Details
Congenital cytomegalovirus infection (cCMV), a rare disease that affects newborns, is the leading non-genetic cause of hearing loss and the most common cause of intellectual disability caused by a viral infection. There are no FDA-approved products to treat or prevent cCMV. The American Academy of Pediatrics recommends six months of treatment with valganciclovir (vGCV) for newborns with moderate to severe cCMV to improve hearing and neurodevelopment outcomes, but the extent of uptake of this recommendation is unknown. Multiple factors may influence providers’ decisions regarding who to treat and for how long, but this information has not been systematically collected.
The Division of Antivirals (DAV)/the Office of New Drugs (OND)/Center for Drug Evaluation and Research (CDER), in partnership with the Office of Pediatric Therapeutics (OPT)/ Office of the Commissioner (OC), is conducting a regulatory science project to improve our understanding of the current landscape for diagnosis, treatment, and outcomes of infants with congenital CMV (cCMV) in the United States. The aim of the project is to explore how Real World Data can be used to augment our understanding of cCMV in the United States. This knowledge will help FDA establish a framework for cCMV drug development.
The Sentinel Distributed Database (SDD) was selected as the data source for this study because it enables the project team to access data from a large population and remove bias related to site selection. The study is being conducted in two parts. Part 1 is a claims-based analysis in the SDD designed to evaluate trends in cCMV diagnosis over time, trends in the use of valganciclovir/ganciclovir [(v)GCV] for the treatment of cCMV, and the correlation between baseline disease manifestations and the decision to treat with (v)GCV. Data collection is complete and analysis is ongoing.
In Part 2, we performed a retrospective chart review using electronic health records (EHR) from Sentinel Data Partners who have Integrated Delivery Systems. This will enable the study team to address more detailed questions that can only be answered though review of patient-level data, including factors affecting the diagnosis and management of cCMV in the United States, as well as the impact of antiviral treatment on clinical outcomes.
This project is funded by the Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration (FDA).
Additional Information
Contributors
Prabha Viswanathan, MD; Office of Pediatric Therapeutics (OPT), Office of Clinical Policy and Programs (OCPP), Office of the Commissioner, Food and Drug Administration, Silver Spring, MD
Aimee Hodowanec, MD; Takashi Komatsu, PhD; Natalie Pica, MD, PhD; Division of Antivirals, Office of Infectious Disease, Office of New Drugs, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD
Jency Daniel, MD; Division of Anti-Infectives, Office of Infectious Diseases, Office of New Drugs, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD
Trish Bright, PhD, MSPH; Danijela Stojanovic, PharmD, PhD; Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD
Ann McMahon, MD, MSc; Office of Pediatric Therapeutics, Office of Clinical Policy and Programs, Office of the Commissioner, Food and Drug Administration, Silver Spring, MD
Adebola Ajao, PhD; Division of Epidemiology, Office of Pharmacovigilance and Epidemiology, Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD
Hengrui Sun, PhD; Division of Biometrics IV, Office of Biostatistics, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD
Amy Bishara, MD; Clinical Review Branch 2, Division of Vaccines and Related Product Applications (DVRPA), Office of Vaccines Research and Review, Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD
Ashish Rai, PhD, MSPH, MBBS; Susan Forrow; Joy Kolonoski, MPH; Samantha Smith; Department of Population Medicine, Harvard Pilgrim Health Care Institute and Harvard Medical School, Boston, MA