NGVB

National Gene Vector Biorepository



POLICY AND PROCEDURE MANUAL

Version 4 - June 19, 2015


INDEX

Statement of Purpose 1.0
Eligibility Requirements for Receiving NGVB Resources 2.0
NGVB External and Internal Advisory Boards 3.0
Advertising of NGVB Resources 4.0
Application Process 5.0
Responsibility of the Investigator 6.0
Criteria for Termination of Support 7.0
NGVB Coordinating Center Responsibilities 8.0
Conflict of Interest 9.0
Authorship Issues 10.0
Commercial Affiliations 11.0
Confidentiality 12.0

1.0 STATEMENT OF PURPOSE

The National Gene Vector Biorepository and Coordinating Center (NGVB), funded by the National Heart, Lung and Blood Institute (NHLBI), National Institutes of Health (NIH), is charged with enhancing the research and clinical environment for gene therapy investigators. The NGVB will provide the following: (1) maintain a database of gene therapy pharmacology and toxicology studies (Pharm/Tox database) to support Investigational New Drug (IND) submissions to the Food and Drug Administration (FDA); (2) develop a reagent repository to support academic gene therapy investigators; (3) provide archiving services to academic investigators; (4) provide educational material about gene therapy; (5) provide testing services to assist investigators in meeting the FDA requirements for post-trial monitoring; and (6) conduct research related to gene therapy safety.

2.0 ELIGIBILITY REQUIREMENTS FOR RECEIVING NGVB RESOURCES

To be eligible to receive NGVB reagents and archiving services, an investigator must: (1) qualify as a not-for-profit organization, or government agency eligible to receive NIH funding and also have the capability to perform scientific studies; (2) submit a request for NGVB services following directions listed on the NGVB website, (3) agree to post-distribution monitoring as defined in Section 6.0, (4) follow the guidelines of the NIH, FDA, and the local Institutional Biosafety Committee (IBC) and Institutional Review Board (IRB), (5) agree to assurance and certification requirements as described in the Application for a Public Health Service Grant (PHS 398 Section III.G, http://grants.nih.gov/grants/funding/phs398/phs398.pdf), and (6) disclose other forms of grant support for all research projects using NGVB services or reagents.

The NGVB Pharm/Tox database and educational resources are provided on the NGVB website (www.NGVBCC.org) are open to all researchers, commercial entities, and other interested parties.

3.0 NGVB EXTERNAL AND INTERNAL ADVISORY BOARD

3.1 Function of the NGVB External Advisory Board

The External Advisory Board will meet annually to review NGVB activities. As needed, conference calls will be convened to discuss issues related to operations and overall objectives. The board will be composed of four or more experts in gene therapy. Members of the NIH, FDA and OBA will also be invited to participate as non-voting members of the board.

3.2 Function of the NGVB Internal Advisory Board

The NGVB Internal Advisory Board will be composed of at least three academic faculty from Indiana University with expertise in genetics, ethics, and/or clinical research. The board will receive bi-annual reports of NGVB activities and meet at least annually to discuss operations and objectives.

3.3 Appointments to the NGVB Advisory Boards

There are no term limits for the NGVB Internal Advisory Board. Individuals on the External Advisory Board will be appointed for 3 year terms. Terms are renewable.

4.0 ADVERTISING OF NGVB RESOURCES

The NGVB will be advertised at scientific meetings or through email notifications as a means of informing investigators of NGVB services. The information will also be available on the NGVB website at www.ngvbcc.org. When possible, additional advertisements will be included in NIH publications and through the World Wide Web.

5.0 APPLICATION PROCESS

5.1 Pharm/Tox Data

Pharm/Tox database information is open to everyone. Summary content is available through the NGVB website. Detailed information does require the investigator to register with the NGVB in order to facilitate the reporting of use statistics to the NIH.

5.2 Obtaining Reagent Repository, Testing, and Archive Services

Requests for reagents, testing, or archiving services require that the investigator complete an on-line request for services form as directed on the NGVB website. The investigator must: (1) provide sufficient information to establish that they meet the eligibility requirements defined in Section 2.0 above; (2) complete material transfer agreements (MTAs) associated with reagents; (3) when clinical samples are involved, attest that subjects have given consent to the storage of samples in an IRB approved informed consent document; and (4) complete the required NGVB forms prior to submitting material for the NGVB testing and archiving services. Once the required information is provided, the investigator will be contacted within 3 working days by the NGVB Coordinating Center to arrange sample submission or reagent shipping.

