Study On The Safeguard System Of Drug Use For Rare Diseases In Our Country

It is one of the hot topics in the world to improve the survival rate and quality of life of rare disease groups,establish a sound medication guarantee system for rare diseases,and realize the equality of medical resources for citizens.Healthy people is an important prerequisite and guarantee for national prosperity and rejuvenation,and an important symbol of a happy life for the people.Due to the difficulty,high cost and small market size of rare disease drug research and development,the commercial rate of return is highly uncertain.Therefore,pharmaceutical enterprises lack motivation and enthusiasm for research and development.Some patients with rare diseases are faced with difficult and expensive drugs.How to overcome the passive situation of "no medicine available" or "no medicine available" for patients with rare diseases through system construction,and improve the level of medication guarantee for rare diseases is not only a common public health governance issue,but also relates to the stability of the whole society and affects the happiness and satisfaction of people’s life.This article mainly uses the literature research method,through searching a large number of laws and regulations related to rare disease drugs,as a basis to analyze the current situation of the drug protection system for rare diseases in China.Literature search engines such as China National Knowledge Network,Wanfang Data Knowledge Service Platform and VIP database were used to search literature and journal articles related to drug protection for rare diseases,and references were used as reference theoretical basis.The article also uses the interdisciplinary research method,based on legal issues,comprehensive medicine,pharmacy,sociology,management and other fields,from the academic perspective to analyze the problems and explore solutions.In addition,by the comparative analysis method,the article pointed out the shortcomings in the current system construction by comparing it with the developed countries or regions such as the United States,Australia and the European Union.On the basis of combining Chinese national conditions,the author pointed out that the successful experience of these countries would be used to improve the relevant systems of drug use protection for rare diseases.Strengthening the security system of drug use for rare diseases is of great significance to improve the availability of drugs for rare diseases,guarantee the basic medical rights of patients with rare diseases,improve our medical security system,and boost the development of pharmaceutical industry.But up to now,the drug security system for rare diseases is not perfect,and a perfect drug security system for rare diseases has not been established,and it has not been scientific and standardized.In the context of fully understanding the supply situation of drugs for rare diseases and the current situation of drug use for patients,this article put forward suggestions of drug security system for rare diseases in line with the situation of Chinese economy and social development level and foreign relevant system experience: Clear the legal definition of rare diseases;Optimize the incentive system for R&D of rare disease drugs;Improve the management system of medication guarantee for rare diseases;We should establish a multi-level drug support system for rare diseases and a multi-level drug support system for rare diseases.