Evidence map›Paper›PMID 25304026›Full record

ArticleOrphanet journal of rare diseases2014

Orphan drugs expenditure in the Netherlands in the period 2006-2012.

Tim A Kanters, Adri Steenhoek, Leona Hakkaart

Abstract readHistorical Article
In one paragraph

Article in Orphanet journal of rare diseases, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed, 2 pooled it
–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

10 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Pooled it
  2. Pooled it
  3. Review
  4. Article
  5. Public spending on orphan medicines: a review of the literature.Journal of pharmaceutical policy and practice · 2020
    Article
  6. Article
  7. Review
  8. Negotiating prices of drugs for rare diseases.Bulletin of the World Health Organization · 2016
    Article
  9. Article
  10. Impact of orphan drugs on Latvian budget.Orphanet journal of rare diseases · 2016
    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

3 authors.

Tim A KantersInstitute for Medical Technology Assessment, Department of Health Policy & Management, Erasmus University Rotterdam, Burgemeester Oudlaan 50, Rotterdam, 3000DR, The Netherlands. kanters@bmg.eur.nl.
Adri Steenhoek
Leona Hakkaart

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThe relatively low budget impact of orphan drugs is often used as an argument in reimbursement decisions. However, overall, the budget impact of orphan drugs can still be substantial. In this study, we assess the uptake and budget impact of orphan drugs in the Netherlands.

methodsWe examined the number of orphan drugs, the number of patients and budget impact of orphan drugs in the Netherlands in the period 2006 to 2012, both for inpatient and outpatient orphan drugs. Budget impact was provided in absolute numbers and relative to total pharmaceutical spending.

resultsThe number of orphan drugs and patients treated increased substantially over the period studied. Overall, budget impact increased substantially over a period of six years, both in absolute terms (326% increase) as well as relative to total pharmaceutical spending (278% increase). Growth rates decreased over time. In 2012, 17% of available drugs had an individual budget impact of more than €10 million per year.

conclusionsIndividual budget impact of orphan drugs is often limited, although exceptions exist. However, in total, the budget impact of orphan drugs is considerable and has grown substantially over the years. This could potentially influence reimbursement decisions for orphan drugs in the future.

Indexed as

Drug CostsBudgetsHistory, 21st CenturyHumansNetherlandsOrphan Drug Production

Identifiers

PMID25304026
PMCPMC4195995

What OpenQuestion holds

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LicenceCC BY
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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.