Pivotal Studies

Pivotal studies are trials conducted after the early clinical development program establishes the overall efficacy and safety of the drug. These trials are an expansion of the clinical trials program.

These trials are designed to satisfy the requirements of the regulatory agencies. Primary and secondary end points are chosen based on FDA recommendations or previous clinical data. There are guidances for identifying primary end points for certain disease states, such as female sexual dysfunction, for example. Please refer to the FDA guidance for clinical studies (see Further Reading).

Pivotal trials are the sponsor's phase 3 trials. These studies are expanded controlled and uncontrolled trials after preliminary evidence of effectiveness and safety has been established in the previous phases. This phase establishes the clinical requirements of regulatory agencies and provides the company's marketing department with information on which to base marketing themes.

Phase 3 studies are usually designed as double-blind, placebo-controlled trials that include an increase in patient number, exposure, and length of drug administration. It is important during phase 3 studies to arrange several meetings with the regulatory agencies to discuss the format of the registration application, any new indications, and the core of the clinical submission.