Navigating rare disease drug development requires coordinated, adaptive strategies as science and regulation evolve in parallel. Small, geographically dispersed patient populations, limited natural history data, and heterogeneous disease progression make trial design and execution unusually complex. At the same time,...
Kenneth Ndugga-Kabuye
Since PDUFA VII was signed into law in September 2022, the U.S. Food and Drug Administration (FDA) has introduced a series of initiatives aimed at addressing the persistent challenges of rare disease development. Collectively, these efforts form a more complete...
In recent years, the pharmaceutical landscape for rare diseases has witnessed a remarkable evolution. Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than 300 million people worldwidei and account for a...
Cell therapies, despite their transformative potential, face significant barriers that can hinder their development and commercialization. Safety concerns, manufacturing challenges, analytical complexities and scale-up difficulties present substantial risks. Competition from other drug modalities, coupled with the inherent complexity of clinical...
Today there are over 10,000 rare diseases affecting more than 30 million people in the U.S.1 Many of these disorders are life-threatening, and more than 90% do not have a U.S. FDA-approved treatment2. Novel gene therapies offer hope for improving...
Cell & Gene Therapy
Where Biomarkers and Gene Therapy Trials Intersect: Benefits Abound
We’re currently experiencing breakthrough times for clinical research in gene therapies. These complex treatments offer much-needed hope to more than 30 million U.S. patients affected by over 1000 rare diseases1, many of which are life-threatening and the vast majority of...