Cancer Control Research - Canadian Centre for Applied Research in Cancer Control (ARCC), BC Cancer, 675 West 10th Avenue, Vancouver, British Columbia, V5Z 1L3, Canada.
School of Population and Public Health, Faculty of Medicine, University of British Columbia, Vancouver, British Columbia, Canada.
Pharmacoeconomics. 2020 Jan;38(1):57-68. doi: 10.1007/s40273-019-00834-0.
A limited evidence base and lack of clear clinical guidelines challenge healthcare systems' adoption of precision medicine. The effect of these conditions on demand is not understood.
This research estimated the public's preferences and demand for precision medicine outcomes.
A discrete-choice experiment survey was conducted with an online sample of the US public who had recent healthcare experience. Statistical analysis was undertaken using an error components mixed logit model. The responsiveness of demand in the context of a changing evidence base was estimated through the price elasticity of demand. External validation was examined using real-world demand for the 21-gene recurrence score assay for breast cancer.
In total, 1124 (of 1849) individuals completed the web-based survey. The most important outcomes were survival gains with statistical uncertainty, cost of testing, and medical expert agreement on changing care based on test results. The value ($US, year 2017 values) for a test where most (vs. few) experts agreed to changing treatment based on test results was $US1100 (95% confidence interval [CI] 916-1286). Respondents were willing to pay $US265 (95% CI 46-486) for a test that could result in greater certainty around life-expectancy gains. The predicted demand of the assay was 9% in 2005 and 66% in 2014, compared with real-world uptake of 7% and 71% (root-mean-square prediction error 0.11). Demand was sensitive to price (1% increase in price resulted in > 1% change in demand) when first introduced and insensitive to price (1% increase in price resulted in < 0.1% change in demand) as the evidence base became established.
Evidence of external validity was found. Demand was weak and responsive to price in the near term because of uncertainty and an immature evidence base. Clear communication of precision medicine outcomes and uncertainty is crucial in allowing healthcare to align with individual preferences.
有限的证据基础和缺乏明确的临床指南，对医疗保健系统采用精准医学构成了挑战。目前尚不清楚这些情况对需求的影响。
本研究旨在评估公众对精准医学结果的偏好和需求。
采用离散选择实验调查方法，对有近期医疗保健经验的美国公众进行在线调查。采用误差成分混合对数模型进行统计分析。通过需求的价格弹性来估计在证据基础不断变化的情况下需求的响应能力。通过对乳腺癌 21 基因复发评分检测的实际需求来检验外部有效性。
共有 1849 名受访者中的 1124 名（61%）完成了在线调查。最重要的结果是具有统计学不确定性的生存获益、检测成本，以及根据检测结果改变治疗方案时医疗专家的意见一致性。如果大多数（而非少数）专家根据检测结果同意改变治疗方案，则愿意为一项检测支付的金额为 1100 美元（95%置信区间 [CI] 916 美元至 1286 美元）。受访者愿意为一项可以更确定地增加预期寿命获益的检测支付 265 美元（95% CI 46 美元至 486 美元）。与 2005 年的 9%和 2014 年的 66%相比，预测该检测在 2014 年的实际使用率为 7%，在 2014 年为 71%（均方根预测误差 0.11）。在引入初期，需求对价格敏感（价格上涨 1%，需求变化超过 1%），而当证据基础确立后，需求对价格不敏感（价格上涨 1%，需求变化不到 0.1%）。
发现了外部有效性的证据。由于不确定性和不成熟的证据基础，近期需求较弱且对价格敏感。精准医学结果和不确定性的清晰沟通对于使医疗保健符合个人偏好至关重要。
