Measuring what matters to rare disease patients - reflections on the work by the IRDiRC taskforce on patient-centered outcome measures
Measuring what matters to rare disease patients - reflections on the work by the IRDiRC taskforce on patient-centered outcome measures
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DOI：
10.1186/s13023-017-0718-x
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发表时间：
2017-11-02
影响因子：
3.7
通讯作者：
Cano, Stefan J.
中科院分区：
文献类型：
--
作者：
Morel, Thomas;Cano, Stefan J.
我们评估真正反映患者未满足的需求、希望和担忧的结果的能力至关重要。然而，目前的许多临床研究和实践福尔斯都没有达到这一目标，因为选择的结果指标没有充分反映患者的价值。在本意见中，我们讨论了以患者为中心的结局指标（PCOM），它有可能系统地纳入患者的观点来衡量对患者最重要的结局。我们主张加强多方利益相关者的合作，以罕见病患者和家庭为中心，开发PCOM。除了推进患者输入的科学之外，PCOM是将护理或观察到的治疗益处转化为患者益处的“可解释”度量的强大工具，从而有助于证明临床有效性。我们提出混合方法心理测量研究作为提供适合罕见疾病的PCOM的最佳途径，因为这种方法将定性和定量研究方法与有效利用小样本数据的明确目标结合在一起。而且，无论是选择开发一种全新的PCOM，还是选择或调整现有的结果指标用于罕见疾病，锚点都是相同的：患者，他们对罕见疾病的日常经历，他们的偏好，核心概念和价值观。最终，现有的价值框架、登记和基于结果的合同在很大程度上无法始终如一地衡量对患者重要的全部结果。我们认为，在罕见疾病中更多地使用PCOM将使以患者为中心的护理快速通道。
Our ability to evaluate outcomes which genuinely reflect patients' unmet needs, hopes and concerns is of pivotal importance. However, much current clinical research and practice falls short of this objective by selecting outcome measures which do not capture patient value to the fullest. In this Opinion, we discuss Patient-Centered Outcomes Measures (PCOMs), which have the potential to systematically incorporate patient perspectives to measure those outcomes that matter most to patients. We argue for greater multi-stakeholder collaboration to develop PCOMs, with rare disease patients and families at the center. Beyond advancing the science of patient input, PCOMs are powerful tools to translate care or observed treatment benefit into an 'interpretable' measure of patient benefit, and thereby help demonstrate clinical effectiveness. We propose mixed methods psychometric research as the best route to deliver fit-for-purpose PCOMs in rare diseases, as this methodology brings together qualitative and quantitative research methods in tandem with the explicit aim to efficiently utilise data from small samples. And, whether one opts to develop a brand-new PCOM or to select or adapt an existing outcome measure for use in a rare disease, the anchors remain the same: patients, their daily experience of the rare disease, their preferences, core concepts and values. Ultimately, existing value frameworks, registries, and outcomes-based contracts largely fall short of consistently measuring the full range of outcomes that matter to patients. We argue that greater use of PCOMs in rare diseases would enable a fast track to Patient-Centered Care.