Henderson Raymond H, French Declan, Stewart Elaine, Smart Dave, Idica Adam, Redmond Sandra, Eckstein Markus, Clark Jordan, Sullivan Richard, Keeling Peter, Lawler Mark
Patrick G. Johnston Centre for Cancer Research, Queen's University, Belfast, UK.
Queen's Management School, Queen's University Belfast, Belfast, UK.
J Pharm Policy Pract. 2023 Jul 5;16(1):84. doi: 10.1186/s40545-023-00590-9.
Precision oncology medicines represent a paradigm shift compared to non-precision oncology medicines in cancer therapy, in some situations delivering more clinical benefit, and potentially lowering healthcare costs. We determined whether employing a companion diagnostic (CDx) approach during oncology medicines development delivers effective therapies that are within the cost constraints of current health systems. R&D costs of developing a medicine are subject to debate, with average estimates ranging from $765 million (m) to $4.6 billion (b). Our aim was to determine whether precision oncology medicines are cheaper to bring from R&D to market; a secondary goal was to determine whether precision oncology medicines have a greater return on investment (ROI).
Data on oncology medicines approved between 1997 and 2020 by the US Food and Drug Administration (FDA) were analysed from the Securities and Exchange Commission (SEC) filings. Data were compiled from 10-K, 10-Q, and 20-F financial performance filings on medicines' development costs through their R&D lifetime. Clinical trial data were split into clinical trial phases 1-3 and probability of success (POS) of trials was calculated, along with preclinical costs. Cost-of-capital (CoC) approach was applied and, if appropriate, a tax rebate was subtracted from the total.
Data on 42 precision and 29 non-precision oncology medicines from 56 companies listed by the National Cancer Institute which had complete data available were analysed. Estimated mean cost to deliver a new oncology medicine was $4.4b (95% CI, $3.6-5.2b). Costs to bring a precision oncology medicine to market were $1.1b less ($3.5b; 95% CI, $2.7-4.5b) compared to non-precision oncology medicines ($4.6b; 95% CI, $3.5-6.1b). The key driver of costs was POS of clinical trials, accounting for a difference of $591.3 m. Additional data analysis illustrated that there was a 27% increase in return on investment (ROI) of precision oncology medicines over non-precision oncology medicines.
Our results provide an accurate estimate of the R&D spend required to bring an oncology medicine to market. Deployment of a CDx at the earliest stage substantially lowers the cost associated with oncology medicines development, potentially making them available to more patients, while staying within the cost constraints of cancer health systems.
与非精准肿瘤学药物相比，精准肿瘤学药物在癌症治疗中代表了一种范式转变，在某些情况下能带来更多临床益处，并有可能降低医疗成本。我们确定在肿瘤学药物研发过程中采用伴随诊断（CDx）方法是否能提供符合当前卫生系统成本限制的有效疗法。开发一种药物的研发成本存在争议，平均估计范围从7.65亿美元（m）到46亿美元（b）。我们的目的是确定精准肿瘤学药物从研发到上市的成本是否更低；第二个目标是确定精准肿瘤学药物是否有更高的投资回报率（ROI）。
从美国证券交易委员会（SEC）文件中分析了1997年至2020年期间美国食品药品监督管理局（FDA）批准的肿瘤学药物数据。数据来自关于药物在其研发生命周期内的研发成本的10-K、10-Q和20-F财务业绩文件。临床试验数据被分为1-3期临床试验，并计算试验成功概率（POS）以及临床前成本。应用资本成本（CoC）方法，并在适当时从总额中减去退税。
分析了美国国立癌症研究所列出并拥有完整可用数据的56家公司的42种精准肿瘤学药物和29种非精准肿瘤学药物的数据。推出一种新的肿瘤学药物的估计平均成本为44亿美元（95%置信区间，36亿至52亿美元）。与非精准肿瘤学药物（46亿美元；95%置信区间，35亿至61亿美元）相比，将一种精准肿瘤学药物推向市场的成本低11亿美元（35亿美元；95%置信区间：27亿至45亿美元）。成本的关键驱动因素是临床试验的POS，占比差异为5.913亿美元。进一步的数据分析表明，精准肿瘤学药物的投资回报率比非精准肿瘤学药物高出27%。
我们的结果准确估计了将一种肿瘤学药物推向市场所需的研发支出。尽早部署CDx可大幅降低与肿瘤学药物研发相关的成本，有可能使更多患者能够使用这些药物，同时保持在癌症卫生系统的成本限制范围内。
