■Wang Zipei
The last day of February every year is International Rare Disease Day. Rare disease prevention and treatment is a global problem and a social problem that we must face up to.
Due to the low prevalence, wide variety and limited clinical research, rare diseases are difficult to diagnose, and they also face difficulties such as few available drugs and high drug prices. At the same time, “rare” is only a relative concept. The absolute number of patients with rare diseases in my country is relatively large, and rare diseases are not far away from us.
From formulating a list of rare diseases, to establishing a national collaborative network for diagnosis and treatment of rare diseases, to including dozens of drugs for rare diseases into the national medical insurance list, my country is on the road to solving the problem of rare disease prevention and treatment. On January 1 this year, Guangdong included all the negotiated drugs during the agreement period into the scope of “dual-channel” managed drugs, including the rare disease drug Nosinagen Sodium, which has attracted widespread attention due to the “700,000 yuan per injection”. Adhering to the supremacy of the people and life, more and more rare disease patients have seen the dawn of hope.
At present, there is still much room for improvement in the prevention and treatment of rare diseases, including paying more attention to the “tertiary prevention” of rare diseases, increasing investment in the research and development of drugs for rare diseases, and improving the consultation and referral mechanism for rare diseases. It is believed that through the joint efforts of the whole society, rare diseases will eventually bid farewell to the history of “orphan diseases”.