Module 4: Regulatory Pathways and the Drug Approval Process

In the fourth module of the Global DARE Foundation’s Patient Collaborator Training Series, Dr. Mousumi Bose explains how new therapies move from early scientific discovery to FDA approval to becoming commercially available as a therapy. We will learn about each stage of the drug development and regulatory pathway,  from preclinical studies and Investigational New Drug (IND) applications through Phase I–III clinical trials, FDA review, and post-market safety monitoring. The module also highlights how patient collaborators can play an important role in shaping trials and advancing treatments, especially for rare diseases. By understanding the approval process, patients and caregivers are better equipped to advocate for timely, safe, and effective therapies. Summary, script, and slides developed by Mousumi Bose and Natalie Zolnierczyk Speaker Bio: Dr. Mousumi Bose is an Associate Professor in the Department of Nutrition and Food Studies at Montclair State University in Montclair, NJ, and the mother of Ilan Betzer (2010-2011), who was born with a severe form of Zellweger spectrum disorder (ZSD), a peroxisome biogenesis disorder. Through her experience with her son, Dr. Bose refocused her career goals to study rare disease. Currently, Dr. Bose serves as the Co-Chair for the Medical and Scientific Advisory Board of the Global DARE Foundation. Her long-term research goals are to combine her training in nutritional sciences and public health with her knowledge and experience with peroxisomal disorders to help improve the quality of life for families affected by rare metabolic disorders like Adult Refsum Disease. She is also passionate about training the next generation of nutrition professionals in rare metabolic diseases, weaving in lessons on Adult Refsum Disease as well as other rare disorders in her advanced nutrition courses.  Refsum Disease & Global DARE Foundation Did you know that one in 300 people who have Retinitis Pigmentosa also have Refsum disease? Many are diagnosed with Refsum Disease 15-20 years after being diagnosed with Retinitis Pigmentosa. Early diagnosis is so important because dietary therapy can slow sight and hearing loss associated with Refsum and lessen other symptoms. Join the Global DARE Foundation in making a difference for the Refsum community. Please consider making a donation at https://www.defeatadultrefsumeverywhe.... Your support helps fund essential research, increase awareness, and accelerate progress toward better treatments—and one day, a cure—for Refsum Disease. Every gift, no matter the size, brings hope to families living with this ultra-rare condition. Visit our website at https://www.defeatadultrefsumeverywhe... #curerefsum #retinitispigmentosa #lowvision