Cellular_Therapies_and_Banking_Stratgies_RFI.pdf

PROCUREMENT SENSITIVE

FOR OFFICIAL USE ONLY

REQUEST FOR INFORMATION

TITLE: Cellular Therapies and Related Cell Banking Strategies to Treat Acute Radiation Syndrome for Mass

Casualties

1. Background

The Office of the Assistant Secretary for Preparedness and Response (ASPR) leads the nation in preventing, preparing for, responding to, and recovering from the adverse health effects of emergencies and disasters by supporting our communities’ ability to withstand adversity, strengthening our health and response systems, and enhancing national health security.

The Biomedical Advanced Research and Development Authority (BARDA), within the Office of the Assistant Secretary for Preparedness and Response in the U.S. Department of Health and Human Services, provides an integrated, systematic approach to the development and procurement of the necessary vaccines, drugs, therapies, and diagnostic tools for public health medical emergencies.

BARDA supports the advanced research, development, and procurement of medical countermeasures (MCMs) against a variety of Chemical, Biological, Radiological and Nuclear (CBRN) threats and in recent years has significantly expanded its investment in therapeutics for radiation exposure. Current BARDA initiatives include the development of MCMs to mitigate systemic injuries caused by exposure to ionizing radiation.

Ionizing radiation exerts the greatest effect on rapidly dividing cells, such as those of the bone marrow and the gastrointestinal epithelium. Recent advances have led to the licensure of cytokines to mitigate the neutropenia associated with acute radiation syndrome. However, we anticipate that a portion of the patients who become neutropenic will be unresponsive to cytokine treatment, or cytokine therapy will not be an option, leaving these patients susceptible to infection and possible death. Alternative therapies, such as cellular therapies, could provide a possible treatment option to support the patient’s immune system until it has recovered from radiation exposure. Additionally, ionizing radiation damages endothelial cells, and damage to the vascular endothelium likely contributes to the development of systemic and organ specific injury in response to radiation. Cellular therapies may provide the structural, anti‐inflammatory and/or signaling components necessary to allow for healing and recovery of several sequelae throughout the body due to acute radiation syndrome.

Potential cellular therapies for radiation injury include, but are not limited to, embryonic stem cells (ESCs), mesenchymal stem cells (MSCs), progenitor cells, and induced pluripotent stem cells isolated from various tissues such as bone marrow, adipose tissue, placenta, amniotic fluid, cord blood and vascular endothelium. Products that improve homing, engraftment, safety, and/or efficacy of cellular therapies are also of interest.

2. Description

2.1 ASPR/BARDA is issuing this Request for Information (RFI) to identify the readiness of approved cellular therapies and product banking strategies for the treatment of acute radiation syndrome for mass casualties. Information is being requested in order to make potential future requirements better for industry and Government. BARDA seeks pertinent marketplace data on availabilities and capabilities to strengthen BARDA’s understanding of the current and future marketplace, to enhance its ability to obtain quality services and products economically, and to efficiently and lawfully establish potential vendor source files and listings.

2.2 THIS IS NOT A SOLICITATION FOR PROPOSALS. THIS IS A REQUEST FOR INFORMATION ONLY.

This RFI is for planning purposes only and is issued in accordance with FAR Clause 52.215‐3. No solicitation document exists at this time. The information provided in the RFI is subject to change and is not binding on the Government. Issuance of this notice does not constitute any obligation on the part of the Government to procure the supplies or to issue a solicitation. In addition, the Government is under no obligation to pay direct or indirect costs or charges that arise as a result of Responder submission of responses to this RFI or the Government's use of such information. Responses to this notice cannot be accepted as offers. The Government reserves the right to reject, in whole or in part, any Contractor's input resulting from this RFI. No contract will be awarded from this announcement. ASPR/BARDA has not made a commitment to contract any products or services, and release of this RFI should not be construed as a commitment or as authorization to incur cost for which reimbursement would be required or sought. All submissions become Government property and will not be returned. Any information that the vendor considers proprietary should be clearly marked as such. Information is being requested in order to make potential future requirements better for industry and the Government.

3. Description of the information requested

3.1 BARDA is seeking capability statements from qualified businesses (large or small) having the ability to provide cellular therapies for the treatment of acute radiation syndrome when patients are refractory to cytokines or when cytokine treatment is not an option. Banking strategies that allow for efficient use of products are also requested.

3.2 The narrative (including figures, schematics, diagrams, photographs, etc.) should describe (when applicable) the capabilities and include the following, if applicable:

a. Description of currently available cellular therapy or product, including the Technology Readiness Level of the proposed cellular therapy or products for the acute radiation syndrome indication. (See https://www.medicalcountermeasures.gov/federal‐initiatives/guidance/integrated‐trls.aspx),

b. The following should also be addressed:

1. Primary indication and market penetration

2. Non‐clinical studies supporting the ARS indication (24 hours or more post radiation)

3. Clinical studies

4. Product development plan

5. Regulatory strategy

6. Banking implementation plan

7. Section 351 of the Public Health Service Act Compliance, where appropriate

8. Volume and throughput of banking of cellular therapies, where appropriate

9. Previous engagements with the FDA

10. Manufacturing process and release testing

11. Cellular characterization (cell surface markers, immunogenicity, cytokine production, other phenotypic attributes, HLA)

12. Surge capacity: processing and product characterization

13. Strategy for allocation, distribution and shipping of banked product in the event of increased demand

14. Storage conditions and stability testing

15. Potential banking strategies including, but not limited to, the capability to maintain inventory https://www.medicalcountermeasures.gov/federal-initiatives/guidance/integrated-trls.aspx on behalf of the government and rotate it with the commercially distributed product to prevent expiration.

16. Business case to support the ability to maintain potential banking strategies

17. Where appropriate, current HLA repertoire, and cost associated with expanding HLA types banked to cover a greater percentage of populating

c. Respondents’ opinions regarding the difficulty and/or feasibility of the potential requirements or proposed acquisitions, possible solutions and approaches that may currently exist in the marketplace, and information regarding innovative ideas or concepts relative to this proposed RFI are encouraged.

d. All capability statements must provide the following:

1. Company name and address

2. Point of contact

3. Phone/fax/email

4. NAICS Code(s)

5. Business size and status

6. Capability information in response to the project requirements and qualifications identified in this notice

4. Key Assumption: Industry will be able to meet all requirements, as addressed.

5. Restrictions: Responses should be limited to 15 pages and may be sent electronically to Christopher.Scott@hhs.gov. Responses are due no later than July 10 by 5PM US EST.

6. Administrative Information:

a. Contracting Officer: The Contracting Officer is the only person authorized to accept responses to this RFI. All comments and/or questions shall be submitted to the Contracting Officer.

b. The Contracting Officer for administration:

Name: Christopher Scott Address: 200 C St SW, Washington DC 20515 Phone: (202) 205‐8580 Email: Christopher.Scott@hhs.gov mailto:Christopher.Scott@hhs.gov.

mailto:Christopher.Scott@hhs.gov