COMMENTARY

FDA's Hamburg on Balancing the Risks, Benefits for 310M Patients

; Margaret A. Hamburg, MD

Disclosures

November 12, 2014

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A Distinguished Start to a Distinguished Career

Eric J. Topol, MD: Hello. I am Eric Topol, Editor-in-Chief of Medscape. Joining me today is Dr Margaret Hamburg, commissioner of the US Food and Drug Administration (FDA). I am interviewing Dr Hamburg as part of our series on the most interesting people in medicine.

You have a remarkable history. Your parents were both physicians. Your mother was the first African American to graduate from Vassar and Yale Medical School. Your father was president of the Institute of Medicine (IOM). You are perhaps the only family whose members have all been elected to the IOM.

Dr Topol: On your children's birth certificates, you are listed not just as their mother but as the New York City health commissioner.

Dr Hamburg: That's right. They may be the only two kids in the history of New York whose mother signed their birth certificates in two places. We are very proud of that, although when I looked at their birth certificates, I realized that we needed to upgrade them because they aren't suitable for framing.

Dr Topol: You graduated from Harvard Medical School?

Dr Hamburg: Yes, and I did my residency in internal medicine at what is now New York Presbyterian Hospital-Weill Cornell Medical Center.

Dr Topol: You have spent time in both neuropharmacology and infectious diseases.

Dr Hamburg: I had very strong interests in neuroscience and endocrinology, and I did research at the National Institute of Mental Health on the National Institutes of Health (NIH) campus before I started medical school and the first summer after medical school. I was able to do some research in neuroscience at Rockefeller University while I was a medical resident next door at New York Hospital. Those were very powerful, strong interests. I thought that I wanted to subspecialize and do academic medicine and follow in the footsteps of my parents, who were professors and physician-scientists at Stanford Medical School when I was growing up. I took a different path, but then like them I ended up broadening into areas of policy and public service as well.

A Laser Focus on Science

Dr Topol: Maybe you had an influence on them, too. You have quite a rich background in public service, government, and in health and human services at different levels. You were at the NIH and also spent 6 years as the New York City commissioner of health. Did you interact with Rudy Giuliani?

Dr Hamburg: I did. I had the privilege of working as the city's health commissioner for 3 years under Mayor Dinkins and 3 years under Mayor Giuliani. I take great pride in that because I believe that jobs such as health commissioner of New York City or FDA commissioner should be very much professions driven by science, medical care needs, and the best public health practice. To be able to serve under a liberal Democrat and a visible Republican speaks to the ability to serve in a professional role regardless of the more complex politics of the environment, and the importance of these roles in terms of continuity regardless of political party.

Dr Topol: You accomplished a great deal during your tenure in the areas of tuberculosis and HIV.

Dr Hamburg: It was a fascinating time.

Dr Topol: Before you came to the FDA, you were at the Nuclear Threat Initiative for several years. Tell us about that.

Dr Hamburg: It was called the Nuclear Threat Initiative, but it was focused on reducing the threat of weapons of mass destruction. It was a new foundation started in January 2001 by Ted Turner and former Senator Sam Nunn. They asked me to come on board to help start up the foundation and a program on biological threats. I said that I would participate if we could focus not only on biological weapons and biological terrorism, but also on naturally occurring biological threats, including such threats as Ebola, because we all recognize that Mother Nature can be a pretty effective terrorist in her own way. I developed these interests when I was the New York City health commissioner. I was in that position the first time the World Trade Center was bombed. I started taking domestic terrorism very seriously and began to focus on the areas of responsibility for a public health agency and the need for better public health preparedness against a range of threats.

New York City was also a hub for international travel, and this meant that we had to be aware of what was happening elsewhere in the world in terms of imported disease and strategies to address those. It was very similar to what is going on with Ebola now. We were dealing with very serious epidemics of disease in New York City, including HIV and the resurgence of tuberculosis, which now has more frightening forms, including the development of drug-resistant tuberculosis. I became deeply committed to the area of biological threats and the role of public health preparedness in addressing them.

