Cell therapy product administration and safety: data capture and analysis from the Production Assistance for Cellular Therapies (PACT) program

Cell therapy products have become more sophisticated both in type and manufacturing complexity (1-3). As clinicians' and researchers' understanding of disease and cell mechanisms of action continue to grow, cell therapy products are being increasingly used or considered for use in clinical trials (4-12). The safety and efficacy of cell therapy products have been reported within individual clinical trials and through review articles (13-19). These publications are often limited to an individual cell type and also differ by how the data are being collected and reported. The Health Resources and Services Administration (HRSA) has provided funds to study the use of cell therapy in the United States with a goal of building a database similar to that established and maintained by the bone marrow transplant community for years. The European Union has pursued a similar goal. Despite these efforts, there is limited published information that describes cell therapy product administration across both cell types and indications. To this end, The National Institutes of Health, National Heart, Lung and Blood Institute (NHLBI) Production Assistance for Cellular Therapies (PACT) program has collected data over the past 10 years on the administration of cell therapy products manufactured within the program. The PACT program was formed in 2003 through funding from the NHLBI. The program provides clinical cell therapy product manufacturing support to investigators wishing to transition a novel cell therapy from the developmental stage to clinical applications within the purview of the NHLBI. PACT is not responsible for directly monitoring the clinical trials of investigators receiving PACT-manufactured products. PACT has required collection of standardized information on product manufacturing, transport, receipt, administration, and adverse reactions with product administration. The purpose of this data collection is 1) to monitor administration of PACT cell therapy products, and 2) to build a product administration database to identify trends or safety concerns that may be associated with cell therapy product administrations. All clinical trials where PACT has provided manufacturing support were approved by local ethics committees.