D. Attachment 1 - Statement of Work.docx

STATEMENT OF WORK (SOW)

GENERAL INFORMATION

Title of Project:

Biological Therapeutics Product Development Subject Matter Expert Consulting Services to Support the Development of Cell and Gene Therapy Products

Background Information and Objective:

General

The National Institutes of Health (NIH) is the nation’s leading medical research agency and the primary Federal agency whose mission is to seek fundamental knowledge about the nature and behavior of living systems and apply that knowledge to enhance health, lengthen life, reduce illness and disability, and conduct, support, and make medical discoveries that improve people’s health and save lives. The National Institute of Neurological Disorders and Stroke (NINDS) is an institute within the NIH that conducts research into the causes, treatment, and prevention of neurological disorders and stroke. The NINDS mission is to seek fundamental knowledge about the brain and nervous system and use that knowledge to reduce the burden of neurological disease.

The NINDS has a need for biological therapeutics development subject matter expert (SME) consultants to provide technical recommendations and advice that will support the translational development of novel therapeutics (Biologics) for NIH drug discovery and development programs. The support included in this contract will cover all areas of cell and gene therapies, including chemistry, manufacturing, and controls, pharmacokinetics/pharmacodynamics, toxicology, medical writing, bioassay development, regulatory affairs, quality assurance/quality control, and biostatistics.

NIH Drug Development Programs Involving Biologics

This requirement supports the NIH Biologics development projects under the NIH Blueprint Neurotherapeutics Network Biologics (BPN-Biologics) program, the NINDS Ultra-Rare Gene Therapy (URGenT) program, the Helping to End Addiction Long-term Pain Therapeutics Development Program (HEAL-PTDP), and other extramural or intramural NIH drug discovery and development programs.

The NIH Blueprint Neurotherapeutics (BPN) network (https://neuroscienceblueprint.nih.gov/neurotherapeutics/blueprint-neurotherapeutics-bpn- network) is a consortium comprised of the NIH Office of the Director and 14 NIH Institutes and Centers (ICs) that support neuroscience research. BPN provides neuroscience researchers with funding and access to a full range of industry-style drug development services and expertise.

The program, originally focused on small molecules, was launched for projects requiring medicinal chemistry optimization, pharmacokinetics (PK), pharmacodynamics (PD), drug manufacturing and formulation, investigational new drug (IND)-enabling toxicological studies support through Phase I clinical testing and it is now being expanded to include the

Version 11-30-2016-1 FAR Part 11—Describing Agency Needs development of biological therapeutics for neurological diseases (BPN-Biologics). (https://neuroscienceblueprint.nih.gov/neurotherapeutics/bpn-biologics).

The URGenT program (https://www.ninds.nih.gov/Current-Research/Research-Funded- NINDS/Translational-Research/urgent-network) has been established by the NINDS to support the development and clinical evaluation of therapeutics to address ultra-rare genetically based neurological disorders. The network will provide neuroscience researchers with funding and access to a full range of industry-style biologics development services and expertise and in the future may expand to include other NIH institutes. The program is intended to rapidly advance patient-customized therapeutics through manufacturing, preclinical toxicology testing, and evaluation in Phase 1 clinical studies.

The HEAL-PTDP program (https://heal.nih.gov/) is a multi-IC initiative that has been established by the NIH to support preclinical optimization and development of safe, effective, and non- addictive biologic therapeutics to treat pain. The goal of the program is to accelerate the optimization and development of promising small molecule and biologic hits/leads to Phase I clinical trials and readiness for the Early Phase Pain Investigation Clinical Network (EPPIC-Net) or other Phase II clinical studies. Applicants must have a promising biologic hit/lead, robust biological rationale for the intended approach, and identified assays for optimization of the agent. The scope of this program includes optimization and early development activities, IND- enabling studies, development of a PD/ target engagement biomarker, assembly and filing of an IND application and Phase I clinical testing. This is a milestone-driven phased cooperative agreement program involving participation of NIH program staff in the development of the project plan and monitoring of research progress.

