Table 1. Current effective national policies about rare diseases.

Item	Category	Effective time	Policy & Regulation	Content about Rare Diseases
Availability	Development and imitation of drugs	2012 (5)	The Notice of the State Council on National Drug Safety During the 12th Five-Year Plan issued by the State Council.	To encourage the development of orphan drugs and suitable dosage forms for children.
		2017 (6)	The Opinions of the State Council on Reform of the System of Evaluation, Review and Approval of Drugs and Medical Devices issued by the General Office of the State Council.	To support the development of drugs and medical devices for rare diseases.
	Registration and approval	2007 (7)	The Measures for the Administration of Drug Registration issued by former China Food and Drug administration.	Special approval for new drugs with obvious clinical efficacy in the treatment of AIDS, malignant tumors, rare diseases etc.
		2009 (8)	The Notice of the China Food and Drug Administration for the Special Approval and Management of New Drugs issued by former Chinese Food and Drug Administration.	Special approval process for the application of new drugs for the treatment of AIDS, malignant tumors, rare diseases and other diseases with obvious clinical advantages.
		2013 (9)	The Opinions of the China Food and Drug Administration on deepening the reform of evaluation and approval systems and encouraging innovation on drugs issued by former China Food and Drug Administrations.	Prioritize and speed up the evaluation of rare disease drugs.
		2015 (10)	The Opinions of the State Council on Reform of the System of Evaluation, Review and Approval of Drugs and Medical Devices issued by the State Council.	Accelerated evaluation and approval of innovative drugs for the prevention and treatment of diseases such as rare diseases, sub-neoplastic diseases, AIDS and major infectious diseases.
		2016 (11)	The Notice of the General Office of the State Council on issuing the 2015 Major Task List on Deepening the Medical and Health Care System reform issued by the General Office of the State Council.	Further smooth the special channel for evaluation and approval of rare disease drugs and clinical urgently needed drugs.
		2017 (12)	Several Opinions of the General Office of the State Council on Further Reforming and Improving the Policies on Drug Production, Circulation and Use issued by the General Office of the State Council.	Classified Evaluation and approval of drugs for rare diseases, children, the elderly, emergency (rescuing) and Chinese medicine (classical prescription).
		2017 (13)	The Policies of the China Food and Drug Administration regarding Encouraging Innovation and Accelerating the Evaluation and Approval Systems on Drugs and Medical Devices (Consultation Paper) issued by the former China Food and Drug Ministration.	Applicants for rare disease treatments and medical devices may apply for clinical trials for reduction and exemption; and rare-drug treatment drugs and medical devices that have been approved for marketing abroad, supplement relevant research within the prescribed time after listing.
		2017 (14)	Opinions of the China Food and Drug Administrationon encouraging innovative implementation of prioritized evaluation and approval on drugs issued by the former China Food and Drug Administration.	Drug registration for rare diseases can be included in the scope of prioritized evaluation and approval.
		2018 (15)	Notice of the National Medical Products Administration and the National Health Commission on Optimizing Review and Approval of Registration of Medical Products issued by the National Medical Product Administration and the National Health Commission.	Orphan drugs can submit clinical trial data obtained overseas and directly apply for drug listing registration, which meeting the requirements of the Drug Registration Management Measures and related documents may directly approve the import.
		2018 (16)	Interim Measures of the National Medical Products Administration for Protection of Pharmaceutical Test Data (Consultation Paper) issued by the former China Food and Drug Administration.	Orphan drugs are listed as the target of data protection and a 6-year data protection period is granted to them since the indication firstly approved in China.
Adaptability	Diseases list	2017 (17)	Policies of the China Food and Drug Administration regarding Encouraging Innovation and Accelerating the Evaluation and Approval Systems on Drugs and Medical Devices (Consultation Paper) issued by the former China Food and Drug Ministration.	The Health and Family Planning Department would publish a list of rare diseases and establish a registry system for rare patients.
		2018 (18)	The National Rare Disease List by five Authority bodies.	121 rare diseases.
	Diagnosis and Treatment	2018 (19)	The Notice of the National Medical Products Administration of Requesting the List of Urgently Needed New drugs in Clinical (the First Batch) issued by the National Medical Product Administration.	There are 20 drugs' indications related to 12 diseases in the National Rare Disease List.
		2019 (20)	The Notice of the National Medical Products Administration of Requesting the List of Urgently Needed New drugs in Clinical (the Second Batch) issued by the National Medical Product Administration.	Involving 13 drugs treating rare diseases.
		2019 (21)	The Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 Edition) issued by the National Health Commission.	The diagnosis and treatment of 121 rare diseases.
Affordability	Pricing	2019 (22)	The Notice by the Ministry of Finance regarding the Value Added Tax Policies on Drugs for Rare Diseases issued by the Ministry of Finance.	For the first batch of 21 orphan drugs and 4 drug substances, value added tax will be reduced to 3% referring to the anti-cancer drug in import process, and the domestic value added tax may choose the simple method, levied by 3%.
	Inclusion in Medical Insurance	2012 (23)	The Notice of the Ministry of Health, the Ministry of Finance, and the Ministry of Civil Affairs on Effectively Conducting Work for the New rural cooperative Medical System issued by three authority bodies.	12 diseases including chronic myeloid leukemia, hemophilia and cleft lip and palate are preferentially included in the pilot area of major illness prevention.
		2019 (24)	The Work Plan for the adjustment of Medicines List for National Medical Insurance in 2019 (Consultation Paper) issued by the National Healthcare Security Administration.	The national medical Insurance list of drug is adjusted to give priority to the treatment of serious diseases such as national essential drugs, cancer and rare diseases.