At the Helm of the FDA

Dr Topol: Speaking of prepared, no one could have been better prepared to take on the role of FDA commissioner. Has this been a dream job for you?

Dr Hamburg: Not at all. I joke that if someone had asked me even a month before I was approached for the FDA job, "Could you see yourself as FDA commissioner?" I probably would have put down a large sum of money with the answer "No." But it has been a terrific opportunity and it is an inspiring place to work in terms of its critical and unique mission. We are a science-based regulatory agency with a mission to promote and protect the health of the public. We are unique. We matter in peoples' lives every day. The products that we regulate and oversee range from the safety and effectiveness of drugs, vaccines, medical devices, and other biologic and medical products to the safety of the blood supply, the safety of most of the US food supply, dietary supplements, cosmetics, and most recently tobacco products. We regulate products that are estimated to account for a little more than 20 cents of every dollar that consumers spend on products. We matter in terms of impact on the lives of individuals, families, and communities, and, more broadly, on the economy.

Past Challenges and Outcomes

Dr Topol: You are overseeing $1 trillion of the economy every year. That is a lot to look after.

Let's discuss some of the challenges that you have had to face along the way—for example, Plan B back in 2011. You had an entire emergency contraception drug set up and ready to go over-the-counter, and then-Health and Human Services Secretary Kathleen Sebelius said, "No, we are not going to do that." Was that experience upsetting to go through?

Dr Hamburg: It was a difficult situation, but it was a situation where I knew what I needed to do as commissioner and that was quite clear. Our job is to look at the science and the data. The company that made this product (Plan B) had approached us with an application for an over-the-counter product that would lower the age of access to this product. They had completed the studies, and the science clearly supported approval of their application. That is the compass that we need to use for decision-making.

Dr Topol: It seemed to get trumped by politics.

Dr Hamburg: It is a complex political and emotional issue. We all wish that we lived in a world where 14-year-olds didn't need access to emergency contraception, and if they did find themselves in that situation, they would have a trusted adult, either a parent or a healthcare provider, to help them make that decision. However, in that moment of crisis, there also are clear health and behavioral benefits to being able to access this product. We knew from having a long experience with this product, and from the data that had been presented from the studies done by the company, that it was safe and effective and could be used appropriately in the over-the-counter setting by the individuals.

Dr Topol: It eventually got straightened out.

Dr Hamburg: It is now in the marketplace.

Dr Topol: What about the drug compounding issue, trying to regulate these rogue compounding pharmacies? That must have been a tough issue.

Dr Hamburg: This was another complicated area for the FDA and certainly an area that had a strong impact on the health and well-being of the American people. It is an issue that we continue to be deeply involved in. There are compounding pharmacies in almost every neighborhood of this country that are very legitimate pharmacies regulated by the state in terms of pharmacy practice and are available to compound products that people need. If you are in need of a product but you have a swallowing problem, the compounding pharmacy can take the pharmaceutical that you need and prepare it in a way that you can use it. Many pediatric treatment needs are compounded to make them more usable by younger children. That has clear benefits.

The problem was that the industry was evolving as the medical care system was evolving. Some of the compounding pharmacies had shifted into a different mode. Rather than being the "neighborhood compounder" that makes a product with a specific prescription for a specific patient, they were making large quantities of often complex products, such as the sterile injectables that are of particular concern because compounding that is not done correctly can create real quality risks and concerns for patients. These products were being marketed across the country. This had been an area of concern for a while—the gray areas in terms of practice and ensuring the best products for people who need them—but the situation came to a head when there was a contaminated steroid product that led to the deaths of more than 70 people and illness in hundreds more.

Balancing Benefit and Risk in Pain Management

Dr Topol: What about opiates? There is a big concern about opiate abuse. Could you comment on that?

