Commentary
Expanded Pediatric Cochlear Implant Candidacy J. Thomas Roland, MD1, and Susan B. Waltzman, PhD1
No sponsorships or competing interests have been disclosed for this article.
Keyword cochlear implants hearing loss in children evidence Received February 7, 2015; revised February 9, 2015; accepted February 9, 2015.
W
hile cochlear implant technology has revolutionized the management of severe to profound bilateral sensorineural hearing loss in children and is the medical treatment of choice for the majority of hearing-impaired children who do not benefit from traditional amplification, the technology remains unavailable to many who might gain significant benefit. The current Food and Drug Administration (FDA)–approved indication is bilateral severe to profound hearing loss with no or limited benefit from hearing aids in children 12 months of age older, making the technology unavailable to children with profiles not falling within FDA-approved guidelines despite proven benefit. While performance outcome data exist for off-label device usage, we need to determine what is necessary and acceptable to allow for more widespread device usage. One example is the implantation of children younger than 12 months, which is routine in many centers when the level of hearing is confirmed and anatomic and medical issues are addressed. One might argue that the earlier the child receives hearing, the less catchup there is to overcome and the faster oral language develops. Both the safety and efficacy of implantation in this group have been reported extensively in the literature. A second issue relates to the documented benefits of binaural hearing most especially during the formative language learning years. To make use of binaural stimulation, many children receive cochlear implants where useful aidable hearing is present on the opposite ear. The results with bimodal hearing are impressive as amplified residual hearing has been shown to combine well with electric hearing provided by the cochlear implant in the opposite ear. Last, single-sided deafness cochlear implantation (SSD CI) is now being offered as an option when children are candidates for an implant in one ear with the second ear having normal or near-normal hearing. Some early indications show that the
Otolaryngology– Head and Neck Surgery 2015, Vol. 152(4) 592–593 Ó American Academy of Otolaryngology—Head and Neck Surgery Foundation 2015 Reprints and permission: sagepub.com/journalsPermissions.nav DOI: 10.1177/0194599815575276 http://otojournal.org
plastic auditory system seems to be able to combine the different signals and allows for the fusing and integration of information; however, several important questions with SSD CI exist. At what duration of deafness is the neural gate closed and SSD CI will not be of benefit? Is it ethical and logical to implant a well-functioning and well-integrated SSD child at age 5 or 7 years? Since many children and adults with SSD adapt well, what is the basis and level of evidence needed for SSD CI to become mainstream? FDA indications and guidelines are just that, indications and guidelines, and not law. Reimbursement often follows these indications, and many third-party payers refuse to cover costs associated with off-label use of approved devices. Providers and institutions are at some financial risk of having to refund reimbursement payments, occasionally with interest and penalties. FDA indications are changed when manufacturers submit postmarket amendment of indication requests, accompanied by good, reliable outcomes and safety data often as a result of clinical trials to the FDA. Unfortunately, clinical trials are expensive and often take years to complete, during which time children are deprived of treatment. There are many examples of health care providers employing off-label use of approved medications and devices in medicine. One example is intratympanic injection of gentamycin, an ototoxic aminoglycoside antibiotic, which is commonly used to treated vertigo in recalcitrant Me´nie`re’s disease. Physicians suggest off-label therapies when they feel there are no other approved alternatives available or when the positive results of the therapy exceed currently available treatments. It is very important for medical/surgical teams who employ off-label use of cochlear implants to collect and report outcomes so that new indications can be changed and reimbursement unchallenged. It is also equally important for manufacturers of pharmaceuticals and devices to use their resources to submit available data to the FDA to facilitate new indications. Several CI centers, including ours, are actively involved in many of these projects, and emerging safety and efficacy
1 NYU Langone Medical Center, Otolaryngology, New York, New York, USA
Corresponding Author: J. Thomas Roland, MD, NYU Langone Medical Center, Otolaryngology, 550 First Ave, Suite 7Q, New York, NY 10016, USA. Email: [email protected]
Downloaded from oto.sagepub.com at GEORGETOWN UNIV MED CTR on May 25, 2015
Roland and Waltzman
593
data suggest that implanting appropriate candidates with bilateral cochlear implants younger than 12 months provides excellent results. Similarly, results in children with greater amounts of residual hearing have been impressive. In addition, SSD CI in young children with short durations of deafness has been shown to be safe, and data are emerging regarding efficacy. Rigorous data collection coupled with accurate reporting of results will hopefully lead to the expansion of guidelines, bringing more widespread usage and benefits to appropriate pediatric candidates. To facilitate data acceptance, these studies should be initially undertaken and/or led by centers with clinical and research teams that possess clinical trial experience and track records of peerreviewed publications. Good-quality, well-conducted studies
are imperative if we are to expand accepted therapies to those in need. Rigorous data collection can result in irrefutable evidence that will assist our regulatory agencies in changing indications and recommendations. Author Contributions J. Thomas Roland, wrote the commentary; Susan B. Waltzman, edited the commentary.
Disclosures Competing interests: None. Sponsorships: None. Funding source: None.
