SynthesisInternational journal of environmental research and public health2021
Bayesian Approaches for Confirmatory Trials in Rare Diseases: Opportunities and Challenges.
Synthesis in International journal of environmental research and public health, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 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
9 citing papers in PubMed.
- Patient-centred and fit-for-purpose clinical trials in rare cancers: lessons from sarcomas for operationalising 2024 WHO Guidance and ICH E6(R3).EClinicalMedicine · 2026Review
- A Bayesian framework to evaluate non-inferiority in randomized controlled trials of uncommon conditions.Journal of clinical epidemiology · 2026Article
- Key challenges in developing a gene therapy for Usher syndrome: machine-assisted scoping review.Journal of community genetics · 2024Article
- Targeted therapy for rare lung cancers: Status, challenges, and prospects.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Review
- Improving resource utilisation in SLE drug development through innovative trial design.Lupus science & medicine · 2023Review
- New prospectives on treatment opportunities in RASopathies.American journal of medical genetics. Part C, Seminars in medical genetics · 2022Review
- [Progress and Application of Bayesian Approach in the Early Research and Development of New Anticancer Drugs].Zhongguo fei ai za zhi = Chinese journal of lung cancer · 2022Article
- Accrual-Monitoring Practices for Various Disease Trials among AACI Member Cancer Centers.Clinics and practice · 2022Article
- Application of Bayesian methods to accelerate rare disease drug development: scopes and hurdles.Orphanet journal of rare diseases · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The aim of this narrative review is to introduce the reader to Bayesian methods that, in our opinion, appear to be the most important in the context of rare diseases. A disease is defined as rare depending on the prevalence of the affected patients in the considered population, for example, about 1 in 1500 people in U.S.; about 1 in 2500 people in Japan; and fewer than 1 in 2000 people in Europe. There are between 6000 and 8000 rare diseases and the main issue in drug development is linked to the challenge of achieving robust evidence from clinical trials in small populations. A better use of all available information can help the development process and Bayesian statistics can provide a solid framework at the design stage, during the conduct of the trial, and at the analysis stage. The focus of this manuscript is to provide a review of Bayesian methods for sample size computation or reassessment during phase II or phase III trial, for response adaptive randomization and of for meta-analysis in rare disease. Challenges regarding prior distribution choice, computational burden and dissemination are also discussed.
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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.