Attachment 1: Statement of Work

STATEMENT OF WORK (SOW) SERVICES

Senior Advisor Subject Matter Expert (SME) with Patient Advocay Expertise for the NINDS Ultra-rare Gene-based Therapy (URGenT) Network
External Oversight Committee (EOC) Subject Matter Expert (SME) Services

GENERAL INFORMATION

Title of Project:
Senior Advisor Subject Matter Expert (SME) for Ultra-rare Gene-based Therapy Network to act as External Oversight Committee (EOC) Subject Matter Expert (SME) with experience in Patient Advocacy.
Statement of Need, Purpose, and/or Objective:
The purpose of this requirement is to procure Senior Advisor 2 [subject matter expert (SME) services] in support of the National Institute of Neurological Disorders and Stroke (NINDS) Ultrarare Gene-based Therapy (URGenT) Network to accelerate development of drugs for rare diseases. The NINDS is seeking to procure a highly experienced executive-level, drug discovery and development Senior Advisor 2 with experience in the area of Patient Advocacy to provide feedback to the NINDS program leadership and technical guidance on drug discovery and development projects within the URGenT portfolio. The Contractor will serve as a member of the URGenT External Counselor Board (ECB), which provides strategic guidance on URGenT projects and on occasion other NINDS drug discovery and development programs. Background Information:
The URGenT Network supports the development of state-of-the-art gene-based therapies for ultrarare neurological diseases, which affect approximately one in fifty thousand people. Altogether, around 7,000 known rare and ultra-rare diseases affect 30 million people in the US. Many are lifethreatening and few have FDA-approved treatments. About 45% of rare diseases, including ultrarare diseases, are neurological disorders, and 90% of rare childhood disorders have major neurological effects. 85% of rare and ultra-rare diseases are single gene disorders, making them excellent candidates for gene-based therapy. The URGenT program provides funding and resources to advance gene-based therapies for ultra-rare neurological diseases from late-stage pre-clinical development into clinical testing. The goals of the URGenT program are to: 1.
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Accelerate advancement of discoveries into the clinic.
Provide resources and expertise not currently available to applicants. Deliver therapeutics to patients with ultra-rare neurological diseases. Standardize and harmonize best practices and protocols for the development of genebased therapies for ultra-rare diseases.