There have been major improvements in the regulatory environment in the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) countries over the last decade, which have lead to a decrease in the time it takes to bring new drugs to market as well as an increase in the number of medicines that become available. Furthermore, the introduction, formalisation and wider use of diverse regulatory pathways and designations have played a major role in this process, particularly for medicines developed in response to unmet medical need.

As part of an ongoing study to monitor regulatory performance, CIRS has analysed the trends in new medicines’ approval between 2005 and 2014 by three regulatory authorities; the US Food and Drug Authority (FDA), the European Medicines Agency (EMA) and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), focusing on the use of:

  • Facilitated regulatory pathways (FRP). FRPs for the purposes of this briefing specifically include:
    • Expedited review to accelerate regulatory assessment: in this report refers to EMA Accelerated Assessment, FDA Priority Review, PMDA Priority Review
    • Other pathways to enable the availability, review and/or approval of medicines; in this report include EMA Conditional Approval, EMA Exceptional Circumstances, FDA Fast Track, FDA Breakthrough Therapy, FDA Accelerated Approval
  • Orphan designations within each of the three agencies