The Landscape of Approved Drugs With Pediatric Orphan Indications in Saudi Arabia

OBJECTIVES: This study aims to assess the types of orphan drugs approved and their availability and accessibility to pediatric patients, comparing these aspects with the United States (US) to identify gaps and opportunities for improvement in Saudi Arabia (SA).

METHODS: Data on all drugs approved with orphan designations and authorized for marketing in Saudi Arabia and the United States until June 2024, were derived from regulatory body databases. Additionally, the public prices of drugs with pediatric orphan indications in Saudi Arabia were collected. Data was analyzed by summary descriptive statistics.

RESULTS: As of June 2024, the Saudi Food and Drug Authority (SFDA) has approved 19 drugs with 50 pediatric orphan indications, which is only 10% of the drugs approved with pediatric orphan indications in the US. 18% of the approved orphan pediatric indications in SA were approved for genetic diseases. The median public price was $927.3 for the drug package (IQR = $1,072.4, range $20.6 to $ 10,511.3).

CONCLUSIONS: The study shows significant gaps between Saudi Arabia and the US in terms of pediatric orphan drugs availability and approval rates, with only 10% of approvals and notable affordability concerns. In Saudi Arabia, there is an urgent need for focused initiatives to increase the availability of drugs with orphan indications to children with rare diseases.