Kicking off the PDUFA VI Reauthorization Process

By: Theresa M. Mullin, Ph.D.

The Prescription Drug User Fee Act (PDUFA) authorizes FDA to collect fees from pharmaceutical companies to help fund the agency’s drug review work. PDUFA’s intent is to provide additional funding for FDA to hire staff, improve systems, and establish a better-managed review process that enables us to do more timely reviews of human drug applications.

Theresa MullinAs a result, many important new drug therapies have been made available to patients sooner without compromising FDA’s high standards for safety, efficacy, and quality.

As part of FDA’s agreement with industry during each reauthorization of the Act, the agency agrees to certain performance goals to enhance the process of drug review. The goals, now 30 in total, apply to many review processes, including the review of original new drug applications, resubmissions, and supplemental applications. Since the first user fee law was passed in 1992, PDUFA has been reauthorized four times. The current legislation, PDUFA V, is set to expire in September 2017.

On July 15, 2015, FDA gathered stakeholder perspectives during a meeting on what features the agency should propose in the reauthorization of PDUFA for fiscal years 2018 – 2022. Attendees included patient advocates, consumer advocates, representatives of professional health care associations, biopharmaceutical industry representatives, academic researchers, policy analysts, and others. FDA received mostly positive feedback on our progress under PDUFA V and helpful input about the future for PDUFA VI.

Highlights of progress noted during this meeting include:

  • FDA continues to meet or exceed most review goals.
  • The program is experiencing high rates of approvals for novel products during their first submission. This includes a historically high number of approvals for novel products treating rare diseases (17 orphan drug approvals in 2014).
  • There are improved and increased communication functions and practices between FDA and new drug companies, or sponsors. This includes implementation of a structured risk-benefit framework within the review process.
  • The Patient-Focused Drug Development program has been successful in systematically obtaining patient perspectives on certain diseases and related treatments.

Meeting participants also contributed a number of ideas for further enhancements, including:

  • Further efforts to involve the patient perspective in drug development processes;
  • Building on FDA’s Sentinel System for active surveillance of safety issues for medical products, including expanding its use as a source of data; and
  • Enhancing regulatory science initiatives, including the use of patient-reported outcomes and biomarkers.

More detailed information about the meeting is available at PDUFA Meetings, which includes a webcast of the one-day meeting, the agenda, access to the docket for online public comments, and (soon to follow) a complete written transcript of the public meeting’s proceedings.

PDUFA was designed to enable FDA to fulfill its mission to protect and promote public health by helping to more effectively bring to market critical new medicines for patients. FDA is strongly committed to the components, enhancements, and initiatives that constitute this program and have made it so successful. The public feedback received during the July 2015 meeting indicates that we are on the right track in achieving our goals under PDUFA V, and FDA looks forward to PDUFA VI.

Theresa M. Mullin, Ph.D., is Director of FDA’s Office of Strategic Programs in the Center for Drug Evaluation and Research

Frances Oldham Kelsey, Ph.D., M.D.: A Pioneer in Public Health and Protection of Patients

By: Stephen M. Ostroff, M.D.

Acting FDA Commissioner, Stephen Ostroff, M.D.

Stephen M. Ostroff, M.D., is Acting Commissioner of the Food and Drug Administration

Last week our nation lost a true pioneer in public health and consumer protection.

Frances Oldham Kelsey, Ph.D., M.D., who joined FDA in 1960 as a medical officer, was known worldwide as a leader in drug safety and the protection of patients. She established that reputation in one of first FDA assignments: reviewing the marketing application for a drug called thalidomide, which was already available in dozens of countries around the world.

Despite constant pressure from the company, Dr. Kelsey refused to approve thalidomide because of inadequate evidence about its safety. As a result of Dr. Kelsey’s expertise, diligence, and integrity, the drug was never approved in the United States and Americans were largely spared the tragic birth defects and deaths experienced by patients in those countries where thalidomide was available.

