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  5. Opening Remarks, Prescription Drug User Fee Act Public Meeting November 14, 2005
  1. Prescription Drug User Fee Amendments

Opening Remarks, Prescription Drug User Fee Act Public Meeting November 14, 2005


COMMISSIONER VON ESCHENBACH: Good morning, and thank you Debbie. I want to begin by thanking you, and also Theresa Mullin for the tremendous effort and hard work in putting this very, very important meeting together.

And I also really appreciate and thank all of you. I want to thank you on behalf of not just the FDA, but the entire community for your interest and your effort and your commitment in coming here. I recognize how difficult it can be to get on the NIH campus. I just came from another meeting, and not only could they not get on the campus but, in their meeting room, they didn't even have any lights. So you're actually a little further ahead than some of the other groups meeting here this morning at NIH.

But it is not just your effort in coming here, it's the effort that you put in throughout the entire year to work collaboratively and collectively together to help us as a community enhance the health and welfare of the American people.

All of you here today play a very crucial role in helping the FDA fulfill our responsibility and part of that very important public health mission.

Clinical drug review is one of the backbones of FDA. And it's thanks to people who have supported our drug review programs that we are continuing to bring safe, effective and affordable products to American consumers; and that we continue in that mission to product and advance the health and welfare of Americans and, in fact, people around the entire world.

So it's a privilege to be with you and to join you for this important discussion about how we can better promote scientific innovation, and maximize the efficiency of the medical development process, especially through the reauthorization of the Prescription Drug User Fee Act.

This has never been a more critical goal than it is today. We are in the midst of a virtual revolution in biomedical research. But despite the tremendous progress of recent decades that's led us to this very exciting time, most medical experts still believe that the most exciting and most important innovations are still ahead of us. And so today we are spending more on biomedical research and development than ever before. We are discovering and learning and understanding about diseases at their very fundamental genetic, molecular and cellular level. And not only are we understanding the disease processes, but we're understand the person affected by those disease processes.

I have seen this research first-hand here at the NIH. And I, too, join all of the others who are extraordinarily optimistic about the future; so much so that four years ago, at NCI, when I became the director, we set a goal for the cancer program that we would capitalize upon this tremendous progress in biomedical research, and work to eliminate the suffering and death due to cancer, and bring that about by 2015.

But now, having had the privilege to serve as the FDA's Acting Commissioner, I can see that this research being translated into safe and effective new medicines is a critically important step if we're going to have that impact on the patient and the public we are dedicated to serve.

We are seeing opportunities emerge at the FDA as we are overseeing more and more investigational new drugs, and more innovative and targeted therapies than have ever before been possible or imagined.

PDUFA has, in fact, enabled a lot of this progress to be made available in a timely way to be able to use these opportunities created into interventions that could be delivered to patients in the form of safe and effective medicines; medicines that are approved by the FDA through a process that is more rigorous and more transparent, and more efficient than it has ever been before; and, in large part due to what the opportunities have been provided for by PDUFA.

PDUFA has enabled FDA to obtain the needed resources and adopt modern tools to review human drugs and to make much needed process improvements, such as management reforms and, most importantly, our opportunities in integrating modern information technologies.

But even with all these promising new treatments that patients have available as a result of improvements that PDUFA has enabled, it is still more important to do more. It is still obvious how much more work yet needs to get done.

And so at the same time that more innovative treatments are reaching patients there are, in fact, fewer new product applications reaching us than at any time in more than a decade. There is now appearing a fundamental disconnect between the tremendous potential that's been for 21st century biomedical technology, and the actual products that are reaching our patients in the future.

There is a disconnect between good ideas in the lab, and even proof-of-concepts, versus the safe and effective treatments that are actually being provided reliably to patients.

And so as we face a real challenge today to bring the promise of the 21st century medical breakthroughs to patients, our challenge for all of us is to find ways to make drugs and other new innovations both safe and affordable, and to continue to promote high levels of medical innovation that are then delivered to patients.

Our challenge is to continue to adapt our regulatory approaches to accommodate and accelerate the pace of biomedical innovation and the introduction of safe and effective medicines.

I believe that the reauthorization of PDUFA IV is critical and essential to enable a lot of these opportunities to be made possible. The discussion today marks the beginning of a public process to see how we can use new PDUFA legislation to continue to advance biomedical progress.

To meet this challenge effectively we need to do much more than ever before. We need to improve the process for developing safe and effective new medical products by making it as clear, as fast and as cost efficient as possible. And once new treatments have been developed, we need to continue to reduce the cost and improve the quality and reliability of making them--like we have done in other high tech industries--opportunities that are impacting patients' lives.

