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Scott Gottlieb, MD - Annual Cancer Progress Conference

This text contains Dr. Gottlieb's prepared remarks. It should be used with the understanding that some material may have been added or deleted during actual delivery.

Speech before
Seventeenth Annual Cancer Progress Conference,
New York, New York

Remarks by
Scott Gottlieb, MD

Deputy Commissioner for Medical and Scientific Affairs
Food and Drug Administration

March 07, 2006

Thank you for inviting me to speak today. It’s an honor to be here with so many people who are working on the front lines of medicine -- working to uncover the basic discoveries that will enable development of new and effective treatments for cancer, and those here today working to help support that research. Today, I first want to thank you for these efforts and for the collaborations we have with many of you. And I want to talk about how to build on it, about some additional steps I believe we need to take together to improve the process for developing new medical treatments and for turning sound scientific ideas into practical solutions for cancer patients.

I think we all agree that biomedical science has more to offer than at any other period in history. New sciences like genomics and proteomics might allow us to tailor treatments to be highly effective against the underlying causes of specific diseases in each individual patient – and perhaps even to prevent those diseases from occurring or progressing in the first place. New engineering applications of nanotechnology that can get the right treatment to the right place in each individual with far fewer side effects and complications than in the past. And next-generation information technologies, to turn the explosion of health information into far greater useful knowledge at the fingertips of doctors and patients, so people can make better informed, more effective decisions to improve their health than is possible today.

But all of these things are by no means a sure thing. I believe that whether or not these things come to pass will depend in large part on what happens in our institutions, our labs, and our offices. That’s because more than ever before, we need to share our expertise and work together on finding common-sense practical solutions, of plowing new roads to reach goals that seemed improbable. We need new and better ways to help people make sense of all of the information out there that may be relevant to improving their own health. We need to make safe and effective medical innovations more quickly available to the patients who need them. And we especially need to be concerned about keeping health care and especially cancer care affordable and accessible while embracing the incentives that encourage all of this biomedical progress and innovation in the first place.

Don’t get me wrong, we’re already making a lot of progress in biomedical science. Right now there are more than 400 drugs to treat cancer in development. And it appears that progress toward highly targeted drugs may increase significantly in the next several years, as there are more targeted and genomically-derived investigational new drug applications being filed with the FDA than ever before. In fact, in 2005, FDA received and reviewed 956 INDs for cancer products, 149 from commercial sponsors; 489 from research organizations; while 318 were for single patient use. We approved seven New Drug Applications (NDAs) for oncology or oncology related indications, and of these four were NDA approvals of priority new molecular entities (NMEs). In addition, the office approved twenty-four efficacy supplements -- nineteen NDA supplements and five biologic license applications (BLA) supplements. And we’re expecting to receive even more applications for new cancer drugs in 2006.

If these applications are any harbinger, thanks to the explosion in the discovery of new treatments, the medicines that we may see in the years ahead have the potential to be far more targeted and perhaps less toxic than ever before.
They have the potential to slow or even prevent clinically important cancers far more effectively than in the past. These are all encouraging signs that the prospects for practical benefits from medical innovation are starting to improve.

But it is continuing to get more expensive and more uncertain to transform new scientific discoveries into treatments. And so we face some challenges along the way. In contrast to many other fields of applied science, when it comes to drug development, we have not made so much progress in improving the process of developing and refining good ideas into products and services that the public can use. The plain truth is that many of the most dramatic scientific advances that we’ve recently made in the labs haven’t yet transformed medical treatments. In 2002, at around the same time that we were declaring victory in sequencing all of the human genome, the number of applications and approvals for truly new drugs at the FDA was reaching a 20-year low. Meanwhile, the National Institutes of Health estimates the overall cost for cancer was $156.7 billion in the year 2001. That’s an astonishing figure -- larger than the gross domestic products of all but a few nations. And according to the consulting firm McKinsey and Company, the cancer bill worldwide could triple by the year 2010.

Because the cost and uncertainty of medical innovation means rising costs and access problems for health care, from a public health standpoint, this just isn’t an acceptable situation. To achieve the goals of biomedical innovation and affordable health care, we are going to have to work together to find new and better ways to turn scientific discoveries into practical medical solutions that can be safely used by patients. That means finding better ways to develop new technologies that can allow us to bring new medical products to patients faster, and more safely, and at a lower cost.

But right now, the opposite is happening, and that should be a concern to all of us. Right now, drug development is becoming even less predictable than in the past, with attrition rates going up, and the number of new products making it through going down, while the cost of developing new drugs, and the barriers to bring new drugs to the market, continue to increase significantly. The plain truth is that a lot of the new technologies we’ve developed over the last five years – proteomics, genomics, microarrays – have only added to the cost of discovery and development without making the process any faster or any more certain. No wonder that health care costs are going up, and no wonder that we haven’t yet fulfilled the promise of what should be the biomedical century. No wonder medicine is getting so expensive, and no wonder that, despite all of the biomedical promise and all of these new sciences, patients aren’t yet seeing much benefit from them.

