Medical network - December 30 little-known, rare people use is about "orphan drug" of life, once again entered the field of public policy.
Premier li keqiang on December 21, chaired a state council executive meeting, by "much starker choices-and graver consequences-in" health and health planning, deployment of the next five years we will deepen reform of the medical and health system. Meeting to determine the key tasks during the period of "much starker choices-and graver consequences-in" deepening reform. One of them, is to improve the drug supply security system, support low-cost drugs, "orphan drug" designation, such as children's drugs production.
This is a piece of encouraging news.
Director of the center for the development of Chinese rare diseases, such as the party of the first financial journalist huang said it is great for rare diseases group, hope relevant rules can come as soon as possible, to support the orphan drug production can implement.
"Orphan drug" designation, which is a rare disease drugs. The world health organization (WHO) defines a rare disease with patients accounted for 0.65 ‰ ~ 1 ‰ of the total population disease; In the United States, is a rare disease refers to the affected patients under 200000 people (limited to the United States) of the disease. Because of the high development cost, the market is small, so the enterprise research and development of attitude is not positive, "underserved" is the biggest problem facing rare diseases group.
As a kind of rare diseases, 2014 "ice bucket challenge" activity "frozen" people obtained the unprecedented attention, but more rare disease group in suffering in silence, is not known.
From the public to the exploration of business
Yellow as party in rare diseases group has a strong appeal. He is a rare disease, height 1 meter he with pseudo achondroplasia disorder, at the age of three or four, parents find his body, unlike other age child, holding his doctor visits.
Starting in 2008, huang in rare diseases have been engaged in public welfare spread, rare diseases development center in China, established a non-profit organizations hatching different rare disease group, promote the spread of rare diseases, rescue, and legislation.
Confirmation of the world's nearly 7000 kinds of rare diseases, 80% were hereditary disease caused by a genetic defect, about 50% of rare diseases at birth or childhood-onset, about 10% of human diseases.
Rare diseases specialist branch committee, director of the Shanghai medical association schuster mm said that the current known rare diseases, 90% belong to severe illness, but treatment is only less than 5%.
In may this year, huang in party started a new business exploration, he opened a company called root health in shenzhen, as CEO.
While some questioned when he doing business and the public, but such as huang side retorted, "I know the most rare diseases group needs, public welfare and cannot completely solve the actual problem, the rest depends on business." Lack of market is the most patients with rare, "we are patient, we etc."
This decision is let him "reality, China has more than 1000 rare disease, diagnosis of patients with less than 40%, rare disease more than 200000 new born every year, rare diseases diagnosis difficult, confirmed the time an average of more than 5 years, average diagnostic cost more than $50000. Although rare diseases development center will also receive some help, but a lot of problem is not solved, he hopes the commercial path rare patients misdiagnosed, difficult, and to reduce the cost.
Heart is uneasy to rush about, is a rare disease family daily status.
This spring, a children's hospital near the west lake in hangzhou, Mr. Zhou and his wife took her two daughters from wenzhou yueqing, a group of special children's care party. His daughter, eight years old, from the age of 1 was found to have an leite syndrome, "the daughter of eight or nine month will call mom and dad, everything is normal, but in about 1 year old, she seemed to be suddenly stupid, look dull, don't call us anymore." Mr Zhou said the media, the daughter also often rub hands, walk also crooked. Mr Zhou took her two daughters in several local hospital, was diagnosed with epilepsy. Questioned the results of Mr. Zhou took her two daughters to seek treatment. In Shanghai, Mr. Zhou for the first time I heard the word "leite syndrome". "But experts in Shanghai told me that specific diagnosis also get Beijing." After travelling more, through the check blood gene, daughter was diagnosed with leite syndrome.
Root health chief operating officer (COO) Tan Xuesong worked in huawei's 16 years, because of the daughter leite syndrome was diagnosed in 2014 Tan Xuesong from huawei, founded in shenzhen leite, institute of rare diseases, after joining life health.
To help the patient to find the right doctor, is only the first step. Health has such as party, said huang foundation and the national more than 200 top rare disease expert, diagnosis and treatment for the patient referral service is not to collect fees, fees from a third party. Health at the same time, the diagnosis is foundation to help patients achieve the first step, but it's not the end, the future really want to do is the patient after precipitation accumulation of large data, research cooperation and drug companies for drug development and disease.
Orphan drug research and development of the domestic cold abroad
Orphan drug research and development is the key to rare diseases one's speciality, is also a lot of drug and domestic institutions don't want to do.
"To put it bluntly drugs for rare diseases in China don't make money," such as party, said huang rare disease variety, but the number of single disease, drugs are generally more expensive, most not included in the health care, the underlying social welfare not shop good, most patients lack of purchasing power, and research and development of new drugs cost time and money, so a lot of companies are not willing to commit, some risk investment institutions are reluctant to invest in research and development of rare diseases.
