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When the human probability of things happen in 1/14 billion individuals, it is often not a lucky thing, rare disease patients are such a "special" existence, porcelain dolls (bone dystrophy), Spider-Man (Mafan syndrome), frozen people (sexual muscular dystrophy), moon children (alchemy), marble babies (bone petrification), penguin families (small brain atrophy) ... became synths of their "image".
multiple sclerosis (MS) as a rare disease, good for young and old, according to the 2018 Chinese Medical Association Neurology Credit Association released the "China Multiple Sclerosis Patient Survival Report" estimates that China has about 30,000 MS patients, about two-thirds of patients diagnosed with the disease takes 1 to 5 years, and 12% of patients can be diagnosed for more than 6 years, and most patients do not receive standard treatment.
data shows that the treatment process for rare diseases such as MS is a difficult road, difficult to diagnose, difficult to treat and difficult to obtain drugs is the plight of patients with rare diseases.
Novartis Pharmaceuticals, with its intention of "committing to China" and its "commitment to improving the health and quality of life of its peopleChinese through innovative products and services," has never stopped exploring rare diseases.
novarhua scientists have investigated and studied the treatment of more than 40 rare diseases and are constantly exploring new ways and targets for the use of rare diseases, especially in the development of new MS drugs.
Focus on MS drug use, multiple protection patients in the future August 1, in order to improve the field of rare diseases on the S1P receptament regulator awareness level, open up the field barriers to share resources, Novartis Pharmaceuticals Special Joint Health Media conducted an online seminar on the theme of "THE status and future of MS drug use", this meeting was chaired by the health media executive CEO Wang Na, and a number of heavyweight guests attended the meeting, China Rare Diseases Alliance Executive Chairman, China Hospital Association Vice President Professor Li Linkang special address.
Professor Li Linkang said he was very pleased to have such an opportunity to conduct in-depth exchanges on the issue of MS drug use alone, and greatly affirmed the importance and support of the national government, the China Rare Disease Alliance, pharmaceutical companies and others in the rare disease and its drugs.
In recent years, whether in speeding up the review and approval of rare disease drugs, rare disease drug innovation, drug supply support, or in the rare disease diagnosis and treatment, drug reimbursement, China has introduced relevant policies, Dalian Medical University School of Public Health Professor Yuan Ni in the "domestic and foreign rare diseases and drug reimbursement policy" in the theme speech elaborated on the development of China's rare disease medical security system.
Capital Medical University affiliated with Beijing Tiantan Hospital Professor Zhang Xinghu through clinical reality and vivid case, a systematic analysis of the S1P receptonous regulator - Fingomod treatment plan, triggered a further exploration of MS drugs, MS patients with limited types of drugs, but the new drug research and development path is still developing forward, Novartis Pharmaceuticals in this regard made a good demonstration.
Professor Zhang Xinghu introduced Fingomod's clinical research to the realization of NEDA-4 four-control July 19, 2019, Novartis Pharmaceuticals (China) announced that the State Drug Administration approved Jeringa? (Fingomod) is used to treat multiple sclerosis (RMS) in patients 10 years of age or older, which is currently the only multiple sclerosis treatment (DMT) drug with childhood adaptation, and the only one of the three DMTs currently available in clinical practice that achieves NEDA-4 triple control.
two-role mechanism, fingomod has been approved in more than 80 countries since its global listing, benefiting nearly 300,000 patients.
Looking at the drugs currently on the market and in phase II and III of clinical, the drug development strategy for MS is divided into three main types: new immunomodulants, targeted S1P receptommune regulators and neurolytic agents, of which targeting S1P receptommune regulators are relatively new MS target drugs.
Professor Mei Dan of Beijing Concord Hospital on ms new drug research and development and the content of the new target mechanism also brought a wonderful speech, Professor Medan from China's MS epidemiological data, through the introduction of MS pathological mechanism in-depth analysis to MS clinical development and outcome. professor
Medan affirmed the clinical efficacy of fingomod but the path to the birth of new drugs such as fingomod is often challenging, and Fingomod has been approved by the FDA since 2010 as the world's first oral DMT drug, with nearly 300,000 patients worldwide testing for efficacy and safety, and 14 years of clinical studies confirming the long-term safety of fingomod and the low rate of adverse events.
, Professor Zhong Mingkang of Huashan Hospital, affiliated with Fudan University, elaborated on the challenges and opportunities of MS drug development.
as Professor Zhong Mingkang said, the drug from the first discovery to the application of clinical needs to go through layers of assessment and verification, the clinical research evaluation index of the drug is very critical.
