Center of Excellence in Regulatory Science and Innovation
The Yale-Mayo CERSI conducts high-quality, high-impact collaborative research to support several areas of focus in the FDA strategic plan for regulatory science. Research topic areas include: adoption/de-adoption of FDA-approved medical products, postmarket surveillance, development and application of novel analytics, and patient-centered regulatory decision-making.
For comments or questions pertaining to our CERSI research projects, contact the Yale-Mayo Clinic CERSI at CERSI@yale.edu.
Generic drugs are approved based on bioequivalence to the brand name agents. However, there are sometimes concerns among patients and clinicians that generic and brand name drugs are not equivalent and have differing effects. Building on our ongoing research, we will test different methods to compare the effectiveness and safety of generic and brand name drugs using a large administrative claims data source that includes information on privately insured and Medicare Advantage enrollees of all ages. Further, we will create packages that will ease the implementation of these methods for future research.
Non-Federal Entity Collaborators: Anirban Basu, PhD, Co-Investigator (University of Washington)
A novel sync-for-science mobile application has been developed that unobtrusively enables patients to provide their own outcomes (through short questionnaires and through synchronizing data from mobile health trackers) to the FDA after they have received a procedure that utilizes medical devices. In addition, with user permission, this application draws data from the electronic medical record to complement patient-reported data. In this project, we will conduct a pilot study testing this mobile health application to enable the FDA to conduct post-market surveillance of two procedures that use medical devices: the multiple devices (including sutures and stapler) used to perform bariatric surgeries (either sleeve gastrectomy or gastric bypass) in patients seeking weight loss and an ablation catheter when used in patients with atrial fibrillation seeking a return to sinus rhythm. Patients will be enrolled before receiving each of the devices and then will be asked to report specific symptoms related to their need for the procedure and those that may be expected at baseline (enrollment, which is pre-procedure), and 1, 4, and 8 weeks post-procedure. Additionally, patients will be asked 2-3 short questions every 3-4 days for the first 30 days post-procedure related to post-procedure symptoms. We will also test if these patients’ electronic health record data from multiple health systems where they receive care can be synchronized into a research-ready database. Patients will also be provided with syncable devices to provide additional insights into their health and health outcomes. Finally, we will test the feasibility of obtaining medication data from pharmacies or the current needs to create a functional system that can integrate pharmacy data into the mobile application. Integration of these multiple data sources (patient-reported outcomes, wearable/mobile device data, electronic health record data, and pharmacy data) have the potential to ultimately enable a more robust and thorough post-marketing surveillance strategy by leveraging the potential of digital health technologies. Non-Federal entities involved in this project include Me2Health (the developers of the Hugo mHealth application), Johnson & Johnson (collaborator- provides input on project and funds to Me2Health for development of Hugo mHealth application) and AliveCor (donated Kardia Mobile devices for this project).
ClinicalTrials.gov Identifier: NCT03436082
Non-Federal Entity Collaborators: Karla Childers, MSJ, Paul Coplan, ScD, MBA, and Stephen Johnston, MSc (Johnson and Johnson)
This project seeks to understand how linking laboratory data to insurance claims can help examine a drug’s performance after approval. We will conduct a case study looking at renal function and the performance of oral anticoagulant drugs in patients with atrial fibrillation (AF). The 30 million patients with AF are at nearly a five-fold risk of stroke. Lifelong oral anticoagulation is recommended in most patients with AF to prevent stroke. However, treatment decisions can be complicated by the presence of chronic kidney disease (CKD), as poor renal function increases the risks of both stroke and bleeding ( a major complication of oral anticoagulation treatment), and may change the risk-benefit ratio of different treatment options. This project proposes to answer three important questions pertaining to the impact of renal function on oral anticoagulation treatment in patients with AF. First, in patients with severe-to-no renal impairment, we will assess the comparative effectiveness and safety of different oral anticoagulant drugs across the range of renal function. Second, in patients on dialysis who have substantial risks of both stroke and bleeding, we will compare different potential treatment options, including warfarin, non-vitamin K antagonist oral anticoagulants (NOACs), and no treatment. Third, we will use novel analytic methods to identify patient characteristics that contribute to the heterogeneity in treatment effects.
We will answer these questions by leveraging the power of a large observational database, OptumLabs Data Warehouse, which contains over 160 million privately insured and Medicare Advantage enrollees of all ages, races, and from 50 states and the USRDS data. The proposed work could provide important new evidence on the safety and effectiveness of oral anticoagulants in relation to renal function and will help physicians and patients make a choice among different treatment options.
Non-Federal Entity Collaborators: Brahmajee Nallamothu, MD, MPH, Rajiv Saran, MD, MS, MBBS, and Konstantinos Siontis, MD - Co-Investigators (University of Michigan)
Non-Federal Entity Collaborators: James Hummel, MD- Co-Investigator (University of Wisconsin School of Medicine)
This project proposes to advance our understanding of cardiogenic shock with the ultimate aim of enabling patients and providers to estimate risk and develop optimal, individualized treatment plans. Specifically, we will use the NCDR CathPCI and ACTION-GWTG registries, two national registries of patients with acute myocardial infarction (ACTION-GWTG) and patients undergoing stent procedures (CathPCI). Given the substantial morbidity and mortality associated with cardiogenic shock, the proposed work will enable us to advance our understanding of this condition, develop better treatment approaches, and will enhance regulatory science by improving the safety and effectiveness of mechanical circulatory support devices through helping target them to patients who will benefit. Collaborators from Texas A&M University are involved in this project.