Post-Marketing Regulation & Studies
Recent ODAC meetings suggest the FDA will expand focus beyond applications with pending approval decisions to refining trial designs and drug labels for competitors.
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; Intercept’s COBALT trial of obeticholic acid was negatively impacted by product safety labeling changes and a high crossover rate to commercial drug.
Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.
A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
Even as US FDA expressed concern about the perioperative trial design, AstraZeneca’s ability to snag NSCLC indication without demonstrating the benefits of both the neoadjuvant and adjuvant phases of therapy highlights slow pace of agency policy shifts.
The first engineered cell therapy for a solid tumor could be submitted for full approval in synovial sarcoma next year.
The European Medicines Agency is seeking feedback on how to standardize the prospective planning and reporting of cardiovascular safety endpoints in oncology trials.
The head of CBER's Office of Therapeutic Products also touted hiring achievements and new communication plans with sponsors at an Alliance for Regenerative Medicine meeting.
The US FDA’s new draft guidance notes its own efforts to combat misinformation and offers some creative examples to illustrate its advice, but avoids any potentially “controversial” contemporary situations.
The FDA creates a new advertising and promotion regulation safe harbor for companies who narrowly respond online to internet-based misinformation about their drugs.
Europe’s recently launched electronic RWD catalogs as of June contained 216 registered data sources and 2,840 studies distributed across various regions of the world.
A new requirement for drug companies on how much safety stock to maintain and a mechanism for extending the expiration date of a lot or batch of a drug if there is a shortage that could cause harm are among the measures Health Canada is proposing to adopt.
The European Medicines Agency is reviewing the known risk of agranulocytosis in people taking medicines containing the painkiller metamizole, which is approved for use in most of the EU member states but banned in the US, the UK and other countries around the world.
FDA leadership weighs in on limitations of AI as part of rollout of new technology meeting program that looks to give industry and other stakeholders a chance to inform future regulation.
The unanimous decision that the Alliance for Hippocratic Medicine lacks standing to challenge the FDA’s relaxation of the abortion pill REMS leaves unclear whether the court would have deferred to the agency’s expertise on the merits.
Feedback is being sought on revised guidelines that tell companies how they should make post-authorization changes to their drug approvals once new rules take effect in January 2025.
Ninety-two approvals, seven revocations and myriad standard-of-care treatments are all discussed in a first-of-its kind study examining the data and nuances of oncology submissions processed under Health Canada’s 26-year-old notice of compliance with conditions (NOC/c) pathway.
An internationally harmonized framework for generating adequate evidence using fit-for-purpose real-world data to address regulatory questions on the safety of medicines is out for public consultation.
The Duchenne muscular dystrophy treatment missed its primary endpoint of time to stand from supine in the RACER53 study, raising more questions about using dystrophin changes to support accelerated approval and the ability of functional endpoints to detect clinical change.