How FDA Ensures the Safety of Blood Products

By: Richard J. Davey, MD

You might be aware that FDA plays a key role in assuring the safety and efficacy of products such as drugs, vaccines and foods. But less well known is the agency’s critical regulatory oversight of our nation’s blood supply.

Scientist using pipet with test tube and blood Blood saves and improves the lives of millions of individuals each year.    It is used for transfusions for example, to replace blood loss during surgery, childbirth or following a traumatic injury. Blood and blood products are also a key part of the medical treatment of serious illnesses such as cancer, organ failure, or sickle cell disease. Additionally, blood and blood products are used to prevent and treat many medical conditions, such as infections and blood clotting disorders.

The Office of Blood Research and Review, which is part of FDA’s Center for Biologics Evaluation and Research, regulates both whole blood and blood components for transfusion, as well as various other blood products. FDA works with blood collecting establishments, product manufacturers, health care providers, patient organizations, academic researchers, and other government agencies to ensure a safe and adequate blood supply. 

Officer donating plasmaFDA goes to great lengths to ensure that our blood products are safe. FDA reviews and approves new products, conduct research, issue guidances and regulations, conduct inspections of manufacturing facilities, and communicates with manufacturers, consumers and health care providers about blood product safety and efficacy issues. FDA also assists in product recalls when necessary, and works with regulatory agencies in other nations as well as international organizations such as the World Health Organization.  

Blood donation is another key area where FDA plays a major role. Beginning with safe donations, we regulate the processes by which blood is collected and manufactured

Each year, nearly 11 million volunteer donors provide about 17,000,000 donations of blood and blood components for transfusion to about 4.5 million recipients, according to the National Blood Collection and Utilization Survey Report.  About a third of these volunteers are first-time donors. And, more than 18 million units of Source Plasma are donated each year for use in manufacturing plasma-derived products, such as clotting factors and immune globulins.

To make sure blood and blood products are safe for recipients, FDA has established a system of five layers of safety for donated blood. Maintaining an adequate supply of blood and blood components is vitally important, and to help achieve this goal the U.S. Department of Health and Human Services recognizes and participates in World Blood Donor Day

We hope this information gives you a better idea of how FDA helps keep blood and blood products safe and available for you and your family. And, we urge you to consider helping to save lives by donating blood today!

Richard J. Davey, MD, is Director of CBER’s Division of Blood Applications, Office of Blood Research and Review

FDA Voice Interviews Deborah M. Autor, Esq.

En Espanol

FDA Voice: Thanks for agreeing to talk with us – can you tell us a little about your job at FDA?

Deputy Commissioner Autor: My goal is to make FDA’s response to globalization challenges and import safety a top priority. A little bit of background – last July 2011, Commissioner Hamburg established the Office of Global Regulatory Operations and Policy (“GO” or “Global Operations”) as one of FDA’s new Directorates. This office provides executive oversight, strategic leadership, and policy direction to FDA’s domestic and international product efforts. I provide broad direction and support to the Offices of Regulatory Affairs and International Programs and I also work on making sure FDA integrates its domestic and international programs to promote and protect public health.

Deborah M. Autor, Esq.FDA Voice: What brought you to GO?

Deputy Commissioner Autor:  There have been fundamental global shifts that have major implications for consumers – and transforming FDA from a domestically focused agency operating in a globalized economy to a truly globally focused public health agency that oversees a worldwide enterprise is a now both a necessity and monumental challenge. Given my almost twenty years of professional experience working on issues related to FDA-regulated products, when Commissioner Hamburg offered me this opportunity to lead GO and help with transforming FDA into a modern public health regulator in a globalized economy, I welcomed the opportunity.

FDA Voice: Tell us more about what you were doing before GO.

Deputy Commissioner Autor: I joined FDA in 2001, and from 2006 to 2011, I served as Director of the Office of Compliance at CDER. Before that, I was a trial attorney at the U.S. Department of Justice, Office of Consumer Litigation, and in private legal practice before that. So pretty much my entire professional career has been focused on FDA-related issues.

FDA Voice: What’s the favorite part of your job at FDA?

Deputy Commissioner Autor: By far, the favorite part of my job is working with the passionate and dedicated people at FDA. They exemplify the best in public service and it’s a privilege to work collaboratively with such great people on critical public health issues.

FDA Voice: What major changes have you witnessed in FDA over the past ten years?

