FDA Seeks Comment on Proposed Health IT Strategy That Aims to Promote Innovation

By: Bakul Patel

Health information technology (IT) offers many benefits to the American people and health care providers. Health IT products, technologies and services can prevent medical errors, improve efficiency and health care quality, reduce costs and increase consumer engagement. They also can help with identification of, and quick response to, public health threats, and further health research. But while health IT benefits are far-reaching, technology can bring risks to patients if not designed, developed, implemented, or maintained properly.

Bakul PatelI’m glad to share that FDA, along with the Office of the National Coordinator for Health Information Technology (ONC), and the Federal Communications Commission (FCC), has recently released a report outlining our proposed framework for health IT. This report fulfills the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA) requirement that we develop a proposed strategy and recommendations on an appropriate, risk-based regulatory framework pertaining to health IT that promotes innovation, protects patient safety and avoids regulatory duplication.

We’ve developed a proposed framework that fulfills this requirement and would allow Americans to reap the benefits of health IT. This is a growing field with many benefits. For example, electronic health records allow providers to access accurate patient data. And computer-aided detection software can analyze an electrocardiogram (EKG) signal and help determine if a person is having a heart attack, helping providers give timely treatment.

In health IT, the best approach is a risk-based approach. We believe risk assessment should primarily focus on the function of the health IT product, not its platform. So we’ve identified three categories of health IT. The first is administrative and relates to functions such as billing and scheduling. This area has minimal risks for patient safety and does not require additional oversight.

The second relates to health management functions, including provider order entry, electronic communication and patient identification. In this category, we’ve identified four priority areas that can be scaled and applied throughout the health IT product lifecycle:

•              Promote the use of quality management principles;

•              Identify, develop, and adopt standards and best practices;

•              Leverage conformity assessment tools; and

•              Create an environment of learning and continual improvement.

Since safety risks for products in the health management category are sufficiently low, even if a technology in this category meets the definition of a medical device, FDA does not intend to focus oversight on it. Rather, FDA intends to focus our attention on the third category, which relates to medical device functions, such as computer-aided detection software and radiation treatment software. Such products are already FDA’s focus because they generally pose greater risk to patients than the products in the other two categories.

We’re not recommending that new areas of FDA oversight are needed.

Along with ONC and FCC, we will continue to develop this framework through public engagement, especially in fostering the further development of a quality-focused culture for health IT. We do not believe that regulation should be, or needs to be, the first approach used to reach this outcome. The agencies are holding a three day public workshop on May 13, 14, and 15 at the National Institute of Standards and Technology to discuss the framework and its components. We encourage consumers, providers, and health care organizations to register for the workshop and to submit comments. Health IT has brought, and continues to bring, many benefits and we are looking forward to engaging with you on this important topic.

Bakul Patel is senior policy advisor in FDA’s Center for Devices and Radiological Health.

For more information please visit these Web links:

FDASIA Health IT Report

Public Workshop – Proposed Risk-Based Regulatory Framework and Strategy for Health Information Technology, May 13-15, 2014

Proposed Risk-Based Regulatory Framework and Strategy for Health Information Technology Report; Notice to Public of Availability of the Report and Web Site Location; Request for Comments

Homing in Faster on Potentially Deadly Bacteria in Food

By: Nilda E. Villegas

Whether an outbreak of foodborne illnesses is caused by Listeria in cantaloupe, E.coli in salad or Salmonella in peanut butter, the faster the disease-causing bacteria are identified, the more illnesses can be averted.

Nilda VillegasLast year scientists based in FDA’s San Juan Laboratory were endowed with seed funding through a Health and Human Services HHSignite award to adapt traditional chemistry methods for the detection of foodborne pathogens. HHSignite (beta) is an internal competitive seed-funding opportunity to test new and unconventional ideas within HHS.

The team is being led by LCDR José Moreno of the U.S. Public Health Service, a microbiologist in FDA’s San Juan Laboratory. Working with Moreno on this project are Joseph Bloom, Ph.D., San Juan Laboratory science advisor, supervisory chemists Adalberto Cajigas and Héctor Espinet, science advisor Osvaldo Rosario, Ph.D., chemist Miguel A. Martínez, and staff fellow Fernando González, Ph.D.  FDA’s San Juan scientists were among 13 teams within HHS to receive such funding when Secretary Sebelius announced the finalists in June 2013.