The NIH anticipates needing 1 consultant.Period of Performance:

The total period of performance (PoP) includes a one-year base period and one-year option year. The anticipated PoP dates are as follows:

Base Period: 12 month Period of Performance – Qty 20 hours Option Line 00001 – Qty NTE 380 hours Option Line 00002 – Travel NTE $2400.00

Option Year 1: 12 month Period of Performance – Qty 20 hours Option Line 00003 – Qty NTE 380 hours Option Line 00004 – Travel NTE $2400.00

Place of Performance:

The primary place of performance will be the Contractor site. Conference calls and email will be the primary mode of communication; however, the Contractor may be required to work at Government facilities or the facilities of selected principal investigators on an as needed basis.

SCOPE OF WORK

General Requirements:

Independently and not as an agent of the Government, the Contractor shall furnish all the necessary services, qualified personnel, material, equipment, and facilities not otherwise provided by the Government that are needed to support NIH biologics programs, including NIH BPN-Biologics, URGenT, and HEAL PTDP.

The Contractor shall provide this support on an ad hoc basis or while serving on NIH Biologics and HEAL Lead Development Teams (LDTs) and URGENT Multi-Disciplinary Project Teams (MPTs), which include both SME consultants and NIH staff. These teams will map out a product development and clinical evaluation research strategy, including milestones, and oversee manufacturing and nonclinical study services by Contract Manufacturing Organizations (CMOs) and Contract Research Organizations (CROs).

SME consultants will be expected to provide feedback and guidance on project milestones, development strategy, study design, and data interpretation through in-person meetings, conference calls, and email. Specific consulting responsibilities will depend on the expertise of the individual consultant and the needs of individual biologics projects and programs.

Consultants may be involved in multiple projects.

SMEs will be required on a part-time basis; hours will vary depending on the needs of the projects they are assigned to. The NIH estimates that the required level-of-effort will be 20-400 hours per year.

Specific Requirements:

The contractor shall provide consulting services in the following Performance Area.

Performance Area 1: Drug discovery and development for cell and gene therapies

Subject matter experts in drug discovery and development for cell and gene therapies focusing on preclinical development. Consultants will be expected to provide senior-level expertise in cell and gene therapies and contribute feedback and guidance on projects to the NIH and to LDT members through conference calls and by email.

1) Knowledge of drug discovery and development process.

a. Demonstrated senior-level oversight of research and development efforts for therapeutic products

b. Broad experience across the entire drug discovery and development process is especially desired, with a focus on cell and gene therapies.

c. Have managed a portfolio of comprehensive, milestone-driven pre-clinical drug development cell and gene-based therapeutics projects; planned, organized and directed inter-disciplinary drug research or development team efforts; developed policies, program directions, and procedures surrounding the administration of drug research programs.

d. Managed milestone-driven research and development projects in the area of cell and gene-based therapeutics, which require well-defined goals and objectives achieved with greatest efficiency. Developed and implemented quantitative and qualitative reviews of milestones to determine if they appropriately translated into accelerated progress of therapeutic candidates toward Investigational New Drug (IND) applications to the FDA, and provided clear indicators of a project's continued success or emergent difficulties.

e. Managed a portfolio of preclinical drug discovery and development projects (e.g., cooperative agreements) in the area of cell and gene-based therapeutics with responsibility for all aspects of administration (i.e., organizing and managing). Secured and implemented scientific advice from senior leaders and colleagues and applied drug discovery and development expertise.

2) Ability to analyze and evaluate milestones to manage a drug development program.

a. Developed project plans and associated communications documents. Managed the day-to-day activities of assigned Programs and Projects; directed and managed project development from beginning to end; and estimated the resources needed to achieve Program and Project goals. Managed key relationships internal and external to an organization.

b. Reviewed, analyzed, and evaluated pre-clinical drug development data

c. Provided an in-depth review, analysis, evaluation, and recommendation of milestones at the beginning of a funding year or funding cycle (depending on the Program or Project) to support a determination as to whether a project should be continued. This in-depth review included assessment and recommendations to ensure that individual projects were competitively evaluated against the broader research landscape.

