Real-world data rounds out value picture of drugs

几十年来，随机临床试验一直是证明潜在新药的金标准，这是安全有效的。

然而，如今，付款人，决策者甚至患者都要求更多。他们想知道该药物在复杂的，不可预测的现实世界中如何表现 - 在哪个患者中最有效，如何与其他疗法进行比较以及哪些治疗方法最具成本效益。

格伦·舒莫克（Glen Schumock）说：“现实世界的证据说，而不仅仅是在受控（临床试验）环境中查看药物的影响，让我们了解实际临床实践中发生的情况。”，Pharm.D。，博士，伊利诺伊大学芝加哥大学药学系统，成果和政策部教授兼校长。

这就是为什么生物制药公司和学术机构越来越多地雇用研究人员和分析师团队获得数据的访问，以更好地了解医生和患者如何在诊所，医院和患者家中使用处方药。

Randomized clinical trials remain important

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.

That 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.

“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.

Additionally, 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.

“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.

Abbvie健康经济学和成果研究副总裁Tony Hebden博士说，现实世界中的数据来自许多来源，并采用了许多形式。他将其定义为“在常规的随机临床试验之外收集的信息，这些信息用于了解代表常规临床实践的环境和人群中疾病，治疗模式，患者行为和产品绩效的负担。”

这样的数据分为三个宽大的存储桶：

• 临床数据，有助于评估药物的安全性和有效性
• Economic data, used to evaluate its value
• 人文数据，通常专注于对患者重要的生活质量措施

研究人员从保险公司，医疗保健系统或国家付款人那里购买了许多匿名原始数据。这样的系统提供大量数据。赫布登说：“尽管临床试验可能有几百名患者，但一名注册表可能有十万个有关使用特定药物的记录。”

这使分析师可以通过提出问题来寻找模式，例如“医学X或药物的价值是什么？”或“所有的医生都为适当的患者开药》和“最常使用什么剂量？”

Real-world data isn't perfect