Presentation (CTI)

OBJECTIVES: To estimate the annual cost per patient associated with orphan drug treatment, disaggregated by therapeutic area, from the perspective of the public health sector in Mexico.
METHODS: Orphan drugs available to the public health sector in Mexico up to November 2025 and their dosage registered in the National Compendium of Health Supplies (CNIS) were identified. They were classified according to the specialty of treatment and the organ affected in the case of oncological diseases. The information on the cost per pharmaceutical presentation was obtained from the transparency portals of the Government of Mexico. The annual cost per patient for each drug was estimated using the microcosting methodology based on the reported dosage, without considering drug waste, and a descriptive statistical analysis was performed. The costs and results of the analysis are expressed in Mexican pesos (MXN).
RESULTS: We identified 114 presentations of orphan drugs available in the public health sector of Mexico, indicated for 56 diseases in different stages (120 indications); the cost of 103 presentations (90.35%) was obtained. The average annual cost per patient of orphan drug use is $2,986,214. The average cost in rare congenital diseases is $3,480,571, while in low-prevalence oncological diseases it is $2,228,731. Orphan drugs indicated for neurological diseases represent the group with the highest average annual cost: MXN$ 6,101,770. The estimated cost range is $5,014 to $35,829,557, with a median of $1,643,625.
CONCLUSIONS: The cost associated with treatment with orphan drugs in Mexico's public sector represents a high economic burden, which means an additional barrier to access to these treatments. Alternative financing models are needed to ensure the treatment of patients with low-prevalence diseases, which would allow equitable access to health services.