Trials

The evolution of a new medicine takes many years as it is evaluated thoroughly before it can be prescribed to treat patients.

The chemical compound, which eventually becomes a medicine having the potential to treat disease, is first evaluated in the laboratory. This usually includes the use of computer models, cells and animal research. This medicine is evaluated in humans in what are called “clinical trials” only if the laboratory results are promising.

Clinical trials are compulsory for all new medicines. Regulators will only give approval for a new medicine if these trials demonstrate it has a favourable benefit: risk profile.

A clinical trial is a research study in human volunteers, conducted in strictly controlled settings, evaluating new medical treatments for patients. The aim of such trials is to determine whether a new treatment option is safe, effective and better than currently available treatments. These trials have to be approved by several regulatory authorities and ethics committees before they can commence. Patients also have to give their informed consent to take part in such a study.

There are different types or phases of clinical trials. In phase I trials a small group of either healthy volunteers or patients with the specific disease under evaluation, will receive a new treatment to determine its safety, metabolism and dose ranges. Phase II trials are conducted in a larger group of patients with the disease under study, to assess clinical efficacy of the new treatment. Phase III trials involve a large number of patients to test the clinical effectiveness of the new treatment, normally in comparison to any established treatment available, and to monitor for any side effects. Phase IV studies are post-marketing studies to delineate additional information including the drug's benefits, optimal use and side effects.

Clinical trials are carefully planned to make sure that participants are protected and that the research yields valid results. A detailed plan for a clinical trial, known as a “protocol”, is prepared by the company and reviewed and approved by an independent ethics committee before trials can go ahead.

All clinical trials are conducted according to the guidelines developed by the concerned regulators. These guidelines provide an internationally accepted ethical and scientific quality standard for designing, conducting, recording and reporting trials.