Abstract Details

Title Costs, Healthcare Resource Utilization, and Patient Characteristics Associated with CDKL5 Deficiency Disorder: A Retrospective Analysis of the US Closed Claims Database MarketScan

Topic Global Health and Neuroepidemiology

Presentation(s) P5 - Poster Session 5 (11:45 AM-12:45 PM)

Poster/Presentation
Number 20-008

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.

Authors/Disclosures
Jaya S. Khushalani, MD, PhD PRESENTER	Dr. Khushalani has received personal compensation for serving as an employee of UCB. Dr. Khushalani has or had stock in UCB.
Bersabeh B. Sile	Miss Sile has nothing to disclose.
Jenna S. Roberts, PhD	Dr. Roberts has received personal compensation for serving as an employee of UCB Pharma. Dr. Roberts has stock in UCB Pharma.
Anna Kuba	Ms. Kuba has received personal compensation for serving as an employee of UCB.
Marcus Brunnert	Mr. Brunnert has received personal compensation for serving as an employee of UCB Biosciences GmbH. Mr. Brunnert has stock in Amgen.
Sophia E. Marsh, PhD	Dr. Marsh has received personal compensation for serving as an employee of UCB. Dr. Marsh has stock in UCB.
Melinda S. Martin, PhD	Dr. Martin has received personal compensation for serving as an employee of UCB. Dr. Martin has received stock or an ownership interest from UCB.
Rajsekar R. Rajaraman, MD (UCLA Medical Center)	Dr. Rajaraman has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Marinus Pharmacueticals.
Scott T. Demarest, MD (University of Colorado Health Science Center, Child Neurology)	The institution of Dr. Demarest has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. The institution of Dr. Demarest has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Capsida. The institution of Dr. Demarest has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Lundbeck. The institution of Dr. Demarest has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mahzi. The institution of Dr. Demarest has received personal compensation in the range of $500-$4,999 for serving as a Consultant for neurocrine. The institution of Dr. Demarest has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Praxis. The institution of Dr. Demarest has received research support from NIH. Dr. Demarest has a non-compensated relationship as a SMAB with SLC6A1 Connect that is relevant to AAN interests or activities. Dr. Demarest has a non-compensated relationship as a SMAB with Ring 14 USA that is relevant to AAN interests or activities. Dr. Demarest has a non-compensated relationship as a SMAB with FamilieSCN2A that is relevant to AAN interests or activities.

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