Orphan drug research and development progress 5 pictures in seconds

Text | Dr.

The last day of February is the 14th International Rare Disease Day.

The so-called rare disease is a disease with a very low incidence.

Drugs used for the prevention, treatment, and diagnosis of rare diseases are also called "orphan drugs".

1.

In order to improve the accessibility of drugs, since the State Council issued Circular 44 in 2015, my country has accelerated the speed of drug review and approval, especially for orphan drugs.

In order to give priority to improving the review speed of some urgently needed drugs, the State Food and Drug Administration has opened a priority review channel.

(2) FDA application

In recent years, with the rapid improvement of my country's overall pharmaceutical research and development capabilities, Chinese pharmaceutical companies have also made regrettable progress in the research and development of orphan drugs.

2.

According to the "China Rare Disease Medical Security City Report" statistics, in the 10 years before the 1983 "Orphan Drug Act" was introduced in the United States, there were only 10 rare disease drugs on the market, and after 1983, 503 drugs received "orphan drugs" Identified and successfully listed, involving 731 rare disease indications (as of September 2018).

In addition to the United States, the European Union also started early in the development of orphan drugs.

3.

In 2000, the number of indications for adult & child, adult and child orphan drug approved by the European Union were 9, 3 and 2, respectively.

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".

Xiumei, who has been the world's drug king for 9 years, was approved as an "orphan drug" in 2008 for juvenile rheumatoid arthritis indications.

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, my country 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 my country and developed countries is constantly being narrowed, and the availability of medicines for patients with rare diseases is rapidly increasing.