This “near miss” spurred Congress and the White House to revive pending proposals to revitalize the oversight and regulation of pharmaceutical products. In the wake of the thalidomide episode, the Kefauver-Harris Drug Amendments became law in 1962. That law mandates “substantial evidence” of a drug’s effectiveness (in addition to evidence of safety that was previously required) and today continues to provide this road map for how unapproved pharmaceutical products are tested in humans.

Dr. Kelsey

Frances Oldham Kelsey, Ph.D., M.D.

It is fair to state that these amendments, as implemented by FDA, ushered in the modern era of science-based proof that the medicines we use are both safe and effective, a level of evidence that created a standard still in effect today.

Dr. Kelsey’s original work on the thalidomide application stands today as a legendary example of how FDA carries out its public health mission: judicious exercise of authority and oversight to protect consumers and patients.

Dr. Kelsey joined FDA during a different era and was a trailblazer in many ways, including the role of women in science. And yet, although this is a new era, our mission endures: to promote innovation while protecting the health and welfare of Americans. And FDA continues to be defined by the same rigor, dedication, and integrity that informed Dr. Kelsey’s work.

Our nation owes a great debt of gratitude to Dr. Frances Oldham Kelsey for her decades of service to public health.

Stephen M. Ostroff, M.D., is Acting Commissioner of the Food and Drug Administration

More information about Dr. Kelsey’s life and career is available in her “Autobiographical Reflections,” which FDA released on the occasion of her 100th birthday in 2014.

Need a Guidance Document? We’ve Got You Covered

By: Chris Mulieri, PMP

We all understand the frustration of searching online for something and not finding it. The Food and Drug Administration recently helped end this problem by making it faster and easier to find our guidance documents – some of the most requested items on our website.

Chris MulieriGuidance documents represent FDA’s current thinking on a particular subject. Currently, there are about 3,100 of them – and the list is growing.

FDA’s Web & Digital Media team and the Office of Information Management and Technology have created a dynamic search list on one site so you can go to just one page and find the guidance documents you need, no matter where they are on This search tool is powerful and easy to use. Now you can go to just one search box to find what you need in moments, instead of the 10 different pages on FDA’s website where guidance documents are posted.

It doesn’t matter if it’s a guidance document on devices, drugs, biologics, tobacco, veterinary medicine, or foods – it’s all there.

We did this as part of FDA’s Transparency Initiative and in response to the feedback we got from our stakeholders via the American Customer Service Index (ACSI) online survey. They told us just how hard and time-consuming it was for them to find these important documents. So we decided to do something about it.

It’s not practical for us to put these documents all in one place. So, we assembled a working group with representatives from each of FDA’s Centers (which post the guidance documents on their own sites) and developed the search criteria.

In addition, we tagged the documents with metadata (search terms) needed to make search and filtering functions work as intended. Now, the list automatically populates as you enter search terms and filters. Each column is sortable.

You can narrow your search by filtering on different categories, including product, date issued, FDA organization, document type, and subject. Refine your search by draft guidance, final guidance, whether it’s open for comment, or by comment closing date.

How are we doing?

Since the launch of the guidance search in December 2014, page views have increased from about 22,000 to more than 136,000 for the first quarter of 2015. For the first time, the page is among the top visited on FDA’s website. And we’ve seen improved user satisfaction, reflected in the feedback in the ACSI responses.

We hope you’ll try the new guidance document search page soon and let us know what you think.

Chris Mulieri, PMP, is FDA’s Director, Web & Digital Media, Office of External Affairs.

What’s New in Health Disparities?

By: Jovonni Spinner, MPH, CHES

In June 2015, I presented at the Health Disparities, Education, Awareness, Research, and Training (HDEART) workshop at Prairie View A&M University, near Houston. This annual workshop brought together nationally recognized leaders to discuss genomics, communications, bioethics, and other minority health issues, as well as disease-specific health programs, such as cancer, maternal health, and smoking cessation.

