CureSCI_Sought_Notice_Warrior_1.pdf

Sources Sought Notice

Whole-person Assessment of Response and Risk after transformative therapies:

Investigating Outcomes and Resilience in Sickle Cell Disease (WARRIOR-SCD)

Introduction

This Sources Sought Notice is for interested small businesses. This notice is NOT a request for proposals and does not commit the Government to award a contract now or in the future. No solicitation is available at this time. The purpose of this notice is to obtain information regarding: (1) the availability and capability of qualified small business sources; (2) whether small businesses are classified as HUBZone; service-disabled, veteran-owned; 8(a); veteran- owned; woman-owned; or small disadvantaged; and (3) their size classification relative to the North American Industry Classification System (NAICS) code for the proposed acquisition. Your responses to the information requested will assist the Government in determining the appropriate acquisition method, including whether a set-aside is possible. The NAICS Code for this acquisition is 541715 – Research and Development in the Physical, Engineering, and Life Sciences (except Nanotechnology and Biotechnology). Small business organizations must have their size status certified by the Small Business Administration. An organization that is not considered a small business under NAICS code 541715 should not submit a response to this notice.

Background

The National Heart, Lung, and Blood Institute (NHLBI) requires contractor support to continue building a cohort of individuals with sickle cell disease (SCD) who have received gene therapy (GT) or hematopoietic cell transplantation (HCT) and to sustain long-term follow-up (LTFU) activities for enrolled participants. While both therapies are available, it has not been established which is safer, more durable, or most effective, nor have rigorous comparisons to untreated persons with SCD who have chosen not to undergo one of these treatments undertaken.

While GT can ameliorate a major complication of SCD, veno-occlusive events, the treatment is considered transformative rather than curative since the beta-globin defect persists in unmodified cells and both modified and unmodified stem cells coexist in the recipient. It is unknown if organ toxicity has been halted or the functions improved. Whether the benefits of GT are durable is unclear: the longest available follow-up spans only about 8 years, involves few patients, and partly reflects manufacturing processes no longer representative of current practice. Allogeneic HCT replaces the recipient's blood-forming cells with a donor's, correcting sickling but risking serious immunologic complications such as graft-versus-host disease. Both approaches use chemotherapy, and the true risk profiles are not well characterized. Both therapies carry a documented risk of myeloid malignancy, with a 10-year incidence of leukemia or MDS of 1.7% across more than 1,000 SCD transplants. Because this risk accrues and can manifest late, short-term safety data are insufficient to characterize either intervention's true risk.

The Food and Drug Administration (FDA) requires 15 years of follow-up for GT recipients, but no infrastructure existed to systematically follow outcomes over that horizon, or to compare them systematically to non-curative standard-of-care management. To address this gap, NHLBI established two patient cohorts within its CureSCi. This is to continue the WARRIOR-SCD (Whole-person Assessment of Response and Risk after transformative therapies: Investigating Outcomes and Resilience in Sickle Cell Disease; (https://cibmtr.org/CIBMTR/Collaborations/WARRIOR-SCD), a 15-year effort to collect long-term clinical outcomes, patient-reported outcomes (PROs), and related operational data from treatment centers across the US, along with biospecimen collection and genetic testing for eligible participants. The second NHLBI-funded effort, the NHDR (Natural History Data Resource; (https://curesicklecell-nhdr.rti.org/ )), is building a comparator cohort of SCD patients who have not received GT or HCT. WARRIOR-SCD and NHDR are both part of NHLBI's CureSCi LTFU Consortium.

WARRIOR-SCD focuses on outcomes of importance to people living with SCD, providers, researchers, and the FDA: acute and chronic pain; end-organ function (brain, lung, heart, kidney, endocrine); other SCD-specific complications (e.g., retinopathy, priapism); complications of conditioning and the GT/HCT procedures (e.g., graft-versus-host disease, graft persistence or function); development of malignancy, including MDS, leukemia, and solid tumors; reproductive health; quality of life; and mortality. The study leverages the infrastructure of the Center for International Blood and Marrow Research (CIBMTR), an existing NIH-supported data system for HCT, cell therapy, and GT.

