest, Hungary.ORCID http://orcid.org/0000-0003-2556-397X
Tamás ZeleiSyreon Research Institute, Budapest, Hungary.ORCID http://orcid.org/0000-0003-1961-9607
Balázs NagySyreon Research Institute, Budapest, Hungary.ORCID http://orcid.org/0000-0003-4125-7905
Rositsa Koleva-KolarovaHealth Economics Research Centre, University of Oxford, Oxford, UK.ORCID http://orcid.org/0000-0002-8924-4671
Apostolos TsiachristasHealth Economics Research Centre, University of Oxford, Oxford, UK.ORCID http://orcid.org/0000-0002-4662-8915
Sarah WordsworthHealth Economics Research Centre, University of Oxford, Oxford, UK.ORCID http://orcid.org/0000-0002-2361-3040
Maureen Rutten-van MölkenInstitute for Medical Technology Assessment, Erasmus University Rotterdam, P.O. Box 1738, 3000 DR, Rotterdam, The Netherlands.ORCID http://orcid.org/0000-0001-8706-3159
HEcoPerMed Consortium
Erasmus University Rotterdam · NLSyreon Research Institute (Hungary) · HUUniversity of Oxford · GBErasmus MC · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveThe objective of this study was to develop guidance contributing to improved consistency and quality in economic evaluations of personalised medicine (PM), given current ambiguity about how to measure the value of PM as well as considerable variation in the methodology and reporting in economic evaluations of PM.

methodsA targeted literature review of methodological papers was performed for an overview of modelling challenges in PM. Expert interviews were held to discuss best modelling practice. A systematic literature review of economic evaluations of PM was conducted to gain insight into current modelling practice. The findings were synthesised and used to develop a set of draft recommendations. The draft recommendations were discussed at a stakeholder workshop and subsequently finalised.

resultsTwenty-two methodological papers were identified. Some argued that the challenges in modelling PM can be addressed within existing methodological frameworks, others disagreed. Eighteen experts were interviewed. They believed large uncertainty to be a key concern. Out of 195 economic evaluations of PM identified, 56% addressed none of the identified modelling challenges. A set of 23 recommendations was developed. Eight recommendations focus on the modelling of test-treatment pathways. The use of non-randomised controlled trial data is discouraged but several recommendations are provided in case randomised controlled trial data are unavailable. The parameterisation of structural uncertainty is recommended. Other recommendations consider perspective and discounting; premature survival data; additional value elements; patient and clinician compliance; and managed entry agreements.

conclusionsThis study provides a comprehensive list of recommendations to modellers of PM and to evaluators and reviewers of PM models.

Indexed as

Cost-Benefit AnalysisPrecision MedicineGuidelines as TopicHumansUncertainty

Identifiers

PMID33860928
PMCPMC8200346
OpenAlexW3153403476

What Socratic holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

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 Socratic graph.