3) Skill in applying Project Management tools to manage drug discovery and development programs.

a. Ensured that projects were managed and executed in line with the rules and governance of an organization and cognizant regulating bodies.

b. Prepared a variety of routine and special reports as requested by senior management. Researched and prepared reports, briefing materials, and other compilations of Program and Project activities and policies for use in policy development, program planning, evaluation and implementation, and presentation to senior advisors. Researched and wrote scientific review presentations, position papers, talking point papers, and briefing materials for senior staff as requested.

c. Has a track-record of successfully building and leading product development teams

d. Defined project objectives; planned, executed, and finalized projects according to strict deadlines and within budget; oversaw quality control, assembled data, and managed program budgets.

4) Ability to communicate clearly, logically and persuasively on complex scientific issues.

a. Has attended and presented drug research findings at conferences and meetings with high ranking scientists in academia, industry, or government.

b. Developed and delivered progress reports, proposals, requirements documentation, and presentations.

c. Has built, developed, and grown relationships vital to the success of a Program or Project to establish worldwide collaborative relationships among government, academia, and industry that are focused on the search for therapeutic interventions and treatments for neurological disorders.

d. Has experience in team leadership, consulting, or service on strategic and advisory boards .

e. A significant track-record of scientific accomplishment as evidenced by publications and/or contributions to the development of marketed products.

f. Must be able to work in a virtual, cross-functional team environment and manage competing priorities.

g. Must participate actively in project team discussions.

h. Must have strong interpersonal skills, attention to detail, excellent organizational, writing, communication, presentation, and documentation skills as well as computer and software literacy.

i. Must have experience working with multi-disciplinary teams at a senior level preferably in the biopharmaceutical industry, especially teams with members from outside organizations and members at various management levels.

Key Personnel:

The Contractor employee working under this award shall be considered key personnel. Key Personnel requirements are as follows:

General Requirements:

a) Demonstrated senior-level oversight of research and development efforts for cell and gene-based therapeutics products

b) A track-record of successfully building and leading product development teams

c) A significant track-record of scientific accomplishment as evidenced by publications and/or contributions to the development of marketed products

d) Experiences in team leadership, consulting, or service on strategic and advisory boards are desired

e) Must be able to work in a virtual, cross-functional team environment and manage competing priorities

f) Must participate actively in project team discussions

g) Must have strong interpersonal skills, an attention to detail, excellent organizational, writing, communication, presentation, and documentation skills as well as computer and software literacy

h) Broad experience across the entire cell and gene-based therapy discovery and development process is especially desired

i) Must have experience working with multi-disciplinary teams at a senior level preferably in the biopharmaceutical industry, especially teams with members from outside organizations and members at various management levels Education and Specific Work Experience Requirements:

a) A MS, PhD, or MD in a relevant scientific discipline is preferred. If a BS or MS, PhD-level experience in biopharmaceutical industry is required.

b) Must have at least 10 years of relevant professional experience

c) Must have at least 10 years demonstrated experience in biologics product development

d) Must have at least 10 years of experience in managing or being a senior leader in biologics development projects in the biopharmaceutical industry

e) SME consultants supporting the URGenT program must also have at least 5 years of experience in supporting rare and/or ultra-rare biologics product development programs

f) SME consultants supporting BPN-Biologics must have at least 10 years of experience across broad biological therapeutics product development programs, including cellular therapy, gene therapy and immunotherapy

g) Experience with biologics development for nervous system conditions is preferred but not required

h) Experience interacting with the FDA or other regulatory authorities preferred

i) Extensive understanding of FDA or other regulatory agency requirements for IND submissions and clinical trial execution (and when applicable for ultra-rare diseases) is preferred

The proposed Key Personnel will become subject to the provisions of Health and Human Services Acquisition Regulation (HHSAR) Clause HHSAR 352.237-75 Key Personnel as follows:

The key personnel specified in this contract are essential to work performance. At least 30 days prior to the contractor voluntarily diverting any of the specified individuals to other programs or contracts the Contractor shall notify the Contracting Officer and shall submit a justification for the diversion or replacement and a request to replace the individual. The request must identify the proposed replacement and provide an explanation of how the replacement's skills, experience, and credentials meet or exceed the requirements of the contract. If the employee of the contractor is terminated for cause or separates from the contractor voluntarily with less than thirty days’ notice, the Contractor shall provide the maximum notice practicable under the circumstances. The Contractor shall not divert, replace, or announce any such change to key personnel without the written consent of the Contracting Officer. The contract will be modified to add or delete key personnel as necessary to reflect the agreement of the parties.

