FDA’s New Roadmap for Progress: Strategic Priorities 2014-2018

By: Margaret A. Hamburg, M.D

The U.S. Food and Drug Administration regulates products that represent about 20 cents of every dollar American consumers spend on products. This includes the safety and effectiveness of drugs, medical devices, and vaccines, the safety of blood supply to food supply, cosmetics, dietary supplements, products that emit radiation, and more recently, tobacco. This fact can be easy to gloss over, but if one pauses for a moment to reflect on this fact, it is clear that the FDA’s regulatory role is large and truly meaningful to all of our everyday lives.

Margaret Hamburg, M.D.When the FDA was first established, our regulated industries were predominantly local, the volume of imported products was low, and even the movement of goods across country was minimal. But times have changed, and so have the strategies we employ to address those changes. Over the last five years alone, the FDA’s regulatory portfolio has increased to now include regulating tobacco products, developing a new global system for protecting food safety, and addressing challenges created by the global expansion of research, commerce and trade.

In fact, more often than not today, a drug or medical product that ends up on the shelves of an American drugstore or in our hospitals will come, at least in part, from some foreign source. Nearly 40 percent of finished medicines that Americans now take are made elsewhere, as are about 50 percent of all medical devices. Approximately 80 percent of the manufacturers of active pharmaceutical ingredients used in the United States are located outside our borders.

These and other new challenges and transformative developments in global science, technology and trade are rapidly altering the environment in which we work to fulfill our broad public health mission. In order to continue to carry out that mission, we need a set of clearly defined priorities and goals, as well as the strategies for reaching them. Therefore, I am pleased to announce the release of a revised set of FDA Strategic Priorities which will guide the agency in how we continue to promote and protect the health of the American public.

The new Strategic Priorities document sets the path for our Agency over the next four years. It establishes a framework for integrating our five strategic priorities – regulatory science, globalization, safety and quality, smart regulation, and stewardship.

Although each priority is significant in and of itself, the priorities are also interconnected and must not be addressed in isolation. In addition, this new roadmap sets forth FDA’s core mission goals and objectives, such as improving and safeguarding access to the products FDA regulates – and promoting better informed decisions about their use.

The Strategic Plan has been in development for more than a year and was created by a hard-working team of talented and knowledgeable FDA employees representing programs from across the agency. While this team drove the Plan’s creation, it is backed by the commitment of all of us at the FDA. My hope is that these priorities, which will be repeatedly cited in our speeches, policies and writings, will serve as our foundational guidepost, providing the strategic direction to help the agency continue to provide the level of service and protection the American people deserve.

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

Reflecting on New Food Protections in the Wake of PCA Convictions

By: Howard Sklamberg and Michael R. Taylor

Last Friday, Sept. 19, 2014, a federal jury in Georgia returned guilty verdicts against two former officials of and one broker for the Peanut Corporation of America (PCA) in connection with practices that led to a 46-state outbreak of Salmonella poisoning in 2009. While there were more than 700 reported cases, including nine deaths, epidemiological projections by the Centers for Disease Control and Prevention put the total number of illnesses at more than 22,000.

Howard Sklamberg

Howard Sklamberg

The convictions arise from the unlawful sale of Salmonella-tainted peanuts and peanut products. According to the evidence, the trio participated in a scheme to fabricate documents stating that the foods were free of disease-causing bacteria when, in fact, there had either been no testing or the testing had revealed the presence of such bacteria.

We can’t tell you this will never happen again. But we can say that five years after the original actions that led to the PCA convictions, we have stronger protections in place – with more coming soon – that will help prevent foodborne illnesses while empowering us to act swiftly against those who flaunt food-safety regulations.

The FDA Food Safety Modernization Act (FSMA), signed into law in 2011, was passed by Congress with the intent of building a safety net that would provide protections at all points along the global food-supply chain – from farm to table.

FSMA empowers FDA to facilitate the growth of a food-safety culture, working with federal and state agencies, and with farmers, food manufacturers and importers, to bring about widespread compliance with the new regulations mandated by the food safety law.

Michael R. Taylor is FDA’s Deputy Commissioner for Foods and Veterinary Medicine.

Michael R. Taylor

We are still in the midst of rulemaking designed to help keep food safety problems from happening in the first place. We can never prevent all problems and will still have to be vigilant and take action when problems do occur. But having a prevention-based system in place will go a long way to keeping food safe for American consumers. We have proposed seven rules since January 2013 to implement such a system, and last week we issued supplemental proposals to make practical and targeted changes to four of those rules related to the safety of produce, human and animal food products, and imported foods. We are working on implementation strategies well in advance of the 2015 dates on which the rules will be final so we will be ready to put these new requirements in place. We are also working on a plan for outreach and education, so that everyone involved in providing our food – including growers, food processers and importers — will know what the rules require and will have adequate time to plan.

