On Sept. 29, 2023, the U.S. Food and Drug Administration announced it was taking steps to help further accelerate the development of novel drug and biological products for rare diseases.

Selected participants of the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program will be able to obtain frequent advice and regular ad-hoc communication with FDA staff to address product-specific development issues, including, but not limited to, clinical study design, choice of control group and fine-tuning the choice of patient population.