Jovonni SpinnerWe know health disparities exist and minorities fare worse for many health outcomes. That is old news. The workshop promoted an open discussion and offered fresh ideas on bio-psychosocial approaches to address health disparities that will improve health equity.

The FDA’s George Strait moderated my panel, “Health Inequities, Health Communication, and the Media.” I spoke with three other public health experts and researchers about how to use communication strategies and collaborative models to reduce health disparities.

We focused on implicit and explicit bias among physicians, developing and implementing public health programs, and building a diverse health care workforce. We also discussed how changes in the private-practice model affect African-American physicians and their efforts to reduce health disparities.

I specifically talked about how the FDA Office of Minority Health (OMH) is building a robust outreach and communications program. OMH partners with minority-serving institutions to better engage minority groups, raise awareness around specific diseases, and develop linguistically and culturally appropriate health educational materials.

The HDEART panel was an excellent platform to raise the visibility of FDA’s role in improving minority health because the audience was filled with health care practitioners, researchers, and social workers who are engaged in these issues and did not know about us.

Here are some salient action items that emerged from the workshop:

  • Support and increase funding for health disparities research;
  • Implement strategies to remove communication and structural barriers;
  • Improve literacy skills by investing in early childhood education;
  • Recognize that multiple factors influence health equity and access to health care, including individual health behaviors, and social and environmental factors;
  • Scale up innovative public health programs that have a positive effect on health outcomes in minority communities; and
  • Find creative ways to reach the underserved; for example, use telemedicine to reach vulnerable and rural populations who do not have medical providers easily accessible.

During the lectures, I thought about how to apply this newfound knowledge to the work we do in OMH. Two areas came to mind: we can work to remove communication barriers and we can support health disparities research.

Moving forward, we can come up with strategies to:

  • Build and strengthen our partnerships to reach a wider audience;
  • Support our extramural and intramural research programs and facilitate scaling up successful projects; and
  • Use innovative communication strategies to reach our audience.

We live in a global society where disease knows no borders. It is our job as a public health agency to employ a holistic approach to improving health equity. Diverse populations are not one dimensional, so one-dimensional solutions will not be enough. We need to identify factors that influence health and tackle the problem from all angles. Only then can we make progress in closing the disparity gap and improve health equity for all!

More information about FDA’s OMH can be found here:

Follow us on Twitter @FDAOMH

More information about the HDEART Workshop can be found here:

Jovonni Spinner, M.P.H., C.H.E.S., is a Public Health Advisor in FDA’s Office of Minority Health

‘Quality Metrics’: FDA’s plan for a key set of measurements to help ensure manufacturers are producing quality medications

By: Ashley Boam, MSBE and Mary Malarkey

Yesterday, we took an important step in advancing the quality of medications with the release of draft guidance for the pharmaceutical industry called, “Request for Quality Metrics.”

Ashley Boam

Ashley Boam, FDA’s acting Director, Office of Policy for Pharmaceutical Quality, Office of Pharmaceutical Quality, Center for Drug Evaluation and Research

In these technical terms, that may not sound like much. But – in plain language – this document describes a set of measurements to help the agency evaluate the quality of the facilities and the processes that manufacturers use to make FDA-regulated drugs and biologics. These include prescription drugs and certain biological products. The guidance also encourages these manufacturers to conduct robust quality measurements on their own products.

It’s critically important for patients, health care professionals, caregivers, payers, and others to have confidence in how medications are made. “Quality metrics,” or the measures used to assess the quality of drug and biologic manufacturing, can help us achieve this goal.

We expect that these measurements will strengthen our efforts to ensure that FDA-regulated medications are not only demonstrated to be safe and effective, but also continually manufactured under strict quality standards.

We believe a careful analysis of quality metrics can help FDA better identify which facilities are at the highest risk for quality problems. This will help us use our inspection resources most efficiently and effectively.

