Established by FDA to Expedite Patient Access to Medications

Susannah Motl, Sarah J. Miller, Patrick Burns


Am J Health Syst Pharm. 2003;60(4) 

In This Article


Approximately 10-15 years elapse between the discovery of a new drug in the laboratory and its therapeutic use in the clinical setting.[1] One factor influencing the duration of drug development is the Food and Drug Administration's (FDA's) review of an agent's efficacy and safety data. In 2001, FDA's Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) approved 31 new molecular entities.[2,3,4] The average time from sponsor submission of a new drug application (NDA) or biologics license application (BLA) to FDA approval of these compounds was 18.6 months, an increase from 17.2 months in 2000, 12.9 months in 1999, and 12.5 months in 1998.

FDA has implemented several programs intended to reduce the review time of a new pharmaceutical and accelerate patient access to medications for the treatment of serious or life-threatening diseases. This review article provides an overview of these initiatives, with a focus on fast-track designation, priority review, accelerated approval, and orphan drug status. Imatinib mesylate (Gleevec, Novartis), which was approved for three phases (chronic, accelerated, and blast crisis) of chronic myelogenous leukemia (CML) in May 2001 and gastrointestinal stromal tumors (GIST) in February 2002, serves as an example of drug development focused on expedited patient access. The information on the clinical development and approval of imatinib was available through the Freedom of Information Act. Imatinib will be discussed throughout this review to illustrate the highlights and opportunities of each FDA program. Finally, the clinical implications of the programs will be described.


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