Request for Information Production Assistance for Cellular Therapies (PACT)

Notice Number: NOT-HL-12-165

Release Date: January 17, 2013
Response Date: February 22, 2013

National Heart, Lung, and Blood Institute (NHLBI)

Purpose

The National Heart, Lung, and Blood Institute (NHLBI) seeks comments regarding the continuance of the Production Assistance for Cellular Therapies (PACT) program. This Request for Information (RFI) is requesting comments to assist NHLBI staff in the assessment of the value of the program as well as how the program could be changed to meet the needs of investigators conducting research in the areas of heart, lung and blood diseases with an interest in cellular therapies.

The NHLBI PACT program was established to provide cell therapy investigators with translational and clinical services that are not deemed suitable for inclusion in a research project grant application. Examples of these services include the scale up of cell manufacture into quantities sufficient for a clinical trial, the establishment of a master cell bank for clinical production, the manufacture of clinical grade cells for Investigational New Drug (IND) enabling studies to satisfy FDA requirements, and the characterization of cell populations intended for clinical use. In addition, the PACT program supplies clinical grade products, produced in a manner that is compliant with applicable regulatory requirements, to early phase clinical trials. The current PACT program will end January 15, 2015, and the NHLBI would like feedback from the scientific community regarding the value of renewing the PACT program.

This RFI is soliciting feedback from the scientific and clinical research communities, other interested organizations, and the public on the renewal of the PACT program and the potential services that could be offered. Comments can include but are not limited to the following areas of concern:

1. Benefits to the scientific community from the continuation of a program such as PACT.

2. Provision of the following translational services is under consideration:

• Transfer and optimize cell manufacturing protocols from pre-clinical laboratory to GMP clinical production methods
• Produce GLP/GMP cell products for use in relevant animal safety studies
• Develop purity/potency assays and other release criteria for cell products
• Establish GMP-compliant cell production standard operating procedures (SOPs)
• Preparation for IND submission and assist in Chemistry, Manufacturing, and Controls (CMC) section development
• Preparation for a phase I trial

Comments can describe the usefulness of these services to investigators conducting research in the areas of heart, lung and blood diseases with an interest in cellular therapies. Comments can also include additional services that would be helpful.

3. Any ancillary services that a program such as PACT should provide, for example, testing scaffolds for cell therapy that has the goal of tissue regeneration and/or conducting and publishing the results of technical studies that could benefit the cell therapy community.

4. Resources (information and services) for HIV-related cell therapy needed for the scientific community.

5. Possibility of a catalogue of cell therapy products including publicly available data about cell source, manufacturing practices, potency assays, and clinical studies (domestic and international) as a useful resource for the scientific community.

Response to this RFI is voluntary. All interested parties are invited to respond. Any personal identifiers (e.g. names, addresses, E-mail addresses, etc.) will be removed when responses are compiled. Only the de-identified comments will be used. Proprietary, classified, confidential, or sensitive information should not be included in your response. The United States government reserves the right to use any non-proprietary technical information in any resultant solicitation(s).

This RFI is for information and planning purposes only and should not be construed as a solicitation or as an obligation on the part of the United States government to provide support for any ideas identified in response to it. Please note that the United States government will not pay for the preparation of any information submitted or for its use of that information. Responses will be compiled and shared internally with staff from the NHLBI, and with the National Heart, Lung, and Blood Advisory Council, with one or more subcommittees of the Council, and with scientific working groups convened by the NHLBI, as appropriate. In all cases where responses are shared, the names of the respondents will be withheld.

Interested extramural investigators and other interested parties are invited to respond.

Responses to the RFI will be accepted until February 22, 2013. You will not receive individualized feedback on any suggestions. No basis for claims against the United States government shall arise as a result of a response to this request for information or from the Unites States government’s use of such information.

All comments must be submitted via email as text or as an attached electronic document. Microsoft Word documents are preferred. Please submit your response to: [email protected].

Please direct all inquiries to:

Traci Heath Mondoro, Ph.D.
Deputy Branch Chief
Transfusion Medicine and Cellular Therapeutics Branch
Division of Blood Diseases and Resources
National Heart, Lung, and Blood Institute
Room 9140, MSC 7950
6701 Rockledge Drive
Bethesda, MD 20892
Telephone: 301-435-0065
FAX: 301-480-0867
Email: [email protected]