ReviewOrphanet journal of rare diseases2018
Recent advances in methodology for clinical trials in small populations: the InSPiRe project.
Review in Orphanet journal of rare diseases, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 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
22 citing papers in PubMed, 4 syntheses or guidelines pooled it.
- In Silico Clinical Trials in Drug Development: A Systematic Review.Therapeutic innovation & regulatory science · 2026Pooled it
- Bayesian Approaches for Confirmatory Trials in Rare Diseases: Opportunities and Challenges.International journal of environmental research and public health · 2021Pooled it
- Systematic review of statistical methods for safety data in malaria chemoprevention in pregnancy trials.Malaria journal · 2020Pooled it
- Agreement between results of meta-analyses from case reports and clinical studies, regarding efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II (MPS-II). A new tool for evidence-based medicine in rare diseases.Orphanet journal of rare diseases · 2019Pooled it
- Trial
- Addressing clinical uncertainties in ATMP reimbursement: a review of methodological guidelines and European practice.Frontiers in pharmacology · 2026Review
- A Pharmacometrics-Informed Trial Simulation Framework for Optimizing Study Designs for Disease-Modifying Treatments in Rare Neurological Disorders.CPT: pharmacometrics & systems pharmacology · 2025Article
- Barriers to Healthcare Access During the Coronavirus Disease 2019 (COVID-19) Pandemic: A Cross-Sectional Study Among Romanian Patients with Chronic Illnesses and Confirmed SARS-CoV-2 Infection.Healthcare (Basel, Switzerland) · 2025Article
- Augmenting Insufficiently Accruing Oncology Clinical Trials Using Generative Models: Validation Study.Journal of medical Internet research · 2025Article
- Primary lymphoma of the uterine cervix: A systematic review and integrated analysis of case reports and series.Oncology letters · 2025Article
- Innovative methodologies for rare diseases clinical trials.Orphanet journal of rare diseases · 2024Article
- Study design challenges and strategies in clinical trials for rare diseases: Lessons learned from pantothenate kinase-associated neurodegeneration.Frontiers in neurology · 2023Review
- New prospectives on treatment opportunities in RASopathies.American journal of medical genetics. Part C, Seminars in medical genetics · 2022Review
- Article
- Accrual-Monitoring Practices for Various Disease Trials among AACI Member Cancer Centers.Clinics and practice · 2022Article
- Current landscape of clinical development and approval of advanced therapies.Molecular therapy. Methods & clinical development · 2021Review
- N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine-A Review.Cancers · 2021Review
- Spinocerebellar ataxia clinical trials: opportunities and challenges.Annals of clinical and translational neurology · 2021Review
- Direct and indirect costs and cost-driving factors in adults with tuberous sclerosis complex: a multicenter cohort study and a review of the literature.Orphanet journal of rare diseases · 2021Review
- Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union.Frontiers in pharmacology · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
22 authors.
Funding
Abstract
Where there are a limited number of patients, such as in a rare disease, clinical trials in these small populations present several challenges, including statistical issues. This led to an EU FP7 call for proposals in 2013. One of the three projects funded was the Innovative Methodology for Small Populations Research (InSPiRe) project. This paper summarizes the main results of the project, which was completed in 2017.The InSPiRe project has led to development of novel statistical methodology for clinical trials in small populations in four areas. We have explored new decision-making methods for small population clinical trials using a Bayesian decision-theoretic framework to compare costs with potential benefits, developed approaches for targeted treatment trials, enabling simultaneous identification of subgroups and confirmation of treatment effect for these patients, worked on early phase clinical trial design and on extrapolation from adult to pediatric studies, developing methods to enable use of pharmacokinetics and pharmacodynamics data, and also developed improved robust meta-analysis methods for a small number of trials to support the planning, analysis and interpretation of a trial as well as enabling extrapolation between patient groups. In addition to scientific publications, we have contributed to regulatory guidance and produced free software in order to facilitate implementation of the novel methods.
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.