The research plan was developed by experts across the relevant medical domains together with individuals living with SCD, treated and untreated. WARRIOR-SCD is designed to enroll approximately 700 participants over the first 5 years, following each annually for at least 5 years post-treatment, with follow-up potentially extending to 15 years or longer subject to funding and programmatic direction.

Comprehensive longitudinal clinical data, PROs, and biospecimens (under separate IRB-approved protocols) will be collected throughout. Participant retention, data quality monitoring, and provider/participant engagement are key priorities. Eligible patients are consented and enrolled prior to receiving therapy and asked to participate in the PRO study and specimen collection; consent allows hospitals to submit clinical data and outcomes on the participant's behalf. All clinical visits are standard of care. The goal is to assess long-term safety and efficacy of GT and HCT in individuals with SCD.

THIS NOTICE ADDRESSES THE Whole-person Assessment of Response and Risk after transformative therapies: Investigating Outcomes and Resilience in Sickle Cell Disease (WARRIOR-SCD) ONLY.

It is anticipated that one (1) award may be made on or about April 2028 for a period of ten (10) years.

Objectives

1. Build a cohort of approximately 700 patients treated with GT and HCT, by enrolling new patients and continuing annual longitudinal follow-up of enrolled participants.

2. Test whether treated participants have preserved organ function relative to baseline and mortality/quality-of-life outcomes comparable to the general US population.

3. Test whether treated participants have preserved organ function and superior mortality/quality-of-life outcomes compared to individuals with SCD who do not receive GT and HCT.

4. Continue to build data and specimen resources to support FDA long-term follow-up reporting requirements for gene therapy and future biomedical research.

https://cibmtr.org/CIBMTR/Collaborations/WARRIOR-SCD https://curesicklecell-nhdr.rti.org/

5. Engage WARRIOR-SCD communities (participants and site research teams) to support retention and health education.

6. Support independently funded, investigator-initiated ancillary studies using study data or specimens.

WARRIOR-SCD is one component of the CureSCi LTFU Consortium (a separate contract supports other components) and is expected to collaborate with consortium members, NHLBI, and the contractor capturing data from the NHDR (individuals with SCD receiving treatments other than GT/HCT).

Project Requirements

The overall purpose is to continue to implement, manage, and support a multi-center program for individuals with SCD who have undergone GT or HCT, as described at https://cibmtr.org/CIBMTR/Collaborations/WARRIOR-SCD#objectives. The Contractor will be responsible for study design, procedures, data management, statistical analysis, participant engagement, biospecimen coordination, support for testing, and close-out — and to disseminate findings and provide sharable datasets/biospecimens to the external research community. The Contractor has latitude to determine specific processes, tools, and staffing approaches to meet performance standards, subject to Government-approved plans and applicable federal, NIH, and NHLBI requirements.

The important overall technical requirements associated with this proposed acquisition for the WARRIOR-SCD include the following:

The Contractor shall perform all work in a timely, professional, and scientifically sound manner.

The Contractor shall be capable of:

A. General Performance Requirements

1. Maintain accurate, complete, and current project documentation.

2. Coordinate effectively with NHLBI staff, participating sites, repositories, committees, and authorized subcontractors.

3. Provide qualified staff sufficient to perform all contract requirements without interruption, including but not limited to clinical expertise in SCD, expertise required for collection of GT and HCT outcomes data, PROs, and biospecimens collection tracking, as well as the bioinformatics and technological infrastructure needed to coordinate and efficiently conduct the WARRIOR-SCD study.

4. Maintain continuity of operations throughout the period of performance.

5. Notify the Contracting Officer’s Representative (COR) promptly of actual or anticipated issues affecting cost, schedule, technical performance, data integrity, or operational continuity.

6. Comply with all applicable Federal, NIH, NHLBI, human subjects, privacy, security, biospecimen, records management, and data-sharing requirements.

