The epidemiology and healthcare burden of rare diseases requiring hospitalisation among adult patients in Langkawi, Malaysia: Insights from a pilot study.

In Malaysia, rare diseases affect fewer than 1 in 4,000 people. As of 2020, 491 rare diseases have been recorded in Malaysia, but with limited epidemiological data. As the first study in Malaysia, this retrospective cohort study examined the epidemiology and admission-related healthcare costs for adult rare disease patients in Langkawi. Among the 38 patients, rheumatological rare diseases topped the list (39.5%). The annual admission rate for rare diseases was 0.9%. Langkawi patients had lengthy hospital stays (9.7 days) and a 7.9% mortality rate. 23.7% of patients defaulted to follow-up, and 7.9% were referred to a tertiary hospital due to inadequate equipment or speciality care. Admission costs were Malaysian Ringgits (MYR) 244,598.63 (~US Dollars (USD) 51,280), with 80.2% from medication. The average healthcare resource utilisation was MYR 6,436.81/ patient/year (~USD 1,350/patient/year).

Cost-effectiveness analysis of granisetron-based versus standard antiemetic regimens in low-emetogenic chemotherapy: a hospital-based perspective from Malaysia.

BACKGROUND: In a prospective cohort study of antiemetic therapy conducted in Malaysia, a total of 94 patients received low emetogenic chemotherapy (LEC) with or without granisetron injections as the primary prophylaxis for chemotherapy-induced nausea and vomiting (CINV). This study is a retrospective cost analysis of two antiemetic regimens from the payer perspective. MATERIALS AND METHODS: This cost evaluation refers to 2011, the year in which the observation was conducted. Direct costs incurred by hospitals including the drug acquisition, materials and time spent for clinical activities from prescribing to dispensing of home medications were evaluated (MYR 1=$0.32 USD). As reported to be significantly different between two regimens (96.1% vs 81.0%; p=0.017), the complete response rate of acute emesis which was defined as a patient successfully treated without any emesis episode within 24 hours after LEC was used as the main indicator for effectiveness. RESULTS: Antiemetic drug acquisition cost per patient was 40.7 times higher for the granisetron-based regimen than for the standard regimen (MYR 64.3 vs 1.58). When both the costs for materials and clinical activities were included, the total cost per patient was 8.68 times higher for the granisetron-based regimen (MYR 73.5 vs 8.47). Considering the complete response rates, the mean cost per successfully treated patient in granisetron group was 7.31 times higher (MYR 76.5 vs 10.5). The incremental cost-effectiveness ratio (ICER) with granisetron-based regimen, relative to the standard regimen, was MYR 430.7. It was found to be most sensitive to the change of antiemetic effects of granisetron-based regimen. CONCLUSIONS: While providing a better efficacy in acute emesis control, the low incidence of acute emesis and high ICER makes use of granisetron as primary prophylaxis in LEC controversial.