Medical network - on January 13, 2014, a campaign to help the rare disease of muscle atrophy in patients with amyotrophic lateral sclerosis (also known as "frozen") of the ice bucket challenge activities popular in the world, make a lot of people awareness of the concept of "rare diseases". Having fewer single disease, rare disease medication use is also known as "orphan drug" designation. How to make such a handful of people can also enjoy the welfare of the community and care? On December 21, 2016, the state council executive meeting to determine the key tasks during the period of "much starker choices-and graver consequences-in" deepen reform, put forward to improve the drug supply security system, support low-cost drugs, "orphan drug" designation, such as children's drugs production.
At present, the drug supply security problems which rare disease? What are the cause of these problems? What are the possible improvement way? With these problems, the economic daily reporter visited patients, experts, drug companies and the relevant departments, etc.
Innovative "orphan drug" is hard to find "made in China"
Recently, the reporter saw in Beijing mucopolysaccharides product storage disease in patients with type I laugh. Mucopolysaccharides storage product syndrome is a rare recessive genetic disease, patients' body whole body stiff joints, corneal opacity, hepatosplenomegaly, respiratory congestion and other symptoms. Conditions to smile has brought great pain and inconvenience, "I can't even wash and wear socks". At the same time, zhang also faces a helpless smile the reality: domestic mucopolysaccharides in patients with no cure for a long time. At present, there is no effective drugs for the disease types of domestic, thanks Marlene Aldurazyme produced by our company by the United States is against the disease type I wonder drug. It went public in 2003, however, did not introduce the domestic.
With a smile situation there are many similar patients. Reporters visited many patients and experts found that the current domestic many rare diseases totally dependent on imported drugs, drugs, if not listed in the domestic, face no cure situation, innovation "orphan drug" difficult to see "made in China".
Rare disease control and prevention in shandong province association of hang jx, increasing domestic drug firms "orphan drug" research and development capabilities, production of "made in China" in the "orphan drug", is the basic way to solve the domestic rare disease medication plight. But compared with developed countries, our country "orphan drug" lagging in research and development ability, innovative "orphan drug" output is almost blank. Capital, researcher at the institute of pediatrics Fang song for the economic journal reporter said: "the current innovative drugs in the world" orphan drug "has accounted for about half of proportion," orphan drug research and development ability has become a research and development of medical field innovation ability is an important embodiment of. In this regard, our country is in a very awkward position."
What are causes of the gap? Non-profit organization development, director of the center for rare diseases such as huang think, domestic drug firms "orphan drug" research and development, largely due to the lack of incentive mechanism. Several countries in Europe and the us has a different type of incentive mechanism to promote research and development of "orphan drug" designation.
Related research literature shows that before 1983, the United States of "orphan drug research and development" also struggling. Turning point in the United States the orphan drug act, published in 1983. In 2002, the United States, the rare diseases act, make the research on rare diseases, with a clear legal protection rare disease research fund also increased gradually, makes the United States developed "orphan drug" security system will be improved. At present, the research and development of American drug companies in the "orphan drug" enjoy clinical research grant funds to support the whole process and the tax credit, at the same time, the drug post-marketing, can give the enterprise market monopoly period of seven years. Since the orphan drug act, the United States "orphan drug" registration number increased significantly. From 1972 to 1972, only 10 "orphan drug", from 1983 to 1983, there are nearly 500 "orphan drug" designation, with annual sales of more than $40 billion. At the same time, the policy of "orphan drug" also attracted the world many countries and regions to follow.
In contrast, the current our country special legislation for rare diseases problems or blank, "orphan drug" policy only referring to drug safety supervision system, etc. Several drug companies told reporters that the national level of basic unified pharmaceutical r&d incentive policies in innovative drug level, drug companies to get the incentive, "orphan drug" can only with other innovation drug indistinguishably into medicine relevant incentive policies for channel. Listed almost the same cost of two classes of drugs, incentive mechanism and no difference, but the market demand is very different, and drug companies to research and development focus on the market demand for it is not surprising that more conventional drugs.
