Research on the Construction of Social Security Systems for Rare Diseases
With the advancement of society and technological development, rare diseases—though having a low incidence and being relatively uncommon—have become increasingly recognized due to their high treatment costs. China's policies in the field of rare diseases are still in the nascent stage, lacking clear, reasonable, and comprehensive policy practices. This paper systematically reviews the definitions of rare diseases, the pricing of orphan drugs, and relevant social security policies, both domestically and internationally. It offers the following recommendations: (1) The definition of rare diseases in China should be based on the number of patients affected; (2) The government should increase support for the research and development of orphan drugs; (3) After including certain rare diseases in medical insurance, the government should conduct a thorough and accurate evaluation of the policy's impact, including its effect on medical insurance rates; (4) In the long term, the government should provide guidance and, to some extent, regulate reproductive behavior among rare disease patients.
[1]Tambuyzer E,2010, Rare Diseases, Orphan Drugs and Their Regulation: Questions and Misconceptions. Nature Reviews Drug Discovery,9(12):921–929.
[2]Liu H,2012, Research on the Establishment of Orphan Drug Policies in China, thesis, Heilongjiang University of Traditional Chinese Medicine.
[3]Reaves ND,2003, A Model of Effective Health Policy: The 1983 Orphan Drug Act. Health Soc Policy,17(4):61–71.
[4]Picavet E, Cassiman D, Simoens S,2012, Evaluating and Improving Orphan Drug Regulations in Europe: A Delphi Policy Study. Health Policy,108(1):1–9.
[5]Aronson JK,2006, Rare Diseases and Orphan Drugs. British Journal of Clinical Pharmacology,2006,333:127.
[6]Ulrich LC,2010. Efforts to Develop Orphan Drugs: The FDA Experience.
[7]Xu X, Zhu L, Huang H,2017, Overview of International Orphan Drug R&D Incentive Policies and Their Implications for China. International Journal of Pharmaceutical Research,44(2):112–116.
[8]Du T, Gong Z, Gao Y,2015, Policies, Strategies, and Practices for Orphan Drug Development in the United States.Pharmaceutical Progress,2015(8):566–570.
[9]Mao Y, Gao Y, Du T,2016, Comparative analysis of orphan drug policies in Various Countries. Pharmaceutical Progress,2016(6):429–436.
[10]Garau M, Mestre-Ferrandiz J,2009, Access Mechanisms for Orphan Drugs: A Comparative Study of Selected European Countries. OHE Briefing,2009:1–32.
[11]Lin Y, Wu X,2012, Lessons from Taiwan's Legal System for Rare Drugs. Legal Research,2012(6):87–91.
[12]Simoens S,2011, Pricing and Reimbursement of Orphan Drugs: The Need for More Transparency. Orphanet Journal of Rare Diseases,6:42.
[13]Stolk P, Heemstra HE, Leufkens HG, et al.,2009, No Difference in Between-Country Variability in use of Newly Approved Orphan and Non-Orphan Medicinal Products - A Pilot Study. Orphanet Journal of Rare Diseases,4(27).
[14]Daniel MG, Pawlik TM, Fader AN, et al.,2016, The Orphan Drug Act: Restoring the Mission to Rare Diseases.American Journal of Clinical Oncology,39:210–213.
[15]Li Y,2014, Discussion on the Formulation of Relevant Policies for Rare Diseases in China: An Analysis Based on the Living Conditions of the Rare Disease Population. China Soft Science,2014(2):77–89.
[16]He J, Zhang Y, Xia S, et al.,2012, The EU Rare Disease Protection System and its Implications. China Health Policy Research,5(7):52–59.
[17]Rtlein GB, de Varax AM, Letellier M,2004, Study on Orphan Drugs: Phase I-Overview of the Conditions for Marketing Orphan Drugs in Europe. ALCIMED.
[18]Michaud JP, Modi R, Phillips MI,2013, Is Orphan Drug Pricing Blowing a Bubble? The Unique Situation of Orphan Drugs and Why High Prices will Likely Persist. Expert Opinion on Orphan Drugs,1(9):675–679.
