Pivotal Clinical Trial: A clinical trial or study intended to provide evidence for a new treatment approval by the United States Food & Drug Administration (FDA)
This month we take a quick look at term that’s used in the field of biomedical research and drug development, that has both a figurative and an official technical/literal meaning – both of which essentially indicate the same thing: “Pivotal Trial.”
The term is significant at this moment, given that NBTS just published both our Second Quarter (Q2) update of newly opened brain tumor clinical trials and our half-year update specifically on phase III clinical trials that are enrolling for brain tumor patients.
A pivotal trial or pivotal study is a clinical trial or study that intends to provide the ultimate evidence and data that the FDA uses to decide whether or not to approve a potential new medicine.
A pivotal trial is usually a phase III clinical trial, but in exceptional cases, a phase II study can be used as a pivotal trial. They are sometimes also refered to as “registration trials.”
LITERAL & FIGURATIVE USE
In an official/regulatory setting the term is actually most often used for the rare phase II trial that is being used as the basis for seeking a new drug approval (i.e. “pivotal phase II trial”), since phase III trials in oncology are assumed to be pivotal trials.
However, in the figurative sense phase III trials are often colloquially termed “pivotal” in the sense that they are the most important, critical trials to determining whether a potential new treatment is safe and effective enough to receive FDA approval. Hence, why we’ve used the headline, “Potential Brain Tumor Treatments Entering the Pivotal Phase of Evaluation,” in our two reports to date on current, open phase III clinical trials for brain tumors. This is the make-or-break phase for clinical trials and for potential new treatments trying to make it to market.
HOW IT WORKS
The Food and Drug Administration‘s New Drug Application (NDA) is the vehicle in the United States through which drug-maker (or other trial “sponsor”) formally propose that the FDA approve a new medicine for sale and marketing to patients and doctors.
A pivotal study that is a trial designed & executed to get statistically significant evidence of efficacy and safety (as compared to the current standard treatments already approved) as required by the FDA for an NDA.
Once all the data is collected and analyzed for the pivotal trial – again, typically a phase III trial or pivotal phase II trial – the company can submit an NDA to the FDA. The NDA includes data from the trials, the preclinical information, and details about how the treatment is manufactured. The FDA then reviews the NDA and decides whether to approve the new medicine or not (this process contains a number of steps, including, often, a review committee hearing called an ODAC…but that’s a topic for a different day).
SOURCES, REFERENCES & FURTHER READING