FDA’s Take on the Executive Order and National Strategy to Combat Antibiotic-Resistant Bacteria

By: Margaret A. Hamburg, M.D.

Few issues in public health today are as critical and time urgent as combating the growing threat of antibiotic resistance. We are delighted to stand with the White House in the development and response to the President’s Executive Order and the National Combating Antibiotic-Resistant Bacteria (CARB) Strategy. Fighting antibiotic resistance is both a public health and national security priority. FDA has played a key role in the development of this important effort, and we already have made strides on many fronts to make sure that we have effective antibiotics for the future.

Margaret Hamburg, M.D.Antibiotics are precious medicines that have saved millions of lives by treating infections caused by bacteria. But their misuse, and overuse, has serious health consequences and has contributed to antibiotic resistance—in which these drugs become less effective, or ineffective, against harmful bacteria.

The consequences of antibiotic resistance must not be underestimated. With each passing day, concern mounts that more patients will have few or no therapeutic options because of resistance to available therapies. In fact, the Centers for Disease Control and Prevention (CDC) estimates that each year at least 2 million illnesses and 23,000 deaths in the United States are caused by antibiotic-resistant bacteria.

It is a high priority for FDA to work with our partners to find solutions for this serious public health problem.

To address the need for effective antibiotics, FDA is working hard to ensure development of new strategies. These include vaccines to help prevent infection with bacteria in the first place; devices to aid in the accurate diagnosis of the cause of infection and of resistant infections; and new drugs to treat patients with serious infections for whom we have few, or no, treatment options because of resistance to currently available antibiotics.

We have been engaging with outside groups to advance the science of clinical trials. For instance, we have worked with the Clinical Trials Transformation Initiative on increasing the efficiency of clinical trials; with the Engelberg Center for Health Care Reform at the Brookings Institution to address overarching issues in antibiotic development, such as the major technical and financial barriers; and with the Biomarkers Consortium of the Foundation for the National Institutes of Health (NIH) on endpoints for studying antibiotics in clinical trials. In fact, we recently joined NIH to hold a workshop to examine the technical challenges related to antibacterial product development and to discuss innovative regulatory and clinical trial approaches for bringing new products to market. (The final agenda and presentations are available online.)

FDA also has been actively implementing the Generating Antibiotics Incentives Now (GAIN) Act, a provision within the Food and Drug Administration Safety and Innovation Act (FDASIA) to promote the development of antibacterial and antifungal drugs. To date, FDA has granted 57 Qualified Infectious Disease Product (QIDP) designations under GAIN to 39 different unique molecules. Antibiotics that have a QIDP designation receive, upon request, priority review, typically shaving four months off review times, and fast track designation, which results in early consultation, including on clinical trial design, between FDA and antibiotic sponsors. QIDPs also can receive an additional five years of marketing exclusivity in addition to existing exclusivity periods at the time of approval. We’re pleased that already three QIDP designated antibacterial drugs have been approved in the past few months: Dalbavancin in May 2014, Tedizolid Phosphate in June 2014 and Oritavancin in August 2014.

Furthermore, FDA promotes the appropriate and responsible use of antibiotics in clinical medicine. Antibiotic labels contain information for health care professionals and patients on appropriate use. And we work to improve the integrity of the global supply chain for pharmaceutical products to minimize the chance of a patient receiving a substandard drug, which in some instances could promote antimicrobial resistance.

In addition, FDA has developed—and is working to implement—two strategies to ensure the judicious use of medically important antimicrobial drugs in food-producing animals. This is a vital step to better protect antibiotic effectiveness for both human and animal populations. Accordingly, we asked the manufacturers of these antibiotics used in food-producing animals to remove all growth promotion indications. Once their labels have been changed, the products can no longer be used legally for growth promotion purposes, or without veterinary oversight. We have now secured the commitment of all 26 affected animal health companies, and 31 products have been withdrawn from the market. Two other companies have implemented label changes and we will be working with the other companies to make sure that they do so as well. The second track will ensure that all remaining therapeutic uses of the affected medically important antibiotics in food-producing animals will take place under the supervision of a veterinarian. The agency continues to work under a three-year transition period, and we remain encouraged by the process.

