Australian Health Services Research Institute, Sydney Business School, University of Wollongong, Room 255, Building 40, Wollongong, NSW, 3522, Australia.
Pharmacoeconomics. 2013 May;31(5):393-401. doi: 10.1007/s40273-013-0038-5.
Risk sharing arrangements relate to adjusting payments for new health technologies given evidence of their performance over time. Such arrangements rely on prospective information regarding the incremental net benefit of the new technology, and its use in practice. However, once the new technology has been adopted in a particular jurisdiction, randomized clinical trials within that jurisdiction are likely to be infeasible and unethical in the cases where they would be most helpful, i.e. with current evidence of positive while uncertain incremental health and net monetary benefit. Informed patients in these cases would likely be reluctant to participate in a trial, preferring instead to receive the new technology with certainty. Consequently, informing risk sharing arrangements within a jurisdiction is problematic given the infeasibility of collecting prospective trial data. To overcome such problems, we demonstrate that global trials facilitate trialling post adoption, leading to more complete and robust risk sharing arrangements that mitigate the impact of costs of reversal on expected value of information in jurisdictions who adopt while a global trial is undertaken. More generally, optimally designed global trials offer distinct advantages over locally optimal solutions for decision makers and manufacturers alike: avoiding opportunity costs of delay in jurisdictions that adopt; overcoming barriers to evidence collection; and improving levels of expected implementation. Further, the greater strength and translatability of evidence across jurisdictions inherent in optimal global trial design reduces barriers to translation across jurisdictions characteristic of local trials. Consequently, efficiently designed global trials better align the interests of decision makers and manufacturers, increasing the feasibility of risk sharing and the expected strength of evidence over local trials, up until the point that current evidence is globally sufficient.
风险分担安排涉及根据新医疗技术的性能随时间推移的证据来调整支付。这种安排依赖于关于新技术的增量净效益及其在实践中的使用的前瞻性信息。然而,一旦新技术在特定司法管辖区得到采用,在该司法管辖区内进行随机临床试验在最有帮助的情况下可能是不可行和不道德的,即在当前有证据表明积极但不确定的增量健康和净货币效益的情况下。在这些情况下,知情的患者可能不愿意参与试验,而是更愿意确定性地接受新技术。因此,由于无法收集前瞻性试验数据,在司法管辖区内告知风险分担安排是有问题的。为了解决这些问题,我们证明全球试验促进了采用后的试验,从而制定了更完整和稳健的风险分担安排,减轻了在进行全球试验期间采用的司法管辖区中成本逆转对信息预期价值的影响。更一般地说,对于决策者和制造商来说,经过优化设计的全球试验具有明显优于局部最优解决方案的优势:避免在采用的司法管辖区中延迟的机会成本;克服证据收集的障碍;并提高预期实施水平。此外,在最佳全球试验设计中固有的跨司法管辖区的证据更强有力和更具可翻译性,降低了局部试验特有的跨司法管辖区翻译的障碍。因此,设计合理的全球试验更好地协调了决策者和制造商的利益,增加了风险分担的可行性,并提高了证据的预期强度,直到当前的证据在全球范围内足够为止。