THE ASIA-PACIFIC CRO

With a streamlined regulatory environment, including no requirement for an IND, and the supportive R&D Government refund of up to 43.5% on clinical research spend, Australia has become a preferred destination for early phase clinical trials.

A growing number of biotechnology companies, both in the West and in Asia, are specializing in gene therapies and other regenerative treatments boosted by the approval of the first genetic therapy agent (tisagenlecleusel) by the FDA in 2017.

Over 600 sites across Asia-Pacific have been involved in the clinical development of now approved IO drugs, and hundreds more are experienced in managing clinical trials with immunotherapies for both monotherapy and combination therapies.

We have observed an unprecedented effort in the clinical development of Orphan drugs in the recent years. In fact, in 2018, the majority of new drugs approved by the FDA and EMA were Orphan medicines.

The objective of this report is to describe the Orphan drug development landscape and explain how the positioning of the Asia region has been evolving in the global picture.