A successful strategy to combat antibiotic-resistant bacteria will require effort and input from all involved groups, including from health care professionals and patients themselves. For our part, FDA continues to work with government partners, product developers and the scientific community as well as other critical stakeholders to address the unique and complex regulatory, scientific and policy challenges associated with this public health issue. The Executive Order and CARB strategy announced today will clearly boost our and the nation’s efforts to meet these challenges more effectively.

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

Teachers Learn About the Science Behind Food Safety and Nutrition

By: Sharmi Das

This past July, middle and high school teachers got a taste of the latest food safety and nutrition information at the FDA’s 15th annual summer training program for school teachers. The week-long program aims to improve food safety and nutrition education in U.S. schools so students are better armed to make nutritious food choices and understand the proper handling of food.

Sharmi DasThe program, called Science and Our Food Supply, uses a curriculum co-developed by FDA and the National Science Teachers Association (NSTA). To date, 652 teachers (from all 50 states, the District of Columbia, and some U.S. Territories) have completed the weeklong training. This year, 32 teachers from 22 states participated in the program and will return to their own schools to teach the curriculum this school year. This class included teachers who specialize in biology, chemistry, food science, health, and family and consumer sciences such as culinary arts.

The creation of a strong network of teachers and students around the country who are “food savvy” is a measure of the program’s success. The program’s participants will reach an estimated 3,200 new students, as well as 640 additional teachers in daylong train-the-teacher sessions, this coming school year. These estimates conservatively reflect the reach of this program – many teachers report using this curriculum to educate consumer groups within their communities, as well as sharing their new knowledge with their own friends and family.

Teachers participating in the 15th year of the Science and Our Food Supply program, FDA/CFSAN

Teachers participating in the 15th year of the Science and Our Food Supply program, FDA/CFSAN

During the weeklong program, teachers learned about the journey food takes from farm to table. The training included basic microbiology techniques in a University of Maryland teaching lab, along with introductions to the latest food safety and nutrition research from scientists at FDA’s Center for Food Safety and Applied Nutrition (CFSAN) and USDA’s Beltsville Agricultural Research Center (BARC). The teachers heard about new and ongoing research on food safety, nutrition and nutrition labeling, food allergies and food allergen labeling, and cosmetics safety.

In these settings, the teachers not only listened to presentations about relevant topics, but were given ample time to interact with the speakers. Teachers were excited at the opportunity to “ask the experts” many of the questions their students had brought up in class. Lively discussions on the topics of food allergies, color additives, and cosmetics helped teachers understand FDA’s role in regulating several products.

Teachers participating in the 15th year of the Science and Our Food Supply program, FDA/CFSAN

Train the trainer…. Teachers learning about nutrition and food safety through the Science and Our Food Supply program at FDA/CFSAN

At the end of the week, teachers reported on the many ways the training and curriculum will be used to teach their students about food safety and nutrition. “I feel like I can better explain and allow them to investigate better/healthier food choices,” said Victoria Obenchain, of Moraga, Calif. The teachers were also given a variety of educational materials and resources to help them use the curriculum in their own schools.

The FDA-NSTA training program immerses educators in the world and culture of food safety and nutrition for a full week. This experience provides school teachers with a comprehensive farm-to-table perspective on food safety and nutrition, allows them to become familiar with the many and varied backgrounds of professionals in this field, and helps to educate thousands of students about Science and Our Food Supply.

Sharmi Das is the Director of the Division of Education, Outreach and Information in the Office of Analytics and Outreach in FDA’s Center for Food Safety and Applied Nutrition

Clinical Trials: Enhancing Data Quality, Encouraging Participation and Improving Transparency

By: Margaret A. Hamburg, M.D.

Today FDA is announcing important steps that the agency plans to take to enhance the collection and availability of clinical trial data on demographic subgroups – patient populations divided by sex, race/ethnicity or age.

