Recently, Huashan Hospital received a reply from the State Food and Drug Administration to temporarily import "paretide"(note: a rare disease drug). The drug import process was successfully started and the patient was given the drug yesterday. This is the first time that Shanghai has successfully imported urgently needed clinical drugs that have not been registered and marketed in China in accordance with my country's "Work Plan for the Temporary Import of Drugs in Urgent Clinical Need".

From rapid approval of urgently needed clinical drugs, overseas procurement, convenient customs clearance to standardized use and other processes, Shanghai's "ice-breaking" of the national new policy has collaborated with medical institutions and multiple government departments to provide treatment for rare diseases in the city that do not yet have effective treatment drugs. Or other critically ill patients bring new hope, and the wind vane is of far-reaching significance.

After receiving the first drug injection, the patient is currently in stable condition

In recent years, benefiting from the development of the domestic pharmaceutical industry and the import of foreign drugs, my country's drug demand for common and frequently-occurring diseases has been greatly met, but there are still some urgently needed drugs that have not yet been marketed domestically. In order to further improve drug supply guarantees and meet people's specific urgently needed drug needs, the National Health Commission and the State Food and Drug Administration jointly issued the "Work Plan for the Temporary Import of Drugs in Urgent Clinical Need" in 2022 to provide institutional guarantees for the import application and use management of relevant drugs.

The three vials of "paretide" that Huashan Hospital applied for import this time are the "ice-breaking" exploration of this solution in Shanghai. The patient received his first injection yesterday and is currently in stable condition.

"Pareotide" is used to treat patients with acromegaly who are not suitable for surgery or have not yet been cured, and who are not well controlled with first-generation somatostatin analogues. Acromegaly can cause multiple system and multiple organ complications throughout the body. If not treated, the average life expectancy will be shortened by at least 10 years. In 2022, it will be included in my country's "Second Batch of Rare Diseases Catalogue", saying that these patients are "struggling on the verge of death." It is no exaggeration.

"This 34-year-old patient has been diagnosed with acromegaly for more than three years and has previously undergone surgery and gamma knife treatment." Professor Zhang Chaoyun, deputy director of the Rare Disease Center of Huashan Hospital, told reporters that this rare disease will not only cause a series of problems such as facial changes, diabetes, and high blood pressure, but also cause heart diseases such as heart failure. If hormones do not drop to normal levels, it will endanger life. Unfortunately, the patient's hormone indicators still did not reach normal after being treated with various existing domestic regimens, and he urgently needed "paretide".

Try first and accumulate experience for the import of urgently needed drugs in clinical need

As the National Medical Center for Neurology, Huashan Hospital is the largest diagnosis and treatment center for acromegaly in China. Two to three hundred patients with acromegaly are operated on every year, of which 20% to 30% undergo surgery combined with first-generation Somatostatin analogues. After comprehensive treatment, the condition has not subsided. Due to the fact that "paretide" has not yet been marketed in China, their clinical needs have not been met for a long time.

Can you try a new path for patients? In March this year, Huashan Hospital submitted an application for the import of "parretide." The Shanghai City Health Commission and the Shanghai City Food and Drug Administration took the initiative to contact Huashan Hospital and conduct a detailed interpretation of the national plan. Under the guidance of the Municipal Health Commission and the Food and Drug Administration, Huashan Hospital established a working group headed by Zhao Zhongbo, director and vice president of the hospital's Rare Disease Center, to coordinate the multidisciplinary medical team of the medical department, the pharmacy department, and the "golden pituitary" to quickly formulate a temporary imported drug management system, safety precautions, and risk disposal plans, and established an expert group to apply to the State Food and Drug Administration based on clinical needs.

Thanks to the efforts of all parties, these three life-saving drugs have finally arrived in Shanghai recently. It is worth mentioning that it is not enough to leave Shanghai. Medicine can be used in Shanghai without flying overseas for treatment, which greatly reduces the treatment cost of patients.

The Shanghai City Food and Drug Administration stated that temporary import of urgently needed drugs that have not been registered and marketed in our country is a useful supplement to the current drug registration system. This pilot trial by Huashan Hospital has provided valuable experience for the future import of domestically unmarketed drugs urgently needed clinically in this city. The drug regulatory department will also track the use of the drug and follow up on the application needs of medical institutions to ensure that more people can both use the drug and use it well.