Department of Public Health and Community Medicine, Tufts Center for the Study of Drug Development, Tufts University School of Medicine, Tufts University, Boston, Massachusetts, USA.
Clin Pharmacol Ther. 2014 Jan;95(1):98-109. doi: 10.1038/clpt.2013.155. Epub 2013 Aug 5.
Broadly speaking, the goals of the US Food and Drug Administration (FDA) special-designation programs--orphan, priority review, accelerated approval, and fast track--have been to expedite and sustain development and facilitate authorization of new medicines for unmet medical needs through so-called "push-pull" incentives. Although generally successful over time, their success has been confined to certain therapeutic areas and, within those areas, certain diseases. Times have changed. The research and development (R&D) burdens and public health urgency that acted as an impetus for the FDA to intervene more actively for certain disease areas are now broadly experienced across many disease areas. This betokens the need for the FDA to make designation and implementation decisions with a view that reaches beyond the immediate horizons of political expediency and patient advocacy to encompass the broader expanse of factors that now influence R&D decisions--global competitiveness, the needs of investors, emerging sponsors, and patient-focused drug development.
广义而言，美国食品和药物管理局 (FDA) 特殊指定计划（孤儿药、优先审查、加速批准和快速通道）的目标一直是通过所谓的“推-拉”激励措施，加快和维持未满足医疗需求的新药开发，并促进其批准。尽管随着时间的推移它们总体上取得了成功，但它们的成功仅限于某些治疗领域，并且在这些领域内，也仅限于某些疾病。时代已经变了。现在，许多疾病领域都广泛感受到了促使 FDA 更积极干预某些疾病领域的研发负担和公共卫生紧迫性。这意味着 FDA 需要做出指定和实施决策，不仅要考虑当前的政治权宜之计和患者倡导，还要考虑现在影响研发决策的更广泛因素——全球竞争力、投资者的需求、新兴赞助商和以患者为中心的药物开发。
