FDA Works with Partners to Establish Important Therapeutic Area Data Standards

By:  Janet Woodcock, M.D.

Clarification, November 5, 2012:  CFAST is a joint initiative of CDISC and the Critical Path Institute.  The FDA and TransCelerate Biopharma, Inc. are partnering with CFAST in its Therapeutic Area Standards Program.

A new partnership between the FDA, the Clinical Data Interchange Standards Consortium (CDISC), and the Critical Path Institute (C-Path) was officially launched today at the CDISC International Interchange in Baltimore. This partnership, called the Coalition For Accelerating Standards and Therapies or CFAST, will bring together clinical data experts from the FDA, the pharmaceutical industry, and the information technology sector, to develop and maintain data standards tailored to individual diseases and therapeutic areas.

Janet Woodcock, M.D.While I was preparing my keynote address for the Interchange, I began thinking of how history provides us lessons for the future, and the Great Baltimore Fire of 1904 is a particular example that stands out in my mind. In this tragedy, thousands of firefighters from surrounding cities and states responded to Baltimore’s call for help, but they were unable to assist in putting out the fire because their hoses and equipment were not compatible with the Baltimore hydrant connections.  This terrible event made the need for standardized fire-fighting equipment and connections devastatingly clear.

As part of the changing tide of drug regulation, we are seeing ever-increasing streams of data coming into the agency. However, much like standardized fire-fighting equipment, we need to develop standardized definitions for individual diseases and the therapeutic approaches to treat them to be able to tap into this data stream.

Standardized data elements that are common to all clinical trials, such as age and gender, have been established using CDISC terminology. However, data elements that are unique for a particular disease or therapeutic area still need to be developed so that the data from multiple trials can be more easily grouped for reporting and analysis.

In short, establishing common standards for data reporting will provide new opportunities to transform the massive amount of data from drug studies on specific diseases into useful information to potentially speed the delivery of new therapies to patients.

We at the FDA are excited to be a member of this very important partnership. We believe that CFAST will provide an important resource for drug development and research that will result in enhancements in the evaluation of safe and innovative therapies for the public.

More about the priority therapeutic areas for standards development.
To read more on data standards, please read:  CK Cooper et al. Drug Information Journal. 46(5) 521-22.

Janet Woodcock, M.D. is the Director of the FDA’s Center for Drug Evaluation and Research.

National Mammography Day: Supporting Quality Mammography

By: Marsha B. Henderson, M.R.C.P. and Helen Barr, M.D.

Like millions of women, we go each year to get a mammogram. For us the experience is not just about healthy living. It is also a reminder of the value of our hard work at FDA. Each time we see the FDA certificate in our mammography facility showing that the facility meets FDA’s high standards, we are reminded of the commitment and dedication of FDA employees to supporting mammography services.
Marsha B. Henderson, M.R.C.P. and Helen Barr, M.D.

Marsha B. Henderson, M.R.C.P. (left) and Helen Barr, M.D.

Under the Mammography Quality Standards Act and Program, FDA employees work to ensure that women can go anywhere in the country and expect to get reliable, high quality breast images. We certify and inspect mammography facilities establishing uniform standards for mammography equipment and staff training. Thanks to these efforts, there are over 8,600 certified mammography facilities in rural and urban communities across the country.

We didn’t stop there. Early on, we realized that regulation was only part of what was needed. FDA recognized that it should also help raise awareness about the importance of mammography. Through the Pink Ribbon Sunday Program, we formed outreach partnerships to teach women the facts about mammography screening. When the Pink Ribbon Sunday Program began in the 1990s, we targeted African American and Latino women because they were least likely to get a mammogram. However, the program quickly spread from minority churches to businesses, sororities, health centers, and other national organizations reaching women from all backgrounds.

Over the years, FDA has touched millions of lives through our mammography initiatives. We have chosen today – National Mammography Day – to thank our colleagues at FDA and our partners in the health care community and state and local governments for their efforts.  We also encourage you to help connect the women in your community to our free mammography resources. Your efforts can help raise awareness, provide hope, and maybe even save a life.

Marsha Henderson, M.C.R.P., is FDA’s Assistant Commissioner for Women’s Health in the Office of the Commissioner

Helen Barr, M.D., is FDA’s Director of the Division of Mammography Quality Standards at the Center for Devices and Radiological Health

Offering Hope: How FDA Engages With the Cancer Community

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

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 (via elliott at dhead online) . 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

BeSafeRx: FDA Helping Consumers Avoid Risks of Online Prescription Drug Purchases

By: Ilisa Bernstein, Pharm.D., J.D.

