The University of Chicago Medical Center, Section of Hematology/Oncology, USA.
COTA, Inc., Boston, MA, USA.
Blood Rev. 2022 May;53:100913. doi: 10.1016/j.blre.2021.100913. Epub 2022 Mar 3.
The landscape for evidence generation in hematologic malignancies is rapidly evolving. While randomized controlled trials (RCTs) remain the gold standard in support of drug efficacy, approval and use, the supplemental use of real-world data (RWD), generated as part of routine healthcare delivery, and real-world evidence (RWE), the insights derived from RWD, in this setting has become increasingly common. There is a wide variety of sources of RWD, each with its own strengths and weaknesses that need to be considered when determining its appropriate use in RWE generation. RWD and RWE have historically been utilized in the post-approval setting to assess real-world application, efficacy, and safety of approved therapies. However, due to increasing awareness of the advantages of additional sources of information, RWE sourced from clinical data are being increasingly used to provide context for regulatory decision-making across several diseases including hematologic malignancies. Today, many commercial vendors offer fully aggregated, de-identified and standardized real-world clinical data. To maximize the potential of RWD and RWE, important considerations are needed to ensure patient privacy and to reduce the potential for biases and residual confounding. Continued collaboration among researchers, regulators and industry partners are needed to optimize evidence generation to ensure that new therapies reach patients as quickly and safely as possible.
血液恶性肿瘤的证据生成格局正在迅速发展。虽然随机对照试验（RCT）仍然是支持药物疗效、批准和使用的金标准，但在这种情况下，越来越多地补充使用真实世界数据（RWD），即常规医疗保健提供过程中生成的数据，以及真实世界证据（RWE），即从 RWD 中得出的见解，RWD 和 RWE 在批准后评估中被广泛用于评估批准疗法的实际应用、疗效和安全性。然而，由于越来越意识到额外信息来源的优势，RWE 来源于临床数据，越来越多地用于为包括血液恶性肿瘤在内的多种疾病的监管决策提供背景。如今，许多商业供应商提供完全聚合、去识别和标准化的真实世界临床数据。为了最大限度地发挥 RWD 和 RWE 的潜力，需要考虑确保患者隐私和减少潜在偏差和残余混杂的重要因素。研究人员、监管机构和行业合作伙伴需要继续合作，以优化证据生成，确保新疗法尽快且安全地惠及患者。
