Syreon Research Institute, Budapest, Hungary.
Center of Health Technology Assessment, Semmelweis University, Budapest, Hungary.
J Manag Care Spec Pharm. 2021 Jul;27(7):936-947. doi: 10.18553/jmcp.2021.27.7.936.
Inclusion of patient experience (PEx) in health technology assessment (HTA) has become increasingly important; however, no harmonized approach exists to help manufacturers or decision makers ensure PEx considerations are fair, consistent, and thorough within global HTA frameworks. To develop a proposal for including PEx in the HTA frameworks of health technologies. A systematic literature review (SLR) on existing value frameworks (VFs) was conducted to capture how PEx-related value judgment is currently considered. Guided by the results of the SLR, a research group including HTA experts and patient representatives used an iterative process to develop potential value domains to capture PEx, in accordance with international guidelines. Subsequently, a panel of international payer experts was used to challenge the proposed PEx domains and provide recommendations for implementation. The SLR found 61 VFs and multi-criteria decision analyses (MCDAs) that considered PEx; however, PEx-related value elements were often referred to superficially, without clear definitions. Five potential PEx domains, with proposed measures for each, were developed and refined using expert feedback: (1) responsiveness to patient's individual needs, (2) improved health literacy and empowerment, (3) patient and caregiver reported outcomes, (4) household's financial burden, and (5) improved access for vulnerable patient populations. A flexible approach for framework implementation was proposed. Proposed PEx domains could be implemented at multiple levels of healthcare decision making to formalize consideration of PEx in the assessment of value, either through the extension of existing VFs or to create new PEx-focused VFs and more holistic decision making tools. This study was funded and sponsored by UCB Pharma. The funding agreement ensured the authors' independence in designing the study, interpreting the data, writing, and publishing the report. Charokopou, Mountain, and Szegvari are employed by UCB Pharma. Inotai, Jakab, and Kalo are employed by Syreon Research Institute, which received funding from UCB Pharma for this research. Brixner has received fees from AbbVie, Elevar, Millcreek Outcomes Group, Novartis, Sanofi, UCB Pharma, and Xcenda. Campbell has received grants and contracts from the PhRMA Foundation and the Institute for Clinical and Economic Review. During a sabbatical leave, Campbell collaborated with Syreon Research Institute on research projects that included funding from UCB Pharma. Hawkins has received consultancy fees from UCB Pharma. Kristensen has received speakers bureau fees from Pfizer, AbbVie, Amgen, UCB Pharma, Celgene, Bristol-Myers Squibb, MSD, Novartis, Eli Lilly, and Janssen Pharmaceuticals and consultancy fees from UCB Pharma.
将患者体验（PEx）纳入健康技术评估（HTA）变得越来越重要；然而，在全球 HTA 框架内，没有一种统一的方法可以帮助制造商或决策者确保 PEx 考虑因素是公平、一致和全面的。目的 制定纳入健康技术 HTA 框架的 PEx 提案。对现有价值框架（VF）进行了系统文献综述（SLR），以了解当前如何考虑与 PEx 相关的价值判断。根据 SLR 的结果，一个包括 HTA 专家和患者代表的研究小组按照国际准则，使用迭代过程开发了潜在的价值领域，以捕获 PEx。随后，一组国际支付方专家用于挑战拟议的 PEx 领域，并提供实施建议。SLR 发现了 61 个考虑了 PEx 的 VF 和多准则决策分析（MCDA）；然而，PEx 相关的价值元素往往只是表面上提到，没有明确的定义。使用专家反馈，开发和完善了五个潜在的 PEx 领域，每个领域都提出了相应的措施：（1）对患者个体需求的响应能力，（2）提高健康素养和赋权，（3）患者和护理人员报告的结果，（4）家庭的经济负担，以及（5）为弱势患者群体改善获得途径。提出了一种灵活的框架实施方法。拟议的 PEx 领域可以在医疗决策的多个层面实施，以正式考虑在价值评估中纳入 PEx，方法是扩展现有 VF 或创建新的专注于 PEx 的 VF 和更全面的决策工具。本研究由 UCB 制药公司资助和赞助。资助协议确保了作者在设计研究、解释数据、撰写和发表报告方面的独立性。Charokopou、Mountain 和 Szegvari 受雇于 UCB 制药公司。Inotai、Jakab 和 Kalo 受雇于 Syreon 研究所，该研究所从 UCB 制药公司获得了这项研究的资金。Brixner 从 AbbVie、Elevar、Millcreek Outcomes Group、Novartis、Sanofi、UCB 制药公司和 Xcenda 获得了费用。坎贝尔从 PhRMA 基金会和临床与经济审查研究所获得了赠款和合同。在休假期间，坎贝尔与 Syreon 研究所合作开展了研究项目，其中包括 UCB 制药公司的资助。霍金斯从 UCB 制药公司获得咨询费。Kristensen 从辉瑞、艾伯维、安进、UCB 制药公司、Celgene、百时美施贵宝、默沙东、诺华、礼来和杨森制药公司获得演讲费，并从 UCB 制药公司获得咨询费。
