REGULATORY CORNER

In May 2024, FDA issued the draft guidance, Platform Technology Designation Program for Drug Development.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.

In this blog, I will be referencing data in the public domain to compare and contrast the studies submitted to FDA to support label expansions for two marketed products and the similarities and differences in the FDA review and outcomes. By way of disclosure, PROMETRIKA has not worked on either of the two products discussed and all information presented is in the public domain (the October 5, 2023 Oncologic Drugs Advisory Committee (ODAC) FDA slides and ODAC vote for the first and the April 1, 2022 Clinical Review and Evaluation and Statistical Review for the second).

The Cures Act of December 2016 added section 505F to the Federal Food, Drug, and Cosmetic Act (FD&C Act), requiring FDA to create a framework for the use of real-world evidence (RWE) in regulatory decision-making. In response, the draft FDA guideline entitled, “Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products,” issued in August 2023, provides very interesting and useful information for consideration in the planning of a marketing application (NDA/BLA) for a drug or biologic product based on RWE.