ArticleFrontiers in pharmacology2022
Valuing the "Burden" and Impact of Rare Diseases: A Scoping Review.
Article in Frontiers in pharmacology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers, 4 of them syntheses 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
35 citing papers in PubMed, 4 syntheses or guidelines pooled it, 53 citations in OpenAlex.
- Self-reported perspective in rare genetic diseases: a systematic review of patient-reported outcome measures classified using the international classification of functioning, disability and health framework.Journal of patient-reported outcomes · 2026Pooled it
- Employment and work ability in individuals living with rare diseases: a systematic literature review.Orphanet journal of rare diseases · 2025Pooled it
- Measuring the health benefits of genome and exome sequencing: a systematic review of economic evaluations.Frontiers in public health · 2025Pooled it
- Assessing the effectiveness of measurement scales in evaluating the health-related quality of life in rare disease patients after treatment: a systematic review.Health and quality of life outcomes · 2024Pooled it
- Content validity, face validity and comprehensiveness of generic quality-of-life measures in adults and children with rare genetic conditions and their carers: a think aloud qualitative study.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2026Article
- The social phenomenon of rare diseases: a logical-modal analysis.Frontiers in sociology · 2026Article
- Genomics in Health and Biomedicine.Advances in experimental medicine and biology · 2026Review
- Development of a multidimensional disease burden assessment system for childhood neuroblastoma based on the social determinants of health theory.Frontiers in oncology · 2026Article
- Evolution and synergy of healthcare security policies for rare diseases in China: a quantitative content analysis.Frontiers in public health · 2026Article
- Extracellular vesicles as minimally invasive biomarkers and therapeutic platforms in rare neurological diseases.Frontiers in aging · 2026Review
- The Patient-Reported Outcomes Measurement Information System perspective of adults with long-standing atypical hemolytic uremic syndrome.Research and practice in thrombosis and haemostasis · 2025Article
- The UK National screening committee, the newborn genomes programme, and the ethical conundrum for UK newborn screening.Journal of community genetics · 2025Article
- Pivotal Studies for Drugs About to Be Launched for Rare Diseases: Will They Better Support Health Technology Assessment and Market Access than in the Past?Journal of market access & health policy · 2025Article
- Case Report: Neuropsychological Profile of a Patient With Intravascular Large B-Cell Lymphoma Following Infection and Vaccination.Archives of clinical neuropsychology : the official journal of the National Academy of Neuropsychologists · 2025Article
- Optimizing Patient Access to Orphan Medicinal Products: Lessons from Central and Eastern Europe.Journal of market access & health policy · 2025Article
- A retrospective study of insurance coverage status and economic cost of rare diseases in Hainan Province.Scientific reports · 2025Article
- Economic Burden of Haemophilia from a Societal Perspective: A Scoping Review.PharmacoEconomics - open · 2025Review
- Rare disease publishing trends worldwide and in China: A CiteSpace-based bibliometric study.Intractable & rare diseases research · 2025Article
- "Lack" and "Finally": A Qualitative Analysis of Barriers and Facilitators in Rare Disease Healthcare.International journal of environmental research and public health · 2025Article
- The role of public health in rare diseases: hemophilia as an example.Frontiers in public health · 2025Review
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 at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
PubMed holds no abstract for this paper.
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.