Implementation and outcomes of clinical trials in immune-mediated hearing loss and other rare diseases

Abstract

Clinical trials for rare diseases can be challenging to design, meet targeted enrollment, and obtain sufficient evidence of efficacy for FDA labeling of new drugs to treat these orphan diseases. Autoimmune inner ear disease, and related diseases of immune mediated hearing loss are yet to be classified orphan diseases. In this chapter, we have addressed some of the unique challenges in designing clinical trials for Autoimmune Inner Ear Disease, Sudden Sensorineural Hearing Loss, Meniere's Disease and Autoinflammatory Diseases.