Cella David, Chen Chieh-I, Quek Ruben G W, Uribarren Ainhoa, Reaney Matthew, Mastey Vera, Collyar Deborah, Chassany Olivier
Northwestern University Feinberg School of Medicine, Chicago, IL, United States.
Regeneron Pharmaceuticals Inc., Tarrytown, NY, United States.
Front Pharmacol. 2022 Oct 17;13:1031992. doi: 10.3389/fphar.2022.1031992. eCollection 2022.
Regulatory agencies encourage the incorporation of the patient voices throughout clinical drug development. Patient-Reported Outcomes (PROs) offer one way of doing this and their use has markedly increased in many therapeutic areas, particularly oncology, in recent years. However, few oncology drug labels include PRO data and those which do, offer little consistency. To provide multidisciplinary perspectives (patient, pharmaceutical industry, PRO researcher, regulatory expert) on PRO data in oncology drug labels. PRO data in the labels of drugs approved by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) for oncology indications between 2010 and 2020 were critically reviewed by authors who provided their insights on the advantages and disadvantages/gaps. Forty-six oncology drugs included PRO data in their labels. Differences were observed between FDA and EMA PRO labeling (e.g., PRO concept, use of tables and graphs to display PROs or reference to clinical meaningfulness). In providing their perspectives on the number and nature of PROs in labels, authors noted limitations including: the low proportion of oncology drugs with PRO labeling, limited PRO information in labels, lack of patient-friendly language, and potential bias towards positive outcomes. Lack of consistency within- and between-agencies was noted. Despite regulatory agencies' commitment to incorporate patient voices in regulatory decisions, availability of PRO information is limited in oncology drug labels. While several PRO guidance documents are available from regulatory and Health Technology Assessment agencies, harmonization of PRO guidance for labeling inclusion around the world is needed to better inform prescribers and consequently their patients in the process of shared medical decisions.
监管机构鼓励在临床药物研发过程中纳入患者的意见。患者报告结局（PROs）提供了一种实现这一目标的方式，近年来，其在许多治疗领域，尤其是肿瘤学领域的应用显著增加。然而，很少有肿瘤学药物标签包含PRO数据，即便有包含的，其一致性也很差。为了提供关于肿瘤学药物标签中PRO数据的多学科观点（患者、制药行业、PRO研究人员、监管专家），对2010年至2020年间美国食品药品监督管理局（FDA）和欧洲药品管理局（EMA）批准的用于肿瘤学适应症的药物标签中的PRO数据进行了严格审查，作者们对其优缺点/差距发表了见解。46种肿瘤学药物在其标签中包含了PRO数据。观察到FDA和EMA的PRO标签之间存在差异（例如，PRO概念、用于展示PROs的表格和图表的使用或对临床意义的提及）。在阐述他们对标签中PROs的数量和性质的观点时，作者们指出了一些局限性，包括：带有PRO标签的肿瘤学药物比例较低、标签中的PRO信息有限、缺乏对患者友好的语言以及对积极结果的潜在偏向。注意到机构内部和机构之间缺乏一致性。尽管监管机构致力于在监管决策中纳入患者意见，但肿瘤学药物标签中PRO信息的可用性仍然有限。虽然监管机构和卫生技术评估机构提供了几份PRO指导文件，但仍需要在全球范围内统一关于纳入标签的PRO指导，以便在共同医疗决策过程中更好地为开处方者及其患者提供信息。