Mary Malarkey

Mary Malarkey, FDA’s Director, Office of Compliance and Biologics Quality, Center for Biologics Evaluation and Research

Quality is also directly connected to a consistent supply of needed medications. Over the years, there have been disruptions in the availability of some drug and biological products due to manufacturing production flaws. We believe that a company’s own robust quality measurement system, along with our quality measurements, can help manufacturers better identify factors that may predict manufacturing problems – and move us a step closer toward reducing and controlling these disruptions.

FDA has been working for many years on solutions to encourage and support the modernization of pharmaceutical manufacturing, such as the use of risk-based regulatory strategies for oversight. Our quality metrics initiative is one of several approaches we believe will further support this effort.

We encourage patients, prescribers, industry and others to submit comments regarding our Quality Metrics draft guidance. We’ll also be hosting a public meeting on August 24, 2015. We’ll use this input to help create a final guidance to support the reporting and calculation of quality measurements.

Yesterday’s draft guidance is an important step on a shared path toward improved drug quality throughout the pharmaceutical industry. We look forward to receiving comments, finalizing the guidance, and receiving the first set of reports.

In the meantime, we’ll be working with others to support industry’s use of robust quality metrics programs and to understand the best way to use quality metrics to improve manufacturing quality and FDA’s regulatory decision making.

We also will continue to emphasize the importance of quality in the pharmaceutical industry for companies that make medications and for the patients who receive them.

Ashley Boam is FDA’s acting Director, Office of Policy for Pharmaceutical Quality, Office of Pharmaceutical Quality, Center for Drug Evaluation and Research

Mary Malarkey is FDA’s Director, Office of Compliance and Biologics Quality, Center for Biologics Evaluation and Research

FDA Science Forum 2015: Views of FDA

FDA’s 2015 Science Forum attracted more than 800 people from the scientific community. Here’s what some attendees said about the innovative research going on at the agency and why FDA can be a valuable collaborator in research aimed at transforming food safety and medical product development. If you couldn’t attend the FDA science forum, you can still see all the presentations on our web site.

More Collaboration, Research Needed to Develop Cures

By: Robert Califf, M.D.

The U.S. Food and Drug Administration’s drug approval process—the final stage of drug development—is the fastest in the world, which means Americans typically have first access to new drugs when they are demonstrated to be safe and effective. But even as our agency has transformed the approval process—approving 51 new molecular entities and biological products last year alone, including more new orphan drugs for rare diseases than in any previous year—drug discovery and development is not keeping pace for many diseases.

Robert CaliffIn many cases, what’s holding back progress is a lack of understanding of the biology of disease, as we outline in a new report we are releasing today that compares diseases where there is a robust pipeline of new therapies with certain diseases that have few known treatments or cures.

For instance, when it comes to cancer, HIV/AIDS, and other viral infections, decades of intense research have given the scientific community and the FDA critical insight on how to develop effective treatments. Ongoing research has led to the discovery of biomarkers, which are characteristics that are objectively measured and evaluated as indicators of normal biological processes, pathogenic processes or response to a therapeutic intervention. Some types of biomarkers give insight on the genetic and metabolic characteristics that alter patients’ responsiveness to particular drugs, and others give insight into whether drugs in development are likely to work. This deep knowledge has resulted in important breakthroughs, rapid drug development and speedy FDA approvals.

While additional research is needed for all diseases, the paucity of reliable biomarkers in some diseases highlights the critical need for more research if we are to make much needed progress. Examples include Alzheimer’s and many rare diseases, as we outline in the new report released today. In these cases, the scientific community still lacks basic information about what causes these diseases and how they can be slowed and treated. When research does not offer answers to important scientific questions, cures cannot be developed. And when viable cures are not in the pipeline, focusing on regulation will not improve the situation, since FDA can only approve therapies with evidence for safety and effectiveness.