What you may not see in the language of the proposed rules is that FDA is reinventing itself to become as much a coach as a cop. In partnership with state and federal agencies, we will help provide technical assistance and education, and we will provide guidance to help farmers and industry build a framework that protects their products from contamination and, if potential hazards are identified, enables them to act immediately to control them.

That’s not to say that we won’t use tough enforcement tools when necessary, as evidenced by the PCA prosecution. When consumers are at risk, FSMA has given FDA greater authority to take action, in addition to bringing a civil action or seeking criminal prosecution. We can issue mandatory recalls when a company fails to voluntarily recall unsafe food; we can detain products that are potentially in violation of the law to keep them from being moved; and in certain situations we can suspend the registration of a facility to prohibit it from distributing food.

The law gives us these powers. But the vast majority of farmers, manufacturers and importers are committed to providing safe food. FSMA also gives us the power to work with them in partnership to keep foods safer than ever for you and your family.

Howard Sklamberg is FDA’s Deputy Commissioner for Global Regulatory Operations and Policy

Michael R. Taylor is FDA’s Deputy Commissioner for Foods and Veterinary Medicine

Read the U.S. Department of Justice press release:

http://www.justice.gov/opa/pr/peanut-corporation-america-former-officials-and-broker-convicted-criminal-charges-related

Celebrating 30 years of easier access to cost-saving generic drugs

By: Margaret A. Hamburg, M.D.

Thirty years ago today, President Ronald Reagan signed into law the Drug Price Competition and Patent Term Restoration Act of 1984, better known today as the Hatch-Waxman Amendments. This law, championed by Senator Orrin Hatch and Representative Henry A. Waxman, made it easier for generic drugs to enter the market, and has greatly expanded access to important—often life-saving—drugs. Over the 10-year period 2003 through 2012, generic drug use is estimated to have generated more than $1.2 trillion in savings to the health care system and to have benefitted the health and well-being of innumerable lives.

Margaret Hamburg, M.D.Thanks to the insight of its creators, one of the strengths of this law is the fact that it provided financial incentives for pharmaceutical companies that develop and manufacture new and innovative trade name products. Under the law, sponsors of qualifying trade name drugs are provided an opportunity to extend a patent to make up for patent life lost during the process of testing and approval of the product.

The law also, however, provided a clear pathway to market for generic drugs. Before Hatch-Waxman, little more than a third of branded prescription drug products even had a generic available, and those that were available were not as widely used. Today, most drugs that go off patent face competition from cost-saving generic drugs. As a result, about 85 percent of all prescriptions filled are for generic versions.

Importantly, while Hatch-Waxman has provided powerful cost savings for American consumers, its value in providing greater access to medication cannot be overlooked. For over 30 years, millions of consumers who otherwise would not have been able to afford needed medication now have access to lower-cost, quality, generic drugs that are just as safe and effective as their brand-name counterparts.

Despite the enormous success of Hatch-Waxman, FDA faces challenges as we continue efforts to ensure access to affordable and quality generic drugs.

FDA is working to reduce the current backlog of generic drug applications for new generic drug products. Fortunately, the Generic Drug User Fee Amendments of 2012, GDUFA for short, provides additional funding for FDA’s generic drug program. We’re allocating significant time and money towards reducing the backlog.

As our world economy experiences greater globalization, it has becoming increasingly important for FDA to allocate its resources based on potential risk around the globe. More than 80 percent of the ingredients used to make our drugs now come from overseas suppliers. FDA is committed to working to ensure that, no matter where the ingredients are from or where the drugs are made, the American public can be assured their products are safe. GDUFA funding also helps FDA address global inspections, and we are diligently working to monitor production across the globe.

FDA salutes the vision of Senator Hatch and Representative Waxman. Their landmark legislation has improved the health of generations of Americans. And we’re proud of the role FDA has had in implementing Hatch-Waxman and helping to assure its success. We look forward to continuing to enhance Americans’ access to safe, effective, and affordable generic prescription drugs.

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

The FDA Drug Shortage Assistance Award… Recognizing manufacturers who help prevent or alleviate drug shortages

By: Douglas C. Throckmorton, M.D.

