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我们如何在肿瘤学及其他领域实现精准医疗的承诺?一份切实可行的行动路线图。

How can we deliver on the promise of precision medicine in oncology and beyond? A practical roadmap for action.

作者信息

Baird Anne-Marie, Westphalen C Benedikt, Blum Sandra, Nafria Begonya, Knott Tanya, Sargeant Ify, Harnik Helena, Brooke Nicholas, Wicki Nicole, Wong-Rieger Durhane

机构信息

Lung Cancer Europe (LuCE) Bern Switzerland.

From Testing to Targeted Treatments (FT3) Program Team, The Synergist Brussels Belgium.

出版信息

Health Sci Rep. 2023 Jun 22;6(6):e1349. doi: 10.1002/hsr2.1349. eCollection 2023 Jun.

Abstract

BACKGROUND

Precision medicine (PM) is a form of personalized medicine that recognizes that individuals with the same condition may have different underlying factors and uses molecular information to provide tailored treatments. This approach can improve treatment outcomes and transform lives through favorable risk/benefit ratios, avoidance of ineffective interventions, and possible cost savings, as evidenced in the field of lung cancer and other oncology/therapeutic settings, including cardiac disease, diabetes, and rare diseases. However, the potential benefits of PM have yet to be fully realized.

DISCUSSION

There are many barriers to the implementation of PM in clinical practice, including fragmentation of the PM landscape, siloed approaches to address shared challenges, unwarranted variation in availability and access to PM, lack of standardization, and limited understanding of patients' experience and needs throughout the PM pathway. We believe that a diverse, intersectoral multistakeholder collaboration, with three main pillars of activity: generation of data to demonstrate the benefit of PM, education to support informed decision-making, and addressing barriers across the patient pathway, is necessary to reach the shared goal of making PM an accessible and sustainable reality. Besides healthcare providers, researchers, policymakers/regulators/payers, and industry representatives, patients in particular must be equal partners and should be central to the PM approach-from early research through to clinical trials and approval of new treatments-to ensure it represents their entire experience and identifies barriers, solutions, and opportunities at the point of delivery.

CONCLUSION

We propose a practical and iterative roadmap to advance PM and call for all stakeholders across the healthcare system to employ a collaborative, cocreated, patient-centered methodology to close gaps and fully realize the potential of PM.

摘要

背景

精准医学(PM)是个性化医疗的一种形式,它认识到患有相同疾病的个体可能有不同的潜在因素,并利用分子信息提供量身定制的治疗方案。这种方法可以改善治疗效果,并通过有利的风险/收益比、避免无效干预以及可能节省成本来改变生活,肺癌和其他肿瘤学/治疗领域(包括心脏病、糖尿病和罕见病)的情况已证明了这一点。然而,精准医学的潜在益处尚未得到充分实现。

讨论

在临床实践中实施精准医学存在许多障碍,包括精准医学领域的碎片化、解决共同挑战的孤立方法、精准医学的可及性和获取方面存在不必要的差异、缺乏标准化,以及在整个精准医学过程中对患者体验和需求的理解有限。我们认为,需要开展多部门、多利益相关方的多元化合作,其活动主要有三大支柱:生成数据以证明精准医学的益处、开展教育以支持明智的决策,以及解决患者就医过程中的障碍,以实现使精准医学成为可及且可持续现实这一共同目标。除了医疗服务提供者、研究人员、政策制定者/监管者/支付方和行业代表外,患者尤其必须成为平等的合作伙伴,并且应该在精准医学方法中处于核心地位——从早期研究到临床试验以及新疗法的批准——以确保它代表了患者的全部体验,并在交付点识别出障碍、解决方案和机会。

结论

我们提出了一个推进精准医学的实用且迭代的路线图,并呼吁医疗系统中的所有利益相关方采用协作式、共同创建的、以患者为中心的方法来弥合差距,充分实现精准医学的潜力。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6be/10286856/7ed4ce446287/HSR2-6-e1349-g003.jpg

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