We also need to develop means of providing better and timelier information on the risks and benefits of medical treatments after they've been approved, so people can make fully informed treatment decisions. We need to empower doctors and patients to get the most value out of the medical products that are on the market.

All of this is the critical path; the critical path for therapeutic development that we have been talking about at FDA; the path for moving from scientific breakthroughs, backed by NIH and the many research foundations that are creating this opportunity and discovery, to then to actual development of treatments that make the most difference in patients' lives. This encompasses not just the science of developing drugs and devices, but also the know-how for turning an experimental pharmaceutical or biological molecule revealed in a test tube into an effective medical product that can be safely delivered to a patient. This include all the many steps that need to occur after a new compound has been removed from the laboratory and is placed into and through clinical trials to become a finally finished drug.

One of the major objectives at FDA is to clarify this critical path; simplify it; and help promising medical products navigate through it. To do so, we need to think about all 100 percent of the process, from the pre-clinical phase through the clinical trial period, through the eventual commercialization of the treatment.

How do we do this?

We need superior development science to translate basic discoveries into new and better medical treatments. We need to take the effort required to develop some better tools for developing medical technologies. And we need a knowledge base, built not just on ideas from biomedical research, but on reliable insights into the performance of drugs after they are approved. So we can continue to learn about risks and benefits in the post-market phase.

Enabling these safe and effective new medical technologies is a fundamental part of FDA's principal mission to protect and advance the public's health. And it is a fundamental part of the goals of PDUFA. At FDA, we are already developing needed information systems and working on collaborations with the National Science Foundation and the NIH, and other research groups, to help provide guidance and support for applied research studies that have the goal of providing the knowledge needed to make the development pathway more clear and more efficient. And we continue to improve our own processes for evaluating new drugs and to advance our commitment for enabling their development through meetings and feedback that we give to scientists every day.

I mentioned the word "collaboration." I mention it because it is a simple fact that we cannot do this alone. Even with all that we are doing, good medical ideas will not necessarily get translated into public health solutions without the collaborative, cooperative integration of all of our efforts; collaborations with our partners from drug-development companies and health care organizations, to academic institutions and, in fact, other government agencies, along with patient advocacy groups and consumers. All of these are key to making medical innovation a reality.

And collaboration is going to be a key in enabling us to gain the feedback that we need to make sure that PDUFA IV is as forward-looking and as smart as it needs to be in order to advance the development of biomedicine and drug development. I'm confident that we'll bridge the gabs in medical innovation and succeed in providing both safe and affordable products to Americans nationwide.

The opportunities that PDUFA enables are a big part--in fact, perhaps a crucial part--of achieving this promise.

And so today we begin a discussions; a discussion of how we can use this important legislation to move forward; to move forward to seize these opportunities on behalf of Americans. In doing so, we will realize the full public health benefits of the incredible innovations that are being made in biomedical research. And I trust we will not only fulfill but exceed the wonderful promise that many envisioned when they labeled this "the biomedical century."

I'm convinced that because of the work of FDA, and the work of this community, in helping to move opportunities like PDUFA forward to enhance and enable strategies like critical path, that you will make possible the fulfillment of a commitment and a goal just like the one we made at NCI to eliminate suffering and death due to cancer.

Our opportunities go far beyond just being able to eliminate the suffering and death due to cancer, as incredible as that one initiative may be. What is occurring at FDA, what PDUFA will make possible, and what the critical path will lead us to is not just a transformation of one disease, but a transformation of all diseases; an opportunity to improve the welfare and health of all people and all diseases. And your work and your participation and your cooperation and your contributions today are a critically important step. And I wish you well in the intensive work of today, and thank the panel and participants for your commitment and contributions. Thank you.


MS. HENDERSON: Thank you, Dr. von Eschenbach.

Our first panel will include presentations from the FDA. And these individuals will also serve as our listening panel for today. And I'd like to briefly introduce them. I will introduce them all before they speak.

Our first speaker, and second to my left, is Dr. Scott Gottlieb. Scott is the Deputy Commissioner for Medical and Scientific Affairs at the FDA. Immediately to my left is Dr. Janet Woodcock. Janet is the Deputy Commissioner for Operations, and the Chief Operating Officer at FDA.

To the left of Scott is Dr. Steven Galson, who is the Director of the Center for Drug Evaluation and Research. And to his left is Dr. Jesse Goodman, who is Director for our Center for Biologics Evaluation and Research.

We'll start with Dr. Gottlieb.

Panel I - Presentations by FDA

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