FDA Updates for the Week of June 19, 2023

• Northern Ireland’s Health Minister has praised a Public Health Agency warning regarding drug use, describing the guidance as "practical." • The statement comes amid legal proceedings where the Minister and his wife, Eleanor Donaldson of Dromore, Co Down, face criminal charges.

• The Centers for Disease Control and Prevention (CDC) is seeking public and agency comments on a proposed continuing information collection project. • The project focuses on the "Use of the Cyclosporiasis National Hypothesis Generating Questionnaire (CNHGQ)" during investigations of foodborne disease clusters and outbreaks.

• Researchers are analyzing the current debate regarding the construction of layperson summaries for clinical trial results within the European Union. • The authors propose a "reader-centered" approach that prioritizes the most critical information for patients, specifically the efficacy and safety of the drug.

• Following the resignation of Commissioner Marty Makary, the FDA is seeking a new leader to oversee the agency's commitment to accelerating treatment access for rare disease patients. • The urgency is highlighted by UX111 gene therapy, which clinical trials show can reduce toxin buildup for over eight years and slow cognitive decline if administered early.

• Author of the new book "Decolonising Disease: Pandemics, Public Health and Pathogenic Novels" explores the intersection of colonialism, imperialism, and epidemic disease. • The work draws critical parallels between the COVID-19 pandemic and historical Ebola outbreaks, specifically those in Congo/Zaire and Sudan in 1976 and West Africa from 2014–16.

• WHO advisory groups have selected several candidates for clinical trial evaluation during the current outbreak response, including remdesivir, Maftivimab®, and vaccine candidates rVSV and ChAdOx1 Bundibugyo. • New research highlights durable results from PCSK9 base-editing (VERVE-102) and examines the impact of GLP-1 agonists like semaglutide on health outcomes.

• Uganda ordered the closure of its border with Congo on Wednesday, May 27, 2026, following a surge of suspected Ebola cases in the neighboring country. • The decision follows the confirmation of Ebola cases within Uganda after local health workers were exposed to infected Congolese patients.

• Regulatory bodies are increasingly integrating real-world evidence (RWE) into the drug approval process to supplement traditional randomized clinical trials. • This shift utilizes real-world data (RWD) sourced from electronic health records, administrative claims databases, disease registries, and digital health tools.

• The acting director of the U.S. NIH’s infectious disease institute has resigned amidst concurrent outbreaks of hantavirus and Ebola. • Political tension has risen over the Affordable Medicine Act, with critics arguing the legislation failed to provide sufficient relief to Virginians outside of Medicare or hold pharmaceutical companies accountable.

• Unproven treatments, including ivermectin, vitamin D, and zinc, are being promoted on social media as cures for hantavirus following a recent cruise ship outbreak. • While vitamin D and zinc are known to support general immune regulation, there is currently no hantavirus-specific clinical evidence to support their use for this disease.

• The Ebola outbreak is continuing to worsen, creating significant collateral health and safety risks specifically for women and girls. • In a surprising leadership change, Kyle Diamantas has been appointed as the new acting head of the FDA.