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Clinical Trials Phases Of Clinical Trials Phase 1 Clinical Study Primary End Point: Safety Phase 2 Clinical Study Primary End Point: Efficacy Increase in cardiac output. Phase 3 Clinical Study Primary End Point: Efficacy Reduction in Mortality Rate Phase 4 Clinical Study Primary End Point: Efficacy Market Driven

Clinical trials are a set of procedures in medical research and drug development that are conducted to allow safety (or more specifically, information about adverse drug reactions and adverse effects of other treatments) and efficacy data to be collected for health interventions (e.g., drugs, diagnostics, devices, therapy protocols). These trials can take place only after satisfactory information has been gathered on the quality of the non-clinical safety, and Health Authority/Ethics Committee approval is granted in the country where the trial is taking place. Depending on the type of product and the stage of its development, investigators enroll healthy volunteers and/or patients into small pilot studies initially, followed by larger scale studies in patients that often compare the new product with the currently prescribed treatment. As positive safety and efficacy data are gathered, the number of patients is typically increased. Clinical trials can vary in size from a single center in one country to multicenter trials in multiple countries. Due to the sizable cost a full series of clinical trials may incur, the burden of paying for all the necessary people and services is usually borne by the sponsor who may be a governmental organization, a pharmaceutical, or biotechnology company. Since the diversity of roles may exceed resources of the sponsor, often a clinical trial is managed by an outsourced partner such as a contract research organization or a clinical trials unit in the academic sector

Phases Of Clinical Research

Phase 1 Clinical Study Primary End Point: Safety

Most phase 1 studies are not conducted in people who have the disease in question (the exception being drugs with low therapeutic indices for use in lifethreatening diseases such as cancer and AIDS). The end point for this first in human study is usually safety and is conducted in human volunteers. The safety outcomes are assessed after single and multiple dose administrations.

Phase 2 Clinical Study Primary End Point: Efficacy Increase in cardiac output.

Phase 2 studies are usually exploratory studies in each candidate indication to determine which are most suitable for further development. In the case of CHF, the end point is efficacy as indicated by increased cardiac output measured by echocardiography. Phase 2 studies often explore the most appropriate dose of drug to use too, looking for the best efficacy without causing undue safety risks.

Phase 3 Clinical Study Primary End Point: Efficacy Reduction in Mortality Rate

In the case of CHF, the end point used in Phase 2 studies will not suffice for a Phase 3 study as the FDA and the market place requires the demonstration of clinical relevance and benefit. Typically, the end point would be reduction in mortality rate and would therefore require the study to follow patients for a longer period of time.

Phase 4 Clinical Study Primary End Point: Efficacy Market Driven

If the Phase 3 clinical study for reduction in mortality rate in CHF demonstrates the new drug is effective and it gains FDA approval, then Phase 4 studies can be used to expand the indications to capture more of the CHF market. For example, treatment of CHF in patients over the age of 65; use in combination with beta-adrenergic blockers to treat CHF; and the avoidance of hospitalization for exacerbations of CHF.