Marrecca Fiore, Margaret Hamburg, MD
October 17, 2013
Editor's Note :
US Food and Drug Administration (FDA) Commissioner Margaret Hamburg, MD, spoke with Medscape during the recent National Health Research Forum in Washington, DC, and discussed clearing the hurdles to drug and device approvals, advancing personalized medicine, and smart spending to leverage scientific enterprise.
Medscape: Your panel today talked about "clearing the hurdles to a research and healthcare renaissance." Some researchers and clinicians see the FDA as a hurdle to getting new lifesaving medications and medical devices to patients. Can you discuss what the FDA is doing right now to streamline and speed up the drug and device approval process?
Dr. Hamburg: Sure. When you look at what is required to move an important discovery in science into a real-world product that patients and consumers want and need, obviously we play a critical role. We are responsible for reviewing the data to really examine critical questions about safety and efficacy. We see that as a clear value for patients and their families, and also for the industry sponsors of these products. Because if the product doesn't work, if it is going to cause problems, it is not in the industry's best interest either.
We need to all work together, and I think part of the discussion in the conference today was how important it is to have all the voices and perspectives represented around the table. FDA is one important player in that process, and we play a critical role because we sit in a position where we can look on one side and see what the unmet medical and public health needs are, and we can also see what is in the pipeline.
But we have an obligation to do our jobs as effectively and efficiently as possible. During my tenure as commissioner, I have worked hard to make sure that, number one, we are as engaged in the science as we can possibly be, and to make sure that we have people with the right scientific expertise and experience to appropriately review applications that come before us. More important, to engage with the scientific community early and often so that, as products are being developed, as new discoveries are moving through the pipeline, we help identify what kinds of data are going to be needed, what kinds of questions have to be asked and answered as part of the ultimate review process, and our ability to approve a drug to go into the marketplace. And we know that by working together in that way, we can actually speed the development process and the review process.
We also have to commit ourselves to making sure that our business processes are as efficient as possible so that we don't sit on applications, so that we return phone calls, so that we have the kind of mechanisms to hire the people that we need, train them properly, etc. We have focused very much on really strengthening our ability to do our job, but we see our job as part of a bigger mission to really advance biomedical product innovation.
Record Numbers of Drug and Device Approvals
Medscape: Correct me if I am wrong, but I believe you said that the FDA has actually been approving more drugs and devices than in the past.
Dr. Hamburg: In the past couple of years, we have had record numbers of new medical product approvals and, more important, we approve more drugs first. If you compare us with other countries, we approve new drugs more quickly. I think we are providing a critical and unique service to the American people.
We can always do better. We must do better, but I think one of the lessons from our panel today -- and certainly a lesson from my experience at FDA and working as a bench scientist and a healthcare provider -- is that we have to always recognize that real, sustainable progress doesn't happen by operating in a silo. We need this kind of partnership and coordination across all of the components and all of the stakeholders [in the approval process] to really make the difference we want to make.
Medscape: Turning to clinical trials, there are some who believe that the randomized, placebo-controlled trial is somewhat antiquated and not always the best method of gauging real-world results when it comes to new drugs and devices. How is the FDA working to improve the way it tests new products?
Dr. Hamburg: We are so far down that path that this question is almost irrelevant, to be honest. Obviously, the randomized controlled clinical trial is the absolute gold standard in terms of rigorous science and getting really solid answers. But there are lots of other ways to get robust scientific answers without using that, and there are many circumstances where you simply can't do a randomized controlled clinical trial for ethnical reasons or practical reasons. We look at [this kind of trial] as one tool in our toolbox, and we use many other strategies in terms of the kinds of clinical data that we will use for review.
We have also funded a lot of work in this area to really look at innovative clinical trial design, working with our academic partners and in some cases with industry. We see it as an area ripe for continuing collaboration and development of new strategies and approaches. But it is critically important, as we learn more and do more, that we become increasingly flexible and innovative in our approaches.
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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