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Moving New Drugs from Discovery to Delivery

Remarks as Delivered by Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
Washington, DC
November 10, 2011

I first want to thank Ellen Sigal and Mark McClellan for all your work over the years to help support the FDA, to ensure progress in science and innovation, and most importantly, to help bring health and hope to cancer patients and their families. 

Additionally, I’m very grateful to the Friends of Cancer Research…and The Engelberg Center for Health Care Reform at the Brookings Institute…for co-hosting this always anticipated conference. This is an open forum for collaboration that is truly unparalleled in the cancer community…and we need more opportunities for the kind of discussion—followed by action--that this forum so successfully provides.  

Finally, I want to thank all of you here today—from academia, industry, government, the non-profit community…from the halls of science, health care clinics and the corridors of industry—for your dedication to translating discovery into delivery for oncology drugs.  All of you are working to open new windows on treatment and new doors to prevention…and to help ensure the day when cancer statistics will only count survivors.

Being here with so many people who are dedicated to moving cancer treatments and therapies from scientific bench to patient bedside, I’m reminded that it was exactly 65 years ago—this year—that the modern era of cancer treatment was born. 

In 1946, two Yale researchers, Louis Goodman and Alfred Gilman, published the original paper that demonstrated that nitrogen mustard—a substance previously used for chemical warfare--could induce tumor regression.

The first step in the long road of treating cancer with pharmacological agents, it was based on some very innovative methods, and it was a groundbreaking discovery.  Unfortunately, we know that discovery isn’t enough. 

Discovery alone can’t confront cancer…or ease suffering…or extend life.  For that, we must translate those discoveries into real world products…and we must ensure the delivery of those products—those promising treatments and therapies—to patients, as safely and swiftly as possible.

That is what all of us want.   It’s what patients need and it is what they expect. And it’s what the FDA is working and striving for every day. 

I’m proud to say that in Fiscal Year 2011—as we just highlighted last week in a new report—we approved 35 novel medicines—many of which were groundbreaking…and all represent real medical advances for patients and for health care. Seven of these new medications represent important new opportunities in the realm of cancer.

We approved two major drugs for late-stage metastatic melanoma, the most dangerous type of skin cancer.  Yervoy, is the very first drug ever shown to be effective in extending the lives of patients with metastatic melanoma.  And Zelabrof was simultaneously approved with a first-of-its-kind companion diagnostic test—BRAF V600E—which will determine if a particular patient will respond to the new medication. 

Additionally, we approved the first drug to treat Hodgkins lymphoma in 30 years, as well as new drugs for late-stage prostate cancer; thyroid cancer; metastatic breast cancer; and late stage lung cancer.

And not only are we bringing new drugs to market, we’re doing it quickly and efficiently…and as fast as or faster than other countries…while still maintaining a gold standard for drug safety and effectiveness.

FDA approved nearly 70 percent of these 35 new drugs before any other regulatory agency in the world.  This reinforces the findings of the recent Friends of Cancer Research study published in the journal Health Affairs, which compared the FDA and the European Medicines Agency oncology approvals between 2003 and 2010.  It found that of the 23 cancer drugs approved by both agencies, all 23 were approved first in the U.S.

However, even with these impressive outcomes, we recognize that uncertainty in the regulatory approval process can sometimes impede the delivery of new medical products.

We have been working hard to improve the transparency, consistency and predictability of our regulatory pathways, as well as to provide increased guidance to industry about our standards, expectations and approaches.

In this regard, I’m extremely pleased to announce that the FDA has finalized its guidance on therapeutic cancer vaccines.  The guidance provides extensive, comprehensive advice—and it should enhance certainty for developers--regarding the FDA's expectations for clinical development of therapeutic cancer vaccines.

As you know, there’s been increasing interest in such vaccines in the last few years. We are also finalizing another relevant guidance on streamlining data collection in late stage clinical trials and post market studies; an effort that very much reflects the work of this group. This guidance should be published soon. We hope that these new guidances will help contribute to the pipeline of new treatments.

This is a critical time to address the overall robustness of our pipeline. I suspect that we all agree that we must accelerate the successful translation of research into treatments and therapies…and to do so I think we must work together on several overlapping challenges. These are challenges which are directly inspired by the birth of the modern era of cancer treatment. And they are the challenges of innovation, regulation, and partnership. Let me say a little about each.

The first challenge is that—like Goodman and Gilman—we must endeavor to devise new, and better—and more innovative—ways of doing things in order to provide novel treatments and therapies.

In fact, developing innovative approaches has always been a cornerstone of our work at the FDA. One hundred years ago in the early days of the Agency, Burton J. Howard, a chief of the Bureau of Chemistry's microchemical laboratory, needed a way to prove his suspicion that worm-eaten refuse was regularly found in cans of ketchup. Using a standard blood-counting cell and a compound microscope, he developed the “Howard Mold Count.”

Although it was simple and straightforward, it was a new and innovative way of doing things.  A century later, we’re still striving to find new and innovative ways of doing things.

We’ve been working on novel ways to expedite the development and delivery of new drugs.  Our drug approval pathways now include Fast Track…Accelerated Approval… Priority Review…and Expanded Access programs.  These programs are designed to speed the testing, availability, and approval of drugs in different ways.  I think it is notable that almost half of the 35 new drugs approved this past fiscal year were approved under “priority review”—where the FDA has a six month goal to complete its review for safety and effectiveness.