As part of our continuing commitment to prevent drug shortages and minimize their impact on public health, FDA has launched the FDA Drug Shortage Assistance Award.

Douglas C. Throckmorton, M.D.This award recognizes efforts of drug manufacturers who have cooperated with FDA and implemented strategies to help provide medically necessary drugs in short supply for patients, while maintaining federally mandated quality standards.

Recently, FDA announced the first recipients of the award: Guerbet Group and Clinigen Group plc, two companies who worked diligently with FDA to help ensure adequate supplies of important medicines for patients in need.

Guerbet Group worked with FDA to help alleviate the shortage of ethiodized oil injection, an important imaging agent for a variety of patients with certain forms of liver cancer. The company’s work included acquiring the new drug application (NDA) for Ethiodol, a form of ethiodized oil; submitting the relevant applications to restart its manufacture under the trade name Lipiodol; and gaining additional approval for a medically necessary indication that was of critical concern during the shortage. Clinigen helped ensure supplies of a medication needed for patients with AIDS who also have a serious eye condition called CMV (cytomegalovirus) retinitis. The company’s work included acquiring the NDA for Foscavir (foscarnet sodium) injection, and submitting the relevant applications to return the product to market.

The FDA Drug Shortage Assistance Award is given to drug manufacturers who, among other factors, have demonstrated a strong commitment to preventing or alleviating a shortage of a medically necessary drug, by:

  • Taking one or more actions to alleviate or prevent a drug shortage, such as increasing production or submitting an application for approval of a drug in shortage;
  • Making a significant impact on public health; and
  • Using a facility that was substantially compliant with current good manufacturing practice (CGMP) for at least one inspection prior to the intervention and during the time the candidate used it to manufacture the shortage drug.

FDA plans to continue to recognize manufacturers with this award based on their ability to meet the criteria.

FDA is committed to preventing and reducing the impact of drug shortages. The FDA Drug Shortage Assistance Award is one of the tools outlined in our Strategic Plan for Preventing and Mitigating Drug Shortages to help address ongoing drug shortages in our nation’s health care system.

On behalf of patients in need of critical medication, FDA thanks Guerbet Group and Clinigen Group plc for their commitment to help ensure access to quality medications, and we offer our sincere congratulations to these award recipients. Shining a spotlight on the efforts of drugs manufacturers who have made outstanding efforts in this area will hopefully inspire other manufacturers to follow suit.

Douglas Throckmorton, M.D., is Deputy Center Director for Regulatory Programs in FDA’s Center for Drug Evaluation and Research

Three encouraging steps towards new antibiotics

By: Janet Woodcock, M.D.

You may have been hearing about a variety of Federal Government actions to address the growing need for new antibiotics. For instance, in an FDA Voice blog last week Commissioner Hamburg discussed the President’s national strategy for Combating Antibiotic Resistant Bacteria (CARB) and our collaboration with a wide variety of organizations to address this issue. You may have also noticed another recent blog talking about FDA’s work on the Generating Antibiotics Incentives Now Act (GAIN Act), the Antibacterial Drug Development Task Force, a public meeting, a Federal Register Notice, and multiple guidance documents, all aimed at building up the nation’s arsenal of effective antimicrobial drugs.

Janet WoodcockThere are many government activities in this area, so I’d like to boil things down a bit. A critical fact is that our efforts are starting to show signs of success. Over the last few months, FDA has approved three new antibiotics to treat patients with acute bacterial skin and skin structure infections (ABSSSI) caused by bacteria like Staphylococcus aureus, including methicillin-resistant strains, also known as MRSA infections.

  • On May 23, FDA approved Dalvance (dalbavancin), an injectable drug, administered intravenously in two doses one week apart.
  • On June 20, FDA approved Sivextro (tedizolid phosphate), available for intravenous and oral use, administered once daily for six days.
  • On August 6, FDA approved Orbactiv (oritavancin), an injectable drug administered as a single dose to comprise a full course of therapy.

In these approvals, the drug’s manufacturer was able to take advantage of recently enacted incentives to help bring new antimicrobials to market. Each of these drugs was approved after being designated as a Qualified Infectious Disease Product (QIDP) under the GAIN Act. As part of this QIDP designation, FDA’s review of the drug application was expedited. The designation also qualified the drugs for five years of marketing exclusivity to be added to certain exclusivity already provided by the Food, Drug, and Cosmetic Act. To date, FDA has granted the QIDP designation to 39 antibiotics under development.

