世界で一番高い薬の値段の決め方。How the most expensive treatment decide its price.
Share for Smileの楓子です。
English follows Japanese.
ではなぜこのような高額な薬になったのか？製造コストがすごくかかるということではなく、ノバルティスは下記の理由をあげています。
(1) 10年間スピンラザという既存のSMAに対する薬（4か月に１回服用）を使用する半分の金額
(2)通常の遺伝性希少疾患を治すのにひつような半分の金額
(3)費用対効果（既存の技術を新しい技術で置き換えることで余分にかかる費用を、生存率や生活の質の改善分で割ることで算出する「増分費用効果（ICER）」で評価される）は非営利経済評価機関が小児希少疾患に設定した上限を超えない
このことから、このような価格設定になったのだそうです。少し頭をよぎってしまうのが人ひとりの命にどれだけの価値があるのか、ということ。
利益を上げ、効率化を目指さなくてはいけない製薬会社は対象の患者が多い糖尿病や高血圧のような一般的疾患に焦点をあて研究を進めています。このような珍しい病気に研究費をあて、さらに遺伝子治療薬という最新の治療法の開発を今後増やしていけるようにした最初の一歩だったのはとてもすごいと思います。ただ、これだけの値段で日本のようにほとんどを国が負担するとなるととても大きな問題になってしまいそうです。でも、自己負担額を2万円切る程度で治療できるというのは、この病気を患っている子供さんのご両親も迷わず治療させてあげられるという利点もあります。
この治療費や国の治療費負担の仕方など様々な国で問題になっています。これからたくさんの「高額だけど効果がある」といった治療薬などが開発されていくと思うので、このような事態に備えて準備を始めなければいけないと思います。
The most expensive treatment is Zolgensma which is used to treat spinal muscular atrophy (SMA) developed by a pharmaceutical company in Switzerland called Novartis. This allows about an hour of intravenous injections and the treatment will be done treating SMA, but the price of this treatment in Japan is about 167million yen and in US which the price of the drug is not regulated costs about 2.3million USD. In Japan, because SMA is one of the diseases under Research into Treatment for Specific Child Chronic Diseases Program, so the maximum amount that one has to pay in their own expense is about 20000 yen.
Then, why did this treatment drug become this expensive? This is not because the cost of production is expensive, and here is the reasons why.
(1)It is 50% of the 10yrs Spinraza use which is another existing drug for SMA that needs to be administered once in 4 months
(2)It is 50% of the price that it requires to treat genetic rare disease in general
(3)Price-performance ratio (calculated by dividing the price difference when existing technology is replaced with the new technology by incremental cost-effectiveness (quality of life or effectiveness) is not does not exceed upper limit of the price that Non-profit economic evaluation agency set for paediatric rare disease.
From those reasons, the price became this expensive. But when you hear this, it kind of makes us think that if the pharmaceutical companies are setting the price of a drug depending on how worth the life is.
A lot of pharmaceutical companies need to focus on the more common disease to raise more profit efficiently as research takes a lot of money and time. I think the development of Zolgensma was amazing in a way that they can treat the rare disease and also that they made a way forward for all the genetic treatments that are currently becoming more and more popular. However, when it comes to covering that amount per person by a country, it may become problematic because Japan and other countries may not be able to spend that much money on the healthcare. But if the government didn't cover this amount, then not many families will have an access to this treatment.
There are ongoing debates on whether the country should cover the cost of the treatment, but I really think that because there might be more treatable but expensive drugs developed, government should start thinking about how to manage the cost and how available they are going to make to the patients.
