BIOLOGICS SUBJECT MATTER EXPERT (SME) CONSULTING SERVICES

生物制品主题专家 (SME) 咨询服务

• 批准号: 10788054
• 负责人: SUSAN BENJAMIN
• 金额: $ 22.08万
• 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 2023-04-14
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10788054
• 关键词: Address; Antibodies; Antisense Oligonucleotides; Area; Biochemistry; Biological; Biological Assay; Biological Process; Biological Products; Biological Response Modifier Therapy; Cell Therapy; Cells; Chemistry; Clinical; Clinical Research; Collaborations; Complex; Contractor; Contracts; Data; Data Analyses; Development; Development Plans; Electronic Mail; Evaluation Studies; Feedback; Formulation; Government; Guidelines; In Vitro; Investigational Drugs; Investigational New Drug Application; Label; Leadership; Lentivirus; Liposomes; Logistics; Methodology; Methods; Modality; Needs Assessment; Oligonucleotides; Peptides; Performance; Persons; Pharmacology; Pharmacology Study; Pharmacology and Toxicology; Polymers; Preparation; Principal Investigator; Process; Protocols documentation; Published Comment; Rare Diseases; Recombinant Proteins; Recommendation; Regulation; Regulatory Affairs; Reporting; Resources; Risk; Role; Running; Services; Small Interfering RNA; Specific qualifier value; Strategic Planning; Telephone; Testing; Therapeutic; Toxicology; United States National Institutes of Health; Viral; Viral Vector; Visit; Work; analytical method; design; evaluation/testing; improved; in silico; in vivo Model; lead optimization; manufacture; meetings; member; method development; model development; nanoparticle; nervous system disorder; operation; pharmacokinetics and pharmacodynamics; pre-clinical; preclinical evaluation; programs; protein purification; research and development; research clinical testing; safety study; safety testing; scale up; screening; symposium; therapeutic development; timeline

The consultants will be expected to provide feedback and guidance on scientific processes and gap areas/needs assessments through in-person meetings, conference calls, and email. The consultants will also be required to provide information on the ultra-rare disease space, preferably with a focus on neurological disorders. This will assist the NIH with better understanding the requirements for product developers to move their therapeutics through the research and development stage into clinical evaluation studies as well as development strategies and alternatives. Specific consulting responsibilities will depend on the expertise of the consultants and the needs of the program. 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 3: Toxicology SME(s) Toxicology SME consultants will be expected to provide senior-level expertise and contribute feedback and guidance on projects to the NIH and to LDT members through conference calls and by email. The role of the consultants may include but are not limited to the following responsibilities and tasks: a) Help establish toxicology endpoints and milestones for a project, recommend bioassays to be incorporated into a testing funnel or development plan, design toxicology and pharmacology studies, and interpret data delivered by CROs. b) In partnership with other consultants, contractors, and the NIH staff, advise, strategically plan, and manage programs to facilitate lead optimization, IND enabling, and clinical studies c) Serve as a resource to the LDT for toxicology requirements, pharmacology and logistics to conduct biologics development activities for biologics from the preclinical stage through to the initial clinical stage d) Serve as the toxicology expert in order to identify potential developmental challenges and suggest strategies to address these challenges e) Collaborate on design of investigative toxicology studies in support of biologics development f) Review toxicology sections of regulatory filings provided by CROs or PIs g) Collaborate on the design of investigative studies in support of biologics development. h) Provide expert support of preclinical strategy development including that for neurological diseases i) Support in vitro and in vivo model development for neurological disease j) Support design and interpretation of in silico analyses and in vitro/ ex vivo screening strategies for oligonucleotides k) Support of design PK/PD and toxicology safety studies 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

预计顾问将提供有关科学过程的反馈和指导 差距区域/需求通过面对面的会议，电话会议和电子邮件进行评估。 还将要求顾问提供有关超稀有疾病空间的信息， 优选地关注神经系统疾病。这将有助于NIH更好 了解产品开发人员将其治疗剂通过 研发阶段临床评估研究和发展 策略和替代方案。具体的咨询责任将取决于 顾问和计划的需求。 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 绩效区域3：毒理学中小企业 有毒理学中小企业顾问将有望通过电话会议和电子邮件向NIH提供高级专业知识，并向NIH和LDT成员提供有关项目的反馈和指导。顾问的作用可能包括但不限于以下职责和任务： a）帮助建立项目的毒理学终点和里程碑，建议将生物测定纳入测试渠道或开发计划，设计毒理学和药理学研究，并解释CROS提供的数据。 b）与其他顾问，承包商和NIH员工合作，咨询，战略计划和管理计划，以促进铅优化，IND启用和临床研究 c）作为LDT的毒理学需求，药理学和物流的资源 d）充当毒理学专家，以确定潜在的发展挑战并提出解决这些挑战的策略 e）在支持生物制剂开发的研究毒理学研究设计上合作 f）审查CRO或PIS提供的监管文件的毒理学部分 g）在支持生物制剂开发方面的调查研究设计上合作。 h）提供临床前策略开发的专家支持，包括神经系统疾病 i）支持神经疾病的体外和体内模型开发 j）支持硅分析和体外/ Ex Vivo中的设计和解释 寡核苷酸的筛选策略 k）设计PK/PD和毒理学安全研究 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