Many activities conducted within FDA’s Sentinel System are aimed at gaining a better understanding of the pediatric patient population. These include studies that estimate rates of specific health outcomes, as well as studies that examine prevalent and incident use of medical products, among pediatric patients in the Sentinel System. Descriptions of efforts led by the Center for Drug Evaluation and Research are shown below. Please visit the links to learn more about each area of activity.
Beta-blockers are only approved for two pediatric indications in the United States (i.e., hypertension and hemangioma), but are commonly used for a variety of off-label conditions. Little is known about the magnitude and patterns of their use in the pediatric population.
This poster examines utilization patterns of beta-blockers among the pediatric population in the United States. It was presented at the 39th International Conference on Pharmacoepidemiology and Therapeutic Risk Management.
In this request, we examined counts of individuals with a beta blocker dispensing of interest among the pediatric population in the Sentinel Distributed Database (SDD). This request is a follow up to a previous analysis to add additional stratified analyses. This request contains three reports:
- Report 1: contains counts of individuals among the pediatric population with evidence of a new beta blocker dispensing.
- Report 2: contains counts of individuals among the pediatric population without requiring evidence that a beta blocker dispensing be new.
- Report 3: contains counts of individuals among the pediatric population with evidence of a new beta blocker prescription.
The study period includes data from January 1, 2008 through May 31, 2022. We distributed this request to eight Sentinel Data Partners on September 27, 2022.
In this request, we examined counts of individuals with a beta blocker dispensing of interest among the pediatric population in the Sentinel Distributed Database (SDD). This request contains two reports:
- Report 1: contains counts of individuals among the pediatric population with evidence of a new beta blocker dispensing.
- Report 2: contains counts of individuals among the pediatric population without requiring evidence that a beta blocker dispensing be new.
The study period includes data from January 1, 2008 to September 30, 2021. We distributed this request to eight Sentinel Data Partners on March 28, 2022.
This was a retrospective descriptive study among children <18 years hospitalized from February 20, 2020, through September 30, 2021, using inpatient EHR data from HCA Healthcare. Hospitals were in 18 states, with Texas, Florida, Virginia, and Colorado heavily represented. We examined patient demographics on admission and analyzed the frequency of admission by month across the study period. We examined conditions that may increase risk of serious complications from COVID-19, including cancer, chronic lung diseases, chronic cardiovascular disease (e.g., congenital heart disease), liver or renal disorders, immunocompromised state, diabetes, obesity, developmental disorders, and smoking. We also assessed how often patients had SARS-CoV-2 polymerase chain reaction (PCR) tests performed within the hospital system.
This presentation provides an overview of the Sentinel System and how the pediatric population is characterized in Sentinel, as well as a brief review of select pediatric-focused Sentinel studies.
It was presented on March 24, 2023 for the Clinical Futures Visiting Speaker Seminar Series at the Children’s Hospital of Philadelphia (CHOP) Research Institute.
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).
This poster describes demographics, enrollment, and health characteristics among pediatric members in the Sentinel Distributed Database. It was presented at the 38th International Conference on Pharmacoepidemiology and Therapeutic Risk Management.
This analysis includes two reports:
- Report 1: We examined the trends in diagnosis of congenital cytomegalovirus disease (cCMV) or cytomegalovirus infection (CMV). We also assessed clinical characteristics, laboratory tests performed for CMV diagnosis, hearing loss and hematologic outcomes among infants with CMV and those who used valganciclovir/ganciclovir treatment.
- Report 2: We assessed follow-up time and distribution of valganciclovir or ganciclovir treatment in infants with cCMV. We also assessed clinical characteristics, laboratory tests performed for CMV diagnosis, hearing loss and hematologic outcomes among infants with CMV who used valganciclovir/ganciclovir treatment.
The study period includes data from January 1, 2008 through May 31, 2021. We distributed this request to 12 Sentinel Data Partners on December 9, 2021.
The proposed goal of this project was to pilot, via two use cases, the ability to more routinely and broadly understand how FDA might conduct “on-demand quality assurance” for selected vital signs and laboratory results in a within cohort fashion. The first use case evaluated a cohort of pediatric patients with hypertension. The second use case evaluated a cohort of new adult users of sodium-glucose co-transporter-2 (SGLT-2) inhibitors.
Vital signs and laboratory measures were evaluated for each use case. Measures included blood pressure as well as smoking use/status, height, and weight. Laboratory tests included Hemoglobin A1c and creatinine.
A comparison of the identification using a claims-only phenotype versus a clinical data (e.g., electronic health record based) phenotype was conducted. Two reports are posted from the first use case that evaluated a cohort of pediatric patients with hypertension. In the first report, the hypertension algorithm is simplified. The second report utilized a more complex hypertension algorithm to define events of interest. Two reports are posted from the second use case that evaluated counts of individuals with SGLT-2 inhibitor and sitagliptin dispensings in the Sentinel Distributed Database (SDD) along with baseline conditions and other medication use. The first report did not include integrated delivery systems data in the analysis; the second report only included integrated delivery systems data in the analysis.
In this request, we estimated rates of hypertension in a pediatric population within the Sentinel Distributed Database (SDD).
The study period included data from January 1, 2008 to April 30, 2019. We distributed the request to 15 Data Partners on September 26, 2019.