A case for a science-informed perspective on health care reform.
A case for a science-informed perspective on health care reform.
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以科学视角看待医疗保健改革的案例。
DOI：
10.1172/jci41038
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发表时间：
2009
期刊：
影响因子：
--
通讯作者：
Mischel,Paul
中科院分区：
文献类型：
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作者：
Mischel,Paul
作为一名当选为美国临床调查学会代表的内科科学家，我希望至少能为一些内科科学家发声，他们不知疲倦地将自己的发现从实验室转化为改善患者护理的工作。我们工作是因为我们相信，阐明疾病的分子基础将转化为更有效的治疗方法，减轻疾病痛苦的负担，改善数百万美国人(以及全球人民)的生活。这就是驱使我们前进的原因。我们中的许多人还认为，分子医学通过针对最有可能从中受益的患者亚群进行更具体、毒性更低的治疗，也可以降低医疗成本，改善护理。因此，我们对医疗保健辩论的结果深感关注。作为个人，我希望医疗改革是人道和普遍的，在范围上是普遍的，在细节上是科学的--简而言之，我们国家必须成为文明的希望灯塔。通过应用强大的技术来研究人类患者的基因、蛋白质和代谢物，我们了解到各种不同的分子变化可能是表型相似疾病的基础。最初被认为是一种同质疾病的实体更有可能代表通向一种常见疾病表型的多条致病途径。当针对特定基因或生化改变的药物被用作治疗时，这种区别不仅仅是学术上的。普通美国人不需要了解分子生物学就能认识到，他们的医疗保健可能会受到这种影响。以癌症为例。几乎每个美国人都会受到癌症的挑战，无论是他们自己的癌症，还是他们亲人的癌症。我们都可能在生命中的某个时候熟悉传统抗癌治疗的痛苦和失望，包括细胞毒性化疗和放射治疗，这些治疗是有毒的，只能略微延长大多数癌症患者的生命，这些患者的癌症是在晚期被发现的。分子医学有望改变这一局面。对癌症基础的研究已经开始产生针对癌细胞所需特定分子病变的新疗法，为更有效、毒性更低的疗法提供了可能性。这些靶向性病变通常只存在于该类型癌症患者的一小部分中。事实上，大多数患有这种癌症的患者都不会受益。传统的衡量标准会将这些新的靶向治疗归入“不起作用”类别，但对于患有特定靶向病变的患者来说，这些治疗可能是挽救生命的。可以想象，在未来，每个患者的癌症都会被分析出有针对性的病变，然后定制个性化的治疗计划，以最好地治疗他们的疾病。因此，这一结果关系到所有美国人的利益。实现这一未来的道路将需要具有前瞻性的、科学知情的医疗改革，在精心设计的临床试验中，开发、测试并将生物标记物纳入新疗法的疗效确定中，以便我们能够更好地回答这样一个问题：哪些有效，哪些无效？通过问：“什么对谁有效？”我相信，如果大多数美国人认识到这一事实，他们会很乐意投资于个性化他们的事业的过程。我不是经济学家。我声称，在理解塑造这一挑战的预算问题的全部范围方面，我没有专业知识。然而，我认为，这不仅仅是一场关于削减成本和改善获得护理的机会的辩论。这也是开发一个框架来评估…正在进行的新测试和治疗的机会
As a physician-scientist elected to represent the American Society for Clinical Investigation, I hope to provide a voice for at least some of the physician-scientists who tirelessly devote their energies to translating their findings from the laboratory to improve patient care. We labor because we believe elucidating the molecular underpinnings of disease will translate into more effective treatments that ease the burden of disease suffering and improve the lives of millions of Americans (and people across the globe). That is what drives us. Many of us also believe that molecular medicine, by directing more specific, less toxic treatments to subsets of patients most likely to benefit from them, can also reduce medical costs and improve care. Thus, we are deeply invested in the outcome of the health care debate. As an individual, I hope for health care reform that is humane and pervasive, universal in scope, and science-informed in its details—in short, the kind of health care reform our country must have to be the civilized beacon of hope it deserves to be. Through application of powerful technologies to study genes, proteins, and metabolites in human patients, we have learned that a variety of different molecular alterations can underlie phenotypically similar diseases. An entity originally considered as one homogeneous disease more likely represents multiple pathogenic routes toward a common disease phenotype. This distinction is more than academic when drugs that target specific genetic or biochemical alterations are used as treatments. The average American does not need to understand molecular biology to recognize that their health care could be greatly affected by this. Take cancer as an example. Virtually every American will be challenged by cancer, either their own or in a loved one. We are all likely to become familiar at some point in our lives with the pain and disappointment of traditional anticancer treatments, including cytotoxic chemotherapy and radiation, that are toxic and only modestly improve the lifespan for most patients whose cancers are detected at an advanced stage. Molecular medicine promises to change this. Studying the underpinnings of cancer has begun to yield new treatments that target specific molecular lesions needed by the cancer cells, providing the potential for much more effective, less toxic treatments. These targetable lesions are usually only present in a subset of patients with that cancer type. In fact, most patients with that type of cancer won’t benefit. Traditional metrics would put these new targeted treatments into the “doesn’t work” category, yet for patients with the specific targetable lesion, these treatments may be life saving. One can imagine a future in which each patient’s cancer is analyzed for targetable lesions and then a personalized treatment plan is tailored to best treat their disease. Thus, all Americans have a stake in the outcome. A path toward realizing this future will require forward-thinking, scienceinformed health care reform that develops, tests, and incorporates biomarkers into the determination of efficacy of new treatments in well-designed clinical trials so that we can better answer the question “What works and what doesn’t?” by asking,“What works for whom?” I believe that if most Americans were to recognize this fact, they would gladly invest in a process to personalize their care.I am not an economist. I claim no expertise in understanding the full scope of budgetary issues shaping this challenge. However, I believe that this is a debate about more than just cutting costs and improving access to care. It is also an opportunity to develop a framework for evaluating new tests and treatments that are being …