Margaret Hamburg, M.D.Section 907 of the 2012 FDA Safety and Innovation Act directed us to take a closer look at the extent to which clinical trial participation and the inclusion of safety and effectiveness data by demographic subgroups is included in medical product applications, report our findings, and then, within one year, produce an action plan with recommendations for improvements.

Our report, issued on August 20, 2013, found that the agency’s statutes, regulations, and policies generally give product sponsors a solid framework for providing data in their applications on the inclusion and analysis of demographic subgroups. Overall, sponsors are describing the demographic profiles of their clinical trial participants, and the majority of applications submitted to FDA include demographic subset analyses. We also found that FDA shares this information with the public in a variety of ways. Now, one year later, we’re releasing the FDA Action Plan to Enhance the Collection and Availability of Demographic Subgroup Data, which we developed after extensive interaction with stakeholders.

The action plan includes 27 action items that are designed to meet three overarching priorities – improving the completeness and quality of demographic subgroup data collection, reporting and analysis (quality); identifying barriers to subgroup enrollment in clinical trials and employing strategies to encourage greater participation (participation); and, making demographic subgroup data more available and transparent (transparency).

In addition to the action plan, we’re publishing a final guidance entitled, “Evaluation of Sex-Specific Data in Medical Device Clinical Studies.” It was written in response to the fact that certain medical devices may yield different responses in women than men, and yet women are under-represented in some medical device studies. This has led to less information for women regarding the risks and benefits of using these devices.

The guidance includes recommended methods for clinical study design and conduct to increase enrollment of men and women, if needed, and ways to analyze data for sex differences. FDA has held a series of public workshops to raise awareness about common strategies for enhancing recruitment and retention of women in medical device clinical trials. Fully integrating this final guidance into the templates used by FDA’s reviewers of medical devices, and providing a webinar for industry on how to use the guidance, comprise one of the 27 items in our action plan.

I hope you’ll find that the action plan is responsive and pragmatic and, most importantly, when fully implemented, it will improve medical care and public health. Many of the steps it outlines will have a broad impact on the work of FDA’s medical product centers and will require great thought and planning as they are implemented, depending on current evidence and available resources. The action items range from relatively short-term goals that can be achieved in a year, to others that will take 1-3 years, to a small number that will require a longer period, 3-5 years, to achieve.

Although the plan certainly places significant responsibilities on FDA’s medical product centers and other FDA offices, it also engages our partners inside and outside of government to share the responsibility for this important mission. For example, industry is being asked to help develop and share best practices for encouraging broad clinical trial participation, and the National Institutes of Health will be participating in several research projects with FDA.

We know that richer information is collected when different subgroups are enrolled in pivotal studies for medical products. This kind of enrollment in turn gives us greater assurance in the safety and effectiveness of the medical products used by a diverse population.

To set the plan in motion quickly, FDA is setting up a steering committee that will oversee implementation, come up with metrics for measuring progress and be responsible for planning a public meeting to be held within 18 months after release of the plan. FDA has already set up a website where the public will be able to track the agency’s implementation progress. That website will be updated on a regular basis.

Also, we’re reopening our Section 907 public docket to solicit comments for the action plan. I encourage everyone to review the document and consider how you might be able to partner with FDA and others in encouraging necessary and appropriate demographic subgroup diversity and representation.

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

Providing Easy Access to Medical Device Reports Submitted to FDA since the Early 1990s

By: Taha A. Kass-Hout, M.D., M.S. and Jeffrey Shuren, M.D., J.D.

Taha Kass-Hout

Taha A. Kass-Hout, M.D., M.S.

In addition to food and drugs, FDA has regulatory oversight of tens of thousands of medical devices ranging from bandages and prosthetics to heart valves and robotics. These products are used by millions of Americans, and they are essential, well-performing tools of modern healthcare, but occasionally they present a safety issue due to risks not identified in prior studies, a malfunction, a problem with manufacturing, or misuse.

These incidents are collected in a publicly available FDA database called MAUDE – short for Manufacturer and User Facility Device Experience. As part of the openFDA project, there is now an Application Programming Interface (API) for this dataset, which provides a way for software to interact directly with the data. This API will allow developers and researchers to easily query thousands of reports dating back to the early 1990s.

