Category Archives: Other Topics

Celebrating American Heart Month—Making Heart Healthy Choices in 2013

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By: Janelle Derbis, PharmD

Each year, nearly half of all Americans make New Year’s resolutions. They often include losing weight, starting an exercise program, quitting smoking, and making healthier food choices—all of which contribute to a heart-healthy lifestyle. February is American Heart Month, and the timing couldn’t be better to make these lifestyle changes, especially since heart disease is the leading cause of death in the U.S.

FDA joins in the commemoration of American Heart Month by highlighting agency initiatives to help Americans reduce their risk of heart disease.

Achieve a healthy weight. Obesity contributes to a number of health conditions, including high blood pressure and high cholesterol. To help obese and overweight Americans who have been unsuccessful in getting their weight under control with diet and exercise alone, FDA approved two long-term weight management medications in 2012—Belviq and Qsymia. These are the first medications the agency has approved for the treatment of chronic weight management in 13 years.

Quit smoking. Smoking and tobacco use contribute to many health hazards, including heart disease. Nearly half of adult Americans are at risk for heart disease and stroke, and over 20% are at risk due to cigarette smoking. To address the huge public health problem of tobacco use, FDA is building a national tobacco product regulation program to reduce the impact of tobacco use on the nation’s health. Using powerful new regulatory tools provided by the law, FDA’s work supports the objective of the Department of Health and Human Services to end the epidemic of tobacco-related death and disease in America. In November 2012, HHS announced the availability of a new comprehensive tobacco website, BeTobaccoFree.gov, a providing one-stop access to the best and most up-to-date tobacco-related information from across its agencies. This consolidated resource includes general information on tobacco, federal and state laws and policies, health statistics, and evidence-based methods on how to quit.

Eat right. Consumers can eat for a healthy heart and choose foods that are lower in salt, cholesterol, and trans-fat by reading the Nutrition Facts label on food and beverage packages.  In January 2013, the agency announced it is planning to update the Nutrition Facts label based on the latest science-based nutrition recommendations. The updates are still being formulated, and public input will be sought when they are proposed.

Lower cholesterol levels.  Making lifestyle modifications can help reduce cholesterol levels. However, hereditary issues can make some people more likely to have high cholesterol levels regardless of diet and exercise. The good news is there are treatment options for people who are unable to lower their cholesterol levels. There are several FDA-approved cholesterol lowering medications on the U.S. market.

In December 2012, FDA approved Juxtapid for a rare cholesterol disorder called homozygous familial hypercholesterolemia (HoFH), an inherited condition that makes the body unable to remove the “bad” cholesterol (LDL cholesterol) from the blood, which  can lead to heart attacks and death before age 30. The approval of Juxtapid is an example of how FDA provides the scientific and regulatory advice needed to bring new treatment options to market.

Control high blood pressure.  Adopting a healthy lifestyle can help prevent high blood pressure. If lifestyle modifications are not enough to lower your blood pressure so that it is within the normal limit (less than 120 over 80), medications are often prescribed. There are many FDA-approved medications to treat high blood pressure so talk with your health care provider to determine which is best for you.

In April 2012, FDA approved the first generic versions of Avapro (Irbesartan) and Avalide (Irbesartan and Hydrochlorothiazide) for the treatment of high blood pressure.  Generic drugs such as these provide safe and effective alternatives to brand-name drugs. 

Exercise. And lastly, physical activity is an essential component of a healthy lifestyle and when done in combination with healthy eating can help prevent heart disease. In 2013, make a commitment to exercising on a routine basis and keep your heart strong!

To receive up-to-date information on heart-related drug and device approvals, safety announcements, and notices of upcoming meetings, subscribe to FDA’s CardioBeat or visit FDA’s cardiovascular webpage.

Janelle Derbis, PharmD, co-manages the Cardiovascular and Endocrine Liaison Program (CELP), at FDA’s Office of Special Health Issues.

FDA’s Special Role: Ensuring Food Safety at the Inauguration

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By: Margaret A. Hamburg, M.D.

With the presidential inauguration just days away, it’s an exciting time to be in Washington, D.C. The police are setting up parade routes and security. Hotels and restaurants are bracing for crowds.

