REGULATORY CORNER

In August 2025, FDA issued the draft guidance, Approaches to Assessment of Overall Survival in Oncology Clinical Trials. This guidance is based in part on discussions held at a joint FDA, American Association for Cancer Research (AACR), and American Statistical Association (ASA) public workshop held in July 2023. It also puts into writing feedback that I have received from the Agency on oncology clinical trials and guidance that I have related to our oncology clients.

Continuation of marketing approval of a product differs between the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). Marketing applications approved by the EMA are valid for 5 years and must be renewed to continue marketing of the product. If conditional approval was granted by EMA, a yearly reassessment is required. In the US, the situation is quite different. Once an application is approved by FDA, this approval continues unless the company requests that FDA withdraw the application or the FDA initiates a withdrawal process.

In May 2024, FDA issued the draft guidance, Platform Technology Designation Program for Drug Development. This guidance is applicable to drug products approved through the New Drug Application (NDA) or Abbreviated New Drug Application (ANDA) routes and biological products approved through the Biological License Application (BLA) route.

I found this guidance very interesting and informative. One of the first questions I receive from clients is, “Is this something that is applicable to my program?” The answer to this question has both a good news and a bad news component…

When I first read the guideline, Considerations for the Conduct of Clinical Trials of Medical Products During Major Disruptions Due to Disasters and Public Health Emergencies, which FDA issued in September 2023, my initial reaction was, ‘do we really need such a guideline?’ We have just gotten over the impact of the COVID pandemic on clinical trials, and business is getting back to usual or normal or whatever we want to call our new normal. That said, I found some interesting and very useful information in this guidance. Having lived through one pandemic, one thing I have learned is that we need to be prepared for the unexpected. While I hope that I will not experience another pandemic, other natural disasters and disruptions in clinical trial are increasingly more likely, especially in our current environment of global clinical studies.

I used to think of a Benefit Risk Assessment as part of a marketing application. However, FDA’s recent guidance, Benefit-Risk Assessment for New Drug and Biologic Products, published in October 2023, clearly highlights the role that Benefit-Risk Assessments play throughout the lifecycle of a product, not only at the time of New Drug Application (NDA) or Biologic Licensing Application (BLA) review.