So we have some great opportunities but also some important challenges to face together. And I think that the innovators and scientific institutions can play a critical role in determining just what kind of a century this will be. We need to work more closely together to address all of the steps that encompass the critical path for therapeutic development – for moving from the scientific breakthroughs backed by your institutions and private research to actual treatments that make a real difference in patients’ lives. This encompasses not just the science of developing drugs and devices and other medical products, but also the know-how for turning an experimental medicine into a finished product that can be used safely and effectively.

To solve these challenges, we need better research into new clinical trial designs such as enriched trials, which may allow us to learn more about new drugs in shorter periods of time. We need better methods for demonstrating early in the development process that new drugs can be easily absorbed, aren’t toxic to the liver or the kidney, or can be manufactured consistently and in sufficient quantities. And we need better research into validated biomarkers that could be used as endpoints in clinical trials or could be coupled with diagnostic tests to help guide treatment in the post-market.

At FDA, we’re already doing a lot to address this critical path – to make it less costly, and more certain, especially when it comes to new safe and effective cancer products. FDA has launched a series of new initiatives on improving medical innovation. These initiatives have several elements.

One element of our initiatives for improving medical innovation is the development of “quality systems” for our review procedures. The idea is to build on our professional staff expertise to identify and apply best management practices internally to our review processes. We want to make sure we are doing all we can not only to continue to reduce the time it takes to review new treatments, but also to help product developers get their applications right the first time – to do the R&D work that demonstrates safety and effectiveness as quickly and efficiently as possible.

FDA is also working on new guidance documents that I believe can bring more efficiency and predictability and modern approaches to the process of proving that new treatments are safe and effective. We’re doing this with collaboration and new support from outside experts, including support from the National Institutes of Health. The idea is to bring together experts in government, in academics, and in the private sector to help lay out the pathway for developing new treatments – to make sure that the clinical endpoints for studies, the study designs themselves, the whole clinical development pathway is as clear as possible to the scientists and clinicians who have a good idea.

One of these guidance documents will focus specifically on developing the information to validate a drug and a corresponding diagnostic test within the same registration trial. Under the leadership of Dr. Rick Pazdur and the dedicated professional staff of the Office of Oncology Products, we’re also developing other guidances, as part of an overall effort to improve the process for developing safe and effective cancer products. And over the next year we have more workshops already scheduled to explore other important developing issues, including clinical trial endpoints in ovarian cancer, and one on accelerating anticancer agent development and validation.

A second element of this broad initiative is to work to modernize the science involved in the drug development process itself. As part of our critical path initiative, led by Deputy Commissioner Dr. Janet Woodcock, we are working to define places in the drug development process where we more modern science can result in better approaches to answering important questions about the safety and efficacy of drugs. As part of our critical path initiative, we recently announced collaboration with the NCI and CMS and the Lymphoma Society to undertake a study to validate the use of FDG-PET as a tool for measuring tumor response in lymphoma drug trials. We also recently unveiled a broad collaboration with the NCI on cancer biomarkers. We’ll soon be releasing our critical path opportunities list, which will identify other areas where we believe broader collaboration around identifying more modern scientific approaches to drug development can improve the process. Our list will pay particular focus to new biomarkers and tools for measuring safety and efficacy, as well as more adaptive clinical trial designs.

A third element is to improve our use of modern information tools to help make sure we’re evaluating and developing information as efficiently as possible. Now that modern information technology has had a fundamental impact on so many parts of our lives, there's also no good reason we can't have a fundamentally better system for helping to protect consumers and assure the safe and effective use of new drugs. There’s no reason we can’t have a much more effective system for monitoring the way new drugs are used in order to more easily and quickly spot potential side effects and to provide appropriate warnings to doctors and patients. There’s no reason we can’t more easily continue learning more about new drugs after they’re approved, without having to start a long and expensive trial every time we have a question to answer about a new drugs safety or its effectiveness. And there’s no reason that we can’t continue to provide people with up-to-date information about how to derive the most benefit from the medical products they use.

To these ends, we’re implementing a new system in partnership with the NCI to take better advantage of information technology in the clinical trials process. Our new system for submitting electronic INDs is part of a much larger effort – the "Cancer Biomedical Informatics Grid" project, developed under the leadership of FDA’s Acting Commissioner Dr. Andrew von Eschenbach. For one thing, it allows us to review these applications faster and hopefully get new treatments to patients more quickly, and at a lower cost. Because the submission is quite straightforward, it could make filing an eIND could be as simple as sending a secure e-mail. In order to make sure we’re making the best use of the information that we generate during clinical trials, it’s important that we make as much of this information as possible electronic so that it can be more easily queried, evaluated, and archived.

