Learning objectives Trial designs
4 important questions on Learning objectives Trial designs
In how many cases of rare diseases are no treatments yet?
What are important topics for the methodology of trials for rare diseases?
Heterogeneity => different phenotypes
Treatment goals => what is important for the patient
Vulnerability
What is the cycle to think about before starting a trial?
Performing: what design, level of evidence, what is the burden on the patient, other medicines that impact your medicine
Monitoring: when, what
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What is a very personalized way of outcome measuring?
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