Once key scientific questions are answered, we can use a variety of tools to reduce the length and cost of initial clinical trials for drug approval for these disease areas, and we can provide guidance to industry including advice on how to develop additional reliable biomarkers. For instance, we’ve improved the efficiency and predictability of clinical drug development by developing tools such as biomarkers and surrogate endpoints—markers of drug effect that do not directly represent an improvement in how a patient feels or functions, but are reasonably likely to predict a clinical benefit. Thus, for example, lowering a patient’s blood pressure can be used as a surrogate for the clinical benefit of preventing heart attack. Such tools have modernized clinical trial designs and may dramatically reduce the length and cost of drug development. They also can help target drugs to specific patients who can benefit most, thereby limiting the number and size of clinical trials.

These are exciting times as we experience simultaneous revolutions in the biological and information sciences. We expect that the astounding increase in knowledge of biological systems enabled by whole genome sequencing, cloud computing, social media, and wearable devices to monitor physiology will create challenges to traditional thinking. And we are confident that this increased knowledge will continue to expand the pipeline of new therapies. This report emphasizes that we are prepared to deal with the product of this scientific investment by using regulatory paradigms that match the state of the science and by supporting dissemination of the latest knowledge applied to drug development.

In this paradigm that takes advantage of the depth of this new biomedical information, it will be critical to continue to support ongoing clinical trials and observational studies to ensure sufficient knowledge of the benefit-risk profile of therapies as they evolve into broad use. Even the best of the current surrogates such as systolic blood pressure cannot substitute for the entire cumulative effects of a drug on the intended biological target and for off-target effects.

We will continue to work to speed patient access to therapies shown to be safe and effective through our existing programs that allow for expedited review, development, and approval of certain medical products. To encourage innovation, we also will continue to work with other government agencies and the healthcare community, including members of patient groups, academia, and industry. It will take a collaborative effort to improve our nation’s understanding of certain diseases and to translate any resulting scientific discoveries into cures.

Robert Califf, M.D., is FDA’s Deputy Commissioner for Medical Products and Tobacco.

More information can be found at: Innovation at FDA.

Celebrating the 3rd Anniversary of the FDA Safety and Innovation Act

By: Stephen M. Ostroff, M.D.

Anniversaries are celebrated for many different reasons. Sometimes it is to recognize the enduring strength of an institution. Other times it offers an opportunity to gauge success or progress.

Acting FDA Commissioner, Stephen Ostroff, M.D.One commemoration that falls into the latter category is today’s third anniversary of the signing of the landmark Food and Drug Administration Safety and Innovation Act or, as it is known in the world of Washington acronyms, FDASIA.

FDASIA gave FDA authority to collect user fees from industry over five years, beginning in 2012, to fund reviews of innovator drugs, medical devices, generic drugs, and biosimilar biological products.

It also promotes innovation to speed patient access to safe and effective products, increases stakeholder involvement in FDA processes, and enhances the safety of the drug supply chain. Just as important, FDASIA improves the agency’s ability to help prevent drug shortages.

FDA has made great strides to implement this important law since President Obama signed it, issuing more than 35 draft and final guidances, more than 10 proposed and final rules, three strategic plans, 14 reports to Congress, 18 public reports, and 13 public meetings designed to solicit input from a vast assortment of stakeholders.

All told, we have completed more than 70% of the law’s deliverables and we continue to maintain our commitment to a transparent and accessible implementation plan that allows the public to follow our progress.

Our work on additional action items continues.

Just two days ago we completed another task – issuing a final rule that requires all manufacturers of certain medically important drug and biologic products to give FDA early notification of potential drug shortages and to report the reasons for that potential shortage.

This step is the latest in a series of changes FDA has made to significantly reduce drug shortages. Those efforts have helped to prevent 282 shortages in 2012, 170 in 2013, and 101 in 2014.

This progress is but one example of how FDA’s work under FDASIA is making an important difference for patients and health care professionals who depend on these products.

One of the most significant provisions of FDASIA was the creation of a new Breakthrough Therapy designation for drugs and biologics intended for serious or life-threatening illnesses where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies.

