A Conversation with FDA Commissioner Margaret Hamburg

Margaret Hamburg was one of the longest serving commissioners of the U.S. Food and Drug Administration (FDA) in fifty years. With Dr. Hamburg at the helm, the FDA confronted the challenge of fulfilling its regulatory mandate in an era when the production of food, drugs, and medical devices increasingly occurs overseas and in complex supply chains. It began oversight of tobacco products, expanded the Agency’s efforts on counterfeit medicines internationally, and approved its first biosimilar drug and over the counter use of the emergency contraceptive Plan B. The FDA also faced major public health issues, including antibiotic-resistant bacteria, electronic cigarettes, generic drug shortages, a fungal meningitis outbreak, Ebola, and the abuse of opioid painkillers. FDA Commissioner Margaret Hamburg joins CFR’s Thomas Bollyky to discuss the lessons and accomplishments of her tenure.

BOLLYKY:  Great.  We're -- we're going to get started if everybody could take a seat.

 

Terrific.  Well, I'm pleased to welcome you to the Council on Foreign Relations.  I'm Tom Bollyky.  I'm very pleased and honored to welcome Dr. Margaret Hamburg here today.  As most of you hopefully know, she is stepping down as the twenty-first commissioner of the Food and Drug Administration.  She is also one of the longest-serving in the last fifty years.

 

This is her second-to-last day on the job, so it's very generous of her to agree to spend part of it with us.

 

I also want to acknowledge and welcome her parents, Dr. David and Beatrix Hamburg, who, as many of you will know, have had their own various illustrious -- very illustrious careers.  It's -- I'm pleased to have them here today.  They're here to support their daughter and keep the rest of us in line.

 

(APPLAUSE)

 

There's no shortage of topics we can discuss here today.  Dr. Hamburg has had a very eventful tenure.  With her at the helm, FDA began its oversight of tobacco products, it approved its first biosimilar and has confronted public health issues ranging from antibiotic-resistant bacteria, to Ebola, to generic drug shortages, to electronic cigarettes.  So there's been no shortages of challenges.  All those are fair game.

 

What I want to -- what I will focus my questions on, though, is the recent efforts of the FDA to try to re-vamp its efforts to pursue its domestic public health mandate in an increasingly globalized world.

 

Between 2007 and 2015, U.S. imports of FDA-regulated products tripled. Forty percent of our medicines come from overseas, 80 percent of the active pharmaceutical ingredients for those medicines are made by manufacturers abroad, half of U.S. medical devices are imported, half of our fresh fruits, a quarter of our vegetables and up to 90 percent of our seafood comes from overseas.

 

FDA is responsible -- in 2012, FDA was responsible for twenty-four million shipments of products that originated from more than one hundred fifty countries from one hundred thirty thousand importers and three hundred thousand foreign facilities.

 

Oversight of this volume of goods at U.S. borders, which was the traditional method of addressing imported products, is -- is not possible.  How to address this global challenge has been an early priority of Dr. Hamburg's tenure at the FDA.  And today we get to hear about how it's gone.

 

The format for today's event is Dr. Hamburg will give ten minutes of opening remarks, I will have the privilege of asking her a few questions after that, and then we will turn it over to the audience.

 

Before we get started, though, CFR requires me that I read you a disclaimer, which is appropriate for an event about the Food and Drug Administration.  I will resist the urge to issue it in the style of our pharmaceutical advertisements by speaking very fast over images of an older woman cycling with her grandchildren or an older man painting a fence with an attractive woman far too young for him.

 

(LAUGHTER)

 

But I digress.

 

Today's meeting is on the record.  Anything you can and will say will most likely be used against you, so be prepared. 

 

If you have a cell phone, I would ask that you turn it off at this time as it does interfere with the sound system.

 

And with that, I will turn it over to Dr. Hamburg.

 

HAMBURG:  OK.  Well, thank you, Tom.

 

And thank you.  Actually, Tom has been an important partner for me over the years in thinking about some of these challenges of globalization and putting some of what we do at FDA in a broader context.  So I've been very grateful for that partnership.  And I appreciate this opportunity now.

 

Also, since you noted the presence of my parents, I have to recognize that there is more than one person in this room that strongly advised me against taking this job, my parents being the first two, Gail Cassell another one, or at least highly skeptical, and I think John Monahan was another one with the various warnings:  you'll get eaten alive; why would you want a job where the very definition is that someone is always mad at you; and don't you have better things to do with your time?

 

But it has been an amazing experience and I've been, you know, so grateful for the opportunity to, number one, get to know the FDA better and understand its critical and unique importance as an agency and its role in American society and beyond, but also to have been at the helm of this great institution during a period that I think really has been so critical in terms of better defining FDA and its role and really repositioning it for the challenges of today and certainly for the future.

 

Tom touched a little bit on this.  But you know, in terms of addressing the challenges posed by globalization, addressing the challenges posed by the dramatic advances in science and technology and the need for innovation, the increasing breadth and complexity of the products that we regulate, and taking on responsibility for implementing a series of very important historic and transformative new laws. 

