Rare medicine research and development: in the solitude

The 2016-08-10 16:06:34

Source: of chaste tree hunan - hubei daily (wuhan)

Reporter cui more than yu Hu tendril correspondent li-li CAI Xu Hui

5 kinds of domestic rare medicine be vividly portrayed

From a rare disease and helpless eyes, big pharmaceutical (China) co., LTD. Read a kind of responsibility. Yesterday, the enterprise, according to the 5 kinds of rare diseases are developing drugs.

Ammonia hexene acid, used in the treatment of "bowknot of tuberous sclerosis (TSC). This is a kind of brain, eyes, heart, such as multiple organ with benign tumor of genetic disease, characterized by epilepsy, developmental delays, etc. A TSC patients per 6000 people, China at least 100000.

At present, the drug market in France, Korea, Canada, has a domestic enterprises applying for import, not approved.

"This means that we should be the drug in imitation, benefit domestic patients." Broad medicine Xie Guofan chairman said, "now, we are in ammonia hexene acid formulation process zoom in trial production, next year is expected to declare clinical trials."

Intense, several other rare medicine also clear handsome eyebrow: used in the treatment of Ⅲ or Ⅳ pulmonary artery high pressure wave raw temple, is about to start the bioequivalence test; Treatment of high blood ammonia, glutamic acid, entered the stage of stability test; For idiopathic thrombocytopenic purpura ai curvelet palmer, baida against mdr-tb quinoline piece, also took to the pharmaceutical research track.

Rare patients to use drugs to maintain life, due to the rare drug dependence on imports, many patients give up treatment for overburdened.

In September 2014, the broad medicine research Chinese acad med sci and China in Beijing signed the drugs for rare diseases "strategic cooperation agreement, the Chinese native medicine companies open scale of rare diseases drugs research and development of" ice-breaking trip."

Less than two years, surprises, 5 kinds of major progress was made in domestic rare medicine research and development.

Everything can only be groping forward

Assistant general manager of the broad medicine Yang Bo introduction, the world health organization (who) defines a rare disease with patients for 0.65 per thousand population - 1 ‰ disease or pathological changes, the existing 5781 kinds of rare diseases, U.S. data covers more than 6800. And our country, there is no clearly defined so far.

In addition, compared with more than 30 foreign countries policy stimulus rare medicine development, although some policies in our country, but the lack of systematic. Such as the 2007 "drug registration management method" is put forward to new drugs to carry out special approval "to" cure disease, 2012 will "encourage rare diseases medicine research and development," write "national drug safety" twelfth five-year "plan", but no matching rules, difficult to fall to the ground.

Rare medicine demand is narrow, r&d risk big, the local drug firms focus on power is insufficient, have fled.

"As a drug firms walked out of the taihang mountains of red, ought to bear." Yuanda pharmaceutical chairman Xie Guofan said.

But from an existing small tablets, push down its distribution ratio, prescription craft, etc., talk easy.

The term is commonly used medicine "touch" is the r&d team. All start from zero, no experience, can be used for reference only feeling.

Ammonia acid hexylene team members Tian Junfeng that: "at first, we choose grignard reaction route, but two or three months test, repeatedly try. Originally, grignard reagent in water, air humidity is high, and wuhan test can not succeed."

He introduces, rare medicine chemical molecules play a "smoke screen" is good, experienced tester, it is hard to identify impurities or drug molecules, puzzled for half a year, from hundreds of times test to find a niche in the solvent, precipitation crystallization required.

Rare medicine wide prospect of market

Enterprise must consider development efficiency, the market prospect of rare medicine, the broad medicine have been made.

"We have to provide novartis wave raw piece of API production, accidentally learned that the drug sales over $1 billion. In fact, rare diseases is relatively rare, in absolute terms, the world rare disease exceeds 350 million. This is a huge market, and less competition." Yang Bo said.

At the same time, the rare medicine also attracted much attention. In 2014, the National People's Congress standing committee, the financial committee of the National People's Congress members ri-tu wu suggest, in the national drug reserves increase treating specific diseases, the variety of rare diseases, and through timely adjust, ensure the hospital drug supply. Also has the National People's Congress on behalf of the proposal, as soon as possible to develop our country "law on the prevention and control of rare diseases, speed up the rare diseases and drug registration for examination and approval, and research and development production.

In January, the general office of national health development planning commission decided to form a "rare disease diagnosis and treatment with security experts committee, promote the standardized diagnosis and treatment of rare diseases and ensure rare diseases basic demand; In march, the state council general office issued the guiding opinions on promoting the healthy development of the pharmaceutical industry, pointed out in the field of rare diseases, key generic market potential, foreign patent medicines for urgent clinical needs. "For a rare drug research and development, the examination and approval, production and other incentives, like curling sunshine, light up the future." Xie Guofan said.

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What is a generic

Generics, it refers to the original drug in dosage, safety and efficacy, effect and indication on the same kind of imitation, have lower health care costs, enhance the level of medical treatment, and other important economic and social benefits. Patent medicine protection expires, other countries and pharmaceutical factory can produce generic drugs.

China has about 170000 drug approval number, belong to chemicals. There are 107000, more than 95% of whom are generic drugs. At present, the generic basic can meet the needs of public administration, but face problems such as quality, low level repeated production.

For improving the quality of generic drugs, 2012, national food drug administration began pushing for generic drugs quality consistency evaluation work. In February this year, the general office of the state council about the opinions of the consistency to carry out the quality of generic drugs and curative effect evaluation ", mark generics consistency evaluation into the implementation stage.