As of last month, 315 requests for this special designation have been received and 93 drugs and biologics have been granted breakthrough status. Expedited development is underway for the majority of these breakthrough designated products, while 26 breakthrough therapy drug/indication combinations have already been approved and are now on the market for use by patients. This program, which, along with fast track, accelerated approval, and priority review, was the topic of FDA’s final guidance on our expedited review programs, also has helped facilitate earlier and continuing consultation and advice by FDA for industry researchers and product developers.

In large part, as a result of these expedited programs, we saw the approval of a record number of new drugs in 2014 for the treatment of both rare diseases and more common conditions like various forms of cancer and hepatitis C. We also saw the approval of a record number of biologics, including new vaccines for meningococcus type B.

Innovation is being promoted under FDASIA through greater patient engagement, including a five-year Patient Focused Drug Development program to learn from patients about the impact of their disease on their daily lives. Since its creation, we have held 14 meetings with patients on subjects such as chronic fatigue syndrome, lung cancer, HIV, and narcolepsy.

As this strategy makes clear, knowledge and understanding of a patient’s perspective on disease are critical. But equally significant is the importance of ensuring adequate data quality and transparency in research to develop new treatments. That brings up another area of great progress under FDASIA: addressing the longstanding concern about representation of women and minorities in clinical trials that support marketing applications for medical products.

In 2014, in response to Congress’s request in Section 907 of FDASIA, we produced an Action Plan to help close gaps in data quality, clinical trial participation, and data access. We have issued a guidance document on the “Evaluation of Sex-Specific Data in Medical Device Clinical Studies,” and we’re working to promote clinical trial participation by women and minorities. We also are posting on our website easy-to-understand Drug Trials Snapshots which provide the breakdown of clinical trial participants by age, race, and sex for newly-approved drugs and biologics. Snapshots also summarize whether there were differences in efficacy and safety among different subgroups.

Part of our efforts to implement and achieve the goals of FDASIA is helping us address the enormous global changes affecting FDA’s responsibilities.

With roughly 40 percent of finished drugs coming from outside our borders, and 80 percent of active ingredient manufacturers being located outside of the U.S., protecting the U.S. drug supply chain and making sure that patients have access to the drugs they need is a continuing priority for FDA.

FDASIA includes a set of provisions, contained in Title VII of the statute, which gave FDA new authorities to address the challenges posed by an increasingly global drug supply chain.

Given the enormity of FDA’s responsibilities, including the many new responsibilities authorized by Congress, combined with the budgetary challenges we face in this time of fiscal limitations, user fee funds play a critical role in FDA’s continued progress and excellence, including providing critical support to our staff of experts and helping maintain the high quality of their work.

Looking ahead, we have begun to plan for the next reauthorization of our user fee programs, beginning with a series of stakeholder meetings that began last month.

And, some of the themes advanced in FDASIA – encouraging antibiotic drug development, patient engagement, and the importance of biomarkers – are being considered by Congress as part of the 21st Century Cures initiative now making its way through Congress.

FDASIA provided enormous new responsibilities but also presented many promising opportunities. As we continue our progress in implementing this landmark law, we anticipate that we will continue to meet – and even exceed – the goals of the law as we strive to fulfill our mission to protect and promote the health of the American public.

Stephen M. Ostroff, M.D., is Acting Commissioner of the Food and Drug Administration

FDA China Office’s Lixia Wang Wins Award for HHS Locally-Employed Staff

By: Mary Lou Valdez

The FDA’s mission to ensure that food is safe and medical products are effective for use by U.S. consumers and patients is widely recognized.

Lou Valdez

Mary Lou Valdez, FDA’s Associate Commissioner for International Programs

A lesser-known fact is that the U.S. Department of Health and Human Services (HHS) employs more than 1,700 locally-employed (LE) staff in 60 countries to uphold its mission across five operating divisions, including FDA. Dr. Lixia Wang, a locally-employed staff member working for FDA in China, exemplifies the contributions of our LE staff and is the recipient of the annual HHS LE Staff of the Year Award.