As a pharmacist who cares about patients’ individual medication needs, I am delighted to share my thoughts on FDA’s latest effort to protect patients from fraudulent, illegal online pharmacies. We’ve just launched BeSafeRx – Know Your Online Pharmacy, a national campaign to educate consumers about the risks of buying prescription medications over the Internet.

Ilisa Bernstein, Pharm.D., J.D.It’s troubling to hear that some patients don’t recognize the need to carefully select where they buy their prescription medicines, or that the “pharmacy” they’re buying from might not actually be a pharmacy at all. Too often it seems that it has become second nature for many consumers to buy clothes, electronics or even medicines on the Internet. But the reality is that purchasing drugs from Internet sources that are not known to be reliable is a risky business, and today, with the sale of counterfeit drugs escalating worldwide, perhaps riskier than ever.

How risky? According to a recent FDA survey, nearly one in four of the surveyed Internet users reported having purchased prescription medicine online. This fact alone is not surprising, considering that many people, especially those with prescription drug insurance, use the Internet to get their prescriptions safely filled from legitimate and reputable pharmacies. Unfortunately, in many other cases—far more than we’d like to see—consumers are surfing the Web for cheaper or more convenient sources. Our survey showed that about 29 percent of survey participants said they were unsure about how to safely purchase medicine online. Why is this important? Because, according to reviews by the National Association of Boards of Pharmacy (NABP), less than 3 percent of online pharmacies comply with U.S laws and NABP practice standards, making it critically important for online consumers to understand how to recognize fraudulent, illegal online pharmacies and how to identify a safe, legal online pharmacy.

That’s why I’m so excited about the BeSafe Rx campaign! We’re providing consumers with practical and useful tools to help them make informed decisions about their online purchases. All this useful information is just a click away

When I was in pharmacy practice I wanted nothing but the best for my patients, and now, as a pharmacist with FDA, I want nothing but the best for the American public. BeSafe Rx helps FDA protect public health, and I’m proud to be a part of it. So be safe, and make sure you know your online pharmacy!

Ilisa B.G. Bernstein, Pharm.D., J.D., is the Director (Acting), Office of Compliance in FDA’s Center for Drug Evaluation and Research

Strengthening Science at FDA to Improve Food Safety

By: Michael R. Taylor, J.D.

Science is the foundation—the critical underpinning—of everything FDA does to protect public health, and food safety is no exception.   Congress recognized this basic fact when it enacted the FDA Food Safety Modernization Act (FSMA), which is all about harnessing science to understand and prevent food safety hazards.

Mike Taylor, J.D.We are called on to develop science- and risk-based safety standards for a wide variety of fresh produce operators and processing facilities that work under highly diverse conditions and may confront different hazards.  In overseeing implementation of these standards, we are expected to prioritize our domestic inspections and import oversight based on level of risk.  And, as an overarching guide to how we target our risk reduction efforts, Congress has directed FDA to identify what we consider the most significant contaminants in food and devise strategies for addressing them. 

These scientific demands are compounded by the changing nature of the food supply itself and the risks it poses.  We are seeing a dizzying array of new foods in the marketplace, many produced using new technologies.  We are seeing new pathogens emerge, and “not so new” pathogens that show up in foods where we never expected them to be, such as the new strains of disease-causing E. coli in sprouts and diverse strains of Salmonella in produce and peanut butter.   We’re also seeing changes in the practices and profile of the consumers we seek to protect, including an increase in the percentage of the population that is particularly “at risk” for foodborne illness—including the elderly, pregnant women, and immune-compromised individuals.

To meet these challenges, we need new scientific data, new scientific tools, and a new scientific understanding of food safety problems and solutions.  FDA is blessed with a strong cadre of scientists who are tackling these needs through both basic and applied research.  They need and have our strong support.   To take full advantage of our scientific capacity, however, we must move forward strategically.

This means defining our mission needs carefully, prioritizing our research and other scientific efforts in accordance with those defined needs, and building bridges among scientists inside and outside FDA to generate synergies, make best use of resources and widely share the fruits of our collective scientific effort.  

To guide these strategic efforts, we recently created the position of chief science officer and research director.  Since July, Dr. David White has been acting in that position and chairing our Science and Research Steering Committee (SRSC).   Consisting of laboratory and scientific leaders from across FDA’s food safety and veterinary medicine program as well as the National Center for Toxicological Research, the Office of International Programs and the Office of the Chief Scientist, the SRSC has been working for two years, much of it under the leadership of Dr. Jeff Farrar, to strengthen research priority-setting and coordination.  

