The State Of Trial Design & Evidence Generation In Rare Clinical Disease

By Derk Arts

Globally, an estimated 2.6 to 4.5 million people are affected by rare diseases. However, conducting clinical trials for these conditions presents unique challenges due to the small patient populations and limited existing research. With limited treatment options available, each breakthrough in rare disease research holds significant promise and hope for patients.

Fortunately, there is ongoing innovation in this field. New trial designs, the utilization of real-world data (RWD) and real-world evidence (RWE), and the development of synthetic control arms are all crucial elements that have the potential to expedite breakthroughs and approvals in the rare disease landscape.

Discover how innovative trial designs and evidence-generation methods are poised to drive advancements in the field of rare diseases by accessing the full article.