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Science, Collaboration and Smart Regulation: Catalyst for Medical Opportunity

Remarks by Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
CED Life Science Conference
Raleigh, North Carolina
February 26, 2014

Good afternoon. Thank you Roger [Jeffs], for that kind introduction, and for your work in organizing today’s conference. I also want to thank CED, the North Carolina Biotechnology Center, and North Carolina Biosciences, for their sponsorship of this meeting. I also want to thank my good friend and long-time colleague Bill Roper, for inviting me to participate in today’s meeting.

It is a pleasure to be with all of you today here in the "Triangle" — the heart of so much extraordinary medical and scientific research, as well as a beautiful area of the country.

I’m really delighted to join you for this conference, which brings together key representatives from across the spectrum of life sciences and healthcare — companies representing the vanguard of drug and device development, academic institutions, patient advocacy groups, funders of research, medical providers, and of course government agencies.

I also want to congratulate Ralph Snyderman, who is being honored here with the Life Science Leadership Award. I have known, and known of, Ralph for a very long time.  He is an excellent choice for this award for obvious reasons: it reflects his broad background and contributions in academia and industry and his remarkable record of leadership, research and accomplishment in groundbreaking fields like personalized medicine. Congratulations, Ralph.

Like Ralph, the broad range of stakeholders and supporters here today, both individually and collectively, play an important role in the convergence of medicine, technology and regulation by digging deep into the science, but also by connecting with others, exchanging ideas, and building for the future.

I don’t use the word "collectively" lightly. How we work together is essential to our success. Obviously, the life sciences industry plays an incredibly important role as an engine for innovation and economic growth in this region and beyond. But the remarkable success achieved here in Triangle Park derives in no small part from the presence of so many great biomedical research institutions and opportunities for collaboration created by their proximity.

But it is more than simply good science that helps to advance biomedical product innovation—though you certainly have a lot of that here.  It is also understanding the needs of patients and the health care system.

A healthy innovation ecosystem must be able to identify and understand critical health care requirements and, especially, unmet or inadequately funded medical and public health needs. Here again, you have several great health care institutions and systems to provide the context for this important work.

And enhancing opportunities for biomedical product innovation also requires the engagement of regulators as well. In short, it demands a broad community of support, not just to take full advantage of the remarkable opportunities for creativity and innovation in science and technology, but to translate that innovation into the products, therapies, and services that make a real difference in the lives of patients … and all our lives. 

And make no mistake. The opportunities today are extraordinary. More than in any other time, science offers us enormous potential to transform the prevention, diagnosis and treatment of many diseases — even cures — through better and more innovative therapies and approaches. I noticed that one of this morning’s sessions was called "Innovation Showcase." That title might just as easily have been applied to this entire conference or, in many respects, to medical science generally.

Today, I want to talk with you about some of these advances, not only the promise they hold, but also the challenges they pose, and how the FDA is working in unprecedented ways to address these challenges and improve health and patient care by working in partnership with many different stakeholders.

Indeed, the FDA of today — the role we play, the products we review, and the processes we employ to carry out our public health mission — is something that the early leaders of FDA probably could only have dreamed of. To appreciate just how far we’ve come it may help to recall a pivotal episode in FDA’s history, which had a particular impact on this area of the country.

It occurred at a time when the agency didn’t even have the authority to evaluate the efficacy and safety of new drugs before they were marketed — an important responsibility we take for granted today. 

Some of you may be familiar with the 1937 case involving Elixir Sulfanilamide, used to treat some infections. Although at the time, the drug had been used beneficially in tablet and powder forms, that year there was a demand — mostly in Southern states, for some reason – for a liquid version. The manufacturer quickly developed a good-tasting and pleasant-smelling liquid medicine and sent more than 600 shipments into the marketplace.

But there was no legal requirement to conduct safety studies at the time so no toxicity tests were conducted. Without such tests — and without FDA review — there was no mention on the drug label that one of the chemicals used to help liquefy the drug was diethylene glycol, a chemical closely related to the principal hazardous ingredient in antifreeze and, as we all know now, a deadly poison.

The drug, laced with this toxic chemical, caused more than 100 deaths, including some in the Raleigh area. The FDA acted quickly to take Elixir Sulfanilamide off pharmacy shelves, but it could only do so through its power to remove misbranded drugs from the market. The FDA simply lacked the authority to prevent the tragedy from occurring.