 

The tobacco law was actually passed within a few weeks of my becoming commissioner back in 2009, really giving us, for the first time, the responsibilities to regulate tobacco products, manufacturing, distribution, and marketing, but also requiring us to build a whole new infrastructure at FDA and the first new product center in literally decades.

 

Also, the Food Safety Modernization Act which is going to make a huge difference in the lives of all Americans because we're shifting from a focus on reacting to problems after they occur to actually preventing food-borne illness, looking at a risk-based approach of where are the vulnerabilities in the entire life cycle of these products.  And also taking on really the recognition that we have a globalized food supply and we have to address it in that way.

 

New authorities and other aspects of our work in terms of expanding our drug regulatory oversight, quality, new regulatory systems for speeding innovation and, you know, really addressing some of the critical track-and-trace issues on the medical products side that are comparable on the food side in terms of really knowing where products are coming from and being able to trace the complexity of their supply chains.

 

And all of these things really matter for Americans.

 

And I think while everyone recognizes the words FDA and they think, yes, I understand what the FDA does, it is hard to really appreciate, I think, both the breadth and depth of our responsibilities, all the different products that we regulate, which actually all told are over twenty cents of every dollar that consumers spend on products that are FDA-regulated products.

 

And you know, they're things that really matter to people.  The safety, efficacy, and quality of human and animal drugs, the safety efficacy and quality of devices, vaccines, biologics, the safety of the majority of the nation's food supply, the safety of the blood supply and other tissue products, dietary supplements, cosmetics, things that emit radiation, and tobacco.  It is a lot.

 

And I think the extraordinary thing is that there is no other agency in government, there is no other not-for-profit or private-sector entity that really has the set of responsibilities that we do to oversee these products.  So if we are not doing our job and doing it well, there really is no other option.

 

And people, I think, have great confidence and trust that these products will be safe, they will work in the way that is promised and they will be available in a timely and meaningful way.

 

But really, every day people are working really hard at the FDA here in Washington and in fact now around the world to make that possible.  And I really think that FDA, you know, historically and to the present, has been both under-appreciated and underfunded to do its job.

 

As I look forward at the challenges ahead, our ability to really live up to the responsibility that the American people have vested in us is a serious concern.  You know, over and over again there are examples where our responsibilities and mandates outstrip the resources that we have.  And we need the continuing support to make the difference that we make in people's lives.

 

But in turn, we need to always be adapting what we do to ensure that we are meeting the needs and expectations of the American public and, frankly, the world beyond because we remain really the gold standard for regulation around the world.

 

But it's been a very important time.  I think I'm going to leave the agency stronger, more engaged, and a more effective agency, frankly, than the one that I found and better-positioned for the challenges of the future.  And I also feel very good that I'm leaving behind a strong and committed leadership team to carry on the mission.

 

BOLLYKY:  Great.  Great.  Well, thank you for those opening remarks.

 

In talking about what FDA has done to address this global challenge or the need to operate in a more globalized world, you talked a bit about the issue of legislative mandates and authorities as well as resources, both of which are, of course, at the mercy of Congress at the particular moment.

 

What I want to focus on, at least initially, is what the agency can do, assuming that our political environment remains what it is and we don't see changes in those legislative mandates and we don't see a surge in funding, whether we would want to or not.

 

It strikes as there are a couple of options of levers that FDA might be able to pursue to advance this.  And it's often said and obviously true that FDA oversees twenty cents of every dollar that U.S. consumers spend, but there's a flip-side of that story, which is FDA also oversees a significant share of what the rest of the world makes.

 

We are nearly 40 percent of the global pharmaceutical market in the United States.  We are 60 percent of the global market for electronic cigarettes.  Currently, our U.S. seafood imports and exports are 20 percent of the global market.

 

What ability does that market power give to FDA?  That has obviously enormous economic implications for producers, it has enormous influence on the way other countries regulate.

 

Six years into this position, is that something that can be leveraged, or is that more just simply a challenge and it's best not to focus on the market forces that drive these concerns?

 

HAMBURG:  Well, I guess from the perspective of the FDA really, our mission is to make sure that the American people are protected from unnecessary health risks and that we are able to fulfill our mandate in all these different arenas.  And when it comes to the import of goods, it really is assuring safety and quality and the integrity of the supply chain that these products follow.

 

And there, I think it's in our vested interest to really work in fuller partnership with countries around the world, both with counterpart regulatory authorities and, you know, with other stakeholders, including industry to try to raise standards everywhere.

 

I mean, first of all, it's sort of a given that we can't use our old model, as you were saying, of inspecting things as they come over the border.  We have to be smarter about how we do inspections and provide oversight of safety and quality.  And that means a more risk-based approach for border screenings, which we're doing now with using, you know, more high-tech systems of tracking and targeting based on the nature of the product, the history of the manufacturer, environmental or other circumstances that might influence quality, intelligence gathered from other sources, et cetera, to really target in on, you know, which container do you take off the ship and truly inspect.

 

But much more importantly, it's this, you know, extending beyond the borders and really developing the working relationships in other countries and regions to try to ensure a higher level of quality, but also raise the quality in these countries.

