ArticlePLoS medicine2017
Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage.
Article in PLoS medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 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
18 citing papers in PubMed.
- 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
- Operational description of rare diseases: a reference to improve the recognition and visibility of rare diseases.Orphanet journal of rare diseases · 2024Article
- 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
- Defining rare conditions in the era of personalized medicine.Nature reviews. Drug discovery · 2023Article
- 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
- Pharmaceutical policy and innovation for rare diseases: A narrative review.F1000Research · 2023Review
- Molecular biomarkers, network biomarkers, and dynamic network biomarkers for diagnosis and prediction of rare diseases.Fundamental research · 2022Review
- Comparison of US and EU Prices for Orphan Drugs in the Perspective of the Considered US Orphan Drugs Act Modifications and Discussed Price-Regulation Mechanisms Adjustments in US and European Union.International journal of environmental research and public health · 2022Article
- Rare Diseases and Otorhinolaryngology, Head and Neck Surgery.Laryngo- rhino- otologie · 2021Article
- Assessment of Clinical Trials Supporting US Food and Drug Administration Approval of Novel Therapeutic Agents, 1995-2017.JAMA network open · 2020Article
- Precision Medicines' Impact on Orphan Drug Designation.Clinical and translational science · 2019Article
- Application of orphan drug designation to cancer treatments (2008-2017): a comprehensive and comparative analysis of the USA and EU.BMJ open · 2019Article
- Estimating the size of the United States market for new antibiotics with activity against carbapenem-resistant Enterobacteriaceae.Antimicrobial agents and chemotherapy · 2019Article
- The impact of the Orphan Drug Act on Food and Drug Administration-approved therapies for rare skin diseases and skin-related cancers.Journal of the American Academy of Dermatology · 2019Article
- [Orphan drugs from the perspective of the Drug Commission of the German Medical Association : Opportunities and challenges].Der Internist · 2019Review
- Biomarkers: Delivering on the expectation of molecularly driven, quantitative health.Experimental biology and medicine (Maywood, N.J.) · 2018Review
- What Is the Purpose of the Orphan Drug Act?PLoS medicine · 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
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Aaron Kesselheim and colleagues examine orphan-designated drugs approved between 2009 and 2015 in the United States.
Indexed as
Identifiers
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.