ArticleBMJ open2019
Application of orphan drug designation to cancer treatments (2008-2017): a comprehensive and comparative analysis of the USA and EU.
Article in BMJ open, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 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.
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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
21 citing papers in PubMed, 1 synthesis or guideline pooled it, 30 citations in OpenAlex.
- Efficacy of Immune Checkpoint Inhibitors in Rare Tumours: A Systematic Review.Frontiers in immunology · 2021Pooled it
- Orphan Designation for Drugs Approved in the United States and European Union: A Comparative Analysis.Clinical pharmacology and therapeutics · 2026Article
- Discordance in orphan drug approvals between the U.S. Food and Drug Administration and the European Medicines Agency: A retrospective observational analysis.PLoS medicine · 2026Observational
- Drug and country characteristics associated with global approval and launch of FDA-approved novel therapeutics.Health affairs scholar · 2026Article
- Targeted Therapies, Novel Antibodies, and Immunotherapies in Advanced Non-Small Cell Lung Cancer: Clinical Evidence and Drug Approval Patterns.Clinical cancer research : an official journal of the American Association for Cancer Research · 2024Review
- Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy.The European journal of health economics : HEPAC : health economics in prevention and care · 2024Review
- Article
- Continued cancer drug approvals in Japan and Europe after market withdrawal in the United States: A comparative study of accelerated approvals.Clinical and translational science · 2024Article
- FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications: cross sectional analysis.BMJ (Clinical research ed.) · 2023Article
- Rare molecular subtypes of lung cancer.Nature reviews. Clinical oncology · 2023Review
- Pharmaceutical policy and innovation for rare diseases: A narrative review.F1000Research · 2023Review
- Long way to go: Progress of orphan drug accessibility in China from 2017 to 2022.Frontiers in pharmacology · 2023Article
- Towards Better Pharmaceutical Provision in Europe-Who Decides the Future?Healthcare (Basel, Switzerland) · 2022Article
- Initial and supplementary indication approval of new targeted cancer drugs by the FDA, EMA, Health Canada, and TGA.Investigational new drugs · 2022Article
- The impact of the rare disease and Orphan Drug Act in Taiwan.Journal of food and drug analysis · 2021Article
- Article
- Removal of the EMA orphan designation upon request of the sponsor: cui prodest?European journal of clinical pharmacology · 2021Article
- Rare Diseases and Otorhinolaryngology, Head and Neck Surgery.Laryngo- rhino- otologie · 2021Article
- The Evaluation of Orphan Drugs by the German Joint Federal Committee-An Eight-Year Review.Deutsches Arzteblatt international · 2020Article
- Health technology assessment and reimbursement policy for oncology orphan drugs in Central and Eastern Europe.Orphanet journal of rare diseases · 2020Article
Corrections and comments
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Authors and funding
2 authors at 1 institution in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveTo determine differences in the characteristics of cancer drugs designated as orphan drugs by the Food and Drug Administration (FDA) and European Medicines Agency (EMA). DESIGN AND
settingIdentification of all cancer drugs (initial or supplementary indication) with orphan status approved by the FDA between 2008-2017 based on publicly accessible reports. The European public assessment reports (EPAR) was searched to determine whether these FDA-approved drugs were also approved by the EMA.
main outcome measuresExtraction of active ingredient, trade name, approval date and approved indication from two FDA data sources (Orphan Drug Product Designation Database, Drugs@FDA) and comparison with the same data from EPAR.
resultsThe FDA approved 135 cancer drugs with orphan indications that met our inclusion criteria, of which 101 (75%) were also approved by the EMA. 80/101 (79%) were first approved in the USA. Only 41/101 (41%) also received orphan designation by the EMA. 33/101 (33%) were approved for biomarker-based indications in the USA, however, only nine approved cancer drug indications by the EMA were biomarker-derived drugs. 78% (47/60) of approved cancer drugs that were only approved in the USA with orphan status were indicated for solid tumours, 22% (13/60) had indications for non-solid tumours. By contrast, out of those approved cancer drugs that received orphan designation by both agencies, 20% (8/41) were indicated for solid, and 80% (33/41) for non-solid tumours.
conclusionsOrphan designation was intended to encourage drug development for rare conditions. This study shows that the FDA approves more cancer drugs with such designations compared with the EMA, especially for subgroups of more prevalent cancers. One reason for the difference could be that the European Union requires demonstration of significant benefit for drugs that target the same indication as a drug already on the market to earn the orphan designation.
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