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Friends of Cancer Research Brookings, 2013 Conference on Clinical Cancer Research

Remarks by Margaret Hamburg, MD
Commissioner of Food and Drugs
2013 Conference on Clinical Cancer Research
Friends of Cancer Research Brookings/Engelberg Center for Health Care Reform
Washington, DC
November 7, 2013

Good afternoon. Thank you Ellen for that kind introduction. I also want to recognize Mark McClellan, one of my most distinguished predecessors as FDA commissioner. Both of you are valued friends and colleagues.

The two of you have played such an important role in building this conference, supporting development of innovative strategies to study and treat cancer, helping to turn the complex process of scientific discovery into the delivery of tangible solutions, and in forging a strong and supportive ongoing relationship with FDA and other government agencies.

I am delighted to be with you once again for your annual conference, which offers such an invaluable opportunity to join so many leading experts in the field – providers, patients, researchers, advocates, industry representatives, and government representatives -- for serious and productive conversations about cutting edge issues in cancer research.

For six years these gatherings have provided a key forum to discuss new pathways, frameworks and strategies.  Truly, this conference, and of course the day-to-day work undertaken by Friends of Cancer Research, plays such an essential role in the world of cancer research, and most importantly, in addressing the vital needs of patients. And these efforts also underscore the fundamental value and demonstrated success of public-private partnerships—across sectors and across disciplines-- to advance cancer research.

In a field that’s too often limited by silos, the Friends-Brookings Conference is one of the best venues for open, public collaboration on behalf of patients everywhere. This conference is really a model for how all parties can come together, and it is a forum that we at the FDA so appreciate and utilize for input, problem solving and guidance.
And by bringing together experts from so many sectors, you ensure that ideas that originate at the Friends-Brookings Conference don’t end up lost in transcripts and white papers—they go on to profoundly impact the way we approach cancer treatment and care.
It is interesting to note that it was in this week back in 1937 –just over 75 years ago--that the National Advisory Cancer Council held its first meeting here in Washington. The Council was authorized that year under the same law that created the National Cancer Institute. I was actually surprised to learn that it was NCI which pioneered the federal funding of biomedical research for academic investigators, and that vision for cooperation across sectors in support of cancer research was a good one.

Looking around us here today, it’s exciting to see how far we’ve come with these kinds of support and collaboration.  And we need to continue to bring new vision—and resources—to advance this important work.

Certainly this is a wonderful opportunity to recognize and applaud the vision and action that this Brookings/Friends of Cancer Research-led collaboration has brought to the field of cancer research. And I’m particularly delighted that FDA is a partner. 

To this end, I want to recognize several of my FDA colleagues who are participating in today’s conference. In just the last session you heard from Dr. Janet Woodcock, who provides such excellent and forward-looking leadership of our Center for Drug Evaluation and Research (CDER).  And earlier this morning, the first panel on Immunotherapies included CDER’s Lead Medical Officer, Amy McKee, as well as Celia Witten, FDA’s Director of Cellular, Tissue, and Gene Therapies in the Center for Biologics Evaluation and Research (CBER). This afternoon’s panel will include Rick Pazdur, the director of our Office of Hematology and Oncology Products within CDER, who I suspect is very well known to all of you here. And of course I want to pay tribute to many other hard-working FDA scientists here today who are playing an important role in the work this group is doing.

I draw attention to these officials not to minimize the impressive array of other top experts participating here today, but to highlight the strong and continuing collaboration and cooperation between FDA, the Friends of Cancer Research, and the many other stakeholders. Our work together centers on achieving a balance that allows us to promote smart regulation that encourages innovation and leads to the best possible treatment and care options for patients by employing the best available science and drawing on the best available minds.

The partnerships formed here have led to the development of a number of opportunities for advances in research, and in streamlining regulatory systems so that research can be translated into viable products in a reasonable time. In 2011, this conference proposed innovative ways to expedite FDA approval of drugs that showed promise in early trials.

Out of this developed the idea of a special pathway for Breakthrough Therapies, which was codified in the Food and Drug Safety and Modernization Act of 2012 (or FDASIA). Breakthrough Therapy complements other existing FDA programs for expedited development and review of new drugs, with the ultimate goal to benefit patients with unmet medical needs.

Sponsors that receive a breakthrough designation will usually receive more intensive and earlier engagement and guidance on an efficient drug development program, as well as greater involvement of senior FDA officials.

Now, less than two years later, we have already seen 30 drugs designated as breakthrough therapies. And just last week FDA approved the first drug to receive breakthrough therapy designation, Gazyva, to treat chronic lymphocytic leukemia. It is not surprising that the first breakthrough approval was for a cancer drug, given that so much exciting breakthrough science is happening in the oncology realm and provides the basis for new, more innovative drug discovery and development.
And I want to recognize another significant breakthrough—an important an important advance that has emerged through this group’s collaborative work—and that is the Lung Cancer Master Protocol. 

As you just heard from the previous panel, the Protocol has blossomed from an innovative idea born here just last year into a tangible and truly revolutionary new structure designed to take advantage of the opportunities in personalized (or precision) medicine, and will help usher us into this exciting new era of research and treatment.

Today’s announcement of a lung cancer clinical trial infrastructure that will provide for multi-drug, multi-arm trials in which the same patient sample will be screened for multiple genetic traits at the same time is an exciting development with promising benefits for both the industry and patients. As a result of this approach, a rich amount of data will be collected more quickly and at lower cost.