Dr Hamburg: The FDA is always working in an environment in which complex decisions must be made, balancing risks and benefits and working within a legal regulatory framework. The opiate issue is a huge area of concern in terms of the public health epidemic that we are facing with opiate addiction, abuse and misuse, overdose, and preventable death. At the same time, many patients need effective pain management either for acute postsurgical pain or for the treatment of chronic conditions. We need to balance those issues. We need to ensure access to safe and effective pain medication but recognize that there is a broader context of abuse.

We are doing several things related to this issue. We are working with the research community and with product sponsors to try to develop opiate drugs that are more resistant to abuse, and that is a challenge. The science and technology are evolving, but there are approaches to making some of these less easily abused. For example, if crushed, they turn into a gel, so they can't be injected or snorted. Other interesting and more sophisticated technologies are in development. It's a difficult challenge. Most of the abuse is with oral products, and that is the hardest to address because you want to have the drug be available to the patient when they take it for the treatment of pain.

We are also trying to work with the scientific community and product developers to find other pain medications, especially in the chronic disease arenas. Opiates are often not the most appropriate treatment, but they may end up being the only available treatment for patients. We are seeing new categories of treatment for certain kinds of pain. That will make a difference in providing better care for patients and also reducing the public health epidemic of opiate abuse.

Dr Topol: That sounds like a great solution.

Dr Hamburg: We need to do a better job with treatment. We don't have the best drugs for the treatment of addiction. As physicians, we are not always adequately trained about how to recognize addiction, and we don't have the addiction treatment networks that we need when referrals need to be made. We all need to learn more about appropriate use of these powerful opiate drugs.

e-Cigarettes: FDA’s Proposal to Regulate

Dr Topol: You touched on tobacco and addiction. That brings me to e-cigarettes. Do you have any thoughts about those?

Dr Hamburg: Legislation was passed several years ago that, for the first time, gave the FDA the authority to regulate tobacco products, but it was cigarettes, smokeless tobacco, and roll-your-own tobacco, and some tobacco products that are currently in the marketplace weren't included in that initial legislation. We have undertaken a process to extend our regulatory authority over these other tobacco products, including e-cigarettes. That is a critical first step.

Then we will need to look very hard at e-cigarettes. The public health and medical communities are divided on the appropriate role of e-cigarettes. Many people are deeply concerned (and I can understand why) about the attractiveness of these new products, especially to young people. We are seeing an uptake in use in adolescent populations, but the numbers are still relatively small. These products are flavored. You can buy mint Oreo-flavored e-cigarettes and other flavors that are clearly targeted to attract children. We don't want to see young people start a nicotine addiction and then go on to combustible cigarettes and other tobacco products that have not only nicotine but other carcinogens and toxins as well.

Many people are encouraged that e-cigarettes can be a useful tool for smoking cessation and shifting away from the harms of traditional combustible cigarettes. The FDA is putting a lot of money into research to better understand e-cigarettes and other tobacco products and their uses. We are working with academic institutions, doing grants, and working with the NIH on a major longitudinal study that will give us a lot more important information as we continue to address the critical challenge of tobacco use and smoking, which remains the leading preventable cause of death in this country.

Dietary Supplements: Postmarket Role Only

Dr Topol: It's amazing. We still don't seem to be in touch with that fact. Speaking of a lot of money, supplements are a $30-$40 billion/year industry. Does the FDA have authority over supplements?

Dr Hamburg: We do not review dietary supplements for safety and effectiveness before they go into the marketplace the way that we do for medical drugs and devices.

Dr Topol: Is that because that industry has lobbied to prevent that?

Dr Hamburg: The legal constructs and the regulatory framework that we have been given by Congress asks us to monitor the safety of dietary supplements in the marketplace. Companies are required to report serious adverse events to us. We have the power, when there is a problem, to recall dietary supplements, and we have the authority to monitor the production of dietary supplements in terms of the quality of manufacturing, but that is only one aspect. Many American consumers assume that the FDA is doing more in the regulatory oversight of these products and that we are engaged in assessing the premarket safety and efficacy of these products.

Personal Genetics and Genomics: Doing It Right

Dr Topol: Another area that is somewhat controversial is consumer genomics and such companies as 23andMe and others. Some people want to get their genomic data, and a lot of other companies are marketing some very questionable genomic-type data. Do you have any thoughts about where that might be headed over time?

