University of South Alabama College of Medicine, Departments of Psychiatry and Neurology, Mobile, AL, USA.
Independent Medical Consultant, Doylestown, PA, USA.
Adv Neurobiol. 2023;30:169-180. doi: 10.1007/978-3-031-21054-9_6.
A prescriber might ask if a new medication is a good option for use in the patients he or she sees in clinic, with their particular blends of demographic and comorbid clinical characteristics. Is this medicine more effective, safe, tolerable, or affordable than the options used in the past? A payer may ask if the new medication offers a more effective, cost-efficient, or convenient alternative to those treatments already being covered. These are the types of questions that are often difficult to answer on the basis of the clinical trials used to support a medication's initial approval, which are generally designed to evaluate a medication's efficacy, safety, and tolerability in narrowly defined patient populations. Consequently, in order to answer the questions most relevant to key stakeholders (i.e., regulators, patients, and clinicians), it is important to continue to examine a medication's impact and characteristics after it has received regulatory approval. Such studies vary in their purpose, scope, and methodology. In this chapter, we review the types of questions most likely to be investigated after regulatory approval, the methods generally used to investigate them, and the characteristics typically considered when prioritizing the allocation of resources.
医生可能会询问某种新药是否适合他/她在临床中看到的患者使用，因为这些患者具有特定的人口统计学和合并症临床特征组合。与过去使用的药物相比，这种药物在疗效、安全性、耐受性或可负担性方面是否更优？支付方可能会询问新药物相对于已涵盖的治疗方法是否具有更有效、更具成本效益或更方便的替代方案。这些通常是难以根据支持药物初始批准的临床试验来回答的问题，临床试验通常旨在评估药物在定义明确的患者人群中的疗效、安全性和耐受性。因此，为了回答与关键利益相关者（即监管机构、患者和临床医生）最相关的问题，重要的是在药物获得监管批准后继续检查药物的影响和特征。这些研究在目的、范围和方法上有所不同。在本章中，我们将回顾监管批准后最有可能调查的问题类型、通常用于调查这些问题的方法以及在优先分配资源时通常考虑的特征。
