Costs, Healthcare Resource Utilization, and Patient Characteristics Associated with CDKL5 Deficiency Disorder: A Retrospective Analysis of the US Closed Claims Database MarketScan
Jaya Khushalani1, Bersabeh Sile1, Jenna Roberts1, Anna Kuba1, Marcus Brunnert1, Sophia E. Marsh1, Melinda Martin1, Rajsekar Rajaraman2, Scott Demarest3
1UCB, 2UCLA Mattel Children’s Hospital, 3Children’s Hospital Colorado, Anschutz Medical Campus
Objective:
To describe costs, healthcare resource utilization (HCRU), and patient characteristics associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in the MarketScan database.
Background:
CDD is an ultra-rare X-linked developmental and epileptic encephalopathy caused by pathogenic variants in the CDKL5 gene. Literature on cost and HCRU in CDD is lacking.
Design/Methods:
In this retrospective study, we assessed patient characteristics, HCRU, and costs associated with CDD in patients included in MarketScan within the patient selection period (Jul-01-2020–Mar-31-2024). Eligible patients had ≥2 CDD claims (ICD-10, G40.42) ≥1 month apart, and ≥6 months continuous enrollment pre first recorded CDD diagnosis. Costs were adjusted to 2024 values.
Results:
Overall, 56 patients with CDD met the eligibility criteria; 77% were female, 75% were aged <18 years [median age; 11.5 years]; 61%, 37%, and 2% of patients, respectively, were insured via Medicaid, Commercial, and Medicare. Most patients had a prior seizure-related diagnosis: 48% had a claim for Rett syndrome, 27% for Lennox-Gastaut syndrome, and 25% for infantile spasms prior to their CDD diagnosis. HCRU burden among CDD patients was high; 17% required hospitalization within 6 months post CDD diagnosis, 67% of which were seizure-related hospitalizations. Mean (SD) duration of seizure-related hospitalizations was 7 (8) days. Median total costs [ER+hospitalizations+outpatient+pharmacy] incurred per patient was $25,861, with outpatient costs being the main contributor (median, $11,009). Outpatient costs contributed 60% to the total costs, with hospitalization contributing 15%, ER 1%, and pharmacy 24%. Of pharmacy costs, 92% were for an anti-seizure medication claim.
Conclusions:
Costs for patients with CDD were higher than previously published estimates for epilepsy patients, reflecting the high burden of CDD for patients, payers, and health systems. These data suggest there is an urgent unmet need for effective seizure control for CDD patients, given the high cost and high HCRU burden associated with the disease.
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