NICE首席長(zhǎng)官Andrew Dillon稱，獨(dú)立評(píng)估委員會(huì)對(duì)Afinitor進(jìn)行了審查，承認(rèn)該藥可能能夠使乳腺癌的生長(zhǎng)和擴(kuò)散延緩約4個(gè)月，但與依西美坦（exemestane）單藥相比，Afinitor究竟能使患者生命延長(zhǎng)多久存在不確定性。因此，對(duì)于國(guó)家服務(wù)系統(tǒng)（NHS）而言，該藥并不是一個(gè)符合成本-效益的選擇。
盡管NICE拒絕Afinitor用于新診患者，但表示，已經(jīng)接受Afinitor治療的患者應(yīng)繼續(xù)使用該藥，直到患者和醫(yī)生認(rèn)為適合停止使用時(shí)。
Afinitor于2012年7月獲歐盟（EU）批準(zhǔn)，聯(lián)合依西美坦（exemestane）用于激素受體陽(yáng)性（HR+）及人表皮生長(zhǎng)因子受體2陰性（HER2-）晚期乳腺癌（HR+型晚期乳腺癌）的治療，適用人群為接受非甾體芳香化酶抑制劑類藥物治療后復(fù)發(fā)或進(jìn)展型、且無(wú)內(nèi)臟疾病癥狀的絕經(jīng)后婦女患者。
Afinitor靶向于PI3K/AKT/mTOR信號(hào)通路，該通路在多種類型腫瘤中處于過(guò)度激活（hyperactived）狀態(tài)。mTOR是一種蛋白，是細(xì)胞分裂、血管生長(zhǎng)、細(xì)胞代謝中的一種重要調(diào)節(jié)因子。已有數(shù)據(jù)證實(shí)，阻斷mTOR是一種行之有效的方法，能夠使現(xiàn)有晚期乳腺癌療法的臨床利益最大化。
Afinitor是獲批用于乳腺癌治療的首個(gè)mTOR抑制劑，該藥先前已獲批用于治療腎癌和胰腺癌等4種類型癌癥。
目前，Afinitor已獲包括美國(guó)、歐盟各國(guó)在內(nèi)的65個(gè)國(guó)家批準(zhǔn)，與exemestane聯(lián)和用于激素受體陽(yáng)性、HER2陰性（HR+/HER2-）晚期乳腺癌絕經(jīng)后女性的治療。
HR+/HER2-晚期乳腺癌是乳腺癌中的最常見(jiàn)類型，約70%的侵入性乳腺癌在確診時(shí)為HR+。（生物谷Bioon.com）
英文原文：'Breast cancer drug not cost-effective', says NICE
The National Institute for Health and Care Excellence (NICE) has found that a breast cancer treatment should not be recommended for use by the NHS because it is not a good use of limited NHS money.
The new guidance advises that the drug everolimus (also called Afinitor and manufactured by Novartis Pharmaceuticals) should not be routinely provided as a treatment for postmenopausal women[1] with HER2 negative, hormone-receptor-positive advanced breast cancer[2].
Sir Andrew Dillon, NICE Chief Executive, said: “NICE assesses treatments and recommends those which are found to be the most clinically and cost effective. With limited NHS funds, it's important we make recommendations based on how well a treatment works compared to alternative treatments in the NHS, as well as any associated side effects and the cost that the health service is being asked to pay.
“The independent appraisal committee, which reviewed everolimus, acknowledged it could delay the growth and spread of breast cancer by around four months, but there were uncertainties relating to how long it could extend a person's life compared with the drug exemestane alone.
“Using the evidence that was available, the committee concluded that everolimus is not a cost-effective option for the NHS.”
Despite not recommending the treatment, the guidance does say that women already receiving everolimus should continue with it until they and their doctor thinks it's appropriate to stop.
Notes to Editors
The final guidance for everolimus in combination with exemestane for treating locally advanced or metastatic HER2 negative hormone-receptor-positive breast cancer after endocrine therapy is available on the NICE website.
Breast cancer is the most common cancer in the UK with nearly 50,000 women and 400 men diagnosed each year[3]. Everolimus is an oral treatment that works by blocking a protein in the body that regulates the division of tumour cells and growth of blood vessels. This can help stop cancer cells from multiplying and spreading. The manufacturer estimates that around 1,500 people would be eligible to receive everolimus, if it were to be recommended.
The recommended dosage of everolimus is 10mg taken once a day. Treatment should continue for as long as it stops the growth and spread of cancer cells or until the patient cannot tolerate the side effects. Side effects that are severe and/or intolerable may be managed by reducing the dosage to 5mg daily or temporarily stopping treatment followed by reintroduction at 5mg daily. The price for a pack of 10mg tablets (30 tablets per pack) is ￡2,970 (excluding VAT; ‘British National Formulary' [BNF] edition 64).
The independent Appraisal Committee assessed the evidence submitted to this appraisal, including clinical trial data and evidence by clinical experts and patient representatives. The evidence submitted to the committee by the manufacturer highlighted that median progression-free survival (the time point in the trial at which the 50% of people experience either disease progression or death) with everolimus (with exemestane) was 4.6 months longer than with exemestane alone. However, the committee concluded that the clinical trial data generated uncertainty relating to the efficacy of the treatment compared with relevant chemotherapy regimens and how much the treatment may extend overall survival.
The manufacturer of everolimus had agreed a patient access scheme with the Department of Health and this was included in the calculation of cost-effectiveness. However, this did not alter the Committee's conclusion that everolimus is not cost effective as a treatment for HER2 negative, hormone-receptor-positive advanced breast cancer.
The Scottish Medicines Consortium (SMC) published guidance in July 2013 which does not recommend everolimus as a treatment for hormone receptor-positive, HER2 negative advanced breast cancer, in combination with exemestane, in postmenopausal women without symptomatic visceral disease after recurrence or progression following a non-steroidal aromatase inhibitor.
In 2009, NICE published a clinical guideline on the care of people with early and locally advanced breast cancer and another for advanced breast cancer. Both guidelines include recommendations for clinicians on a range of treatment options for patients.
