Clearfield Elizabeth, Kim Benjamin, Ford Sarah, Connell Nathan T, Santaella Maria E, Lavin Michelle, Degenaar-Dujardin Manon E L, Ayoub Emily, Flood Veronica H, Rotellini Dawn, Skinner Mark W, James Paula
Institute for Policy Advancement Ltd, Washington, District of Columbia, USA.
Vega Therapeutics Inc, South San Francisco, USA.
Haemophilia. 2024 Nov;30(6):1357-1365. doi: 10.1111/hae.15122. Epub 2024 Nov 27.
Treatment options are expanding for von Willebrand disease (VWD). A core outcome set (COS)-a minimum set of agreed-upon outcomes to be used in every clinical trial for a given condition-provides guidance on which outcomes are most important to measure to ensure necessary data is collected for a variety of stakeholders and enable comparison across products and trials.
coreVWD aimed to develop a COS for trials for prophylaxis and perioperative treatments for VWD.
A modified Delphi consensus process was used to condense/prioritize a long list of potential outcomes. Over three Delphi rounds, a multi-stakeholder panel (patients, clinicians, pharmaceutical company representatives, HTA organizations, payer, and government organization representatives) rated each outcome from 1 (not important to include in a COS) to 9 (essential to include). Outcomes were eliminated or retained based on pre-determined criteria; a special provision to elevate patient priorities was included. An in-person consensus meeting was held after Delphi round 2.
Thirty-nine panellists participated. The final COS for prophylaxis treatment included 18 outcomes, seven of which are part of a special subset selected for women, girls and people with the potential to menstruate. There were 11 outcomes in the final perioperative branch COS. Six outcomes overlapped both COS.
The coreVWD COS represents a consensus list of outcomes for clinical trials for both factor and non-factor VWD therapies. These outcomes will be useful across the lifecycle of a product, from clinical development through regulatory and market access phases and into patient-provider decision-making.
血管性血友病（VWD）的治疗选择正在不断扩展。核心结局集（COS）——针对特定病症在每项临床试验中使用的一组最低限度的商定结局——为确定哪些结局对于测量最为重要提供了指导，以确保为各类利益相关者收集必要的数据，并能够对不同产品和试验进行比较。
coreVWD旨在为VWD的预防和围手术期治疗试验制定一个COS。
采用改良的德尔菲共识法对一长串潜在结局进行浓缩/排序。在三轮德尔菲过程中，一个多利益相关者小组（患者、临床医生、制药公司代表、卫生技术评估组织、支付方和政府组织代表）对每个结局从1（对纳入COS不重要）到9（必须纳入）进行评分。根据预先确定的标准对结局进行剔除或保留；其中包括一项提升患者优先事项的特殊规定。在第二轮德尔菲之后举行了一次面对面的共识会议。
39名小组成员参与。预防治疗的最终COS包括18项结局，其中7项是为女性、女孩和有月经可能的人群选定的一个特殊子集的一部分。围手术期分支的最终COS中有11项结局。有6项结局在两个COS中都有。
coreVWD COS代表了因子和非因子VWD疗法临床试验结局的共识清单。这些结局在产品的整个生命周期中都将有用，从临床开发到监管和市场准入阶段，再到患者与医疗服务提供者的决策过程。
