Orphan Exclusivity, Drug-Device Designation Court Rulings Against FDA Would Be Fixed In User Fee Bill

The UK government has acknowledged that sponsors of clinical trials for rare diseases face challenges around patient recruitment and trial design, which will be addressed in its clinical trials reform, a lawyer says.

Clinical trial sponsors in the rare disease space face “big safety demands” from regulators, which can be challenging for those with limited resources. A CRO founder makes the case for “embedding automation” into the clinical trial process to keep pace with regulatory requirements.

The termination of the HHS Secretary’s Committee on Human Research Protections closed an important forum for discussing pediatrics and pregnancy in clinical trial design, a former SACHRP chair said.

FDA regulation was painted as an obstacle to US dominance in the cell and gene therapy space even as panelists at an agency event praised the Office of Therapeutic Products' track record under Nicole Verdun.