We’ve also been showing increased flexibility when it comes to clinical trials.  Adaptive clinical trial designs like the I-SPY study is one great example. And again, if you look at the list of new drug approvals just released, you can see clinical requirements were streamlined to permit smaller, shorter or fewer studies wherever possible. Many have been surprised to see the number of drugs approved on the basis of single arm studies or using studies with extremely small patient populations.

In October, we released our Innovation Report, and creating an expedited drug development pathway for important targeted therapies is one of its major areas of focus.

We know that sometimes, during the development of a new treatment for a serious or life threatening disease that has few therapeutic options, a new treatment option appears to perform better in early clinical trials than the “standard of care.”   There’s agreement that such a drug should be developed more quickly.  But there’s no consensus on how to simultaneously speed-up development and safeguard patients.

In response, the FDA is working on two immediate and related steps toward expedited drug development. First, we’re developing a draft guidance on enrichment strategies for clinical trial development. 

Second, as an example of an expedited pathway, the Center for Drug Evaluation and Research will publish a draft guidance on the use of pathologic complete response–when no clinical evidence of disease remains—as a surrogate endpoint for accelerated approval in primary high-risk breast cancer. This guidance will outline a relatively seamless pathway that could be followed from a multi-drug screening trial to an accelerated approval.

But much of our ability to modernize and streamline our regulatory pathways—and to increase regulatory certainty--  depends on strengthening the science base that must be the foundation of all our work.

That’s also our second challenge: To make advances in regulatory science—the science that underpins drug evaluation and approval—a top priority.  Last year, I focused my remarks at this Conference on the importance of regulatory science…on why it matters…on why we must invest in regulatory science to bridge the gap between discovery and delivery.  Today, I want to talk a little about what we’re specifically doing to advance and strengthen regulatory science.

This has been a top priority  of mine since becoming Commissioner. And in these efforts, I have been grateful for the guidance and support of many of you in this room, and most certainly Ellen and Friends of Cancer Research and Mark in his many roles.

  Through our “Advancing Regulatory Science Initiative,” we have been attempting to define some of the important opportunities before us, and we are devoting time and resources in an effort to ensure that badly needed investments are made in regulatory science.

 For example, last year, the FDA and the National Institutes of Health launched a new NIH-FDA Council and a Regulatory Science Initiative to encourage research in this field.  We awarded our first set of grants in regulatory science and we hope to be able to expand this program soon.

We have tried to develop and strengthen productive collaborations, such as The Biomarkers Consortium. It’s an innovative public-private partnership that has proven its value in helping to identify and characterize new biological markers for regulatory use.

We are optimistic that we will be able to catalyze more such opportunities as the Reagan-Udall Foundation, a nonprofit organization, created to support the mission of the FDA, is now getting up and running under the able leadership of Mark McClellan, Ellen Sigal and others

Although resources have been more limited than hoped, we have been able to establish a few Centers of Excellence in Regulatory Science. Over the summer we inaugurated one Center in Arkansas focused on nanotechnology, involving FDA’s National Center for Toxicology Research, several academic institutions in the State and the Governor’s office. Last month we announced two new regional Centers for regulatory Science and Innovation, one at Georgetown University and the other at the University of Maryland.

Additionally, our Critical Path Initiative—begun with the vision and leadership of our CDER Director, Dr Janet Woodcock-- has created a nationwide network of scientists who search for and test ways to streamline drug development and review.

As we work to accelerate innovation and strengthen regulatory science, it is increasingly clear that our most effective strategies are grounded in partnership. 

I think we also recognize that this must include an expanded effort to increase our partnership with patients and advocacy groups to ensure that patient needs are being met. Strikingly, our drug user fee negotiations with industry included a new patient-centered drug development focus in the drafting of our PDUFA V plan. Under the proposed agreement, the FDA will develop a five year plan to further develop and implement a structured “benefit-risk assessment” in the new drug approval process.  And over the period of PDUFA V, we will initiate a public process to nominate a set of disease areas—20 in all—that could benefit from a more systematic and expansive approach to obtaining the patient perspective on disease severity or unmet medical need. 

We’ll begin by inviting public comment on what diseases we should focus on…on which therapies patients think are needed…and on where we need more information and on how we should be thinking about risks and benefits. We then plan on holding four public meetings a year—for five years—with each meeting focused on a different disease area.

The meetings will include participation of FDA review divisions, the relevant patient advocacy community, and other interested stakeholders. After each meeting, we’ll publish an analysis of the input that is relevant to our consideration of disease severity and unmet medical need. 

This is just part of the process of continuing to listen and learn…and ensuring that we always engage in a true dialogue.

These are difficult, often fractious times, but together, we must make sure that we remain focused on the things that really matter…the things that will make a real and enduring difference. Frankly, this is one of the real strengths of this Conference on Cancer Clinical Research—now in its 4th year. Bringing together the right people around the right topics, you have focused and you have made a difference.

It is sobering to realize that next month will mark forty years since we witnessed a milestone in the fight against cancer with the signing of the National Cancer Act…and the beginning of our national effort to defeat cancer. In the long years that have followed, we’ve won many battles…but the struggle is far from over. 

Of course, we’ll never be able to declare victory without meeting the overlapping challenges I’ve just touched on: Accelerating innovation…strengthening regulatory science…and continuing to work as partners. 

But if we meet those challenges, we’ll be able to better ensure the translation of discovery into delivery…and to hasten the day when we may be able to remove cancer from the headlines and consign it to the history books. 

So I will stop talking and let you get back to your important work.

Thank you for your time, your attention and all of the important contributions you are making.