Types of Research Trial

By Louise Murchison

Types of Studies Case Report: These describe the medical history of a particular patient which is
normally a description of a single unusual or interesting case

Case-Series: A simple descriptive account of interesting characteristics observed in

a group of patients. They generally involve patients seen over a relatively short period of time. Generally case-series studies do not include control subjects. They often serve as a precursor to other studies.

Case-Control Studies: Begin with the absence or presence of an outcome then look backward (retrospective analysis) in time to try to identify possible causes or risk factors.
Cases = individuals with a particular disease or outcome; Controls = individuals without the disease or outcome.

Cohort Studies: Subjects with or without a risk factor of interest are followed over a
period of time (prospective analysis) and the occurrence of an outcome of interest is measured. When a randomised trial cannot be performed (e.g. smokers vs nonsmokers for CHD), cohort studies are the gold standard of research.

Cohort study Prospective RISK FACTOR Retrospective Case control study Cross-sectional Studies: Analyze data collected on a group of subjects at one time rather than over a period of time. Subjects are selected and information is collected from them at a particular point in time. This has led them to also be referred to as prevalence studies. Examples of cross-sectional studies include surveys and polls. Randomized Trials: Patients who have particular characteristics are randomized to receive one of two or more treatment regimens and after a period of time particular outcome measures are analysed. This allows treatment groups to be balanced for known and unknown variables that may influence the disease process and response to treatment so that a fair comparison can be made between the different interventions. Systematic Review: Give an overview of primary studies which contain an explicit statement of objectives, materials and methods (i.e. The Cochrane Collaboration is an example). OUTCOME

Meta-analysis: Involves the mathematical synthesis of the results of two or more (usually much more!) published studies and combines the results permitting an overall conclusion to be made.

Phases of Drug Development

Phase of Trial Design Features Number of Participants Data Collected

Pre-clinical

Animal and/or laboratory studies Investigation of a new drug in humans

Drug properties and potential uses Early Clinical Pharmacology and Safety

Phase I

20-50

Phase II

Provides preliminary efficacy and safety data. Randomised controlled trials

50-500

Therapeutic exploration, Initial Efficacy & Safety

Phase III

1000+

Therapeutic confirmation, Efficacy & Safety

Phase IV

Long term effects, cost

2000-10,000+

Therapeutic use, postmarketing Studies