Background

Health systems experience difficult trade-offs when paying for new drugs. In England, funding recommendations by the National Institute for Health and Care Excellence (NICE) for new drugs might generate health gains, but inevitably result in forgone health as the funds cannot be used for alternative treatments and services. We aimed to evaluate the population-health impact of NICE recommendations for new drugs during 2000–20.

Methods

For this retrospective analysis, we identified technology appraisals for new drugs in England published in NICE's publicly available database of appraisals between 2000 and 2020. We excluded products with terminated appraisals, not recommended, or subsequently withdrawn from the market and excluded appraisals in programmes focusing on medical devices, diagnostics, or interventional procedures. We included drugs that underwent NICE appraisal within 5 years of initial regulatory approval. We collected data on drug name, appraised indication, and specific features of both the drug and its appraisal. We noted the value for money offered by new drugs, expressed as the incremental cost-effectiveness ratio (ICER), and data on health benefits, expressed as quality-adjusted life-years (QALYs). We estimated the number of patients receiving new drugs recommended by NICE using proprietary data on the total volumes of new drugs sold in England between Jan 1, 2000, and Dec 31, 2020. We calculated the net health effect of each appraisal using the difference between the incremental QALY gains from implementing the new drug within the National Health Service (NHS) and the estimated QALYs that could hypothetically be obtained by reallocating the same funds to other NHS services or treatments. We obtained forgone QALYs by dividing the incremental cost of the new drug by the health-opportunity cost of NHS expenditure.

Findings

NICE appraised 332 unique pharmaceuticals between 2000 and 2020; 276 (83%) had positive recommendations. Of these 276, 207 (75%) had a NICE appraisal within 5 years of regulatory approval. We included 183 (88%) of 207 drugs in this analysis, after excluding drugs that did not meet eligibility criteria. The median QALY gain across all 339 appraisals was 0·49 (IQR 0·15–1·13), equivalent to an additional half a year in full health. Median ICER for recommending new drugs increased from £21 545 (IQR 14 175–26 173) per QALY gained for 14 appraisals published between 2000 and 2004 to £28 555 (19 556–33 712) for 165 appraisals published between 2015 and 2020 (p=0·014). Median ICER varied by therapeutic area, ranging from £6478 (3526–12 912) for 12 appraisals of anti-infective drugs to £30 000 (22 395–45 870) for 144 appraisals of oncology drugs (p<0·0001). New drugs generated an estimated 3·75 million additional QALYs across 19·82 million patients who received new drugs recommended by NICE. The use of new drugs resulted in an estimated additional cost to the NHS of £75·1 billion. If the resources allocated to new drugs had been spent on existing services in the NHS, an estimated 5·00 million additional QALYs could have been generated during 2000–20. Overall, the cumulative population-health impact of drugs recommended by NICE was negative, with a net loss of approximately 1·25 million QALYs.

Interpretation

During 2000–20, NHS coverage of new drugs displaced more population health than it generated. Our results highlight the inherent trade-offs between individuals who directly benefit from new drugs and those who forgo health due to the reallocation of resources towards new drugs.

Funding

The Commonwealth Fund.

中文翻译：

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2000-20 年英格兰国家卫生与临床优化研究所推荐的新药对人口健康的影响：回顾性分析

 背景

卫生系统在支付新药费用时需要做出艰难的权衡。在英格兰，国家卫生与临床优化研究所 （NICE） 为新药提供的资金建议可能会带来健康收益，但不可避免地会导致健康丧失，因为这些资金不能用于替代治疗和服务。我们旨在评估 2000-20 年期间 NICE 对新药的建议对人口健康的影响。

 方法

对于这项回顾性分析，我们确定了 2000 年至 2020 年间在 NICE 公开可用的评估数据库中发布的英格兰新药的技术评估。我们排除了评估终止、不推荐或随后退出市场的产品，并排除了专注于医疗器械、诊断或介入程序的计划中的评估。我们纳入了在初始监管批准后 5 年内接受 NICE 评估的药物。我们收集了有关药物名称、评估适应症和药物及其评估的具体特征的数据。我们注意到新药提供的物有所值，以增量成本效益比 （ICER） 表示，以及健康益处数据，以质量调整生命年 （QALY） 表示。我们使用 2000 年 1 月 1 日至 2020 年 12 月 31 日期间在英格兰销售的新药总量的专有数据估计了接受 NICE 推荐的新药的患者人数。我们使用在国家卫生服务 （NHS） 内实施新药的增量 QALY 收益与假设通过将相同资金重新分配给其他 NHS 服务或治疗而获得的估计 QALY 之间的差异，计算了每次评估的净健康影响。我们通过将新药的增量成本除以 NHS 支出的健康机会成本来获得放弃的 QALY。

 发现

NICE 在 2000 年至 2020 年期间评估了 332 种独特的药物;276 人 （83%） 有积极的建议。在这 276 家中，有 207 家 （75%） 在监管部门批准后的 5 年内进行了 NICE 评估。在排除不符合纳入标准的药物后，我们在这项分析中纳入了 207 种药物中的 183 种 （88%）。所有 339 次评估的 QALY 增益中位数为 0·49 （IQR 0·15–1·13），相当于在完全健康状态下额外增加半年。推荐新药的 ICER 中位数从 2000 年至 2004 年间发布的 14 次评估每获得 QALY 21 545 英镑（IQR 14 175-26 173）增加到 2015 年至 2020 年间发布的 165 次评估的 28 555 英镑（19 556-33 712）（p=0·014）。ICER 中位数因治疗领域而异，从 12 次抗感染药物评估的 6478 英镑 （3526-12 912） 到144 次肿瘤药物评估的 30 000 英镑 （22 395-45 870） 不等 （p<0·0001）。新药在接受 NICE 推荐的新药的 1982 万患者中产生了大约 375 万个额外的 QALY。据估计，新药的使用给 NHS 带来了 751 亿英镑的额外成本。如果分配给新药的资源用于 NHS 的现有服务，估计在 2000-20 年期间可以产生 5·00 万个额外的 QALY。总体而言，NICE 推荐的药物对人口健康的累积影响是负面的，净损失约为 1·2500 万 QALY。

 解释

在 2000-20 年期间，NHS 对新药的覆盖取代了更多的人口健康，而不是它产生的。我们的结果强调了直接从新药中受益的个人与因将资源重新分配给新药而放弃健康的人之间的内在权衡。

 资金

 联邦基金。