Margaret Hamburg, M.D.And the FDA is playing an important part as well. For certain events of national significance, such as U.S.-based Olympic games, national political conventions and U.S.-hosted summits of world leaders, we are called upon to marshal our expertise. This week, at the request of the U.S. Secret Service and D.C. Department of Health, we’ve assembled a team of 35 FDA staff from across the U.S. including 18 experts in retail foods and field inspection. Their mission? To work closely with the D.C. Department of Health, local county health departments in Maryland and Virginia, and the FDA Baltimore District Office to make sure that the food served at the inaugural ceremony and parade, balls and galas is safe to eat.

When you think about it, it’s not such an unusual role for FDA. After all, the agency works hand-in-hand each day with state and local public health agencies throughout the U.S. to ensure food safety.

And that’s what we’ll be doing this week. Our team of regional food experts will work with local health departments to protect food from contamination. We’ll review menus and observe food preparation, storage and service. We’ll train kitchen staff about risk factors, such as cleanliness, food temperatures and refrigeration. And information on food sources and supply chains at venues and vendors will be questioned so that if any foodborne illness is reported, we’ll have data to trace it back to the source.  

2009 inaugural salads

Data from the 2009 inauguration tells us that our inspections covered more than 100,000 meals. We expect similar coverage this time around.

It’s a privilege and an honor, but it’s also an enormous responsibility. Fortunately, we come prepared. We tackle this important challenge armed with years of valuable knowledge and experience in careful, data-based, cutting-edge science. We protect Americans from foodborne illness based on lessons learned over a long period of time. 

In ancient Rome, the emperors had special food tasters to make sure their feasts were safe and poison-free. Washington, D.C., isn’t ancient Rome, of course, and the “poisons” we are looking out for may be dangerous microbes.

But just as the Secret Service is responsible for overall security at the inaugural events, FDA is responsible for managing food safety and security in the retail food venues. We’ll work with the chefs and food services and facilities staff in D.C. to ensure that food safety standards are met. As the FDA Food Safety Modernization Act makes clear, our focus will be on preventing food safety problems before they happen.

The standards that FDA and our local health departments will be enforcing in the nation’s capital are the same as those we have in place every day for you and your families. Whether the lucky guests are at an inaugural ball or eating at a stand along the parade route, they can rest assured – as can you - that the regulations are the same for both, and that FDA is doing its part to help ensure that the food we all eat will be safe.

Margaret A. Hamburg, M.D., is the Commissioner of the Food and Drug Administration

A New Law Advances Public Health: New Web Page Tracks Progress

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By: Malcolm Bertoni and Leslie Kux

After Congress passes a law that affects how FDA carries out its public health mission, we must begin the task of implementing the law — that is, putting the law into effect and enforcing it.

For a major piece of legislation like the Food and Drug Administration Safety and Innovation Act (FDASIA), signed into law in July, this is a complex undertaking.  

Malcolm Bertoni

FDASIA is a 140-page law divided into 11 separate sections, officially known as “titles,” which address different aspects of drug and device law. FDASIA reauthorizes and makes some changes to user fee programs that provide FDA with the resources we need to maintain a predictable and efficient review process for human drugs, biological products (such as vaccines), and medical devices.  

FDASIA also creates two new user fee programs:  one for generic drugs and another for biosimilar biologics. These new programs will allow FDA to enhance its efforts to ensure that American consumers have more timely access to safe, high quality, affordable medicines. The law also gives the agency new authority to protect the safety of the drug supply chain, which is so important when these products arrive from all corners of the world; to combat drug shortages; and to improve products used to treat children. The law includes many other provisions, including those involving drug innovation and device regulation.

The requirements of the individual provisions vary; some direct FDA to write new regulations or guidance documents that will help industry meet the law’s requirements, while some call for the agency to issue reports or develop strategic plans. Some provisions set specific timetables for action, others don’t.

Leslie Kux

The successful implementation of FDASIA is one of our top priorities. To ensure its success, FDA set up a steering committee shortly after the law was passed to oversee the task of integrating the requirements of FDASIA into the agency’s ongoing workload. One of the committee’s projects has been to create a table that tracks what FDA must do to comply with the statute.

Today we are making available a website that will allow you to follow the agency’s progress in accomplishing the actions required by the new law. The website includes a table that lists information about FDASIA tasks such as the citation to the section of the law, a description of the task, the statutory due date, and the name of a primary contact person. The table will also include links to pertinent documents as they are completed and published.

Initially, the table will include only those requirements with a due date set by Congress. In 2013, other requirements that were not given a specific due date will be added, along with FDA’s target completion date.

FDASIA is an important law with significant provisions affecting industry, patients, consumers and health care providers. We will be updating the website on a regular basis as part of our commitment to transparency about our FDASIA implementation.