China is a key supplier of goods imported to the U.S. The FDA has made significant regulatory in-roads since establishing its China post in 2008 and in many ways these are due to the contributions made by Dr. Wang and other LE staff in the world’s most populous country.

For example, Dr. Wang was essential in the negotiations of bilateral agreements for the placement of additional staff in China. With Dr. Wang’s contributions, FDA finalized these important agreements, which pave the way for FDA to more than triple its staff size in China. This move will bolster FDA’s work to protect and promote the health of consumers and patients in the U.S. and around the world.

Dr. Lixia Wang

Dr. Lixia Wang in Beijing

Dr. Wang, who has served as Medical Research Scientist for FDA’s China Office since 2009, was cited for her central role in negotiations concerning the Implementing Arrangements with China’s Food and Drug Administration, and Implementing Arrangement Between the Food and Drug Administration and the General Administration of Quality Supervision, Inspection and Quarantine.

In addition, Dr. Wang has made significant contributions to the HHS mission in China since joining U.S. Embassy Beijing in 2006. From 2006 to 2009, she served as senior local staff in the Office of the HHS Health Attaché, and played a key role in strengthening U.S.-China collaboration on infectious disease.

During that time, she also supported the FDA response to emerging problems associated with melamine in dairy and pet products, and worked to address contaminated blood thinner sourced from China. She played a key role in the 2007 negotiations of binding agreements with the Chinese Government on the safety of FDA-regulated products, and on the opening of FDA’s first-ever overseas office in 2008.

FDA congratulates Dr. Wang, and takes pride in the recognition for excellence and commitment to global public health that she brings to FDA with this award.

Mary Lou Valdez is FDA’s Associate Commissioner for International Programs

A Global Fight Against Dangerous Counterfeit and Unapproved Medical Products: From Operation Pangea to FDA’s Global Strategic Framework

By: Howard Sklamberg, J.D., George Karavetsos, J.D., and Cynthia Schnedar, J.D.

Unfolding earlier this month was a global cooperative effort, which included the Food and Drug Administration, to combat the online sale and distribution of potentially counterfeit and illegal medical products. Operation Pangea VIII was a project of massive scope, a lightning move by 115 countries that resulted in more than 2,400 websites being taken offline and the seizure of $81 million worth of potential dangerous illegal medicines and medical devices worldwide.

Howard Sklamberg

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

It’s a sad and cruel fact that drug and device counterfeiting and adulteration pose serious threats to public health. Unapproved and misbranded prescription drug products and unapproved/uncleared medical devices offered for sale on the Internet are potentially dangerous. The illegal sale of these medicines and devices bypasses both the existing safety controls required by the FDA and the protections provided when these products are used under a licensed practitioner’s supervision.

FDA is dedicated to sustaining and expanding the fight against counterfeits as part of our global strategy that leverages resources and expertise, engages the private and public sectors, and is data-driven and risk-based.

We have developed a Global Strategic Framework for counterfeit and substandard medical products (sometimes known by the acronym SSFFC, for Substandard, Spurious, Falsely-Labeled, Falsified, Counterfeit) to help protect consumers by reducing their exposure to counterfeit and substandard medical products.

The framework is focused on three pillars: Prevention, Detection, and Response.

What’s needed is better prevention of market entry of counterfeit and substandard products. Better detection of these products. And, more efficient response when counterfeit and substandard products are found.

FDA developed this framework in order to lay out a strategic vision of what is needed and how these needs can be met globally. There are areas where our expertise can and does contribute to prevention, detection, and response, but there are other areas where other U.S. federal and local government agencies, foreign counterparts, industry, healthcare professionals, consumer and patients, non-governmental organizations, procurement and donor organizations, standards bodies, and others have a role.

George Karavetsos

George Karavetsos, J.D., FDA’s Director, Office of Criminal Investigations

It is important for all players fighting to combat counterfeit and substandard drugs and devices to understand exactly how to best use our resources, knowledge, and experience, and leverage the work of others. This framework helps shape what roles we can play, minimize duplication of effort, and strengthen our global might in this fight against the criminals.

