本文關(guān)鍵詞： 罕見病用藥 研制 政府促進(jìn) 義務(wù)　出處：《西南大學(xué)》2014年碩士論文　論文類型：學(xué)位論文

【摘要】：罕見病是一類長(zhǎng)期不為人們熟悉的疾病,用于醫(yī)治這類疾病的藥物由于適用人群小,社會(huì)關(guān)注度不高,市場(chǎng)預(yù)期利潤(rùn)低而長(zhǎng)期不為科研機(jī)構(gòu)和制藥企業(yè)所重視。正是由于這些原因,我國(guó)的罕見病用藥長(zhǎng)期依賴進(jìn)口,這一情況造成了嚴(yán)重的市場(chǎng)失靈問題。2007年10月1日施行的《藥品注冊(cè)管理辦法》和2009年1月7日施行的《新藥注冊(cè)特殊審批管理規(guī)定》雖然都規(guī)定了對(duì)創(chuàng)制新藥、治療疑難危重疾病的新藥實(shí)行特殊審批,但是由于具體實(shí)施細(xì)則的缺失,使這些規(guī)定長(zhǎng)期無法落到實(shí)處。一方面,患者因病亟需藥品,另一方面作為藥品研制主體的科研機(jī)構(gòu)和制藥企業(yè),卻因風(fēng)險(xiǎn)高、利潤(rùn)低、法律規(guī)定的不完善和相關(guān)促進(jìn)制度的缺失而對(duì)罕見病用藥的研制望而卻步。 在現(xiàn)代社會(huì)的經(jīng)濟(jì)生活中,當(dāng)面臨無法通過市場(chǎng)自行調(diào)節(jié)來解決的問題時(shí),國(guó)家干預(yù)就成為了最好的選擇。為了進(jìn)一步促進(jìn)我國(guó)罕見病用藥的研制,政府應(yīng)當(dāng)在對(duì)域內(nèi)外的相關(guān)制度進(jìn)行研究和借鑒的基礎(chǔ)上,多管齊下,做到實(shí)體與程序并舉,利用國(guó)家資金扶持、知識(shí)產(chǎn)權(quán)保護(hù)、建立特殊審批機(jī)制等手段初步建立系統(tǒng)的罕見病用藥研制政府促進(jìn)制度并不斷進(jìn)行完善。建立并完善我國(guó)的罕見病用藥研制政府促進(jìn)制度是保證人民群眾健康的需要,是完善我國(guó)社會(huì)保障制度的需要,更是政府應(yīng)當(dāng)履行的一項(xiàng)重要義務(wù)。 具體的制度構(gòu)建是我國(guó)罕見病用藥研制政府促進(jìn)義務(wù)的核心和落腳點(diǎn)。在進(jìn)行具體制度構(gòu)建時(shí),應(yīng)當(dāng)做好相關(guān)理論研究,特別是罕見病用藥研制政府促進(jìn)制度涉及到法學(xué)、經(jīng)濟(jì)學(xué)、醫(yī)學(xué)、藥事學(xué)等多個(gè)學(xué)科,更應(yīng)當(dāng)重視基礎(chǔ)理論的研究,如果盲目進(jìn)行制度建設(shè)不但可能浪費(fèi)本已緊缺的人力和無力還有可能造成相關(guān)制度不具備科學(xué)性和可操作性而成為“空中樓閣”。由于我國(guó)的罕見病用藥研制政府促進(jìn)義務(wù)長(zhǎng)期缺位,在進(jìn)行制度構(gòu)建時(shí)更應(yīng)當(dāng)慎重,在做好基礎(chǔ)理論研究的同時(shí)還應(yīng)當(dāng)對(duì)域外的相關(guān)典型制度進(jìn)行分析和總結(jié),對(duì)域外的有益經(jīng)驗(yàn)進(jìn)行借鑒和消化,使之進(jìn)一步本土化更加適應(yīng)我國(guó)的國(guó)情從而解決罕見病用藥市場(chǎng)失靈問題。 本文共分為四部分： 第一部分為政府促進(jìn)罕見病用藥研制相關(guān)基本理論,著重對(duì)與罕見病用藥研制有關(guān)的基礎(chǔ)理論問題進(jìn)行了探討。具體包括：罕見病的概念、我國(guó)罕見病現(xiàn)狀、罕見病用藥的內(nèi)涵以及外延、罕見病用藥研制的概念以及罕見病用藥研制政府促進(jìn)的概念和意義等。 第二部分為我國(guó)罕見病用藥研制政府促進(jìn)義務(wù)的現(xiàn)狀,該部分對(duì)我國(guó)現(xiàn)有的與罕見病用藥研制政府促進(jìn)義務(wù)有關(guān)的法規(guī)進(jìn)行了梳理,在梳理現(xiàn)有法律規(guī)定的基礎(chǔ)上總結(jié)了罕見病用藥研制政府促進(jìn)在立法上存在的不足,并且對(duì)這些不足存在的原因進(jìn)行了分析。 第三部分為域外罕見病用藥研制政府促進(jìn)義務(wù)考察與借鑒。主要分為兩部分內(nèi)容,首先對(duì)域外有關(guān)國(guó)家和地區(qū)(歐盟、美國(guó)、日本和我國(guó)臺(tái)灣地區(qū))的罕見病用藥研制政府促進(jìn)義務(wù)進(jìn)行了考察。其次,在對(duì)域外相關(guān)制度進(jìn)行考察的基礎(chǔ)上,分析了域外相關(guān)制度可供我國(guó)借鑒的經(jīng)驗(yàn)。 第四部分為全文的核心和落腳點(diǎn),其主要內(nèi)容為完善我國(guó)罕見病用藥研制政府促進(jìn)義務(wù)的對(duì)策建議。在通過法律明確我國(guó)罕見病范圍的基礎(chǔ)上,利用基金扶持、稅收優(yōu)惠政策、價(jià)格調(diào)控措施等具體手段降低罕見病用藥研制主體的成本和風(fēng)險(xiǎn)從而激勵(lì)他們進(jìn)行罕見病用藥的研制。
[Abstract]:A rare disease is a kind of long term for people not familiar with the disease, for the treatment of this disease due to drug for the crowd is small, the degree of social concern is not high, the market is expected to low profits and long-term attention to the scientific research institutions and pharmaceutical enterprises. It is for these reasons, a rare disease medication of our long-term dependence on imports, this a situation has caused serious market failure during.2007 October 1st implementation of the "measures on the administration of drug registration and the January 7, 2009 implementation of the" special approval of new drug registration management regulations although provides for the development of new