Deputy Commissioner Autor: Global economic conditions and innovations in technology, science, and communications have dramatically changed the landscape of FDA regulated products and, in addition, global production of FDA-regulated goods and materials has exploded over the past 10 years. For example, FDA-regulated products are currently imported from more than 150 countries, with more than 130,000 importers of record, and from more than 300,000 foreign facilities. In 2011, nearly 24 million shipments of food, devices, drugs, cosmetics, radiation-emitting products, and tobacco products arrived at U.S. ports of entry. Ten years ago, that number was closer to 6 million. So you can see how globalization has fundamentally changed the economic landscape in the U.S., and it will continue to have major implications for FDA.

FDA Voice: How can we meet the challenges of globalization?

Deputy Commissioner Autor: We have come up with a plan, and last June, 2011, we published a special report, “Pathway to Global Product Safety and Quality,” which lays out our global strategy and action plan. I hope people will read the plan, but in short it lays out an approach focusing on four pillars of: building global coalitions of regulators; establishing global information sharing and networks; increasing intelligence-gathering and risk analytics; and, leveraging third parties. The Pathway focuses on how FDA, given its limited resources, can have the greatest impact on public health–a goal we all share.

FDA Voice: How do you envision FDA ten years from now? 

Deputy Commissioner Autor: Our goal for the future is to build a public health safety net for consumers around the world, created, supported, and maintained by global coalitions of regulators. In the future, we will be operating under a model that relies on strengthened collaboration with other regulators, improved information sharing and gathering, data-driven risk analytics, and the smart allocation of resources, leveraging the combined efforts of government, industry, and public- and private-sector third parties. It is clear that FDA, and our many partners, are ready to face the global challenge together!

FDA Voice: Thank you so much for your time!

Deborah M. Autor, Esq., is FDA’s Deputy Commissioner for Global Regulatory Operations and Policy

Hope on the Horizon

By: Margaret Hamburg, M.D.

This week we were once again reminded that the critical issue of drug shortages isn’t about industry, it isn’t about government, or even about the drugs themselves. It is about getting people the treatments they trust, they need, and they rely on. One person who understands this all too well is Sara Stuckey from Lincoln, Illinois. Sara’s son, six-year old Nate, has been on the cancer drug methatrexate since he was diagnosed with leukemia back in 2009. The drug is the preferred treatment for the disease. Unfortunately, due to the shortage, Nate’s doctor informed Sara that he only had enough of the drug for Nate’s treatment this week. 

At a briefing Tuesday hosted by the FDA for representatives from industry, health professional and patient organizations, as well as the FDA team that has been working day and night to address this problem, Sara Stuckey shared her story.
Watch videos from the FDA Progress on Drug Shortages briefing.
Sara talked about Nate, the little boy, who is the “laid-back” one in her family. She explained how the family has learned to be flexible, working around Nate’s disease, compromised immune system, and chemotherapy treatment. For this family and many others, methotrexate is a life-saving drug. So, when Sara heard about a possible shortage of the drug, she talked to Nate’s clinic. They said there was just enough supply for a week but that there were alternatives. Still, Sara was left with many questions and concerns – the same questions any parent would be asking if their child faced a life-threatening illness.
Sara Stuckey

Sara Stuckey speaks at "FDA Progress on Drug Shortages," a briefing held on February 21, 2012, about her son, six-year old Nate.

View photos from the FDA Progress on Drug Shortages briefing.
The good news for Sara and Nate – and many other children and families – is that the shortage has been averted.
  • FDA has prioritized review of and approved a preservative-free methotrexate generic drug manufactured by APP Pharmaceuticals and expects that product to become available in March and continue indefinitely.
  • At Tuesday’s briefing, the CEO of Hospira, another company that manufactures the drug, said that supplies are being ensured for months to come. Hospira expedited release of additional supplies, resulting in 31,000 vials of new product – enough for more than one month’s worth of demand – being shipped to hundreds of U.S. hospitals and treatment centers on Tuesday.
  • And FDA is actively working with other manufacturers of methotrexate who have also stepped up to increase production in order to meet patient needs, including Mylan and Sandoz Pharmaceuticals.

To remedy the critical shortage of another cancer drug Doxil (liposomal doxorubicin), the FDA took proactive steps needed to increase available supply for patients in the U.S. For Doxil, there will be temporary importation of a replacement drug, Lipodox (liposomal doxorubicin), which is expected to end the shortage and fully meet patient needs in the coming weeks.