 

What's striking to me -- and occasionally, people within the FDA have actually sort of stopped me and said, wait a minute, we're not a development agency, you know, we already have hard jobs, don't give us another task.  But at the end of the day, especially when you look at where products are now coming from, including some of our most vulnerable products, like some of the food commodities you mentioned are coming from countries with very immature regulatory systems, and it in the long run will be much better for us if we can help raise the regulatory capacities in those countries and regions so that they will more closely approximate standards of safety and quality that the American people expect than trying to, you know, really inspect our way out of the problem or limit imports that actually the public wants because of safety concerns.

 

And it has huge, broader benefits.  Because if we can raise the standards in those countries, we actually enhance their health and well-being as nations, their economies.  It's a very good form of economic development.  And it increases, I think, security for all of us.

 

So you know, I think I've gotten a little bit off your question, I guess, but...

 

BOLLYKY:  No, I think that's great.

 

HAMBURG:  But I think, you know, that in the long term we've got to be thinking in very new ways about what the world looks like and what our role as a regulator is in this environment. 

 

We also need to be working to try to harmonize standards and approaches so that we're not all independently doing separate, discrete things, but working together and we're now developing systems where we're actually sharing information with other regulatory authorities in new ways.  But not just sharing information, we're actually sharing workload because it's crazy for us to be going into a set of facilities in China and India and Brazil when there are other countries that also want to know what's going on in those facilities.  We can, you know, divide and conquer, so to speak.

 

And again, as part of what we're trying to do is working together now as more of a coalition of regulators, as we're doing inspectional work and as we're creating a stronger presence in other countries, also trying to educate and train both industry and counterpart regulators on those countries to, you know, keep striving to raise standards.

 

BOLLYKY:  Great.  A couple of things I want to say there.  First, I want to acknowledge Patrick Kelly and the Institute of Medicine which did a great report, I think, on this issue and looking at the development and health links of regulatory systems in low- and middle-income countries, because I think that accords nicely with the comment you made.

 

I guess I would ask, particularly with a new commissioner coming in, what do you see as the strategies that are likely to be most impactful in moving that forward?  Is it trusted importer programs?  Obviously, the TSA has moved to this global entry model.  Are there ways of trying to reward access to U.S. markets for better performance?  Obviously, trade considerations there, but something that might be done. 

 

Is it these networks that you've talked about?  There has been some good progress of regulators working together, but they are typically under the format of some close cultural ties like the New Zealand and Australia or within the European Union. 

 

Which of these collaborations among regulators do you see as moving forward?  What gives you hope in this space?

 

HAMBURG:  Well, I think we're going to use a lot of different models.  You know, certainly you mentioned, you know, the sort of trusted traveler model.  I mean, that's part of what we're doing at the borders is, you know, we're rewarding companies with good, strong track records and a record of transparency and data sharing with us as well, trying to move certain kinds of things through more quickly so we can scrutinize the things where we have greater concern.

 

We're also, I think, you know, really committed to the notion of creating alliances where we have agreements for sharing information and sharing the workload, as I said, and that's going to be, the partnership.

 

You know, industry has an important role to play here in terms of, you know, stepping up to the plate. It's clearly in their interests to ensure a higher level of quality, et cetera.  And you know, so working with industry to have systems that we can validate, but where they are auditing and using, certainly on the food side, third-party auditors is really part and parcel of the Food Safety Modernization Act.

 

You know, we're never going to be able to do it all ourselves and we've got to figure out how to do it more effectively and in true partnership.

 

One of the things that I've worked hard on is actually trying to create a new, formal global governance mechanism for regulatory authorities around the world.  And that is not an easy task.  Every country obviously has its own sovereign laws that are important and are the law of the land.  You can't just ignore them. 

 

Countries also have their own, you know, values and cultures and histories and traditions that all affect the regulatory framework.  But we really do need to find ways to cut across some of that into the fundamentals so that we can better leverage limited resources and, you know, can really create this global safety net that's so important.

 

BOLLYKY:  One last question, then I'm going to turn it over to the audience, so start thinking of your questions.

 

Six years into this effort to address FDA's role in this globalized world, are U.S. consumers safer from imported drugs and foods than they were six years ago?  And is there something in particular you'd point at, too, that would make you feel confident that that is the case?

 

HAMBURG:  Well, you know, I'm a little superstitious about even -- you know, I think that we have dramatically transformed our systems for oversight of imported foods and medical products, and that does make a difference.

 

I think we've seen fewer major problems in recent years.  That doesn't mean we're not still very, very vulnerable and vulnerable in fundamental ways that have to do with both the naturally occurring opportunities for contamination or other adulteration of a product, but also we have to recognize the possibility of deliberate adulteration and that can either be, at its extreme end, you know, a terrorism kind of activity, and I think we recognize the very real vulnerabilities there, but also economic adulteration. 