[19]Hyry HI, ROOS JCP, Cox TM,2015, Orphan Drugs: Expensive yet Necessary. QJM: An International Journal of Medicine,108:269–272.
[20]Hemphill TA,2010, Extraordinary Pricing of Orphan Drugs: Is it a Socially Responsible Strategy for the US Pharmaceutical Industry? Journal of Business Ethics,94(2):225–242.
[21]Hughes-Wilson W, Palma A, Schuurman A, et al.,2012, Paying for the Orphan Drug System: Break or Bend? Is it Time for a New Evaluation System for Payers in Europe to take Account of New Rare Disease Treatment? Orphanet Journal of Rare Diseases,7(1):961–974.
[22]Chai Y, Wu Y, Xin X, et al.,2015, Discussion on the Legislation of Rare Diseases in China. New West,2015(8):75–76.
[23]Cote A, Keating B,2012, What is Wrong with Orphan Drug Policies? Value in Health,15(8):1185–1191.
[24]Kanavos P, Nicod E,2012, What is Wrong with Orphan Drug Policies? Suggestions for Ways Forward. Value in Health,15(8):1182–1184.
[25]McCabe C, Claxton K, Tsuchiya A,2005, Orphan Drugs and the NHS: Should We Value Rarity? Bmj,331(7523):1016–1019.
[26]Xiang W,2016, Study on the Medical Security System for Rare Diseases in Jiangsu Province, thesis, Nanjing University.
[27]Paz MPDL, Groft SC,2010, Rare Diseases Epidemiology. Advances in Experimental Medicine & Biology,686(193):101–102.
[28]Zhang W,2014, Medical Security for Rare Diseases from the Perspective of Equal Rights. Theoretical Exploration,2014(5):117–120.
[29]Zhang L,2011, Ethics and Policy Research on Rare Diseases in China, thesis, Peking Union Medical College.
[30]Zhang N,2013, Research on the Legal System for the Management of Rare Drugs in China, thesis, Nanjing University of Traditional Chinese Medicine.
[31]Gong S, Zhang M, Deng X, et al.,2006, Reflections on the Definitions of Rare Diseases and Rare Drugs in China.Chinese Journal of New Drugs,15(15):1225–1229.
[32]Cheng Y,2011, Dilemmas and Solutions of the Legal System for Rare Diseases. Hebei Law Science,29(5):10–18.
[33]Song P, Gao J, Inagaki Y, et al.,2012, Rare Diseases, Orphan Drugs, and Their Regulation in Asia: Current Status and Future Perspectives. Intractable Rare Dis Res,1(1):3–9.
[34]Li Y,2006, A New Exploration of the Goal Positioning of Social Security System Reform in China. Society,2006(02):175–188.
[35]Zheng G,2010, Social Security Reform and Future Development in China. Journal of Renmin University of China,2010(05):2–14.
[36]Wang JB, Guo JJ, Yang L, et al.,2010, Rare Diseases and Legislation in China. The Lancet,375:708–709.
[37]China Rare Disease Network. www.hanjianbing.org
[38]Chen Y, Wu L,2014, A Preliminary Exploration of Rare Disease Definitions in China.
[39]US Food and Drug Administration. http://www.rarediseases.org/
[40]European Medicines Agency (EMEA), Regulation No.141/2000 of the European Parliament and Council on Orphan Medicinal Products.
[41]Australia Orphan Drug Program,1998.
[42]South Korea Rare/Intractable Disease Center. http://helpline.nih.go.kr/cdchelp/
[43]Taiwan Rare Disease Special Nutritional Products and Orphan Drug Logistics Center. http://www.rdfdlm.tw/
[44]European Medicines Agency. http://www.ema.europa.eu/
[45]Central People's Government of the People's Republic of China,2017, The General Office of the CPC Central Committee and the General Office of the State Council issued the "Opinions on Deepening the Reform of the Review and Approval System to Encourage Innovation in Drugs and Medical Devices." https://www.gov.cn/xinwen/2017-10/08/content_5230105.htm
[46]Zhejiang Provincial Department of Civil Affairs. http://www.zjmz.gov.cn/il.htma=si&id=8aaf80155717920d0157417ef0e202a9