ArticleOrphanet journal of rare diseases2019
Comparing access to orphan medicinal products in Europe.
Article in Orphanet journal of rare diseases, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 53 papers, 2 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.
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Who cites it
53 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Reimbursement decision-making system in Poland systematically compared to other countries.Frontiers in pharmacology · 2023Pooled it
- A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries.Frontiers in public health · 2019Pooled it
- Rebalancing innovation, affordability, and access for orphan drugs in the European Union.The Lancet regional health. Europe · 2026Review
- Access to orphan drugs in adults with inherited metabolic diseases in Switzerland: a single-center retrospective cohort study.Orphanet journal of rare diseases · 2026Article
- Controlled access to lumasiran in primary hyperoxaluria type 1: evaluation of a new access route for orphan drugs in the Netherlands.Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association · 2025Article
- Access to Orphan Medicinal Products in Bulgaria: An Analysis of the Positive Drug List and Individual Access Schemes.Healthcare (Basel, Switzerland) · 2025Article
- The PICO Puzzle: Can Public Data Predict EU HTA Expectations for All EU Countries?Journal of market access & health policy · 2025Article
- Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union.Therapeutic innovation & regulatory science · 2025Article
- Access to child-appropriate medicines: an exploratory survey of the use of paediatric use marketing authorisation products in the UK.European journal of pediatrics · 2025Article
- Views and opinions of the general public about the reimbursement of expensive medicines in the Netherlands.PloS one · 2025Article
- The clinical assessment of studies on orphan drugs in relation to the EMA's authorization marketing decisions in Europe.Frontiers in pharmacology · 2025Article
- EUROCOVER-CLL: Reimbursement and accessibility of new treatments in relapsed/refractory chronic lymphocytic leukemia.Frontiers in pharmacology · 2025Article
- Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals.Journal of inherited metabolic disease · 2025Article
- Health technology assessment of medicinal products in Greece: a 5-year (2018-2023) review of timelines and productivity.International journal of technology assessment in health care · 2024Article
- AOTMiT reimbursement recommendations compared to other HTA agencies.The European journal of health economics : HEPAC : health economics in prevention and care · 2024Article
- The impact of proposed price regulations on new patented medicine launches in Canada: a retrospective cohort study.CMAJ : Canadian Medical Association journal = journal de l'Association medicale canadienne · 2024Article
- Project SATURN- a real-world evidence data collaboration with existing European datasets in Osteogenesis Imperfecta to support future therapies.Orphanet journal of rare diseases · 2024Article
- A Review of Current Approaches to Evaluating and Reimbursing New Medicines in a Subset of OECD Countries.Applied health economics and health policy · 2024Review
- Rare diseases: still on the fringes of universal health coverage in Europe.The Lancet regional health. Europe · 2024Review
- Clinical aspects of reimbursement policies for orphan drugs in Central and Eastern European countries.Frontiers in pharmacology · 2024Article
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesThe primary objective of this study was to compare the availability and access of orphan medicinal products (OMPs) in the devolved nations in the United Kingdom (UK), France, Germany, Italy and Spain. Availability is defined as the possibility to prescribe OMPs. Access refers to their full or partial reimbursement by the public health service.
methodsData were collated on: marketing authorisations, Health Technology Assessment (HTA) decisions, commissioning, and reimbursement decisions, and respective dates of these events for all the OMPs centrally authorised. Indicators of availability of and access to OMPs were calculated in each country and compared.
resultsWe found that since the implementation of the OMPs Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorisation in the European Union. These OMPs are most widely accessible in Germany and France. In the other countries between 30 and 60% of OMPs are reimbursed. In particular in England, less than 50% of centrally authorised OMPs are routinely funded by the NHS, with one-third of these recommended by NICE. In Germany reimbursement is automatically granted to all medicines which receive a marketing authorisation, immediately after authorisation - but since 2011, there is an evaluation and potentially a pricing negotiation between companies and sickness funds (third party payers). In the other countries, the shortest time from authorisation to a reimbursement decision is observed in Italy and France where it takes 18.6 and 19.5 months respectively on average.
conclusionsMarketing authorisation granted to OMPs is only the first step, as medicines reach patients when reimbursement decisions are implemented by national health systems (this applies to non-OMPs too). We found that more than a half of centrally authorised OMPs were available in the five selected countries, but that access to patients was further restricted by different national reimbursement policies, especially in the UK, Italy and Spain.
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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.