Rare Disease – Global Clinical Trial Landscape (2018-2022)

Rare diseases, as defined by the World Health Organization (WHO), are severe conditions affecting fewer than one in every 1,000 individuals and are often life-threatening. They collectively impact an estimated 260-440 million people worldwide, emphasizing their significance in the medical landscape. Rare diseases come with multiple challenges, including delayed diagnosis and a lack of expertise, particularly in chronic diseases where a cure remains elusive.

These conditions pose a global challenge. The Asia-Pacific (APAC) region, with around 260 million individuals, has a substantial rare disease population, mainly children. Europe and the United States each have over 30 million affected people.

From 2018 to 2022, rare disease clinical trials witnessed steady growth, with over 16,000 studies globally. APAC has the fastest 5-year compound annual growth rate (CAGR), while North America and Europe have been reported to conduct the most trials overall. Other notable contributors include the United States, Mainland China, the United Kingdom, Germany, and France.

For rare disease research, new therapeutic approaches include small molecule drugs, monoclonal antibodies, CAR-T cell therapy, gene therapy, enzyme replacement therapy, protein replacement therapy, and antisense oligonucleotides (ASOs). North America, particularly the United States, leads in venture funding, highlighting its commitment to rare disease research. However, biotech firms are expanding their biologic drug pipelines.

With all this data in mind, it is clear that rare diseases present a global challenge with ongoing clinical trials, innovative treatments, and growing venture funding. Discover why the collaboration and expertise of organizations like Novotech are vital in advancing rare disease research and improving the lives of millions affected.