Pharmacological research in the field of rare diseases arouses great interest but faces very difficult methodological challenges because of the small number of affected people.
The course deals with the methodological problems concerning the design of trials on rare diseases, emphasizing the similarities and peculiarities of this context compared to trials on common diseases.
The types of design and the analysis methodologies (e.g. Bayesian adaptive randomization trials, Group-sequential designs) on rare diseases studies are discussed.
Finally, the role of meta-analysis in the assessment of treatments for rare diseases is explained.


  • Theoretical bases of clinical trials
  • Planning a clinical trial: the essential elements of a protocol
  • Methods of randomization
  • Conditions of blindness and placebo
  • The different types of experimental design
  • Controlled clinical trials: statistical issues
  • Sample size calculation
  • Non-inferiority and superiority trials
  • Statistical analysis of trial results
  • Data representation and summary measures
  • P-values and confidence intervals
  • Guide for a correct use of statistical tests
  • Univariate and multivariate analyses
  • Efficacy measures (relative and absolute risk, number needed to treat)
  • GCP: general overview
  • Critical reading of an article on the effectiveness of a treatment
  • Guide to the critical reading of a scientific article
  • Exercise in small groups: reading and evaluation of a scientific article