China leads rare disease trials from 2020

According to GlobalData’s Clinical Trials Database, non-industry sponsored trials lead rare disease indications, accounting for 55.6% of all trials since 2004.

Rare disease indications can focus on specific patient populations, which may not provide enough financial incentive for pharmaceutical companies to invest heavily in research and development in these areas. Non-industry sponsors, such as academic institutions or government organisations, may step in to fill this gap due to their focus on advancing scientific knowledge and public health rather than profit.

Non-industry sponsors may additionally be more experienced at navigating incentives or may receive grants and funding specifically aimed at rare disease research. Lastly, non-industry sponsors may collaborate with industry partners to
leverage resources and expertise. This collaboration can be crucial in pooling the necessary resources to conduct trials for diseases with limited patient populations.
Asia-Pacific (APAC) leads in total number of rare disease trials. Since 2017, APAC surpassed North America in rare disease trials and has since maintained dominance in this space. Rare disease trials are dominant in APAC due to significant unmet medical needs in several regions and increased population sizes where patient accrual is significantly more attainable. Cooperations such the Asia-Pacific cooperation is actively trying to increase research and development for rare diseases within APAC. Conducting trials within APAC may also seem more cost-effective for sponsors due to increased emerging markets and increased availability of trial sites with a specialised focus on rare diseases. Europe, the Middle East and Africa, and South and Central America remain far behind the leading regions. Historically, the US has held research supremacy, especially since the nation has the most overall trials. However, since 2020, China has dominated rare disease trials. This could be attributed to increased funding. China has also streamlined their processes for reviewing rare disease drugs, which facilitates quicker market access.