CPPCC representative lights up hope for patients suffering from rare diseases

“Every life is precious and every patient needs meticulous caring,” said nurse Du Liqun from Nanning, southwest China’s Guangxi Zhuang autonomous region, after learning a medicine that treats spinal muscular atrophy (SMA) entered the country’s new medical insurance catalogue in December last year.

Chinese national health insurance negotiator helped cut down the bidding price of the medicine from around 700,000 yuan ($110,847) per dose to 33,000 yuan.

Du, who works as the head nurse at the HIV department of the Fourth Hospital of Nanning, is a member of the National Committee of the Chinese People’s Political Consultative Conference (CPPCC). How to better ensure the right to life and health of patients suffering from rare diseases has been a focus of her work over the past years.

“Sparse clinical cases and lack of experiences of treatment result in high frequency of misdiagnoses, missed diagnoses and expensive medication of rare disease patients,” Du introduced, and that’s why she drafted a proposal on further enhancing medical security for rare disease patients based on her visits to primary-level medical workers, as well as patients and their families.

Surveys found that the society still doesn’t have inadequate understanding of rare diseases, Du told People’s Daily, explaining that relevant screenings can reduce the occurrence of rare diseases and their long-term impacts on people’s health, as around 80 percent of rare diseases are inherited.

She said prevention measures shall be taken as a primary approach to controlling rare diseases, which should be performed in such links as premarital check-up, antenatal care and neonatal screening.

“To include more medicines which treat rare diseases in the national insurance catalogue and to lower treatment expenses are also vital for the promotion of rare disease control.” That’s what Du wrote on her notebook after visiting a large number of patients suffering from rare diseases.

“I met a six-year-old child suffering from a rare disease earlier this year, whose mother told me that the price of a medicine for treating the disease has dropped from 700,000 yuan per shot to 33,000 yuan. She said it brought hope to her family, and also smiles on the face of her child,” Du noted.

Du learned that there were only two patients taking this medicine at the Maternity and Child Health Care of Guangxi Zhuang Autonomous Region, and the number rose to more than 20 after the price reduction. There are more patients registering for it, she said.

To improve the multi-level medical security system for rare disease patients will help relieve the patients’ economic burden and improve their life quality, Du said.

At this year’s annual session of the CPPCC, Du has actively shared her research and thinking. “In recent years, relevant parties have gradually raised their attention on major and frequent diseases, and I hope there will be more caring for patients suffering from rare diseases,” she said.