Methodological Foundations of Clinical Research

  • Antonella BacchieriEmail author
  • Giovanni Della Cioppa
Part of the Health Informatics book series (HI)


This chapter focuses on clinical experiments, discussing the phases of the pharmaceutical development process. We review the conceptual framework and classification of biomedical studies and look at their distinctive characteristics. Biomedical studies are classified into two main categories, observational and experimental, which are then further classified into subcategories of prospective and retrospective and community and clinical, respectively. We review the basic concepts of experimental design, including defining study samples and calculating sample size, where the sample is the group of subjects on which the study is performed. Choosing a sample involves both qualitative and quantitative considerations, and the sample must be representative of the population under study. We then discuss treatments, including those that are the object of the experiment (study treatments) and those that are not (concomitant treatments). Minimizing bias through the use of randomization, blinding, and a priori definition of the statistical analysis is also discussed. Finally, we briefly look at innovative approaches, for example, how adaptive clinical trials can shorten the time and reduce the cost of classical research programs or how targeted designs can allow a more efficient use of patients in rare conditions.


Phase I, II, III, and IV trials Classification of biomedical studies Observational study Experimental study Equivalence/non-inferiority studies Superiority versus non-inferiority studies Crossover designs Parallel group designs Adaptive clinical trials Targeted designs 


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Copyright information

© Springer International Publishing 2019

Authors and Affiliations

  1. 1.CROS NT srl and Clinical R&D Consultants srlsVeronaItaly
  2. 2.Clinical R&D Consultants srlsRomeItaly

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