 

And we have experienced that in ways that had profound impact on the American population and others as well when there was the tainted Heparin that came out of China back in I guess it was 2007 and the melamine-contaminated pet food and dairy products, which, you know, took a toll on companion pets and on people.  And those were examples of an intentional adulteration trying to make a product more cheaply, adding a substitute for an important component of the product, not realizing that it was going to actually cause, you know, serious, negative health impacts.

 

BOLLYKY:  With that, I'm going to turn it over to the audience.  If you would raise your hands, I will try to call on you in the order that I see you.  And if you'll just state your name and your affiliation prior to asking a question.

 

Gail?

 

QUESTION:  Gail Cassell.

 

BOLLYKY:  And there's a microphone coming to you.

 

QUESTION:  I'm sorry.  Oh, I'm sorry.  First of all, that night you called, I thought I tried to convince you to take it.  I tried to be balanced.

 

(LAUGHTER)

 

I would just like to applaud your efforts, though.  I have watched very closely and am very proud of what you've done.  And so thank you very much.

 

In particular with respect to the foreign inspections and what's going on internationally, having spent a fair amount of time in China, Russia, and India and met some of your inspectors firsthand that were on the ground and living in those countries, it makes a huge difference.

 

In 2007 when the Science Board issued its report on mission at risk, that year the appropriated funds for FDA were less than the Montgomery County School District.  And one of the big problems, of course, is having enough resources not only to do what you need to do domestically, but having these increased activities overseas, and rightly so, I'm sure drains both sides of the budget.

 

So today, can you tell us where we are and what your views are towards the future in terms of some of the most critical needs that you're not able to meet or have not been able to meet, especially on the international side?

 

HAMBURG:  Well, thank you for the question and thank you for your past work.  Gail actually chaired a subcommittee of our Science Board back in 2007 and issued a very important report that was called "FDA's Science and Mission At Risk" I think.  And it really shone a spotlight on the terrible disconnect between what FDA needed to do, was required to do and expected to do, and the resources that we were given to do it, and really reminded people about the fundamental importance of FDA as a science-based, data-driven agency and that if we didn't have the resources to support that, you know, everything else would not be possible.

 

You know, the resource issue is such a huge one.  And it's actually startling to me how under-resourced we are, given our importance as an agency.  I mean, some of you in this room have probably already heard me quote the statistic.  But if you look at our budget authority, what the government gives us to support our activities, and you divide it up by every American, it's eight dollars a year.  And you know, it's hard to find, even in our poorest communities, a household that isn't using at least eight dollars worth of FDA-regulated products every single day.

 

And you think about the sort of cascading impacts that the FDA has on the health of individuals and families and communities, the health of our health-care system and the health of our economy and, ultimately, our global economic competitiveness as well, and then you look at how we are supported, you know, and you sort of have to wonder if people are really thinking about return on investment.  Because I think there are very few agencies that actually deliver more for the resources that we're given than the FDA. 

 

And I think everyone recognizes that if we are not functioning well, not only are we not delivering, but we're a drag on the systems.  But if we can function well, you know, and many people refute this, I suspect, but I do believe that our regulatory role enhances industry.  It certainly enhances health, safety, and quality of life.

 

But we have to be able to do it well and adequately resourced.  And the international issues, you know, are just compounding our problems enormously.  We've been able -- you know, this is another long discussion, but we do have a certain amount of support from user fees that are negotiated carefully every five years with industry.

 

We had a big breakthrough a few years ago with the generic drug industry who agreed to pay user fees for the first time to enable us to do a better job dealing with generic drug applications, which as you all, I think, know really matters in terms of making important drugs more available to people.  And now I think 80 or 85 percent of prescriptions filled in this country are generic drugs, and that's saved, you know, billions of dollars and has saved lives because people that couldn't afford drugs can get them now.

 

But many of those generic drugs are made in other countries.  India, of course, has a very high concentration.  And we're required by law to inspect those facilities before we can approve a generic drug.  And getting people over there and doing those inspections was just enormously challenging.  We're getting farther and farther behind.

 

Now we have resources that more closely match the task and we are getting out from under; we're not there yet.  But I think by 2017, as we committed, we will have the backlog taken care of and we will, you know, then be in a better position going forward.

 

But you know, people like to say, you know, doing more with less.  But at a certain point you do less with less, and I think, you know, we have to keep a close eye on the FDA to make sure that in fact we have the resources that we need to do the job.

 

We've tried to be as smart as we can about available resources.  We've tried to leverage resources.  We've tried to partner in ways that can extend our reach.  But at the end of the day, you know, there are critical tasks to be done and we need to be equipped to do them.

 

BOLLYKY:  Great.

 

Doug?

 

QUESTION:  Thank you.  Very interesting discussion.  My question -- first of all, Doug Raymond, Endgame.  My question has to do with some of the public health risks that are behavioral in addition to the substances.  So I think about our obesity epidemic which, you know, relates to the underlying healthfulness of our food supply, but also to people's behavior.  What role do you think government should have in addressing that problem?

 

HAMBURG:  Well, FDA, I think, does have an important role and I think we have made some important contributions in recent years.

