| The definition of rare diseases is not uniform and depends on the legislation and policies adopted by each region or country.Orphan drugs are neglected by pharmaceutical companies because of high investment cost,difficult research and development,and small demand.Without incentives,many orphan drugs would not be developed and approved because they are unprofitable for the pharmaceutical industry.In order to solve the plight of patients with rare diseases faced by few drugs,expensive drugs and no cure,meet the drug needs of patients with rare diseases,and protect the life and right to health of patients with rare diseases,many countries have introduced relevant legislation and incentive measures to encourage pharmaceutical enterprises to invest in the research and development and production of orphan drugs.Compared with the rapid development of orphan drug markets abroad,the development of orphan drugs for rare diseases in China is in its infancy.The main purpose of this paper is to improve the accessibility of orphan drugs through legal protection,so as to provide some theoretical references for the formulation and improvement of specific orphan drug legislation and policies,so as to alleviate the current plight of patients with rare diseases who have no drugs available and cannot afford medicinal use.The main way is to sort out the current legislation status and existing deficiencies of orphan drugs for rare diseases in China,compare and analyze the legal framework and regulatory environment of orphan drugs in the United States,the European Union and Japan,and believe that it is necessary to build a legal guarantee system to encourage the development of orphan drugs in China.Then,based on the basic national conditions,economic level,medical conditions,the current living conditions of patients with rare diseases and other factors,the author puts forward some concrete suggestions on how to improve the accessibility of orphan drugs by law: First,in order to improve the accessibility of orphan drugs and promote the development and innovation of orphan drugs,it is suggested to improve the legislation on orphan drugs for rare diseases,clarify the legislative definition of rare diseases,promote the development of orphan drug clinical trials,improve the market exclusivity of orphan drugs and other legal safeguards.Second,due to the small number of patients with rare diseases and the limited market size of orphan drugs,pharmaceutical enterprises may lack the enthusiasm to research and develop orphan drugs.Through providing tax incentives,research and development fund support and other policy measures,pharmaceutical enterprises are encouraged to invest in the research and development and innovation of orphan drugs.Third,improve the affordability of orphan drugs,reduce the economic burden of patients with rare diseases,including medical assistance,drug access,medical insurance and other aspects,so that patients can get fair and timely treatment.Fourth,raise the social attention of rare diseases,improve the medical service system,and establish a rare disease information platform.Legal protection can raise the issue of rare diseases to the national level and promote the attention of all sectors of society to rare diseases.This will help protect the rights and interests of patients with rare diseases and ensure that their legitimate rights and interests are safeguarded.Fifth,while accelerating the marketing and approval of domestic drugs,strengthen international cooperation in the research and development of orphan drugs for rare diseases,promote transnational cooperation and information sharing,and help China cooperate with other countries in the research and development of rare diseases and orphan drugs,so as to jointly meet the challenges of global rare diseases. |
