Aix Marseille Univ, APHM, INSERM, Inst Neurosci Syst, UMR 1106, University Hospital Federation DHUNE, Service de Pharmacologie Clinique et Pharmacovigilance, 13005 Marseille, France.
Obmann NF Kinder/Obmann NF Patients United/Obmann EUPATI Austria, 1230 Wien, Austria.
Therapie. 2021 Mar-Apr;76(2):111-119. doi: 10.1016/j.therap.2020.12.007. Epub 2020 Dec 5.
Clinical trials involving brain disorders are notoriously difficult to set up and run. Innovative ways to develop effective prevention and treatment strategies for central nervous system (CNS) diseases are urgently needed. New approaches that are likely to renew or at least modify the paradigms used so far have been recently proposed. Quantitative systems pharmacology (QSP) uses mathematical computerized models to characterize biological systems, disease processes and CNS drug pharmacology. Integrated experimental medicine should increase the probability and predictability of success while controlling clinical trials costs. Finally, the societal perspective and patient empowerment also offer additional approaches to demonstrate the benefit of a new drug in the CNS field.
涉及脑部疾病的临床试验通常很难开展和实施。急需创新方法来制定针对中枢神经系统 (CNS) 疾病的有效预防和治疗策略。最近提出了一些新的方法，这些方法可能会更新或至少修改迄今为止使用的范例。定量系统药理学 (QSP) 使用数学计算机模型来描述生物系统、疾病过程和中枢神经系统药物药理学。综合实验医学应该在控制临床试验成本的同时，提高成功的可能性和可预测性。最后，从社会角度和患者赋权的角度来看，也为在 CNS 领域展示新药的益处提供了额外的方法。