But the subsequent outcry led Congress to pass the 1938 Food, Drug and Cosmetic Act, which provided FDA with the authority to review medicines for safety before they could be marketed, something that today seems only common sense.

Beyond this important improvement in the law that strengthened FDA’s oversight, there is another important lesson to draw from this bit of history.

And that is that even when FDA lacked the legal authority to prevent dangerous drugs from entering the market, the Agency was nonetheless already deeply engaged in applying the best available science to the other actions it could take, whether they involved tracking the source of harmful drugs, working to remove them from the market, or analyzing the chemical makeup of marketed products.

That application of scientific expertise to the regulatory framework has helped FDA build a long and distinguished history of helping to support the development of safer and more effective medical products, as well as their quality manufacture, efforts which continue to this day.

It’s particularly important now, at a time when there is so much potential for new science and innovation.  From targeted delivery of a treatment to a tumor, to blood tracking using Radio Frequency identification, to artificial retinas, to discussions you may have read about today in the papers concerning oocyte modification interventions for mitrochondrial disease, it’s getting harder and harder to differentiate between today’s science and what used to be considered science fiction. 

And as we combine new insights into the underlying mechanisms of disease and human biology with ever more powerful computers and IT capabilities, advances in engineering, materials development and other technologies, we increasingly are in a position to harness the power of these advances to create exciting and novel products and approaches, including the ability to tailor medical treatments to the specific characteristics and needs of individual or patient subgroups. Such personalized or precision medicine is no longer just a promise, but a growing reality.  

For example, an increasing number of cancer drugs today are used with a diagnostic that can help determine whether a patient will respond to that treatment based on genetic characteristics of the patient’s tumor. 

And now we’re preparing for whole genome sequencing that would give the ability to take a broader look at a patient’s genetic makeup or the genetic characteristics of a tumor or a microorganism to help in diagnosing disease, identifying the cause of symptoms and assuring the use of the best possible treatment or technology in a targeted way. Last November we approved four diagnostic devices that can be used for next generation sequencing.

Personalized medicine means other new approaches as well … such as the progress we are seeing in regenerative medicine and stem cell therapy, in which a patient’s own cells can be used to replace or regenerate their missing or damaged tissues, and new technologies to meet individual medical needs through the process of additive manufacturing, or 3-D printing.
As I have been learning, 3-D printing has a growing place in medicine.  For example, it allows for complex and even moving parts to be created for use in the body, and, more importantly, to be designed from patient imaging such as a CT or MRI, allowing for an exact replacement. Such 3-D printing recently was used to replace a piece of a patient’s skull and to fabricate a bioresorbable tracheal splint for a critically ill infant, and more applications are potentially in the offing.

Still other important technological developments in healthcare involve tools that many of us use every day. Take, for example, health related mobile apps, which have the potential to provide enormous benefit to consumers, health care professionals, and patients. Estimates are that by 2015, 500 million smartphone users across the globe will be using a health care app.

Needless to say, these apps range in both sophistication and medical significance, from a tool to help individuals maintain a certain bodyweight by tracking calories and exercise, to one that allows a smartphone to produce and send electrocardiograms (EKGs) or test blood glucose.

And, not surprisingly, the differences in the potential risks various apps can present are just as great as the range of new medical apps we are seeing developed.

That’s why the FDA recently issued a guidance to cover this emerging field of technology. This is a narrowly tailored guidance, which is intended to help promote innovation while protecting patients, and focuses on the risk and functionality of what is actually a fairly limited group of apps deemed of the highest risk. 

While this guidance covers computer software rather than pharmacology or medical device engineering, the fundamental goals are the same — to ensure that the best available science is applied to assess the overall benefits and risks to users and support the development of new products that will make a real difference for patients.

To me, this is the ultimate benefit of any scientific or medical development — how it helps to improve the care and treatment of patients…or prevent disease in the first place. After all, innovation is important not because it creates something that is new, but something that is new and that makes a meaningful, positive difference in people’s lives.

And critical discoveries in science that can make an enduring difference for health and disease aren’t just automatically translated into products with the proven benefits, reliability, stability and quality that patients need and expect. Moving from discovery to bedside involves real expertise, focused attention, determination and investments in the right kind of scientific study for such translation to occur.

