On December 19, Mr. Huang of Danzhou, Hainan, took his 1-year-old son to Guangzhou for an injection of Nusinersen, a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients. Next year, his child should be able to access this medicine in Hainan at a much reduced price. The world's first therapy to treat SMA, a rare genetic disorder that affects muscle control, was added to the China’s national list of medicines eligible for reimbursement on the 3rd of December, 2021.

Hainan Boao Lecheng International Medical Tourism Pilot Zone (Boao Hope City) has also set up a medical center as a global drug information platform for rare diseases, giving rare disease patients access to innovative medicines from around the world.

CNY 550,000 per dose for SMA treatment

In December 2020, Mr. Huang's son was diagnosed with Spinal Muscular Atrophy (SMA). "We never wanted to give up after the diagnosis," said Mr. Huang. He found out that Nusinersen is a life-saving medicine for his child, but the cost of treatment is high. It costs more than 500,000 yuan per dose, and the patient needs the medication throughout their lifetime.

Most children who have SMA, which results in death in 95 percent of patients up to 18 months of age, can be saved with this treatment at a far more affordable price than previously. Patients who have reached adulthood can also benefit from the therapy.

Mr. Huang, whose child was diagnosed with Spinal Muscular Atrophy (SMA) / WANG HONGXU

Mr. Huang had asked several hospitals in Hainan, but none of them had Nusinersen available. With the recommendation of a friend, he finally took his child to Guangzhou for medical treatment.

"The sooner the child gets the medicine, the better," said Mr. Huang. He has always hoped that the treatment could be reduced in price in the future or would be covered by medical insurance. Before his son’s first course of treatment was complete, Mr. Huang's hopes came true.

Mr. Huang speaking with  Director Pang Qiming about his son's medication. WANG HONGXU

A new hope for financial relief

On December 3, the announcement was made that seven rare disease medications had been added to China’s list of medicines eligible for coverage under the country’s national health insurance. The price of the SMA treatment medicine was reduced to 33,000 yuan per dose and it is now also covered under China’s national medical insurance. Mr. Huang and his wife cried tears of joy when they heard the news, and went around telling everyone.

Mr. Huang forwarded the news to the "SMA Hainan Group". The group had 17 members, and most of them were parents of children with SMA. Everyone was moved and full of hope.

"In Hainan, we have learned that more than 20 children are suffering from SMA and need treatment, and there may be many children who have not yet come to the hospital," said Dr. Pang Qiming, Director of the Neuroscience Department of the Hainan Women's and Children's Medical Center. The disease severely affects the growth of children, and also brings a lifelong financial burden to the family. It is hoped that these children can be treated in time.

Dr. Pang Qiming, Director of the Neuroscience Department of the Hainan Women's and Children's Medical Center, said that children with SMA should be treated as soon as possible.  WANG HONGXU

To help patients with rare diseases, since China’s National Healthcare Security Administration was established in 2018, it has updated the list of medicines covered by national insurance once a year, and medications for rare diseases are also included. Meanwhile, through negotiating access to treatments for rare diseases, prices have been drastically reduced.

Currently, more than 60 rare disease medications have been approved for sale in China, and over 40 of them have been included on the list of medications eligible for coverage under China’s national insurance, with 25 different rare diseases now having treatments available. A total of 7 rare disease medications were successfully reduced in cost in 2021, with the price dropping an average of 65% per medication.

Advantages of treatment in Hainan

Doctor Pang Qiming, Director of the Neuroscience Department of Hainan Women and Children's Medical Center, visited a hospitalized child suffering from a rare disease. WANG HONGXU

For the treatment of rare diseases, Hainan has an advantage. Boao Lecheng Hope City can license and import urgently needed rare disease medications that are available abroad but not in China. At the same time, it can expand the scope of imported health foods and medical equipment for health care, and can import and prescribe special medicated foods  that are available overseas but not in China.

To this end, Boao Lecheng has established a rare disease clinical medicine center and has brought in 50 rare disease medications. Lecheng is expected to become a rare disease treatment center for patients from around China.