FDA Taking Genomic Testing to the Next Level

By: Adam Berger and Zivana Tezak

President Obama’s Precision Medicine Initiative (PMI) envisions a day when the specific differences between people – genetic, environmental, lifestyle – will be used to customize the healthcare that we receive. Many of the current efforts toward achieving this goal have focused on analyzing and interpreting a person’s unique genetic makeup, including the identification of genetic alterations that may impact his or her health.

Adam Berger

Adam C. Berger, Ph.D., is Senior Staff Fellow on the Personalized Medicine Staff at FDA’s Office of In Vitro Diagnostics and Radiological Health, Center for Devices and Radiological Health

Next generation sequencing (NGS) technologies have significantly advanced the ability to derive more comprehensive genetic information on individuals in a relatively inexpensive and fast manner. In order to help achieve the goals of the PMI, FDA is developing new regulatory strategies for NGS-based clinical tests. We aim to ensure that these tests provide accurate, reproducible, and meaningful results relevant to a person’s medical condition while continuing to foster innovation so that people have access to the best available results generated by the most cutting-edge medical technologies.

In December 2014, we issued a preliminary discussion paper describing how FDA might go about creating a modern, flexible and dynamic regulatory system for NGS, which could potentially be applied to many other types of genomic tests. We expanded on this discussion by holding a workshop in February 2015 with a variety of stakeholders and received many helpful comments. To further advance this stakeholder conversation, we are holding two back-to-back public workshops on November 12th and 13th, 2015. The first will focus on analytical performance evaluation standards, including potential ways to develop these standards, which can be used by test developers to ensure their tests produce accurate and reliable results. We believe that stakeholder input about ongoing community standardization efforts is essential to construct flexible analytical evaluation approaches for a wide variety of NGS tests and modifications.

Zevana Tezak

Zivana Tezak, Ph.D., is Associate Director for Science and Technology at FDA’s Office of In Vitro Diagnostics and Radiological Health, Center for Devices and Radiological Health

The second workshop will address current challenges in clinical validation of NGS tests. A single company, lab, or institution is unlikely to have enough information to definitively determine the clinical importance of test results. The aggregation of clinical information in curated databases will create a “data commons” that could serve as a reliable source of scientific evidence that test developers could use to demonstrate that NGS test results are relevant to a person’s disease or outcome. The workshop will highlight how scientists, patient groups, and private industry can work together to develop high-quality, curated clinical databases of genomic information that associate specific genetic changes with various diseases, such as cardiovascular disease or diabetes.

In advance of these workshops, FDA will be releasing additional discussion papers informed by public input we have received, which will also include some general questions for stakeholder consideration. These documents will provide a high level overview of regulatory considerations for the development of analytical standards and the use of curated clinical databases to support NGS test submissions.

We look forward to engaging with the public on these important issues to ensure that advances in precision medicine rapidly turn into treatments that benefit everyone.

For more information:

President’s Precision Medicine Initiative

Sign up for the workshops or related webinars:

“Standards-Based Approach to Analytical Performance Evaluation of Next Generation Sequencing In Vitro Diagnostic Tests”

“Use of Databases for Establishing the Clinical Relevance of Human Genetic Variants”

Adam C. Berger, Ph.D., is Senior Staff Fellow on the Personalized Medicine Staff at FDA’s Office of In Vitro Diagnostics and Radiological Health, Center for Devices and Radiological Health

Zivana Tezak, Ph.D., is Associate Director for Science and Technology at FDA’s Office of In Vitro Diagnostics and Radiological Health, Center for Devices and Radiological Health

OpenFDA Makes Medical Device-Related Data Easier to Access and Use

By: Taha Kass-Hout, M.D., M.S., Roselie A. Bright, Sc.D., M.S., P.M.P. and Ann Ferriter

Taha Kass-Hout

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

OpenFDA is releasing information on medical devices that could spur innovation and advance scientific research.

OpenFDA’s Application Programming Interface (API) expands on the previous openFDA resources concerning medical device-related adverse events and recalls by incorporating information from the medical device product life cycle. This includes current data on device classification (6,000 records), 24,000 registrations of device companies and establishments, and the companies’ listings of more than 100,000 devices.

OpenFDA is a project that provides easy access to the many large, important, health data sets collected by FDA. Data since 1976 on 30,000 device premarket approvals (PMAs) and approval supplements, and 141,000 device clearances through premarket notifications (510(k)s) and granted de novo requests are now available on openFDA. Additionally, more details about device recalls (9,500 records going back to 2002) and adverse event reports (4.2 million records since 1991) were added. Although this information has been available in our public databases for many years, now developers can more easily access and use the data.

Roselie Bright

Roselie A. Bright, Sc.D., M.S., P.M.P., manages openFDA and is in FDA’s Office of Health Informatics, Office of the Chief Scientist

The flexible openFDA interface works well even when greater demands are made on it and is designed on a common platform so developers can harmonize and integrate data from various sources and build their own applications. For example, developers could develop a smartphone app to search all the recalls associated with a particular type of device or find all companies that manufacture certain types of devices.

