• The GTRP Clinical Coordinating Center.
• The GTRP Core Laboratory from whom services are requested (if applicable).
• Selected members of the GTRP Scientific Review Board (if RSA is other than preclinical or regulatory).
• The GTRP Steering Committee (if RSA is other than preclinical or regulatory).
• The NHLBI Gene Therapy Group.

• The Clinical Coordinating Center reviews all RSA submissions for:
  
  
  • Completeness of the RSA form
  • Regulatory status of any associated clinical protocol
  • Submission of ancillary documents such as the Conflict of Interest Statement and, if applicable, the Investigator and Institution Certifications and Agreement signed document

• The Clinical Coordinating Center reviews RSAs for Regulatory services for clarity, completeness, and feasibility, including required timelines, of providing the service .

• For Core Laboratory services the Laboratory reviews for clarity, completeness, and feasibility of providing the service.  Their feasibility assessment includes a cost analysis for providing the service and the timelines required.

• Select GTRP Scientific Review Board members review RSAs for full GMP-grade vector production and clinical trial funding assistance; RSAs for pharmacology/toxicology testing are reviewed on a case-by-case basis determined by the NHLBI Gene Therapy Group. The Scientific Review Board assesses:
  
  
  • The soundness of the supporting scientific data
  • The design of the proposed study
  • The overall scientific merit of the study

• The GTRP Steering Committee reviews the same types of RSAs reviewed by the Scientific Review Board.  The Steering Committee reviews the RSA package including:
  
  
  • RSA form and documents submitted by the investigator
  • The Feasibility Assessment provided by the Core Laboratory or CCC
  • Assessments provided by the Scientific Review Board members (for noted RSA types only)
  • Both scientific and cost aspects of the RSA.

The Steering Committee makes recommendations to the Gene Therapy Group based on the RSA’s scientific merit, compatibility with the goals of the Program, and cost merit in terms of the overall Program scope and budget.

• The NHLBI Gene Therapy Group reviews RSAs for preclinical vector production and regulatory support considering, respectively, the Preclinical Core Laboratory’s or Clinical Coordinating Center’s Feasibility Assessment in conjunction with the investigator’s RSA submission.  For other RSA types the Gene Therapy Group additionally reviews the recommendations of the Scientific Review Board and the Steering Committee.  Final disposition of all RSAs is made by the NHLBI Gene Therapy Group.

• The Clinical Coordinating Center and the Core Laboratories review RSAs on an ongoing basis.
• Scientific Review Board members are selected for each RSA requiring SRB review after the Feasibility Form has been completed by the Core Laboratory (if Laboratory services were requested).  SRB reviews are ongoing and may require as much as two week’s time following assignment of a reviewer.
• The Steering Committee conducts reviews at face-to-face meetings held twice annually.
• The Gene Therapy Group conducts reviews at Program meetings held approximately every two weeks.

Applicant investigators have access, via the GTRP website and a secure password, to a summary page listing each RSA he/she has submitted.  A status is displayed with each RSA.  The RSA status may include such designations as:

• Preliminary RSA Submitted
• Final RSA Submitted
• Feasibility Assessment Submitted
• Scientific Review Board Review Scheduled
• Scientific Review Board Review Completed on February 28, 2008
• Steering Committee Review Scheduled for April 15, 2008
• Gene Therapy Group Review scheduled for December 15, 2007

These are examples of some of the RSA status categories.  Only the current (most recent) status applicable to the RSA is displayed on the Investigator’s RSA summary page. 

An applicant investigator should contact the Clinical Coordinating Center with questions concerning the review of their RSA submission (all types of RSAs) or with other questions regarding the submission and review process. CCCemail

For questions regarding the completion of the RSA form, the investigator should contact the individual laboratory from whom they are requesting service.  To do this click on the laboratory of interest from the GTRP Main Home Page.  Laboratory contact information is available on the individual Laboratory page.   For questions regarding the completion of Regulatory RSAs and Clinical Trial Funding Assistance RSAs, the investigator should contact the Clinical Coordinating Center. CCCemail