The API can be a powerful tool for generating hypotheses for further investigation or inquiry and can inform the development of safer, more effective technologies. For example, it can help identify new, potential safety signals as well as which classes of devices may be associated with particular adverse events.

Jeffrey Shuren

Jeffrey Shuren, M.D., J.D.

There are some necessary caveats to this API. The dataset is a record of reports submitted to FDA, and not a definitive accounting of every incident with every device. It may contain incomplete, inaccurate, unverified, or biased data. Thus, it cannot be used to determine incidence. And the appearance of a device in a report does not mean that cause-and-effect has been determined. Therefore, these data should be used in the context of other available information. It’s also important to note that the data made available under this initiative do not contain anything that potentially could be used to identify individuals or reveal other private information.

This API is the latest in a series of openFDA releases that have made publicly available data more easily accessed and queried. We believe that these tools can be used by developers and researchers to make insights that fuel new, innovative products (such as mobile apps and websites), and that help protect and promote the public’s health. Over the last two months, openFDA has released several APIs related to drugs, food, and devices. Together, they help provide perspective on the work FDA is doing, and make the public health data the agency is developing easier to access and utilize.

By design, openFDA is a research and development project that draws on community involvement. We are actively involved in the openFDA communities on GitHub and StackExchange, and encourage people interested in the project to participate in those communities. Together, we can make openFDA into a more useful, more powerful resource for the protection and advancement of the public health.

In addition to providing datasets, openFDA encourages innovative use of the agency’s publicly available data by highlighting potential data applications, and providing a place for communities to interact with one another and with FDA domain experts.

Taha A. Kass-Hout, M.D., M.S., is FDA’s Chief Health Informatics Officer and Director of FDA’s Office of Informatics and Technology Innovation

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

See more at: http://blogs.fda.gov/fdavoice/#sthash.COpthK14.dpuf

Providing Easy Public Access to Prescription Drug, Over-the-Counter Drug, and Biological Product Labeling

By: Taha A. Kass-Hout, M.D., M.S.

Every prescription drug (including biological drug products) approved by FDA for human use comes with FDA-approved labeling. The labeling contains information necessary to inform healthcare providers about the safe and effective use of the drug for its approved use(s). Once a prescription drug is approved, the labeling may be updated as new information becomes available, including, for example, new approved uses, new dosing recommendations, and new safety information. Thus, the approved labeling is a “living document” that changes over time to reflect increased knowledge about the safety and effectiveness of the drug.

Taha Kass-HoutIn some cases, the approved labeling for a prescription drug can be extensive, consisting of 20,000 words or more. This amount of information, while important to guide safe and effective use of the drug, can present formidable challenges. For example, it can be a daunting task to study more than one labeling to better understand a class of drugs, or to compare drugs, and to keep up with their regular changes. Although they have been publicly available for many years on FDA’s website, now this labeling is available on openFDA through an Application Programming Interface (API), which provides a way for software to interact directly with the data.

For several years, the labeling has been posted publicly in Structured Product Labeling (SPL) format at http://labels.fda.gov/. The SPL format enhances the ability to electronically access, search, and sort information in the labeling. The SPL files are also available at the National Library of Medicine’s DailyMed site and can be downloaded. We’ve created an API for the data to supplement (not replace) these resources, and to provide easy and timely access to changes or updates to the labeling.

The openFDA drug product label API provides access to the data for nearly 60,000 prescription and over-the-counter (OTC) drug labeling. The prescription labeling includes sections such as the “Indications and Usage” and “Adverse Reactions” sections and the OTC labeling includes “Purpose” and “Uses” headings and so forth.

This API can be used, for instance, to identify those medications that have a Boxed Warning, that have lactose as an inactive ingredient, that have a known interaction with grapefruit juice (or other fruit juices and where the labeling states “the concomitant use of DRUG-X with grapefruit juice is not recommended”), and to answer other queries.

