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E-mail China.org.cn, October 16, 2024
Cnr.cn:
My question is about medications for rare diseases. We know that in recent years, the issue of medication for patients with rare diseases has attracted widespread attention from society, and I am also very concerned about it. What measures has the NMPA taken to encourage innovation, R&D and introduction of medicines for rare diseases? Thank you.
Huang Guo:
Thank you for your question. Medications for rare diseases indeed require the attention of the entire society. To meet the medication needs of patients with rare diseases, the NMPA has continuously introduced a series of incentive policies and promoted R&D and marketing of medicines for rare diseases through multiple channels. In summary, there are mainly three channels:
First, we're encouraging independent innovation. By promoting the R&D of new medicines in China, we aim to address the lack of available treatments for certain rare diseases. As you may know, we have established accelerated pathways for rare disease drug applications, including breakthrough therapy and medicine designations, conditional approvals, and priority review and approval procedures. Technically, we're enhancing the efficiency of R&D and market launch for rare disease drugs through several methods. These efforts include strengthening communication and guidance during the R&D process, allowing rolling submissions of data, reasonably determining alternative clinical endpoint s, and shortening review timelines. In other words, our goal is speed.
Second, we're expediting the import process to accelerate the entry of rare disease drugs being researched or produced overseas into the Chinese market. On the one hand, we're supporting multinational pharmaceutical companies in conducting simultaneous R&D, application, and market launch in China. To this end, we've implemented a series of measures, such as establishing an implied license system for clinical trials and accepting overseas clinical trial data. On the other hand, we're encouraging the import of rare disease medicines already marketed abroad. Currently, we're soliciting the public's opinions on the Announcement on Issues Pertaining to the Review and Approval of Overseas New Drugs Urgently Needed in Clinical Settings. For drugs that meet the requirements, we're implementing measures to encourage import applications, including waiving clinical trials, granting priority review and approval, shortening inspection timelines, and reducing the number of inspection batches and samples.
Third, a temporary import channel to try and ensure the availability of urgently needed clinical drugs under special circumstances. In 2022, we collaborated with the National Health Commission to develop and release the Work Plan for the Temporary Import of Urgently Needed Clinical Medications. Over the past two years, this channel has ensured the supply of urgently needed medications like clobazam and sapropterin hydrochloride for rare diseases.
In addition, we have strengthened collaboration with relevant local authorities and departments to create more channels for the clinical use of drugs for rare diseases. Our measures include supporting medical institutions within Hainan's Boao Lecheng International Medical Tourism Pilot Zone and the Guangdong-Hong Kong-Macao Greater Bay Area with importing urgently needed clinical medications, as well as supporting the establishment of a pilot zone in the Beijing Tianzhu Comprehensive Bonded Zone to ensure the supply of rare disease medications.
Altogether, these measures have led to a significant increase in both the number and speed of rare disease medications entering the market in recent years. Since 2018, over 130 rare disease drugs have been approved for the market, with as many as 37 being approved from January to August 2024.
In the next step, we will continue to make sustained efforts to build and develop these important channels for ensuring the availability of rare disease medications. We will closely collaborate with health, healthcare security and other departments to increase policy support and encourage companies to innovate and conduct research and development, so that more drugs for rare diseases can become available. Thank you!
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