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Regulatory and Funding Strategies to Develop a Safety Study of an Auditory Brainstem Implant in Young Children Who Are Deaf

  • Special Section on Pediatrics: Original Research
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Abstract

Background

In the US, a child born deaf with abnormal inner ear anatomy and for whom a cochlear implant fails to provide benefit has no approved surgical treatment options. The auditory brainstem implant (ABI) was developed in the US for patients with Neurofibromatosis 2. A European surgical team has advanced ABI use in young children, with promising outcomes. Clinical teams face a number of challenges, from regulatory issues to funding. The strategic decisions leading to the safety and early efficacy protocol of the ABI for young children with congenital deafness are described.

Methods

A sponsor-investigator pre–Investigational Device Exemption (IDE) was significantly altered in discussion with the Food and Drug Administration (FDA), followed by approval of a revised protocol. A critical step involved securing external funding through an NIH U01 clinical trial grant, and enrollment began in 2014.

Early Results

The protocol focused on strict inclusion criteria, clear stopping rules, and rigorous interim safety data review before proceeding with enrollment. To date, 4 of the 7 enrolled subjects met criteria for implantation. A single expected serious adverse event occurred, which resolved completely without sequela.

Conclusions

Promising innovative treatments face a number of obstacles along the pathway to full commercialization. A strategy that included early conversations with the FDA and the device manufacturer, and successfully obtaining external funding, resulted in an approved IDE protocol. Early results indicated that the risks, though not minimal, can be successfully mitigated. These young children appear to benefit audiologically from the ABI.

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Author information

Authors and Affiliations

  1. USC Tina and Rick Caruso Department of Otolaryngology-Head and Neck Surgery, Keck School of Medicine of University of Southern California, Los Angeles, CA, USA

    Laurel M. Fisher PhD, Laurie S. Eisenberg PhD & Robert V. Shannon PhD

  2. Billy and Audrey Wilder Endowed Chair in Neurosurgery; Division Chief, Neurosurgery; Director, Neurosurgery Fellowship Program, Children’s Hospital Los Angeles, Los Angeles, CA, USA

    Mark Krieger MD

  3. Huntington Medical Research Institutes, Pasadena, CA, USA

    Eric P. Wilkinson MD

  4. House Clinic, Los Angeles, CA, USA

    Eric P. Wilkinson MD

Authors
  1. Laurel M. Fisher PhD
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  2. Laurie S. Eisenberg PhD
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  3. Mark Krieger MD
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  4. Eric P. Wilkinson MD
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  5. Robert V. Shannon PhD
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Consortia

Los Angeles Pediatric ABI Team

Corresponding author

Correspondence to Laurel M. Fisher PhD.

Additional information

Author Note

The members of the Los Angeles Pediatric ABI Team are as follows: Keck School of Medicine of University of Southern California: Laurie S. Eisenberg, PhD, Co-Principal Investigator, Robert V. Shannon, PhD, Co-Investigator, Laurel M. Fisher, PhD., Co-Investigator, Amy S. Martinez, MA, Research Audiologist, Clinical Coordinator, Margaret Winter, MS, Lead ABI Audiologist, Jamie Glater, AuD, ABI Audiologist, Dianne Hammes-Ganguly, MA, Speech Language Pathologist, Debra Schrader, Educational Liaison. Children’s Hospital Los Angeles: Mark Krieger, MD, Chief, Pediatric Neurosurgery. Huntington Medical Research Institutes, House Clinic: Eric P. Wilkinson, MD, Co-Principal Investigator, Marc S. Schwartz, MD, Neurosurgeon

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Fisher, L.M., Eisenberg, L.S., Krieger, M. et al. Regulatory and Funding Strategies to Develop a Safety Study of an Auditory Brainstem Implant in Young Children Who Are Deaf. Ther Innov Regul Sci 49, 659–665 (2015). https://doi.org/10.1177/2168479015599559

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  • DOI: https://doi.org/10.1177/2168479015599559

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