Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs

Chapter 2

Accelerated Approval

Louis Cabanilla and Christopher P. Milne

2.1 Introduction

The development and approval of a new drug is a complex and time-consuming procedure, which takes many years to accomplish. By the time a drug receives FDA approval, extensive lab and clinical work must be performed to ensure that the drug is both safe and effective, and months or years spent by the FDA reviewing the drug’s application. Although this level of quality control is a great benefit to people who use the drug, the extensive amount of time spent on development and regulatory submission review represents a barrier for a person with a serious or life threatening disease for which no treatment exists, or for a person for whom available treatments have failed. In such cases, a speedy development and regulatory review are of the utmost importance. A patient with no or few therapeutic options is often willing to accept a higher risk to benefit ratio including the use of a treatment whose efficacy is predicated on indirect measures of the expected clinical benefit.

2.2 Accelerated Development Versus Expanded Access in the U.S.A.

In the United States, an emphasis has been placed on making potentially life saving drugs available as soon as possible. In some instances, this process involves making experimental drugs available to patients who are not enrolled in clinical trials, but more typically it involves programs designed to decrease the time to market for these important drugs. This movement ...