BIOLOGICS SUBJECT MATTER EXPERT (SME) CONSULTING SERVICES

生物制品主题专家 (SME) 咨询服务

• 批准号: 10719215
• 负责人: CLYDE SCHULTZ
• 金额: $ 0.6万
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-04-15 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10719215
• 关键词: Address; Antisense Oligonucleotides; Area; Biological; Biological Products; Biological Response Modifier Therapy; Cell Therapy; Cells; Chemistry; Clinical Research; Complex; Consult; Contractor; Contracts; Data; Development; Electronic Mail; Ensure; Feedback; Formulation; Government; Guidelines; Investigational Drugs; Investigational New Drug Application; Label; Leadership; Lentivirus; Liposomes; Methodology; Methods; Modality; Oligonucleotides; Peptide antibodies; Performance; Polymers; Preparation; Principal Investigator; Process; Proteins; Protocols documentation; Published Comment; Recombinant Proteins; Regulation; Regulatory Affairs; Reporting; Risk; Role; Running; Services; Site; Small Interfering RNA; Strategic Planning; Telephone; Testing; Therapeutic; Time; TimeLine; Toxicology; United States National Institutes of Health; Viral; Viral Vector; Visit; Work; analytical method; base; design; evaluation/testing; meetings; member; method development; nanoparticle; operation; preclinical evaluation; product development; programs; quality assurance; research clinical testing; safety testing; scale up; symposium; therapeutic development

Performance Area 1: Biologics Chemistry, Manufacturing and Controls (CMC) SME(s) Subject matter experts in manufacturing of the various biological therapeutics platforms, including but not limited to, modalities such as therapeutic oligonucleotides (e.g., antisense oligonucleotide -ASOs, small interfering RNAs -siRNAs, etc.), AAV, lentivirus or other viral vector therapeutics, purified proteins, recombinant proteins, peptides, antibody-based and cell-based therapeutics, and ex vivo cellular therapies are required. Biologics CMC consultants will be expected to provide senior-level chemistry, manufacturing and controls expertise and contribute feedback and guidance on projects to the NIH and to LDT members through conference calls and by email. Please note that, for Performance Area 1, the Government will consider SME consultants with expertise in one or more manufacturing modalities. The role of the CMC SME consultant may include, but is not limited to, the following responsibilities and tasks: a) Review and comment on the adequacy of biologic manufacturing, including scale-up, analytical method development, and process development (upstream and downstream processing) approaches proposed by Principal Investigators (PIs) and CMOs b) Provide guidance on delivery methods, which may include complex formulations such as polymer, liposomes, and other nanoparticle constructs c) Provide expert advice on master and working cell and viral bank development and testing d) Provide strategy and feedback on biologics process development and manufacturing plans proposed by the team e) Provide feedback on therapeutic oligonucleotide sequences and synthesis processes proposed by the team f) Provide feedback on proposed biologics formulations g) Provide feedback on critical quality attributes, release testing, etc. h) Evaluate CMC data on a weekly or biweekly basis i) Serve as the CMC expert in order to identify potential product manufacturing challenges and suggest strategies to address these challenges j) Accompany NIH staff or visit, at NIH staff request, CROs and CMOs under contract to the NIH. k) Inspect facilities and discuss appropriateness of CRO/CMO proposed methodology l) CMC consultant may be asked to evaluate the potential tractability (including surrounding IP space) of a proposed biological therapeutic m) Collaborate on design of investigative studies in support of therapeutics development projects n) In partnership with other consultants, contractors, and the NIH staff, advise, strategically plan, and manage CMC to facilitate preclinical evaluation and safety testing, Investigational New Drug (IND) submissions, and clinical studies o) Facilitate LDT discussions via telephone and email regarding assigned CMC efforts p) Provide strategic guidance for the continued development and improvement of the BPN-Biologics, HEAL PTDP, and URGenT biologics programs Performance Area 5 – Regulatory Affairs and Regulatory Operations SME(s) Regulatory Affairs consultants will provide advice and leadership in support of regulatory affairs strategy for NIH biologics programs. Consultants will be asked to provide guidance on IND-enabling studies to ensure that teams meet the guidelines and timelines of IND applications. Consultants will serve as the principal regulatory contact with NIH staff, PIs, and regulatory agencies, and may also be the primary regulatory representative to the FDA. The roles of the Regulatory Affairs and Regulatory Operations SME consultants may include but are not limited to the following responsibilities and tasks: a) Work within project teams to develop regulatory strategies, identify regulatory risks, and enable timely approval b) Advise NIH staff and project teams on issues related to regulatory strategy and identify areas of concern regarding developing regulations c) Provide input on and review specifications, methods, SOPs, protocols, reports, labeling, etc. for regulatory compliance d) Develop and manage timelines for regulatory submissions e) Work effectively to coordinate the activities of CROs and other consultants in the preparation of regulatory submissions as necessary f) Prepare and submit IND applications to the appropriate regulatory agency g) Prepare for and run regulatory agency meetings Performance Area 8 – Quality Assurance (QA) SME(s) The Quality Assurance SME Consultant will provide quality oversight of activities supporting product development and clinical evaluation of biological therapeutics under NIH biologics programs. The Quality Assurance SME will: a) Draft, review, and approve relevant sections of regulatory filings. b) Advise product development team on best practices and processes c) Ensure CRO/CMO compliance to regulations and quality requirements d) Be responsible for conducting quality checks e) Be responsible for deviation assessment f) Support audits of manufacturing CMO facilities and studies g) Support audits of toxicology CRO facilities and studies h) Accompany NIH staff or visit, at NIH staff request, CROs and CMOs under contract to the NIH. Inspect facilities and discuss appropriateness of CRO/CMO proposed methodology i) Conduct other site audits and study report audits as required

