Assessing the "Drug Loss" and "Drug Lag" for Rare Diseases in China: A Comparative Analysis with the United States (2001-2024).

WHY IT MATTERS

If you have a rare disease in China, this research directly impacts whether you can access treatments that may already be approved and available in the United States, potentially identifying barriers your doctor or patient advocacy groups can help address.

Researchers compared how quickly rare disease drugs become available in China versus the United States between 2001 and 2024. The study looked at a problem called 'drug loss' — when medicines are approved in the US but never reach Chinese patients — and 'drug lag' — when there's a long delay before Chinese approval. The findings help the Chinese government understand what's blocking patients from getting treatments that already exist elsewhere in the world.

Assessing the "Drug Loss" and "Drug Lag" for Rare Diseases in China: A Comparative Analysis with the United States (2001-2024). Abstract: Recently, the Chinese government has prioritized enhancing access to rare disease drugs, with the initial objective of addressing the challenge of drugs being "available abroad but not domestically." The United States, a leader in the development of treatments for rare diseases, serves as a benchmark for assessing a nation's progress in this field. This study examined the progression and influencing factors of the availability gap through the application of two principal metrics: "drug loss" and "drug lag" at the indication level. We conducted a retrospective analysis of rare disease indications approved in the United States and China from 2001 to 2024, focusing on diseases listed in China's official Catalogs of Rare Diseases. Our study indicated that "drug loss" in China had continued to Authors: Wang et al. Journal: Clinical pharmacology and therapeutics MeSH: China, United States, Rare Diseases, Humans, Orphan Drug Production, Retrospective Studies, Drug Approval, United States Food and Drug Administration