“There are currently more than 7,000 rare species known in the world, and the number of affected people is more than 300 million. The diagnosis and treatment of rare diseases is far behind common diseases. Only 5% of patients have medical treatment, but Most of these costs are extremely high, most patients are unbearable. “
Recently held in Beijing, the 12th International Symposium on Rare and Orphan Drugs and the 6th China Rare Disease Summit Forum, Peking Union Medical College Hospital Vice President Zhang Shuyang disclosed the above data.
State Food and Drug Administration Drug Evaluation Center, a senior adjudicator Zhang Jie is further added, “more than 7,000 kinds of rare diseases have a specific therapeutic effect of only 400 kinds of 500 kinds of drugs. China’s listed less than 20%, the vast majority of imported drugs. ”
Fortunately, China’s rare disease drug import approval speed is accelerating, the relevant support policies have also been introduced, according to Zhang Jie introduced, there are more than 130 kinds of rare drugs in the country was approved.
According to Zhang Shuo Yang’s introduction, the official definition of the first batch of rare disease directory is also being studied, including more than 100 kinds of disease, then the relevant rare disease drug import approval and health insurance payments or will benefit.
directory priority included in the treatment of rare diseases
7000 kinds of rare diseases, the official directory will include which, to comply with what criteria, why not a one-time income list?
Zhang Shu Yang expressed, in the first directory contains about 100 kinds of rare diseases, and the future of health insurance policies linked to at least have some way to solve (the patient burden is too big problem), “otherwise vain days, touching the ground Zhang Yi also stressed that the review of rare drugs need to consider the incidence of disease, severity, drug accessibility, the basis of foreign listing and China’s medical practice, etc.
At present, there is no official definition of rare diseases and relevant laws and regulations in China.
At present, there is no official definition of rare diseases in China and the relevant laws and regulations. > National Food and Drug Administration drug review center biological products clinical department minister Gao Chenyan also expressed confusion: rare disease directory how to develop? Rare disease medication and orphan drug is the same? What is the equivalent range?
We hope that the health management department issued a list of rare diseases, the clinical needs to tell us The Among the various policies, there is no word for ‘orphans’, and all policies are rare drugs. But in the end which is rare disease? “In the actual operation, since the above-mentioned part of the guiding principle has not yet been refined, and because the official definition of” orphan drug “has not yet been established,” which medicine is orphan drug “Prerequisites are still a problem, and priority is given to” priority “is more elusive.
” No state, no official disease definition, no national policy of support, almost no health insurance claims. Zheng Weiyi, chairman of Nanjing Connaught Pharmaceutical Technology Co., Ltd., who was the expert of the National “Thousand Talents Program”, described the status of “orphans” in China in an interview with surging news.
It is in view of this, this rare disease official directory of the development, will have priority included in the treatment of rare diseases.
orphan drug Medicare payment is still to be broken
“rare disease drug research and development The cost is 25 times the common drugs, so many drugs desperate. “Social and economic conditions, market access and payment policies are the core factors that determine whether a patient can get a therapeutic drug.”
Some patients because of the economic burden is too heavy to give up medication.
For pharmaceutical companies, orphans can not be included in the medical insurance, which means that the market more The small amount of research and development costs are difficult to recover, and unprofitable.
Qingdao Social Insurance Research Association, vice president and secretary general Liu Junshuai pointed out that China’s rare disease protection is a point-like breakthrough, but also There is no complete breakthrough in the depth, such as rare diseases in the concept, definition, directory, legal and epidemiological data and other basic parts, did not achieve a breakthrough.Second, the entire security system, including health insurance, relief, charity, are not The formation of a steady-state structure, only part of the local breakthrough, only part of the local breakthrough in the disease, only some of the local part of the patient’s organization some breakthrough.
Liu Jun-shu recommended, rare disease protection priority Practice, should first be selected to obtain a strong sense and easy to operate matters, the construction of national treatment center, focusing on the disease Diagnosis, from the availability of medical services to start; can cure rare disease treatment, focusing on drug protection, from orphans can pay to start; refractory rare disease benefits, focusing on comprehensive protection, from the full life cycle security to start ; Clinical epidemiology of the study, focusing on basic data, starting from the easy to operate the basic factors in the practice of slowly hatching rare disease directory.
Previously, the relevant departments have repeatedly for the “orphan drug” issue of documents.
Gao Chenyan said that in 2015 – 2017, the state introduced a series of policies to encourage innovative medical equipment for the draft (No. 52 ~ 55 bulletin), hoping to promote rare disease drug research and development. For example, Bulletin No. 55, the applicant in the application of drugs listed in the application, can also submit experimental data to apply for protection. On the both belong to the innovative drug and is a rare disease medication, children’s special medicine, given 10 years of data protection period; is a rare new drug treatment drugs, children’s special medicine, given 3 years of data protection period.
Medicare payments are still difficult.
The future is likely to be similar to the practice of Zhejiang, Shanghai and other places as a sample, by the local first to explore.
The meeting also revealed another good news that the world’s first drug for the treatment of rare disease C-type menopausal disease (NPC) MacGeda capsules Ze Weicai, will be global The lowest price in China.
“lowest price” for about 30 million a year, but at least “no drug available” dilemma will soon be resolved.