Conference: Accelerating Drug Product Development and Approval: A Critical Need in Response to National Emergencies

会议：加速药品开发和审批：应对国家紧急情况的迫切需要

• 批准号: 2232197
• 负责人: Gintaras Reklaitis
• 金额: $ 8.5万
• 依托单位: National Institute for Pharmaceutical Technology and Education
• 依托单位国家: 美国
• 项目类别: Standard Grant
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-02-15 至 2024-01-31
• 项目状态: 已结题
• 来源: https://www.nsf.gov/awardsearch/showAward?AWD_ID=2232197&HistoricalAwards=false
• 关键词: Conference; Accelerating; Drug; Product; Development

The drug discovery, development, and regulatory evaluation process, from the point where a molecule is identified as a potential therapeutic agent to when it is approved for commercialization as a drug product, can take up to 15 years. This lengthy process involves many stages, including the determination of efficacy, safety, toxicity, and therapeutic regime, identification of target populations, side effects, and interactions, selection of a feasible synthetic pathway, and development of a product formulation and a reliable manufacturing process. This lengthy time frame makes it hard to respond to emergencies requiring new therapies, deprives patients of faster access to life-saving medicines, decreases the profitability period of pharmaceutical companies, and contributes to the high cost of new medicines. Moreover, the COVID-19 pandemic has made clear the need for faster drug development and regulatory evaluation process. To address these issues, a workshop is proposed to bring together a focused group of thought leaders from academia, industry, and regulatory agencies to discuss the critical elements needed to accelerate drug discovery, development, and regulatory evaluation based on recent scientific and technical advances in medicine, biology, materials science, biostatistics, and advanced manufacturing technologies. Acceleration mechanisms will be examined from multiple perspectives, including the strength of the underlying science, technical feasibility, and regulatory acceptability. The systemic brainstorming and analysis with the participation of experts across the relevant disciplines will enable significant outcomes, including the production of a strategic plan to implement the workshop findings.The project leadership team propose to organize and conduct a structured workshop that will bring together a focused group of thought leaders from academia, industry, and regulatory agencies, to discuss the critical elements needed to accelerate drug discovery, development, and regulatory evaluation based on recent scientific and technical advances in medicine, biology, materials science, biostatistics, and advanced manufacturing technologies. These discussions will be led by pre-selected panelists with established track records in their respective fields. Acceleration mechanisms will be examined from multiple perspectives, including the strength of the underlying science, technical feasibility, and regulatory acceptability. The systemic brainstorming and analysis with the participation of experts across the relevant disciplines will generate significant outcomes, which would not be achieved in any existing scientific communication forum or conferences. Acceleration mechanisms deemed to be potentially effective will be selected for further analysis by a focused multidisciplinary team of workshop participants, which will be tasked to integrate these mechanisms into a strategic plan and an implementation pathway. These documents, which will be the main output of the workshop, will serve as a blueprint for the Federal Government, industry, and academia to direct efforts in this field. The workshop will be held in the Washington, DC area 24-26 January 2023 and will be attended by leaders from industry, academia, and government organizations such as NSF, NIH, NIST, and FDA. The proposed workshop will achieve two goals, to: 1) identify Scientific, Technological, and Regulatory mechanisms capable of significantly reducing the drug discovery, development, and commercializing time frame with improved efficiency and reduced risks to patients; and 2) integrate these mechanisms into a coherent strategy and integrated implementation plan.This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.

药物发现、开发和监管评估过程，从分子被确定为潜在治疗剂到被批准作为药品商业化，可能需要长达 15 年的时间。这个漫长的过程涉及许多阶段，包括确定功效、安全性、毒性和治疗方案，确定目标人群、副作用和相互作用，选择可行的合成途径，以及开发产品配方和可靠的制造工艺。如此漫长的时间框架使得难以应对需要新疗法的紧急情况，使患者无法更快地获得救命药物，缩短了制药公司的盈利期限，并导致新药成本高昂。此外，COVID-19 大流行明确表明需要加快药物开发和监管评估流程。为了解决这些问题，建议召开一次研讨会，将来自学术界、工业界和监管机构的思想领袖聚集在一起，根据最近的科学和技术进展，讨论加速药物发现、开发和监管评估所需的关键要素。医学、生物学、材料科学、生物统计学和先进制造技术。加速机制将从多个角度进行审查，包括基础科学的强度、技术可行性和监管可接受性。相关学科专家参与的系统性集思广益和分析将取得重大成果，包括制定战略计划来实施研讨会的结果。项目领导团队建议组织和举办一次结构化研讨会，汇集重点关注的人员来自学术界、工业界和监管机构的思想领袖组成的小组，根据医学、生物学、材料科学、生物统计学和先进制造技术的最新科学技术进展，讨论加速药物发现、开发和监管评估所需的关键要素。这些讨论将由预先选定的在各自领域拥有良好记录的小组成员主持。加速机制将从多个角度进行审查，包括基础科学的强度、技术可行性和监管可接受性。相关学科专家参与的系统性头脑风暴和分析将产生重大成果，这是任何现有的科学传播论坛或会议都无法实现的。由研讨会参与者组成的多学科重点团队将选择被认为可能有效的加速机制进行进一步分析，该团队的任务是将这些机制整合到战略计划和实施路径中。这些文件将成为研讨会的主要成果，将作为联邦政府、工业界和学术界指导这一领域工作的蓝图。该研讨会将于 2023 年 1 月 24 日至 26 日在华盛顿特区举行，来自行业、学术界和 NSF、NIH、NIST 和 FDA 等政府组织的领导人将出席。拟议的研讨会将实现两个目标：1）确定能够显着缩短药物发现、开发和商业化时间框架、提高效率并降低患者风险的科学、技术和监管机制； 2) 将这些机制整合到一个连贯的战略和综合实施计划中。该奖项反映了 NSF 的法定使命，并通过使用基金会的智力价值和更广泛的影响审查标准进行评估，被认为值得支持。

项目成果

WANTED DEAD OR ALIVE: New Thinking to Incentivize Drug Development

生死攸关：激励药物开发的新思维

• DOI: 10.1007/s11095-024-03666-6
• 发表时间: 2024
• 期刊: Pharmaceutical Research
• 影响因子: 3.7
• 作者: Kinch, Michael S.

Accelerating Drug Product Development and Approval: Early Development and Evaluation

加速药品开发和审批：早期开发和评估

• DOI: 10.1007/s11095-023-03566-1
• 发表时间: 2024
• 期刊: Pharmaceutical Research
• 影响因子: 3.7
• 作者: Bak, Annette;Burlage, Rubi;Greene, Nigel;Nambiar, Prabu;Lu, Xiuling;Templeton, Allen
• 通讯作者: Templeton, Allen