HEALTH STATE UTILITIES ASSOCIATED WITH OTOFERLIN-RELATED HEARING LOSS
Author(s)
Louis S. Matza, PhD1, Timothy A. Howell, MA2, Nick C. Patel, PharmD, PhD3, Peter Quon, MPH4, Paul Sommer, MBA5, Ivette Cejas, PhD6, Donald M. Goldberg, PhD7, Jameel Muzaffar, MBBS8, Chad Ruffin, MD9.
1Senior Research Leader, Thermo Fisher Scientific, Bethesda, MD, USA, 2Thermo Fisher Scientific, Wilmington, NC, USA, 3Regeneron, Katy, TX, USA, 4Regeneron, Lake Success, NY, USA, 5Regeneron, Tarrytown, NY, USA, 6Children's Hearing Program, University of Miami, Miami, FL, USA, 7Cleveland Clinic, Cleveland, OH, USA, 8University Hospitals Birmingham NHS Foundation Trust, Birmingham, United Kingdom, 9Chad Ruffin, MD PLLC, Seattle, WA, USA.
1Senior Research Leader, Thermo Fisher Scientific, Bethesda, MD, USA, 2Thermo Fisher Scientific, Wilmington, NC, USA, 3Regeneron, Katy, TX, USA, 4Regeneron, Lake Success, NY, USA, 5Regeneron, Tarrytown, NY, USA, 6Children's Hearing Program, University of Miami, Miami, FL, USA, 7Cleveland Clinic, Cleveland, OH, USA, 8University Hospitals Birmingham NHS Foundation Trust, Birmingham, United Kingdom, 9Chad Ruffin, MD PLLC, Seattle, WA, USA.
OBJECTIVES: Otoferlin-related hearing loss (HL) results from sequence variations in the OTOF gene. Available treatment includes cochlear implantation, and in the US, a gene therapy to restore otoferlin production and auditory signalling was recently granted accelerated approval based on improvement of hearing sensitivity. Cost-utility analyses (CUAs) examining the value of new treatments require health state utilities as inputs. The purpose of this study was to estimate utilities associated with varying degrees of congenital, non-syndromic, monogenic sensorineural HL.
METHODS: Two sets of four health state vignettes (child and adult) depicting varying degrees of HL with associated assistive devices (i.e., hearing aids, cochlear implants) were developed based on published literature, clinician interviews, and patient interviews. Health states were valued in time trade-off interviews with general population respondents in the UK (London and Edinburgh). Participants completed the exercise separately with the adult and child health states.
RESULTS: A total of 232 participants completed interviews (52.2% female; mean age = 47.3 years). Mean (SD) utilities for adult health states were 0.97 (0.05) for the cured state (successful gene therapy, no remaining HL), 0.93 (0.09) for mild HL with hearing aid, 0.89 (0.13) for moderate/moderately-severe HL with hearing aid, and 0.83 (0.18) for severe/profound HL with cochlear implant. Mean (SD) utilities for child health states were 0.98 (0.03) for cured state, 0.94 (0.06) for mild HL with hearing aid, 0.92 (0.09) for moderate/moderately-severe HL with hearing aid, and 0.89 (0.11) for severe/profound HL with cochlear implant.
CONCLUSIONS: Lower utilities were associated with health states that described higher degrees of HL. As seen in previous studies estimating both adult and child utilities for corresponding health states, mean child utilities were higher than mean adult utilities. Utilities from this study may be useful in models examining cost-effectiveness of gene therapies to treat congenital, non-syndromic, monogenic sensorineural HL.
METHODS: Two sets of four health state vignettes (child and adult) depicting varying degrees of HL with associated assistive devices (i.e., hearing aids, cochlear implants) were developed based on published literature, clinician interviews, and patient interviews. Health states were valued in time trade-off interviews with general population respondents in the UK (London and Edinburgh). Participants completed the exercise separately with the adult and child health states.
RESULTS: A total of 232 participants completed interviews (52.2% female; mean age = 47.3 years). Mean (SD) utilities for adult health states were 0.97 (0.05) for the cured state (successful gene therapy, no remaining HL), 0.93 (0.09) for mild HL with hearing aid, 0.89 (0.13) for moderate/moderately-severe HL with hearing aid, and 0.83 (0.18) for severe/profound HL with cochlear implant. Mean (SD) utilities for child health states were 0.98 (0.03) for cured state, 0.94 (0.06) for mild HL with hearing aid, 0.92 (0.09) for moderate/moderately-severe HL with hearing aid, and 0.89 (0.11) for severe/profound HL with cochlear implant.
CONCLUSIONS: Lower utilities were associated with health states that described higher degrees of HL. As seen in previous studies estimating both adult and child utilities for corresponding health states, mean child utilities were higher than mean adult utilities. Utilities from this study may be useful in models examining cost-effectiveness of gene therapies to treat congenital, non-syndromic, monogenic sensorineural HL.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR9
Topic
Patient-Centered Research
Topic Subcategory
Health State Utilities
Disease
Genetic, Regenerative & Curative Therapies, No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases