Reliable Evidence from Small Populations: Rare-Disease Design Options
Evaluate precision, Bayesian methods, repeated measures, adaptive designs and external controls when populations are limited.
Educational articles on statistical methods, SPSS usage, and academic research.
Evaluate precision, Bayesian methods, repeated measures, adaptive designs and external controls when populations are limited.
Plan PRS access, protocol fields, outcome definitions, quality control and record updates using official ClinicalTrials.gov guidance.
Treat consent as an ongoing process, with clear key information, risks, voluntariness, data use and comprehension checks.
Check scientific validity, risk–benefit balance, consent, privacy and protocol consistency before ethics review.
A pilot study should test feasibility rather than prove effectiveness. Plan progression criteria, sample size and transparent reporting.
Define eligibility criteria that protect safety and measurement quality without unnecessarily restricting generalisability.