现实世界的数据轮价值的药物

For decades, randomized clinical trials have been the gold standard for demonstrating a potential new medicine is safe and effective.
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\r\nNowadays, however, payers, policymakers and even patients are demanding more. They want to know how that drug will behave in the complex, unpredictable real world – in which patients it will be most effective, how it compares with other treatments, and which treatments are most cost effective.
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\r\n“Real-world evidence is saying, rather than just looking at the effects of the drug in a controlled (clinical trial) environment, let's understand what happens in actual clinical practice,” and use that to inform treatment and reimbursement decisions, says Glen Schumock, Pharm.D., Ph.D., professor and head of the Department of Pharmacy Systems, Outcomes and Policy at the University of Illinois at Chicago.
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\r\nThat’s why biopharmaceutical companies and academic institutions increasingly are employing teams of researchers and analysts, with access to terabytes of data, to better understand how prescription drugs are being used by physicians and patients in clinics, hospitals and patients’ homes.

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几十年来,随机临床试验已经证明一个潜在的新药的黄金标准是安全的和有效的。

现在,然而,纳税人,决策者甚至病人要求更多。他们想知道毒品行为的复杂的,不可预知的现实世界——在哪些患者最有效的,它是如何与其他治疗方法相比,哪种治疗方式是最有效的。

“真实的证据是说,而不是看着的影响药物在控制(临床试验)环境中,让我们了解在实际临床实践中,到底发生了什么”,用它来通知治疗和补偿决定,格伦Schumock Pharm.D说。博士,教授和部门负责人药房系统,结果和政策在芝加哥伊利诺伊大学。

这就是为什么生物制药公司和学术机构越来越多地采用团队的人员和分析师,与tb的数据,更好地了解处方药正被医生和病人在诊所、医院和病人的家庭。

That doesn’t mean randomized clinical trials are going away. They remain the gold standard for determining whether a new drug is safe and efficacious. They are required by global regulatory bodies, including the U.S. Food and Drug Administration, the European Medicines Agency and many others for the approval of new medicines.
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\r\nClinical trials are carefully designed, focusing on very specific types of patients. Those patients take their medicines according to a pre-defined schedule, attend frequent follow-up visits and have routine lab tests. This enables clinical trials to answer the questions, “is this medicine safe and effective for its intended use?”

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这并不意味着随机临床试验准备外出。他们仍然是黄金标准确定一个新的药物是安全的和有效的。他们要求全球监管机构,包括美国食品和药物管理局,欧洲药品局和其他许多新药的批准。

临床试验是精心设计,专注于特定类型的病人。患者把他们的药物根据预定义的安排,参加后续互访频繁,常规实验室测试。这使临床试验来回答这个问题,“这药是安全的和有效的,它?”

Once a new medicine is approved, doctors may choose to prescribe it for patients who differ in some ways from participants in the clinical trials. They might have other health conditions besides the approved indication or disease, for example, or they might take additional medications that clinical trial participants were not allowed to take. As a result, clinical trials don’t perfectly predict what will happen in the real world. The new drug may work better in some patients than others, and patients may notice different side effects than those observed in clinical trials.
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\r\nThat creates challenges for payers, who must decide whether to include newly approved drugs on their formularies and what co-pays to charge. The limited information from clinical trials may not be enough to make these decisions. This is where real-world evidence can play a role.
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\r\n“Studies using real-world data answer the question `does this drug work under actual conditions, and how does it compare to what is already available?’ ” Schumock says.
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\r\nAdditionally, regulators and biopharmaceutical companies use real-world evidence to monitor the safety profile of prescription medicines. Such evidence can lead to label enhancements and extensions.
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\r\n“In the end you really need both kinds of studies. We will continue to have randomized clinical trials as the gold standard for drug development, but we also need to have these Phase 4 or comparative effectiveness or real-world evidence studies,” Schumock says.