That’s where the FDA comes in. And for this goal to be met, the FDA must work closely with industry and academic researchers, as well as health care providers, patients and advocates, and other stakeholders. We all play a role as we strive to foster the development of new drugs and new medical technologies that will improve medical care, or more broadly promote public health.

And for the FDA, I believe we are in the midst of a critical transformation in how we approach our mission of promoting and protecting the health of the public as we oversee the safety, efficacy and quality of drugs and medical devices. We are advancing a new regulatory mindset and sense of engagement that I think is enabling us to move forward in important new ways, as we seek to support innovation and more rapidly bridge the gap between good ideas and meaningful products for the people who, in fact, rely on our work.

Part of this work focuses on advancing regulatory science, especially at the pre-competitive stage. For example, FDA’s Center for Devices and Radiological Health partnered with Life Science Alley in Minnesota to form the nonprofit research consortium, the Medical Device Innovation Consortium with a membership that is as diverse as the groups represented here. Some of the MDIC’s current projects include Patient Centered Outcomes Research and Clinical Trial Innovation and Reform, and the development of computer models and simulations. 

We also are casting a wider net to solicit ideas and potential best practices. The Entrepreneur in Residence program at FDA’s Center for Devices may be the best example of that. A group of world-class entrepreneurs and innovators spent time at CDRH to help develop solutions to specific problems.

This group was responsible for shaping two of the device center’s three strategic priorities for 2014 that deal with clinical trials and reconsidering the balance between premarket and postmarket requirements.

Many of the changes in regulatory approach are possible because of the kinds of advances in science and technology I spoke of earlier. Frankly, better science means better products, making our job of review and approval more streamlined and straightforward.

Many are possible because of new collaborations and partnerships.

But the changes underway at FDA also relate to important new responsibilities and authorities the agency has been given to develop expedited review tools, which we are using to promote innovation, and help patients get earlier access to promising new drugs.  

I want to spend a few minutes discussing some of these developments, which I hope will serve as an introduction to the discussion the panel on accelerated approvals you will be having later this afternoon.

At the FDA, we are currently pursuing a number of strategies to help streamline the review process, seeking strategies to enhance the effectiveness and efficiency of our regulatory system…on both the business process side and with respect to regulatory pathways.

It is the latter, the regulatory pathway side, that I want to focus on now.  You likely know that we have been using several mechanisms — priority review, fast track, and accelerated approval — to speed review as appropriate. These have clearly made a difference. However, the most recent, and increasingly significant of our approaches to help expedite drug review and approvals is the breakthrough therapy designation, which was included in the landmark Food and Drug Administration Safety and Innovation Act — or FDASIA.  Since that law was passed, we have already seen 40 drugs designated for review as breakthrough therapies, and three of those have received approval.

One of the great benefits of this new pathway is the opportunity for engagement with FDA reviewers and high-level FDA scientists and managers during earlier stages of drug development.

FDA’s Center for Drugs is also working more broadly to improve its communication to sponsors during drug development, including setting up a dedicated communication and training staff that can provide training, receive feedback and serve as an important point of contact for industry.

All of this can really matter, though it is very resources intensive for us.  But we know from our work in recent years that earlier and more ongoing communication during the development process can reduce the overall time of both development and review, and can result in better products. To me these seem like important goals to strive for.

And we can see results.  In 2013, FDA’s Center for Drug Evaluation and Research approved 27 novel new medicines, or new molecular entities.  These are strong numbers, but it is not just a question of numbers, but also that these drugs will address important medical needs and conditions.  One out of three was the first in its class.  Nearly three-quarters were approved in the U.S. before anywhere else.  And a large number were approved using expedited approval pathways.

Yet we are never without our critics. Some have suggested that we are moving too fast or sacrificing standards and consistency — for speed — in  the review process. But I think this misconstrues the mandate to add flexibility and tailor clinical trial requirements for each drug and the disease area for which it is being studied.

Certainly when Congress created the "breakthrough" pathway, it recognized that the responsibility to help translate the newest discoveries and research into treatments for patients who are critically in need as the goal of drugs developed through the accelerated approval process.  And that means a rigid, "one size fits all" approach is not the answer.