Among many other things, the SRSC has launched an annual research conference to bring researchers together and last week convened a second annual program-wide research prioritization meeting in which program and research leaders collaborated in shaping our 2013 research agenda.

This work could not be timelier as we face constrained resources and must pay careful attention to priorities and the public health payoff from our research.   The work FDA scientists are doing now to study how common and persistent Salmonella can be in tomatoes and to develop rapid methods to detect Salmonella in animal feed and pet food are good examples of the research we need to be doing.   We are also working at a time of exciting opportunity to take the scientific underpinnings of our public health work to entirely new levels. It is utterly essential to public health and to the success of the nation’s food system that we meet today’s scientific challenges and take full advantage of today’s scientific opportunities.  We are committed to that.  We are fortunate to be working with talented scientists who are more than up to the task.  And we look forward to doing this work in partnership with the scientific community at large.

Michael Taylor is Deputy Commissioner for Foods at FDA

Celebrating a Public Health Milestone: The Kefauver-Harris Drug Amendments

By: Margaret Hamburg, M.D.

Margaret Hamburg, M.D.My predecessor, FDA Commissioner George Larrick, once said that, with the exception of 1906 and 1938, no date had more significance in the agency’s history than October 10, 1962, when President Kennedy signed into law the Kefauver-Harris Drug Amendments.  Commissioner Larrick stood behind the President at that momentous event.

The amendments were the response by Congress to the birth of thousands of malformed children, in Europe and elsewhere, whose mothers had used thalidomide during pregnancy.  The U.S. was spared that tragedy, thanks to the vigilance of FDA’s Dr. Frances Kelsey.

Fountain pen enscribed "The President - The White House"

With this pen, which he presented to Dr. Frances Kelsey, President John Kennedy signed into law the 1962 Kefauver-Harris Drug Amendments.

The time was therefore ripe for reform to protect and to promote public health, and it came in the form of legislation proposed by Sen. Estes Kefauver.  The Drug Amendments of 1962 strengthened the Federal Food, Drug and Cosmetic Act by requiring solid and rigorous science-based evidence that drugs are both effective and safe and in doing so the law laid the foundation for today’s modern pharmaceutical industry. 

To comply with the new amendments, manufacturers were now required to test their drugs in a truly scientific manner — typically, by conducting two well-designed and controlled clinical trials.  Companies also had to adhere to good manufacturing practices and monitor safety reports after the drugs reached the market.

With the benefit of hindsight, we can now see that the Drug Amendments of 1962 did much more than strengthen FDA’s authorities to ensure that our medications are safe and effective. The requirements of this law have since then been adopted world-wide, and became the “gold standard” for science-based decisions involving drugs.

In short, the Drug Amendments of 1962 merit our great praise on their fiftieth anniversary: they have saved countless lives around the world.

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

Breast Cancer Awareness Month: Heartened By Signs of Progress

By: Margaret Hamburg, M.D.

For more than two decades, October has served as a time for us to honor and remember those affected by breast cancer, a disease that strikes almost a quarter of a million women every year and kills more than 35,000 each year.  We’ve likely all known someone who has been touched by this disease. Fortunately, as we recognize another Breast Cancer Awareness Month, we can be heartened by signs of progress in combating this disease.

Margaret Hamburg, M.D.Recently, researchers announced they had identified four distinct types of breast cancer, information that could fundamentally reshape our scientific understanding of this disease.  It could help explain why a drug therapy for one type of cancer may not work for another. The very real possibility that these findings—the product of a federal project called the Cancer Genome Atlas—will lead to new treatments is among the best news I’ve heard this year. 

And there’s much more work going on. The fact is that scientists in the federal government, academia, and industry and patient groups and patients are making supreme efforts to make cancer, if not curable in all cases, at least a treatable, chronic disease. Today, 40 percent of all drug development is in cancer research, and a significant part of that work is focused on breast cancer. At FDA, we’re proud that our experts contribute to this crusade by providing invaluable guidance to researchers and companies that are developing drugs as well as diagnostic and treatment devices for cancer.

Thanks to these many efforts, physicians have a steadily increasing number of therapies and tools to use in confronting breast cancer. In the last two years, our agency has added three drugs to the 16 previously approved treatments for late-stage breast cancer and approved two state-of-the-art tools for screening and detecting breast cancer in its earliest stages, when it is most treatable.

