What is the Difference Between Required and Nice to Know?


  • February 13, 2012

This actually can be a very good question to ask, especially when considering elements of drug development. Often, there are multiple roads that lead to Rome, or in this case an acceptable clinical trial proposal or NDA filing. Why is this the case? The answer is reflected in the regulations and how they are written in order to allow for advances in science without having to be constantly revised. It is also an element of science and a residual of us all being human.

First of all, one should understand what is written in the FDA regulations. If you actually have the time to read them, you will find them to be quite specific in some areas (e.g. annual reports must be filed for an IND within 60 days of the anniversary of the IND filing), while they remain fairly ambiguous in other areas (e.g. specifically what studies need to be done to get a drug approved). The regulations talk about demonstrating proof of safety and efficacy, but recognize that an exhaustive list addressing every situation cannot be generated. This is where the regulations provide the FDA authority to issue guidance to share the agency’s thoughts on specific elements of the drug development process with the public. The guidances are in fact non-binding, but provide a sponsor with an idea of the points that need to be addressed in order to demonstrate safety and efficacy of their product. A sponsor may propose alternative approaches to addressing these points to the Agency. The ability to pursue these alternative approaches will depend on the robustness of the proposed approach and the other data collected on the drug.

Secondly, science is an ever moving target and our understanding of things can change drastically with just one new piece of information. This may be reflected at the scale of an individual program or a whole class of drugs. It can even change how to treat a disease (e.g. the discovery that AIDS is caused by a retrovirus). Asking the question “Is this study necessary, given what we now know about X?” is very helpful in creating a streamlined development strategy.

Finally, everyone is human. It is very possible that an explanation was not clear and led to a misunderstanding of the underlying dataset. It is also possible that not all of the information was available for review. If you believe that you have already collected data that addresses a specific request, you should find a tactful way to direct the reviewer to the pertinent piece of information so that they can consider and possibly refine their recommendations.

Posted by Theresa Allio, Senior Consultant. For more information, please contact Jeff Antos at jeff.antos@weinberggroup.com.