Still, we cannot stop there. At Tuesday’s briefing, we heard from representatives of the American Cancer Society, the American Society of Clinical Oncology, the Children’s Oncology Group, and APP, as well as Hospira. All spoke forcefully for working together to prevent future shortages from affecting the lives of both children and adults. It is enough for families to cope with disease; adding real or potential shortages to their list of worries is more than unfortunate.

Members of the FDA Drug Shortages team

Members of the FDA Drug Shortages team

This event reminded us of the human impact of drug shortages – as well as what can be done when we work together. We hope this can be a model for future efforts as we move forward, working with our partners in industry, with health professionals, and with patient advocates to find answers that will work for families like Nate’s.

While there is no simple solution to resolving drug shortages, we are doing all that we can to make sure patients have access to the critical medicines they need when they need them. I’d like to give a special thanks to the FDA Drug Shortages team and all the other staff throughout our agency for their hard work and leadership on this topic.

We are making progress. Hope is on the horizon. 

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

A Valentine for Your Heart

By: Margaret Hamburg, M.D.

Valentine’s Day, when we celebrate matters of the heart, is the perfect reminder that February is American Heart Month.  There’s no better gift for those you love, or for yourself, than to help ensure a healthy heart.

Heart disease is the leading cause of death in the United States.  One in every three deaths in our country is from heart disease and stroke.  That’s equal to 2,200 deaths each day, every day, or more than 800,000 per year.  But more than abstract statistics, every victim is a parent or child, spouse or sibling, grandparent or friend.  There’s not a single family, not a single individual, who hasn’t been touched by this devastating epidemic.

Margaret Hamburg, M.D.Cardiovascular disease is also very expensive.  Together, heart disease and stroke hospitalizations in 2010 cost the nation more than $444 billion in health care expenses and lost productivity, and they are among the leading causes of disability.  One out of every six health care dollars is spent on cardiovascular disease treatment, and cardiovascular disease accounts for the greatest disparity in life expectancy across our racially and ethnically diverse population.

Too often, when we think of heart disease, we think that it only affects men.  Nothing could be further from the truth.  Nearly 43 million women, one-third of all women in America, are living with or are at risk for heart disease.  More women die from heart disease than from anything else, more than from all forms of cancer combined, and it’s largely preventable.

The FDA Office of Women’s Health (OWH) maintains a web page which provides links and resources related to heart health.  The website features the OWH heart disease in women fact sheet and publications on hypertension, cholesterol, smoking cessations, and other ways to promote a healthy heart.

As a physician, I know that hereditary issues play a role in heart disease.  But, the majority of risk factors are controllable or treatable, regardless of your age or physical ability.  Make healthy eating choices.  Don’t smoke.  Reduce sodium and transfats in your diet.  Regularly check your blood pressure and cholesterol levels.  Maintain a healthy weight.  Manage stress.  And, get active.  All of these things will help you enjoy a healthy heart and lifestyle.  Use this American Heart Month to educate yourself about heart disease, its risk factors, and ways to beat it.

One way the Department of Health and Human Services (HHS) is helping Americans achieve healthier hearts and lives is through the “Million Hearts” initiative.  It’s a new public-private partnership launched in September of 2011 that’s trying to prevent one million heart attacks and strokes by 2017.  The “Million Hearts” initiative will focus, coordinate, and enhance cardiovascular disease prevention activities across the public and private sectors through a wide range of activities targeted at improving clinical care and empowering Americans to make healthy choices.

Within HHS, the Centers for Disease Control and Prevention and the Centers for Medicare and Medicaid Services are helping us lead the “Million Hearts” initiative, working alongside many other federal agencies.  Key private-sector partners include the American Heart Association, the American Medical Association, the YMCA, other non-profit agencies, communities and health systems.

Preventing one million heart attacks by 2017 is an ambitious goal that will require work and a steady commitment to change from each one of us.  Visit Million Hearts to learn more about the steps you can take to help reach this national goal, and to enjoy a healthier lifestyle.  Additionally, take the pledge to be one in a Million Hearts!

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

FDA Assists in the Success against Epidemic Meningitis in Africa

By: Marc Kusinitz, PhD

Meningococcal meningitis, a disease that sweeps across sub-Saharan Africa in an area called the meningitis belt, is losing its power to inflict illness and death. Scientists from FDA made a critical contribution in developing the technology needed to manufacture a vaccine against this terrible disease, and at an affordable cost for African nations like Burkina Faso, Chad, Ethiopia, and Niger.