We believe the NCI can be especially helpful in helping us continue to move toward this electronic system when it comes to tracking the information that’s developed by clinical trials -- in part because the universe of cancer doctors and the NCI’s cancer cooperative groups are among the most technologically proficient. Many of the bottlenecks in clinical trials occur because of delays in translating information from the bedside to the drug developers and eventually to the FDA. In many cases, doctors working in the NCI’s cooperative groups are already entering this information electronically. Their experience proves that this kind of electronic information makes the process not only faster, but also better because more of the important information can be made easily available and queried by drug developers and reviewers here at the FDA.

Having more of this information available electronically will allow clinical trials that today would take many months and cost many millions of dollars to be done far more quickly and easily. For example, instead of requiring researchers to cull through patient medical files every time new questions arise about a new treatment – all in order to piece together fragments of information scribbled inside paper medical records -- if more of this information was available electronically these queries could be accomplished at minimal expense and in a matter of minutes.

And finally, based on this very promising collaboration, we also believe that the NCI can be especially helpful to us for post market trials, when drugs continue to be tested after they make it onto the market. Since many new cancer drugs are approved under accelerated programs -- based on their effect on a surrogate marker such as their ability to shrink tumors on x-ray scans -- it is also important to continue to test these drugs after they are approved to make sure that the benefits that were observed in the clinical development process actually translate into meaningful gains in comfort and life expectancy for patients. One of the quality systems we will be developing will specifically focus on the design and implementation of post market studies, to make sure that FDA is working closely with sponsors to design optimal and practical studies that answer the most important clinical questions and can be brought to timely completion.

We need to do all these things to make the process for bringing new medical innovations to patients more timely, less expensive, and fraught with fewer risks. We need to do these things because right now, while the potential for scientific breakthroughs is greater than ever, we haven’t yet succeeded as well in turning the recent basic science discoveries into safe and effective new treatments for the patients. Until we do, unless we do, too many people will continue to suffer.

In addition to working to reduce the cost and improve the efficiency of bringing better medical treatments to patients, we also need to work to help prevent cancer and other diseases by helping people make positive lifestyle changes, particularly diet changes, and even prevent the onset of cancer.

Toward these same ends, we also need a better system for evaluating new treatments aimed at chemoprevention, especially for people who are at high risk but who have not yet developed the disease. We can’t wait many years and enroll thousands of patients in very large and expensive clinical trials each time we want to evaluate a new preventative treatment.

To these ends, we will be sponsoring a workshop with DIA to look specifically at the appropriate clinical trial criteria for pursuing prevention-oriented claims on drug labels. We’ll also be addressing this issue through the development of some specific guidance documents. One such guidance document that we plan to undertake will focus on the clinical evaluation of agents to lower the risk of developing sporadic colorectal adenomas. We also need better ways to make use of good epidemiological data. We need to find better clinical endpoints and trial designs that are just as scientifically rigorous as our current practices, but allow us to determine whether a drug can lower the incidence of a certain cancer without waiting many years every time. And we need to have a clear way to more easily determine whether the benefits of these new preventative treatments outweigh their risks. This is going to require all of us to work together on developing the critical path for evaluating these kinds of preventative treatments and for making these determinations.

Finally, as the potential for new cancer treatments continues to improve, with more targeted and less toxic medicines going into development than ever before, we need to make sure we have robust and responsible approaches to making potentially lifesaving medicines available to patients who are out of therapeutic options, even before these products have undergone full FDA approval.

Our Office of Special Health Opportunities, led by Terry Toigo, works closely with patient groups and individual patients to make sure the opportunities for expanded access are as clear and accessible as possible. Dr. Pazdur and his team have also worked hard over recent years to continue to improve our own internal process, including approving many individual INDs for patients seeking access to unapproved cancer drugs.

Right now, we are also working on finalizing two new rules that we believe are an important step that will help enable more promising new medicines to be made available to cancer patients through our existing expanded access programs.

The first rule would better describe the types of investigational uses for which a sponsor may be able to charge for a drug offered as part of an expanded access program and the types of costs that can be recovered. The proposed rule is intended to permit charging for a broader range of investigational uses than is explicitly permitted in current regulations. The goal is to help encourage more sponsors to make drugs available under investigational uses, especially in cases where costs may be an obstacle to doing so.

The second new proposed rule would allow FDA to amend its regulations on investigational new drug applications to describe the ways in which patients may obtain investigational drugs for treatment use. Under the proposal, treatment use of investigational drugs would be available to individual patients, including in emergencies; intermediate size patient populations; and larger populations under a treatment protocol or IND. The proposed rule is aimed at improving access to investigational drugs for patients with serious or life-threatening diseases or people suffering from conditions that lack other therapeutic options, where they may benefit from investigational therapies.

These two rules have been the result of a lot of public discussion and have benefited from the feedback we have received from patient groups. They will help advance our ability to more effectively make promising medicines available to patients out of good therapeutic options, but these rules are just one step, and there is still a lot that we want to do to improve access to experimental medicines under our expanded access programs.

Over the coming months, at FDA we will be working on other new ideas for the best ways to work with all of these new programs I discussed here today, to address the barriers on the critical path to better medicines for patients. We hope to work with you on these new efforts and learn from your expertise. Thank you.