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Scott Gottlieb, M.D. - National Coalition for Cancer Survivors

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
National Coalition for Cancer Survivorship:
Industry Roundtable on Expanded Access

Remarks by
Scott Gottlieb, M.D.

Deputy Commissioner for Medical and Scientific Affairs

January 6, 2006

As we start the New Year, if you think about all the worthwhile things we might do together, it is hard to imagine that there are any more important undertakings than helping people overcome a serious illness.

Thanks to the dedication of scientists and doctors and especially patients -- thanks to many of you here today -- there are more reasons for thanks and hope than ever before when it comes to treating cancer.

From tests that precisely diagnose molecular signatures of different cancers to drugs that can target those signals, the translation of biomedical sciences into new and better practical treatments is already making a real difference in lives of people with cancer.

We have made so much progress in the last few decades that it is easy to take the pace of improvement we have seen in cancer treatment for granted. People with many forms of cancer, including previously fatal forms like the cancer that claimed the life of our President's little sister a half-century ago – many people who at one time would have died, today can and should expect a cure.

And today, the new sciences of genomics, and proteomics, and nanotechnology, as well as advances in our understanding of immunity and the molecular basis of disease – these sciences are just starting to have an impact on patient care. Because of this scientific progress, many experts now believe that our understanding of the mechanics of cancer are finally sufficiently advanced that we can envision transforming strides against the unconquered diseases.

We are already starting to see these scientific achievements show up in some recent FDA product approvals.

But it is time to more fully transform these hopes into a new reality of cancer treatment.

And I think many of us would agree that these scientific advances are not being turned into practical, safe and effective treatments that patients can use, at the pace that we would hope for, or even expect, given all of our investments in cancer research.

It is not too late to make sure that all of the recent scientific advances are able to be turned into practical medical solutions. In fact, I think this is a pivotal time to make changes today in our approach to drug development that will unlock a lot of this scientific potential.

I want to tell you about some of the things we are doing at FDA, under the leadership of Dr. Rick Pazdur, Dr. Steve Galson, Dr. Janet Woodcock and many others, to help make sure this progress is made available to patients who need access to new and better treatments.

And I also want to discuss with you my view of how we can do a better job of making promising new treatments available to the sickest patients, even before medicines are formally approved for marketing, by taking more effective steps to use our existing legal authorities for providing expanded access to experimental medicines.

Our efforts to improve the process for getting promising new medicines to cancer patients who need better treatments starts with the process for reviewing new drugs to make sure they are safe and effective.

Right now there are more than 400 drugs to treat cancer in development that are undergoing various stages of testing in people to show that they are effective and can be used without excessive toxicity. That's far more drugs in the pipeline than ever before.

And it appears that progress toward highly targeted drugs may increase significantly in the next several years, as there are more genomic investigational new drug applications being filed with the FDA than ever before, more novel immune therapies, and more drugs that we believe apply new science and target very novel and difficult-to-treat forms of cancer.

One measure of this is that the intensity of the work around many of the investigational new drug applications we receive has increased dramatically in recent years. We think this is a reflection of the fact that sponsors are developing more intricate molecules that are aimed at more difficult, unmet medical conditions.

If these new applications we're seeing are any harbinger of the future -- and I believe they are – then the drugs that we're likely to see are going to be even more targeted and effective, and perhaps less toxic than ever before.

And we're likely to have not only new and better drugs, but far better knowledge about how these drugs work. And which of the many available therapies and dosing schedules are actually the best choices for an individual patient, based in turn on a much more solid understanding of why that patient is sick.

But as I said, we're not seeing these new medicines and these new approaches to treatment being developed fast enough for the millions of patients suffering today from cancers that may be more treatable or even cured in just a few more years.

The greatest burden of this situation is borne by cancer patients and their families and friends as they struggle to survive and cope and pay the bills, even though better treatments could be available sooner and could be more affordable – if only the process for developing safe and effective new drugs were less costly and more certain.

For medical progress to continue and for it to be affordable, we need to find better ways today to develop new treatments -- to reduce the costs of finding and developing drugs, and to improve access to better medicines. That is the challenge.

And that is precisely what FDA has been working hard to address over the past year. And I am optimistic that we will make significant strides in these endeavors.

Part of the problem is that the process for developing drugs is becoming too long, too costly and too uncertain. The problem is that the development process itself, the process for turning new discoveries made in the laboratory into effective medical treatments that can be safely used by patients who need them, is not as advanced as the molecules that are being discovered.

So we have increasingly sophisticated drugs being evaluated by tools and approaches to drug development that have not advanced much if at all in many years. This is a problem that involves everyone inside the drug development process, but we believe that at FDA we can take a leadership role in helping to better develop the scientific tools and modern approaches to resolving development problems.