Deliverables and Reporting Requirements:

SME consultants shall be responsible for providing subject matter expertise in the following areas:

1) Needs and/or gap assessment of ultra-rare gene-based therapeutic development processes

2) Advice on technical flow of various stages of the development process

3) Estimation of time, cost, and effort involved in each stage of the development process

Consultants shall also be responsible for submitting ad hoc summary reports of their analyses as needed.

OTHER CONSIDERATIONS

Government Responsibilities:

The Government does not anticipate providing Government-furnished property. Project related data (i.e., Government furnished information) will be provided to the Contractor for performance of work efforts. All data provided to the Contractor must be treated as confidential and shall not be disclosed to any third party.

Project data will be collected and stored by the NIH. NIH will provide data as needed to the Contractor each time it requests service. The Contractor may store data up to 12 months after initially receiving it, after which time the Contractor should either delete the data files and sanitize the computer using National Institute of Standards and Technology (NIST) SP 800-88 (current revision), or if information is still needed, contact the COR for permission to store the data for a longer period of time.

Travel:

The Contractor may be required to travel within the contiguous United States to provide the services described in this SOW. The Contractor shall make its own travel arrangements; the Government shall reimburse all reasonable travel costs up to the Government approved limits.

All travel arrangements must be made in accordance with Federal Travel Regulations in effect at the time of travel and must be approved by the Contracting Officer’s Representative and authorized by the Contracting Officer prior to being finalized or invoiced.

Information System Security Plan:

The Contractor must ensure that any computer system used in the course of this contract shall include the following features:

1. Any computer used in relation to this contract must be patched with most updated IT Security Patches.

2. Any Contractor computer used in relation this contract must have virus protection loaded and running with definition files that are updated on at least a daily basis.

Data Rights:

The NIH shall have unlimited rights to, and ownership of, all deliverables provided under this contract, including reports, analyses, recommendations and all other deliverables. This also includes any additional deliverables required by contract change. The definition of “unlimited rights” is contained in Federal Acquisition Regulation (FAR) 27.401, “Definitions.” FAR clause 52.227-14, “Rights General,” shall be incorporated by reference into any resulting award.

Project related data will be provided to the Contractor for performance of work efforts. All data provided to the Contractor must be treated as confidential and shall not be disclosed to any third party.

Section 508—Electronic and Information Technology Standards:

Section 508 of the Rehabilitation Act of 1973 requires that Federal agencies’ electronic and information technology (EIT) is accessible to people with disabilities. The Federal Acquisition Regulations (FAR) Final Rule for Section 508 (EIT Accessibility) can be found at www.section508.gov and at the Access Board’s Web site at https://www.access- board.gov/508.htm. The contractor must state that they will comply with the requirements of Section 508 or cite a justifiable reason for an exception.

Confidentiality of Information:

Consultant(s) shall guarantee strict confidentiality of the information/data that is provided by the NINDS or by any other participant on a project to which the consultant is assigned. The NINDS has determined that the information/data that the consultant will be provided during the performance of the consulting contract is of a sensitive nature.

Consultant(s) may only disclose the information/data generated for a particular project to other participants on that same project. These specific individuals will be identified when the consultant is assigned to a project.

Disclosure of the information/data, in whole or in part, by the consultant to anyone else can only be made after the consultant receives prior written approval from the Contracting Officer.

Whenever the consultant is uncertain with regard to the proper handling of information/data under the contract, the consultant shall obtain a written determination from the Contracting Officer.

Contractor agrees that it will not use the Confidential Information for any purpose except as set forth within this SOW, unless such time as such Confidential Information becomes public information.

Publications and Publicity:

Not applicable. The Contractor will not write or publish articles of data produced as a result of the SOW requirements.

Non-Personal Service Statement:

Contract employee performing services will be controlled, directed and supervised at all times by management personnel of the contractor. Actions of contractor employees may not be interpreted or implemented in any manner that results in any contractor employee creating or modifying Federal policy, obligating the appropriated funds of the U.S. Government, overseeing the work of Federal employees, providing direct personal services to any Federal employee or otherwise violating the prohibitions set forth in Parts 7.5 and 37.1 of the Federal Acquisition Regulations (FAR).