As the US Food and Drug Administration (FDA) grapples with how to use real-world data for regulatory decisions, two FDA officials wrote in a perspective published last Friday in the Clinical Journal of the American Society of Nephrology that the agency is developing guidance on data quality issues unique to the real-world data setting and related study design considerations. Aliza Thompson and Mary Ross Southworth of the Division of Cardiovascular and Renal Products w...

A European drug regulators’ taskforce on big data published its first report on Friday. The Heads of Medicines Agencies (HMA) and the European Medicines Agency (EMA) formed the HMA-EMA Joint Big Data Taskforce to harness the potential of data in development and oversight, starting with the new report. They examined clinical trial data, spontaneous adverse drug reports (ADRs), social media data and real-world data (RWD) to inform the work and reach “an important mileston...

The US Food and Drug Administration (FDA) issued a request for comment on Monday that sets forth a proposal for a new framework on the regulation of software applications for use with one or more prescription drug products.   The agency’s newly adopted digital health approaches “are still in their early stages,” FDA Commissioner Scott Gottlieb said Monday in remarks at the Reagan Udall Foundation annual public meeting. “A lot of these technologies haven’t advanced as...

The National Evaluation System for health Technology Coordinating Center (NESTcc), which is mutually funded by the US Food and Drug Administration (FDA) and industry, announced Monday it has selected a total of eight real-world evidence (RWE) test cases to evaluate medical device performance in clinical settings.   NESTcc based its selections on applications medical device manufacturers submitted in January under a public call for concepts initiated last year and s...

The National Evaluation System for health Technology Coordinating Center (NESTcc) this week put out three requests for proposals to help it build the case for using real world evidence for regulatory and coverage decisions for medical devices. Background In 2016, the US Food and Drug Administration's (FDA) Center for Devices and Radiological Health (CDRH) awarded a $3 million grant to the Medical Device Innovation Consortium (MDIC) to establish NESTcc. The cente...

A study published in JAMA Internal Medicine on Monday was able to replicate the results of a large randomized controlled clinical trial for the blood pressure drug telmisartan using real world evidence gathered from insurance claims data. The goal of the study, which was conducted by researchers at Harvard Medical School, was to investigate whether health care databases can be used to confirm findings from clinical trials conducted to support additional indications for...

The group of stakeholders building the National Evaluation System for health Technology (NEST) outlined what’s in store for the new future as well as the direction that they want the system to go. "NEST is about how we improve the value, use, and time and cost of using real-world data," Jeff Shuren, director of the US Food and Drug Administration’s (FDA) Center for Device and Radiological Health (CDRH), explained to attendees of AdvaMed’s MedTech conference in San J...

The clinical trials system is "broken" and there needs to be new ways to collect and utilize patient data, Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, told a workshop on real world evidence (RWE) at the National Academies of Sciences, Engineering, and Medicine on Wednesday. The comment came at the end of Woodcock's talk in which she also noted that use of master protocols (ie. protocols for trials that look at multiple therapies in ...

The US Food and Drug Administration (FDA) has finalized a slightly tweaked guidance on medical device companies’ use of real-world data (RWD) and real-world evidence (RWE) in winning approvals for their products. FDA says the guidance is meant to explain the "characteristics and sources of RWD and characteristics of RWE that may be sufficient for use in making various regulatory decisions." In addition to the draft guidance, the final guidance includes further clar...

The US Food and Drug Administration (FDA) says it relied on real-world evidence in its decision to expand the use of Edwards Lifesciences' Sapien 3 transcatheter aortic valve replacement (TAVR) to valve-in-valve procedures. In a blog post Wednesday, Center for Devices and Radiological Health (CDRH) Director Jeffrey Shuren and Bram Zuckerman, director of CDRH's Division of Cardiovascular Devices touted the agency's turnaround from 42nd in the world to approve a TAVR devic...

Top officials at the US Food and Drug Administration (FDA) are defending the agency's flexibility to accelerate the availability of products by considering diverse data sources in product submissions. In an article in Nature Reviews Drug Discovery on Friday, Rachel Sherman, deputy commissioner for medical products and tobacco in the Office of the Commissioner at FDA, writing alongside former FDA Commissioner Robert Califf and other high-ranking agency officials, argue ...

Fifteen top US Food and Drug Administration (FDA) officials published an article in the New England Journal of Medicine on Thursday calling into question some of the potential uses of real-world evidence (RWE) but also acknowledging that real world research and the concepts of a planned intervention and randomization “are entirely compatible." Defining RWE Rachel Sherman, deputy commissioner for medical products and tobacco in the Office of the Commissioner at FDA, wr...