Eastern Research Group Inc, Lexington, Massachusetts.
US Department of Health and Human Services, Office of Inspector General, Washington, DC.
JAMA Netw Open. 2022 Sep 1;5(9):e2231609. doi: 10.1001/jamanetworkopen.2022.31609.
The US medical device market is the world's largest, but estimates of the cost to bring a medical device to market are not available to help inform policy making and regulatory efforts to enhance device safety and innovation.
To estimate the mean expected capitalized cost of developing a novel therapeutic complex medical device.
DESIGN, SETTING, AND PARTICIPANTS: In this economic evaluation, an analytical model of novel therapeutic complex medical device development using data from public and proprietary sources with coverage from 2000 through 2018 was used to estimate the cost, duration, and phase transition success probability associated with each stage of development. Data analysis was completed in September 2021.
Conduct of nonclinical and clinical studies; payment of FDA user fees for novel therapeutic complex medical devices.
Mean development cost (in 2018 US dollars) incurred by developers for an FDA-approved novel therapeutic complex medical device, accounting for failures and cost of capital.
In this economic analysis, the mean development cost for a novel therapeutic complex medical device was $54 million (95% CI, $25 million-$200 million) excluding any postapproval studies that might be required. After accounting for the cost of failed studies and cost of capital, the mean capitalized cost of bringing a novel therapeutic complex medical device to the US market was $522 million (95% CI, $205 million-$3382 million). The key factors associated with this cost were the phase transition probabilities: 46.9% for nonclinical to feasibility study, 48.0% for feasibility to pivotal study, 75.7% pivotal study to FDA premarket approval submission, and 80.5% for FDA premarket approval submission to approval. The nonclinical development stage constituted the largest portion of overall cost at 85.0% with the FDA review stage with the highest phase transition probability accounting for only a small fraction at 0.5%.
In this economic evaluation study, the cost of therapeutic complex medical device development from proof of concept through postapproval stages was assessed accounting for the cost of failures and the cost of capital. Existing estimates did not account for all stages of development, capitalization, or failure costs, which this study suggests were substantial.
美国医疗器械市场是全球最大的，但目前尚无法估算将医疗器械推向市场的成本，这无助于为加强医疗器械安全性和创新性的政策制定和监管工作提供信息。
估算开发新型治疗性复杂医疗器械的平均预期资本成本。
设计、环境和参与者：在这项经济评估中，使用了来自公共和私人来源的数据，这些数据涵盖了 2000 年至 2018 年期间的信息，建立了一个用于开发新型治疗性复杂医疗器械的分析模型，以估算与每个开发阶段相关的成本、持续时间和阶段转换成功率。数据分析于 2021 年 9 月完成。
非临床和临床研究的开展；为新型治疗性复杂医疗器械支付 FDA 用户费用。
开发者为获得 FDA 批准的新型治疗性复杂医疗器械支付的平均开发成本（以 2018 年美元计），包括失败和资本成本。
在这项经济分析中，新型治疗性复杂医疗器械的平均开发成本为 5400 万美元（95%CI，2500 万美元-2 亿美元），不包括可能需要的任何批准后研究。在考虑到研究失败和资本成本后，将新型治疗性复杂医疗器械推向美国市场的资本化成本平均为 5.22 亿美元（95%CI，2.05 亿美元-33.82 亿美元）。与这一成本相关的关键因素是阶段转换概率：从非临床到可行性研究为 46.9%，从可行性到关键研究为 48.0%，从关键研究到 FDA 上市前批准申请为 75.7%，从 FDA 上市前批准申请到批准为 80.5%。非临床开发阶段构成了总成本的最大部分，占 85.0%，而 FDA 审查阶段的阶段转换概率最高，仅占 0.5%。
在这项经济评估研究中，从概念验证到批准后阶段的治疗性复杂医疗器械开发成本进行了评估，考虑到了失败和资本成本。现有估计没有考虑到所有开发阶段、资本化或失败成本，而本研究表明这些成本相当大。