Professor Zhong Mingkang introduced the development process of Fingomod pharmaceutical joint, evidence-based data to support the new development conference, Professor Zhao Zhigang of Beijing Tiantan Hospital affiliated with Capital Medical University presided over and discussed MS drug protection and patient benefits, and also participated in the discussion with Professor Chen Chengjun of Huashan Hospital affiliated with Fudan University, Dong Dong of Xuanwu Hospital of Capital Medical University Professor Huiqing, Professor Huang Dexuan of the Chinese People's Liberation Army General Hospital, Professor Feng Yufei of Peking University People's Hospital, Professor Li Pengmei of the Sino-Japanese Friendship Hospital, Professor Tong Rongsheng of Sichuan Provincial People's Hospital, Professor Yuying Yuying of Xuanwu Hospital of capital medical university, Professor Zhou Yusheng of Nanhua Hospital affiliated with Nanhua University, and Professor Zhang Hua of Beijing Hospital are outstanding experts. More than
experts on MS drug protection and patient benefits of the discussion is well known, new drugs often need to go through a number of preclinical research after a lot of hard work to really enter the clinic, Peking University People's Hospital Professor Feng Yufei specifically recommended that the drug should be more registered after the market research.
Fingomod is listed in our country, the patient efficacy of existing prescriptions is remarkable, as part of the Commitment of the National Drug Administration's Drug Review Center, Novarro has also planned to carry out a PAC STUDY for Fingomod in a number of centers to supplement the relevant Chinese groups of data, will give our clinicians and patients more comprehensive data support, so that more clinicians and patients can be assured of treatment.
after listening to a number of experts, Professor Zhou Yusheng of Nanhua Hospital, affiliated with The University of South China, stressed that the team of clinical pharmacists should also strengthen their participation in clinical treatment and stand in the patient's perspective to ensure the availability of drugs. Professor Chen Also explained that
"in the case of rare disease drugs, the safety of the drug should be guided by clinical pharmacists, and the effectiveness of the drug and the interpretation of the disease mechanism need clinicians and sub-specialists to play a greater role," Chen also stressed that molecularly targeted drugs such as Fingomod can conduct more innovative research.
Notre Dame has never stopped exploring the pace of innovative drug research and development, and after Fingomod, Sinimod was included in the first batch of the FDA in 2019 and was listed as a Class 1 innovative drug in China, which was launched in May 2020 in sync with the world.
, under the dual mechanism of the outer and central, pays more attention to the inflammation control of the outer week, and the early use of the front line during the relapse remission phase will benefit the patient even more.
unlike Fingomod, Sinimod is more institutionally focused on the repair of neurodegeneration in the central region, making it more therapeutic in the population of patients with early signals of progression.
Fingomod and Sinimod's dominant patient population is particularly grateful to the state for the importance it attaches to rare diseases, expressing recognition of the clinical efficacy of Fingomod and looking forward to the price of Fingomod after it enters health insurance.
, after being the first batch of 48 clinically urgently needed new drugs, was officially approved in July 2019 and officially launched in January 2020 and is currently self-costing (recommended retail price of 10,500 yuan/box, 28 tablets a box, one piece a day).
considering the financial burden on MS patients, Novartis pharmaceuticals reiterated that although Fingomod is not yet covered, Novartis is very sincere in its health care negotiations to truly benefit MS patients in our country.
In the future, innovation and development of drugs within reach, Professor Huang Dexuan combined with clinical practice experience stressed that the hospital MDT diagnosis and treatment model should be combined with pharmaceutical experts to further promote the diagnosis and treatment of rare diseases, and suggested that China's MS diagnosis and treatment should be carried out in a central mode.
Zhang Hua stressed the importance of MS early diagnosis and treatment, and pointed out that MS testing, diagnosis, fees and so on should be standardized as far as possible, so as to truly achieve drug access.
Tong Rongsheng re-emphasized the role and value of pharmaceutical services in the diagnosis and treatment of rare diseases, and looked forward to more research on the safety of Fingomod.
" drug monitoring needs urgent attention during the patient's drug use, and needs to pay attention to the symptoms of rebound and how to prevent it.
" Professor Yu Suying has placed special emphasis on drug monitoring and pointed out that the development stage of new drugs such as fingomod is even more so.
As we all know, MS is a long-term progression immune disease, and any immunomodalization treatment for MS patients may result in a recovery of disease activity, which is associated with the volatility of the disease itself, clinically difficult to clearly define disease activity recovery or rebound, and the discontinuation of DMT drugs have a risk of disease activity recovery or rebound.
, since its launch in 2010, Fingomod has been used in nearly 300,000 MS patients in more than 80 countries, and the FDA has reported only 35 rebounds, with a clinically order-of-order rate.
Professor Li Pengmei concluded the meeting with deep expectations about the future of fingomod, "If the efficacy and safety of the drug itself are well supported by evidence-based data, and after access to health insurance to consider the financial burden of patients, I believe that this drug is promising."
"Rare disease drug use is a worldwide problem that requires the multi-faceted efforts of the government, hospitals, pharmaceutical companies and society to promote innovative drug research and development, innovative management model and innovative diagnosis and treatment model", Professor Zhao Zhigang made a final analysis of the wonderful discussion of many experts. At the end of the
meeting, Wang Na, Executive CEO of the health sector, concluded, "In the process of optimizing the implementation of the medical insurance system for rare diseases, I believe that companies with a strong sense of social responsibility, such as Novartis Pharmaceuticals, will continue to bring us good news in the development of new MS drugs."
Tao Ran Source: Health !-- End of Content Presentation -- !-- Determine whether or not to log in to end.