San Juan Laboratory Team: Left to right in the back: Dr. Joseph Bloom, Dr. Osvaldo Rosario and Dr. Fernando González; standing in front Miguel A. Martínez and LCDR José Moreno.

San Juan Laboratory Team: Left to right in the back: Dr. Joseph Bloom, Dr. Osvaldo Rosario and Dr. Fernando González; standing in front Miguel A. Martínez and LCDR José Moreno.

Their proposal is to assess the utility and practicality of coupling CE-MS, which stands for Capillary Electrophoresis coupled with a Mass Spectrometer, that promises to be the workhorse of many analytical chemistry laboratories for use in identifying pathogenic microorganisms through the identification of specific proteins associated with pathogenicity by their fragmentation patterns. This essentially identifies specific “fingerprints” for disease-causing bacteria, such as E. coli, Salmonella, Listeria and Staphylococcus, in foods.

The research undertaken by Moreno and his team could result in faster screening methods for a wide array of pathogens as part of FDA’s food safety and food defense programs.

What’s next?

The San Juan team is continuing their research on this innovative screening tool to reduce testing times even more, and coordinating their efforts with their scientific colleagues in our Office of Regulatory Affairs and in our Center for Food Safety and Applied Nutrition on the mainland to protect the safety of the U.S. food supply.

Nilda E. Villegas is a Public Affairs Specialist in the San Juan District Office, part of FDA’s Office of Regulatory Affairs

Visiting India: The Importance of Biomedical Research and Quality

By: Margaret A. Hamburg, M.D.

As my busy and productive trip to India drew to a close, I had the opportunity for one final meeting and one last memory with a group of some of the country’s most extraordinary women. The occasion was a Women’s Roundtable in Mumbai, organized by the Confederation of Indian Industry (CII). It brought together a diverse collection of female industry and academic leaders in India for a thought-provoking discussion and a wonderful chance to share perspectives with these accomplished women.

Commissioner Hamburg and members of the Women's Roundtable in Mumbai

Commissioner Margaret A. Hamburg, M.D. and members of Women’s Roundtable in Mumbai, India.

We covered a wide range of important and timely topics and could have gone on for much longer had time allowed. We discussed the many challenges that professional women often face in today’s workplace including efforts to achieve work-life balance and the importance of educating, motivating, mentoring and empowering women at every stage of both their professional and personal development. We also focused on a number of pressing issues in biomedical research, clinical trials and the regulatory framework for food and drugs.

What was most striking about this remarkable group of women is the commitment each has made not only to support one another in their diverse professional endeavors, but also to work to improve the lives of people living in India and around the world.

Two themes emerged during our discussion: the importance of biomedical research in India, specifically clinical trial design and enrollment; and the importance of quality and the role these prominent leaders in the pharmaceutical and food production sectors can play in communicating why quality matters.

The group expressed concern about the status of the clinical trial system in India. Many Indians have been wary of the way clinical trials have been conducted and how trial participants are chosen and informed. India has been in the midst of a significant re-examination of its clinical trial infrastructure and the legal, regulatory, ethical and scientific required for future research.

Certainly, we understand the importance of a vibrant, reliable and transparent clinical trials system. The information FDA receives and reviews from clinical trials conducted in the U.S. and abroad is an essential part of the agency’s decision-making for all new and existing drugs. Without it, we would not have important information such as how a drug works, whether it is reasonably safe for patients, and how the human body metabolizes the drug. Additionally, clinical trials may represent a vital mechanism to access an investigational drug for patients living with serious and life-threatening diseases.

Over the years, the FDA has worked closely with academia, industry and the advocacy community to improve transparency around the design and conduct of the clinical trials used to generate data for medical product review and approval. The agency has been working with the health care and research community to improve a clinical trial subject’s understanding of what it means to participate in a clinical trial, as well as the specifics of the trial in which they are considering enrollment.

Because the information we learn from clinical trials has the potential to benefit people living around the world – and the trials may be conducted in a wide range of countries – it is important that government officials, industry, the research community and patient organizations work together to ensure appropriate human subject protections, rigorous clinical trial design, and needed health care. We look forward to continuing our discussions with government, industry and academia on these important topics.

