BIOLOGICS SUBJECT MATTER EXPERT (SME) CONSULTING SERVICES

生物制品主题专家 (SME) 咨询服务

• 批准号: 10829324
• 负责人: CLYDE SCHULTZ
• 金额: $ 1.45万
• 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 2024-04-14
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10829324
• 关键词: Address; Antibodies; Antisense Oligonucleotides; Area; Biochemistry; Biological; Biological Process; Biological Products; Biological Response Modifier Therapy; Cell Therapy; Cells; Chemistry; Clinical Research; Collaborations; Complex; Contractor; Contracts; Data; Development; Electronic Mail; Ensure; Feedback; Formulation; Government; Guidelines; Helping to End Addiction Long-term; Investigational Drugs; Investigational New Drug Application; Label; Leadership; Lentivirus; Liposomes; Methodology; Methods; Modality; Oligonucleotides; Pain; Pain management; Peptides; Performance; Polymers; Preparation; Principal Investigator; Process; Protocols documentation; Published Comment; Recombinant Proteins; Regulation; Regulatory Affairs; Reporting; Risk; Role; Running; Services; Site; Small Interfering RNA; Specific qualifier value; Speed; Strategic Planning; Telephone; Testing; Therapeutic; Toxicology; United States National Institutes of Health; Viral; Viral Vector; Visit; Work; analytical method; clinical pain; design; evaluation/testing; improved; manufacture; meetings; member; method development; nanoparticle; operation; preclinical evaluation; product development; programs; protein purification; quality assurance; research clinical testing; safety testing; scale up; symposium; therapeutic development; timeline

“This study is part of the NIH’s Helping to End Addiction Long-term (HEAL) initiative to speed scientific solutions to understand the basis of pain and enhance clinical pain management.” 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

“这项研究是NIH的一部分，有助于结束长期（治愈）倡议，以加快科学解决方案，以了解疼痛的基础并增强临床疼痛管理。” 性能区域1：生物制剂，制造和控制（CMC）中小型企业 Subject matter experts in manufacturing of the various biologics 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 therapy, purified proteins, recombinant proteins, pepperides, antibody-based and cell-based therapy,并且需要离体细胞疗法。预计Biologics 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成员提供有关项目的反馈和指导。 请注意，对于绩效区域1，政府将考虑具有一种或多种制造方式的专业知识的中小企业顾问。 CMC SME顾问的作用可能包括但不限于以下职责和任务： a）审查和评论生物制造的充分性，包括大规模，分析方法开发和过程开发（上游和下游处理）方法，由首席研究人员（PIS）和CMO提出 b）提供有关递送方法的指导，其中可能包括复杂公式，例如聚合物，脂质体和其他纳米颗粒构建体 c）提供有关主和工作单元以及病毒银行开发和测试的专家建议 d）提供团队提出的生物制度流程开发和制造计划的策略和反馈 e）提供有关团队提出的热寡核苷酸序列和合成过程的反馈 f）提供有关拟议的生物制剂公式的反馈 g）提供有关关键质量属性，发布测试等的反馈。 h）每周或每两周评估CMC数据 i）作为CMC专家，以确定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的工作人员或根据NIH员工要求，CRO和CMO签订了与NIH的合同。 k）检查CRO/CMO提出的方法的设施和讨论适当性 l）可以要求CMC顾问评估拟议的生物疗法的潜在障碍性（包括周围的IP空间） m）在研究支持治疗开发项目的研究研究设计方面合作 n）与其他顾问，承包商和NIH员工合作，咨询，战略计划和管理CMC，以促进临床前评估和安全测试，研究性新药（IND）提交和临床研究 o）通过电话和电子邮件就分配的CMC努力来促进LDT讨论 p）为BPN生物学，治愈PTDP和紧急生物制剂计划的持续发展和改进提供战略指导 绩效领域5 - 监管事务和监管运营中小企业的监管事务顾问将提供建议和领导地位，以支持NIH生物制剂计划的监管事务战略。将要求顾问提供有关辅助研究的指导，以确保团队符合IND应用程序的准则和时间表。顾问将与NIH员工，PI和监管机构担任主要监管联系，并可能是FDA的主要监管代表。 中小企业顾问的监管事务和监管运营的作用可能包括但不限于以下职责： a）在项目团队内部工作以制定监管策略，确定监管风险并及时批准 b）就与监管策略有关的问题向NIH员工和项目团队提供建议，并确定有关制定法规的关注领域 c）提供和审查规格，方法，SOP，协议，报告，标签等的输入。 d）开发和管理监管提交的时间表 e）有效地协调CRO和其他顾问在必要时准备监管提交的活动 f）准备并向适当的监管机构提交IND申请 g）准备并举办监管机构会议 性能区域8 - 质量保证（QA）中小企业（S） 质量保证中小企业顾问将在NIH生物制剂计划下提供支持产品开发和生物疗法临床评估的活动的质量监督。 质量保证中小企业将： a）草稿，审查和批准监管申请的相关部分。 b）关于最佳实践和流程的产品开发团队建议 c）确保CRO/CMO遵守法规和质量要求 d）负责进行质量检查 e）负责出发评估 f）支持制造CMO设施和研究的审核 g）支持对毒理学CRO设施和研究的审核 h）陪同NIH的工作人员或根据NIH员工要求，CRO和CMO签订了与NIH的合同。检查CRO/CMO提议的方法的设施和讨论适当性 i）进行其他现场审核并根据需要进行研究报告审核