OBJECTIVES: This study assessed the trends in new therapeutic biologic license applications (BLA) and new molecular entities (NME) regulated by the Center for Drug Evaluation and Research and approved by the FDA in the period 1980–2017.

METHODS: The following information was collected from FDA online databases: date of approval; orphan designation; market status; discontinuation reasons; risk evaluation and mitigation strategy (REMS), black-boxed warning, medication guide, and date of first generic/biosimilar approval. The Anatomical Therapeutic Chemical (ATC) classification was collected from the WHO. Descriptive statistics, t-test and chi-square tests were performed in the analysis (α=0.05).

RESULTS: The FDA approved 132 BLA and 949 NME in the period of analysis. The annual average±standard deviation of approvals was 3.5±3.2 for BLA and 25.0±7.9 for NME. The approvals of BLA increased from 0.9±1.4 in the 1980s to 7.1±3.9 in the period 2010-2017. BLA represented 4.0%, 7.7%, 15.9%, and 21.5% of the approvals in the 1980s, 1990s, 2000s, and 2010-2017, respectively. 88.6% of the BLA and 85.0% of the NME were marketed as of 31-Dec-2017. The main ATC was antineoplastic and immunomodulating agents (69;52.3%) for BLA, and antiinfectives for systemic use (135;14.2%) for NME. BLA (48.5%) were more likely than NME (22.7%) to have orphan designation (p<0.001), REMS (14.4% vs. 4.4%, p<0.001), black-boxed warning (43.2% vs. 25.8%), and medication guide (30.3% vs. 17.1%). A lower percentage of BLA (6;4.5%) than NME (413;43.5%) experienced market competition (p<0.001). The average number of years without market competition was higher for BLA (17±3.9 years) than for NME (12.6±4.2 years).

CONCLUSIONS: The number of BLA licensed by the FDA increased overtime. In the period 2010-2017, BLA represented more than 1 in 5 new drugs approved by the FDA. BLA were more likely than NME to have an orphan designation, and safety-related FDA decisions.