This API is just one more example of how openFDA is helping make publicly available data more accessible and useful. Since the first API for adverse events was posted on June 2, 2014, there have been more than 2.6 million API accesses with approximately 20,000 internet devices connected to the adverse events API alone, and more than 30,000 unique visitors to the site.

It’s very important to note that the labeling for prescription drugs is proposed by the applicant, reviewed by FDA, and approved by FDA. The labeling for OTC medications is also either approved by FDA or must conform to applicable regulations that govern the content and format of OTC drug labeling that are not pre-approved by FDA.

As a research and development project, openFDA is a work in progress (Beta phase), and we are eager to learn from the developer and research communities what possible uses these data might have. We are also interested in hearing from the community about other publicly available FDA datasets for which an API might prove useful.

We are actively involved in the openFDA communities on GitHub and StackExchange, and encourage people interested in the project to participate in those communities. In addition to providing access to datasets, openFDA encourages innovative use of the agency’s publicly available data by highlighting potential data applications, and providing a place for community interaction with one another and with FDA domain experts.

Over time, we hope that openFDA can become an important resource where developers, researchers, and the public at large will learn about the medications and other FDA-regulated products that protect and promote the health of Americans.

Taha A. Kass-Hout, M.D., M.S., is FDA’s Chief Health Informatics Officer and Director of FDA’s Office of Informatics and Technology Innovation

FDA’s JumpStart program: Supporting drug innovation

By: Lilliam Rosario, Ph.D.

When it comes to public health, the U.S. Department of Health and Human Services (HHS) recognizes that innovation drives success.

Lilliam RosarioAs part of the HHS Innovates program, HHS Secretary Sylvia Mathews Burwell and Deputy Secretary Bill Corr acknowledge excellence in the field with the Secretary’s Pick Award, an honor that identifies and celebrates internal innovation by HHS employees.

I’m proud that this year, the winner of one of three Secretary’s Pick Awards was the Food and Drug Administration’s Office of Computational Science (OCS), part of the Office of Translational Sciences (OTS) in the agency’s Center for Drug Evaluation and Research (CDER). OCS received the award for its work in developing CDER’s JumpStart program, an innovative initiative dedicated to enhancing the efficiency of CDER’s new drug development and review process.

The JumpStart program provides CDER’s new drug review teams with clinical trial data analyses early in the review process when they assess quality, data composition, exploratory analyses, and tools for the analyses. It gives the reviewers a “jump start” on their review providing the information on the quality of the submission as well as analyses to support an effective and efficient evaluation of the medical product submission. You can learn more about JumpStart here. 

Our congratulations to the two other Secretary’s Pick Award recipients, the “Breast Cancer Startup Challenge,” led by the National Cancer Institute, and “Whole Genome Sequencing: Future of Food Safety,” led by the Centers for Disease Control and Prevention. It is a great honor to be recognized side by side with these two innovative programs!

We are proud of the team effort involved in making the JumpStart program a success, and look forward to continued efforts and innovative actions that will help bring safe, effective, and high quality new drug therapies to the American public as efficiently as possible.

For more information on HHS Innovates, visit HHS Innovates Celebrates 7th Round of Innovations!

Lilliam Rosario, Ph.D., is Director, Office of Computational Science, Office of Translational Sciences, at FDA’s Center for Drug Evaluation and Research

Stem cell therapy: FDA regulatory science aims to facilitate development of safe and effective regenerative medicine products

By: Steve Bauer, Ph.D.

One of FDA’s primary missions is to make sure that the products we approve are safe and effective. There is tremendous interest in the development of regenerative medicine, including numerous proposed products that rely on stem cells. Stem cells have the ability to generate more stem cells or to turn into more mature cell types such as nerve- or bone-producing cells. These properties make stem cells potentially well suited for use in regenerative medicine. They might be used in repairing heart, nerve, and brain damage or in treating diabetes and other diseases by repairing or replacing cells and tissues.

Steve Bauer

Steve Bauer, Ph.D., chief of the Cellular and Tissues Therapy Branch, Division of Cellular and Gene Therapies, in the Office of Cellular, Tissue and Gene Therapy at CBER.