By combining the resources of drug companies to test several therapies specifically targeted to individuals with particular genetic makeups -- and to do so in potentially hundreds of clinics throughout the United States – the development of this Protocol vastly increases the chance that we will find more effective treatments, and does so in a creative and more cost-effective way. 

But the promise of this Protocol is not confined to the development of specific lung cancer drugs. Its significance also derives from the model it establishes for other clinical research as well as for future collaborations between FDA, industry and academic researchers.

We have at our disposal a vast array of advances in our understanding of disease, human biology and the science of drug development, new techniques for rapid genomic analysis such as next generation sequencing, the availability of more powerful computers and other technologies and so much more. What this means is we now have the opportunity to greatly expand our ability to find new treatments and tailor them to the characteristics and needs of patients.

At FDA we have been placing increased focus on personalized medicine, adapting and modifying regulatory processes in response to – and in anticipation of – scientific and medical achievements. We just put out a new report this topic that I think is a valuable contribution to the field, which you can find on the FDA website. The Protocol is an important step forward in this domain. It embodies the critical goals of innovation, partnership and a commitment to the best available science.

As you understand, however, even as we move forward with new thinking and action to develop drugs in more effective and more targeted ways for the benefit of patients, we must always think about more than just approval as a guidepost.

Too often the development of a drug is thought about simply in terms of getting approval.  While that is important, we must take more of a dynamic, lifecycle approach; ones that includes research and innovation, oversight and testing, both before and after approval. It requires a continuous assessment with the ongoing balancing of risks and benefits.

And again, partnerships exemplified by the work of those present here and this conference – between regulators and innovators and entrepreneurs and advocates – are critical to building success at every stage of the process.

We must have, for example, the right incentives at the outset to encourage industry and others to invest in the development of new products. More broadly, we must ensure that there is sufficient support and funding from government, industry, academia, and advocates to encourage innovation and development of the opportunities in science to produce the treatments so many diseases require.

Certainly, that is some of what the Master Protocol ultimately aims to achieve. But, as you have been discussing, no matter how much we increase efficiency and minimize costs and time involved, there will continue to be significant risk, in terms of both potential success of the treatments, and potential for return on investment. 

Finally, and perhaps most importantly, we must remember that this process is one of continuing exploration. In our business we rarely deal in absolutes.  This is particularly true when it comes to assessing safety and efficacy of drugs, and balancing apparent risks and benefits. There are always uncertainties and unknowns.

This is certainly the case with the development of drugs approved through an accelerated process. As with all drugs, adverse effects may become apparent only after a drug is marketed and used more widely.  These will require our monitoring, response and adjustment. While these bumps are often disappointing, they should not be viewed as failure.

It is only natural that a process like this is accompanied by risk. And particularly when we expand from a relatively small, clinical trial conducted under carefully controlled conditions, to a much larger population of patients in the real-world setting-- there will likely be additional findings and possibly new safety concerns.

We saw this process recently with the drug Iclusig, which was approved last December for the treatment of chronic myeloid leukemia (CML). Recently, however, we received reports of a higher incidence of strokes, heart attacks and other serious side effects in patients taking this drug. As a result, we asked the manufacturer to suspend marketing and sales of the drug, and they agreed. And we are working aggressively to make sure that it is still available for those patients who believe that the drug is having an overall beneficial effect.

Some critics may suggest that this case demonstrates that Iclusig, or any drug that reached the market as a result of an accelerated pathway, should not have been approved in the first place.  But that’s the wrong lesson to be learned.

Taking on a certain level of risk is an important component of such a regulatory process. Indeed, it would be remarkable if some of these drugs that receive accelerated approval did not have their usage indications modified in some way, including possibly coming off the market either temporarily or permanently, as a result of new information and continuing study. Balancing that risk with the potential benefits of getting promising drugs to sick patients with limited options must continue to be our point of reference.

And the need for continuing oversight, monitoring and study of drugs post approval is true for all drugs, not just those approved with an expedited pathway. But to do so requires building the capacity to measure and track real world experience, including development of a strong electronic reporting system to monitor these results.

That is precisely why FDA has undertaken the Mini-Sentinel, a pilot project intended to create an active surveillance system—The Sentinel System—that can monitor the safety of FDA-regulated medical products using existing health care data from multiple sources; an approach that allows FDA scientists and researchers to acquire information about how patients are responding to a particular drug and strengthen our ability to quickly identify, assess and respond to emerging safety concerns. We currently have a database that includes some 125 million people.

And the American Society of Clinical Oncology (ASCO) is also now developing a quite comprehensive monitoring system of special interest to the oncology community, drawing on their unique network of providers and their patients.

These are important systems that will add to our ability to gain a deeper understanding of the risks, benefits and appropriate use of products throughout their lifecycle.

All of which brings me back to some of the exciting developments on which this conference has been focused. Over the past six years, panels at this conference have addressed some of the most pressing challenges and the most compelling innovations in the fight against cancer.
Through your work here you have explored new pathways and strategies to get cutting-edge treatments to patients more efficiently than ever before, in clinical trials and beyond. 

You have developed public-private partnerships for true collaboration across industries and institutions.

And you have worked to streamline regulatory systems so that medical innovation doesn’t outpace our ability to deliver care to patients. 
There is much important work still to be done…and I so am going to let you get back to it. And I have to warn you, we have high expectations!

Thank you again for the work you are doing, and I look forward to continuing our collaborative efforts.