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新药获得批准后,医生可以给病人开它在某些方面不同参与者的临床试验。他们可能有其他健康问题的人,除了批准或疾病迹象,例如,或者他们可能会采取额外的药物临床试验参与者不允许。因此,临床试验不完全预测在现实世界中会发生什么。这种新药可能比其他人更好地工作在一些病人,病人可能会注意到不同的比在临床试验中观察到的副作用。

为纳税人创造挑战,他们必须决定是否包括新批准药物的处方和自费部分费用。从临床试验的有限信息可能不足以做出这些决策。这就是真实的证据可以扮演一个角色。

“研究使用真实的数据回答这个问题“这药物在实际情况下工作,它如何与可用是什么?’”Schumock说。

此外,监管机构和生物制药公司使用真实的证据监控处方药物的安全性。这样的证据会导致标签增强和扩展。

“最后你真的需要这两种类型的研究。我们将继续有随机临床试验作为药物开发的黄金标准,但我们也需要这些阶段4或比较或真实的证据有效性研究,”Schumock说。狗万正网地址

Real-world data falls into three broad buckets:

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• Clinical data, which helps evaluate a drug’s safety and effectiveness
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• Economic data, used to evaluate its value
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• Humanistic data, typically focused on quality of life measures important to patients
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\r\nResearchers buy much of the anonymized raw data from insurance companies, health care systems or national payers. Such systems provide significant amounts of data. 
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\r\nThat allows analysts to look for patterns by posing questions, such as “what is the value of medicine X or medicine Y?” Or “are all physicians prescribing the medication to the appropriate patients” and “what dosage is used most often?”

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现实世界的数据分为三个大水桶:

• 临床数据,这有助于评估药物的安全性和有效性
• 经济数据,用来评估其价值
• 人文数据,通常专注于重要的病人生活质量的措施

研究人员从保险公司购买匿名的原始数据,卫生保健系统或国家的纳税人。这样的系统提供大量的数据。

分析师可以通过提出问题,寻找规律,如“医学医学X或Y的值是什么?”或“都是医生处方药物适当的病人”和“最常使用剂量是什么?”

Real-world data has limitations, however. If all the patients in an insurance company’s database come from one area of the country, for example, or are of a particular socioeconomic status, the results might not apply to other areas or groups of patients.
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\r\nAlso, most real-world data isn’t as reliable or clean as clinical trial data. It typically is collected to enable payment of insurance claims or to help doctors track an individual patient’s progress.
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\r\nAlso, most commercial databases don’t include much quality of life or other humanistic data, “and that’s a problem,” says Jeremy Gilbert, vice president of PatientsLikeMe, an online patient social network and research platform.
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\r\nMost databases focus primarily on clinical data. “The patient is going to look and ask `what does this do for me? Does it help me live a longer or better life as a result?’” Gilbert adds.

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然而,现实世界的数据有一定的局限性。如果保险公司的数据库中所有病人来自一个地区的国家,例如,或特定的社会经济地位,结果可能不适用于其他领域或组的患者。

同时,大多数真实的数据不可靠或干净的临床试验数据。它通常被收集,使支付保险索赔或帮助医生跟踪单个病人的进步。

同时,大多数商业数据库不包括生活质量或其他人文数据,“这是一个问题,”杰里米•吉尔伯特说,副总裁PatientsLikeMe病人一个在线社交网络和研究平台。

大多数数据库主要侧重于临床数据。“病人要看,问“这为我做什么呢?它帮助我生活较长或更好的结果吗?“吉尔伯特补充道。

That’s why researchers increasingly are looking to newer, more innovative sources of data, including patient networks, to supplement traditional data sources. PatientsLikeMe uses science-based principles to collect and analyze patient-reported data from more than 400,000 members about the things that matter to them. “It puts the value of a drug in the context of a disease," Gilbert says.
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\r\n“We hope that there is patient-generated evidence that can be journal worthy and even find its way into regulatory submissions for new products,” Gilbert says. "If we can get that trifecta of patient-generated data, outcomes data and medical records all in the mix, we'll have a really amazing real-world evidence base to use for all kinds of decisions."

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这就是为什么研究人员越来越多地寻求新的、更具创新性的数据来源,包括病人网络,来补充传统的数据源。PatientsLikeMe使用科学的原则来收集和分析patient-reported数据来自400000多个成员的事情。“它把一种药物的价值上下文中的疾病,”吉尔伯特说。

“我们希望我们有证据表明,杂志值得甚至可以找到进入监管提交新产品,”吉尔伯特说。“如果我们能得到我们的小游戏数据,结果数据和病历都混杂在一起,我们将有一个非常神奇的实际证据的基础上使用的各种各样的决策。”