OBJECTIVES

:

The past 10 years has seen a steady growth in the number of drugs approved by the FDA in the United States, particularly in innovative drug approvals by the Center for Drug Evaluation and Research (CDER) from 26 in 2009 to 59 in 2018. This study investigated the changes in research and development that have led to the introduction of many novel disease classes, that aim at improving quality of life in chronic diseases, and extending survival in life threatening diseases.

METHODS

: Data on the trends in pharmaceutical approvals in the United States were obtained from publicly available information in financial reports and regulatory publications.

RESULTS

: Of the 59 drugs approved by the CDER in 2018, 19 (32%) were first in class, with mechanisms of action that differed from existing therapies. Moreover, 34 (58%) were approved to treat rare ‘orphan’ diseases, which affect fewer than 200,000 patients in the United States, for whom there was previously no treatment option. CDER also designated 24 (41%) of the 59 novel drugs as Fast Track, and a further 14 (24%) as Breakthrough Therapies, which are those that have shown preliminary evidence of effectiveness in serious or life threatening diseases. Both designations are aimed at reducing the development time and speeding up patient access. Further, 42 (71%) of these novel drugs were approved in the US before receiving approval in any other market. Finally, 7 new biosimilar drugs were approved in 2018, bringing the total number of reference biologic products to 9.

CONCLUSIONS

: The United States continues to lead the world in bringing new therapies to the market by responding to both regulatory and financial challenges. Much of the focus has been on the development of immuno-oncology products, orphan drugs, reducing the cost of biologics, and on the opportunities for faster regulatory approvals.