ArticleHealthcare (Basel, Switzerland)2023
Disentangling the Cost of Orphan Drugs Marketed in the United States.
Article in Healthcare (Basel, Switzerland), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 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.
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Who cites it
15 citing papers in PubMed, 30 citations in OpenAlex.
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- Plazomicin: A promising novel antimicrobial in the era of bacterial resistance (Review).Experimental and therapeutic medicine · 2026Review
- The Economics of Drug Development: A Comparison between Orphan and Non-orphan Drugs.Current drug discovery technologies · 2026Article
- Advances in Cell and Gene Therapy for Rare Disease Treatment.International journal of stem cells · 2025Review
- Predictive Analysis for First Submission of Generic Drug Application for Orphan Drug Products Using Random Survival Forest.Clinical and translational science · 2025Article
- Artificial intelligence applications in rare and intractable diseases: Advances, challenges, and future directions.Intractable & rare diseases research · 2025Article
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- A semiautomated microfluidic ELISA for the detection of hemophagocytic lymphohistiocytosis biomarkers.American journal of clinical pathology · 2025Article
- Results of health technology assessments of orphan drugs in Germany-lack of added benefit, evidence gaps, and persisting unmet medical needs.International journal of technology assessment in health care · 2024Article
- Use of Basket Trials to Solve Sleep Problems in Patients with Rare Diseases.Clocks & sleep · 2024Article
- Increasing Diversity, Equity, Inclusion, and Accessibility in Rare Disease Clinical Trials.Pharmaceutical medicine · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The increasing number and high prices of orphan drugs have triggered concern among patients, payers, and policymakers about the affordability of new drugs approved using the incentives set by the Orphan Drug Act (ODA) of 1983. This study evaluated the factors associated to the differences in the treatment cost of new orphan and non-orphan drugs approved by the FDA from 2017 to 2021. A generalized linear model (GLM) with the Gamma log-link analysis was used to ascertain the association of drug characteristics with the treatment costs of orphan and non-orphan drugs. The results of the study showed that the median and interquartile range (IQR) drug cost was USD 218,872 (IQR = USD 23,105) for orphan drugs and USD 12,798 (IQR = USD 57,940) for non-orphan drugs (
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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.