drugs, new drug for the treatment of complicated and serious diseases of the implementation of special approval, but due to the lack of specific rules for the implementation of these Provisions, the term cannot fall the letter. On the one hand, the patients due to need drugs, on the other hand, as the main drug development research institutions and pharmaceutical companies, but because of the high risk, low profits, the law does not The lack of perfect and related promotion system is prohibitive for the development of rare diseases.
In the modern economic life, when faced with not through the market adjust itself to solve the problem, the state intervention has become the best choice. In order to further promote the development of China's rare disease drug, the government should be the basis for research and reference in the relevant system of inside and outside the domain, multi pronged approach to achieve the entities and procedures simultaneously, use of state funds to support the protection of intellectual property rights, establish special approval mechanism establishment of orphan drug system for the government to promote system and constantly improve. Establish and improve China's rare disease drug development to promote the government system is needed to ensure people's health, is to improve the needs of our society security system, is an important duty of the government should perform.
The specific construction of the system is our country's rare disease drug development to promote the core and goal of government obligations. In the construction of the specific system, should do research on related theories, especially the rare disease drug development of the government to promote the system involves the law, economics, medicine, pharmaceutical science, more attention should be given to the basic theory research. If the blind system may not only waste the already scarce human weakness and may also cause the relevant system does not have the scientific and operable and become "castles in the air." due to a rare disease in China with the development of medicine government promote compulsory long-term absence, when constructing the system should be more careful, do a good job in the basic theoretical research at the same time should also analyze and summarize the typical experience of the foreign system, for reference and digest the useful experience of foreign, make it more suitable for further localization The problem of drug market failure of rare diseases should be solved in response to the national conditions of our country.
This article is divided into four parts:
The first part is the government to promote the development of drugs for rare diseases related to the basic theory, focuses on the development of basic theory and issues related to orphan drugs were discussed. Including: the concept of rare diseases, rare disease status in our country, the connotation and extension of rare disease drug development, the concept of drugs for rare diseases and orphan drug development promoted by the government the concept and significance.
絎簩閮ㄥ垎涓烘垜鍥界綍瑙佺梾鐢ㄨ嵂鐮斿埗鏀垮簻淇?jī)杩涗箟鍔＄殑鐜扮姡?

本文編號(hào)：1450519