It is important to understand that no matter what approval pathway is used, the Agency consistently applies the same statutory approval standards of safety and efficacy to all drugs in order to be marketed in the United States. Increased flexibility does not mean abandoning standards…and it certainly does not mean abandoning science. Just the opposite. We need to employ the best science in ways that will increase our efficiency, productivity, and our shared ability to find creative solutions to the challenges that confront us.

But we also must recognize that each new medical product we approve and usher to the market will always involve a balancing of risks and benefits. A certain level of risk is an intangible component of such a regulatory process.

And we must absolutely balance our eagerness to find ways to address the needs of patients for the most serious illnesses by responding as quickly as possible to develop treatments, with the need to ensure that the benefits outweigh the risks.

It is a huge responsibility FDA is charged with, nowhere more so than when dealing with unfolding technologies that offer enormous potential -- but that also may present real risks for people and their health.

As with all drugs and devices, adverse effects may become apparent only after they are marketed and used more widely. It would, in fact, be remarkable if some of the drugs that receive accelerated approval — in fact, any kind of 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.

Such results are disappointing, but they are not a failure of our regulatory system. Rather, we might say that they offer evidence that the system works as intended.

Balancing a product’s risk with its potential benefits to patients must continue to be our point of reference … and action. That is why, over time, we have evolved from the view that we must protect patients from the consequences of research, to a view that we must protect patients through research, in order to assure their access to new and effective medications.  And it is a continuous process throughout the life of a product and its use.

You will hear later today from Nancy Goodman, an inspiring advocate in the fight against pediatric cancer. She understands the challenges of this balancing act, as well as the benefits of collaboration and coordinated communications between government agencies, patient advocates, funders of research, and others — with the goal being to make the investigation and development process in the search for cures.

One such achievement growing directly from Nancy’s efforts is the Creating Hope Act, which was signed into law in 2012. This legislation provides market incentives to pharmaceutical companies to develop drugs for pediatric rare diseases by providing a transferable voucher for priority review.

I’m very pleased to report that just this month — appropriately, on Valentines Day — FDA awarded the first Creating Hope Act voucher to BioMarin for Vimizim to treat Morquio A Syndrome, a rare and serious disease. This is a tribute to Nancy’s hard work, of course, but also a demonstration of what comes from building partnerships in the fight against cancer … as well as understanding the perspectives and necessary alignments of critical needs, opportunities and incentives across all components of our biomedical product innovation ecosystem.

And this brings me back, as I conclude my remarks, to where I began — which is that success, in this unprecedented era of medical discovery and scientific progress, depends on partnership — across disciplines, experience, and sectors.

Ventures like the recently announced Lung Cancer Master Protocol, for instance — a collaboration among FDA, advocacy groups, academia and the drug industry — take advantage of our growing engagement in personalized medicine to undertake a highly innovative clinical trial design and infrastructure that can be used to test multiple new investigational drugs with multiple study arms at multiple sites, using whole genome sequencing to screen the patient population for multiple genetic traits.

It is a highly efficient design that will allow several therapies specifically targeted to individuals with particular genetic characteristics to be studied.

Approaches like this are important, not just because of the results they can deliver for the disease and treatments under study, but for how they can be adapted or extended to other areas of research and to other diseases, bringing together the best minds to focus on what needs to be done … and take action to do it in new and exciting ways.

The United States today is the global focal point for the life sciences, home to the world's most advanced and extensive basic research enterprise and to the most dynamic medical device and biopharmaceutical industries. The benefits to our nation from this leadership have been dramatic and vital to our success.

I’m proud that FDA is playing an increasingly important role. And I can assure you that whether it involves a review of a new drug, the development of a novel clinical trial design or the validation of a biomarker, the application of advances in genome sequencing or nanotechnology, or the development of some other state-of-the-art medical treatments or devices — FDA is committed to uphold our responsibility to ensure the safety and effectiveness of medical products, and, just as important, to working with all of you to help these scientific achievements reach their full potential by building a pathway for continued innovation that matters.

But progress for us depends on more than just the work of our agency.  And successful innovation is more than an individual scientific breakthrough by a single company. It is more than an approval of a single drug, therapy, or trial, no matter how important those may be. I’ve heard it said that "No one can whistle a symphony. It takes a whole orchestra to play it." Well, what we want are medical symphonies. Beautiful music that can change lives.  It can be done — it has been done.  And I look forward to our continuing collaboration to achieve that goal.

Thank you.