Throughout the track of the precision medical noninvasive prenatal genetic testing story has almost been finished, the next there are dozens of companies poured into the tumor niche - for investors and enterprises, the tumor is just need.
Even China's largest gene sequencing company shenzhen genomics co., LTD. (hereinafter referred to as "genomics") also had not put the rare diseases treatment in high priority.
Genomics CEO Yin Ye said in an interview with China business, profit is a rare disease, it is a small circuit. Known to most of the rare diseases treatment or concept or in the phase of clinical trial, scores of orphan drug is rarely, want to have a rare disease detection is a difficult process. Rare diseases in China must go the public way, any country rare diseases are government + folk charity relief.
Orphan drug industry abroad, by contrast, is showing a very hot side.
In the past decade, the United States food and drug administration (FDA) has approved 230 orphan drug. Approved in 2015 especially, 47% more new drugs approved to treat rare diseases.
IMS (IMS research company), according to data from the global orphan drug market annual growth rate averaged 12%, general drug is difficult to achieve this growth. In 2020, the orphan drug markets around the world will amount to $178 billion, will account for 20.2% of the world's prescription drug market.
European and American markets, the yellow as said already proved that the orphan drugs can make money, orphan drug industry is very hot. Some small studies of orphan drug companies in developed after merger or acquisition will be big company a high price.
It is important to establish the incentive mechanism of r&d. At present, the United States, the European Union, Australia, South Korea, Japan, and China's Taiwan and Hong Kong, etc., are introduced in view of the "orphan drug" policy support and the rare disease.
The United States in 1983, first the orphan drug act, funding for clinical research, drug registration for examination and approval procedures, extend patent protection, clinical research fee reduction or exemption from the legislative guarantee.
The FDA specifically set up the "Orphan drug Development Office" (Office of Orphan Products Development, OOPD), a new drug to the examination and approval on the policy support, the first thing to submit application to OOPD, concluded that it is for rare diseases can enter after the "Orphan drug" "green channel", get priority review or accelerated review, and exempted from examination and approval.
The European Union, the United States, Japan and other countries the government also provides a large number of research funding support directly. Such as in the United States, by 2009, OOPD, a total of $246 million to fund more than 480 research projects, direct drive at least 43 rare diseases drugs on the market.
In China, drugs for rare diseases behind the lack of motivation is the lack of incentive and protection mechanism. Although in 2012 the national drug safety "twelfth five-year" plan ", the clear encourage drug research and development of rare disease; Published in 2014 "on deepening the reform of drug approval review's opinions on further encourage drug innovation put forward again to major disease, a rare disease, the elderly and children has better therapeutic effect of drugs given to speed up the review.
"The problem is not detailed rules for the implementation," yellow such as party, said what is what kind of medicine can speed up the approval, process, can speed up how much, is the lack of details. This leads to the examination and approval of drug companies, there is still no basis.
When the rare diseases drugs into health care
"However, relevant policy is difficult to promote the cause of the rare disease can also be understood." Such as huang fang said, countries face a lot of health and people's livelihood problem, rare diseases is not necessarily the priority; At the same time, for a long time, rare diseases because of disease, patients scattered, fighting for the rights in the weak voice.
Now that the domestic research and development dynamics is insufficient, can be imported from abroad orphan drug? In fact, because of the large population base, China has the largest in the world of rare disease drug market, but the orphan drug drug firms products to enter the Chinese barriers. The scholar statistics, from 2000 to 2000, the United States listed 64 kinds of "orphan drug" designation, China only 16 species; Among them, the Chinese market "orphan drug" there are 10 kinds of totally dependent on imports, 1 kind is given priority to with imports.
Imported drugs for the treatment of frozen disease gradually.
Clinical trials is orphan drug to enter China first, according to the regulation, clinical trials have not less than 200 cases. A rare disease, may have to find more than 100 people across the country. At the same time, to complete the introduction of a full range of application, examination and approval procedures, often need 5 ~ 6 years.
Orphan drug such as huang argued, problems to solve, must have a complete set of solutions, including funding, patent protection, such as tax relief policies.
In zhejiang province as an example, after many negotiations, on January 1, 2016, gaucher disease, benzene acetone urine, gradually frozen disease treatment in the field of three kinds of special drug, was included in the scope of a serious illness insurance drug reimbursement in zhejiang province. Gaucher disease is pretty expensive cost of treatment, targeted drug "praise" and each costs about 26500 yuan, patients' families spend more than 100000 yuan per month. The policy has greatly alleviate the patients' family economic pressure. |