Malcolm J. Bertoni is FDA’s Assistant Commissioner for Planning

Leslie Kux is FDA’s Assistant Commissioner for Policy

Modernizing FDA’s Information Technology

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By: Eric D. Perakslis, Ph.D.

As I look back at my first year as chief information officer and chief scientist (informatics) at FDA, I am gratified with all of the progress that has been made. But I am also eager to transform initial wins into consistent and sustained success.

My team at the Office of Information Management (OIM) manages and advances information technology at FDA. Our mission is to provide the organizational structure and services that allow the exchange of information, communication and knowledge that enhance and sustain FDA’s ability to protect and promote the public health.

Our vision is that FDA’s technology and knowledge capability is modern, secure, accessible, cost-effective, and exceeds customer and partner expectations.

Some of our early successes include important contributions to what we call the Innovation Pathways 2.0 initiative, which provides a rapid new way to submit breakthrough medical devices to FDA for approval. This in turn would get such products faster to the patients who need them.

OIM also completed 20 projects in very short time in support of the Food and Drug Administration Safety and Innovation Act (FDASIA), signed into law by President Obama in July 2012. Many of these projects strengthen the process of reviewing and approving important new medical products, while making that process more transparent to the tax-paying public.

In addition, this year we launched a new strategic effort to improve the overall “customer experience” with FDA’s information technology, customers in this case being FDA employees stationed all over the world. Our goal is to provide the technology to ensure that FDA employees can reliably perform their daily duties efficiently and timely regardless of location, whether they’re in an office or in the field, or at home working remotely.

And our improvements to the process of analyzing the health risks presented by medical products of all kinds have helped drive the modernization of the FDA inspection process. This helps ensure that the growing number of products we regulate have a higher level of safety and quality before they reach American consumers.

While these accomplishments are significant, there is still much to be done to modernize information technology at FDA.

The new FDA Information Management Strategic Plan sets the path forward. Its priorities include strengthening real-time connectivity and access to key data and information. This is essential for daily FDA operations and for our connections to the public we serve and to our many partners outside of the agency who depend on FDA for the execution of their own public health missions.

The Strategic Plan also focuses on the availability and usability of data essential to the speed and efficiency of decision-making at FDA. The intent of this goal is to facilitate a learning and knowledge network that will enable FDA to assess the potential risks of regulated products on a scale that routinely handles global sources and large volumes of data. 

Going forward, we will do our best ensure that FDA’s technological needs today and tomorrow are fully covered and in place. There is no more inspirational public health mission than that of the FDA. Every citizen of this great nation depends on us every day and we will never forget that. In many ways this Strategic Plan is as much about what we believe, and our commitment to our customers and mission, as it is about what we deliver.

Eric D. Perakslis, Ph.D., is FDA’s Chief Information Officer and Chief Scientist for Informatics

Working for You During Hurricane Sandy

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By: Margaret A. Hamburg, M.D.

You may think that FDA is simply an organization that reviews medical products or works to keep your food safe. But FDA’s broad public health mission includes mobilizing to protect Americans when natural disasters like Hurricane Sandy strike.

FDA Commissioner Margaret A. Hamburg visits the Emergency Operations Center at the agency’s headquarters in Silver Spring, Md., where FDA experts are monitoring, assessing, and responding to the impact of Hurricane Sandy on FDA regulated facilities and products. Mark Russo, acting director of FDA’s Office of Crisis Management, and other members of the FDA Incident Management Group brief the commissioner on their progress. Click on the photo to enlarge it.

We often think of major storms like Sandy in terms of the risk to life and damage to property. But at FDA we also have to focus on the effects of natural disasters on the safety of the products we regulate and that consumers and patients depend on – everything from fresh produce to canned foods to medicines and high-tech medical devices. Flooding, extreme temperatures, and power outages can create a multitude of safety problems for those products and the facilities where they are made and stored.

FDA’s preparation for hurricanes begins even before the tropical season starts. Every year, we use our Geographic Information System to create and continuously update a set of maps and a list of FDA-regulated facilities, sorted by the type of product the facilities produce. This information prepares FDA offices across the country to act immediately in the event of a natural disaster by showing us the location of the affected facilities.

FDA’s work during public health emergencies is coordinated through our Emergency Operations Center, which is in a constant state of readiness 24/7. The EOC, as we call it, is staffed by FDA experts on such subjects as emergency management and coordination, investigation, epidemiology, public and environmental health, the safety of food and medical products, imported products, legal issues, and cartography.

