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Jul
24

Targeted Drug Development : The FDA perspective – A more approachable regulator?

As I am slowly catching up on my reading and I came across this new white paper type doc from the FDA which came out a few weeks ago called Targeted Drug Development: Why are Many Diseases Lagging Behind?

It makes for an interesting read and of course conjures up images of diseases on a race track.. but should it? Tortoise and hare each being different diseases, Alzheimers and Hepatitis C perhaps in this analogy. 

I did get a sense of the FDA throwing down a challenge and saying that discovery and testing had not kept up, the regulatory side was doing just fine thanks, but that’s just my take.

The diseases chosen were those which billions of dollars / euros have been invested, cancer, diabetes, Alzheimers, HIV/AIDS and Hepatitis C. And then there is a little mention of rare diseases, which appear to be the Cinderella here, they are generally poorly funded, have few patients and have to be creative to make their mark.

Take home messages –

These were the need and use of surrogate endpoints in clinical trials. Flexible trial designs were also mentioned and this could mean many things such as adaptive trials, or anything that veers away from the gold standard randomized placebo controlled trial. The need for the FDA to work closely (collaborate) with companies, academia and patient organizations was also touted as important.

Is this a sign of a more approachable (read warmer and cuddlier) FDA as they push the US as leading the world in the introduction of novel drugs?

Personally I think a 13 page PDF is a bit much, it could have been boiled down to one page. My summary is that even if you spend billions on a big disease over decades you still may not have what it takes to find a treatment. Focus on what clearly matters to the FDA, come up with a good surrogate endpoint, plan a flexible trial, collaborate and voila you should be able to succeed. According to this document it sounds like the FDA has development covered and it is a shot across the bows to discovery to clean up their act and get with the program. Not so fast you might say – I can bet coming soon is a comparable doc from the NIH saying we need more funding to improve the discovery situation. So is this all posturing ready for the next president you might ask? Well I will let you decide, but frankly I wished the FDA would just focus on getting more drugs approved and leave white papers to others.

 

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