FDA has many ongoing activities and initiatives that support the framework goals. To better prevent counterfeit and substandard products from entering the market, we are working on improving the transparency, accountability, and integrity of the supply chain. Specifically, we are focusing on good manufacturing, distribution, and pharmacy practices, and we’re working for a convergence of global standards to create a more level playing field for the legitimate supply chain.

We are also implementing the new track and trace law (the Drug Supply Chain Security Act), which outlines steps to build an electronic, interoperable system to identify and trace certain prescription drugs as they are distributed in the United States, no matter where they originate. This is a collaborative effort whereby FDA is working with drug manufacturers, wholesale drug distributors, repackagers and dispensers (primarily pharmacies) to implement the law and develop the new system over the next eight years. Some of the key goals of this system will be to trace the path of drugs at the package-level through the drug supply chain, help ensure they are legitimate products, and enhance the detection of illegitimate drugs.

Cynthia Schnedar

Cynthia Schnedar, J.D., Director of the Office of Compliance at FDA’s Center for Drug Evaluation and Research

To better detect potentially harmful products before they enter the supply chain on their way to patients, we are focusing on improving information-sharing and communication. Also, as part of our effort for better detection, we are improving our surveillance through more efficient investigations of suspect incidents, and more quickly confirming that products are counterfeit. To this end, we have developed new detection technologies, specifically the handheld device, CD3, which uses wavelength detection to detect counterfeit drugs and packaging at our ports of entry.

Lastly, to better respond to incidents in the most efficient manner, FDA’s Center for Drug Evaluation and Research is developing more effective ways to notify the public of confirmed incidents and quicker removal of counterfeit products from the marketplace.

As for enforcement, we will continue to rely on our skilled professionals in FDA’s Office of Criminal Investigations (OCI) to lead domestic and global investigations to combat counterfeits. For example, OCI’s involvement in the past seven years of Operation Pangea has resulted in the seizure of more than $172 million in unlawful medical products — a real testament to effective international partnership.

We fully recognize that there are sophisticated, global criminal networks engaged in money laundering and the preparation and transportation of illegal products around the world. Importantly, we are meeting this global threat with international collaboration. FDA’s Office of International Programs has engaged with the World Health Organization’s Global Surveillance and Monitoring System, the World Bank, and the U.S. Agency for International Development in securing drug supply chains, reducing the threat of substandard drugs and strengthening regulatory systems.

We also collaborate with many foreign law enforcement organizations. For example, we have an OCI agent permanently assigned to Europol, based in The Hague, Netherlands. We also have a longstanding and solid partnership with the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA). More recently, OCI signed with the French National Gendarmerie a Letter of Intent to increase law enforcement collaboration.

Moreover, the stakes have grown higher in the U.S. as judges around the country recognize the risks of unapproved drugs in the U.S. marketplace. In January 2015, for example, a Turkish exporter of illegal drugs was sentenced to 30 months in federal prison for his part in the scheme.

As underscored by Operation Pangea last week, our actions to protect the health of Americans from the online sale of potentially dangerous illegal medical products will continue. In the longer term, our focus will be prevention, detection and response. We will need a more coordinated, domestic and global approach that leverages resources, expertise, tools, and trainings, and engages stakeholders, other regulators, and law enforcement.

Through our framework for strategically safeguarding supply chain security and integrity and combatting counterfeit and substandard drugs and devices, we know we are on the right path with the right goal: Protecting public health by helping to ensure that the prescription medications and devices used by health care professionals and patients are safe, effective and of high quality.

Howard Sklamberg, J.D., is FDA’s Deputy Commissioner for Global Regulatory Operations and Policy

George Karavetsos, J.D., is FDA’s Director, Office of Criminal Investigations

Cynthia Schnedar, J.D., is FDA’s Director, Office of Compliance, Center for Drug Evaluation and Research