6.0 RESPONSIBILITIES OF THE INVESTIGATOR

6.1 Publication

Investigators will be required to acknowledge the NGVB in all publications, in the same manner that funding support is acknowledged.

6.2 Sharing Pharmacology/Toxicology Data

To receive NGVB support for archiving Pharm/Tox samples, the investigator must agree to submit a summary of the Pharm/Tox data to the NGVB Coordinating Center for inclusion in the NGVB pharmacology/toxicology database.

6.3 Post-distribution Monitoring

The NGVB will monitor use of NGVB services to allow reporting of activities to the NIH. When requested by the NGVB Coordinating Center, investigators agree to supply: (1) the name and number of federal or other grants that utilize NGVB services; (2) publications that acknowledge NGVB resources; and (3) the number of IND or other regulatory submissions that utilize the Pharm/Tox database.

6.4 Material Transfer Agreements

Investigators requesting reagents from the reagent repository will be required to complete a Material Transfer Agreement(s) (MTA) with the owner of the reagent and with Indiana University. Draft MTAs will be provided by the NGVB Coordinating Center, but it will be the responsibility of the investigator to return the completed agreements before a reagent can be shipped.

6.5 Regulatory Compliance

NGVB users must attest that the material will be used in compliance with current regulatory guidelines and approved by the appropriate Boards prior to use (ex. Institutional Review Board, Institutional Biosafety Committee, US Food and Drug Administration, or other applicable agencies).

6.6 Return or Destruction of Samples

All archived samples, reagents, or testing material held within the NGVB remain the property of the submitting investigator. Investigators agree to accept the return of materials if the NGVB program is discontinued by the NIH.

Samples will not be destroyed by the NGVB without written permission of the investigators. Investigators agree to permit the destruction of, or to accept the return of, material that is degraded, expired, or no longer of scientific or clinical value.

Investigators requesting the destruction of material held in the NGVB must provide evidence that the appropriate regulatory requirements for storage or testing have been fulfilled.

7.0 CRITERIA FOR TERMINATION OF SUPPORT

7.1 Non-compliance with NGVB Policies and Procedures

Failure to comply with the guidelines set forth in the NGVB Policies and Procedures will serve as grounds for suspension of NGVB support. The NGVB Coordinating Center Director will submit concerns regarding non-compliance to the investigator. Failure to reply satisfactorily within four weeks will result in suspension of NGVB support. The NGVB External Advisory Board will discuss all cases of non-compliance. NGVB support for an investigator can be terminated by the majority vote of the NGVB External Advisory Board.

7.2 Non-compliance with Post-Distribution Monitoring

Failure to submit required documents (as outlined in Section 6.3 above) will result in a warning letter from the Coordinating Center requesting the needed information. Failure to submit the required documentation within three weeks will result in suspension of NGVB support until the requested documentation is received. The suspended study may not use any NGVB services until the suspension is favorably resolved.

7.3 Termination

Three suspensions for any of the above-mentioned reasons will result in the termination of NGVB support.

8.0 NGVB COORDINATING CENTER RESPONSIBILITIES

8.1 NGVB Advisory Board Meetings

The NGVB Coordinating Center will be responsible for organizing the NGVB Advisory Board Meetings. The NGVB Coordinating Center will be responsible for arranging the meeting location and for distributing the agenda and review materials at least two weeks prior to the meeting. The NGVB Coordinating Center will maintain written minutes of both External and Internal Advisory Board meetings.

Non-Federal external advisors invited to such meetings will be reimbursed for travel expenses and per diem through the NGVB Coordinating Center.

8.2 Review of Requests for NGVB Services

The Coordinating Center will review request forms for NGVB services to insure the responses are consistent with the eligibility requirements stated in Section 2.0 above.

For repository services that require Material Transfer Agreements, the NGVB Coordinating Center will provide draft MTAs from the donor institutes, these will be sent together with the NGVB MTA to the requesting investigator. Upon completion by the investigator and their institution, the MTAs are returned to the Coordinating Center who obtain final signatures from the owner/institutions and the NGVB. Once all of the MTAs are fully executed the reagents are shipped out to the investigators. The NGVB Coordinating Center will ensure the MTA is completed by the owner and requesting investigator prior to shipping the reagent.

