Early phase trials of novel hearing therapeutics: Avenues and opportunities

Anne G M Schilder; Matthew P Su; Rishi Mandavia; Caroline R Anderson; Evie Landry; Tanjinah Ferdous; Helen Blackshaw

doi:10.1016/j.heares.2019.07.003

Early phase trials of novel hearing therapeutics: Avenues and opportunities

Hear Res. 2019 Sep 1:380:175-186. doi: 10.1016/j.heares.2019.07.003. Epub 2019 Jul 7.

Authors

Anne G M Schilder¹, Matthew P Su², Rishi Mandavia³, Caroline R Anderson², Evie Landry⁴, Tanjinah Ferdous², Helen Blackshaw²

Affiliations

¹ evidENT, Ear Institute, University College London, Royal National Throat Nose and Ear Hospital, 330 Gray's Inn Road, London, WC1X 8DA, United Kingdom; National Institute of Health Research (NIHR) University College London Hospitals (UCLH) Biomedical Research Centre, Maple House, Suite A, 1st floor, 149 Tottenham Court Road, London, W1T 7DN, United Kingdom. Electronic address: a.schilder@ucl.ac.uk.
² evidENT, Ear Institute, University College London, Royal National Throat Nose and Ear Hospital, 330 Gray's Inn Road, London, WC1X 8DA, United Kingdom; National Institute of Health Research (NIHR) University College London Hospitals (UCLH) Biomedical Research Centre, Maple House, Suite A, 1st floor, 149 Tottenham Court Road, London, W1T 7DN, United Kingdom.
³ evidENT, Ear Institute, University College London, Royal National Throat Nose and Ear Hospital, 330 Gray's Inn Road, London, WC1X 8DA, United Kingdom; National Institute of Health Research (NIHR) University College London Hospitals (UCLH) Biomedical Research Centre, Maple House, Suite A, 1st floor, 149 Tottenham Court Road, London, W1T 7DN, United Kingdom; NIHR Collaboration for Leadership in Applied Health Research and Care (CLAHRC) North Thames, London, United Kingdom.
⁴ evidENT, Ear Institute, University College London, Royal National Throat Nose and Ear Hospital, 330 Gray's Inn Road, London, WC1X 8DA, United Kingdom; National Institute of Health Research (NIHR) University College London Hospitals (UCLH) Biomedical Research Centre, Maple House, Suite A, 1st floor, 149 Tottenham Court Road, London, W1T 7DN, United Kingdom; Division of Otolaryngology - Head and neck Surgery, University of British Columbia, 2775 Laurel Street, 11th Floor, Vancouver, BCV5Z 1M9, Canada.

PMID: 31319285
DOI: 10.1016/j.heares.2019.07.003

Abstract

Novel hearing therapeutics are rapidly progressing along the innovation pathway and into the clinical trial domain. Because these trials are new to the hearing community, they come with challenges in terms of trial design, regulation and delivery. In this paper, we address the key scientific and operational issues and outline the opportunities for interdisciplinary and international collaboration these trials offer. Vital to the future successful implementation of these therapeutics is to evaluate their potential for adoption into healthcare systems, including consideration of their health economic value. This requires early engagement with all stakeholder groups along the hearing innovation pathway.

Keywords: Aetiology; Clinical trial; Diagnosis; Genetics; Hearing; Therapeutics.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Auditory Perception* / genetics
Clinical Trials as Topic*
Diffusion of Innovation
Genetic Therapy*
Hearing Loss / genetics
Hearing Loss / physiopathology
Hearing Loss / psychology
Hearing Loss / rehabilitation*
Hearing* / genetics
Humans
Persons with Hearing Disabilities / psychology
Persons with Hearing Disabilities / rehabilitation*
Recovery of Function
Research Design*
Stem Cell Transplantation*
Treatment Outcome

Grants and funding

NIHR-RP-011-045/DH_/Department of Health/United Kingdom