ArticleBMJ open2017
Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs.
Article in BMJ open, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers, 1 of them a synthesis 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
12 citing papers in PubMed, 1 synthesis or guideline pooled it, 29 citations in OpenAlex.
- Drug repurposing: a systematic review on root causes, barriers and facilitators.BMC health services research · 2022Pooled it
- Debates over orphan drug pricing: a meta-narrative literature review.Orphanet journal of rare diseases · 2025Review
- Scientific and regulatory progress in advancing paediatric oncology drug development in the EU and in the US.Frontiers in medicine · 2025Article
- Successes and pitfalls in orphan drug development for sickle cell disease.Blood advances · 2024Article
- Innovative research methodologies in the EU regulatory framework: an analysis of EMA qualification procedures from a pediatric perspective.Frontiers in medicine · 2024Article
- How to START? Four pillars to optimally begin your orphan drug development.Orphanet journal of rare diseases · 2023Article
- Making Sure That Orphan Incentives Tip the Right Way in Europe.Healthcare (Basel, Switzerland) · 2022Article
- Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval.Journal of clinical pharmacology · 2020Article
- Application of orphan drug designation to cancer treatments (2008-2017): a comprehensive and comparative analysis of the USA and EU.BMJ open · 2019Article
- [Orphan drugs from the perspective of the Drug Commission of the German Medical Association : Opportunities and challenges].Der Internist · 2019Review
- Innovation for rare diseases and bioethical concerns: A thin thread between medical progress and suffering.World journal of clinical pediatrics · 2018Article
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesThe research and development process in the field of rare diseases is characterised by many well-known difficulties, and a large percentage of orphan medicinal products do not reach the marketing approval.This work aims at identifying orphan medicinal products that failed the developmental process and investigating reasons for and possible factors influencing failures.
designDrugs designated in Europe under Regulation (European Commission) 141/2000 in the period 2000-2012 were investigated in terms of the following failures: (1) marketing authorisation failures (refused or withdrawn) and (2) drugs abandoned by sponsors during development.Possible risk factors for failure were analysed using statistically validated methods.
resultsThis study points out that 437 out of 788 designations are still under development, while 219 failed the developmental process. Among the latter, 34 failed the marketing authorisation process and 185 were abandoned during the developmental process. In the first group of drugs (marketing authorisation failures), 50% reached phase II, 47% reached phase III and 3% reached phase I, while in the second group (abandoned drugs), the majority of orphan medicinal products apparently never started the development process, since no data on 48.1% of them were published and the 3.2% did not progress beyond the non-clinical stage.The reasons for failures of marketing authorisation were: efficacy/safety issues (26), insufficient data (12), quality issues (7), regulatory issues on trials (4) and commercial reasons (1). The main causes for abandoned drugs were efficacy/safety issues (reported in 54 cases), inactive companies (25.4%), change of company strategy (8.1%) and drug competition (10.8%). No information concerning reasons for failure was available for 23.2% of the analysed products.
conclusionsThis analysis shows that failures occurred in 27.8% of all designations granted in Europe, the main reasons being safety and efficacy issues. Moreover, the stage of development reached by drugs represents a specific risk factor for failures.
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What OpenQuestion holds
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.