Meningococcal meningitis can be deadly. It kills 10 percent of people it infects within two days after they start showing symptoms. Although an antimicrobial drug saves large numbers of infected individuals, about 10 percent die from the infection and about 10 percent to 20 percent of survivors develop mental retardation, hearing loss, or seizures. In Africa, most meningococcal disease is caused by the group A meningococcus bacteria, and about half of its victims are working-age adolescents and young adults. The disease results in very significant human, social, and economic losses to the affected communities and countries. Vaccination offers the best chance to prevent the disease and epidemic meningitis. However, an existing vaccine didn’t work very well.

Developing a Vaccine:

In 2001, the Bill & Melinda Gates Foundation agreed to fund the Meningitis Vaccine Project (MVP)–a partnership between the World Health Organization and PATH, a non-profit organization based in Seattle, WA that works with collaborating groups to provide health care technologies and strategies to areas of the world that have limited resources.

Dr. F. Marc LaForce led MVP’s effort to develop, test and license a new type of vaccine against group A meningococcus bacteria, that could protect people before an epidemic begins. The new vaccine, called a conjugate, is a chain of sugars connected to a protein that the immune system responds to very well. When MVP hit a hurdle during the development stage, FDA stepped in. Drs. Robert Lee and Carl E. Frasch, two researchers in the Office of Vaccines Research and Review in FDA’s Center for Biologics Evaluation and Research (CBER), had developed an alternative conjugation technology that was more efficient and less costly. Through a technology transfer agreement, FDA provided the technology to MVP via PATH, with help from the National Institutes of Health.

Scientists at CBER also developed reagents for evaluating the vaccine’s performance and safety and developed methods to monitor the manufacturing process. MVP had partnered with the Serum Institute of India Limited, a developing-country vaccine manufacturer, to make the new conjugated vaccine. In December 2003, two scientists from the Serum Institute came to Drs. Lee and Frasch’s FDA laboratory to learn the conjugation method to manufacture the vaccine.

After preclinical animal studies and a series of clinical trials in people in India and Africa’s meningitis belt to assess its safety and effectiveness, the new vaccine, MenAfriVac, was licensed in Dec. 2009 by India for export to Africa. By June 2010, the WHO had prequalified the vaccine for use in global immunization programs.

Woman Receiving Vaccination

September 2010. A woman in Mali receives MenAfriVac in an early introduction in preparation for the official vaccine program launch in December. Photo: WHO/Mali

Launching the Vaccination Campaign:

MVP launched its vaccination campaign in Dec. 2010, beginning in Burkina Faso and moving on to Mali and Niger and eventually Cameroon, Chad and Nigeria. By the end of 2011, an estimated 55 million people had been vaccinated with MenAfriVac at a cost of only 40 cents per dose.

The contribution of CBER researchers Carl Frasch and Robert Lee was perhaps best summed up by MVP director Dr. LaForce in a news story about their timely contribution of the conjugation technology that enabled the development of MenAfriVac at a price that Africa could afford: “These guys are heroes.”

FDA’s contributions to a major health care project in Africa underscore the agency’s recognition that infectious diseases know no borders. Protecting human health globally is linked to the agency’s core mission of protecting human health in the United States.

Marc Kusinitz is Senior Science Communications Advisor in FDA’s Center for Biological Evaluation and Research

50 Years after Thalidomide: Why Regulation Matters

By: Margaret Hamburg, M.D. 

Fifty years ago, the vigilance of FDA medical officer Dr. Frances Kelsey prevented a public health tragedy of enormous proportion by ensuring that the sedative thalidomide was never approved in the United States.  As many remember, in the early 1960’s, reports were coming in from around the world of countless women who were giving birth to children with extremely deformed limbs and other severe birth defects.  They had taken thalidomide. Although it was being used in many countries, Dr. Kelsey discovered that it hadn’t even been tested on pregnant animals.

Dr. Kelsey’s reaction to thalidomide exemplifies the FDA’s mission: protecting and promoting the health of the American people, using science for regulatory decision-making.
Margaret Hamburg, M.D.Now I know that in some circles regulation is viewed as a roadblock to innovation and economic growth. But in actuality, when done right, regulation isn’t a roadblock; it’s the actual pathway to achieving real and lasting innovation.

