"Orphan drug" is not unpopular: Inventory of policies related to drug research and development for rare diseases

In order to further improve the efficiency of clinical research and development of rare diseases, meet the treatment needs of patients with rare diseases, combine the characteristics of rare diseases, and provide suggestions and references for the development of rare disease drugs and scientific trial design, on October 11, 2021, the CDE official website released the "Rare Diseases" Technical Guidelines for Drug Clinical Research and Development (draft for comments), the time limit for soliciting comments is 1 month from the date of issuance.
The technical guidelines aim to further improve the efficiency of clinical research and development of rare diseases, meet the treatment needs of patients with rare diseases, and combine with rare diseases.
Disease characteristics, provide suggestions and references for rare disease drug research and development and scientific trial design.
This article counts the policies and regulations related to rare disease drug research and development
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PART 0 1.
Definition of rare diseases that broke the game The blue ocean of rare disease drugs is becoming a popular track
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Rare diseases, as the name suggests, refer to those diseases with a very low incidence, and so far there is no clear definition in China
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Due to the small number of patients with rare diseases, low market demand, high research and development costs, and fewer pharmaceutical companies to research and develop, these drugs are also called "orphan drugs
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" Countries around the world have certain differences in the identification standards of rare diseases according to their own country's specific conditions.
See the table below for details
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PART 0 2.
China’s policy dividends support the research and development of rare disease drugs In order to overcome the hard bones of rare diseases, the regulatory authorities have attached great importance to the research and development of rare disease drugs in recent years and issued the following series of regulatory documents to accelerate the review and approval of rare disease drugs, including but Not limited to: PART 0 3.
CDE’s review and consideration of rare disease drugs has always been the medical field due to difficult research and development, low morbidity (narrow audience), relatively small medical research, and high research investment costs in the field of rare diseases.
The weak link in development is also the research and development direction that the major domestic pharmaceutical companies have avoided before
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In order to promote the research of rare diseases, the reform of China's registration system also shows that China attaches great importance to the research and development of drugs for the treatment of rare diseases, and has successively introduced favorable policies to provide policy support at different stages such as the research and development of new drugs for the treatment of rare diseases, the application for marketing, and the post-marketing.
However, there are still many problems in this field that need to be solved urgently.
For example, the official definition of rare diseases and orphan drugs is not yet simple.
CDE's general considerations for the development of rare disease drugs are the "trilogy": 1) Accept overseas data; 2 ) Promote high-level imitation; 3) Encourage the research and development of new drugs
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Regarding whether to accept overseas data, CDE has issued the "Notice on the Release of Technical Guidelines for Accepting Overseas Clinical Trial Data of Drugs" (No.
52 of 2018), which defines the application status of accepting overseas data.
The consideration factors for accepting overseas data review include but Not limited to: 1) The reliability of foreign research data, especially the design, evaluation methods and research results of key clinical trials; 2) Is the dose of foreign research applicable to our patients? 3) Are the morbidity and diagnostic criteria consistent with those in China; 4) What problems are urgently needed to be solved by the existing treatment methods? 5) the difficulty of risk control, including the requirements for prescribers and patients of safety monitoring; expert consultation will be held, if necessary, strengthen communication with regulators and so on
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The review of rare disease drugs will comprehensively consider factors such as the incidence, severity, availability of drugs, the basis for foreign marketing, and China’s practice.
The state will continue to increase the value-added tax concessions granted to rare disease drugs, and will continue to update them.
We will well support investment in rare diseases and encourage the development of drugs tailored to the needs of rare patients to further improve the health and well-being of the people
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References [1][2] Yang Zhimin, "Regulations on Registration, Review and Registration of Drugs for Rare Diseases", cphi, antibody circle, etc.