To prepare for Hurricane Sandy, we consulted with other public health and preparedness agencies, received regular updates from the National Hurricane Center, and conferred with sister agencies during telephone conferences conducted by the Federal Emergency Management Agency (FEMA). And to help the public prepare, we posted information on our website about ensuring the safety of food and medical supplies.

This map in FDA’s Emergency Operations Center shows the concentration of FDA-regulated facilities in each affected county. Combined with an overlay of the projected rainfall amounts provided by the National Weather Service, the FDA is able to tell at a glance which facilities producing FDA-regulated products would be impacted by Hurricane Sandy. Click on the map to enlarge it.

As Sandy moved up the East Coast, the experts in our EOC swung into action. We created a series of maps for FDA field offices overlaid with the predicted storm track and FDA-regulated companies in the storm’s path to give us a picture of Sandy’s potential impact. Monitoring reports from our field offices, we produced situation reports, which we fed to FEMA and other agencies. This awareness was crucial in strengthening the agencies’ preparedness and immediate response efforts.

In the wake of Hurricane Sandy, some of our most important work is just beginning. FDA’s district offices are reaching out to the affected states and regulated industries to assess the full impact of this devastating storm. Our offices are using detailed and specialized maps, as well as historical information about facilities, to decide which manufacturing plants to inspect first.

The first priority might be a damaged plant that manufactures complex monoclonal antibodies to treat cancer, a flooded facility that produces medical devices, or a cold storage food warehouse that has lost power. As we assess the damage, we need to inspect facilities and focus on situations that pose the greatest risk to public health and safety.

The Emergency Operations Center is staffed during a disaster like Hurricane Sandy by a FDA Incident Management Group comprised of personnel who are expert in a variety of public health and regulatory areas. They work with FDA Incident Management Teams in the field to monitor the impact of the storm and help shape the agency’s response. Click on the photo to enlarge it.

In the case of an unprecedented natural disaster like Sandy, state and local governments may ask for help with tasks they normally perform, such as inspecting restaurants, school and hospital cafeterias, and facilities where diagnostic x-ray and mammography equipment are installed. Working with our parent Department of Health and Human Services and FEMA, FDA can provide expert teams of inspectors with specialized training to assess and help correct problems.

Fortunately, storms like Sandy occur rarely. But when they do, FDA employees are on the job, dedicated to protecting the public health and working to keep you safe.

Margaret Hamburg, M.D., is Commissioner of the U. S. Food and Drug Administration

For these and similar images go to FDA’s Flickr photostream.

Offering Hope: How FDA Engages With the Cancer Community

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By: Deborah Miller, Ph.D., M.P.H., R.N.

Deborah Miller, Ph.D., M.P.H., R.N.It’s October and the pink ribbons representing breast cancer awareness month are again a common sighting. These ribbons are reminders that breast cancer is still to be overcome. Breast cancer remains the most common cancer among American women, except for skin cancers. Just about everyone knows someone affected by cancer in general, and many have been touched by breast cancer in some way.

For many years, I worked at the Government Accountability Office (GAO), where I became familiar with FDA. I joined FDA’s Office of Special Health Issues (OSHI) in September 2008 because I wanted to be involved more directly with patients again after working for years during my earlier career with seriously ill patients and their families as a neonatal nurse, research nurse, and hospice volunteer.

Like a lot of people, I have experience with cancer – personal, family members, and friends. As the manager of OSHI’s Cancer Liaison Program, I’ve had many experiences that have enhanced my compassion, respect, and patience as I strive to explain FDA’s role in medical product development and regulation to patients with breast and other cancers.

FDA’s Cancer Liaison Program interacts with many cancer patients and family members asking for help. The program seeks to meet the needs of patients and their families in three basic ways. Listening, educating, and assisting.

First and foremost, we listen to patients and caregivers. They tell us their story – when they were diagnosed, treatments they have tried, providers they have seen, and tests they have been through. Often, they tell us they’re scared.

Some of these patients have been dealing with cancer for a number of years, and they tell us that the approved therapies have not worked or have stopped working. Some have considered or joined a clinical trial of an investigational therapy. Some call with the hope of obtaining a “promising” new investigational product that they have heard about in the news and are convinced may be their last hope.

Secondly, we educate. We spend a significant amount of time explaining to patients and family members how cancer drug development, clinical trials, and expanded access, (known in the community as compassionate use) work. We explain FDA’s role, and what we can and cannot do for patients, and try to guide patients toward practical and appropriate options.

