Zhao Zhao (pseudonym) is a 12 year old child. On a winter morning in December 2020, his parents took him to a hospital in Shanghai. Dr. Li, the coffee spots on Zhao Zhao's body have been around for a long time. At that time, he thought it was a degenerative birthmark. Now that the child is 12 years old, the coffee spots have not disappeared, but instead the bubbles have grown even more severe. In summer, he dares not wear shorts or short sleeves, and the child is very insecure, "Zhao Zhao's mother said.

The so-called bubble disease refers to scattered tumors on the skin of Zhao Zhao, resembling "bubbles" of varying sizes. This is called a type 1 symptom of neurofibromatosis, and people affectionately refer to this type of children as "bubble babies"

Can bubble disease be cured? "Zhao Zhao's mother asked a question that no one could answer as soon as she spoke. This is a chronic syndrome in the child, with bumps on the surface that look terrible. However, don't worry, there are treatment precedents internationally. "Dr. Li comforted Zhao Zhao's mother. Zhao Zhao's diagnosis was not too late, but it may lead to complications such as learning difficulties, spinal torsion, hypertension, etc. The child is still young, so it is recommended to use an "orphan medicine" treatment instead of surgery As a doctor for neurofibromatosis, Dr. Li plans to treat it conservatively.

Orphan drugs, also known as rare disease drugs, have limited market demand and high research and development costs, and pharmaceutical companies are unwilling to invest in research and development. The research and development of orphan drugs in China is still in a blank, relying mainly on imports.

After multidisciplinary consultations, we feel that there is no other effective treatment method. Attempting smectinib is currently the only possible effective method, and it is the first drug to be granted the qualification to treat orphan neurofibromatosis by the US FDA. However, it has not yet been listed in China, and our hospital does not have it. It is not available in Shanghai, only in foreign countries, "said Dr. Li.

I have heard in the 'Bubble Home' group that many people are purchasing on behalf of the United States, "said Zhao Zhao's mother. It's too late to purchase on behalf of others. Treatment takes one or two days and the tumor is growing rapidly. "Dr. Li was also very anxious.

Your side is a medical special zone, a green channel, first try, international triple synchronization, help find the "life-saving drug" - smectinib... "A pharmacy related person contacted a friend from the pilot area of Boao Lecheng in Hainan.

Smetinib? There's a pile of tininib drugs here, I'll check... "said a friend from Lecheng who was going to the drug store to search.

As early as October 2020, Boao Super Hospital submitted China's first application for "Simetinib imported clinically urgently needed drugs". Due to the nine national policies of three synchronizations and four concessions, the Hainan Food and Drug Administration quickly reviewed and approved the application. This is currently the only "green fast track for imported drugs" in China

According to the policy, patients can not only apply for the use of smectinib in the Lecheng pilot area, but also apply for medication to be taken out of the park. Boao Super Hospital submitted a medication application as soon as possible, and departments such as Hainan Provincial Drug Administration, Health Commission, Haikou Customs, and Lecheng Management Bureau worked together to ensure that Bubble Baby can use the medication as soon as it arrives at Boao Super Hospital, allowing the child's condition to be well controlled.

Shanghai Boao, a journey of two thousand miles. Thanks to the special policy granted by the government to Lecheng Pilot Zone, Bubble Babies can access imported life-saving drugs without leaving the country, all of which are due to the four words "doctors are kind-hearted".