Table 1 Incentive policies on orphan drug development
Effective time | Title | Theme | Subtheme | Related Content |
|---|---|---|---|---|
2012-1 | The 12th Five-Year Plan for National Drug Safety [14] | National strategy | National strategy | Strengthen the quality supervision of a drug product throughout its lifecycle, and encourage the R&D of orphan drugs and pediatric dosage forms. |
2013-2 | Opinions on Deepening the Reform of the Review & Approval System and Encouraging Drug Innovation [15] | Expedited approval | Priority review | Implement priority review for orphan drugs; accelerate the registration application of innovative drugs for rare diseases. |
2015-8 | Opinions of the State Council on the Reform of Review and Approval System of Drugs and Medical Devices [16] | Expedited approval | Accelerated review and approval | Accelerate the review and approval of innovative drugs for AIDS, malignant tumors, major infectious diseases and rare diseases. |
2015-11 | Announcement on Certain Relevant Policies on the Review and Approval of Drug Registration [17] | Expedited approval | Accelerated review and approval | Set up a separate queue to accelerate the review and approval of innovative drugs for the prevention or treatment of HIV/AIDS, malignant tumors, severe infectious diseases, and rare diseases. |
2016-2 | Opinions on Solving the Backlog of Drug Registration Applications and Implementing Priority Review and Approval [18] | Expedited approval; technical support;; safety and efficacy requirements | Priority review and approval; protocol assistance; scientific advice/consultation; clinical trial exemption | Drugs for the prevention and treatment of rare diseases can be granted priority review and approval; the CDE provides scientific advice for the development of clinical protocol and consultations for decision making. For orphan drugs, applicants can apply for abbreviation or waiver of clinical trials, and the CDE will give review advice based on technical review needs and the actual situation of Chinese patients. |
2016-4 | Notice of the CPC Central Committee and the State Council on Issuing the Key Tasks of Deepening the Health Care System Reform [19] | Expedited approval | Accelerated review and approval | Further unblock the green channels for the review and approval of drugs for rare diseases or in urgent clinical need or for the special patient population, and accelerate the progress of registration review. |
2017-2 | 13th Five-Year Plan for National Drug Safety [20] | Expedited approval | Accelerated review and approval | Accelerate review and approval of drugs and medical devices for rare diseases and speed up the launch of orphan drugs with independent intellectual property rights. |
2017-10 | Opinions on Deepening the Reform on Review and Approval System and Encouraging Innovation on Drugs and Medical Devices [21] | Safety and efficacy requirements, exclusivity, expedited approval | Clinical trial exemption; data protection; conditional approval | Propose to publish a national list of rare diseases and establish a registration system for patients with rare diseases; applicants for drugs and medical devices for rare diseases can apply for waiver or abbreviation of clinical trials and submit an application for the protection of independent and yet undisclosed study data; marketing application of the same medical product developed by other applicants will not be approved during the data protection period; conditional approval can be granted for drugs and medical devices for rare diseases approved overseas. |
2017-12 | Opinions of the China Food and Drug Administration on Priority Review and Approval to Encourage Drug Innovation [22] | Expedited approval; safety and efficacy requirements; | Priority review and approval; clinical trial exemption; | Priority review and approval can be granted for orphan drugs with significant clinical advantages. Applicants can apply for abbreviation or waiver of clinical trials for orphan drugs. |
2018-4 | Interim Measures for Implementing Pharmaceutical Study Data Protection (Draft for Public Review) [23] | Exclusivity | Data protection | Orphan drugs are included in the scope of data protection and can be entitled to a six-year data exclusivity upon the date of first approval of the relevant indication in China. |
2018-5 | Announcement on Optimizing Review & Approval of Drug Registration[24] | Safety and efficacy requirements | Acceptance of overseas clinical trial data | Applicants of orphan drugs approved overseas can directly submit registration application by using overseas clinical trial data if the ethnic/racial difference has been proved to be non-existent. |
2018-7 | Technical Guidelines for Accepting Overseas Clinical Trial Data of Drugs [25] | Safety and efficacy requirements | Acceptance of overseas clinical trial data | Based on the quality of overseas clinical trial data, its acceptance by NMPA will be divided into fully accepted, partially accepted and rejected. For application of orphan drugs whose overseas clinical trial data are categorized as “partially accepted”, the NMPA may grant a conditional acceptance of the overseas clinical trial data subject to the collection of further efficacy and safety data for evaluation after market authorization. |
2018-10 | The Review and Approval Process for Overseas Approved New Drugs with Urgent Clinical Need [26] | Expedited approval | Accelerated review and approval | Establish special channels for the review and approval of orphan drugs that have been marketed in the US, EU or Japan in the past decade but not yet in China; technical review of orphan drugs should be completed within three months (six-month for other overseas approved new drugs) upon the reception of application dossier. |
2019-5 | Key Considerations in Using Real-World Evidence to Support Drug Development (Draft for Public Review) [27] | Technical support | Protocol assistance | Encourage the use of real-world evidence to evaluate drug safety and efficacy; for clinical trials of orphan drugs, external controls based on real-world data in natural disease cohorts can be considered. |
2019-12 | Drug Administration Act [28] | National strategy | National strategy | Encourage the development of new drugs indicated for rare diseases. |
2020-1 | Provisions for Drug Registration [29] | Expedited approval | Priority review and approval | Applicants of innovative drugs and modified new drugs indicated for rare diseases can apply for priority review and approval for marketing authorization; for urgently needed orphan drugs that have been marketed overseas but not yet in China, the review procedure should be completed within 70 days. |
2020-12 | Administrative Measures for Communication of Drug R&D and Technical Review [30] | Technical support | Scientific advice/consultation | Provide applicants with a variety of proactive communication channels such as face-to-face meetings, video conferences and written responses for consultation regarding key technical issues that are not covered by currently issued guidelines for drug R&D and evaluation; applicants of orphan drugs can propose a communication meeting with CDE to jointly discuss relevant technical issues, particularly regarding priority review and approval. The consensus reached by both parties can be used as an important reference for drug development and evaluation. |
2021-12 | Technical Guidelines for Clinical Development of Orphan Drugs [31] | Technical support | Scientific advice/consultation | The CDE suggests innovative and widely accepted approaches to overcome common challenges in clinical development of orphan drugs to further improve the efficiency in orphan drug development. |
2022-5 | Implementing Regulations of the Drug Administration Law of the People’s Republic of China (Draft for Public Review) [32] | Expedited approval ; exclusivity | Priority review and approval; market exclusivity | Encourage the R&D of orphan drugs and indication expansion of existing drugs to cover rare diseases; implement priority review and approval for orphan drugs with urgent clinical needs and speed up the marketing process; grant 7-year exclusivity for approved orphan drugs to prohibit generic manufacturers from securing a marketing authorization. |
2022-6 | Guidelines for Statistics of Clinical Research of Orphan Drugs (Draft for Public Review) [33] | Technical support | Scientific advice/consultation | The CDE provides guidance for sponsors to conduct rare disease clinical research, especially by addressing key statistical issues in research. |
CDE, Center for Drug Evaluation; NMPA, National Medical Products Administration | ||||