Quality was a recurring theme during my visit to India. I was pleased that the women who participated in the roundtable were as committed to quality as I am.  As leaders in their industries and academic institutions, these women are uniquely positioned to help reinforce the message that quality matters and to educate the next generation of leaders in their respective industries and organizations about the importance of building quality into everything they do. And I was delighted that the group appreciated how smart regulation can help shape, support, and strengthen the product development and manufacturing processes that can help ensure continuous quality. In the words of moderator Swati Piramal, one of the highest ranking and most eminent leaders in Indian Pharma today, “good regulators make good companies.” And that combination can lead to better, safer and higher quality products – which benefits the health of people in both our nations and around the world.

Margaret A. Hamburg, M.D., is Commissioner of Food and Drugs

Another Strong Year for Novel New Drug Approvals

By: John K. Jenkins, M.D.

Last year marked another strong year for FDA approvals of novel new drugs, known as new molecular entities (NMEs).  In 2013, FDA’s Center for Drug Evaluation and Research (CDER) approved 27 NMEs last year – about the same as the 26 average NME approvals per year since the beginning of this decade.

John JenkinsMore important than the quantity of novel new drugs approved in 2013 is their quality – and the important new roles many of these drugs can serve in advancing medical care and the health of patients. We now have new safe and effective treatments for a wide range of serious medical conditions, such as late-stage breast cancer, chronic hepatitis C, metastatic melanoma, mantle cell lymphoma, chronic lymphocytic leukemia, homozygous familial hypercholesterolemia, pulmonary arterial hypertension, and many more. Some of these medications offer new hope to patients who previously had few or no treatment options.

Here are a few highlights of these approvals:

  • One-third of the NMEs approved in 2013 were identified by FDA as “first-in-class,” for example, drugs that use a new and unique mechanism of action for treating a medical condition;
  • One-third were also approved to treat rare or “orphan” diseases that affect 200,000 or fewer Americans who often have few or no drug treatment options;
  • Almost half of the 27 NMEs approved last year (13 of 27), were designated in one or more categories of Fast Track, Breakthrough, Priority Review, or Accelerated Approval. Each of these designations helps speed the development and/or approval process and is designed to help bring important medications to the market as quickly as possible;
  • Although FDA’s regulatory processes differ widely from those of foreign regulatory authorities, almost three-quarters (74%) of the NMEs approved by FDA in 2013 were approved first in the United States before any other country.

All of us at FDA are pleased and proud to be part of a team that helped bring these new drugs to market as safely and efficiently as possible. As always, while striving for efficiency in our review and approval of applications for new drugs, compromises were not made in our standards. To be approved, each NME had to demonstrate that it was safe and effective before being approved.

My colleagues and I look forward to another productive year serving the American public!

For more details about 2013’s approvals, please visit The Novel New Drugs Summary at: http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DrugInnovation/UCM381803.pdf

John K. Jenkins, M.D., is Director, Office of New Drugs, at FDA’s Center for Drug Evaluation and Research

Setting the Bar for Blood Glucose Meter Performance

By: Courtney Lias

Courtney Lias is Director of the Division of Chemistry and Toxicology Devices within the Office of In Vitro Diagnostics and Radiological Devices at FDA’s Center for Devices and Radiological HealthMany of the nearly 19 million Americans diagnosed with diabetes must monitor their blood glucose (sugar) frequently throughout the day using an at-home meter to make sure that their blood glucose is within a safe range. The ability to measure blood glucose at home has given people with this serious and chronic condition the ability to better control their blood sugar and thus avoid potential complications.

In the last 10 years there has been much advancement in the development of glucose meters. They are now smaller, require a smaller blood sample for each test and produce faster results.  However, their accuracy has improved little.

At FDA’s public meeting in March 2010 on this topic, the clinical and patient communities challenged the agency to improve performance of glucose meters. Feedback gathered from that meeting directly informed the creation of two draft guidance documents released this week. These documents set forth recommendations, which are justified to help ensure that these important devices are designed to be more accurate and reliable for the patients who need them. To address this need, this week we are proposing new recommendations for labeling, meter performance evaluation, manufacturing controls, and cleaning and disinfection procedures to help improve the accuracy and performance of blood glucose meters.

FDA recognized the need to optimize the safe use of blood glucose meters in two distinct settings: self-monitoring using devices purchased over-the-counter, and use in a clinical setting by health care professionals. FDA believes that by distinguishing where these devices are used, they can be better designed to meet the needs of their intended populations and ensure greater safety and efficacy.

