Regional variability and efficient drug development based on ICH E17 guideline; Multi-regional clinical trials

Abstract

Globalisation of drug development has increased the use of MRCTs (multi-regional clinical trials) for regulatory submissions in multiple regions. However, it may be challenging to conduct a drug development programme globally, in part due to distinct and sometimes conflicting requirements from different regulatory authorities. At the same time, regulatory authorities face increasing challenges in evaluating data from MRCTs for drug approval. Data from MRCTs are often submitted to multiple regulatory authorities without a previously harmonised regulatory view on the study plan. To increase the acceptability of MRCTs in global regulatory submissions, ICH established E17 expert working group for creating a new international harmonized guideline entitled "General Principles for Planning and Design of Multi-Regional Clinical Trials" in June 2014. The guideline was finalized in November 2017 after having a public consultation on the draft guideline that was published in the summer of 2016. This guideline addresses strategic programme issues and issues that are specific to the planning and design of confirmatory MRCTs. It encourages to evaluate impacts of ethnic factors (intrinsic and extrinsic factors) on drug responses throughout drug development programme from an early stage. MRCTs, which are properly designed and executed according to this guideline, may facilitate more efficient drug development and increase the possibility of submitting marketing authorisation applications to multiple regulatory authorities in different regions simultaneously, thus providing earlier access to new drugs worldwide.

In this presentation, the key concepts of the final E17 guideline with recent review experiences of MRCT data in Japan will be explained and discussed.