Recent advances in methodology for clinical trials in small populations: the InSPiRe project

Value of information methods to design a clinical trial in a small population to optimise a health economic utility function

(2018) Michael Pearce et al.   BMC Medical Research Methodology

Approaches to sample size calculation for clinical trials in rare diseases

(2018) Frank Miller et al.   PHARMACEUTICAL STATISTICS

Robustness of testing procedures for confirmatory subpopulation analyses based on a continuous biomarker

(2018) Alexandra Christine Graf et al.   STATISTICAL METHODS IN MEDICAL RESEARCH

Flexible alpha allocation strategies for confirmatory adaptive enrichment clinical trials with a prespecified subgroup

(2018) Toshifumi Sugitani et al.   STATISTICS IN MEDICINE

A design-by-treatment interaction model for network meta-analysis and meta-regression with integrated nested Laplace approximations

(2018) Burak Kürsad Günhan et al.   Research Synthesis Methods

Discrete Approximation of a Mixture Distribution via Restricted Divergence

(2017) Christian Röver et al.   JOURNAL OF COMPUTATIONAL AND GRAPHICAL STATISTICS

Does the low prevalence affect the sample size of interventional clinical trials of rare diseases? An analysis of data from the aggregate analysis of clinicaltrials.gov

(2017) Siew Wan Hee et al.   Orphanet Journal of Rare Diseases

Bayesian treatment comparison using parametric mixture priors computed from elicited histograms

(2017) Peter F Thall et al.   STATISTICAL METHODS IN MEDICAL RESEARCH

Determination of the optimal sample size for a clinical trial accounting for the population size

(2016) Nigel Stallard et al.   BIOMETRICAL JOURNAL

Meta-analysis of two studies in the presence of heterogeneity with applications in rare diseases

(2016) Tim Friede et al.   BIOMETRICAL JOURNAL

Directions for new developments on statistical design and analysis of small population group trials

(2016) Ralf-Dieter Hilgers et al.   Orphanet Journal of Rare Diseases

Systematic reviews in paediatric multiple sclerosis and Creutzfeldt-Jakob disease exemplify shortcomings in methods used to evaluate therapies in rare conditions

(2016) Steffen Unkel et al.   Orphanet Journal of Rare Diseases

Optimizing Trial Designs for Targeted Therapies

(2016) Thomas Ondra et al.   PLoS One

Unified approach for extrapolation and bridging of adult information in early-phase dose-finding paediatric studies

(2016) Caroline Petit et al.   STATISTICAL METHODS IN MEDICAL RESEARCH

Meta-analysis of few small studies in orphan diseases

(2016) Tim Friede et al.   Research Synthesis Methods

Designing a Pediatric Study for an Antimalarial Drug by Using Information from Adults

(2015) Caroline Petit et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Hartung-Knapp-Sidik-Jonkman approach and its modification for random-effects meta-analysis with few studies

(2015) Christian Röver et al.   BMC Medical Research Methodology

Methods for identification and confirmation of targeted subgroups in clinical trials: A systematic review

(2015) Thomas Ondra et al.   Journal of Biopharmaceutical Statistics

Decision-theoretic designs for small trials and pilot studies: A review

(2015) Siew Wan Hee et al.   STATISTICAL METHODS IN MEDICAL RESEARCH

Sharing clinical trial data on patient level: Opportunities and challenges

(2014) Franz Koenig et al.   BIOMETRICAL JOURNAL

Adaptive designs for subpopulation analysis optimizing utility functions

(2014) Alexandra C. Graf et al.   BIOMETRICAL JOURNAL

Interleukin-2 receptor antagonists for pediatric liver transplant recipients: A systematic review and meta-analysis of controlled studies

(2014) Nicola D. Crins et al.   PEDIATRIC TRANSPLANTATION

Adaptive enrichment designs for clinical trials

(2013) N. Simon et al.   BIOSTATISTICS