Because stem cells can change based on their surroundings, whether during growth outside of the body or following injection into the body, ensuring the safety of effective regenerative medicine products can be challenging. One type of adult stem cell, the multipotent marrow stromal cell (MSC) — more popularly called the mesenchymal stem cell — is the subject of a great deal of research in regenerative medicine. These cells can divide repeatedly, making additional cells, and under the right conditions can be turned into a variety of more specialized and mature types of cells. Depending upon the culture conditions, these more specialized cells have the potential to produce cartilage, bone, and fat, and help with control of inflammation and immunity.

MSCs can be obtained from bone marrow and adipose tissue (fat) and can be grown outside of the body to produce the large numbers needed for many proposed clinical trials. Donated MSCs can also suppress the immune system in individuals who receive them, preventing their rejection and allowing cells from one donor to potentially treat many different people, unlike most other cells or tissues.

But there are still scientific questions to answer about MSCs. A particularly important set of questions is how the manufacturing of these cells outside of the body could affect their potential healing properties and their safety. FDA scientists believe that answering these questions will improve the way MSCs are characterized and thereby facilitate the development of products made from MSCs. For this reason, the FDA’s Center for Biologics Evaluation and Research assembled seven of its laboratories into a consortium to develop tests and techniques that will help answer these types of questions as these products move through the development process.

Using bone-marrow-derived MSCs from eight different human donors, the consortium has published scientific articles on the following topics:

  • Evaluation of the ability of human MSCs to suppress activation of certain types of mouse immune cells in order to reduce variation in MSC immune suppression assays that use T-cells from human donors who might have many different T-cells. The mouse cells come from a genetically modified strain in which all of the mouse immune T-cells are identical.
  • Creation of a large database of MSC proteins (a total of 7753) that enabled us to demonstrate the large variability among proteins from different MSC samples. This database will enhance our understanding of MSC biology and help define the variability among various MSC samples.
  • Identification of 84 proteins (14 identified for the first time) on the surface of MSCs that may be useful for tracking these cells as they grow, divide, and differentiate to produce specific tissues.
  • Development of techniques that enable scientists to quantify the ability of MSCs to multiply and to differentiate into specific cell types.
  • Identification of specific genes that distinguish aging MSCs grown in cell culture, which could facilitate development of tests that evaluate the quality of MSCs before they are used to treat patients.

These contributions are part of the overall effort of FDA to bring safe and effective stem cell-based therapies to the many patients who could potentially benefit from this type of regenerative medicine.

Steve Bauer, Ph.D., is the chief of the Cellular and Tissues Therapy Branch, Division of Cellular and Gene Therapies, in the Office of Cellular, Tissue and Gene Therapy at FDA’s Center for Biologics Evaluation and Research.

FDA Researchers Build Partnerships to Advance Innovations

By: David G. White, Ph.D.

Last week, FDA scientists and researchers presented more than 160 abstracts at the 4th Annual Food and Drug Administration Foods and Veterinary Medicine Science and Research Conference:  that’s more than 160 research projects focused on protecting the health of people and animals. The presentations and posters at the conference were shared among approximately 300 FDA researchers and other staff members who came to hear the latest on our science and research accomplishments.

David White and Heather Tate discuss poster

Heather Tate, author of “NARMS investigation of an increase in Salmonella serotype IIIa 18:z4,z23:- isolated from retail meats and humans,” discussing her poster with David G. White, Ph.D., Chief Science Officer and Research Director, FDA Office of Foods and Veterinary Medicine, at the 4th Annual FDA Foods and Veterinary Medicine Science and Research Conference.

FDA research in the food and veterinary medicine arena covers many different fields of study, from foodborne pathogens to nanotechnology, food allergens, dietary supplements and much more. For example, research is being conducted to improve detection methods for numerous microbial pathogens and chemical hazards that may contaminate the foods you and your pets eat. The diverse research portfolio of this conference showcased all the advancements in science and technology that the FDA is investing in to protect the health of people and animals.