To deal with this challenge we recently announced a major new medical technology development initiative that we call our Critical Path Initiative. This initiative is aimed at ensuring we have regulatory processes that are as efficient and up-to-date as possible.

Among some of the things we're working on and will announce soon are a collaboration to develop and validate new biomarkers for diagnosing and treating certain cancers, and the development of better science around the use of imaging tools such as PET scans for tracking the progress of cancer treatments in diseases such as lymphoma.

We will be holding a workshop soon to look at endpoints in certain kinds of cancer. We have also undertaken new collaborations with the National Cancer Institute.

These are just some of the things we are doing. I'd like to take a moment to tell you more about these efforts.

Our challenges don't just relate to the scientific tools that we use to evaluate drugs as they are developed, but also the approaches that we take.

For example, the way in which we design clinical trials or the manner in which we test new drugs on patients.

This is especially true when it comes to cancer, where we are very sensitive to concerns about the shortage of patients willing to enter trials, or the need to sometimes require placebo trials, where patients are randomized to receive sugar pills in order to get the most rigorous, scientifically valid information about a new medicine.

Here again, there are alternatives to placebo trials that may allow drugs to be tested in different kinds of protocols that are more accommodating to patients' desires to get access to experimental medicines. But these kinds of alternative clinical trial designs also require us to improve the science for how we develop and evaluate drugs.

The kinds of opportunities we are exploring under our critical path initiative include many opportunities to study alternative clinical trial designs that may, in some cases, yield better information about the safety and effectiveness of new medicines.

These opportunities will be the subject of a critical path opportunities list that we will soon release.

To give you one example: right now, the usual approach to developing and testing drugs is very empiric, and by that I mean that testing new drugs is often guided first and foremost by statistics.

We test drugs on carefully controlled groups of patients to look for improvements or side effects that can be explained only by the new medicine. We look for overall response rates. We look at statistics. As doctors, we say things like forty percent of patients respond to a medicine.

But even at the end of an exhaustive and well-conducted clinical development plan, even after drugs are tested on thousands of patients, we often don't know which forty percent of all patients are most likely to respond, or why.

This approach was all we could do when we did not really understand how most new drugs worked.

But today, more and more of the drugs that are being developed are based on a clear understanding of the molecular basis of cancer, where the mechanism of the disease and the drug is clearly understood.

And so as one part of our Critical Path Initiative, we are looking at tools and clinical trial designs that can enable us to take a more mechanistic approach to testing and evaluating new medicines.

It behooves us to see if there are better approaches to clinical development and clinical trials that allow us to leverage this information to learn more about why drugs work, and why they sometimes don't during clinical development.

So that medicine is not drive by an empiric approach to statistics – so that as a doctor I don't have to settle for the fact that half my patients or more won't respond to a medicine I prescribe. But we can know more about which people will respond, and do more to get the right treatment to the right patient at the right time.

I would like to close by talking a little more specifically about the issue we are gathered to discuss today - expanded access to unapproved drugs.

The current FDA programs I have discussed here today, the initiatives to improve the science of cancer drug development, are tied closely to our efforts to improve access to promising therapies.

We still believe that participation in clinical trials is the best way for patients to get access to unapproved drugs. But often, patients who are out of treatment options are not eligible for clinical trials.

We also believe that working with the academic community, the NIH, and drug developers to shorten drug, biologic and medical device development time will mean patients will have access to these products sooner. Development time is different than FDA review time, and this is an important distinction. Because most of the decade of more that a promising drug will spend in development is spent being tested, sometimes by needlessly outdated and expensive and inefficient approaches by sponsors.

Today, with the help of the dedicated staff in FDA's cancer program, we are reviewing and approving drugs in time periods that are as fast, or faster, than any country in the world with a comparable system of protecting human subjects. We are doing so while maintaining our longstanding standards for safety and effectiveness.

But even with all of these steps to improve the process for developing new cancer drugs, and reviewing them inside FDA, that doesn't directly address the needs of patients who are dying from their disease, and want access to promising experimental medicines as a last chance of quelling and even potentially reversing their disease.

This is the problem you gathered here today to discuss, and I look forward to the presentations that are scheduled for the morning. I hope we can discuss how we can take new steps to use our existing regulatory authorities, and perhaps consider new ones, in order to make promising drugs accessible to patients who are out of useful options, even before these drugs are approved for marketing.

Right now, we are working on finalizing two new rules that we believe 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. This includes uses for which charging was not previously expressly permitted.

The agency is also proposing changes to the provisions describing the types of costs that can be recovered when charging for an investigational drug.

The proposed rule is intended to permit charging for a broader range of investigational uses than is explicitly permitted in current regulations. It is also intended to provide greater specificity concerning what costs a sponsor can recover when it charges for an investigational drug.

