ArticlePLoS medicine2017
What Is the Purpose of the Orphan Drug Act?
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 34 papers, 1 of them a synthesis that pooled it.
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
34 citing papers in PubMed, 1 synthesis or guideline pooled it.
- A systematic review on the economic burden of interstitial lung disease and the cost-effectiveness of current therapies.BMC pulmonary medicine · 2022Pooled it
- Assessment of United States Food and Drug Administration Approval of Cell and Gene Therapies.Clinical pharmacology and therapeutics · 2026Article
- Article
- Systematic mapping of rare genetic disease studies using UK primary care electronic health records.European journal of human genetics : EJHG · 2026Review
- Barriers and Facilitators to the 3 Sides of Extended Reality-Rehabilitation Adoption: Scoping Review.Journal of medical Internet research · 2026Article
- How to evaluate rare disease policy effectiveness based on policy modeling consistency (PMC) index model: A quantitative assessment of policy implementation in China.Intractable & rare diseases research · 2026Article
- Building a National Policy for Rare Disease in Brazil.Journal of community genetics · 2025Review
- Rare Diseases, Spotlighting Amyotrophic Lateral Sclerosis, Huntington's Disease, and Myasthenia Gravis: Insights from Landscape Analysis of Current Research.Biochemistry · 2025Review
- Article
- Prevalence of Individuals With Multiple Diagnosed Genetic Diseases in the Undiagnosed Diseases Network.American journal of medical genetics. Part A · 2025Article
- The Metabolic Treatabolome and Inborn Errors of Metabolism Knowledgebase therapy tool: Do not miss the opportunity to treat!Journal of inherited metabolic disease · 2025Review
- The role of public health in rare diseases: hemophilia as an example.Frontiers in public health · 2025Review
- Application of observational research methods to real-world studies for rare disease drugs: A scoping review protocol.PloS one · 2025Article
- Review
- Valuing the Societal Impact of Medicines and Other Health Technologies: A User Guide to Current Best Practices.Forum for health economics & policy · 2024Article
- Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach.Molecular genetics and metabolism · 2024Review
- Analysis of affordability differences for rare diseases in China: a comparison across disease types and regions.International journal for equity in health · 2024Article
- A deep learning transformer model predicts high rates of undiagnosed rare disease in large electronic health systems.medRxiv : the preprint server for health sciences · 2023Article
- Phenotype-Driven Molecular Genetic Test Recommendation for Diagnosing Pediatric Rare Disorders.Research square · 2023Article
- Cross-national comparative study of orphan drug policies in Saudi Arabia, the United States, and the European Union.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Matthew Herder suggests it may be time to re-examine the purpose of the U.S. Orphan Drug Act.
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.