One of these devices, which was approved last year, is the first X-ray device that provides three-dimensional images of the breast, a level of detail that may help the examining doctor make a more accurate diagnosis, which could lead to more successful treatments and spare the need for additional tests for many women. 

And just a few weeks ago, FDA approved the first ultrasound device that can be used together with standard mammography to check dense breasts for early signs of cancer. This is an important advance for those women with dense breasts, who account for about 4 in 10 women who undergo mammography. Their dense breast tissue had been difficult to examine, putting these women at risk for late detection of breast cancer when the disease is difficult to treat.

FDA is involved in cutting-edge breast cancer research, working closely with outside scientists to help identify and develop promising new treatments. One major effort is to develop individualized therapies focused on the genetic make-up of specific breast tumors.  Our agency is cooperating with scientists in the National Institutes of Health, academia and industry in an innovative study that could expedite the development of more tailored treatments for breast cancer.  Patients with breast cancer are already among the earliest benefactors of the efforts towards targeted or personalized treatments, and more are in the works.

Breast cancer patients are playing a significant role in this broad-based, many-faceted effort to defeat cancer. Patients who participate in research are the backbone of scientific advances, because only through careful and systematic analysis of their disease can progress be made. Breast cancer patients are serving as patient representatives, playing an important role as members of advisory committees that help us to evaluate new drugs. They also provide essential information on what level of risks patients are willing to accept in order to gain access to potentially beneficial drugs.

While progress was made during the last two decades, a tremendous amount of work remains. I am confident, however, that through continued engagement with patients, the health professional community, academia and industry, breast cancer patients will have a wider range of available diagnostics and therapies, taking us one step closer to identifying a cure.

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

Treating Children with Cancer

September is National Childhood Cancer Awareness Month. Watch the below video in which two FDA experts discuss existing and new efforts to encourage the development of medicines for kids with cancer.

The conversation is between Robert “Skip” Nelson, M.D., Ph.D., deputy director and senior pediatric ethicist in FDA’s Office of Pediatric Therapeutics, and Gregory Reaman, M.D., associate director of the Office of Hematology and Oncology Products.

It begins with a discussion of FDA’s role in evaluating medications used to treat children with cancer and what measures are underway to encourage further development of these important drugs.

For More Information


New Pediatric Labeling Information Database

Cancer Liaison Program

Office of Hematology and Oncology Products

Friends of Cancer Research Honors Our Dr. Richard Pazdur

By: Margaret Hamburg, M.D.

One of the privileges of being the head of the Food and Drug Administration is being able to report on the accomplishments of the dedicated and highly skilled employees who are advancing the public health mission of our agency. Last night, I had the pleasure of participating in an event honoring one of our scientists for work that makes us particularly proud: his outstanding contribution to the fight against cancer.

Margaret Hamburg, M.D. and Richard Pazdur, M.D.Dr. Richard Pazdur, the director of our Office of Hematology and Oncology Products, joined Senator John McCain and Neera Tanden, president of the Center for American Progress, as a recipient of the Cancer Leadership Award from the Friends of Cancer Research, an important organization that helps advance medical progress.

Senator McCain and Ms. Tandem have been tireless advocates for public investment in cancer research, getting cancer drugs to market more quickly, and developing prevention strategies that are known to reduce the risk of cancer. Dr. Pazdur, who was honored for his leadership in cancer research, makes sure that the drugs used to treat cancer patients are not only available for patients who need them as fast as science will allow, but above all, that they are safe and effective.

Achieving these three goals requires highly sophisticated and frequently very difficult decisions. Thanks to Dr. Pazdur, who arrived at FDA more than a decade ago as a highly regarded physician and researcher, our agency is up to the task. He has helped make FDA a more transparent, innovative and compassionate organization grounded in solid science.

At FDA, we appreciate both his professional and personal qualities. As an FDA official and scientist, Dr. Pazdur explores hard data with industry, academia, and patient advocates in search of innovative approaches to expediting the approval of new drugs. When talking to cancer patients and their advocates, Rick Pazdur, the physician, responds with deeply-felt empathy. But even this great compassion does not weaken his resolve to perform his foremost duty, which is to ensure that the benefits of cancer drugs are not outweighed by their risks.

Dr. Pazdur’s leadership is a hallmark of FDA’s pursuit of science in the service of public health. I am proud to congratulate him on his richly deserved award on behalf of our entire agency.

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