Regulatory issues for evaluation of therapies to prevent or arrest disease progression

The Federal Food Drug and Cosmetic Act gives authority to the US Food and Drug Administration (FDA) to regulate foods, human and veterinary drugs, biological products, and cosmetics which are to be introduced into interstate commerce. The approval of pharmaceutical products for the treatment and/or prevention of periodontal diseases requires presentation of evidence that supports the safety and efficacy of the particular drug therapy in question. Sponsors need to carefully consider each step in the overall development plan beginning with an Investigational New Drug Application and concluding with submission of a New Drug Application for pre-market approval. This paper focuses specifically on the design, conduct, and analysis of clinical trials of drugs for the prevention or treatment of periodontal diseases. It is based upon a guidance document under development at the time of this presentation by FDA's Center for Drug Evaluation and Research that will include separate chemistry, toxicology, and biopharmaceutics sections. Unlike more formal FDA guidelines, a guidance document can be more easily and regularly updated to reflect changes in the state of the art of periodontal diagnosis and treatment.