Do patient access schemes for high-cost cancer drugs deliver value to society?-lessons from the NHS Cancer Drugs Fund.
Do patient access schemes for high-cost cancer drugs deliver value to society?-lessons from the NHS Cancer Drugs Fund.
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DOI：
10.1093/annonc/mdx110
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发表时间：
2017-08-01
期刊：
影响因子：
--
通讯作者：
Sullivan R
中科院分区：
文献类型：
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作者：
Aggarwal A;Fojo T;Chamberlain C;Davis C;Sullivan R
NHS癌症药物基金(CDF)成立于2010年，旨在减少延误和改善癌症药物的获取，包括那些以前经过评估但未获得NICE(国家健康与护理卓越研究所)批准的癌症药物。在13亿英镑的支出之后，英国议会在2016年的一次审查中将CDF重新合理化为NICE。本文根据六个价值标准对CDF的潜在价值进行了分析。这包括经过验证的临床受益表、NICE定义的成本效益标准以及对真实世界数据的评估。分析的重点是29种获批的抗癌药物，涉及可能在2015年1月通过CDF开出的47种适应症。在CDF批准的47个适应症中，只有18个(38%)报告了在统计学上显著的OS益处，总体中位生存期为3.1ms月(1.4vs月-15.7ms月)( )。根据临床受益表进行评估时，47个药物适应症中只有23个(48%)和9个(18%)分别符合ASCO和ESMO标准。NICE此前拒绝了CDF批准的26种适应症(55%)，因为它们没有达到成本效益阈值。四种药物--贝伐单抗、西妥昔单抗、伊维洛莫司和拉帕替尼--代表了CDF的大部分申请，总共批准了18种不同的适应症。其中13种适应症随后在2015年1月被CDF摘牌，原因是临床益处证据不足--这些数据自最初批准以来没有变化。我们的结论是，CDF没有为患者或社会带来有意义的价值。没有经验证据表明，相对于手术和放射治疗等其他癌症领域或其他非癌症药物的伴随投资，只有药物保护的癌症基金是可行的。癌症护理中所有药物和干预措施的报销决定应通过适当的卫生技术评估程序作出。
The NHS Cancer Drugs Fund (CDF) was established in 2010 to reduce delays and improve access to cancer drugs, including those that had been previously appraised but not approved by NICE (National Institute for Health and Care Excellence). After 1.3 billion GBP expenditure, a UK parliamentary review in 2016 rationalized the CDF back into NICE. This paper analyses the potential value delivered by the CDF according to six value criteria. This includes validated clinical benefits scales, cost-effectiveness criteria as defined by NICE and an assessment of real-world data. The analysis focuses on 29 cancer drugs approved for 47 indications that could be prescribed through the CDF in January 2015. Of the 47 CDF approved indications, only 18 (38%) reported a statistically significant OS benefit, with an overall median survival of 3.1 months (1.4–15.7 months). When assessed according to clinical benefit scales, only 23 (48%) and 9 (18%) of the 47 drug indications met ASCO and ESMO criteria, respectively. NICE had previously rejected 26 (55%) of the CDF approved indications because they did not meet cost-effectiveness thresholds. Four drugs—bevacizumab, cetuximab, everolimus and lapatinib—represented the bulk of CDF applications and were approved for a total of 18 separate indications. Thirteen of these indications were subsequently delisted by the CDF in January 2015 due to insufficient evidence for clinical benefit—data which were unchanged since their initial approval. We conclude the CDF has not delivered meaningful value to patients or society. There is no empirical evidence to support a ‘drug only’ ring fenced cancer fund relative to concomitant investments in other cancer domains such as surgery and radiotherapy, or other noncancer medicines. Reimbursement decisions for all drugs and interventions within cancer care should be made through appropriate health technology appraisal processes.