Since 2006 the National People's Congress zhao-qi sun has insisted on rare diseases relevant legislation and Suggestions. , in his view, to change the status of the domestic research and development of "orphan drug" lack of power, must through the special legislative policy tilt, "special legislation to speed up the rare diseases and rare disease is to solve the current difficulties and 'orphan drug research and development lag problem of the most pressing needs". With positive policies to promote market forces to solve the problem of "orphan drugs" research and development and production, the view is also Shared by many experts and scholars.
"Orphan drug" designation, the world's market is in a stage of rapid growth, let our country medicine enterprise, however development lag is missing in the huge "orphan drug" market share more profit opportunities. Medical market research institutions Evaluate Pharma released 2015 "orphan drug" market report shows that the current global "orphan drug" market has nearly 12% annual growth rate, by contrast, in the first half of 2015, the global generic drugs market growth rate is only 5.9%. Report predicts that by 2020, the global "orphan drug" sales will reach $178 billion, "orphan drug" designation in the proportion of the total amount of all prescription sales is expected to more than 20%. The world's largest pharmaceutical giant also aim at the market, such as mergers and acquisitions way to expand their share in "orphan drug" market. At the same time, our domestic drug firms production "orphan drug" had almost are generic drugs, medicine gap huge profit margins and innovation, lead to drug companies in our country is difficult to get high profits in the "orphan drug" market, its innovation ability and market value has been greatly restricted.
Higher prices could "orphan drug" into the health care
For drug firms, innovative drug r&d often require huge investment. And "orphan drug" market demand is very small, drug firms to profit by selling covers the research and development and listing costs, and get a good return, tend to use patented drugs price set too high.
Aldurazyme mucopolysaccharides drugs, for example, according to the listing price, each patient drug costs at least $2 million a year, which makes patients at medicine market. A smile tells a reporter, even if there are ways to buy medicine from abroad, also could not afford, the price is too expensive, so I can only do a regular check, the body which organs appear problem, do some local treatment." Zhang laughed, said the she is able to link to the domestic patients have more than 300 people, treatment condition basic similar with her.
Thanks Marlene Cao Wen said China market access manager, drugs, if can't go into medical insurance directory, patients are difficult to use drugs in China. "At present in domestic promotion orphan drug costs, including clinical trials and register for examination and approval of investment, health care, patients with medicine and so on, at the same time Aldurazyme prices high, almost impossible to rely on patients out-of-pocket medical, must under the medicare payments to get market, domestic currently does not have such requirement." Cao Wen said Aldurazyme listed in several countries and regions, is through into the medical insurance directory, and patients to reduce the economic burden for the market. Cao Wen also said that in view of the Aldurazyme listed on domestic approved, drug companies with multiple local government departments to communicate, but there is no progress, said all the relevant departments around the drug after get registered qualification to consider whether incorporated in the medical insurance directory.
In our country, because of the rare disease specific generally do not belong to the basic drugs, whether in health care decisions generally relevant departments in many places. But for places, to determine whether drugs into health care should be carried out after the drug registration and the relevant calculation, therefore, in accordance with relevant provisions of the both, the practice of across departments.
Promote more "orphan drug" into the medical insurance directory, pressure have how old? The ministry of human resources and social security, according to data published in China in 2015, the urban basic medical insurance fund revenue totaled 1.1193 trillion yuan, 931.2 billion yuan, at the end of town primary medical treatment is the balance as a whole fund is 811.4 billion yuan RMB, earning more than spending, balance the number average pay is about 10 months. 2009 people club department, the ministry of finance issued "on further strengthening the basic medical insurance fund management guidance", points out that as a whole the area town worker is basic medical treatment insurance balance as a whole fund accumulated in principle should be controlled in 6-9 months average pay. Slightly higher current balance principle range, showing the current medical insurance fund in our country still has strong ability to pay.
However, there are a lot of concerns about voice. Hang jx believes that because of China's rare disease types and large population, the high price of imported "orphan drug" designation, if mass into health care, is likely to gradually influence to other common disease, frequently-occurring disease in patients with medical payments, "in fact, this kind of situation has appeared in Europe". At the same time, some experts believe that high medical insurance fund balance of all the differences as a whole the area is covered by several, in some areas, the balance number even has reached more than 20 months average pay levels, but at the same time, many areas have to make ends meet, the regions are quite different.
