ReviewOrphanet journal of rare diseases2022
Application of Bayesian methods to accelerate rare disease drug development: scopes and hurdles.
Review in Orphanet journal of rare diseases, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers, 3 of them syntheses that pooled it.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
29 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- A systematic review of sample size determination in Bayesian randomized clinical trials: full Bayesian methods are rarely used.BMC medical research methodology · 2026Pooled it
- Cell-Based Therapies for Spinocerebellar Degenerations: A Systematic Review of Human Clinical Evidence.Cerebellum (London, England) · 2026Pooled it
- Sensitivity analysis in Bayesian clinical trials was underused and poorly reported: a systematic survey.BMC medical research methodology · 2026Pooled it
- Design, simulate, refine: simulation-guided clinical trials for accelerated drug development.Nature reviews. Drug discovery · 2026Review
- Rett syndrome: MECP2 biology, multisystem pathophysiology, and the evolving therapeutic landscape.European journal of pediatrics · 2026Review
- Pruritus is common in primary sclerosing cholangitis, persists over time, and its intensity is associated with disease severity: a multicentre, prospective observational study.Hepatology (Baltimore, Md.) · 2026Observational
- From Small Data to Big Decisions: How Clinical Pharmacology Shapes Rare Disease Development.Journal of clinical pharmacology · 2026Review
- The design of a small Bayesian hybrid-controlled trial utilizing time-to-event data from six published sources that used best alternative care to treat liver-predominant metastasis from primary ocular melanoma.Contemporary clinical trials communications · 2026Article
- Artificial Intelligence in Rare Diseases: Workflow-Integrated Precision Kidney Care.Clinics and practice · 2026Review
- Mavodelpar in patients with primary mitochondrial myopathy: a phase 1 trial.Scientific reports · 2026Article
- Can We Trust PAICs in Rare Diseases? Methodological Challenges and Limitations.Journal of market access & health policy · 2026Article
- The added value of Bayesian interim analysis in randomized phase II and phase III clinical trials in hemato-oncology.Haematologica · 2026Article
- Reimagining care of people living with rare diseases with artificial intelligence.PLoS medicine · 2026Article
- General Commentary: From hospital exemption to evidence: a statistical framework for leveraging real-world data.Frontiers in immunology · 2026Article
- Co-creation process of an app for people with rare diseases - a citizen science approach.Orphanet journal of rare diseases · 2025Article
- Use of Bayesian techniques in clinical trials for rheumatoid arthritis and systemic sclerosis: a scoping review.BMC rheumatology · 2025Article
- Prasinezumab: A Bayesian Perspective on Its Efficacy.Movement disorders : official journal of the Movement Disorder Society · 2025Article
- Navigating challenges in pediatric trial conduct: integrating bayesian sequential design with semiparametric elicitation for handling primary and secondary endpoints.BMC medical research methodology · 2025Article
- Biomarkers of disease progression in progressive supranuclear palsy for use in clinical trials.Brain communications · 2025Review
- Telitacicept demonstrates high probability of efficacy in myasthenia gravis: a Bayesian real-world study.Frontiers in neurology · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundDesign and analysis of clinical trials for rare and ultra-rare disease pose unique challenges to the practitioners. Meeting conventional power requirements is infeasible for diseases where sample sizes are inherently very small. Moreover, rare disease populations are generally heterogeneous and widely dispersed, which complicates study enrollment and design. Leveraging all available information in rare and ultra-rare disease trials can improve both drug development and informed decision-making processes. MAIN TEXT: Bayesian statistics provides a formal framework for combining all relevant information at all stages of the clinical trial, including trial design, execution, and analysis. This manuscript provides an overview of different Bayesian methods applicable to clinical trials in rare disease. We present real or hypothetical case studies that address the key needs of rare disease drug development highlighting several specific Bayesian examples of clinical trials. Advantages and hurdles of these approaches are discussed in detail. In addition, we emphasize the practical and regulatory aspects in the context of real-life applications.
conclusionThe use of innovative trial designs such as master protocols and complex adaptive designs in conjunction with a Bayesian approach may help to reduce sample size, select the correct treatment and population, and accurately and reliably assess the treatment effect in the rare disease setting.
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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.