van Overbeeke Eline, Hauber Brett, Michelsen Sissel, Goldman Michel, Simoens Steven, Huys Isabelle
Clinical Pharmacology and Pharmacotherapy, University of Leuven, Leuven, Belgium.
Health Preference Assessment, RTI Health Solutions, Durham, NC, United States.
Front Med (Lausanne). 2021 Mar 9;8:595797. doi: 10.3389/fmed.2021.595797. eCollection 2021.
Gene therapies are innovative therapies that are increasingly being developed. However, health technology assessment (HTA) and payer decision making on these therapies is impeded by uncertainties, especially regarding long-term outcomes. Through measuring patient preferences regarding gene therapies, the importance of unique elements that go beyond health gain can be quantified and inform value assessments. We designed a study, namely the Patient preferences to Assess Value IN Gene therapies (PAVING) study, that can inform HTA and payers by investigating trade-offs that adult Belgian hemophilia A and B patients are willing to make when asked to choose between a standard of care and gene therapy. An eight-step approach was taken to establish the protocol for this study: (1) stated preference method selection, (2) initial attributes identification, (3) stakeholder (HTA and payer) needs identification, (4) patient relevant attributes and information needs identification, (5) level identification and choice task construction, (6) educational tool design, (7) survey integration, and (8) piloting and pretesting. In the end, a threshold technique survey was designed using the attributes "Annual bleeding rate," "Chance to stop prophylaxis," "Time that side effects have been studied," and "Quality of Life." The Medical Ethics Committee of UZ KU Leuven/Research approved the study. Results from the study will be presented to stakeholders and patients at conferences and in peer-reviewed journals. We hope that results from the PAVING study can inform decision makers on the acceptability of uncertainties and the value of gene therapies to patients.
基因疗法是一种正在不断发展的创新疗法。然而，健康技术评估（HTA）以及支付方对这些疗法的决策受到不确定性的阻碍，尤其是关于长期疗效的不确定性。通过衡量患者对基因疗法的偏好，可以量化那些超越健康获益的独特因素的重要性，并为价值评估提供参考。我们设计了一项研究，即基因疗法价值评估中的患者偏好（PAVING）研究，该研究通过调查成年比利时血友病A和B患者在被要求在标准治疗和基因疗法之间进行选择时愿意做出的权衡，为健康技术评估和支付方提供参考。本研究采用了八步法来制定方案：（1）选择陈述偏好方法；（2）识别初始属性；（3）识别利益相关者（健康技术评估机构和支付方）的需求；（4）识别患者相关属性和信息需求；（5）确定水平并构建选择任务；（6）设计教育工具；（7）整合调查；（8）进行试点和预测试。最后，利用“年出血率”“停止预防治疗的几率”“副作用研究时长”和“生活质量”等属性设计了一项阈值技术调查。鲁汶大学医院/研究医学伦理委员会批准了该研究。研究结果将在会议上以及同行评审期刊上向利益相关者和患者公布。我们希望PAVING研究的结果能够为决策者提供有关不确定性的可接受性以及基因疗法对患者的价值的信息。
