Global Top 12 Non-Oncology Rare Disease Pharmaceutical Companies: Market Ranking and Pipeline Overview

According to statistics from the U.S. Food and Drug Administration (FDA), there are more than 7,000 known rare diseases worldwide, accounting for 10% of all human diseases. The global population of patients with rare diseases has exceeded 250 million, with nearly 50% being children. Among the more than 7,000 known rare diseases, fewer than 10% have approved therapeutic drugs or treatment regimens.

In May 2018, five departments, including the National Health Commission, the Ministry of Science and Technology, the Ministry of Industry and Information Technology, the National Medical Products Administration, and the National Administration of Traditional Chinese Medicine, jointly announced the First Batch of the Rare Disease Catalog, which includes 121 rare diseases. According to epidemiological literature and public data estimates, these 121 rare diseases affect approximately 3 million patients in mainland China.

Based on the 121 rare diseases included in the “First Batch of Rare Diseases Catalog” and the rare disease drugs approved by the U.S. FDA for these conditions, IQVIA recently presented a comparison of the market launch status of leading global non-oncology rare disease pharmaceutical companies in the United States and China.