Department of Global Health and Social Medicine, King's College London, London, UK
Department of Population Medicine, Harvard Medical School, and Harvard Pilgrim Health Care Institute, Boston, MA, USA.
BMJ. 2023 Mar 29;380:e073711. doi: 10.1136/bmj-2022-073711.
To evaluate the frequency with which relevant and accurate information about the benefits and related uncertainties of anticancer drugs are communicated to patients and clinicians in regulated information sources in Europe.
Document content analysis.
European Medicines Agency.
Anticancer drugs granted a first marketing authorisation by the European Medicines Agency, 2017-19.
Whether written information on a product addressed patients' commonly asked questions about: who and what the drug is used for; how the drug was studied; types of drug benefit expected; and the extent of weak, uncertain, or missing evidence for drug benefits. Information on drug benefits in written sources for clinicians (summaries of product characteristics), patients (patient information leaflets), and the public (public summaries) was compared with information reported in regulatory assessment documents (European public assessment reports).
29 anticancer drugs that received a first marketing authorisation for 32 separate cancer indications in 2017-19 were included. General information about the drug (including information on approved indications and how the drug works) was frequently reported across regulated information sources aimed at both clinicians and patients. Nearly all summaries of product characteristics communicated full information to clinicians about the number and design of the main studies, the control arm (if any), study sample size, and primary measures of drug benefit. None of the patient information leaflets communicated information to patients about how drugs were studied. 31 (97%) summaries of product characteristics and 25 (78%) public summaries contained information about drug benefits that was accurate and consistent with information in regulatory assessment documents. The presence or absence of evidence that a drug extended survival was reported in 23 (72%) summaries of product characteristics and four (13%) public summaries. None of the patient information leaflets communicated information about the drug benefits that patients might expect based on study findings. Scientific concerns about the reliability of evidence on drug benefits, which were raised by European regulatory assessors for almost all drugs in the study sample, were rarely communicated to clinicians, patients, or the public.
The findings of this study highlight the need to improve the communication of the benefits and related uncertainties of anticancer drugs in regulated information sources in Europe to support evidence informed decision making by patients and their clinicians.
评估在欧洲监管信息来源中，相关和准确的抗癌药物益处及其相关不确定性信息传达给患者和临床医生的频率。
文件内容分析。
欧洲药品管理局。
2017-19 年获得欧洲药品管理局首次营销授权的抗癌药物。
产品书面信息是否针对患者常见问题进行了说明：药物的用途和对象；药物的研究方式；预期的药物获益类型；以及药物获益证据的强弱、不确定或缺失程度。比较了书面来源中针对临床医生（产品特性摘要）、患者（患者信息传单）和公众（公众摘要）的药物获益信息与监管评估文件（欧洲公共评估报告）中报告的信息。
纳入了 2017-19 年 32 种不同癌症适应证的 29 种首次获得营销授权的抗癌药物。药物的一般信息（包括批准适应证和药物作用方式的信息）在面向临床医生和患者的监管信息来源中经常被报道。几乎所有产品特性摘要都向临床医生全面传达了关于主要研究的数量和设计、对照组（如有）、研究样本量以及药物获益的主要测量值的信息。没有患者信息传单向患者传达药物研究的信息。31 份（97%）产品特性摘要和 25 份（78%）公众摘要包含了与监管评估文件中信息一致且准确的药物获益信息。23 份（72%）产品特性摘要和 4 份（13%）公众摘要报告了药物延长生存期的证据存在或不存在。没有患者信息传单向患者传达了基于研究结果可能期望的药物获益信息。研究样本中的几乎所有药物都引起了欧洲监管评估人员对药物获益证据可靠性的关注，但这些问题很少向临床医生、患者或公众传达。
本研究结果强调需要改善欧洲监管信息来源中抗癌药物益处及其相关不确定性的沟通，以支持患者及其临床医生基于证据做出决策。