 

Obviously, the ability for people to understand what's in the food they eat and how that fits into the kind of diets that promote health is very, very key and we have an essential role in aspects of that.  And it matters for obesity, it matters for many other important chronic diseases that are taking more and more of a toll on people in this country, heart disease, stroke, diabetes, et cetera, as well as obesity.

 

But you know, one example is our nutrition facts label which I think everybody has come to know over the years in terms of the information that's listed on the back of the package.  We're trying to update that to reflect current nutrition science and current eating habits so that people can really understand what's in the food that they're eating and hopefully make more-informed choices.

 

Also, we recently finalized regulations around menu labeling, and that will enable people dining out at restaurants that are part of a chain of twenty or more or hopefully not eating from vending machines too much, but the labeling requirements apply to vending machines, too, to get information particularly about calories, but other nutritional information as well.

 

You know, the kinds of things that I'm describing here have to be part of a bigger strategy of education and support for other activities that matter for health as well.  But I think we can make a real contribution.

 

As we've been working on some of these issues, I was surprised to learn how limited some of the fundamental knowledge about nutrition actually is in this country that, you know, the average person actually has no idea how many calories they should be taking in a day.  So we can give them better information about the calories in a product, but they still need to be informed about the bigger picture.

 

But whether it's nutritional information or what we're trying to do.  I see Matt Myers sitting there -- in the smoking area, our opportunity to regulate aspects of the problem, but do that as part of a more comprehensive program that will really lead to lasting behavior change and change in how our society addresses some of these really important health issues is absolutely essential.

 

BOLLYKY:  Libbie, in the center.

 

QUESTION:  Hi.  I'm Libbie Prescott, I'm with the State Department.  And I just want to say thank you for your service and also for elevating the role of international and the possible opportunities for FDA to play in that.

 

My question is a little bit different, so focusing more on the rise of big data.  And the systems that are in place are designed to regulate material products.  And I know that there's been some advances made, particularly in the space of 23andMe and others, but how do you see the future of the intersection between health data and products coming on the market?

 

HAMBURG:  Well, I think, you know, the whole discussion around big data is an interesting one right now.  I worry sometimes it devolves into jargon and people sort of think, collect a lot of data and the answers just fall out.  You know, I think you have to really know what questions you're asking and make sure that both your data is collected in a way that it's usable and that you then mine the data in a way that gives you meaningful answers.

 

But it's really important.  And we are increasingly looking to big-data strategies in many different aspects of what we're doing.  It's a big-data strategy that really underlies our risk-based screening at the borders that I was mentioning, something called Predict.  But it's also big data that enables us to follow drugs as they go into the marketplace, to learn more about emerging safety issues and efficacy and indications for appropriate use of these products where we're now tracking tens of millions of people in terms of electronic health records and existing data through something that's called the Sentinel program, which is a post-market surveillance program.

 

I think it's like 117 million lives or something that we're tracking.  But in any case, it's a big pool of data that we're accessing through electronic health records and existing information from health systems and insurance payments and other things. 

 

And it enables both to look for emerging issues or concerns, and also if there's through another mechanism a concern reported to us we can query this database and ask, you know, does it look like there's some relationship between this particular product and this potentially unexpected, adverse event.

 

So that's proving very, very valuable.  But it's only as good as the data on which it rests.  We still have huge problems with the interoperability of electronic health records.  We have issues about how data is collected so that the case definitions align and that the data reflects not just what might be put in the system for billing purposes, but what actually reflects what the patient has, et cetera.  So that's all important.

 

And then on the front end, you sort of asked about -- as we learn much more about some of the underlying mechanisms of disease, in particular key genetic and other biomarkers, we can look at data and really start to drill down.  Is there a subset of responders to a given product that if you just studied everybody it might get washed out because it's going to work for some people, but not for others and if you pool them all together you won't see the benefit?  But if you can start to identify and tease out, you can actually identify that the drug has important benefits.  Or in some cases, you can identify that there's a subgroup that has an unacceptable adverse event associated with it.  So that's another use of big data that's on the front end in terms of better understanding approvals.

 

So there's going to be a lot more work in this area.  It's an area where we've been scaling up our expertise.  It's also an area where if we're going to really do it right we need to be partnering with the experts external to FDA as well, and we're doing a lot more of that, too.

 

BOLLYKY:  Great. 

 

We have a lot of questions, so I'm going to try to get to everybody.

 

The gentleman in the back right there who's holding up his hand.

 

QUESTION:  Hi, Commissioner.  It's Bill Nitze with Oceana Energy.  And I have a follow-on question actually to your last comment.  I understand that it can cost four hundred million dollars for, say, Pfizer to get full approval of a new drug, just to pick a number.

 

HAMBURG:  Usually people say more than that in fact.

 

QUESTION:  I'm low-balling.  I've heard numbers as high as seven hundred million dollars, but a lot of money.  And my question is, if you were to adopt an approach where you approve new drugs for very limited classes of people and then, based on experience, gradually expanded the range of that approval, would that create a better cost-benefit ratio?