We help bridge the gap between patients, their treating physicians, and FDA scientists who are working to review and approve new treatment options for patients. We strive to provide a human touch for each patient or family member with whom we interact.

Finally, we assist the patients. For example, we try to find potential clinical trials for them, guide them through the expanded access process when it’s appropriate, and work with their healthcare providers throughout the expanded access application process. We give patients, family members, and healthcare providers our contact information so they can reach us to work through regulatory issues at any time, including evenings and weekends.  We periodically call them to see how they’re doing.

And if access to investigational drugs is not practical, we go back to listening. We listen to patients’ expressions of their disappointment, anger, frustration, and fears.

This month, I am thinking about the many breast cancer patients I worked with during this past year who benefited from FDA’s approval of Perjecta in June. But I am equally mindful of the many other patients who did not benefit from the drug and will be calling me, desperately searching for something more.

Deborah Miller, Ph.D., M.P.H., R.N., is the manager of the Cancer Liaison Program in FDA’s Office of Special Health Issues

Looking Back at the Kefauver-Harris Drug Amendments and their Meaning

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By: John P. Swann, Ph. D.

The Drug Amendments of 1962, also known as the Kefauver-Harris Drug Amendments, became law five decades ago. But this law’s importance grows with each passing year, making Americans safer than ever from unsafe and ineffective medicines.

John P. Swann, Ph. D.To understand why this law stands today as a pillar of public health in America, it helps to look at how our history shaped it.

There was a system of drug controls in place as early as 1905 that took effectiveness into account, but it was voluntary and administered privately by the American Medical Association. Congress passed laws that required effectiveness from the early 1940s on, but only for selected medicines, such as insulin and penicillin. In 1941, FDA developed regulations to ensure good manufacturing practices to ensure a product’s quality and purity, but only for one drug category. Technically, the Federal Trade Commission had been regulating drug advertising since 1938, but there was little strength in its hold on this industry. And the 1938 Food, Drug, and Cosmetic Act required evidence of a drug’s safety, but the nature of that proof and oversight over how it should be developed were not that clear.

In 1959 Sen. Estes Kefauver began hearings that focused on the high cost of medicines—reflected in the comprehensive bill he introduced in April 1961. But priorities shifted substantially in the next year with the global thalidomide disaster, narrowly averted here, in which a sedative used to treat morning sickness caused thousands of birth defects around the world. Substantial legislative input from FDA helped shape the law that President Kennedy signed on October 10, 1962. And it changed everything:  requirements for therapeutic viability of drugs, veracity in marketing, the proper conduct of investigations, verifiable production controls, patient protections, actual FDA assent to constitute approval, and rigorous proof as the essential element of a drug application.

FDA assembled clinical experts to advise the Agency on drugs previously approved for safety only. They reviewed the available evidence on the effectiveness of those drugs and found that on average 4 out of 10 drugs approved before 1962 and still marketed—medicines that physicians prescribed to their sick patients—either did not work or needed more—often much more—evidence that they did. In the following years FDA removed more than 1,000 of these from the market. At the same time, the agency further called upon therapeutic experts through the systematic use of advisory committees to offer their insights into approval decisions, decisions that still ultimately rested with FDA.

In sorting out this therapeutic mess from the pre-1962 era, the investigational, manufacturing, and regulatory communities reached an understanding about what constituted acceptable evidence, which generally meant randomized, well-controlled clinical trials. While that definition shifted over the following years to accommodate, for example, the needs of gravely ill patients facing few if any treatment options, these changes did not come at the expense of good clinical evidence. Science remained the benchmark of Kefauver-Harris’s legacy.

So, the Drug Amendments of 1962 elevated medical practice, pharmaceutical manufacturing, and public health by inserting a much greater degree of certainty in the way drugs are tested, manufactured, approved, advertised, prescribed, dispensed, and taken.

John P. Swann, Ph. D., is an FDA historian

FDA Voice interviews Helene D. Clayton-Jeter, O.D., on the Cardiovascular and Endocrine Liaison Program

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FDA Voice: Thank you for blogging with FDA Voice today. Can you tell us about the Cardiovascular and Endocrine Liaison Program in FDA’s Office of Special Health Issues?

Helene Clayton-Jeter, O.D. Helene Clayton-Jeter: Millions of Americans have cardiovascular disease (CVD) and diabetes, and their ranks are expected to swell as the population ages and the obesity epidemic continues.

