ArticleExpert opinion on orphan drugs2022
Drugs and biologics receiving FDA orphan drug designation: an analysis of the most frequently designated products and their repositioning strategies.
Article in Expert opinion on orphan drugs, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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
11 citing papers in PubMed, 18 citations in OpenAlex.
- Personalized drug discovery from rare diseases to cancer.npj drug discovery · 2026Review
- Post-Marketing Requirements for Anticancer Drugs Approved in Japan, 2001-2024: A Cross-Sectional Analysis.CPT: pharmacometrics & systems pharmacology · 2026Article
- The Regulatory Environment Surrounding Cannabis Medicines in the EU, the USA, and Australia.Pharmaceutics · 2025Article
- 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
- Cost of Exempting Sole Orphan Drugs From Medicare Negotiation.JAMA internal medicine · 2024Article
- Landscape Analysis of Generic Availability for Oncologic Drugs.Therapeutic innovation & regulatory science · 2023Article
- A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act.Orphanet journal of rare diseases · 2023Article
- Assessment of Surrogate End Point Trends in Clinical Trials to Approve Oncology Drugs From 2001 to 2020 in Japan.JAMA network open · 2023Article
- Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience.Human gene therapy · 2023Article
- Pharmaceutical policy and innovation for rare diseases: A narrative review.F1000Research · 2023Review
- Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021.JAMA network open · 2022Article
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 at 1 institution in 1 country.
Funding
Abstract
Background: The Orphan Drug Act was created to stimulate the development of drugs and biologics for rare diseases. Investigating products that have received orphan drug designation provide a greater understanding of rare disease drug development, as well as the repositioning business models of developers. Research design and methods: We used a dataset containing all orphan drug designations between 1983 and 2019. To analyze the orphan products, we constructed a variable, 'unique product,' that allowed for the standardization of generic names of drugs and biologics. Additional analysis was performed on the most frequently designated unique products and their repositioning strategies. Results: We found 5,099 orphan drug designations representing 3,269 unique products, of which 508 had an orphan-designated approval from FDA. Unique products with only a single designation represented 2,448 (75%) of the total products and 26 (1%) products had 10 or more designations. Over 60% of these unique products with 10 or more designations were antineoplastics or immunomodulators. Conclusions: The most designated unique products revealed a continuum of repositioning strategies, from the repurposing of approved drugs to parallel indication development programs for recently developed drugs. The fact that over 3,000 unique products have been studied for rare diseases indicates that future repositioning opportunities may become increasingly available.
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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.