# A Data Resource for Blood and Marrow Transplants and Adoptive Cellular Therapy Research

> **NIH NIH U24** · MEDICAL COLLEGE OF WISCONSIN · 2024 · $756,884

## Abstract

PROJECT SUMMARY / ABSRACT
CIBMTR (Center for International Blood and Marrow Transplant Research) represents an international
network of >360 treatment centers and maintains a clinical outcomes database with data from >675,000
hematopoietic cell transplant and adoptive cellular therapy patients, providing a unique resource of data for use
in a variety of communities. U24-CA76518 provides the primary support for development and mainte-
nance of the database and its statistical support. The Centers for Medicare & Medicaid Services (CMS)
Cell and Gene Therapy (CGT) Access Model aims to improve the lives of people with Medicaid insurance living
with rare and severe diseases, such as sickle cell disease (SCD), by increasing access to the transformative
gene therapy treatments. The aim of this supplement is to expand existing CIBMTR infrastructure and core
capabilities to support goals of the CGT Access Model. The new initiative has three new objectives: (1) To
expand CIBMTR’s existing data infrastructure, including completeness and validation, methods to
prospectively link disparate data sources, and protected storage requirements; (2) To develop a CMS CGT
Access Model Plan to describe project-specific extracts supporting statistical analytics along with a validated,
repeatable, and protected data exchange process; and (3) To establish standard CGT Access Model patient-
reported outcomes (PRO) measures and expand PRO electronic data collection and follow-up techniques in
the SCD population. To accomplish these objectives, CIBMTR’s data collection systems will require
expansion with minor modifications in the clinical data systems and more significant modifications to the
ePRO system to accommodate SCD pediatric patient PRO data collection. In a role of honest broker, CIBMTR
will create a separate, secure environment for ongoing linking of disparate data sources and a process to
create a Unique Study ID. CIBMTR will develop a robust plan for implementing the model that will document
project-specific requirements and describe patient inclusion, variable selection, statistical analytics, patient
level linking, dataset delivery, and other design considerations, including protocol development and additional
consent if necessary. Finally, CIBMTR will tailor its standard PRO measures and data collection system to
collect important information about the SCD patient population and will develop new processes to include
innovative techniques for long-term follow-up and patient engagement. The requested supplemental funds will
support the above efforts as well as additional forms reimbursement funding for centers, a center participation
incentive, and PRO survey reimbursement for participants. While the requested center and participant
reimbursement does not fully cover the true cost of data submission, it can be an important influence of
consistent and reliable engagement. The end product of this initiative will be ongoing collection and sharing
of reliable and v...

## Key facts

- **NIH application ID:** 11160283
- **Project number:** 3U24CA076518-27S1
- **Recipient organization:** MEDICAL COLLEGE OF WISCONSIN
- **Principal Investigator:** Marcelo C. Pasquini
- **Activity code:** U24 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $756,884
- **Award type:** 3
- **Project period:** 2024-09-01 → 2028-02-29

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11160283

## Citation

> US National Institutes of Health, RePORTER application 11160283, A Data Resource for Blood and Marrow Transplants and Adoptive Cellular Therapy Research (3U24CA076518-27S1). Retrieved via AI Analytics 2026-06-08 from https://api.ai-analytics.org/grant/nih/11160283. Licensed CC0.

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