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FDA-CMS Summit for Biopharma Executives

Remarks by Margaret A. Hamburg, MD   
Commissioner of Food and Drugs
FDA-CMS Summit for Biopharma Excecutives
Washington, DC
December 12, 2013

Thank you for that introduction, and for inviting me to be with you today. I understand that you have had two excellent and very full days, covering a wide range of important topics.


Coming as the final speaker after such an impressive lineup and rich agenda creates a certain challenge for me.  By now, I suspect that you have participated in numerous fact-filled sessions, enlightening discussions, and had some lively debates. Hopefully all of this has triggered some interesting new ideas and directions. But the breadth and depth of your previous discussions makes it hard for me to know what to add.


So while I may not shed much new light on the issues, I would like to use my time this afternoon to focus on several key themes that will continue to be priorities in the coming months and years for all of us who care about health and the improvement of medical care, whatever sector you work in or whatever your professional discipline.


One theme implicit in most, if not all, of the discussions during these meetings, as well as in the daily work in which we are all engaged, is the impact of the extraordinary advances in science and technology.
 

Historians write about particular eras that stand apart in the history books. These can be identified by important changes that spur human progress during these periods, acquiring names such as the industrial age, the jet age, or the age of computers.


We have clearly entered one of the most remarkable and productive eras in human history for achievements in medical science. Life sciences research, advances in information technology, engineering and other disciplines are coming together in extraordinary ways. More than in any other time, scientific innovation today offers us the extraordinary potential to transform the prevention, diagnosis and treatment of disease—even cures – through better and more targeted therapies. Developments are happening so fast that what might have been termed science fiction just a few years ago… is today simply science.


Consider recent progress in regenerative medicine and stem cell therapy in which a patient’s own cells offers the promise of replacement or regeneration of a patient’s missing or damaged tissues. Advances in sequencing the human genome, combined with greater understanding of the underlying mechanisms of disease and human biology, are making it possible to develop new therapies tailored to the specific characteristics, needs, and preferences of individual patients.


The emerging field of “personalized or “precision” medicine makes possible treatments that simply were not available before -- because both the understanding and the technology did not exist.


For instance, as you have discussed during this meeting, the treatment of some diseases, such as cancer, is quite literally being transformed as a result of new drugs and new treatment strategies.


We are entering an era when next-generation DNA sequencing technology offers the ability to identify the genetic mutations present in a patient’s tumor and enable physicians to use this information to choose the best therapeutic regimen for that individual and rule out drugs likely to be ineffective or have side-effects that would outweigh benefits.


At FDA, we are enormously supportive of this new direction. More information means better products and more appropriate use.


But the rapid pace of scientific advancement requires us to think and act in new ways. We must spur on innovation while protecting patient safety through smart, science based regulation. Toward this end, we are adapting and changing regulatory policies in response to – and in anticipation of – scientific opportunities so that they can best be harnessed to improve health and medical care. And we can see benefits for patients.


Consider, for example, just a few of the drugs FDA has recently approved. 


Last week, we gave a green light to Sovaldi, an oral drug that is the first to demonstrate safety and efficacy to treat certain types of Hepatitis C virus infection without the need for co-administration of interferon, with all its attendant side effects. This novel medication opens up new opportunities to improve treatment for many of the 3 million Americans with chronic hepatitis and even offers the prospect of cure.
 

And last month, the FDA approved Gazyva for previously untreated chronic lymphocytic leukemia (CLL), a slowly progressive blood and bone marrow cancer diagnosed in some 15,000 Americans each year and a disease that takes over 4,500 lives annually. Gazyva works by helping certain immune cells attack cancer cells and represents a significant addition to the treatment options for patients with CLL.
 

Also last month the FDA approved Imbruvica to treat patients with mantle cell lymphoma (MCL), a rare and aggressive type of blood cancer. Imbruvica is the third drug approved to treat MCL, and is intended for patients who have already received another therapy, offering an important additional treatment option.


Each of these drugs is a significant advance for patients, and also represents an example of how new scientific insights are leading to exciting and important innovation in laboratories across the nation. But I also mention them because they demonstrate how FDA is pursuing new strategies to better support the timely translation of important opportunities in science into products that matter for patients.


We are working collaboratively with companies, as well as other researchers and stakeholders, to expedite the development, review and availability of promising drugs.


These three drugs were among the first that FDA designated as “breakthrough therapies” to gain approval. Breakthrough designation allows FDA to expedite a drug’s development at the request of the sponsor if preliminary clinical evidence indicates the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. 


Breakthrough Therapy was part of last year’s landmark Food and Drug Administration Safety and Innovation Act (or FDASIA), and offers real opportunities to get promising drugs more quickly to patients who need them. I should add that FDASIA was passed with overwhelming bipartisan support.