B. Support WARRIOR-SCD Study Core Operations that include 1.Program management and coordination.

2. Site recruitments, onboarding and support

3. Participant accrual, registration and tracking

4. Clinical data collection and reporting

5. Patient-reported outcomes operations

6. Participant retention https://cibmtr.org/CIBMTR/Collaborations/WARRIOR-SCD#objectives

7. Biospecimen collection and coordination

8. Data quality, monitoring and reporting

C. Ancillary studies. The Contractor shall support independently funded ancillary studies by providing Contractor-collected data, in accordance with established procedures and timelines, for NHLBI-approved investigators.

D. Privacy and Data Handling The Contractor shall ensure that all data handling complies with applicable Federal requirements, approved protocols, and NHLBI direction.

E. Transition and Continuity The Contractor shall support orderly transition of activities to a successor contract or contractor, if required by NHLBI. The Contractor shall minimize disruption to ongoing CureSCi LTFU Consortium operations and WARRIOR-SCD study activities.

Anticipated Period of Performance

The Government intends to negotiate one (1) contract award, for the potential period of ten (10) years, with an approximate award date on or around April 2028.

Capability Statements – How to submit a response

Small business concerns that believe they possess the capabilities necessary to undertake this work should submit complete documentation of their capabilities to the Contracting Officer. The capability statements must specifically address each project requirement separately. Additionally, the Capability Statement should include: 1) the total number of employees; 2) the professional qualifications of scientists, medical experts, and technical personnel as it relates to the above outlined requirements; 3) a description of general and specific facilities and equipment available, including computer equipment and software; 4) an outline of previous research projects that are similar to the project requirements in which the organization and proposed personnel have participated; and 5) any other information considered relevant to this program. The Capability Statement must not exceed 15 single-sided or 7.5 double-sided pages in length and use a 12-point font size minimum.

Interested small business organizations are required to identify their size standards in accordance with the Small Business Administration. The Government requests that no proprietary or confidential business data be submitted in response to this notice. However, responses that indicate the information therein is proprietary will be properly safeguarded for Government use only. Capability Statements must include the name and telephone numbers of a point of contact having authority and knowledge to discuss responses with Government representatives. Capability Statements in response to this market survey that do not provide sufficient information for evaluation will be considered non-responsive. When submitting this information, please reference the Sources Sought Notice number 75N98026R00146.

Point of Contact

Elizabeth Bulger, Contracting Officer, Research and Development (R&D) NHLBI Branch, R&D and Professional Services – Division B, Office of Acquisition and Logistics Management (OALM), Office of the Director, NIH; Phone: (301) 435-3498; E-mail: elizabeth.bulger@nih.gov

Department of Health and Human Services, National Institutes of Health, Office of Acquisition and Logistics Management (OALM), Research and Development and Professional Services – Division B, 6701 Rockledge Drive, RKL2, Bethesda, MD 20817 UNITED STATES.

Information Submission Instructions

Interested parties shall submit capability statements via electronic mail and must reference the Sources Sought Notice number 75N98026R00146 in the subject line of the e-mail submission.

The due date for receipt of capability statements is 3:00 p.m. Eastern Standard Time (EST) on October 2, 2026. All responses must be received by the specified due date and time in order to be considered.

Capability statements shall be submitted electronically to the designated Contracting Officer and Contract Specialist as follows:

Elizabeth Bulger, Contracting Officer: elizabeth.bulger@nih.gov Jessica Williams, Contract Specialist: jessica.williams3@nih.gov

This notice does not obligate the Government to award a contract(s) or otherwise pay for the information provided in the response. No proprietary, classified, confidential, or sensitive information should be included in your response. The Government reserves the right to use information provided by respondents for any purpose deemed necessary and legally appropriate. Any organization responding to this notice should ensure that its response is complete and sufficiently detailed to allow the Government to determine the organization’s qualifications to perform the work. Respondents are advised that the Government is under no obligation to acknowledge receipt of the information received or provide feedback to respondents with respect to any information submitted. After review of the responses received, pre-solicitation and solicitation notices may be published in SAM.gov. However, responses to this notice will not be considered adequate responses to a solicitation/Request for Proposal (RFP).

mailto:elizabeth.bulger@nih.gov mailto:elizabeth.bulger@nih.gov mailto:jessica.williams3@nih.gov