Evidence map›Paper›PMID 28095876›Full record

ArticleOrphanet journal of rare diseases2017

Multi-criteria decision analysis (MCDA): testing a proposed MCDA framework for orphan drugs.

C Schey, P F M Krabbe, M J Postma, M P Connolly

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Orphanet journal of rare diseases, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed, 3 pooled it
29.1field-weighted citation impact, top 1% 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

24 citing papers in PubMed, 3 syntheses or guidelines pooled it, 63 citations in OpenAlex.

  1. Pooled it
  2. Multi-criteria decision analysis for health technology assessment: addressing methodological challenges to improve the state of the art.The European journal of health economics : HEPAC : health economics in prevention and care · 2019
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  17. Benefit-risk evaluation: the past, present and future.Therapeutic advances in drug safety · 2019
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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 at 2 institutions in 1 country.

C ScheyUnit of PharmacoEpidemiology & PharmacoEconomics, Department of Pharmacy, University of Groningen, Groningen, The Netherlands. carina@gmasoln.com.
P F M KrabbeDepartment of Epidemiology, (UMCG), University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
M J PostmaUnit of PharmacoEpidemiology & PharmacoEconomics, Department of Pharmacy, University of Groningen, Groningen, The Netherlands.
M P ConnollyUnit of PharmacoEpidemiology & PharmacoEconomics, Department of Pharmacy, University of Groningen, Groningen, The Netherlands.
University of Groningen · NLUniversity Medical Center Groningen · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundSince the introduction of the orphan drugs legislation in Europe, it has been suggested that the general method of assessing drugs for reimbursement is not necessarily suitable for orphan drugs. The National Institute for Health and Clinical Excellence indicated that several criteria other than cost and efficacy could be considered in reimbursement decisions for orphan drugs. This study sought to explore the multi-criteria decision analysis (MCDA) framework proposed by (Orphanet J Rare Dis 7:74, 2012) to a range of orphan drugs, with a view to comparing the aggregate scores to the average annual cost per patient for each product, and thus establishing the merit of MCDA as a tool for assessing the value of orphan drugs in relation to their pricings.

methodsAn MCDA framework was developed using the nine criteria proposed by (Orphanet J Rare Dis 7:74, 2012) for the evaluation of orphan drugs, using the suggested numerical scoring system on a scale of 1 to 3 for each criterion. Correlations between the average annual cost of the drugs and aggregate MCDA scores were tested and plotted graphically. Different weightings for each of the attributes were also tested. A further analysis was conducted to test the impact of including the drug cost as an attribute in the aggregate index scores.

resultsIn the drugs studied, the R

conclusionDespite several limitations of the proposed model, this quantitative study provided insight into using MCDA and its relationship to the average annual costs of the products.

Indexed as

Decision Support TechniquesOrphan Drug ProductionDrug DiscoveryHemoglobinuria, ParoxysmalHypertension, PulmonaryLennox Gastaut SyndromeMucopolysaccharidosis IIMyelodysplastic SyndromesLennox-Gastaut syndromeMCDAMucopolysaccharidosis IIMulti-criteria decision analysisMyelodysplastic syndromesOrphan drugsParoxysmal nocturnal haemoglobinuriaPulmonary arterial hypertensionReimbursement

Identifiers

PMID28095876
PMCPMC5240262
OpenAlexW2578652651

What OpenQuestion holds

Textmetadata
LicenceCC BY
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 OpenQuestion graph.