2021 was the leading year in rare disease trials

According to leading data and analytics company GlobalData’s Clinical Trials Database, over the past 20 years trial activity has steadily increased year on year,
peaking in 2021.

There has been increased demand over the years for rare disease trials. Currently, only 5% of rare disease indications have a licensed treatment available. Incentives by the US Food and Drug Administration and European Medicines Agency such as orphan drug designations and PRIME designations have further driven sponsors to conduct rare disease trials. These incentives increase the feasibility for companies to invest in sponsors focusing on small-scale rare disease trials.

Trial designs have also changed over the past 20 years. Adaptive design, exploratory studies, and the use of real-world evidence help increase the efficiency and efficacy of rare disease trials. 2021 was a record-breaking year. This could be attributed to several reasons, the first being an increase in focus on using ribonucleic acid (RNA)-based therapies for rare disease treatment. Secondly, there was a significant increase in basket and umbrella trials, which allowed for more efficient testing of multiple rare diseases.

Asia-Pacific (APAC) leads in the total number of rare disease trials. In 2017, APAC surpassed North America in rare disease trials and has maintained dominance in this space since then. 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 as the Asia-Pacific cooperation are 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.