When Elise Felicione tried to design a clinical trial for a supplement, 5 things got in the way. It’s a lesson for the ...

FDA's Plausible Mechanism Framework streamlines individualized therapy approvals for ultra-rare diseases.

By Dr. Priyom Bose, Ph.D. Inviting adults to colorectal cancer screening at age 60 shifts diagnoses to earlier stages without ...

As rare disease trials face persistent feasibility challenges, Bayesian designs are gaining momentum by enabling more ...

A key advantage of using RWD in a natural history study is gaining diverse and representative data. RWD covers broad patient populations, including those with comorbidities, various age groups, and ...

This transition is explored in “Embodied Artificial Intelligence in Healthcare: A Systematic Review of Robotic Perception, ...

How do we take on and resolve vein-related pelvic pain in women? PeVD has been associated with venous-origin chronic pelvic ...

News headlines often compress complex science into a simple storyline: If two things are happening at the same time, one must ...

"Disappointing" trial results, but some researchers remain hopeful ...

In an interview, C. Ola Landgren, MD, PhD, delved into the details of the FDA's draft guidance on using MRD as a basis for ...

Divergence in the post-progression interval suggests disparities may concentrate in subsequent-line therapy, surveillance, supportive care, or access to timely interventions outside trial ...

In today’s ACT Brief, we explore how Bayesian trial designs are gaining traction in rare disease research, what priorities clinical operations leaders should focus on in 2026, and how rare disease ...