Historically, devices used in these two settings have been studied using the same methods and standards. However, it has become increasingly clear that meters used in these different settings have unique characteristics and different design specifications. For example, critically ill patients in health care settings may have physiological variables, like abnormal oxygen levels, that could interfere with the accuracy of the blood glucose meter. Patients who use over-the-counter glucose meters and test strips at home vary in age, how much they know about how to use blood glucose tests, and other critical factors that might affect the  accurate use of the device.

To distinguish between FDA recommendations for blood glucose meters used in health-care facilities, and those intended for self-monitoring by lay-persons, the agency is issuing separate draft guidances for each one, that is:

  • prescription-use blood glucose meters, for use in point-of-care professional health-care settings, and
  • blood glucose devices purchased over-the-counter, intended for self-monitoring by lay-persons.

We believe that these recommendations will help ensure that glucose meters meet critical standards for accuracy in the hands of people with diabetes, who rely on them to manage their disease. Please help us in this effort by providing specific comments to these draft guidance documents to let us know if you agree with our recommendations or whether you have suggestions to further improve them.

Improving the quality of blood glucose meters will not solve all challenges for those who live with diabetes, but it may help millions of people to avoid complications and better achieve their health goals.

Courtney Lias is Director of the Division of Chemistry and Toxicology Devices within the Office of In Vitro Diagnostics and Radiological Devices at FDA’s Center for Devices and Radiological Health

Gregory Reaman Helps Make the World a Better Place for Children

By: Richard Pazdur, M.D.

I am privileged to work every day with many physicians and other health care professionals dedicated to advancing public health for all Americans. One of them is Dr. Gregory Reaman, who has been awarded the Leukemia & Lymphoma Society’s prestigious Return of the Child Award. Greg has devoted his career to finding better ways to treat and improve the outcomes for children with cancer.

Richard Pazdur, M.D.This award, presented in December in New Orleans, is given each year to a person who has made a major and lasting scientific or humanitarian contribution to the better understanding, management or treatment of pediatric hematological malignancies. (Hematological malignancies are the types of cancer that affect the blood, bone marrow and lymph nodes.)

Greg was honored for his exceptional leadership and accomplishments in the field of pediatric hematology and oncology during his lengthy career as a clinician and academic researcher and, since 2011, as a member of my team here at FDA. His primary work has focused on clinical trials for children with acute lymphoblastic leukemia (ALL) and the early phases of developing experimental drugs for children. Greg’s leadership in these areas has led to significant improvements in the care of children with ALL, as well as the facilitation of new drug development for children with cancer.

Dr. Gregory Reaman accepts award

Dr. Gregory Reaman (left) accepts The Return of the Child Award Presented by LLS CEO John Walter

Greg is a previous recipient of the Leukemia & Lymphoma Society’s Tree of Life Award, which honors those who have played a major role in improving the quality of life of patients and their families. Through these and many other efforts, his extensive research has helped to advance how pediatric blood cancers are treated today.

At FDA, where he is associate director of oncology sciences, Greg is using new mechanisms created by recent laws to facilitate public discussion and promote drug research and development for children with cancer.

Greg is also executive director emeritus and senior attending physician at Children’s National Medical Center in Washington, D.C. Throughout his distinguished career, Greg has held other leadership positions at Children’s and at the National Childhood Cancer Foundation. He is also a tenured professor of pediatrics at the George Washington University School of Medicine and Health Sciences. 

I know of no greater endeavor in life than to dedicate one’s work to making this world a better place for children, and I know of no individual more deserving of an award for such efforts than Greg. I know I speak for my entire staff and all of us at FDA when I congratulate Greg on his achievements.

Richard Pazdur, M.D., is director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research

We Moved Forward on Many Fronts This Year

By: Margaret A. Hamburg, M.D.

At the FDA, the agency that I’ve had the privilege to lead for the past five years, I am gratified to report that we have a lot to be proud of this year. In fact, this past year’s accomplishments on behalf of public health have been as substantial as any in FDA’s recent history.

Margaret Hamburg, M.D.We moved significantly forward, for example, in creating a system that will reduce foodborne illness, approving novel medical products in cutting-edge areas of science, and continuing to develop our new tobacco control program. We worked successfully with Congress and with regulated industry to reach agreement on a number of difficult issues, while continuing to use the law to the full extent possible to protect consumers and advance public health.

