Washington of Xinhua News Agency on May 8 report (Deng Xian comes to reporter Xu Yuan) the gene therapy hopeful of disease of sweeny of baby of a kind of OK and effective treatment appears on the market in the United States, but this one price or the therapeutics that are as high as 2 million dollar also cause controversy in the United States, fix a price around medicaments especially and problem of insurance manage compensate.
Name of therapy of this one gene is Zolgensma, by enterprise of the famous medicine that cross a state Nuo China is rolled out. American food medicines and chemical reagents controls management board (FDA) will examine and approve a decision at be being made to its this month, japan and European Union also hopeful is made inside this year examine and approve a decision. Analytic personage thinks generally, because this one therapeutics already gathered a large number of clinical data, hopeful is in beautiful day Europe this year 3 big markets are obtained batch.
Gene therapy enters human body through guiding active gene, will treat the disease that causes because of gene blemish. The amyotrophy of spinal cord sex that Zolgensma place treats disease, it is the disease that because of the patient lack controls sarcous key gene one kind and causes.
If be not treated, disease patient meets amyotrophy of spinal cord sex to die before two years old normally. In clinical trial, 12 first little patients that accept treatment already exceeded two years old at present, among them most little patient already can look up by oneself, have a meal with the mouth or sit alone stand.
On April 21, on the parade of paschal beautiful cap that holds in American new York, a children looks the doggie that dresses up meticulously up and down. Xinhua News Agency is sent (Guo Ke is photographed)
But the problem depends on, gene therapy cost is high. Accept of · of presiding apparitor gas pulls Nuo China laborious expresses, zolgensma price may be in 1.5 million to 5 million dollar between. The personage inside course of study predicts final price generally to may be controlled in 2 million dollar.
Nuo China thinks, zolgensma has amyotrophy of sex of remedial spinal cord the tremendous potential of disease, because this is as high as the price of 7 digit,be reasonable. Industry analyst thinks, zolgensma hopeful gives Nuo China every year to bring profit of about 2 billion dollar.
To gene therapy, the price is high be not special case. American food medicines and chemical reagents controls management board 2017 the bottom approves Luxturna of therapy of first gene of American, its price is as high as 850 thousand dollar. Therapy of this kind of gene is used at treating specific transmissibility eye disease.
American food medicines and chemical reagents supervises management board to predict, to 2025, the gene that approves every year and cellular therapeutics hopeful are amounted to 10 go to 20 kinds. As gene therapy large-scale appear on the market, high price also causes the tremendous controversy of medicaments price and insurance manage compensate.
The difficulty that medicaments fixes a price depends on how reaching price consensus. At present common price method is " cost effectiveness " price law, the healthy life estimation that is a patient each years namely is certain benefit, the total benefit that brings through computational medicaments next will undertake medicaments fixes a price.
But it is very difficult that the healthy life that is a patient undertakes appraise is worth. Nuo China thinks of Zolgensma " cost effectiveness " in 4 million to 5 million dollar between. But blame seek profits the organization is clinical the analysis that comments on an institute with economy thinks, of Zolgensma " cost effectiveness " should not exceed 1.5 million dollar. Both appraise is worth six to one, because Nuo China gives a patient each years the appraise watch of healthy life,be tall.
American health is sure to plan union spokesman Kathleen Tangnasen to think, the price is a problem really, the medicaments that can afford because of nobody is no-good. And another issue that accompanies with price photograph is safe, as prospective more gene therapy enters the market, at every turn the remedial charge of millions dollar is to insurance company hard susceptive.
For this, the pharmacy company of Nuo China and therapy of gene of other research and development suggests, with managing in installment compensate means reduces cost; Or carry out " be based on value manage compensate " insurance policy, namely insurance company manages beforehand compensate treats cost, if cure disables an insurance company can obtain partial refund.
But these proposals and presence of current policy of insurance of American medical treatment are a lot of incompatible place. For this, the company of therapy of American scintilla gene that rolls out Luxturna is driving American government approval to undertake is sure to reform pilot plan, will attract insurance company to participate in gene therapy.
