Journal Article

Mandatory Registration and Results Reporting of Real-World Evidence Studies of FDA-Regulated Medical Products

Dhruva, Sanket S., Ross, Joseph S., Shah, Nilay D.

2020

Overview

In June 2017, the Food and Drug Administration (FDA) approved a new indication for use of the Edwards Sapien 3 transcatheter heart valve (Edwards Lifesciences, Irvine, California), 2 years after the initial approval of the device. This approval was unique because the underlying evidence was generated from a postmarket registry of patients receiving transcatheter aortic valve replacement, not from premarket clinical trials. Registries, along with electronic health records (EHRs) and claims data, are among the many data sources generated outside of traditional clinical research settings being used for real-world evidence (RWE).1 Such RWE-based approvals represent not only the future of generating evidence of the benefits and risks of medical products to inform regulatory approvals, but also decisions by payers, patients, and clinicians. Accordingly, to ensure that this evidence is rigorous and reliable, steps must be taken to require registration and results reporting of all RWE studies of FDA-regulated products.Such calls have been made previously for all observational studies,2-4 but recent changes at FDA heighten its pertinence for RWE studies. In 2017, FDA's Center for Devices and Radiologic Health (CDRH) released a Guidance Document entitled Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices. In December 2018, FDA met the 21st Century Cures Act provisions specifying that the agency create a program to evaluate use of RWE \"to help support the approval of a new indication for a drug already approved ... or to help support or satisfy drug postapproval study requirements.\"5 FDA has been shifting toward a life-cycle approach to medical product evaluation, increasingly relying on safety and effectiveness evaluations after market approval, as opposed to premarket, to facilitate earlier patient access to novel medical products.

• Publisher: Elsevier Inc,Elsevier, Inc,Frontline Medical Communications Inc,Elsevier Limited
• Subject: Access to Information / Aortic valve / Clinical Decision-Making / Clinical trials / Decision making / Device Approval - legislation & jurisprudence / Device Approval - standards / Electronic health records / Electronic medical records / FDA approval / Health care / Humans / Journalism, Medical / Laws, regulations and rules / Management / Mandatory Reporting / Medicaid / Medical equipment / Medical journals / Medicare / Patients / Product Surveillance, Postmarketing - methods / Product Surveillance, Postmarketing - standards / Public Reporting of Healthcare Data / Registration / Registries - standards / Risk Assessment / Statistical analysis / Task forces / United States