And Breakthrough Therapy can be used in conjunction with several other regulatory pathways for speeding development and review of critical and promising drugs, such as Fast Track, Priority Review and our Accelerated Approval program. Earlier today you heard from the director of FDA’s Orphan Drug office, Dr Gayatri Rao. This program offers an additional regulatory approach to help support and speed the development and review of drugs needed to treat rare conditions or disease, and has demonstrated its importance and value.


FDA’s ability to adapt and strengthen its regulatory role in the face of changing technologies and constant innovation is essential. New challenges will continue to arise with new scientific developments, and will be accompanied by increasing commercial opportunities and applications.  Keeping up with these challenges is an enormous responsibility, and one FDA takes very seriously. But let me say, our underlying responsibility remains the same -- to ensure the safety and effectiveness of drugs and other medical products.


Ultimately, the review of each new product we approve and usher to the market is based on rigorous study and evaluation of hard data and the best available science.


And regardless of the speed of the development or the review, that product must demonstrate that its benefits outweigh its risks. The FDA does not compromise its safety or efficacy standards in exchange for rapid approval.  Moreover, our responsibility for oversight extends after approval when the products are in the marketplace—and we can often learn a great deal more about a product when it is in real-world use.


This underscores another point often lost in discussions relating to the FDA’s work -- that regulation, when done right, is a key driver of innovation in the laboratory and across society.


It can support—in fact shape—more appropriate, streamlined, and time and cost-effective product development. Moreover, smart, science-based regulation instills consumer confidence in products and treatments. It levels the playing field for business and promotes economic development. It decreases the threat of litigation. It prevents recalls that threaten industry reputation and consumer trust— and that also levy huge preventable costs on individual companies and entire industries. And it spurs industry to excellence.


In the area of personalized therapies, for instance, the FDA can play a particularly significant role as a driver of innovation.


Our efforts encourage the development of new tools to evaluate targeted therapeutics. They promote greater collaboration in key areas of research. They define and streamline regulatory pathways and policies. And they apply new knowledge in product reviews.


Whether it involves advances in sequencing the human genome or the development of some remarkable new drug, the addition of rigorous regulatory science from the FDA helps to ensure the safety and effectiveness of innovative new products; sees that these scientific achievements quickly reach their full potential; and builds a pathway for continued innovation.


But our effectiveness comes as a result of partnership. It is to this subject that I want to turn to next. I can promise you that FDA will continue to promote innovation through rigorous application of regulatory science. 


The opportunities offered by new scientific breakthroughs and technological innovations – along with the creativity that spurs them – will be significantly furthered through increased collaboration and by transforming how we approach research, clinical care, and patient engagement.


We need to develop new ways to work with partners across government, as well as with industry, academia, the private sector, NGOs, philanthropic groups, and of course the public that we serve.


We have already formed a variety of partnerships and forged many agreements to help facilitate scientific research and development, inform regulatory science, and strengthen opportunities in clinical trial design, clinical pharmacology, and translational science. By enhancing these kinds of collaborative ventures, the FDA and the greater scientific and stakeholder community can chart a course to pursue science-based strategies to overcome barriers to innovation while optimizing safety and benefits for patients.


Perhaps nowhere is the need for us to expand our approach to developing new partnerships greater than in our response to the growing challenges created by the expanding global marketplace.


As you likely know, products used here at home are increasingly manufactured, in whole or in part, in other countries. For example, 40 percent of all finished drugs used in this country and 80 percent of the active ingredients are made outside the United States. And as globally sourced materials are turned into finished goods, their ultimate distribution in the US may follow an astonishingly complex supply chain.


All of this clearly creates potential vulnerabilities in the U.S drug supply and increased pressure on regulatory authorities to ensure the quality and safety of these products. How we respond on a domestic level, and how we interact and cooperate with other nations in a productive way that addresses both economic and security needs, will have an enormous impact on public health.


Last week, I had the opportunity to attend a meeting of international Heads of Medicines Regulatory Authorities. As regulators we share many common challenges. Globalization for sure, and the challenge of protecting the integrity of our supply chains.


But also: the increasing complexity of new drug products and drug development and keeping up with the fast pace of change in science and technology; growing geographic distribution of markets; greater demands for public accountability and transparency in our work, and budgetary and political challenges to regulatory oversight. And of course, there is also the overriding burden of maintaining quality in the face of all these pressures.


All of which brings me to my final theme-- the principle at the heart of both global and domestic health and regulatory challenges, and the backdrop for everything else I have been talking about today – and that is quality.


