Summary authored by Takeo Fujii, MD, MPH

FDA and Oncology Product Development

Takeo Fujii, MD, MPH

Dr. Goulart’s presentation entitled “FDA and Oncology Product Development” provided a great overview of the regulatory process in new drug development and great insights on the role of the Food and Drug Administration (FDA). The entire process from the beginning of basic laboratory finding to the new drug application (NDA) and the post-marketing process after the approval of drugs was clearly introduced. He also shared the history of development of regulatory processesin drug development.

There are several learning points that I want to emphasize from his lecture. First, the concept of Investigational New Drug (IND) was explained sufficiently and the difference between IND and Investigational Device Exemption (IDE) was also clarified very well. He noted that the FDA hasa pre-IND consultation program in which applicants can discuss the non-clinical and clinical data necessary to IND application with FDA staff at the early stage of the drug development plan.IND can be exempt for certain situations. For example, cases where drugs are investigated or approved for specific types of malignancies may be candidates for IND exemption if the new investigation involves minimal modifications in the drug label or population assessed. Dr. Goulart emphasized the importance of early and close communication with the FDA at any level of drug development. 

Second, he clarified the concepts of four different expedited drug development processes. This is important for researchers to understand but is often not easy for us to understand the difference correctly.  (1) “Fast Track Designation” is the accelerated development and review process of new drugs or biologics that will be used to treat serious or life-threatening health conditions and fill clinical unmet needs based on clinical and non-clinical data. The requests are usually submitted during or after IND submission. (2) “Breakthrough Therapy Designation” is intended to expedite the review process of drugs for serious conditions diseases with preliminary data indicating that drugs might provide substantial improvement over the available treatments. (3) “Priority Review Designation” means that the FDA will take regulatory action within 6 months, in contrast to 10 months under the standard review process. This is granted if the drugs would significantly improve the safety or efficacy of the treatment, diagnosis, or prevention of serious diseases, if approved.  (4) “Accelerated Approval” is the approval process of new drugs that demonstrate efficacy based on “surrogate endpoints” or an intermediate clinical endpoint. This approval is granted only for serious or life-threatening conditions. The surrogate endpoints need to be what is thought to predict clinical benefit but is not itself a measure of clinical benefit.  Confirmatory clinical trials with the primary endpoint of the anticipated clinical benefit such as overall survival must be conducted. If the confirmatory clinical trials fail to show clinical benefits, the FDA may withdraw the approval, but the ultimate regulatory decision depends on the specific context and may not necessarily involve withdrawal of the drug from the U.S. market.