Publication date: 23 Sep 2024
The U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) has initiated the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program to help accelerate the development of new treatments for rare diseases. Selected companies will be able to obtain support from FDA staff to address clinical study design, control groups, choice of patient population, leveraging nonclinical information and product characterization. Among the conditions being explored at the program's onset are NGLY1 Deficiency, Isolated Methylmalonic Acidemia, Canavan Disease, Rett Syndrome, Friedreich's Ataxia, Vanishing White Matter Disease and Mucopolysaccharidosis Type IIIA. See the following link for a program announcement and list of participants: https://www.fda.gov/science-research/clinical-trials-and-human-subject-protection/support-clinical-trials-advancing-rare-disease-therapeutics-start-pilot-program.