Department of Pharmacy, University of Washington, Seattle, WA 98195, USA.
Med Care. 2012 May;50(5):388-93. doi: 10.1097/MLR.0b013e3182422a3b.
Systematic approaches to stakeholder-informed research prioritization are a central focus of comparative effectiveness research. Genomic testing in cancer is an ideal area to refine such approaches given rapid innovation and potentially significant impacts on patient outcomes.
To develop and pilot test a stakeholder-informed approach to prioritizing genomic tests for future study in collaboration with the cancer clinical trials consortium SWOG.
We conducted a landscape analysis to identify genomic tests in oncology using a systematic search of published and unpublished studies, and expert consultation. Clinically valid tests suitable for evaluation in a comparative study were presented to an external stakeholder group. Domains to guide the prioritization process were identified with stakeholder input, and stakeholders ranked tests using multiple voting rounds.
A stakeholder group was created including representatives from patient-advocacy groups, payers, test developers, regulators, policy makers, and community-based oncologists. We identified 9 domains for research prioritization with stakeholder feedback: population impact; current standard of care, strength of association; potential clinical benefits, potential clinical harms, economic impacts, evidence of need, trial feasibility, and market factors. The landscape analysis identified 635 studies; of 9 tests deemed to have sufficient clinical validity, 6 were presented to stakeholders. Two tests in lung cancer (ERCC1 and EGFR) and 1 test in breast cancer (CEA/CA15-3/CA27.29) were identified as top research priorities.
Use of a diverse stakeholder group to inform research prioritization is feasible in a pragmatic and timely manner. Additional research is needed to optimize search strategies, stakeholder group composition, and integration with existing prioritization mechanisms.
系统的利益相关者知情的研究优先级制定方法是比较有效性研究的核心关注点。鉴于癌症基因检测的快速创新和对患者结局的潜在重大影响，该方法是完善此类方法的理想领域。
与癌症临床试验联盟 SWOG 合作，开发并试点测试一种基于利益相关者的方法，为未来的研究确定基因组检测的优先级。
我们进行了一项景观分析，通过对已发表和未发表的研究进行系统搜索以及专家咨询，确定了肿瘤学中的基因组检测。将适合在比较研究中评估的临床有效检测呈递给外部利益相关者群体。在利益相关者的投入下确定了指导优先级制定过程的领域，利益相关者使用多轮投票对检测进行排名。
创建了一个利益相关者小组，其中包括来自患者倡导团体、支付方、检测开发商、监管机构、政策制定者和社区肿瘤学家的代表。我们通过利益相关者反馈确定了 9 个研究优先级领域：人群影响、当前的护理标准、关联强度、潜在临床获益、潜在临床危害、经济影响、需求证据、试验可行性和市场因素。景观分析确定了 635 项研究；在被认为具有足够临床有效性的 9 项检测中，有 6 项被提交给了利益相关者。肺癌（ERCC1 和 EGFR）中的 2 项检测和乳腺癌（CEA/CA15-3/CA27.29）中的 1 项检测被确定为首要研究重点。
以多样化的利益相关者群体为依据进行研究优先级制定是可行的，且具有务实性和及时性。需要进一步研究来优化搜索策略、利益相关者群体组成以及与现有优先级制定机制的整合。