To help fight the spread of these conditions, Janelle Derbis, PharmD, and I established the Cardiovascular and Endocrine Liaison Program (CELP) in 2011. We facilitate interactions among patients, health professionals, and the FDA Centers that regulate the medical products that are used to treat diabetes, hypertension and heart disease.

I was an optometrist for 21 years before joining FDA. Treating diabetes and hypertension is near and dear to my heart because many of my patients were affected by these conditions. And Janelle brings her background as a pharmacist to this work.

Patients with these conditions and the health professionals who treat them want an effective, consistent way to interact with FDA and to become part of FDA’s decision-making processes.

FDA Voice: What is the public impact of these two medical conditions?

Clayton-Jeter: Heart disease and stroke are the first and third leading causes of death, respectively, for both men and women in the United States, accounting for more than one-third of all deaths, according to the Centers for Disease Control and Prevention. An estimated 23.6 million people in the U.S.—approximately 7.8% of the population—have diabetes. This chronic illness is the number one cause of blindness in the U.S and one of the leading causes of disability. Complications associated with these conditions can result in significant costs to patients and to the health care system.

FDA Voice: Can you describe some of the CELP tools and activities?

Clayton-Jeter: We recently launched two new web pages, one for diabetes and one for CVD. Both include information on product safety, new drug and device approvals and clearances, prevention and wellness information, and opportunities for public comment.
We also launched two e-mail lists: FDA DiabetesMonitor and FDA CardioBeat. These e-mail lists deliver FDA updates on safety and regulatory issues related to diabetes and CVD, including product approvals, safety warnings, notices of upcoming public meetings, and notices of draft regulatory guidances.

CELP takes advantage of our office’s existing communication tools to facilitate and moderate stakeholder calls on new product approvals. These calls help patient and health professional groups better explain the issues to their constituents because their representatives get the information first-hand.

We are working with Medscape—part of WebMD’s health professional network— to provide information about new topics within FDA. Working with Medscape, we have developed two articles on drug-eluting stents — a medical device and a drug used to treat blocked arteries – and risks posed when taking high dose simvastatin – a cholesterol-lowering drug.

FDA Voice: What do you have coming up on the horizon for CELP?

Clayton-Jeter: We plan to introduce more web-based training programs on CVD and diabetes issues. We will also be speaking at patient advocacy and health professional conferences, such as the Second Annual Health Professional Organization Conference scheduled for October 4, 2012 at FDA’s White Oak Campus.

We encourage you to visit the web pages and sign up for the e-mail lists to stay current on FDA announcements in the areas of CVD and diabetes.

Helene Clayton-Jeter, O.D., co-manages the Cardiovascular and Endocrine Liaison Program (CELP), at FDA’s Office of Special Health Issues.

Additional Protections for Children Who Participate in Clinical Research

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By: Robert “Skip” Nelson, M.D., Ph.D.

Parents and guardians of seriously ill children often face difficult decisions about their child’s medical care. As a physician practicing for about 20 years in a pediatric intensive care unit, I knew that many of the interventions I used, while potentially life-saving, had no guarantee of success and carried a risk of significant harm or even death.

Robert "Skip" Nelson, MD PhD When faced with a life-threatening disease for which there are few good options, parents sometimes want to try a promising drug that is still under development. I have lived through this situation with parents many times, both in the intensive care unit and as a medical ethicist. Some parents see the drug as a lifeline where none had existed before. It is a natural instinct for parents to want to leave no stone unturned.

Many of the drugs that we use in children have not been approved by FDA for that use, and may not have been studied at all in children. They are available on the market because they have been studied and approved for use in adults, but not necessarily for the same disease for which they might be used in children. There are data available in adults describing the risks, benefits and side effects of drugs already in the marketplace. This information may reassure us a bit about the potential effects of a drug in children. But when that same drug is studied in children, we often discover that we should have been using a different dose, that the drug does not work, or that children may experience a concerning side effect.

The lack of information about the likely effects of a drug in children is more of a problem when the drug hasn’t yet been approved for any use. Most drugs in the early phases of drug development fail, either because they are ineffective in humans, or because of unacceptable side effects. So while it may be tempting to want to try any experimental drug in children with a life-threatening disease, we must be cautious.

Recently, my colleague Richard Pazdur shared his insight into the FDA “expanded access” regulations that allow patients with serious and life-threatening diseases or conditions access to investigational drugs outside of clinical trials. There are two important values at stake when considering expanded access in children. We want to do everything we can to restore a child to health, but we also want to protect that child, and other current and future children from ineffective, and potentially dangerous, interventions.

