Research Process

There are four phases in the medical research process for adults. Since we work with children, only phases II and III apply to us. Please see the glossary for explanations of technical words.

Phase I

Phase I studies are the initial tests of new drugs in humans. They are small studies, which are usually not placebo controlled. They:

• Usually include healthy volunteers (who may be paid), but may include patients.
• Ascertain safe, tolerable levels.
• Discover initial pharmacokinetics.

Phase II

Phase II studies may be randomised, and they may be placebo controlled. They:

• Include participants who have a specific disease or illness.
• Determine the proper dosages.
• Provide evidence of activity and better evidence of safety.

Phase III

Phase III studies are comparative, controlled studies. They:

• Include participants who have a specific disease or illness.
• Assess the efficacy and safety of a drug, and therefore the balance of risks and benefits.
• Compare the benefits and side effects with those of standard treatments or placebos, or both.

Phase IV

Phase IV studies evaluate medicines that are already available for doctors to prescribe, rather than new medicines that are still being developed. They include participants who have a specific disease or illness. The main reasons for conducting this phase are to find out:

• More about the side effects and safety of a drug in a larger population.
• What the long term risks and benefits are, by conducting long term follow-up.
• How well the medicines work when used in a broader population, or in combination with other treatments.