(232) Real-World Cost-Effectiveness of Adrenocorticotropic Hormone Versus Oral Prednisolone for Children with West Syndrome

Saturday, August 27, 2022

8:00 AM – 6:00 PM CEST

Location: Hall C

Publication Number: 906

Presenting Author(s)

Nagita Devi
– National Institute of Pharmaceutical Education and Research, S.A.S. Nagar, Mohali, Punjab, India

Co-Author(s)

Priyanka Madaan
– Pediatric Neurologist, Division of Pediatric Neurology, Advanced Pediatric Center, Postgraduate Institute of Medical Education and Research (PGIMER), Chandigarh, India, Chandigarh, India
Dipika Bansal, MBBS, MD, DM
– Assistant Professor, National Institute of Pharmaceutical Education and Research (NIPER) S.A.S. Nagar, Punjab, India
Jitendra Kumar Sahu
– Additional Professor, Division of Pediatric Neurology, Advanced Pediatric Center, Postgraduate Institute of Medical Education and Research (PGIMER), Chandigarh, India, Chandigarh, India

Background: Adrenocorticotropic hormone (ACTH) and oral prednisolone are the commonly prescribed first-line treatment options for West syndrome (WS). However, the cost variation between these drugs makes the choice of treatment debatable.

Objectives: To evaluate the patient-level real-world cost-effectiveness of ACTH versus oral prednisolone for children with WS.

Methods: A cost-effectiveness decision tree model was applied from the payer’s perspective to predict the benefit and costs of ACTH and oral prednisolone in children with WS. Model inputs were obtained from real-world parent-reported data at a tertiary care center in North India. The model consisted of two health states either persistent cessation of spasms (within 14 days of treatment onset without any recurrence for ≥ 28 consecutive days) or no sustained cessation of spasms (within the 14 days of the treatment onset). The direct cost of treatment was collected as diagnostic, medications, hospital admissions, traveling, and lodging/ staying cost. Incremental cost-effectiveness ratios (ICERs) for cost per child with spasm cessation were the primary outcome. One-way sensitivity analyses were performed to understand the impact of different variables on the cost-effectiveness model and visualized in the Tornado diagram. Probabilistic sensitivity analyses using Monte Carlo simulation with over 10000 iterations were applied through the uncertainty range for ICER.

Results: Total 93 children (55 for ACTH and 39 for oral prednisolone) with WS were included in the base-case analysis with the mean (SD) age of 10 (3.7) months and males accounted for 73 (78%). Among them, 29/55 (31%) of children from the ACTH group and 13/38 (14%) from the oral prednisolone group achieved complete spasm cessation, p = 0.093). Using the model inputs, ICER for ACTH versus oral steroids and oral prednisolone were ₹ 1,39,447.90 and ₹ 1,06,711.06 per child with complete spasm resolution respectively. This was less than India's total GDP per capita (₹ 1,56,476.66 in 2021), signifying that oral prednisolone was more cost-effective than ACTH. Decision outcomes were robust to the one-way and probabilistic sensitivity analyses.

Conclusions: The study findings provide preliminary insight into the cost-effectiveness of ACTH and oral prednisolone in WS, relevant for developing countries like India. Future studies with a larger sample size are needed in this context.