Healthcare Resource Use of Pediatric and Adult Patients With Seizures Associated With Dravet Syndrome, Lennox-Gastaut Syndrome, or Tuberous Sclerosis Complex in Japan
Author(s)
Michael LoPresti, PhD1, Sooyeol Lim, MSc, MBA1, Shun Takagi, BA (Econ)1, Timothy Mann, MSc2, Javier Sabater, PharmD, MSc2, Jamshaed Siddiqui, MMath2.
1Value & Access Department, INTAGE Healthcare Inc., Tokyo, Japan, 2Jazz Pharmaceuticals, UK Ltd., London, United Kingdom.
1Value & Access Department, INTAGE Healthcare Inc., Tokyo, Japan, 2Jazz Pharmaceuticals, UK Ltd., London, United Kingdom.
OBJECTIVES: To evaluate the healthcare resource use (HCRU) of pediatric and adult patients experiencing seizures associated with Dravet syndrome (DS), Lennox-Gastaut syndrome (LGS) or tuberous sclerosis complex (TSC) in Japan, based on insights from clinical expert interviews.
METHODS: Interviews were conducted in December 2024 with clinical experts in Japan experienced in treating patients with the target conditions. Interviews were blinded, lasted 60 minutes each, and were conducted via telephone and/or video conference. The data collected represented physicians’ perception of HCRU rather than patient-level data. Results for each indication, stratified by pediatrics (2-17 years) and adults (≥18 years), were reviewed and summarized after interview completion.
RESULTS: A total of four interviews were conducted for DS, four for LGS, and six for TSC with six experts (three pediatric neurologists and three neurologists). Across all indications, the average number of hospitalizations and emergency room visits per year were reported as increasing with seizure frequency (range) in both pediatric (respectively, DS, 0-8 and 0-24; LGS, 0-2 and 0-4; TSC, 0-8 and 0.4-6.3) and adult patients (respectively, DS, 0-3 and 0-12; LGS, 0-2 and 0-4; TSC, 0-8 and 0.3-5.3). The average number of rescue medications used per year was also reported as increasing with increasing seizure frequency across all indications in both pediatric and adult patients. HCRU including annual outpatient visits, hospitalizations, rescue medication use, emergency room visits, and intensive care unit admissions were slightly lower in patients with TSC experiencing focal seizures with impairment versus generalized seizures for both pediatric and adult patients.
CONCLUSIONS: A trend of increasing HCRU was reported across all indications as seizure frequency increased. HCRU was marginally lower in adult patients compared with pediatric patients, and slightly lower in TSC patients with focal seizures with impairment than those with generalized seizures.
METHODS: Interviews were conducted in December 2024 with clinical experts in Japan experienced in treating patients with the target conditions. Interviews were blinded, lasted 60 minutes each, and were conducted via telephone and/or video conference. The data collected represented physicians’ perception of HCRU rather than patient-level data. Results for each indication, stratified by pediatrics (2-17 years) and adults (≥18 years), were reviewed and summarized after interview completion.
RESULTS: A total of four interviews were conducted for DS, four for LGS, and six for TSC with six experts (three pediatric neurologists and three neurologists). Across all indications, the average number of hospitalizations and emergency room visits per year were reported as increasing with seizure frequency (range) in both pediatric (respectively, DS, 0-8 and 0-24; LGS, 0-2 and 0-4; TSC, 0-8 and 0.4-6.3) and adult patients (respectively, DS, 0-3 and 0-12; LGS, 0-2 and 0-4; TSC, 0-8 and 0.3-5.3). The average number of rescue medications used per year was also reported as increasing with increasing seizure frequency across all indications in both pediatric and adult patients. HCRU including annual outpatient visits, hospitalizations, rescue medication use, emergency room visits, and intensive care unit admissions were slightly lower in patients with TSC experiencing focal seizures with impairment versus generalized seizures for both pediatric and adult patients.
CONCLUSIONS: A trend of increasing HCRU was reported across all indications as seizure frequency increased. HCRU was marginally lower in adult patients compared with pediatric patients, and slightly lower in TSC patients with focal seizures with impairment than those with generalized seizures.
Conference/Value in Health Info
2025-09, ISPOR Real-World Evidence Summit 2025, Tokyo, Japan
Value in Health Regional, Volume 49S (September 2025)
Code
RWD266
Topic Subcategory
Health & Insurance Records Systems
Disease
SDC: Neurological Disorders