The research presented was the highlight of the conference, but we are making equally important advancements as an organization. We have come very far in terms of our communication and collaboration among foods, cosmetics, and animal health researchers across different components of the FDA. There are so many parts of FDA involved in these areas of research that our top priority is to be sure we are working together and using our resources strategically. We must make sure our projects are more than just interesting – they must be focused on our highest public health priorities.

One of the major themes of the conference was that partnerships are critical to fostering innovation. This was emphasized by Deputy Commissioner for Foods and Veterinary Medicine Mike Taylor, who noted in his opening remarks the terrific effort of everyone who worked on the Whole Genome Sequencing project – a major undertaking that was recently a finalist and a Secretary’s Pick for the Department of Health and Human Services (HHS) Innovates award.

FDA Science and Research Conference

Tammy Barnaba, author of “Surveillance of Probiotic Ingredients in Dietary Supplements and Microbial Variations Between Product Lots,” explaining data from her poster to Laurenda Carter, another attendee, at the 4th Annual FDA Foods and Veterinary Medicine Science and Research Conference.

This project was launched to showcase the capacity of this technology to revolutionize foodborne disease tracking, and it was a true collaboration among many laboratories within FDA (Center for Food Safety and Applied Nutrition and Office of Regulatory Affairs), the Centers for Disease Control and Prevention (CDC), the National Institutes of Health (NIH) and the U.S. Department of Agriculture’s Food Safety and Inspection Service (USDA-FSIS).

One of the goals of our Whole Genome Sequencing initiative is to further develop and roll out a pathogen detection network called the GenomeTrakr, which would store genomic data of common foodborne pathogens such as Salmonella and Listeria. This data would enable FDA scientists to determine the exact order of the molecules in an organism’s genetic material, information which can then be used to identify specific strains of bacteria or viruses in foods that are causing illness. Once the strains are identified, scientists from FDA, CDC, USDA and the various states can quickly and efficiently trace the strain back to the origin of contamination so that we can improve the safety of our food supply and protect people from becoming ill.

As Dr. Eric Brown, the director of FDA’s Center for Food Safety and Applied Nutrition (CFSAN) Division of Microbiology in the Office of Regulatory Science, explains: “What genome sequencing allows us to do with food traceback is unprecedented. It’s like upgrading from an old backyard telescope to the Hubble.”

The projects presented at this year’s conference highlight the progress we have made, and the progress we want to continue to make, to expand our partnerships beyond FDA and our sister agencies, such as CDC and USDA, into academia and the private sector.

It’s exciting to see the headway we are making and the commitment of our researchers to protect and promote the health of humans and animals.

David G. White, Ph.D., is Chief Science Officer and Research Director, FDA Office of Foods and Veterinary Medicine

Curbing Risk, Not Medical Innovation, in Personalized Medicine

By: Jeffrey Shuren, M.D., J.D.

Innovative new tests are routinely submitted to the Food and Drug Administration to assure they are safe and effective. They include genetic tests that help oncologists decide whether a patient is a good candidate for a drug that treats melanoma as well as tests that are capable of sequencing the entire human genome.

Jeffrey ShurenBut many tests never undergo FDA premarket review to determine whether they are accurate, reliable, and clinically meaningful. These are laboratory developed tests (LDTs) designed, manufactured and intended to be used in a single laboratory.

FDA has exercised enforcement discretion over LDTs since 1976, when the agency first obtained comprehensive authority to regulate all in vitro diagnostics as medical devices. In those early days, LDTs were relatively simple, low risk, often for rare conditions, and generally only available on a limited basis.

But LDTs have evolved and proliferated because of advances in technology and evolving business models. Today, many LDTs are more complex, have a nationwide reach and have higher-risk uses such as detection of risk for breast cancer and Alzheimer’s disease. And yet they don’t undergo premarket review – or have adequate controls in place to assure proper test design and development, even when they compete with FDA-approved IVD test kits that conventional manufacturers market.