Physicians can apply for expanded access use for a patient by submitting certain medical information about the patient and important scientific and clinical information about the drug and its intended use to FDA. FDA then reviews this information and determines whether certain important criteria are met. These safeguards are in place to avoid exposing patients to unnecessary risks. Even if FDA determines that an expanded access request may go forward, the company manufacturing the drug has to be willing to supply it.

In implementing its expanded access regulations, FDA tries to strike a balance between two social goods: treating an individual child and demonstrating that a medical product is safe and effective. Children with a serious or immediately life-threatening disease for which there is no comparable or satisfactory alternative therapy are able to have access to investigational agents outside of a clinical trial. However, there must be some evidence that the potential benefit to the child justifies the risks of the treatment and that those risks are not unreasonable in the context of the disease to be treated. Providing the investigational drug must not interfere with the ability to initiate, conduct or complete a clinical trial of that drug that could be used to support its marketing approval for the disease. Otherwise, we would never collect the essential data needed to establish that the drug is truly safe and effective for that use, or whether one child’s seemingly miraculous response was the result of the drug or of something else.

FDA strongly supports the inclusion of children in FDA-regulated clinical trials, provided the trials are conducted in an ethical and scientifically sound manner. For the past 15 years, FDA has been actively involved in initiatives aimed at improving medical product research in children (including under the legislative mandates provided by the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act). Children must be protected from exploitation and exposure to unnecessary risks related to their inclusion in clinical research, and from the unwarranted use of investigational drugs outside of a clinical trial.

I am often asked about FDA’s role in protecting children who are receiving experimental treatments and who are participating in clinical trials. FDA’s regulations provide for specific additional protections to help ensure that children who participate in research are involved in ways that protect the children’s rights, safety, and welfare.  Protections include, for example, review of research by an Institutional Review Board (IRB) supplemented by pediatric expertise as appropriate. The risks posed by research interventions must be low if the interventions are done for the sake of knowledge rather than for the child’s medical benefit. For higher risk interventions, the intervention must offer the child a sufficient prospect of direct medical benefit to justify the potential risks. Investigators must also obtain permission from the child’s parent(s) or guardian(s), and in most clinical trials must obtain each child’s assent when the child is developmentally capable of providing it.

My heart goes out to those parents of children with a life-threatening disease for whom there are no satisfactory treatments. My personal belief is that responsible clinicians and investigators caring for critically ill children, parents, and FDA can work together to find the right path forward for our present and future children. 

Robert “Skip” Nelson, M.D., Ph.D., is Senior Pediatric Ethicist in FDA’s Office of Pediatric Therapeutics

FDA Voice Interviews Michelle Yeboah, Dr.P.H., Director of FDA’s Office of Minority Health

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FDA Voice: Dr. Yeboah, thank you for blogging with FDA Voice today. Can you tell us about your job at FDA, and give us some background on the origin and scope of FDA’s Office of Minority Health?

Dr. Yeboah: Absolutely! The FDA Office of Minority Health (OMH) was established in 2010, as mandated by the Affordable Care Act. OMH serves as the principal advisor to Commissioner Hamburg on minority health and health disparities. I’m currently the Acting Director, and have been on board since the inception of the Office.

FDA Voice: What is the mission or vision for the office, and how did you develop it?

Dr. Yeboah: Our regulatory mission is to reduce racial and ethnic health disparities and achieve the highest standard of health for all. In doing so, we focus on three key areas:

1) Strengthening FDA’s capacity to address minority health and health disparities through coordinated leadership on regulatory actions and decision making across the agency.

2) Promoting effective communication and the dissemination of information to the public, particularly underserved, vulnerable populations.

3) Improving and strengthening the research and evaluation of sub-population data associations with race and ethnicity.

This mission for OMH was developed after discussions with health professional groups, academicians, advocacy groups, industry and a cadre of leaders in the area of minority health.

FDA Voice:  Can you tell us more about your initiatives to improve FDA’s ability to address minority health and health disparities among racial and ethnic populations?

Dr. Yeboah: OMH explores the intersection between regulatory science and health disparities. The 2010 US Census estimates that minorities account for 46% of the overall US population, and current health statistics highlight poorer health outcomes for African American, American Indian and Alaska Native, Asian American, Hispanic American, Native Hawaiian and Pacific Islander communities. Across racial and ethnic groups, a disproportionate share of  minority communities are more likely to die from serious health issues such as diabetes, heart disease, cancer and asthma, to name a few.