Each day millions of patients, from babies to adolescents to grandparents, rely on thousands of drug products to help ensure their health and well-being or fight disease. It is vital that they get the medications they need in a timely fashion.  And it is just as critical that we continue to develop innovative new drugs that will offer improved outcomes. But without product quality, none of us can feel completely comfortable that a product is either safe or effective. These concepts are inseparable.


We are all too aware of how lapses in quality really matter, wherever and however they occur. Episodes of product contamination have caused direct harm to individuals, as well as put communities at risk. Also, manufacturing and quality issues can generate recalls that lead to widespread shortages of critical drug products.


Quality is fundamental in so many additional ways. It is how a company builds its reputation and trust in its products. Quality is a very large part of why the American pharmaceutical industry has been the standard against which the rest of the world is measured.  And most importantly, quality is what the public cares about … and expects. And it is why we must renew our focus in this area.


While it is the responsibility of individual companies to manufacture safe, effective, high quality drugs, the FDA plays a major role in helping to ensure that patients in the United States get the quality drug products they require. And increasingly we have been focused on the issue of quality, from strengthening enforcement and inspections, to supporting broad developments that provide new opportunities for building quality into the system.


For example, we are working to encourage advances in pharmaceutical manufacturing technology made over the last decade which offer significant opportunities to strengthen quality.


Forms of “continuous manufacturing,” which includes chemical synthesis of active ingredients, offer a means to prevent shortages and ensure that product quality can be precisely controlled. It allows for a range of strengths or doses of drugs to be prepared more easily, which has important implications for personalized medicine, and for the quality and availability of critically needed drugs.


In addition, recent legislative advances will also make a significant difference in our ability to help ensure high quality products. We were pleased that Congress passed, and the President recently signed into law, the Drug Quality and Security Act.


This law includes important protections and establishes critical steps to build an electronic, interoperable system to identify and trace certain prescription drugs as they are distributed in the United States. It will enhance FDA’s ability to protect consumers from exposure to drugs that may be counterfeit, stolen, contaminated, or otherwise harmful, and will strengthen our ability to detect and remove potentially dangerous drugs from the supply chain. 


Also, the new law also provides a regulatory framework for certain entities that prepare compounded drugs and that register with FDA as outsourcing facilities. If a facility registers as an outsourcing facility, to qualify for the exemptions from certain requirements such as the new drug approval requirements, they must follow the requirements in the new law that are applicable to outsourcing facilities. In addition, outsourcing facilities are subject to current good manufacturing practice requirements, and FDA will be conducting risk-based inspections of these facilities.


While the new law did not provide FDA with all of the additional authorities we sought, it is definitely progress.
 

This new law complements the goals of the Food and Drug Administration Safety and Innovation Act I mentioned earlier. That landmark law gave FDA additional authority to secure the safety and integrity of drugs imported into the U.S. We are moving towards an integrated system for ensuring the integrity of drugs.


This includes provisions allowing FDA to refuse admission of a product if inspection of a manufacturing facility is delayed, limited, or denied; requires companies to provide complete information on threats to the security of the U.S. drug supply chain; and improves current registration and other informational requirements for foreign and domestic manufacturers.


These are important steps that will make both an immediate and long-term difference in the quality of the drug supply in our country.  We are pleased that Congress has recognized the important role FDA plays, and has given us major new responsibilities to help us fulfill our mission. I am hopeful that along with this recognition, Congress will also provide the kind of real resources—not just new mandates and authorities-- that are equal to the challenge.


In our rapidly changing world, it is vital to invest continually in science as an engine of innovation. But it is just as critical that we maintain support for those processes and structures, such as the FDA, which contribute significantly to the meaningful advancement of biomedical product innovation into real-world products for people.


Without that support, we are in danger of not being able to build and sustain the systems and capacity required to undertake the necessary scientific analyses and review, to develop new tools and approaches, to provide ongoing oversight of products in the marketplace, to conduct investigations, and to enhance collaboration in the ways that are vitally needed.


I began my remarks today by highlighting the remarkable period of change and innovation that we are lucky enough to be part of.


For no matter how dramatic the advances in science and technology are, they must not be the end point.  To me, innovation is not just something that is new, but it is something new that makes a meaningful difference. The challenges we are facing cannot be addressed by just one law or one policy or even the many remarkable medical discoveries we are experiencing. 


So I urge us all to seize the opportunities that these developments can provide for patients and for health care as a whole. We need to develop a broad strategic approach that recommits our nation to the advancement of medical product innovation and will ensure that now and in the future, the American people will have access to the care and treatment they need, whether the disease be rare or common and whether the treatment is existing practice or cutting edge.


To do this will require a commitment by all of the players in this ecosystem, and significant collaboration and planning, unlike any prior effort. I look forward to exploring the possibilities and to working with you on the development of this kind of future.


Thank you.