[BostonCard]

I am referring to people I know in pharma who deal with the FDA on a regular basis. They have great respect for the FDA as an institution, and for many (not all) of the people who work there. There is no question that these people are aware that they have effectively absolute power. If they tell you that you must do something in your clinical trial, even if their reason makes no sense, you basically have to do it. Many of the people at the FDA are knowledgeable but not real willing to change their minds. Often they do a great job, but sometimes they don't.

As a group, they are very risk averse. They all seem to be aware that asking you to be more conservative than you want to be has no down side to the FDA. It may delay the treatment availability, in fact it may render the treatment financially infeasible because the trial cost would be too high. However, the public isn't going to know about that, and if somebody complains they can wrap themselves in the "protecting the public" flag.

Everybody recognizes the FDA has a tough job. They have to balance risk vs benefit. Generally they try hard to do that in a professional manner. They will almost universally err on the side of caution. Unfortunately, some of the standards they set are of necessity somewhat arbitrary, as the "right answer" isn't known. That is an area where they are often too hard-headed in my friends view. Some endpoints that have what appears to be reasonable justification are rejected out of hand. That is hard to deal with.

My personal experience with the FDA is that they are tough but fair and rarely arbitrary or capricious. I know that it is often review division dependent, so my experience may not be universal.  I know for a fact that FDA reviewers are willing to change their minds if the data support doing so.  To quote a former FDA reviewer friend of mine, "in God we trust; all others must bring data".

Also, the FDA rarely will reject an endpoint out of hand, but unless it is a hard endpoint or one that has been the basis for approval in the past, they will require a strong justification and/or validation before allowing it.  Most often, we will get an answer like, "prior to acceptance of X as an endpoint, it will need to be validated as a meaningful and relevant outcome through x, y, and z."

BC

[BostonCard]

I am referring to people I know in pharma who deal with the FDA on a regular basis. They have great respect for the FDA as an institution, and for many (not all) of the people who work there. There is no question that these people are aware that they have effectively absolute power. If they tell you that you must do something in your clinical trial, even if their reason makes no sense, you basically have to do it. Many of the people at the FDA are knowledgeable but not real willing to change their minds. Often they do a great job, but sometimes they don't.

As a group, they are very risk averse. They all seem to be aware that asking you to be more conservative than you want to be has no down side to the FDA. It may delay the treatment availability, in fact it may render the treatment financially infeasible because the trial cost would be too high. However, the public isn't going to know about that, and if somebody complains they can wrap themselves in the "protecting the public" flag.

Everybody recognizes the FDA has a tough job. They have to balance risk vs benefit. Generally they try hard to do that in a professional manner. They will almost universally err on the side of caution. Unfortunately, some of the standards they set are of necessity somewhat arbitrary, as the "right answer" isn't known. That is an area where they are often too hard-headed in my friends view. Some endpoints that have what appears to be reasonable justification are rejected out of hand. That is hard to deal with.

My personal experience with the FDA is that they are tough but fair and rarely arbitrary or capricious. I know that it is often review division dependent, so my experience may not be universal.  I know for a fact that FDA reviewers are willing to change their minds if the data support doing so.  To quote a former FDA reviewer friend of mine, "in God we trust; all others must bring data".

Also, the FDA rarely will reject an endpoint out of hand, but unless it is a hard endpoint or one that has been the basis for approval in the past, they will require a strong justification and/or validation before allowing it.  Most often, we will get an answer like, "prior to acceptance of X as an endpoint, it will need to be validated as a meaningful and relevant outcome through x, y, and z."

BC