 

And on the other side of the issue, I've heard that certain already-approved drugs, for example, show promise against Alzheimer's disease -- Amyloid, Plaques and Tangles, and so forth.  And so can you do the reverse in the sense of looking at already-approved drugs and approving them from targeted uses that were never thought of before?  So any comments on how this issue is being managed?

 

HAMBURG:  OK.  Well, first, I think it's important to understand that the costs of developing a drug are not costs that are imposed exclusively by the FDA and the regulatory process.  It's the scientific research to identify a promising discovery that might bear on a disease and then develop it, understand, you know, whether in fact this is something worth developing and then the costs of actually developing it.  We have an important role to play in that process because we do have standards and expectations for a demonstration of safety and efficacy and also that the product can be reliably manufactured, reproduced, scaled up.

 

But it is certainly the case that if we work closely as partners in the scientific process, we can actually accelerate part of that process.  And I think this is one of the recognitions of a new way of doing business for the FDA, because it's not that we're, you know, going into business with the product sponsor, but we are sitting down with them early to say these are the kind of studies that you should be doing in order to really get the data that you're going to need for product development, these are the studies you don't have to do, these are the ones that you should do.

 

And also, working in partnership with academia, industry, and government to invest in certain kinds of research that give us new tools and understandings about the underlying nature of disease and new tools to streamline studies that aren't being done by one particular product sponsor, but can advance our understanding of whole areas of disease, like if we can identify and validate surrogate markers or understand a genetic marker that can be used to speed a clinical trial, et cetera.

 

So there's a lot going on in the realm of product research and development and innovative approaches that includes FDA, but isn't exclusively over at FDA to try to reduce costs and streamline development, including trying to create a less-cumbersome clinical trial infrastructure, which, again, is not strictly an FDA issue, but we see the impact because that's the part of the development process that takes the most time and is the most costly.

 

In terms of studying drugs and approving drugs that were developed for one purpose, for another, you know, that happens a lot.  It also is the case that physicians can use drugs that are approved for one indication, for treatment of another indication.  We don't regulate the practice of medicine.  And in certain areas of medicine, such as oncology, it happens a lot.

 

We think -- going back to the big-data question -- there are a lot of opportunities since in the real world, in the practice of medicine drugs are being used for off-label indications, to continue to study that so we can really figure out what works and what doesn't.  Because sometimes you believe that something is having an important benefit for patients, and when you study it a little bit more intensively you find out that in fact not.

 

So in the interests of patients, you know, we're always interested in learning more and actually making sure that we don't just go on hunches or anecdotes, but actually develop the database to support the most appropriate uses for patients.

 

But you know, there are some major programs under way.  NIH has one for re-purposing of drugs.  But there are complex dynamics there also in terms of the incentives for industry because for many of these products that have already been approved for one use they're no longer on patent, and so the incentives to actually do the studies to identify other potential uses are fewer.

 

But there's a lot going on.  And you know, what's exciting to me is that it's the advances in science and technology that are driving the opportunities that ultimately will make better products for patients and, I think, over time will make the process more streamlined and help to bring costs down.  We're not there yet, but I think it will come.

 

BOLLYKY:  Great.

 

Given our time, I'm going to take questions in batches of two, people I've seen holding their hand for a long time.

 

So this gentleman here in the bow tie, John Monahan.  We'll take those two, then Sabrina and the woman here at the front table who's been very patiently waiting.  And I'll try to circle through.  But for this first round, the two over here.

 

QUESTION:  Rick Gilmore, I'm chairman of the Global Food Safety Forum, and we have an office in Beijing.  And my question is, China still has the highest ratio of cargo rejections of any foreign supplier of food or food ingredients to the United States.  I wonder if you'd comment on the notable achievements, which I think are considerable, that FDA has made vis-a-vis China and the regulatory environment there in the time of your tenure.

 

BOLLYKY:  And then John.

 

QUESTION:  This is John Monahan, Georgetown University.  First, I want to thank you, Peggy, for your public service.  I for one am grateful and I think millions of people should be grateful that you didn't take my advice about the challenges of the position.

 

(LAUGHTER)

 

My question goes back a little bit related to where this gentleman's question goes, but to improving regulatory capacity in low- and middle-income countries.  It seems obviously there's an argument to leadership in countries about protecting the health and safety of populations.  But there's also an argument about the ability to strengthen a country's economic position and its competitiveness with a robust regulatory system.  It seems to me that's a hard argument to make.  It's been more or less successful, but I would be interested in your reflections on having made that argument multiple times over the last few years about how can we make that more persuasively.

 

HAMBURG:  OK.  It's hard to answer both quickly.  You know, as far as China goes, and China is one country with lots and lots of exports to the United States, but there are other countries as well that have similar issues, you know, we are seeing more openness, we're seeing more ability to work with their regulators and their industries, we're seeing more interest in understanding our standards and meeting them.

 

They also, I think, are recognizing that we're serious when we say we have standards.  And if they don't let us into one of their food facilities to do an inspection, for example, we don't let their products come in.

 

And obviously, when there's a threshold in terms of repeated problems, we just put an import alert on, and every single thing from that company or everything in that product category is going to get inspected.

