The NHLBI Gene Therapy Resource Program
Social & Scientific Systems serves as the Clinical Coordinating Center for the National Heart, Lung, and Blood Institute's (NHLBI) Gene Therapy Resource Program. In this capacity, we provide planning, implementation, and management support to the NHLBI Gene Therapy Group in the actualization of the program. Our staff developed a Web-based system for investigators to register and submit applications for program resources, including the following:
- Gene therapy vectors for preclinical and clinical studies
- Pharmacology and toxicology testing
- Regulatory support for gene therapy trials
- Funding assistance for enrollment to clinical studies
Our staff coordinates the program activities of three vector laboratories and a pharmacology and toxicology laboratory for the Gene Therapy Group. We provide an array of regulatory support services to investigators and manage and disburse program funds allocated for approved requests for clinical trial funding assistance.
We coordinate and manage the activities of a peer review board established to assess the scientific merit of service applications. The database and Web site our staff developed provide the Coordinating Center with the following capabilities:
- Match studies (included as part of a request for program services) with the scientific expertise of board members
- Assign specific board members to assess a given study or request
- Monitor and track submission of a completed assessment form by each assigned board member
This customized peer review management system provides easy but controlled access to needed documents and forms for the reviewer and a high level of confidentiality and security regarding the assessments provided.
In addition, as the Coordinating Center, we use the Web site and database to manage assessments by two additional review groups—the Steering Committee and the NHLBI Gene Therapy Group—as well as reviews by the associated laboratory and the Clinical Coordinating Center. This complexprogram requires the coordination of numerous requesting investigators, five service areas, and five reviewing entities.
Our staff also provides coordination and management support for the program's Steering Committee; for program awareness efforts, including materials design and production; and for program policy and procedure development and implementation.