Post-Marketing Regulation & Studies

Some advisory committee members said adding quantitative data on misuse, abuse, opioid use disorder and overdose to labeling would be helpful, but others worried the two epidemiological studies were not sufficiently generalizable to a broader population.

In an unusual move, Tracy Beth Høeg, a special assistant to FDA Commissioner Martin Makary, is leading the continued negotiations on Novavax’s delayed COVID-19 vaccine approval.

Pink Sheet reporter and editors discuss the most recent senior staff departures at the FDA and their impact on the agency, as well as Commissioner Martin Makary’s plans for a new approval pathway and a combined adverse event database as outlined in an interview with a podcast host.

In discussing FDA’s adverse event monitoring, Makary also seemed to falsely imply the agency did not fully investigate the myocarditis signal with COVID-19 vaccines.

Regulatory authorities from multiple regions are considering extending a pilot project related to collaborative assessments of post-approval chemistry, manufacturing and controls changes, with a focus on the supply of critical medicines.

The pharmaceutical industry was not sufficiently involved in designing updates to England’s health data infrastructure, leading to a “misalignment” between company needs and what is currently being delivered, according to the results of a survey by the Association of the British Pharmaceutical Industry.

Patient advocates and sponsors want the US to reconsider a 20-year-old class warning against the use of antipsychotics in patients with dementia. The agency is weighing the issue, but also defending the current labels of newer agents.

Amid complaints about the challenges of following gene therapy patients for up to 15 years, OTP Director Nicole Verdun said the FDA is considering how to conduct long-term postmarket studies more efficiently.

The agency will consider study enrollment, accrual rates and the number of clinical site activations, among other factors, in determining whether a confirmatory trial is underway prior to accelerated approval, but the new draft guidance does not specify the proportion of patients that must be enrolled at the time of approval.

The Therapeutic Goods Administration is advising companies to get up to speed with its new recall procedure, which is designed to improve the timeliness of recalls, alerts and corrections, and reduce regulatory burden for sponsors.

Himal Thakur said the required long-term safety study can replicate a clinical trial, which is not the intent.

The landscape for primary biliary cholangitis or cirrhosis, PBC, is set to change if Intercept withdraws Ocaliva, which received its fourth safety alert from the US FDA and lost conditional marketing authorization in EU. Pink Sheet studies data from Citeline’s Pharmaprojects and separately Evaluate Pharma to reveal a promising clinical trials landscape and a sizeable market

Whether the FDA will seek withdrawal of the anticoagulant-reversal agent or request additional confirmatory data is unclear.

The US FDA seeks to better understand and predict insertional mutagenesis in gene therapies like bluebird bio's Skysona, which use integrating viral vectors, but a workshop on integration site analysis highlighted the approach's complexity and limitations.

The agency will focus less on new capabilities and more on meeting explicit mandates for the Sentinel active surveillance system, in part due to budgetary realities.

The confirmatory trial for the anticoagulant reversal agent raised safety and efficacy concerns from the agency.

The European Medicines Agency is encouraging companies to submit the type I variations they want to make to their marketing authorizations by the end of this month.

Updated guidance issued by the EU regulator explains how to comply with the provisions of the amended Variations Regulation, which will apply to new submissions from January 2025 and is intended to make lifecycle management “more efficient and future-proof.”

The US FDA Pediatric Advisory Committee uses web-posted reviews of “low safety risk” products to keep up with postmarketing monitoring requirements amid rising interest in pediatric development.

The FDA and sponsors of two new accelerated approval drugs for PBC have taken steps in study design, initiation and reporting transparency to ensure timely completion.