 

We are, you know, we have enough enforcement teeth now that, you know, we're taken seriously, but we don't have the resources to actually keep it up on a long-term basis.  So enforcement is not the long-term strategy.  It's improving the standards.

 

And China's an interesting case.  You probably know more about it than I, and this would be a whole other set of discussions.  But you know, I'm sort of interested in seeing how the Chinese public is going to drive improvements in food safety that are going to benefit their own country and benefit us.  Because they've had enough serious problems in China now that the public is starting to speak up and starting to demand more action. 

 

And I think, you know, for better or for worse, one of their FDA commissioner equivalents was actually executed over some food-safety-related issues at an earlier point in corruption.  So you know, they're sending some signals that they're taking it seriously.

 

When I took this job, I said to my kids, because the initial, you know, press coverage was pretty positive, I said, you know, it's not always going to be that way and sometimes, you know, people are going to say things that are, you know, really very harsh and sometimes it's going to be unfair and sometimes it will be fair because I'm going to make mistakes.  And my daughter very earnestly said, as long as they don't execute you like they did the guy in China.

 

(LAUGHTER)

 

But in any case, you know, I think, you know, this is a dynamic process.  You know, we're always wanting things to happen really fast and change in some of these countries takes time.  And we've had a very hard time in many of the countries that we work in convincing our host country that it's actually a benefit to them to have us in there.  Some people in this room know how hard we fought to get visas for some of our inspectors in China because they really weren't sure they wanted us there.  They let us in, they'd gotten a taste of what we were doing and they weren't really sure that they wanted more.

 

But in fact, one of our advocates for more inspectors was the Chinese FDA because they knew that they were benefiting from our presence, both in terms of industry taking standards seriously, but also they were getting educated because we were letting them come on inspections with us and they were learning how to do a better job and a job that they needed to do.

 

So you know, it's going to take time, but I think we're making progress and I think there is a new level of openness and cooperation in countries, to be honest, that didn't used to want to really work closely with us and were sort of skeptical of some of what we were trying to do and now are having their own problems with imported goods.

 

And so, you know, everybody is increasingly seeing this as a global challenge where the only way to do it is to work together and to strengthen standards.  And I guess that comes back to, you know, the question you were asking, is that not only do we need to do it to improve the quality and safety of products, you know, whether you're a high-income country or a low-income country.

 

But you know, I've been pushing and I think it's really true that as a development strategy, as we think about investments in low- and middle-income countries to strengthen health systems, we need to also include a regulatory component because the health system is not going to be adequate and serve the people well unless there are safe, high-quality products in that health system.  And we know that if we add the component of regulatory capacity, not only do you strengthen the health system, but you're actually going beyond that to strengthen the small and growing industries in those countries. 

 

It's economic development that really works and really matters.  And I think it makes, you know, really for, come back to it, a good return on investment, but the kind of investments that people really haven't been thinking about before.

 

BOLLYKY:  Great.

 

And I would say amen to that, particularly with global production models where you have countries getting a slice of production, the ability to have consistent and adequate regulation, predictable regulation is very important.

 

Sabrina and then the woman up here.  And we have nine minutes left, so keep your questions short and then we'll try to get to as many people as we can.

 

QUESTION:  Commissioner Hamburg, thank you.  My question -- can you talk a little bit about twenty-first...

 

BOLLYKY:  Do you want to say who you are?  Sorry.

 

QUESTION:  Oh, sorry, New York Times -- twenty-first century cures and what you think the sort of objective is there, if they have any -- there's draft legislation in Congress right now -- if it has any point that FDA approvals are too slow, if there's sort of slowness, where it is over the course of drug development, and just sort of talk a little bit about how you see that legislation and how you see kind of, you know, do they have a point in criticizing FDA?

 

HAMBURG:  Well, I think this is a really critical time to be looking at the whole ecosystem that supports medical product development and the contribution of the many components.

 

I think most people, you know, whether you're a venture capitalist, you're a patient advocate or you're a CEO in industry would say that it's much more complicated than just how long it takes for FDA to review a product.  In fact, our FDA drug-review times have, number one, declined enormously over the years.  Number two, we outpace every other developed country in the world substantially, and the majority of new drugs that are approved anywhere in the world are approved in the United States first.

 

If you look at our recent new-drug approvals, last year it was a near twenty-year high in terms of new-drug approvals.  There were not just more drugs, but first-in-class drugs and a large proportion of cases that really offer, you know, new treatments for patients where before there was none or inadequate therapy.

 

And most of them, I think three-quarters, involved some kind of expedited, more streamlined pathway for approval.

 

Where I think we need to be really careful is to confuse the FDA regulatory review process with where the science is.  And it is disturbing to me to hear people talk about if you'd just lower standards we'd get more drugs.  You'd get more drugs, but they wouldn't be drugs that would work and that would have a meaningful benefit for patients.  And it's not a question of how long it takes for the FDA to review the drug, it's a question of where the science is.

