The use of special approval medicines among pediatric patients in a tertiary care hospital: A reality check

BACKGROUND: Special approval medicines (SAMs) are medicines used with approval from the Director General of Health Malaysia when the therapeutic options within regulatory and formulary boundaries appear unsuitable or ineffective to treat the patients. OBJECTIVES: To examine and characterize the use of SAMs among children in a Malaysian tertiary care hospital. METHODS: The named-patient basis SAM application forms, cover letter, pharmacist review summary and patient monitoring forms available at the Pharmacy Department between 1st January 2019 and 31st December 2020 were reviewed. Unprocessed, unapproved and stock-basis applications were excluded. The outcome measures were categories, scope, off-label use and cost of SAM. Per-patient data were analyzed descriptively. RESULTS: Overall, 1010 patients (mean age of 8.7 ± 5.6 years) were involved in 328 SAMs applications. The most common SAMs pharmacological groups were nervous system (n = 371, 36.7%) and antineoplastic and immunomodulating agents (n = 332, 32.9%). Top three SAMs were melatonin (11.5%), scopolamine (7.6%) and cholecalciferol (7.1%). A total of 837 (82.9%) and 513 (50.8%) patients were involved in the SAMs applications for non-formulary and unregistered medicines, respectively. Unregistered, non-formulary medicines were applied for 47.3% (n = 478) of the patients. The majority of the scope for SAMs (64.7%) were to substitute the available alternatives in the national formulary which were ineffective or sub-optimal for the patients. Among the 262 patients with repeat applications, 93.8% reported disease or symptom improvement while 1.9% experienced side effects. Up to 17% of SAMs analyzed in this study were used for off-label indications. The total cost of the SAMs was RM8,748,358.38 (USD 2,090,418.86). CONCLUSION: The use of SAMs among children in this hospital involved unregistered, non-formulary medicines used to substitute the available alternatives in the formulary. A concerted effort is warranted in exploring supplementary mechanisms to enhance the medicine registration process and formulary system towards facilitating enhanced provision of treatment for children.