8.3 Maintenance of Pharmacology/Toxicology Database

The NGVB Coordinating Center will maintain a pharmacology/toxicology database in a manner that is accessible through the NGVB home page. The database will provide information on studies already completed, and will provide examples for investigators seeking to design toxicology studies. When appropriate, the NGVB will coordinate letters of cross-reference so that the FDA may use previously submitted studies in support of new Investigational New Drug applications.

8.4 Facilities and Sample Protection

The NGVB will maintain appropriate infrastructure and facilities to meet the regulatory requirements associated with reagents and samples stored in the NGVB. Storage of materials requiring Good Manufacturing Practices or Good Laboratory Practices will be performed under Standard Operating Procedures with oversight by a Quality Assurance Officer that does not report directly to NGVB personnel. Security of storage facilities will be maintained by restricted cardkey access controlled by NGVB staff, unauthorized individuals entering the facility must be accompanied by trained personnel at all times. Freezers and other essential equipment will be monitored visually on a daily basis and continuously by an electronic Building Management System. A 24/7 call system will be in place and all critical equipment will be under service contract with preventative maintenance programs. Back-up facilities for storage are available on-campus and at off-site locations. Clinical samples are maintained in the Indiana University Sample Storage Facility and managed under Standard Operating Procedures available for investigator review (www.indianaCTSI.org/ictsissf/sops).

8.5 Data Protection

The NGVB will maintain Laboratory Management customized for the fairly diverse programs of the NGVB. Computer security is maintained in strict compliance with HIPAA guidelines and IU's extensive security policies (protect.iu.edu/cybersecurity/policies). The NGVB's dedicated servers are kept in facilities that are accessible only to appropriate system administrators and IT Managers via a card-key access system. Data is backed up to the Massive Data Storage System (MDSS), a rare, if not unique, academic facility that simultaneously mirrors data in Indianapolis and Bloomington, sites 50 miles apart. Hardware and software maintenance contracts provide prompt, expert advice and replacement hardware, as needed. Servers are kept in climate-controlled facilities that are monitored on a 24/7 basis by a professional Operations staff.

Investigators may review the status of their samples by contacting the NGVB Coordinating Center, who will provide the requested information within three working days.

8.6 Release, Distribution or Destruction of Materials

The NGVB and Indiana University serve solely as a storage facility for the materials and claim no rights or ownership of the material. All materials held within the NGVB remain the property of the submitting investigator (owner). Materials will not be released to other investigators or used by the NGVB staff for research purposes, without written permission of the owner. Return or distribution of any reagents will require MTA agreements as described above. Return, distribution or destruction of archived or testing material requires written directions as to the deposition of the material by the owner.

9.0 CONFLICT RESOLUTION

When NGVB users raise concerns regarding the function of the NGVB, the complaint will be forwarded to the NGVB External Advisory Committee for conflict resolution. In conflict resolution, efforts will be made to avoid conflict of interest or the appearance of conflict of interest by the NGVB External Advisory Board. Members must recuse themselves from discussion on any issue (1) involving faculty from the member's institution; (2) is submitted by an investigator with an ongoing or recent collaboration; or (3) if the NGVB Steering Committee member has a financial or fiduciary interest in the institution or issue under discussion.

10.0 AUTHORSHIP ISSUES

The NGVB is a NIH-sponsored service facility. Indiana University faculty and staff will not expect authorship on investigator publications that uses NGVB material if the contribution was limited to the goals stated in Section 1.0 above.

Investigators will be required to acknowledge the NGVB in all publications, in the same manner that funding support is acknowledged.

11.0 COMMERCIAL AFFILIATIONS

It is anticipated that in some instances investigator-initiated research will be conducted in concert with commercial entities. Acknowledging these possibilities, investigators must insure that the commercial entity will not restrict the investigator from publishing data derived from the NGVB-supported project for more than 60 days after being notified of their intent to publish, or in any other way restrict the unqualified publication of results.

12.0 CONFIDENTIALITY

All information submitted to the NGVB related to requests for services and subsequent correspondence is to be held in confidence by the NGVB personnel and the NGVB Internal and External Advisory Boards.