Drugs

Standardized Methodologies for RWE Research Needed: Paper

Three researchers say that despite increasing interest in use of real-world evidence (RWE), there is no standard definition of the term that has been uniformly embraced. Writing online in JAMA Oncology, the researchers say FDA defines RWE as “data regarding the usage, or the potential benefits or risks of a drug derived from sources other than traditional clinical trials.” Further, the agency’s Real-World Evidence Program framework defines real-world data (RWD) as “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.”

“Real-world evidence is obtained from RWD, which encompasses data collected outside randomized clinical trials, ideally in diverse groups such as all Americans, all Europeans, or all global citizens,” the article says. “Real-world evidence can be derived through routine daily clinical practice or through patient interactions in their customary daily living environment. Because greater than 90% of patients with cancer are treated outside randomized clinical trials, RWE presents a unique avenue for obtaining data on patients with characteristics outside those typically required for trial eligibility.”

The researchers propose designing “proper standard methodologies to research RWE and clarifying when decision making is warranted, as output from such research could have regulatory implications.” They suggest that (1) RWD should be meaningful, valid, timely, and transparent to justify decision making based on its analysis; (2) RWE should be used to better clarify potential harms in understudied populations subsequent to marketing of novel therapies, and not used for basic licensure or approval of novel indications; and (3) analyzing RWE output to identify deficiencies that can guide future trials is critical, especially regarding rare diseases and groups that were excluded from original studies.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome