Waldman Scott A, Terzic Andre
Department of Pharmacology and Experimental Therapeutics, Division of Clinical Pharmacology, Department of Medicine, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.
Center for Regenerative Medicine, Mayo Clinic, Rochester, Minnesota, USA.
Clin Pharmacol Ther. 2018 Jan;103(1):8-12. doi: 10.1002/cpt.929.
Deconvoluting key biological mechanisms forms the framework for therapeutic discovery. Strategies that enable effective translation of those insights along the development and regulatory path ultimately drive validated clinical application in patients and populations. Accordingly, parity in What vs. How we transform novel mechanistic insights into therapeutic paradigms is essential in achieving success. Aligning molecular discovery with innovations in structures and processes along the discovery-development-regulation-utilization continuum maximizes the return on public and private investments for next-generation solutions in managing health and disease.
解析关键生物学机制构成了治疗发现的框架。能够沿着研发和监管路径有效转化这些见解的策略最终推动在患者和人群中得到验证的临床应用。因此，在如何将新的机制见解转化为治疗范式方面实现平衡对于取得成功至关重要。使分子发现与发现-开发-监管-应用连续体中的结构和流程创新保持一致，可最大限度地提高公共和私人投资在管理健康与疾病的下一代解决方案方面的回报。
