The developing companies themselves are responsible for carrying out all the research and development. In vitro and animal studies provide initial information on the efficacy and safety of potential new drugs. Investigational New Drug Application. The IND describes the structure and chemistry of the drug, the preclinical results and any other information pertaining to the drug. It also includes a detailed proposal for clinical trials.
Clinical trials test the safety and effectiveness of the drug in human volunteers and patients.
Phase I clinical trials are small studies usually carried out on volunteers to determine the safety of the drug. The investigators are primarily interested in learning how the body reacts to the drug, what side effects are to be expected and what dosage levels can be used before the drug becomes toxic. Some preliminary information regarding effectiveness may also be gained. Phase II clinical trials are small trials on persons having the disease or medical condition to determine whether the drug has some level of therapeutic effect. The trials are usually carried out with a treatment group, who receive the drug, and a matched control group, who receive a placebo.