While there were many significant actions and events to recognize, below are some of the highlights of 2013.

In the foods area, there were many new actions this year that will have a long-standing impact on improving our food supply for consumers. Throughout the year we have been proposing new rules to reach the goals set forth by the FDA Food Safety Modernization Act (FSMA). These science-based standards will help ensure the safety of all foods produced for our market, whether they come from the U.S. or from other countries.

We also took important steps towards reducing artery-clogging trans fat in processed foods, and understanding the health impact of arsenic in rice. With a final rule that defines when baked goods, pastas and other foods can be considered free of gluten, people with celiac disease can have confidence in foods labeled “gluten free.” And we are studying whether adding caffeine to foods may have an effect on the health of young people and others.

There have likewise been many accomplishments in advancing the safety and effectiveness of medical products. We worked closely with Congress on the recently enacted Drug Quality and Security Act, which contains important provisions relating to the oversight of human drug compounding. The law also has provisions to help secure the drug supply chain so that we can better help protect consumers from the dangers of counterfeit, stolen, contaminated, or otherwise harmful drugs.

Using tools provided by last year’s landmark Food and Drug Administration Safety and Innovation Act (FDASIA), we are continuing to improve the speed and efficiency of medical product reviews, including those involving low-cost, high quality generic drugs and innovative new medical devices. The average number of days it takes for pre-market review of a new medical device has been reduced by about one-third since 2010. The percentage of pre-market approval applications that we approve has increased since then, after steadily decreasing each year since 2004.

We launched a powerful new tool to accelerate the development and review of “breakthrough therapies,” allowing FDA to expedite development of a drug or biologic (such as a vaccine) if preliminary clinical evidence indicates that it may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. This offers real opportunities to get promising drugs more quickly to patients who need them. In fact, using this new approach, FDA recently approved two advanced treatments for rare types of cancer and one for hepatitis C. We have also strengthened efforts to ensure product quality, increased protection of the drug supply chain, and reduced drug shortages.

We confronted the growing misuse of powerful opioid pain relievers by advising manufacturers on how to make these drugs harder to abuse with formulations that are more difficult to crush for inhalation or dissolve for injection. And we recommended that hydrocodone combination products be subject to stricter controls to help prevent abuse. 

We took an important step towards fighting the development of antibiotic-resistant bacteria by implementing a voluntary plan to phase out the use of antibiotics to enhance the growth of food-producing animals, and to move any remaining therapeutic uses of these drugs under the oversight of a licensed veterinarian. So-called “production” use is considered a contributing factor in the development of bacteria that are resistant to the antibiotics used in human medical treatment.

In many areas of our work we are supporting the emerging field of personalized medicine. Advances in sequencing the human genome and greater understanding of the underlying mechanisms of disease, combined with increasingly powerful computers and other technologies, are making it possible to tailor medical treatments to the specific characteristics, needs, and preferences of individual patients.

Many cancer drugs today are increasingly used with companion diagnostic tests that can help determine whether a patient will respond to the drug based on the genetic characteristics of the patient’s tumor. In May, FDA approved two drugs and companion diagnostic testing for the treatment of certain melanoma patients with particular genetic mutations.

Advances in science and technology are also seen in the creation of new medical devices. For example, 3-D printing - the making of a three-dimensional solid object from a digital model – was once considered the wave of the future. But in February, FDA cleared for marketing a device created by 3-D printing – a plate used in a surgical repair of the skull that is built specifically for the individual patient.

While we have worked hard to get therapies to patients, we are at the same time using the tools available to us to remove unsafe and dangerous products from the market. In November, we used new enforcement tools provided by the food-safety law to act quickly in the face of a potential danger to public health presented by certain OxyElite Pro products. These supplements had been linked to dozens of cases of acute liver failure and hepatitis. After FDA took action, the manufacturer agreed to recall and destroy the supplements.

Finally, we made significant progress in implementing the letter and spirit of the Family Smoking Prevention and Tobacco Control Act. We have signed contracts with numerous state and local authorities to enforce the ban on the sale of tobacco to children and teens; conducted close to 240,000 inspections; and written more than 12,100 warning letters to retailers. And, in the first quarter of 2014 we will launch a public education campaign aimed at reducing the number of young people who use tobacco products.