Pharmaceutical affairs management at the Beijing university school of medicine and clinical director of the department of pharmacy Shi Lu says, because all parts of the country medical treatment as a whole and fiscal income level differences, if the country can expect a macro policy, according to the region around the incidence and their construction assistance system, such as the level of economic development is a more appropriate way of assistance system construction.
Parts of domestic economic development level is higher, in fact, have begun to gradually explore the more rare diseases into the medical security system. In 2016, zhejiang will include frozen disorder, gaucher disease, benzene acetone urine disease, rare diseases in medical security system. Gradually frozen disorder and benzene acetone urine disease treatment for ten yuan a year, gaucher disease's treatment "and" like in domestic purchases will be expected to each patient at least 2 million yuan a year, and need lifelong medication, the disease to be incorporated into health care system, greatly reduce the economic burden of patients. Is understood to be included in scope of protection of rare diseases, by the basic medical insurance, serious illness insurance, medical treatment, fiscal share step by step a special funds such as resolving the medical costs of compliance.
But in most areas, compared with the patients needs, assistance system construction progress is slow, difficult, expensive medical phenomenon is very outstanding. Two sessions in 2016, zhao-qi sun Suggestions on the national promotion of rare diseases in zhejiang province medical insurance system, to alleviate the burden of patients, "a lot of patients and their families because of sickness poor, Chinese due to illness, become a significant part of our country special poverty".
At the same time, some experts believe that if a high around the "orphan drug" can further into medicare catalog, easing the burden on patients, help to gain more market, "orphan drug" research and development of domestic drug firms is also a powerful incentive.
Listed are looking forward to the "green channel"
In accordance with the relevant provisions, in innovative drug patent protection expires, pharmaceutical factory to produce generic drugs, but require consistency evaluation process, qualified rear can appear on the market. Generic drug prices are usually lower than the innovation medicine many, for many patients with alternatives. However, reporters found that for many rare diseases, use of generic type "orphan drug" also faces many difficulties. This is because the drugs listed the cost is not much difference with other conventional drugs, and generic low prices also lead to drugs low-margin, difficult to cover the listed cost, inhibit the listed companies to promote drugs.
Tao zi is a rare congenital adrenal cortex hyperplasia disease the patient's mother had, for a long time, she has been beset by twists and turns of the road to buy medicine. Hydrocortisone is the treatment of congenital adrenal cortex hyperplasia of essential drugs, in the domestic, Shanghai medicine sym pharma co., LTD., also produce alternative medicines, but only 20 mg dose of the product. Tao zi said: "this disease for the dose of medication accuracy requirement is very high, drug for children is generally 5 mg dose, 20 mg of drug for adults only, if the drug is not accurate, will lead to major side effects." The appropriate dose of the drug can only be bought abroad. Tao zi said she had communication with foreign drug companies, the other says, because the drug profits is too low, can't cover the listed cost, so don't want to enter the Chinese market. She can only act as purchasing agency drugs from abroad, it brings to the tao zi made a lot of policy risk.
To this, the reporter contacted the prescription sym pharmaceutical factory. Prescription sym said, if the company get 5 mg or 10 mg specifications of hydrocortisone acetate production approval, and the market demand, the company also willing to organize production, timely meet the patient's clinical needs. To get production approval, however, the development of drugs and the examination and approval period is longer, the early stage of the cost is very big, if the market is very small, business will have to take into account the burden of a problem.
The same dilemma also reflect on the "new with older drugs". Tao zi is a rare disease, tuberous sclerosis patients mainly taking medications for generics sirolimus. Despite the obvious curative effect, but sirolimus specified indication does not contain tuberous sclerosis, in this case, the need for treating patients with doctors declare research projects. In addition, Beijing, shenzhen and other places some specialized subject hospital outpatient service after through ethical association reagent for the record, also can prescribe medicine, but the patient is a reagent is identity rather than the patient's identity, risk agreement is signed with the doctor, the patient medication process is too complicated.
"We want to be able to build a new 'old drug use, can let more patients on the drug, because it is cheap, patients can afford." Tao zi tells a reporter, although there are four domestic pharmaceutical companies in the production of sirolimus, only the north China pharmaceutical, reiterated his desire to cooperate with the rest of the drug companies, with a negative attitude "so north China pharmaceutical also have very big concern, because if it spent much cost to promote drug indications, is beneficial to the other drug firms, and other drug companies already in many hospitals, higher market share".
