ArticleNature reviews. Drug discovery2016
Worldwide collaboration for orphan drug designation.
Article in Nature reviews. Drug discovery, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
21 citing papers in PubMed.
- CAR-Based Cell and Gene Therapies: Global Clinical Landscape and Emerging Therapeutic Strategies from ClinicalTrials.gov.Biomolecules & therapeutics · 2025Review
- Insights into Support Systems for Orphan Drug Development: A Comparative Study.Therapeutic innovation & regulatory science · 2025Review
- Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Rare disease challenges and potential actions in the Middle East.International journal for equity in health · 2025Article
- Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation.Gene therapy · 2024Review
- Top Selling (2026) Small Molecule Orphan Drugs: A Journey into Their Chemistry.International journal of molecular sciences · 2023Review
- Cucurbitacin B-induced G2/M cell cycle arrest of conjunctival melanoma cells mediated by GRP78-FOXM1-KIF20A pathway.Acta pharmaceutica Sinica. B · 2022Article
- Drug Lag and Associated Factors for Approved Drugs in Korea Compared with the United States.International journal of environmental research and public health · 2022Article
- Opportunities and Challenges in Drug Development for Pediatric Cancers.Cancer discovery · 2021Review
- Drug Discovery and Development in Rare Diseases: Taking a Closer Look at the Tafamidis Story.Drug design, development and therapy · 2021Review
- Regulatory Standards in Orphan Medicinal Product Designation in the EU.Frontiers in medicine · 2021Review
- Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation.Frontiers in oncology · 2021Article
- Lithium's antiviral effects: a potential drug for CoViD-19 disease?International journal of bipolar disorders · 2020Review
- Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.Nature reviews. Drug discovery · 2020Review
- Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network.Orphanet journal of rare diseases · 2020Article
- Future of Rare Diseases Research 2017-2027: An IRDiRC Perspective.Clinical and translational science · 2018Review
- Glioblastoma Treatments: An Account of Recent Industrial Developments.Frontiers in pharmacology · 2018Review
- Progress in Rare Diseases Research 2010-2016: An IRDiRC Perspective.Clinical and translational science · 2018Review
- The International Rare Diseases Research Consortium: Policies and Guidelines to maximize impact.European journal of human genetics : EJHG · 2017Article
- Are products with an orphan designation for oncology indications different from products for other rare indications? A retrospective analysis of European orphan designations granted between 2002-2012.Orphanet journal of rare diseases · 2017Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
PubMed holds no abstract for this paper.
Indexed as
Identifiers
27245396What 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.