All of us take great pride in the skill and vigor with which we overcame the year’s challenges and new demands. And so, as the year draws to a close, I extend my gratitude to the employees at the FDA who work tirelessly on behalf of the American public year in and year out. To all of our stakeholders, my heartfelt wishes for a joyous holiday season and a safe and healthy 2014.

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

Driving Innovation Is a Key Part of Our Mission

By: Margaret A. Hamburg, M.D. 

Ensuring the safety of the food supply and the safety and effectiveness of drugs, devices and biologics has always been at the core of FDA’s responsibility to protect the public health – and always will be.

Margaret Hamburg, M.D.But what is often lost or neglected in discussions about FDA’s vigilance on behalf of consumer and patient safety is how this kind of oversight and regulation, when done right, can be a key driver of innovation throughout society. Whether it involves the development of some remarkable new drug or oversight of a particular product, the addition of rigorous regulatory science from FDA helps to safeguard the safety and effectiveness of innovative new products; ensures that these scientific achievements quickly reach their full potential; and builds a pathway for ongoing innovation. That’s why we continue to adapt and change regulatory policies in response to – and in anticipation of – scientific opportunities so that they can best be harnessed to improve health and medical care.

There are many recent examples of this thoughtful approach to innovative products. For instance, we have put in place a new breakthrough pathway to market for promising drugs that may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. Using this new approach, we have already approved two treatments for rare types of cancer and one for hepatitis C. And there may be considerably more in the weeks and months ahead because many companies have expressed interest. So far we have received 119 requests for designation and granted 35. 

In a very different realm, another example is mobile medical apps. We took a measured and sensible approach that promotes innovation when we finalized guidance related to these devices earlier this year. Although many mobile apps pertain to health, CDRH intends to focus its oversight on a very small subset of those mobile apps that are medical devices. We have called that subset “mobile medical apps” and we are approaching their regulatory oversight within a risk-based framework. We believe this approach will promote innovation while protecting patient safety by focusing on those mobile apps that pose greater risk to patients. 

As explained in the final mobile medical application guidance, our regulation of software as a medical device – and a mobile app is software – is based on risk and functionality, and that functionality should be treated the same regardless of the platform on which it is used. For example, an electrocardiography device – an ECG machine – that measures heart rhythms to help doctors diagnose patients is still an ECG machine regardless of whether it is the size of a bread box or the size of a smartphone. The risks it poses to patients and the importance of assuring for practitioners and patients that it is safe and effective are essentially the same. 

And, just a few weeks ago, we authorized for marketing four innovative gene-sequencing devices. Two of these products comprise a system that allows laboratories to develop clinical tests that can look at a person’s genetic makeup and detect abnormalities that could be responsible for illness. FDA realized the innumerable uses of these systems from the outset, and rather than focusing on specific diseases or areas of the genome, we took a tool-based regulatory approach. We assessed whether the devices overall measure what they are intended to measure accurately, reliably and precisely so that there can be greater confidence in the test results. These types of genome sequencers represent a significant step forward in the ability to generate genomic information that may ultimately improve patient care.  

Each of the new medical products we approve and usher to the market involves a balancing of risks and benefits, which is based on study and evaluation of hard data and the best available science. It is a huge responsibility that FDA is charged with, nowhere more so than when dealing with unfolding technologies that offer enormous potential – but that also may present real risks for people and their health.

We know there will be new and continuing challenges that arise with additional technological developments and advancements in science and medicine. This is why FDA’s regulatory role in these emerging areas continues to develop as well, sometimes even as the technologies themselves are taking shape 

But our goal remains constant – to protect the public health through smart regulation that helps to enhance innovation and ensure that new medical technologies have real value to the people who will use them, and that they are used effectively and safely to address their needs.

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

Gene Sequencing Devices Are ‘Next Generation’

By: Jeffrey Shuren, M.D.

Just for a moment, imagine a scenario in which you have an illness that has eluded diagnosis. The usual suspects have been ruled out and no one knows exactly what’s making you sick. 

Using medical devices that FDA has now cleared for marketing, a laboratory could sequence your genome to look for any abnormalities in your genes that could be responsible for your illness. This information would be relayed to your doctor and used to determine the course of treatment.

This is called “next generation sequencing” because it’s another step towards a future in personalized medical care that few of us could have envisioned even a decade ago. 

