SynthesisOrphanet journal of rare diseases2013
Systematic review of available evidence on 11 high-priced inpatient orphan drugs.
Synthesis in Orphanet journal of rare diseases, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 3 of them syntheses that pooled it.
What it found
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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.
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.
Who cites it
15 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- Issues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review.PharmacoEconomics · 2024Pooled it
- Characteristics of clinical trials in rare vs. common diseases: A register-based Latvian study.PloS one · 2018Pooled it
- Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries.Orphanet journal of rare diseases · 2016Pooled it
- RD-LIVES: A Living Evidence Synthesis System for Rare Disease Treatment Efficacy and Safety.AMIA Joint Summits on Translational Science proceedings. AMIA Joint Summits on Translational Science · 2025Article
- The clinical assessment of studies on orphan drugs in relation to the EMA's authorization marketing decisions in Europe.Frontiers in pharmacology · 2025Article
- A Budget Impact Model of the Addition of Telotristat Ethyl Treatment to the Standard of Care in Patients with Uncontrolled Carcinoid Syndrome.PharmacoEconomics · 2020Article
- Patient access to orphan drugs in France.Orphanet journal of rare diseases · 2019Article
- Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties.Orphanet journal of rare diseases · 2018Article
- Using a meta-narrative literature review and focus groups with key stakeholders to identify perceived challenges and solutions for generating robust evidence on the effectiveness of treatments for rare diseases.Orphanet journal of rare diseases · 2018Review
- Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis.Orphanet journal of rare diseases · 2018Article
- Generic Substitution of Orphan Drugs for the Treatment of Rare Diseases: Exploring the Potential Challenges.Drugs · 2018Review
- Impact of orphan drugs on Latvian budget.Orphanet journal of rare diseases · 2016Article
- Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation.PharmacoEconomics · 2016Article
- Review
- Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries.Orphanet journal of rare diseases · 2013Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundAttention for Evidence Based Medicine (EBM) is growing, but evidence for orphan drugs is argued to be limited and inferior. This study systematically reviews the available evidence on clinical effectiveness, cost-effectiveness and budget impact for orphan drugs.
methodsA systematic review was performed in PubMed, Embase, NHS EED and HTA databases for 11 inpatient orphan drugs listed on the Dutch policy rule on orphan drugs. For included studies, we determined the type of study and various study characteristics.
resultsA total of 338 studies met all inclusion criteria. Almost all studies (96%) focused on clinical effectiveness of the drug. Of these studies, most studies were case studies (41%) or observational studies (39%). However, for all orphan diseases at least one experimental or quasi-experimental study was found, and a randomized clinical trial was available for 60% of the orphan drugs. Eight studies described the cost-effectiveness of an orphan drug; an equal number described an orphan drug's budget impact.
conclusionsDespite the often heard claim that RCTs are not feasible for orphan drugs, we found that an RCT was available in 60% of orphan drugs investigated. Cost-effectiveness and budget impact analyses for orphan drugs are seldom published.
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Registered trials
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.