 

So I hope that twenty-first century cures will really focus on, how do we make sure we're making the right investments in science to really advance our understanding of human biology and the underlying mechanisms of disease to really advance the development of the regulatory tools that we need to be as efficient as possible as well and to make sure that FDA has the resources so that we can do our job as efficiently as possible.  And I think all of that is crucial.

 

So you know, my major -- I'm, you know, enthusiastic about the underlying concept of twenty-first century cures and have actually been in various fora pushing the need for a more strategic approach to biomedical product innovation for a long time.  But I think it's really important to take a clear-eyed look at all the different components that contribute to biomedical product innovation and focus on where the problems really are and not try to fix the wrong problem. 

 

And I think, you know, right now we've got a lot of serious, unmet medical care and public health needs that we need to be able to leverage science to advance new treatments, cures, preventive strategies.  But I think that the answer is not as simple as some people would frame it, which, you know, would be to just reduce standards.

 

BOLLYKY:  Great.

 

Over there and then over here.

 

QUESTION:  Very quickly.  Thank you again for your presentation today.  Reba Carruth, McCourt School, Georgetown University.  Very quickly, I just wanted to follow up on points you made earlier.

 

One of the things I'm noticing is that the FDA and the other regulatory agencies in the United States are really focusing more and more on international regulatory cooperation and interagency cooperation.  So for the FDA, particularly in the area of antibiotic drugs and antimicrobial drugs, this One Health approach is really gaining focus, not just in the U.S., but transatlantically and also with international organizations like the World Health Organization.  Could you talk a little bit about where we can go with this in terms of building on this platform to train and converge regulatory capacity worldwide?

 

HAMBURG:  OK.

 

BOLLYKY:  And one more.  This one here.

 

QUESTION:  Thank you.  Marsha Echols from Howard's Law School, the World Food Law Institute.  On the global framework also, there are many different players in the global marketplace for foods.  So we have small-island countries, we have China and Thailand, we have Brazil, Russia, the United States.  So for those who are sending foods in the United States, there are many different levels of players with different capacities.  And for American companies, those who are involved in international trade distributing, importing, using the ingredients, there are different sizes also. 

 

So what a large mass-producer might be interested and do is different from one of our artisanal producers.  So I wondered whether the idea is for -- whether it's for products coming in, looking at our companies, or exporters from other countries.  Are you saying there's one size that fits all?  Does everyone have to basically follow the same rules?  Are you educating for the same kinds of actions by governments of different sizes?

 

BOLLYKY:  Great.

 

And I want to also say you have three minutes, so make this your closing remarks as well.

 

HAMBURG:  No.  I've repeatedly said one size doesn't fit all, but the point is, you know, when it comes to food safety, for example, whatever you're making, however big you are, you have a responsibility to understand, where are the risks in the growing and the manufacture, the packaging and the distribution of that product?  Where are the risks that can introduce contamination, and how can you shore those up so that your products in fact are safe and wholesome?  And I think that that is what we're trying to get at.

 

Now, let's see, your question -- I think, you know, the fundamental issue there,  you mentioned One Health, is a notion that, you know, we can't operate in isolation, that we have to look at how animal health and human health align, whether it's challenges like Ebola where we know that emerging infectious disease threats more often than not first arise in animal populations and then take hold in human populations and can cause devastating disease, or a problem like antimicrobial resistance where we can't address it alone by focusing on the responsible stewardship of antibiotics in humans or assuring pathways to develop new antibiotics. 

 

We need also to recognize that there's a lot of antibiotic use in animal populations.  It's an ecosystem.  And we need to be working on all of these problems in a comprehensive way. 

 

It comes back in some ways to what we were talking about earlier in terms of behavioral health and behavioral challenges that require not just one magic bullet, but a comprehensive approach.  It comes back to what was just asked about twenty-first century cures and how do you speed new-product development.

 

We have to be thinking in much more comprehensive ways.  And I think that, you know, we are coming out of an era where things were done in stovepipes, where agencies, you know, worked within their walls and where we did not recognize, you know, the critical and essential need for collaboration across components of government, state, local, federal and international. 

 

We didn't recognize the full importance of collaboration across disciplines.  We didn't recognize the importance of collaboration across sectors.

 

And I think, you know, that really in our modern world with all of its complexities, you know, wherever you sit this is going to look a bit different.  But that is one of my big take-homes in terms of my experience with the FDA, was that I came to an agency, you know, that was, you know, a little bit insular. 

 

You know, some people accused us of being a black box, but you know, I have worked hard to sort of open us up, to try to explain what we do, how we do it and why, and to engage with partners in order to get our important tasks done.  I think it's the only way to deal with the complexity of the challenges before us.  It's the only way to deal with the realistic limits on resources as well, and that in fact is dollar and human resources.

 

And I think it is the pathway forward for the future.

 

BOLLYKY:  With that, I just want to say that, from my own experience as somebody having been in government and had been an interagency partner with FDA on a lot of these global issues, it really has been a sea change.  And I think Commissioner Hamburg deserves an enormous amount of credit for that as well as for her service.

 

So please join me in thanking her. 

 

(APPLAUSE)

 

END