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Implementation and Outcomes of Clinical Trials in Immune-Mediated Hearing Loss and Other Rare Diseases

  • Andrea Vambutas
  • Martin L. Lesser
Chapter

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.

Keywords

Autoimmune Inner Ear Disease (AIED) Sudden Sensorineural Hearing Loss (SSNHL) and Meniere’s Disease (MD) Autoinflammatory Disease Rare disease Orphan disease 

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Copyright information

© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Department of Otolaryngology and Molecular MedicineBarbara and Donald Zucker School of Medicine at Hofstra/NorthwellHempsteadUSA
  2. 2.Biostatistics UnitFeinstein Institute for Medical ResearchManhassetUSA
  3. 3.Department of Molecular Medicine and Population HealthBarbara and Donald Zucker School of Medicine at Hofstra/NorthwellHempsteadUSA

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