Clinical Trials II (SL_115)

There are, broadly speaking, two types of studies in biomedical research – randomized experiments and observational studies. Such studies range from pilot animal studies to clinical trials and epidemiological studies. In this course, the term clinical trial is to be understood in a wide sense as a prospective randomised study involving human subjects. The main purpose of a clinical trial is to assess the efficacy and safety of a treatment such as a drug, a medical device or even surgery or certain paramedical treatments such as traditional medicine. The course tries to adopt a patient centric view of clinical trials and thus to cover the safety aspect rather that efficacy as well as various types of ethical issues arising when planning, conducting, reporting clinical trials. While doing so, the emphasis will be on statistical issues whereas guidelines for “ethical statistical practice” will be discussed. Such guidelines are relevant for all types of studies in biomedical research including animal studies but are especially important for studies involving human subjects.
The safety aspect of clinical trials aspect will also be presented on the level of an individual trial either in (i) special safety trials with safety endpoints or in efficacy trials where various types of adverse events are monitored or in (ii) analyses of safety data using meta-analysis methods and pharmacovigilance studies monitoring marketed drugs.
We will also discuss precision medicine, i.e. attempts to explore how treatments can be tailored to individual patients based on relevant biomarkers. This leads us to exploring how such biomarkers can be validated for use in clinical trials and how diagnostic tests can be performed based on these biomarkers.
Finally, decision making based on quantitative criteria e.g. Go/No-Go decisions after each trial is also presented.