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Four, Top10 drug orphan drug identification
According to the statistics of "China Rare Disease Medical Insurance Cities Report", among the top 10 drugs in global sales in 2018, 8 except for Electo and Laitsch have been identified as "orphan drugs" in the United States, and 4 of them are Marketed as an "orphan drug" and gradually expanded for multiple rare disease or non-rare disease indications, and Refumei (lenalidomide), which ranks second in global sales, was approved in the United States for myelodysplastic syndromes and multiple All indications such as myeloma and mantle cell lymphoma are rare diseases and have obtained the status of "orphan drug"
.
Humira, who has been the world's drug king for 9 years, was approved as an "orphan drug" for juvenile rheumatoid arthritis indications in 2008
.
Rituximab, Herceptin, and Avastin, known as the Roche Troika, have been approved for rare disease indications for non-Hodgkin B-cell lymphoma, advanced gastric adenocarcinoma overexpressing HER2, and malignant glioma, respectively
.
Despite the late start, China attaches great importance to the availability of drugs for patients with rare diseases.
In recent years, a series of encouraging policies have been introduced, which greatly increased the enthusiasm of enterprises to develop orphan drugs and accelerated the development of orphan drugs.
The gap between China and developed countries is constantly being narrowed, and the availability of medicines for patients with rare diseases is rapidly increasing
.