SCIO briefing on strengthening drug regulation to ensure drug safety for the public
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E-mail China.org.cn, July 28, 2023
Cnr.cn:
In recent years, medicines for rare diseases have received significant attention. What measures the National Medical Products Administration has taken to encourage innovative research and also the production and supply of medicines for rare diseases? Thank you.
Jiao Hong:
Thank you for your question. Rare disease patients are indeed a group that requires special attention and care, and the management of their medicines is also one of the key areas of focus for the National Medical Products Administration. Now, I will ask Huang Guo to provide more detailed information on this matter.
Huang Guo:
Indeed, we are more confident when the media expresses concern. The inquiries from the media also reflect the general public's awareness and support for treating rare diseases.
As we all know, the prevalence of rare diseases is low. Therefore, the research and development of medicines for these diseases is very challenging, and the market size is small. However, when it comes to every family and each patient, medicines for rare diseases are often lifesaving and cannot be ignored. Therefore, we believe that every medicine for a rare disease deserves our utmost effort.
In recent years, the National Medical Products Administration has seized the opportunity of deepening reforms to the evaluation and approval of medicines, and made significant efforts to accelerate the approval of medicines for rare diseases, striving to provide more options for patients suffering from these illnesses. Due to time constraints, I will primarily introduce two measures.
On the one hand, unleashed policy dividend has sped up the research and development of rare disease medicines. Since 2018, we've provided a special channel in the evaluation and approval link for medicines which are already available in overseas markets and in urgent need for clinical use, including those for treating rare diseases. Under this policy, we've established a specific waiting list, encouraged application of those medicines and expedited their review and approval process. Statistics show that 23 new rare disease medicines have been approved for import and domestic marketing via this special channel. In 2020, we further streamlined the priority review and approval procedure, including new rare disease medicines with significant clinical outcomes into the priority review program. Currently, among all drug marketing applications, the review and approval time for rare disease medicines is the shortest.
On the other hand, we've strengthened technical guidance for enterprises to take fewer detours. Given the exceptionally low incidence of individual rare diseases, the challenges of drug research significantly exceed those of other common diseases. We have provided special policy preferences for new rare disease medicines. Our drug evaluation and approval agency has conducted early participation, interconnected research and evaluation, and provided a full range of services. We've established a special review team to follow up on the research and development of new medicines for rare diseases. Enterprises are allowed to submit research materials on a rolling basis, and a seamless connection mechanism has been established in key phases such as communication, inspection and testing, and comprehensive evaluation. These practices have greatly improved the quality and efficiency of new drug development for rare diseases. We've developed a series of technical guidelines, such as Technical Guidelines for the Clinical Research and Development of Drugs for Rare Diseases and Statistics Guidelines for the Clinical Research and Development of Drugs for Rare Diseases, to guide enterprises in tailoring their approach to the characteristics of rare diseases, promoting flexible designs while maintaining scientific rigor. We encourage them to obtain scientific evidence by making good use of limited patient resources so as to advance clinical research and development.
Through comprehensive measures, China has improved in both the number and speed of rare disease medicine approvals in recent years. To give you some figures, since 2018, China has approved 68 imported and domestically produced drugs for rare diseases. Going forward, the National Medical Products Administration will continue to focus on the needs of rare disease medicines. While ensuring the safety, efficacy, and quality of medicines on the market, we will expedite the evaluation and approval of rare disease drugs, striving to slow the progression of rare diseases and enhance the quality of life for patients.
Thank you!
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