Dr Hamburg: Obviously we have entered an entirely new era, thanks to advances in science and technology. We are very enthusiastic about the opportunities that precision medicine present to develop better drugs that can target individuals in new and important ways by focusing on individual characteristics, disease characteristics, and patient needs. Diagnostics, genomics, and next-generation sequencing are very important components of what is already happening and what will happen in the future.

We support the notion of consumers and their healthcare providers having access to this kind of information, but we feel strongly—whether it is a genetic test or any other kind of a diagnostic test—that the test be accurate and reliable. If important decisions are going to be made, if that information is going to be actionable in critical ways, consumers deserve accurate and reliable information.

Dr Topol: It's such a broad issue. You have to inspect facilities not only in the United States but throughout the whole world. Your responsibilities are beyond comprehension.

Time for a National Drug-Cost Debate?

Dr Topol: The last thing that I want to ask you about is the cost of drugs. In other countries, cost is reviewed by government. In this country we have been immune to that. The FDA reviews everything independently of the cost. Now we have runaway specialty drug costs, such as cancer drugs, and many new approvals, but we don't have any system of checks-and-balances on the cost of these drugs. In other countries, drug costs are a fraction of what Americans are paying. Will we ever get to a time when the cost of drugs is reined in?

Dr Hamburg: We do have a very different system in many ways from other countries. The FDA is prohibited by law from taking cost into account when we do our scientific reviews.

Dr Topol: Can we change the law?

Dr Hamburg: It's a complex question and one that is worthy of discussion. There is value in the FDA focusing on the science. That has been a very important, if not essential, compass for our work, but we have to recognize the broader context in which these products will be used. We have an agency (the Centers for Medicare & Medicaid Services) that makes decisions about reimbursement, and they have a very powerful role in terms of setting costs and what they are willing to pay. They also influence other private payers. There is a reimbursement framework that is active in this ecosystem. We have reached a point where we have to have a national debate on these issues. We have to sort out how we are going to deal with all of this.

My hope is that advances in science and technology will continue to bring us new and better drugs, and perhaps the development costs will be lowered by the increasing sophistication of the drug development process. At the same time, we have to look at the whole ecosystem and recognize that we just can't continue to provide the care that American patients expect without addressing the broader cost issues.

The Rewards of Public Service: 310 Million Patients

Dr Topol: You have been incredibly dedicated to public service and are a great inspiration to the medical community, and also to women in science. Can we close with what you would like to say to the physician audience about what you have learned and what impact you have had on others?

Dr Hamburg: I hope that all physicians and others in the broader medical community will think about public service as an important application of their knowledge, skills, and expertise. Public service is incredibly rewarding. It's a bit of a shift from the care and commitment that you give to an individual patient, to stepping back and doing that for an entire population. When I first became health commissioner of New York City, my great Aunt Winnie was very upset because she wanted me to be a "real doctor." She couldn't understand why I was throwing away my medical education. My father tried to calm her down by saying, "She is a real doctor. She just has about 8 million patients." Now I have more than 310 million patients as FDA commissioner.

Dr Topol: That is a pretty big practice.

Dr Hamburg: Public service is enormously rewarding, and you feel that every day you are making a difference. We need to recognize in medicine that we are working in a much more complex environment, and we have to think about and deal with patients in terms of all of the influences on their lives and all of the different policies and programs that affect them—all of the real-world circumstances, whether it's poverty or other pressures. Being engaged in health policy has made me look at things in a much more textured way. All of the problems before us are complex and multidetermined, and the solutions have to be multifaceted. Most of the real problems that underlie medical care and our desire to promote health are not going to be resolved simply in a doctor's office or in a hospital setting. We have to step back and look at all of the determinants of health.

Dr Topol: We are all grateful to you, not just for your willingness to have this conversation but for all that you have done to look after the American public and set a great standard for the FDA. Thank you so much for joining us for this interesting discussion.

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