FDA's Hamburg on Research Investment and Clearing the Hurdles to Drug Approvals

Marrecca Fiore; Margaret Hamburg, MD


October 17, 2013

In This Article

Advancing Personalized Medicine and Scientific Enterprise

Medscape: One of the FDA's science and research initiatives is to stimulate innovation, clinical trials, and personalized medicine. Personalized medicine and genomic medicine are becoming increasingly important in the treatment of diseases. Can you explain what is being done in this area?

Dr. Hamburg: As we learn a lot more about the underlying mechanisms of disease and the relationship of certain disease processes to certain genetic traits, we also understand that even within one disease category, responsiveness to certain treatments may be different depending on certain genetic traits of the individual -- or in the case of oncology, the tumor.

We have an opportunity now to really deepen these understandings and build on them in terms of the therapies that are developed and how they are used, and that requires some new approaches in terms of the types of therapies that are being developed and the use of diagnostics that are a companion to therapy so that you can identify the subsets of responders and nonresponders. We are very deeply immersed in these activities. We have seen a number of really exciting approvals in recent years linking the diagnostic with the therapeutic, and we have seen some dramatic translations of new scientific understandings into products for people based on this approach. We are going to see more and more as time goes on.

Medscape: Finally, what is on your wish list for the future in terms of research and research funding of drugs and devices?

Dr. Hamburg: When we look at the sort of scientific enterprise overall, the understanding and investment in regulatory science have not been adequate. I think that if we really want to be able to deliver the biomedical products that the public wants and deserves, we have to address that. My wish list would be that we at FDA could get additional resources to help fund research in critical areas that will enable us to apply the advances in science and technology more effectively to the drug development process and the drug review process, whether it is genomics or bioinformatics, bioimaging, new clinical trial designs, etc.

There is so much opportunity, but it hasn't been fully developed. We have started some centers of excellence for regulatory science in academic centers, and it has been very, very productive and very valuable. We have created new partnerships, including precompetitive collaborative research with industry academia and government, including the National Institutes of Health. All of that has demonstrated real benefit in helping to move research and development forward and getting some of these exciting new understandings of disease and health out to people in terms of new products. But it is not enough.

We really need to capitalize on the opportunities [presented at the conference] today -- that would be my wish list. Of course, the reality as we know it is that dollars are constricting, so we have to be as smart as possible about leveraging resources. Part of what we talked about in the conference today was that we are going to have to make some hard choices. We are going to have to think about how we spend limited dollars, but I think that we really need to recognize that this arena of regulatory science is so key, and that it has been the weakest link in the chain of science that is necessary to truly realize the potential of science today.


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