Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI's Patient Groups and Clinical Trials Project
Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI's Patient Groups and Clinical Trials Project
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DOI：
10.1177/2168479017716715
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发表时间：
2018-03-01
影响因子：
1.5
通讯作者：
DiMasi, Joseph
中科院分区：
文献类型：
--
作者：
Levitan, Bennett;Getz, Kenneth;DiMasi, Joseph
背景资料：虽然患者团体、监管机构和申办者越来越多地考虑在临床开发项目的设计和实施中与患者接触，但由于投资回报的不确定性，申办者往往不愿意超越试点项目。我们开发了一种方法来估计patient engagement.Methods的财务价值：预期净现值（ENPV）是一种常见的技术，它集成了成本，时间，收入和风险的关键业务驱动因素到项目战略和投资组合决策的摘要度量。我们评估了患者参与对进入2期或3期的典型肿瘤学开发项目的ENPV的影响。结果：对于II期前项目，避免一次方案修订并改善入组、依从性保留是净现值（NPV）增加6200万美元（第3阶段前为6500万美元），ENPV增加3500万美元（第3阶段前为7500万美元）。与10万美元的患者参与投资相比，NPV和ENPV的增加可能超过投资的500倍。这ENPV增加是加速前阶段2产品推出的21/2年（11/2年前阶段3）。结论：风险调整的财务模型可以评估患者参与的影响。经验数据和主观参数估计的组合表明，有可能避免方案修订和/或改善入组、依从性和保留的参与活动可能会增加相当大的财务价值。这种方法可以帮助赞助商评估患者参与投资决策。
Background: While patient groups, regulators, and sponsors are increasingly considering engaging with patients in the design and conduct of clinical development programs, sponsors are often reluctant to go beyond pilot programs because of uncertainty in the return on investment. We developed an approach to estimate the financial value of patient engagement.Methods: Expected net present value (ENPV) is a common technique that integrates the key business drivers of cost, time, revenue, and risk into a summary metric for project strategy and portfolio decisions. We assessed the impact of patient engagement on ENPV for a typical oncology development program entering phase 2 or phase 3.Results: For a pre-phase 2 project, the cumulative impact of a patient engagement activity that avoids one protocol amendment and improves enrollment, adherence, and retention is an increase in net present value (NPV) of $62MM ($65MM for pre-phase 3) and an increase in ENPV of $35MM ($75MM for pre-phase 3). Compared with an investment of $100,000 in patient engagement, the NPV and ENPV increases can exceed 500-fold the investment. This ENPV increase is the equivalent of accelerating a pre-phase 2 product launch by 21/2 years (11/2 years for pre-phase 3).Conclusions: Risk-adjusted financial models can assess the impact of patient engagement. A combination of empirical data and subjective parameter estimates shows that engagement activities with the potential to avoid protocol amendments and/or improve enrollment, adherence, and retention may add considerable financial value. This approach can help sponsors assess patient engagement investment decisions.