-- Third article in the series of articles on rare disease drugs
In the Ice Bucket Challenge in 2014, the "amyotrophic lateral sclerosis" (ALS) patients attracted the world’s attention, and more people started to pay attention to the rare diseases. In the previous two articles in the series of articles on rare disease drugs, the U.S. and EU basic regulations on orphan drugs, and 2017 drug approval situation are separately introduced. Compared to U.S. and EU, the development of rare disease drugs in China is lags far behind.
Support from regulations and policies can be called one of the important factors restricting orphan drug development. Let’s first talk about the regulations, to see the gap between China and the Europe and America in the development process of rare disease drugs.
China
Current situation in China—there have been no explicit official criteria for the determination of rare diseases in China, let alone the independent regulation, and perfect and systematic approval policy on rare diseases, and the medical insurance reimbursement therefor is very little. The current R&D of "orphan drugs" in China can be said to be still in the beginning stages, there are only a few enterprises involving the rare disease drugs, and someone even vividly described the current embarrassing situation of the orphan drugs in China as "three noes". On the other hand, there have been about 20 million rare disease patients in China, and the drugs for rare diseases almost depend on import, therefore, many rare disease patients have no access to drugs, or have to choose the expensive imported drugs.
China and its relevant departments have been actively striving to create favorable conditions for the R&D of orphan drugs in China in recent years, however, the process is slow due to a lack of systematic and strong support. The followings are articles or content involving rare diseases and drugs therefor in the policies and regulations issued in China as collected and arranged by me:
2007: Measures for the Administration of Drug Registration (CFDA Order No. 28)
Article 32 of the Measures for the Administration of Drug Registration specifies that: Where there are circumstances, regarding rare or special diseases, etc., which request clinical sample size reduction or clinical trial exemption, a request shall be made with the clinical trial application, and reviewed and approved by China Food and Drug Administration; Article 45 defines that special review and approval may be implemented for "new drugs for the treatment of diseases such as AIDS, malignant tumors and rare diseases with significant clinical advantage".
2009: Administrative Provisions for the Special Review and Approval of New Drug Registration
The document specifically describes the "special review and approval" in the Measures for the Administration of Drug Registration: for the rare diseases, it specifies that the Center for Drug Evaluation of CFDA (CDE) shall organize an expert meeting to review and determine within 20 days upon receipt of the applications for special review and approval. And applicants who apply for special review and approval shall formulate corresponding risk control plans and implementation schemes at the time of applying for clinical trial and production.
It encourages the drug innovation oriented by clinical value, and reiterate that "review shall be accelerated for the registration applications of innovative drugs that have better therapeutic effects for major diseases, rare diseases, and diseases of the old and children, possess independent intellectual property, and are listed in the National Science and Technology Plan and Major Project of China, etc.".
It establishes the f...
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