REGULATORY CORNER

When FDA published the guidance, Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases in August 2024, I read it with great interest. At first, I thought that it was a draft guideline, as this was the first time I had seen a guideline addressing dose selection in the oncology setting. But I quickly realized that this final guidance implements the information gathered in the FDA’s Project Optimus.

In May 2024, FDA issued the draft guidance, Platform Technology Designation Program for Drug Development.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

The new FDA draft guidance, Key Information and Facilitating Understanding in Informed Consent, (March 2024) provides some food for thought regarding the informed consent process and how we should organize our informed consents to best inform potential study participants about the studies we are conducting. I’ve recently read Patient H.M. (Luke Dittrich, 2016), about a patient with severe epilepsy who was treated with a lobotomy in an effort to reduce the seizures. While seizures remained severe, the procedure resulted in eliminating the patient’s ability to establish short-term memory and erased or jumbled most of the patient’s long-term memory. While it was noted that this particular patient did consent to the procedure, the book also chronicles stories of asylum patients with mental disorders who were given lobotomies without their or their legal representative’s prior consent. I am always both amazed and horrified when reminded that these types of research methods occurred in my lifetime. The processes we currently follow in generating informed consent forms (ICFs) for use in today’s clinical studies were not always standard procedure. Remembering this and understanding the importance of having each trial subject freely give consent, after being fully informed of the trial’s objectives, risks and benefits, is an interesting, important and relevant topic for consideration as we design and execute 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.