NICE首席长官Andrew Dillon称，独立评估委员会对Afinitor进行了审查，承认该药可能能够使乳腺癌的生长和扩散延缓约4个月，但与依西美坦（exemestane）单药相比，Afinitor究竟能使患者生命延长多久存在不确定性。因此，对于国家服务系统（NHS）而言，该药并不是一个符合成本-效益的选择。
尽管NICE拒绝Afinitor用于新诊患者，但表示，已经接受Afinitor治疗的患者应继续使用该药，直到患者和医生认为适合停止使用时。
Afinitor于2012年7月获欧盟（EU）批准，联合依西美坦（exemestane）用于激素受体阳性（HR+）及人表皮生长因子受体2阴性（HER2-）晚期乳腺癌（HR+型晚期乳腺癌）的治疗，适用人群为接受非甾体芳香化酶抑制剂类药物治疗后复发或进展型、且无内脏疾病症状的绝经后妇女患者。
Afinitor靶向于PI3K/AKT/mTOR信号通路，该通路在多种类型肿瘤中处于过度激活（hyperactived）状态。mTOR是一种蛋白，是细胞分裂、血管生长、细胞代谢中的一种重要调节因子。已有数据证实，阻断mTOR是一种行之有效的方法，能够使现有晚期乳腺癌疗法的临床利益最大化。
Afinitor是获批用于乳腺癌治疗的首个mTOR抑制剂，该药先前已获批用于治疗肾癌和胰腺癌等4种类型癌症。
目前，Afinitor已获包括美国、欧盟各国在内的65个国家批准，与exemestane联和用于激素受体阳性、HER2阴性（HR+/HER2-）晚期乳腺癌绝经后女性的治疗。
HR+/HER2-晚期乳腺癌是乳腺癌中的最常见类型，约70%的侵入性乳腺癌在确诊时为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.
