Case
studies provide real insight in how to choose comparators for rare
diseases and oncology studies. Drug development is a very complex, long,
and expensive process. Once a suitable molecule is identified, then
clinical trials are undertaken to establish the effect of an
intervention.
Treatment effects are
efficiently isolated by controlling for bias, and confounding and
minimising variation. Key features of clinical trials that are used to
meet this objective are randomisation (possibly with stratification),
adherence to intent-to-treat principles, blinding, prospective
evaluation, and use of a control group. Compared to other types of study
designs (e.g., casecontrol studies, cohort studies, and case reports),
randomised trials have high validity, but are more difficult and
expensive to conduct.
The gold standard of
any scientific evidence is data generated from a randomised control
trial. In these studies, a pool of patients is randomly assigned to two
groups that receive different treatment. An experimental group will
receive the investigational medicinal product (IMP) and a control group
can receive a range of different interventions. The most commonly
utilised are a placebo (an inert compound that has no effect on the
endpoints measured) and an active comparator (usually the standard of
care [SOC]) for the indication being tested for. The choice of
medication for the control group depends on the nature of the disease.
For example, oncology patients are always put on medication for ethical
reasons.
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