ArticleBritish journal of clinical pharmacology2011
Access to orphan drugs despite poor quality of clinical evidence.
Article in British journal of clinical pharmacology, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers, 4 of them syntheses that pooled it.
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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.
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Who cites it
32 citing papers in PubMed, 4 syntheses or guidelines pooled it.
- A systematic review of moral reasons on orphan drug reimbursement.Orphanet journal of rare diseases · 2021Pooled 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
- Systematic review of available evidence on 11 high-priced inpatient orphan drugs.Orphanet journal of rare diseases · 2013Pooled it
- Evidence available and used by the Food and Drug Administration for the approval of orphan and nonorphan drugs.Health affairs scholar · 2025Article
- Navigating challenges and opportunities in orphan medicines: A spotlight commentary on rare diseases.British journal of clinical pharmacology · 2025Article
- Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries.Frontiers in pharmacology · 2024Article
- Clinical aspects of reimbursement policies for orphan drugs in Central and Eastern European countries.Frontiers in pharmacology · 2024Article
- Novel approach to decision making for orphan drugs.International journal of technology assessment in health care · 2023Article
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Application of Bayesian methods to accelerate rare disease drug development: scopes and hurdles.Orphanet journal of rare diseases · 2022Review
- Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study.Frontiers in pharmacology · 2022Article
- Article
- Impact of health technology assessment and managed entry schemes on reimbursement decisions of centrally authorised medicinal products in Belgium.European journal of clinical pharmacology · 2019Article
- [Orphan drugs from the perspective of the Drug Commission of the German Medical Association : Opportunities and challenges].Der Internist · 2019Review
- 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
- Characteristics of drugs for ultra-rare diseases versus drugs for other rare diseases in HTA submissions made to the CADTH CDR.Orphanet journal of rare diseases · 2018Review
- Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand.Orphanet journal of rare diseases · 2018Article
Corrections and comments
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Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
aimWe analysed the Belgian reimbursement decisions of orphan drugs as compared with those of innovative drugs for more common but equally severe diseases, with special emphasis on the quality of clinical evidence.
methodsUsing the National Health Insurance Agency administrative database, we evaluated all submitted orphan drug files between 2002 and 2007. A quality analysis of the clinical evidence in the orphan reimbursement files was performed. The evaluation reports of the French 'Haute Autorité de Santé', including the five-point scale parameter 'Service Médical Rendu (SMR), were examined to compare disease severity. Chi-squared tests (at P < 0.05 significance level) were used to compare the outcome of the reimbursement decisions between orphan and non-orphan innovative medicines.
resultsTwenty-five files of orphan drugs and 117 files of non-orphan drugs were evaluated. Twenty-two of 25 (88%) submissions of orphan drugs were granted reimbursement as opposed to 74 of the 117 (63%) non-orphan innovative medicines (P= 0.02). Only 52% of the 25 orphan drug files included a randomized controlled trial as opposed to 84% in a random control sample of 25 non-orphan innovative submissions (P < 0.01). The duration of drug exposure was in most cases far too short in relation to the natural history of the disease.
conclusionsOrphan drug designation predicts reimbursement despite poor quality of clinical evidence. The evidence gap at market authorization should be reduced by post-marketing programmes, in which the centralized regulatory and the local reimbursement authorities collaborate in an efficient way across the European Union member states.
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