The scientific need for understanding health disparities through scientific research is critical to eliminating health inequities and ensuring the highest standard of health for all.

As highlighted in FDA’s Strategic Priorities 2011-2015, expanding efforts to meet the needs of special populations is a crosscutting agency priority. Strengthening FDA’s capacity to understand the complexities associated with minority health and health disparities can lead to better health outcomes for the most vulnerable communities, whether it is through epigenomics, personalized medicine or targeted risk communication among underserved populations.

In May we held a Webinar entitled Steps to Addressing Health Disparities. The Webinar discussed the establishment of the Office of Minority Health at FDA and also discussed agency efforts to ensure the safety, efficacy, and security of regulated products among vulnerable populations.

FDA Voice:  We’d love to hear about the initiatives and partnerships you are working on to address health disparities. I know there are a lot of them, but if you could give us some details that would be great!

Dr. Yeboah: Yes – as we talked about, OMH is looking to find opportunities for collaborating in the areas related to minority health, health disparities and issues of personalized medicine. So far this year we have been able to finalize four University-based research partnerships:

  • University of Hawaii Hilo, College of Pharmacy
  • Meharry Medical College, Center for AIDS Health Disparities Research
  • University of Nebraska, Rural Health Education Network
  • University Of Hawaii Manoa, Office of Public Health Studies

These partnerships are solely focused on examining minority health and health disparities from a regulatory perspective.  For example, we have a research Memoranda of Understanding (MOU) with the University of Hawaii, Hilo College of Pharmacy, to work on reducing disparities in diabetes treatment and outcomes among Asian and Pacific Islander Americans living in Hawaii. And, to date, we have established partnerships with several Universities exploring issues of disparities in diabetes, tobacco and HIV/AIDS.

We have additional University-based research partnerships that are pending and have several collaborations with other organizations including the National Institutes of Health, with whom we are co-sponsoring the 2012 Summit on the Science of Eliminating Health Disparities: Building a Healthier Society, Integrating Science, Policy and Practice, in the fall. We are also partnering with the Centers for Disease Control and National Hispanic Alliance. We look forward to growing additional partnerships to address issues of health disparities from a regulatory perspective.

Find out the details on our MOU’s. We have a lot of exciting and promising activities on the horizon at OMH!

FDA Voice:  How is the Office of Minority Health helping to diversify the FDA Advisory Committees?

Dr. Yeboah: Last year OMH began a new initiative called “Enhancing Diversity on FDA Advisory Committees.” The goal is to broaden the search for qualified advisory committee candidates who have experience with underserved communities.

Advisory committees are essential in supporting FDA’s mission of protecting and promoting the public health by obtaining independent expert advice on scientific, technical, and policy matters. Promoting FDA’s leadership role in addressing health disparities and assuring diverse perspectives on advisory committees are integral to achieving the agency’s public health mission.

The expertise provided by advisory committee members is essential for understanding the safety and efficacy of regulated products on the target population, especially racial and ethnic populations, who are often disproportionately impacted by higher rates of disease, disability and premature death for conditions ranging from cancer to cardiovascular disease and HIV/AIDS to name a few.

It is critical that FDA Advisory Committee members have the expertise and experience necessary to effectively consider the impact of regulated products on racial and ethnic populations. Health professionals from racial and ethnic minority groups or who serve in minority communities often possess valuable experience in the prevention, diagnosis and treatment of disease among racial and ethnic minorities.

FDA Voice:  I understand the Office of Minority Health is also helping to strengthen research with clinical trials. Can you talk about that a bit?

Dr. Yeboah: We collaborated with FDA’s Office of Women’s Health, the Society for Women’s Health Research, and industry representatives, and held the “Dialogues on Diversifying Clinical Trials: Successful Strategies for Engaging Women and Minorities” conference late last year to explore promising practices in clinical trials for the inclusion of women and minority populations. The meeting drew a cadre of professionals from various fields including pharmaceutical industry representatives, health care providers, patient advocacy groups and academia.

OMH continues to pursue the overarching goals discussed at the conference: to increase the participation of underserved populations in clinical trials with resulting improvement in clinical care and positive health outcomes, and to share successful practices in the recruitment, retention and analysis of women and minorities in clinical trials. For additional information about the conference or our office please visit the OMH Webpage.

Michelle Yeboah, Dr.P.H., is Director of FDA’s Office of Minority Health (Acting)