性能领域1：生物制剂化学、生产和控制（CMC）SME 制造各种生物治疗平台的主题专家，包括但不限于治疗性寡核苷酸（例如，反义寡核苷酸-AS 0、小干扰RNA-siRNA等），需要AAV、慢病毒或其他病毒载体治疗剂、纯化蛋白、重组蛋白、肽、基于抗体和基于细胞的治疗剂以及离体细胞治疗。生物制剂CMC顾问将提供高级化学，制造和控制专业知识，并通过电话会议和电子邮件向NIH和LDT成员提供项目反馈和指导。 请注意，对于业绩领域1，政府将考虑在一种或多种制造模式方面具有专门知识的中小企业顾问。 CMC SME顾问的职责可能包括但不限于以下职责和任务： a）、 审查和评论生物制品生产的充分性，包括主要研究者（PI）和CMO提出的规模扩大、分析方法开发和工艺开发（上游和下游加工）方法 B） 提供递送方法的指导，其中可能包括复杂的制剂，如聚合物、脂质体和其他纳米颗粒结构 c）、 为主细胞、工作细胞和病毒库的开发和检测提供专家建议 d）、 对团队提出的生物制剂工艺开发和生产计划提供策略和反馈 e）、 对团队提出的治疗性寡核苷酸序列和合成工艺提供反馈 f）的 提供关于拟定生物制剂的反馈 g）的 提供关键质量属性、放行检测等方面的反馈。 h）的 每周或每两周评估CMC数据 （一） 作为CMC专家，识别潜在的产品制造挑战，并提出解决这些挑战的策略 j）、 陪同NIH工作人员或根据NIH工作人员的要求访问与NIH签订合同的CRO和CMO。 k）的 检查设施并讨论CRO/CMO提议方法的适当性 （一） 可能要求CMC顾问评估拟定生物治疗药物的潜在可处理性（包括周围IP空间） 米）的 合作设计调查性研究，以支持治疗开发项目 n）的 与其他顾问、承包商和NIH工作人员合作，为CMC提供建议、战略规划和管理，以促进临床前评价和安全性测试、新药临床试验（IND）提交和临床研究 o） 通过电话和电子邮件促进LDT讨论分配的CMC工作 p）的范围内 为BPN生物制剂、HEAL PTDP和URGenT生物制剂项目的持续开发和改进提供战略指导 性能领域5 -法规事务和法规运营SME法规事务顾问将提供建议和领导，以支持NIH生物制剂项目的法规事务策略。顾问将被要求提供IND使能研究的指导，以确保团队符合IND申请的指导方针和时间表。顾问将作为NIH工作人员、PI和监管机构的主要监管联系人，也可能是FDA的主要监管代表。 法规事务和法规运营SME顾问的职责可能包括但不限于以下职责和任务： a）、 与项目团队合作，制定监管策略，识别监管风险，并及时批准 B） 就监管策略相关问题向NIH工作人员和项目团队提供建议，并确定有关制定法规的关注领域 c）、 为法规合规性提供质量标准、方法、SOP、方案、报告、标签等的输入和审查 d）、 制定和管理监管提交的时间表 e）、 有效协调CRO和其他顾问在准备监管申报资料时的活动（如必要） f）的 准备并向相应的监管机构提交IND申请 g）的 准备和召开监管机构会议 绩效领域8 -质量保证SME 质量保证SME顾问将对NIH生物制剂项目下支持产品开发和生物疗法临床评价的活动进行质量监督。 质量保证SME将： a）、 起草、审核和批准监管文件的相关章节。 B） 为产品开发团队提供最佳实践和流程建议 c）、 确保CRO/CMO符合法规和质量要求 d）、 负责质量检查 e）、 负责偏差评估 f）的 支持生产CMO设施和研究的稽查 g）的 支持毒理学CRO机构和研究的稽查 h）的 陪同NIH工作人员或根据NIH工作人员的要求访问与NIH签订合同的CRO和CMO。检查设施并讨论CRO/CMO提议方法的适当性 （一） 根据需要进行其他研究中心稽查和研究报告稽查