# Specific drugs for rare diseases in a province of eastern China under catalog management: from 2021 to 2023

## TL;DR

This study examines the availability and supply of rare disease drugs in Shandong Province, China, from 2021 to 2023, highlighting drug shortages and regional disparities.

## Contribution

The study provides the first detailed analysis of rare disease drug supply and utilization in a specific Chinese province using catalog data and procurement records.

## Key findings

- Shandong Province has access to 42 and 28 specific rare disease drugs from the first and second national catalogs, respectively.
- Several drugs, including Spesolimab and Selumetinib, face shortages or low delivery rates below 80%.
- Drug availability in Shandong reached 80%, but regional disparities in drug costs were significant, with Jinan leading at 770 million RMB.

## Abstract

China attaches great importance to the prevention and treatment of rare diseases. The government has successively formulated two rare disease catalogs, and approved a variety of rare diseases treatment drugs. However, the actual supply and utilization of these drugs post-marketing remains unclear.

Based on the first and second list of national rare disease catalog in China, this study sort out the specific therapeutic drugs and extract procurement data from the provincial platform over the past 3 years. Subsequently, the drug allocation, shortages, delivery rate, temporal changes, and spatial distribution were analyzed to gain a comprehensive understanding of the local drug supply situation.

In the first catalog of 121 rare diseases, China has listed 54 specific drugs; in the second catalog of 86 rare diseases, 35 specific therapeutic drugs have been identified. Among these drugs, Shandong Province has access to 42 and 28, respectively. Spesolimab, Sodium Phenylbutyrate, Nitisinone and Emapalumab are currently in short supply, and the delivery rate of 16 drugs such as Selumetinib, Sirolimus (tablet), Octreotide, Dimethyl Fumarate and Lanreotide is below 80%. The number of available drugs increased year by year. The allocation of 19 drugs increased significantly, and 19 drugs were newly developed. The overall procurement cost of drugs increased and then decreased, which may be related to national policies. Additionally, there are significant regional disparities in drug cost, with Jinan, the provincial capital, leading at 770 million RMB.

The number of specific drugs for rare diseases has steadily increased, with the drug availability rate in Shandong Province reaching 80%. This indicates a generally high level of accessibility to drugs for rare diseases in China. However, attention should be given to improving the supply capacity for drugs that are in short supply and have a low delivery rate.