Policy warming up! In order to encourage orphan drugs to go on the market, CFDA has carried out a series of reforms in the past 10 years

Source: Bio research 2017-09-13 orphan drug research enthusiasm was once affected by low incidence rate of return, investment difficulty, and less patient application Many pharmaceutical companies are reluctant to set foot in this field Now, with the improvement of social awareness, the introduction of supporting policies, the increase of diagnosis and treatment means, the development trend of drugs for rare diseases is warming At the 12th International Congress of rare diseases and orphan drugs and the 6th China rare diseases Summit Forum, Gao Chenyan, director of Clinical Department of biological products from the drug review center of the State Food and drug administration, and Zhang Jie, senior reviewer of clinical step of chemical drugs, shared with you the impact of "drug review and approval reform on the listing of orphan drugs" from the level of national policy Rare disease: "rare disease" refers to a kind of disease with a very low prevalence About half of the patients are born or in childhood The disease often progresses rapidly and has a high mortality rate In China, there is no official definition In 2010, the Chinese Medical Association Medical Genetics branch recommended that the disease rate of Chinese population below 50 1/10000 or the incidence rate of newborn less than 1/10000 was defined as a rare disease According to this statistics, the number of rare diseases in China is far beyond 16 million The core clinical needs of patients with rare diseases and their families have always been "seeking doctors and medicine", getting timely and accurate diagnosis, effective and feasible treatment However, because of the low incidence rate, many people have little knowledge of rare diseases Because rare diseases involve many subjects such as children and adults, many doctors have relatively limited knowledge of rare diseases In the past 30 years, more than 500 kinds of rare disease drugs have been listed in the world, but 70% of them have not yet entered the Chinese market Moreover, most of the rare diseases are "incurable", only about 1% of patients have the opportunity to get effective treatment Of the 7000 rare diseases known, only about 400 have specific treatment Data shows that more than 30% of the patients with rare diseases need to go through the diagnosis and treatment of 5-10 doctors, 48.3% of the patients are misdiagnosed as other diseases, and the misdiagnosis time is up to 5-30 years CFDA encourages the research and development of orphan drugs, and the policy "turns warm" in 2007: Measures for the administration of drug registration (Decree No 28 of the Bureau) Article 32 of the measures for the administration of drug registration stipulates that in the case of rare diseases, special diseases, etc., the number of clinical trial cases shall be reduced or clinical trials shall be exempted, which shall be put forward when applying for clinical trials and examined and approved by the State Food and drug administration At the same time, Article 45 makes it clear that "new drugs for treatment of AIDS, malignant tumors, rare diseases and other diseases with obvious clinical treatment advantages" can be subject to special approval 2009: the "special examination and approval" of the "administrative measures for drug registration" is specified in the "administrative provisions for special examination and approval of new drug registration": for rare diseases, it is stipulated that the drug evaluation center of the State Food and drug administration shall organize an expert meeting to review and determine within 20 days after receiving the application for special examination and approval The applicant applying for special examination and approval shall formulate corresponding risk control plan and implementation plan when applying for clinical trial and production 2013: opinions on deepening drug review and approval reform and further encouraging innovation We will encourage clinical value oriented drug innovation, and once again reiterate that "we will speed up the review of applications for registration of innovative drugs that have better therapeutic effect on major diseases, rare diseases, diseases of the elderly and children, have independent intellectual property rights and are listed in major special projects of the national science and technology plan" At the same time, this document defines the priority review area of generic drugs - for generic drug registration applications, drugs that are under clinical supply, insufficient market competition, and affect the accessibility and affordability of public drugs, children's drugs, drugs for rare diseases and other special groups of people, as well as other drugs that are determined to be urgently needed by the market value evaluation, priority review shall be carried out In 2015, the announcement of the State Food and Drug Administration on several policies for drug registration review and approval (No 230) made it clear that "application for registration of innovative drugs for prevention and treatment of diseases such as AIDS, malignant tumors, major infectious diseases and rare diseases" could be queued separately to speed up the review and approval 2016: the first National Expert Committee on diagnosis, treatment and guarantee of rare diseases was established by the health and Family Planning Commission On January 4, the official website of the national health and Family Planning Commission of the people's Republic of China announced that: in order to strengthen the management of rare diseases, promote the standardized diagnosis and treatment of rare diseases, guarantee the basic needs of rare disease medication, and safeguard the health rights and interests of patients with rare diseases, the national health and Family Planning Commission has established an expert committee for the diagnosis and treatment and protection of rare diseases of the national health and Family Planning Commission The responsibilities of the expert committee are: To study and put forward the definition and range of rare diseases in line with China's national conditions, to organize the formulation of technical specifications and clinical routes for the prevention and treatment of rare diseases, and to make suggestions on the prevention, screening, diagnosis and treatment, medication, rehabilitation and security of rare diseases The term of office of the expert committee shall be three years from the date of spontaneous communication 2016: opinions on solving the problem of pressing the application for drug registration to implement the priority review and approval included the application for drug registration with obvious clinical advantages in the scope of "priority review and approval" For rare diseases or other special diseases, an application may be made to reduce the number of clinical trials or to avoid clinical trials when applying for clinical trials According to the needs of technical review and the actual situation of Chinese patients, the drug review center shall make review opinions on whether to agree with the application 2017: relevant policies on encouraging innovation of pharmaceutical and medical devices to accelerate the review and approval of new medical devices on the market (Draft for comments) (No 52-55) No 52 document: support research and development of rare disease treatment drugs and medical devices The Department of health and family planning shall publish the catalogue of rare diseases and establish a registration system for patients with rare diseases Applicants for rare disease treatment drugs and medical devices can apply for relief of clinical trials and accelerate the review and approval of medical devices for rare disease treatment For rare disease treatment drugs and medical devices that have been approved to be listed abroad, they can be approved to be listed conditionally, and relevant research can be made up within the specified time after listing Document No 55: improve the drug test data protection system When submitting the application for drug listing, the applicant may submit the application for protection of test data at the same time For the innovative drugs approved for marketing, the data protection period is 6 years; for the innovative drugs, the data protection period is 10 years; for the new drugs, the data protection period is 3 years Opportunities and challenges such policies as priority review, special approval, reduction of clinical trials, proposed approval for listing, extension of data protection period, and expanded clinical trials will undoubtedly promote the research and development of rare disease drugs and market access However, we still face many challenges How to make a list of rare diseases? Are drugs for rare diseases the same as drugs for orphans? What is the scope of equivalence? What are the differences between orphan drug efficacy and safety evaluation and other drugs? How to assess risks and benefits? Minister Gao Chenyan thinks these are all problems that need to be solved in the treatment of rare diseases Zhang Jie reviewers stressed that the review of rare drugs requires comprehensive consideration of the incidence rate, severity, drug accessibility, the basis of foreign listing and China's medical practice At present, the whole society is paying more and more attention to rare diseases, the industry is no longer only focusing on high-profit products, the scientific research community is gradually deepening the understanding of rare diseases, and the drug review and approval policy is gradually warming We are confident to achieve the common goal: to make more patients with rare diseases available as soon as possible!