J Natl Compr Canc Netw. 2018 Dec;16(12):1460-1466. doi: 10.6004/jnccn.2018.7084.
FDA approvals do not consider cost, but they set the tone for regulatory approvals worldwide, including in countries with universal healthcare where cost-effectiveness, utility, and adoption feasibility are considered rigorously. Data from the pan-Canadian Oncology Drug Review (pCODR), a national drug review system that makes evidence-based funding recommendations to Canada's provinces and territories, were collected. Our objectives were to assess (1) temporal trends in cost and efficacy of drugs reviewed, (2) correlations among magnitude of benefits, cost, and pCODR decisions, and (3) predictors of approvals. A total of 60 drugs for 91 indications were reviewed by pCODR from January 2012 to January 2018. Of the 91 reviews (approved previously by FDA), 18 received negative recommendations on the grounds of inadequate clinical benefits; 87% (64/73) of those approved were conditional on improvement in cost. Surrogate outcomes were used to support approvals in 83% of the reviews, which were not correlated with overall survival (r = +0.16; =.24). Median cost/quality-adjusted life years (QALY) increased by 36% per annum (quantile regression, =.0029), although benefits in overall and progression-free survival were stable (=.21 and .65, respectively). Median-based incremental cost-effectiveness ratio (ICER) of new drugs was $186,403 CAD (range, $7,200 to $2.1 million). Higher ICER was a strong predictor of a negative pCODR recommendation (<.01). A substantial number of cancer drugs that are FDA approved for public use do not meet Canadian standards for efficacy. Cost of cancer drugs increases by a third annually in Canada, but the benefits-measured mostly with surrogates that did not correlate with survival-are stable. With finite resources to share among multiple societal priorities, such as education and preventive health, cancer drug cost may be unsustainable despite price regulation.
FDA 的批准并不考虑成本，但它们为全球监管批准设定了基调，包括在那些普遍实行医疗保健的国家，这些国家会严格考虑成本效益、实用性和采用的可行性。从 pan-Canadian Oncology Drug Review（pCODR）收集了数据，这是一个全国性的药物审查系统，为加拿大的省份和地区提供基于证据的资金建议。我们的目标是评估：（1）审查药物的成本和疗效的时间趋势；（2）效益、成本和 pCODR 决策之间的相关性；（3）批准的预测因素。从 2012 年 1 月到 2018 年 1 月，pCODR 对 91 种适应证的 60 种药物进行了审查。在 91 项审查中（之前已被 FDA 批准），18 项因临床获益不足而被建议否决；在获得批准的 73 项中，有 87%（64/73）是有条件批准，条件是成本得到改善。替代终点在 83%的审查中被用于支持批准，但与总生存期无关（r = +0.16；p=.24）。每 12 个月成本/质量调整生命年（QALY）增加 36%（分位数回归，p =.0029），尽管总生存期和无进展生存期的获益稳定（分别为 p=.21 和 p=.65）。新药物的中位基于增量成本效益比（ICER）为 186403 加元（范围为 7200 加元至 210 万加元）。较高的 ICER 是 pCODR 负面建议的有力预测因素（p<.01）。大量被 FDA 批准用于公共用途的癌症药物不符合加拿大的疗效标准。加拿大癌症药物的成本每年增加三分之一，但主要用与生存无关的替代终点来衡量的效益是稳定的。由于有限的资源要在多个社会优先事项之间共享，例如教育和预防保健，因此，尽管有价格管制，癌症药物的成本可能仍然不可持续。
