" orphan medicine " Very your patient have way can move March 9, 2005, small lovely Rui landed in human world smoothly. However, little Rui mother will not expect to dream, she and son would step to a tragic experience and road that the hardships will be laid from then on. Small Rui was born in two weeks just now, the hospital called, while saying the neonate sifted checking, small the intersection of blood specimen and test of Rui find benzene third index on the high side amino acid, demand to reexamine. After delivering to the blood specimen again, keep waiting for until the afternoon of April 14, little Rui father brings from the hospital back a news that makes the person heart-broken: The amino acid content of benzene third appears to rise constantly, doubt it is " benzene acetonuria disease " in the test of blood specimen (English is abbreviated as PKU) . The recessive gene that the infant is carried by parents is pathogenic, lack third amino acid hydroxy enzyme benzene in the liver, the internal normal supersession of third amino acid of benzene that unable to make, benzene is turned into the noxious substance promptly, cause brain injury, cause infant's intelligence to be low or dull-witted. Treatment of this disease is long, need all the life; Therapeutic method is complicated, can only eat the specialized purpose-built food, and normal people's food is poison, Arsenicum for child, the ones that can be eaten are vegetables and fruit; It is quite expensive to treat the expenses, need 15,000 yuan at least every year. Small Rui has gone to the hospital to collect blood to test for the third time, make a definite diagnosis of it PKU, little Rui parents must go to the hospital to take and treat the milk power at once, will carry on the all-round treatment on the same day. Nowadays, unless after five years, under the control of medicine,whose name is small for Rui can life like normal child, study, but the treatment of more than five years has already one of low Rui let have no money, how to get to the future way? The parents of little Rui are very vast and hazy. At present, the therapeutic medicine of the rare disease patient of our country basically relies on importing abroad, the country has not ensured the policy to the therapeutic expenses of the rare disease yet, the general patient's family is unable to undertake to treat the expenses. So, most rare disease patients do not have medicine that can be cured or bemoaned in the face of medicine. According to the statistical data of the American association of pharmaceutical industry, develop and go on the market a new medicine, the success rate in Europe is 1/4317, U.S.A. is only 1/6155, it does not mean commercial success in listing that new medicine gets permission at the same time. So, few enterprises would like in " orphan medicine " Invest energy on researching and developing. But once succeed, it is expensive and burning and claiming with it, on the most expensive medicine list of price of " Forbes ", 9 kinds of medicines about the rare disease are listed. Treat the rare disease " Popping night the intersection of hematuria and disease " Medicine most been in soliris for price in the world expensive medicines single one of,last 409,500 dollars annual expenseses therapeutic. Of our country rare the intersection of disease and patient exceed ten million a lot of patient can only bemoan in the face of medicine the intersection of China Charity Federation and rare disease succour staff member of office, say when being interviewed by staff reporter, in five or six thousand kinds of rare diseases confirmed at present, the majority does not have medicine that can be managed, only a little part has medicines that can be managed, but this part of " orphan medicines " But the price is very expensive, the general family is difficult to bear the enormous economic pressure. In order to treat " Ge is thanked the disease " Medicine example, one the expenseses therapeutics of year about for 200,000 dollars,let a lot of patient not can only give up not treatinged expensive expenseses therapeutic. There are 134 in China at present " Ge is thanked the disease " The patient succours and plans to present the medicine project and get the free medicine charitably through rare disease. At the beginning of 2001, Mr. Chen's daughter was found in course of the therapeutic cold liver splenomegaly was unusual, in order to look for the cause of disease, Mr. Chen and wife go around all large hospitals in the town taking the daughter. In May of 2001, measured through the bone marrow, Mr. Chen's daughter was made a definite diagnosis of in order that " the dagger-axe was thanked the disease " . The doctor says, only 1/200,000 of ill rate of this kind of disease, if does not treat in time, it is very difficult for the patient to survive. This news is undoubtedly bolt from the blue. Fortunately, one month later, the doctor tells them, specific drug curing this disease has already been developed out by an American drugmaker, but it is extremely expensive, Mr. Chen can only apply to help to health foundation and this drugmaker of the world finally. After going through the anxious waiting of half a year, the Chen 's met free specific drug at last at the beginning of 2002. Unless after using, return to normal daughter health every index,at last can kindergarten, primary school like normal child. Our country does not have official authority that define to rare disease, have not seen the precise report either in data of kinds of Diseases and ill crowd at present. Define 0.65% of incidence of the rare disease according to World Health Organization. ~1%. Calculating, the rare disease patient exceeds ten million the amount of people at the present stage in our country. But prevent and cure and succour the work of the respect at rare disease, our country makes little progress, links such as the research and development, introduction, producing, selling of rare disease medicines,etc. lack policy support, cause rare disease medicines of our country to have blanks to develop the industry. And " orphan medicine " Because the profit is low, market demand is little, research and develop the high cost, domestic pharmacy enterprises are too profitless to go to produce either, caused a lot of rare disease patients to choose expensive imported medicine or have no medicine to use finally. " orphan medicine " Research and develop the introduction of staff member succouring the office according to the rare disease of China Charity Federation slowly in the whole world, until in recent years, with the development of biotechnology, " orphan medicine " in countries such as U.S.A., European Union,etc. Under the guarantee of the relevant law, just develop the therapeutic medicines of a few several kinds of rare diseases. Because of " orphan medicine " The risky current situation researched and developed, a lot of countries have offered the support on the policy to it. For example Japan implemented " management system of using medicine of rare disease " formally in 1993, the whole course that study can enjoy the intersection of fund and subsidy, derate and preferential examination and approval, medicines check again time is lengthened and the national health insurance is paid the favour at. American medicines administration bureau (FDA) Also " orphan medicine " Registration offer express passway procedure,"orphan medicine " Still enjoy and derate and the market of 7 years monopolizes right. The countries all over the world have encouraged the research and development of the rare disease medicine of biological pharmaceuticals to the laws and regulations of the rare disease medicine, also accelerated the listing of the rare disease medicine greatly. Only fewer than 10 rare disease medicines are listed before the rare disease medicine bill was implemented in 1983 in U.S.A., in December by 2008, the rare disease medicine registered in FDA has already been up to 1951 kinds, having the rare disease products that are sanctioned merchandized reaches 325 kinds. Before medicine code enforcement of rare disease in 1999 of European Union, only there are 8 kinds of orphans medicines that were verified and passed, by February of 2009, 619 kinds of rare disease products have been asserted already, 47 kinds of " orphan medicines " Verified and pass. At the beginning of 2009, " new medicine will register the special examination and approval regulation " to issue and implement in our country, will examine and approve the administration of rare disease to list in the special examination and approval range. However, still the rare disease medicine without independent development and production is listed in our country, the patient can only look forward to the country The other medicines company got registration of the rare disease medicine in China as soon as possible. Three methods prevent the rare disease: Premarital checkup, pregnant and careful, neonate sift checking Is third affiliated hospital pediatrics director Cui QiLiang accepting and writing Guangzhou medical college? Bu the intersection of lemon and north ��, chaste tree of Xing, go straight towards � very <
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