Downloaded from oto.sagepub.com at GEORGETOWN UNIV MED CTR on May 25, 2015
Expanded Pediatric Cochlear Implant Candidacy J. Thomas Roland, MD1, and Susan B. Waltzman, PhD1
No sponsorships or competing interests have been disclosed for this article.
Keyword cochlear implants hearing loss in children evidence Received February 7, 2015; revised February 9, 2015; accepted February 9, 2015.
W
hile cochlear implant technology has revolutionized the management of severe to profound bilateral sensorineural hearing loss in children and is the medical treatment of choice for the majority of hearing-impaired children who do not benefit from traditional amplification, the technology remains unavailable to many who might gain significant benefit. The current Food and Drug Administration (FDA)–approved indication is bilateral severe to profound hearing loss with no or limited benefit from hearing aids in children 12 months of age older, making the technology unavailable to children with profiles not falling within FDA-approved guidelines despite proven benefit. While performance outcome data exist for off-label device usage, we need to determine what is necessary and acceptable to allow for more widespread device usage. One example is the implantation of children younger than 12 months, which is routine in many centers when the level of hearing is confirmed and anatomic and medical issues are addressed. One might argue that the earlier the child receives hearing, the less catchup there is to overcome and the faster oral language develops. Both the safety and efficacy of implantation in this group have been reported extensively in the literature. A second issue relates to the documented benefits of binaural hearing most especially during the formative language learning years. To make use of binaural stimulation, many children receive cochlear implants where useful aidable hearing is present on the opposite ear. The results with bimodal hearing are impressive as amplified residual hearing has been shown to combine well with electric hearing provided by the cochlear implant in the opposite ear. Last, single-sided deafness cochlear implantation (SSD CI) is now being offered as an option when children are candidates for an implant in one ear with the second ear having normal or near-normal hearing. Some early indications show that the
Otolaryngology– Head and Neck Surgery 2015, Vol. 152(4) 592–593 Ó American Academy of Otolaryngology—Head and Neck Surgery Foundation 2015 Reprints and permission: sagepub.com/journalsPermissions.nav DOI: 10.1177/0194599815575276 http://otojournal.org
plastic auditory system seems to be able to combine the different signals and allows for the fusing and integration of information; however, several important questions with SSD CI exist. At what duration of deafness is the neural gate closed and SSD CI will not be of benefit? Is it ethical and logical to implant a well-functioning and well-integrated SSD child at age 5 or 7 years? Since many children and adults with SSD adapt well, what is the basis and level of evidence needed for SSD CI to become mainstream? FDA indications and guidelines are just that, indications and guidelines, and not law. Reimbursement often follows these indications, and many third-party payers refuse to cover costs associated with off-label use of approved devices. Providers and institutions are at some financial risk of having to refund reimbursement payments, occasionally with interest and penalties. FDA indications are changed when manufacturers submit postmarket amendment of indication requests, accompanied by good, reliable outcomes and safety data often as a result of clinical trials to the FDA. Unfortunately, clinical trials are expensive and often take years to complete, during which time children are deprived of treatment. There are many examples of health care providers employing off-label use of approved medications and devices in medicine. One example is intratympanic injection of gentamycin, an ototoxic aminoglycoside antibiotic, which is commonly used to treated vertigo in recalcitrant Me´nie`re’s disease. Physicians suggest off-label therapies when they feel there are no other approved alternatives available or when the positive results of the therapy exceed currently available treatments. It is very important for medical/surgical teams who employ off-label use of cochlear implants to collect and report outcomes so that new indications can be changed and reimbursement unchallenged. It is also equally important for manufacturers of pharmaceuticals and devices to use their resources to submit available data to the FDA to facilitate new indications. Several CI centers, including ours, are actively involved in many of these projects, and emerging safety and efficacy
1 NYU Langone Medical Center, Otolaryngology, New York, New York, USA
Corresponding Author: J. Thomas Roland, MD, NYU Langone Medical Center, Otolaryngology, 550 First Ave, Suite 7Q, New York, NY 10016, USA. Email: [email protected]
Downloaded from oto.sagepub.com at GEORGETOWN UNIV MED CTR on May 25, 2015
Roland and Waltzman
593
data suggest that implanting appropriate candidates with bilateral cochlear implants younger than 12 months provides excellent results. Similarly, results in children with greater amounts of residual hearing have been impressive. In addition, SSD CI in young children with short durations of deafness has been shown to be safe, and data are emerging regarding efficacy. Rigorous data collection coupled with accurate reporting of results will hopefully lead to the expansion of guidelines, bringing more widespread usage and benefits to appropriate pediatric candidates. To facilitate data acceptance, these studies should be initially undertaken and/or led by centers with clinical and research teams that possess clinical trial experience and track records of peerreviewed publications. Good-quality, well-conducted studies
are imperative if we are to expand accepted therapies to those in need. Rigorous data collection can result in irrefutable evidence that will assist our regulatory agencies in changing indications and recommendations. Author Contributions J. Thomas Roland, wrote the commentary; Susan B. Waltzman, edited the commentary.
Disclosures Competing interests: None. Sponsorships: None. Funding source: None.
Downloaded from oto.sagepub.com at GEORGETOWN UNIV MED CTR on May 25, 2015
