June 14, 2016 article Open access Directions for new developments on statistical design and analysis of small population group trials Orphanet Journal of Rare Diseases DOI: 10.1186/s13023-016-0464-5 Full text (OA) OpenAlex Authors 0 Author list not loaded yet. Cited by 4 stored of 40 Search Sort Most cited Newest Oldest Patent citations Title Any typearticle review book-chapter conference-paper preprint dissertation book dataset other Any fieldAgricultural and Biological Sciences Arts and Humanities Biochemistry, Genetics and Molecular Biology Business, Management and Accounting Chemical Engineering Chemistry Computer Science Decision Sciences Dentistry Earth and Planetary Sciences Economics, Econometrics and Finance Energy Engineering Environmental Science Health Professions Immunology and Microbiology Materials Science Mathematics Medicine Neuroscience Nursing Pharmacology, Toxicology and Pharmaceutics Physics and Astronomy Psychology Social Sciences Veterinary Open access Does randomization matter in rare disease clinical trials 2026 PLoS ONE article Mathematics Statistical Methods in Clinical Trials Open access 0 citations A Pharmacometrics‐Informed Trial Simulation Framework for Optimizing Study Designs for Disease‐Modifying Treatments in Rare Neurological Disorders 2025 CPT Pharmacometrics & Systems Pharmacology article Mathematics Statistical Methods in Clinical Trials Open access 3 citations Methodological insights from the EPISTOP trial to designing clinical trials in rare diseases—A secondary analysis of a randomized clinical trial 2024 PLoS ONE article Mathematics Statistical Methods in Clinical Trials Open access 2 citations Innovative methodologies for rare diseases clinical trials 2024 Orphanet Journal of Rare Diseases article Mathematics Statistical Methods in Clinical Trials Open access 1 citations 4 results References 0