Development of these three new antibiotics was also helped a great deal by the scientific collaboration among stakeholders dedicated to advancing new antimicrobial therapies. The Biomarkers Consortium of the Foundation for the National Institutes of Health, academic and industry experts, and other contributors made valuable recommendations to the FDA regarding designing scientifically sound studies to show the effectiveness of these drugs in clinical trials.

We still have a long way to go in getting a leg up on building a new and more effective arsenal of antimicrobial products. And once approved, it will be critical for health care professionals to appropriately prescribe these new antibiotics. But with ongoing collaborative, concerted efforts by the many public and private stakeholders, we can continue to advance and help build a national antibacterial research and development enterprise capable of bringing new drugs to the patients who need them. These three approvals are an encouraging start!

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

Learn more by reading Dr. Woodcock’s testimony: 21st Century Cures: Examining Ways to Combat Antibiotic Resistance and Foster New Drug Development.:

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

FDA Works to Mitigate the West Africa Ebola Outbreak

By: Luciana Borio, M.D.

Luciana Borio, M.D.The world is witnessing the devastating effects of the Ebola virus outbreak in West Africa, the worst Ebola outbreak in recorded history. To date, more than two thousand people in Guinea, Liberia, Nigeria and Sierra Leone have become infected, and more than twelve hundred have died. The stories of so many lives lost, and those of so many others fighting for their lives, are heartbreaking and tragic. We at the Food and Drug Administration are dedicated to helping end this outbreak as quickly as possible. And we are working hard to accelerate the development and production of treatments and vaccines to help prevent future outbreaks like this.

The primary approaches to contain the current outbreak remain standard public health measures. However, this outbreak presents complex challenges, in part because there are no FDA-approved treatments or vaccines for the Ebola virus. FDA has an important role during situations like this.

For example, we are working closely with U.S. government agencies that support medical product development – including the National Institutes of Health, the Biomedical Advanced Research and Development Authority, and the U.S. Department of Defense (DoD) – to speed the development and production of medical products that could help mitigate outbreaks like this. And we are working interactively with medical product sponsors to clarify regulatory and data requirements in order to move investigational products forward in development as quickly as possible. We also are in close contact with the World Health Organization and several of our international regulatory counterparts to exchange information about these investigational products for Ebola treatment, and to exchange information about how FDA works to facilitate development of and access to these products.

The experimental vaccines and treatments in development are in the earliest investigational stages and have not been fully tested for safety or efficacy. Only small amounts of some experimental products have been manufactured for testing, which means few courses, if any, are available for companies to make available for compassionate use in response to this outbreak. We are working closely with our U.S. government colleagues to have experimental treatments and vaccines available for clinical evaluation in the next few months. We are hopeful that, in the future, we will have medical products approved and manufactured for wide-scale use to address the Ebola outbreak. However, these products are not at that stage yet.

In the meantime, FDA is doing all we can to alleviate the situation. FDA has one of the world’s most flexible regulatory frameworks, which includes mechanisms to enable access to available investigational medical products when, based on certain criteria such as the balance between expected risk and benefit to the patient, it would be appropriate to use such products.

For example, under certain circumstances, clinicians may request the use of an Emergency Investigational New Drug (EIND) application under the FDA’s Expanded Access program to access investigational products outside of clinical trials for their patients. And under the FDA’s Emergency Use Authorization (EUA) authority, we can allow the use of an unapproved medical product – or an unapproved use of an approved medical product – for a larger population during emergencies, when there are no adequate, approved and available alternatives.

This month, we authorized the use of an Ebola diagnostic test, developed by DoD, under an EUA to detect the Ebola virus in DoD-designated laboratories. This test can help facilitate an effective response to the ongoing outbreak in West Africa by helping to rapidly identify patients infected with Ebola virus and facilitate appropriate containment measures and clinical care.

It is an unfortunate fact that, during outbreaks like this, fraudulent products that claim to prevent, treat or cure a disease rapidly appear on the market. FDA has learned of several fraudulent products that claim to prevent or treat this Ebola virus infection, including so-called natural remedies. Consumers who have seen these fraudulent products or false claims should report them to us. For our part, we will remain vigilant for fraudulent products and false product claims related to the Ebola virus, and will take enforcement actions as warranted to protect public health.

FDA stands ready to work with companies and healthcare providers to speed product development and to facilitate access to investigational products to treat patients when appropriate. We are fully committed to helping end this outbreak as quickly as possible and to sustaining our efforts to help prevent such outbreaks in the future.

Luciana Borio, M.D., is the Assistant Commissioner for Counterterrorism Policy and Acting Deputy Chief Scientist.

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