OIG Says FDA Lacks Comprehensive REMS Data
Office of the Inspector General (OIG) recently released its finding on the FDA's effective of the Risk Evaluation and Mitigation Strategies (REMS), which are for drugs associated with known or potential risks that may outweigh the drugs’ benefits. It reviewed approved REMS since the program's inception in 2008 through 2011 and conducted structured interviews with FDA officials about FDA’s efforts to evaluate REMS components. It also reviewed 49 sponsors’ REMS assessments and FDA’s reviews of these assessments to determine the extent to which sponsors’ assessments were complete, were submitted to FDA within required timeframes, and indicated that REMS were meeting their goals. We also determined whether FDA evaluated theelements to assure safe use (ETASU) of one drug in each year of the program, as required by Federal law. The OIG found, as noted by the report's title, the FDA lacks comprehensive data on REMS. Specifically, nearly half of sponsor assessments for the 49 REMS we reviewed did not include all information requested in FDA assessment plans, and 10 were not submitted to FDA within required timeframes. FDA determined that 7 of the 49 REMS we reviewed met all of their goals. However, FDA has not identified reliable methods to assess the effectiveness of REMS. Finally, FDA’s assessment review times exceeded its goal of 60 days for all but one sponsor assessment, which reduces sponsors’ time to make suggested changes before submitting subsequent assessments.
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Improving Relationships and Diversifying the Site Selection Process
April 17th 2025In this episode of the Applied Clinical Trials Podcast, Liz Beatty, co-founder and chief strategy officer, Inato, discusses a number of topics around site engagement including community-based sites, the role of technology in improving site/sponsor relationships, how increased operational costs are impacting the industry, and more.
Behind the Buzz: Why Clinical Research Leaders Flock to SCOPE Summit
February 7th 2025In this episode, we meet with Micah Lieberman, Executive Conference Director for SCOPE Summit (Summit for Clinical Ops Executives) at Cambridge Innovation Institute. We will dive deep into the critical role of collaboration within the clinical research ecosystem. How do we bring together diverse stakeholders—sponsors, CROs, clinical trial tech innovators, suppliers, patients, sites, advocacy organizations, investors, and non-profits—to share best practices in trial design, program planning, innovation, and clinical operations? We’ll explore why it’s vital for thought leaders to step beyond their own organizations and learn from others, exchanging ideas that drive advancements in clinical research. Additionally, we’ll discuss the pivotal role of scientific conferences like SCOPE Summit in fostering these essential connections and collaborations, helping shape the future of clinical trials. Join us as we uncover how collective wisdom and cross-industry partnerships are transforming the landscape of clinical research.
FDA-Approved Gene Therapy Beqvez Shows Sustained Efficacy, Safety in Long-Term Hemophilia B Trial
April 17th 2025Beqvez (fidanacogene elaparvovec), an FDA-approved one-time gene therapy for hemophilia B, demonstrated sustained factor IX expression, low bleeding rates, and a favorable safety profile over long-term follow-up.
