US Pharm. 2016;41(10):11.

According to the Center for Drug Evaluation and Research, novel drugs are those that offer new and innovative treatments to fulfill unmet medical needs or to advance clinical care and improve public health. The FDA assesses both the quality and the quantity of novel drugs, providing scientific and regulatory advice at every step of the process to advance development.

Approval Rates: Over the last decade, the number of applications for novel drugs ranged from 23 to 41 annually (average: 35), and an average of 29 were approved per year. Over the same period, an average of 82.6% of applications were approved, and the lowest annual approval rate was 51.4% in 2007. The largest number of novel drugs approved (45) occurred in 2015, and this rate was a 9.1% increase over 2014. In 2015, approximately 87% of novel drugs were approved following the first review cycle.

Approval Pathways: There are four pathways that expedite the development and/or approval process in order to bring novel drugs to market as quickly as possible: priority review, fast-track designation, breakthrough therapy, and accelerated approval. For priority review, the review time is <6 months instead of the standard 10 months; the fast-track designation means that certain portions of the application are reviewed ahead of the complete submission. In breakthrough therapy, a clinically significant end point shortened the development time, and accelerated approval is given when a drug intended for a serious or life-threatening illness offers a benefit over current treatments based on a surrogate endpoint. Rates for these pathways in 2015 were 53.3%, 31.1%, 22.2%, and 15.6%, respectively. About three-quarters of these drugs were new molecular entities (by New Drug Application) or new therapeutic biologics (by Biologic License Application). Of the novel drugs approved, 64.4% were drugs first approved in the United States and 35.5% were first-in-class, one indicator of the innovative nature of a drug. First-in-class drugs have unique mechanisms of action for treating the medical condition.

Therapeutic Categories: The novel drugs approved in 2015 included four for multiple myeloma treatment and others for treatment of non–small cell lung cancer, metastatic melanoma, breast cancer, high-risk neuroblastoma, and colorectal cancer. Also approved were novel drugs for treating heart failure and hypercholesterolemia, as well as the first reversal agent for a commonly prescribed blood thinner. Novel drugs were approved for the treatment of cystic fibrosis, irritable bowel syndrome, chronic hepatitis C virus genotype 3 infections, and urinary tract infections. A record number of novel drugs (21) were approved to treat rare or orphan diseases.

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