New medicines are developed as follows:
Preclinical Testing - A pharmaceutical company conducts laboratory and animal studies to show biological activity of the compound against the targeted disease, and the compound is evaluated for safety. These tests take approximately three and one-half years.
Investigational New Drug Application (IND) - After completing preclinical testing, the company files an IND with the FDA to begin to testing the drug in humans. The IND becomes effective if the FDA does not reject it within 30 days. The IND contains information on the results of previous experiments, how, where and by whom the new studies will be conducted; the chemical structure of the compound; how it is thought to work in the body; any toxic effects found in the animal studies; and how the compound is manufactured. In addition, the IND must be reviewed and approved by the Institutional Review Board, located where the studies will be conducted, and progress reports on clinical trials must be submitted at least annually to the FDA.
Clinical Trials, Phase I - These tests take about one year and involve about 20 to 80 normal, healthy volunteers. The tests study a drug's safety profile, including the safe dosage range. The studies also determine how a drug is absorbed, distributed, metabolized and excreted, and the duration of its action.
Clinical Trials, Phase II - In this phase, controlled studies of approximately 100 to 300 volunteer patients (people with the disease), assess the drug's effectiveness. This study takes approximately two years.
Clinical Trials, Phase III - This phase lasts about three years and usually involves 1,000 to 3,000 patients in clinics and hospitals. Physicians monitor patients closely to determine efficacy and identify adverse reactions.
