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...