AML is the most common form of leukemia in adults, accounting for approximately a third of all leukemia cases worldwide.

Ovarian cancer is the 7th most common cancer and 8th leading cause of cancer mortality among women globally.

Lymphoma, including Hodgkin’s lymphoma (HL) and non-Hodgkin’s lymphoma (NHL), is one of the most common cancers worldwide.

Cancer of the skin is by far the most common of all cancers. Melanoma accounts for only about 1% of skin cancers but causes a large majority of skin cancer deaths.

Head and neck cancer (HNC) is the sixth most common cancer globally, with the highest incidence being observed in the Asia region.

Breast cancer is the most frequent cancer, impacting 2.1 million patients each year, and causes the greatest number of cancer-related deaths.

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.