The second new rule would allow FDA to amend its regulations on investigational new drug applications to describe the ways 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 intended to improve access to investigational drugs for patients with serious or life-threatening diseases or conditions who lack other therapeutic options and may benefit from such therapies.

These two rules will help advance our ability to more effectively make promising medicines available to patients who are out of good options. But there is still a lot that we want to do to improve access to experimental medicines under our expanded access programs.

A lot of our ability to do this depends on our capacity to continue to work on all of the better approaches to drug development I mentioned. How we improve the process for testing drugs as part of the drug development process.

I want to begin by saying that this starts with, as Dr. Pazdur has said on a number of occasions, separating the concept of access to a drug from the concept of approval.

It begins by acknowledging that we can have broader access to promising medicines, after they have demonstrated activity against cancer, for patients who are dying of cancer and who out of options and want access to an unapproved drug.

Right now, if a drug company agrees to allow a patient to have access to their unapproved drug, the FDA will review the patient's request after it is submitted by the drug company. If there are no safety concerns that might jeopardize the patient and the patient has no proven treatment options availabe to them, FDA will usually agree to allow the patient to have the drug.

It is important to remember that FDA has important responsibilities when experimental drugs are made available to patients as part of an expanded access program.

First and foremost, we have a responsibility to make sure that patients and their doctors are fully informed about whatever we know about the benefits and risks of any medicine, especially one that is still unapproved.

Patients who pursue experimental medicines, out of a sense of hope, and/or their desire to be altruistic, deserve to know that we have taken every step possible to make sure they are informed of everything we know before they consent to take the unapproved drug.

We also have a responsibility to try and do everything we can to take every possible opportunity to learn more about the benefits and risks of a medicine, and this is especially true in areas of rare diseases or great medical need, such as many cancers where large trials are not always possible or desired because they can slow down the development process.

This perhaps means trying to do more to learn something about an experimental medicine through each and every patient encounter. Because our ability to effectively use new medicines after they are approved is only as reliable as the information we have about a new drugs safety and effectiveness. And that information is only as rigorous and detailed as what the development process enables.

Cancer doctors are more engaged in clinical research than perhaps any other medical field. We can depend on them to develop important information about new drugs after they are approved, through the vast and collaborative research enterprise that the cancer field maintains. But much of the information doctors use to guide prescribing decisions still comes from the development process -- from clinical trials and the information collected when drugs are tested for the first time.

I believe this means that if we're going to test drugs on small populations of patients in order to keep the clinical development process efficient, we also need to take every opportunity to learn everything possible about new medicines, to help doctors make more effective use of them after they are approved.

That gets me to the expanded access programs, and the question of whether we can or should be collecting more information about their effectiveness, in addition to the information we already collect under any IND process, even as we provide broad access to new drugs pre- or per-approval. Can we achieve both of these goals?

I know this is a very difficult question, and I hope we can discuss it here today. There has sometimes been a debate in the larger community about whether we can collect information about a drug in development from expanded access programs while not undermining the incentive or the ability of sponsors to create these programs.

But with the growing availability of very simple data collection mechanisms, like web-based interfaces, and the use of simple registries for data collection to answer some important questions, I think we can learn incremental information about the safety and benefits of these new medicines while we provide broader access to promising unapproved drugs.

I believe doing so can provide an important addition to the traditional development plan. If we decide that collecting data from expanded access programs is useful, we could possibly answer certain questions that are not essential to the approval decision but nonetheless important to clinical practice or to patients. Or perhaps these kinds of large, simple data collection programs or registries can be used to establish an interconnected network of patients and doctors that can be maintained after approval and perhaps satisfy some of the post-marketing follow-up commitments that sponsors are sometimes asked to fulfill.

In short, I think there are a lot of opportunities to use new tools, new ideas, and new approaches, to create broader access to promising medicines while still fulfilling our commitment to patients and doctors to take every opportunity to learn about how new treatments work, and every opportunity to provide patients with as much information as possible to guide their choices.

And I trust we will use the occasion of this important workshop to think creatively about how we can use all of our existing authorities, resources, and ideas to make the most of these opportunities -- to simultaneously fulfill our commitments to patients and to science.

There are many things that we're going to be doing over the next months to work on ways to help accelerate and improve the process for developing safe and effective new cancer treatments, and to encourage product developers to continue to take risks in seek of better cures.

And there are still many more things we need to do in order to help all Americans get the best care possible.

We must help those with needs beyond their means, and we must create a healthy environment for providing high-quality care today and ever-improving care for the future.

We're at a point where we have more knowledge and resources than ever before to make a real difference in the lives of many patients with cancer. And we need to work together to make sure that we use these resources to maximum advantage for patients.