Algorri Marquerita, Cauchon Nina S, Christian Twinkle, O'Connell Chelsea, Vaidya Pujita
Department of Global Regulatory Affairs and Strategy - CMC, Amgen Inc, Thousand Oaks, CA 91320, USA.
Department of Global Regulatory Affairs and Strategy - CMC, Amgen Inc, Thousand Oaks, CA 91320, USA.
J Pharm Sci. 2023 Apr;112(4):922-936. doi: 10.1016/j.xphs.2023.01.029. Epub 2023 Feb 3.
Patient-centric drug development describes the systematic approach to incorporating the patient's perspectives and preferences into the design, assessment, and production of a therapeutic product. While a patient centric approach can be applied at any stage of the drug development lifecycle, an integrated end-to-end strategy is often most effective to create an optimized product for the patient at the earliest possible timepoint. The importance of patient centricity is well recognized by health authorities and biopharmaceutical organizations which have established toolsets, guidances, and methodologies for incorporating patient input during the clinical stage of development. However, in addition to clinical research, there are other significant aspects of product development that profoundly impact the patient experience. Specifically, chemistry, manufacturing, and control (CMC) and device aspects must also be acknowledged and addressed as part of a cohesive patient-centric development strategy. This review explores current applications and regulatory considerations for patient-centric approaches across the product lifecycle, including R&D, early product development, clinical development, device and combination product development, and post-approval change management. Specific topics of discussion include the contributions of product modality, formulation, and devices to the patient experience; usage of the Quality Target Product Profile (QTPP) as a patient-centered design tool; and post-approval product optimization. Future advancements in regulatory data management and information exchange are also explored as potential enablers of patient engagement which support enhanced communication and interconnectivity between stakeholders. Multidisciplinary collaboration between patients, health authorities, health care providers, and the biopharmaceutical industry is ultimately necessary for ensuring that medicinal products, and their corresponding regulatory processes, take on a patient-first mindset that prioritizes patient needs, values, and preferences.
以患者为中心的药物研发描述了一种系统方法，即将患者的观点和偏好纳入治疗产品的设计、评估和生产过程。虽然以患者为中心的方法可应用于药物开发生命周期的任何阶段，但采用端到端的综合策略通常最有效，能够在尽可能早的时间点为患者打造优化产品。卫生当局和生物制药组织已充分认识到以患者为中心的重要性，它们建立了工具集、指南和方法，以便在临床研发阶段纳入患者的意见。然而，除了临床研究之外，产品开发的其他重要方面也会对患者体验产生深远影响。具体而言，化学、制造和控制（CMC）以及器械方面也必须作为以患者为中心的连贯开发策略的一部分得到认可和解决。本综述探讨了在产品生命周期中以患者为中心的方法的当前应用和监管考量，包括研发、早期产品开发、临床开发、器械和组合产品开发以及批准后变更管理。具体讨论的主题包括产品形式、剂型和器械对患者体验的贡献；使用质量目标产品概况（QTPP）作为以患者为中心的设计工具；以及批准后产品优化。还探讨了监管数据管理和信息交换方面的未来进展，将其作为患者参与的潜在推动因素，以支持利益相关者之间加强沟通和互联互通。患者、卫生当局、医疗服务提供者和生物制药行业之间的多学科合作对于确保药品及其相应的监管流程秉持患者至上的理念至关重要，该理念优先考虑患者的需求、价值观和偏好。
