ArticleThe American journal of bioethics : AJOB2023
Rare Disease, Advocacy and Justice: Intersecting Disparities in Research and Clinical Care.
Article in The American journal of bioethics : AJOB, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 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
23 citing papers in PubMed, 37 citations in OpenAlex.
- Rare disease genomics and justice: overview of a workshop at the Fondation Brocher, 22-24 January 2025.Journal of community genetics · 2026Article
- On the Unfairness of the 'Fair-share Principle' for Health Research.Public health ethics · 2026Article
- The CHI-RON Study: Using PCORnet® and Patient Engagement Strategies to Improve Diversity Among Research Participants in the Congenital Heart Initiative.Medical care · 2026Article
- The social phenomenon of rare diseases: a logical-modal analysis.Frontiers in sociology · 2026Article
- "We are ambassadors, we are advocates": rare disease patient advocacy groups as knowledge brokers across health and social systems-a qualitative study from Poland.Frontiers in public health · 2026Article
- Navigating the human gene editing scene and proposing a new governance framework in Africa: from knowledge to policy to action.Frontiers in genome editing · 2026Article
- Assessing rare disease understanding: a novel disease readiness level framework.Orphanet journal of rare diseases · 2025Article
- Trends, lag and characteristics of rare disease drug approval in the USA and China, 1983-2022.Orphanet journal of rare diseases · 2025Article
- Precision diagnostic and therapeutic interventions in rare genetic neurodevelopmental disorders.Pediatric research · 2025Review
- A Zebra in Horse's Clothing: Rethinking the Diagnosis of Rare Diseases.Molecular genetics & genomic medicine · 2025Review
- Optimizing Biomedical Health Efficiency: Unlocking the Full Potential of Life Science Innovation Through System Design.Therapeutic innovation & regulatory science · 2025Article
- Distributional Cost-Effectiveness Analysis in Genomic Medicine: Considerations for Addressing Health Equity.Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research · 2025Article
- Building Better Medicine: Translational Justice and the Quest for Equity in US Healthcare.The American journal of bioethics : AJOB · 2025Article
- Preclinical research (on rare diseases): we need to talk about health equity.Mammalian genome : official journal of the International Mammalian Genome Society · 2025Review
- Article
- Anxiety in Turner syndrome: Engaging community to address barriers and facilitators to diagnosis and care.American journal of medical genetics. Part A · 2024Article
- Increasing Diversity, Equity, Inclusion, and Accessibility in Rare Disease Clinical Trials.Pharmaceutical medicine · 2024Review
- Patient-Centric Approaches: Revolutionizing Rare Disease Drug Research.Infectious disorders drug targets · 2024Article
- Investigation of dermal collagen nanostructures in Ehlers-Danlos Syndrome (EDS) patients.PloS one · 2024Article
- Building advocacy into research.Disease models & mechanisms · 2023Article
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Authors and funding
4 authors at 3 institutions in 1 country.
Funding
Abstract
Rare genetic diseases collectively impact millions of individuals in the United States. These patients and their families share many challenges including delayed diagnosis, lack of knowledgeable providers, and limited economic incentives to develop new therapies for small patient groups. As such, rare disease patients and families often must rely on advocacy, including both self-advocacy to access clinical care and public advocacy to advance research. However, these demands raise serious concerns for equity, as both care and research for a given disease can depend on the education, financial resources, and social capital available to the patients in a given community. In this article, we utilize three case examples to illustrate ethical challenges at the intersection of rare diseases, advocacy and justice, including how reliance on advocacy in rare disease may drive unintended consequences for equity. We conclude with a discussion of opportunities for diverse stakeholders to begin to address these challenges.
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.