First, let’s define some terms. A genome is the complete set of genetic information in your body. This information is held in sequences of DNA, and gene sequencing from your whole blood allows laboratories to look for genetic variations that could hold the key to the causes of disease and the right treatment. 

FDA is clearing the marketing of four gene-sequencing devices. Two of the devices make up the first test system authorized for marketing that allows laboratories to sequence a patient’s genome for any purpose. The software compares the patient’s sequence to a normal human genome sequence used for reference and identifies the differences. 

The other two devices are used to detect changes in the CFTR gene, which can result in cystic fibrosis, a disease inherited through a faulty CFTR gene from both parents. More than 10 million Americans are carriers of cystic fibrosis (they have only one faulty copy), and one of these tests could be used to identify men and women with the faulty CFTR gene. The second test looks for other, perhaps unexpected, mutations in the CFTR gene that could be having an impact on the patient’s health. 

Regulatory science – the science of developing new tools, standards and approaches to assess the safety, effectiveness, and quality of FDA-regulated products – played a key role in FDA’s readiness to assess these revolutionary devices. Knowing the potential of next generation sequencing to advance personalized medicine, FDA researched next generation sequencers to understand how they work and their likely limitations. By the time Illumina (the San Diego-based biotechnology company that developed the next generation sequencing devices authorized for marketing) walked in the door, FDA had the expertise and tools needed to timely review the submissions for the next generation sequencers. 

The regulatory science development efforts that contributed to the timely marketing authorization of these devices will continue to help advance this important technology. We are also collaborating with the National Institute of Standards and Technology – a federal agency that works to advance measurement science, standards and technology – and other agencies to develop human genome materials that can serve as reference materials so that other labs and researchers can assess the performance of their gene sequencers quickly, effectively, and at a lower cost.

We are working on many fronts to achieve the promise of personalized medicine, so that patients can get medical treatments that are right for them. Clearing the marketing of these four devices moves us closer to that goal.

For further perspective, read a new article in the New England Journal of Medicine by FDA Commissioner Margaret A. Hamburg, M.D. and National Institutes of Health Director Francis S. Collins, M.D., Ph.D.

Jeffrey Shuren, M.D., is Director of FDA’s Center for Devices and Radiological Health

FDA Takes a Responsive Approach to Mobile Web

By: Chris Mulieri

Since January 1, over 30 million visitors have come to FDA’s website. We know that they come to FDA.gov to get reliable and up-to-date information on everything from food and drug recalls to medical product alerts to regulations and guidance for industry…and the list goes on.

We also know that an increasing number of our visitors use mobile devices to get this information. In the last year alone, the number of mobile visits to FDA.gov has nearly doubled, and now 25 percent of our visitors use a tablet or smartphone to access the site.

Our Visitors Come First

As director of web and digital media for the FDA, I lead a team that is committed to providing a positive experience for visitors to FDA.gov, including our mobile visitors. We are also responsible for supporting the Digital Government Strategy, issued by the White House, which calls for federal agencies to provide government information on demand and on any device.

In plain English, that means that we need to provide a single FDA.gov site that’s available anytime, anywhere, on any device.

To meet the needs of our mobile visitors without creating a separate mobile website, we turned to a proven web development approach called responsive design. This approach uses special code to ensure that web content is easy to read and scroll across a wide range of devices, from desktop computers to mobile phones. Since the first of the year, mobile-friendly responsive designs have been implemented successfully on a number of large non-government websites, such as NYTimes.com and NPR.org. And now, on FDA.gov as well.

Check Out FDA.gov on Mobile

We are excited to announce that our first responsive pages on FDA.gov launched on November 15. If you are using a smartphone or tablet, go to FDA.gov and check it out. You’ll see our most popular content, such as recalls, news, and safety alerts, is now formatted to fit your screen. We’ve also made it easier for mobile visitors to tell us about a problem with the products that FDA regulates, such as food, drugs, medical devices, and animal food and drugs.

This is just the beginning. In keeping with best practices in web design, we are taking an iterative approach to mobile on FDA.gov. In other words, we will apply the lessons learned from our visitors’ experiences to share more mobile friendly content as it becomes available.

Our ultimate goal is to create one website that will provide a quality experience for all visitors who seek information that will benefit their health and safety.

Chris Mulieri is the director of web and digital media for the Food and Drug Administration