Smart, science-based regulation instills consumer confidence in products and treatments. It levels the playing field for businesses. It decreases the threat of litigation. It prevents recalls that threaten industry reputation and consumer trust, not to mention levying huge preventable costs on individual companies and entire industries. And it spurs industry to excellence.

The tragedy of thalidomide led to changes that strengthened both the regulatory and scientific environment for medical product development and review.

In response to the public uproar, in 1962 Congress enacted the Kefauver-Harris amendments to the Federal Food, Drug and Cosmetic Act. Thanks to these new amendments, manufacturers had to prove that a drug was not only safe, but also effective. Approvals had to be based on sound science. Companies had to monitor safety reports that emerged postmarket and adhere to good manufacturing practices that would lead to consistently safe products. And there were new protections for patients.

The amendments not only benefited patients, they helped industry, raising scientific standards that eventually ushered in today’s sophisticated, science-based life sciences industry.

For the very first time, many companies put in place research and development programs, including the design and implementation of controlled clinical trials. Major therapeutic breakthroughs resulted, including the use of beta blockers in patients after a heart attack and angiotensin-converting enzyme inhibiters to improve survival in patients with heart failure. All of these were good news for public health and for corporate bottom lines. The best drugs and treatments rose to the top, not simply those that were most heavily marketed.

The Harris-Kefauver Amendments created a culture of quality and innovation that laid the foundation for our current regulatory environment which fosters a domestic pharmaceutical industry that is second to none.

Going forward, smart regulation requires regulatory flexibility that responds to changing situations, new information and new challenges. It also demands that we advance regulatory science: the knowledge and tools necessary for the meaningful and timely review of products for safety, efficacy, quality and performance.

Thalidomide, once again, is a good example. It came back on the U.S. market in 1998 after data showed it was safe and effective to treat a complication of leprosy. In an appropriate balancing of benefit and risk, FDA required strong safety monitoring and a strict dispensing plan before approving the drug.

Regulation such as this requires a strong, robust FDA, one endowed with the necessary resources to ensure smart, sound, science-based regulation.

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

Beyond Rulemaking: Building the Scientific Infrastructure for Food Safety under FSMA

By: Michael R. Taylor, J.D.

One year has passed since the FDA Food Safety Modernization Act was enacted, and we are making remarkable progress in developing regulations to implement many of the law’s provisions. It’s certainly a big job, requiring a lot of hard work by many at FDA. 

As we proceed with rulemaking, we also need to be sure we have the infrastructure in place and the resources available to operate in the FSMA environment called for by Congress. FSMA requires industry to focus on preventing problems and on addressing risks in the food supply. But in order to implement the new requirements, FDA will need up-to-date information on what the hazards are, how to detect them, and what steps can be taken all along the farm–to-table chain to minimize them to the extent possible.   

Michael R. Taylor, J.D.

Our existing scientific foundation is strong, but we must be ready for the new challenges posed by FSMA. Our scientific leadership, capacity and partnerships all must be up to the task. One important step we are taking now is to recruit a Chief Science Officer and Research Director to provide strategic direction and lead our research and methods development activities. This individual will work with our scientists and policy experts to be sure that the resources we have available—including expertise and funding—are dedicated to our most critical public health needs. He or she will also need to anticipate our needs for the future.

This means prioritizing research based on our most critical needs and data gaps. This needs to be done strategically across all of our food programs and in concert with other research offices within FDA. It means determining what new methods, such as rapid analytical tests, are needed to identify contaminants in foods and to ensure that industry is meeting our food safety requirements. And, it means collaborating with other research entities in the public and private sectors.

By working closely with our research partners in other government agencies, academia and industry, we can leverage each other’s work and avoid duplication.

FDA chemist uses gas chromatography to measure levels of melamine in food samples. Our Chief Science Officer and Research Director will also make sure we identify and invest in scientific disciplines and specialties that are critical to carry out our mission. Science and technology change rapidly, and we must have the right expertise, training, equipment and facilities to keep up.

A lot is at stake. FSMA provides the framework for the new prevention- and science-based food safety system, but we must have the science to support the framework and make the right decisions to protect the public health. Americans expect us to have the right science at our fingertips to protect themselves and their families. FSMA must be accompanied by change within FDA so we are ready to operate the new food safety system – and we are ready to meet that challenge head on.

Michael Taylor is Deputy Commissioner for Foods at FDA