One side is the lack of incentive mechanism of the obstacle, the other side is related costs. North China pharmaceutical relevant personage JiaoJinPing said, according to the current policy, to adapt to the new card, generally needs about 3 years time of examination and approval, the enterprise needs to drug expenses, clinical treatment, etc., in terms of money and time to need a lot of cost, calculate the balance of payments account, other companies also didn't have a lot of enthusiasm ".
In accordance with the relevant regulations, the new drug clinical trials often need to get enough support cases. As rare diseases often lack enough cases, in a rare disease drugs before entering the market, pharmaceutical companies will submit exemption "clinical trials" or "sample size reduction", but every time need to register new drugs are submitted separately. ZhuGuYing sanofi group communication media department, said the current way of examination and approval will greatly increase the time cost of drug companies, "hope explicit laws and regulations, in order to make clear the principle of rare disease drug can obtain free clinic, or directly to draw lessons from foreign data, and carries on the review of priority, and does not need to apply for alone time".
In fact, to promote the "orphan drug" listed, the state food and drug administration issued a series of new reform of examination and approval document, proposed in view of the rare disease such as special group medicine give preference to speed up the review, the review, but the actual effect is not obvious. Yellow as think, first, because these policies lack of rules, is not yet clear how to perform the relevant authorities. In addition, China's rare diseases drugs and medications for examination and approval of the same channel, lack of separate paths for examination and approval, the examination and approval efficiency also will improve.
(the "tao zi" and "tao" is not her real name)
Basis of diagnosis and treatment of disease
Work began on the road
Promoting the "orphan drug" examination and approval efficiency, green channel is crucial for examination and approval of "orphan drug" designation. Therefore, how to recognized rare diseases and "orphan drug" designation, is also an important factor to influence policy. At present, our country for rare diseases is not yet clear official definition of different countries around the world, according to their own situation, has a different definition. Shi Lu says: "the different definitions, involving the implementation of related policies and measures for the object is different, so how to define a rare disease, not only involves the incidence of a disease, is also an economic problem."
On this question, one step ahead in Shanghai. In February 2016, Shanghai issued "Shanghai main list of rare diseases (2016 edition), 56 kinds of disease were included. Relevant state departments also began to notice this problem, in December 2015, the national health and family planning commission has formed a rare diseases diagnosis and treatment with security expert committee. National health development planning commission for the economic journal reporter said, "at present, the health development planning commission depends on the expert committee has carried out the following work: one is to start the first selection catalogue of rare diseases, according to the 'technical methods of diagnosis and medicine cure or control obviously relieve symptoms, social reflect the prominent' principle, for the first batch of rare diseases disease to the selection criteria and procedures, and start the selection work; the second is to carry out the rare diseases and drug security carding and research, the expert committee has initially rare diseases and drug security situation in our country, began investigating rare diseases diagnosis and treatment and medication status in our country, to study and draft the measures for the management of rare diseases, to improve the level of diagnosis and treatment and the safeguard and advice are put forward. The committee of experts will be based on duty, the rare disease definition and scope of disease".
At the same time, some experts believe that to speed up the "orphan drug" review of the examination and approval, should be very detailed data base of support, to ensure the effectiveness and safety of drugs, the "orphan drug" in our country, research and development of short board, at present, our country epidemiological data distortion seriously, research is weak. So some experts advocate and promote rare diseases registration system unceasingly, to help establish database of rare diseases. "Building a database can complement the lack of epidemiological data, improve the" orphan drug "scientific basis and core knowledge, also can provide quantitative data support for drug development, makes the drug companies more reasonable in drug design." Such as huang fang said.
Inspired, the domestic many rare diseases group also began to collect all kinds of rare diseases related data, including the distribution and the information of diagnosis and treatment of rare diseases, "orphan drug" research and development, production and sales information, as well as the "orphan drug dosage, the clinical use of information, etc., in order to promote the" orphan drug "in the future clinical trials carried out effectively. And rare diseases prevention and control association of shandong province and other rare diseases at the provincial level agencies are organizations nationwide rare disease a baseline survey and so on basic research, and obtained the financial support of national science and technology support plan. |