That’s concerning. Without appropriate safeguards, neither patients nor their health care providers can be assured that these tests are safe and effective. This is particularly troubling when an FDA-approved test is available, because it puts patients at unnecessary and avoidable risk. It also stifles innovation by creating disincentives for conventional manufacturers to invest in developing new, medically important tests.

We believe that LDTs serve an important role in health care and that there are many good tests on the market. Unfortunately, FDA is also aware of faulty or unproven LDTs, including ones that could cause patients to be inappropriately treated for heart disease; cancer patients to be exposed to inappropriate therapies or not get effective therapies; incorrect diagnosis of autism; and unnecessary antibiotic treatments.

That’s why FDA intends to propose a risk-based oversight framework that would appropriately balance assuring that patients and providers receive safe and effective tests with promoting innovation.

It would phase in enforcement of premarket review, quality systems, and adverse event reporting requirements for high- and moderate-risk LDTs over many years, beginning with the highest-risk tests (which include companion diagnostics—crucial to personalized medicine by targeting treatments for cancer, heart disease and other conditions) to give laboratories time to comply. Moreover, we intend to leverage existing programs, such as third party review and third party inspection as appropriate, and explore opportunities to work with entities that have experience with labs, thereby creating more efficiencies for labs to meet applicable FDA requirements.

On the other hand, under our upcoming proposed framework, we intend to continue exercising enforcement discretion with respect to the premarket review requirements for tests that labs make for rare diseases, to address an unmet need, or that are low risk.

Labs and conventional manufacturers serve as vitally important sources of innovative test development. Through smart, appropriately tailored oversight, we can best promote product development by all test developers and best serve patients and their healthcare providers.

When everyone plays by the same rules, innovation and society benefit.

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

Achieving our Mission through Enhanced IT Service Delivery

By: Walter S. Harris, M.B.A, P.M.P.

At its core, FDA is an information- and process-driven organization. Day-in and day-out, FDA’s experts make thousands of weighty and complex decisions by evaluating, and allowing access to, life-sustaining, life-enhancing and life-saving products. This is done using a vast amount of sophisticated and reliable data. And it is done while continuously engaging with consumers, patient representatives, industry, academia and other government agencies.

Walter HarrisSince the establishment of the Office of Information Management and Technology (OIMT) seven months ago, we have fundamentally changed how we support the Agency’s mission — primarily, to increase transparency, and better align functions and resources to achieve more efficient and improved customer support and services. To further these objectives, we have taken the following steps to help transform our service to our internal and external stakeholders.

  • Reorganized the Office of Information Management into a more stable structure that is focused on our customers and the delivery of services. This new IT structure includes robust leadership, increased scientific capability and closer attention to IT’s business and customer needs, including a new IT audit and compliance program.
  • Hired the first Chief Health Informatics Officer (CHIO), Taha Kass-Hout, MD, M.S., to promote and develop innovative enterprise solutions and identify opportunities for transparency and availability of FDA’s public health data to our consumers while ensuring accountability and privacy. With the launch of openFDA, we have demonstrated our ability to respond quickly and accurately to emerging scientific, technological and economic trends.
  • Requested that the CIO Council, FDA’s IT governance board with representation across all of its Centers, focus on opportunities to consolidate IT solutions into capabilities that benefit the agency, eliminating duplication of efforts and creating possibilities for reinvestment.
  • Creating an IT service cost-allocation model that will include a service catalog and identification of cost drivers for IT services.
  • Restructuring our IT portfolio to a service based portfolio model that is in alignment with our cost allocation model.

OIMT, together with IT leaders in the Centers, will transform our IT operation to minimize redundancies, streamline IT, and enhance customer service while lowering IT costs to the agency. We continue to seek opportunities to  identify and tackle issues, improve communications across functional lines, and more fully capitalize on the expertise of our talented staff.

These are exciting endeavors and I am proud of the efforts IT leaders across the FDA have taken to focus on customer service. With a renewed emphasis on service delivery to enable mission outcomes, we are better able to use resources in a manner that will achieve greater efficiency, improve support across the FDA, and provide results that benefit the public health.

Walter S. Harris, M.B.A, P.M.P., is FDA’s Deputy Commissioner for Operations