However, there are some important safeguards to the data released. For instance, the information doesn’t contain anything that potentially could be used to identify individuals or reveal confidential commercial information.

Everything available in these datasets should be understood in the appropriate context. FDA has harmonized the data, but there may be instances when a query does not return a full and complete result. For example, if the name of a manufacturer is listed with different spellings, some variations may not be captured in the result.

Ann Ferriter

Ann M. Ferriter, FDA’s Director of Analysis and Program Operations, Office of Compliance, Center for Devices and Radiological Health

Some datasets are snapshots in time. The 510(k) dataset, for instance, shows who submitted the 510(k), the device name, and other information at the time of clearance. Moreover, the types of information that FDA has collected has changed over the years, which can make it difficult to look at data over time. Also, the data may not have enough information to establish cause and effect, incidence, or prevalence.

By design, openFDA is a research and development project that draws on community involvement. We are active in the openFDA communities on GitHub and StackExchange, and encourage researchers, scientists, and developers to participate in those communities. This API is the latest in a series of openFDA releases that has made publicly available data easier to access. FDA believes that you can use these tools to create innovative products that could help protect and promote public health. In fact, over the last year, there have been dozens of tools created using openFDA resources. We hope these enhanced device data will be put to similar advantageous use. Together, we can make openFDA an even more useful and powerful resource for all.

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

Roselie A. Bright, Sc.D., M.S., P.M.P., manages openFDA and is in FDA’s Office of Health Informatics, Office of the Chief Scientist

Ann M. Ferriter is FDA’s Director of Analysis and Program Operations, Office of Compliance, Center for Devices and Radiological Health

For more information:

Premarket Approval (PMA)

Premarket Notification 510(k)

New Section 513(f)(2) – Evaluation of Automatic Class III Designation, Guidance for Industry and CDRH Staff

What is a Medical Device Recall?

Manufacturer and User Facility Device Experience Database – (MAUDE)

Medical Device Databases

FDA, From a Distance

By: Claudia Heppner, Ph.D.

It is a great honor for me, as a European, to be working for FDA. I am one of the two Locally Employed Staff (Foreign Service nationals) currently working in FDA’s Europe Office in Brussels, Belgium.

Claudia HeppnerI came to this position after serving for 12 years in the European Food Safety Authority (EFSA), which is the European Union (EU) institution that provides independent scientific advice on existing and emerging food safety issues.

Before joining EFSA, I worked with the Secretariat of the EU’s Scientific Committee on Food. I’ve also worked for a multinational company in Belgium and the United Kingdom in the areas of pesticides product discovery and product development, including genetically-engineered plants.

With seven months at FDA under my belt, I enjoy and receive a great deal of satisfaction from my challenging new duties. Together with my colleagues, I am analyzing the range of science and policy issues under discussion in the EU’s decision-making framework. These EU issues span the breadth of FDA-regulated products and may sound familiar to some: updating and streamlining the food safety system; rapid access to innovative medicines; biotech, nanotech, novel foods, mobile and e-health; and, implementation of new legislation on tobacco and electronic cigarettes.

The EU has a complex environment for decision making, involving the “three pillars” (the European Commission, the European Parliament, and the Council of the EU) along with EU organizations that are counterparts to FDA such as the European Medicines Agency, EFSA, and various EU scientific committees.

In addition, each EU Member State (countries that are members of the EU) has its own national law-making bodies and regulatory organizations.

Only the European Commission can propose an EU law. The preparatory steps include: concept papers; a roadmap describing the timeline and significant events; impact assessments examining potential economic, social and environmental consequences; and public consultations.

I quickly learned that the European system is quite different from the legislative process and the notice-and-comment rule making system in the United States. In the Europe Office, we look at each step along the way in the EU decision-making process as a potential opportunity for strategic engagement.

Recently, I wrote a paper that analyzed what the EU is doing to strengthen food regulatory systems in Africa, China, and India. I was struck by the possibilities of what could be achieved through FDA and EU cooperation to help assure the safety of foods shipped to the United States and Europe and to improve public health around the world.

I feel fortunate to be working at FDA and to have the opportunity to broaden my professional horizons. I enjoy the dual focus on science and policy, working on medical product issues as well as foods issues, and observing how a non-EU organization like FDA works.

I look forward to continued learning and to the possibility of contributing to both the U.S. public health and – through FDA’s engagement with the EU – the EU public health.

Claudia Heppner, Ph.D., is a Senior Policy Analyst in FDA’s Europe Office

Find out more about FDA’s Europe Office

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 FDA.gov. 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.

Advancing precision medicine by enabling a collaborative informatics community

By: Taha A. Kass-Hout, M.D., M.S., and David Litwack, Ph.D.

FDA plays an integral role in President Obama’s Precision Medicine Initiative, which foresees the day when an individual’s medical care will be tailored in part based on their unique characteristics and genetic make-up. Yet while more than 80 million genetic variants have been found in the human genome, we don’t understand the role that most of these variants play in health or disease. Achieving the President’s vision requires working collaboratively to ensure the accuracy of genetic tests in detecting and interpreting genetic variants. We are working towards that goal by developing an informatics community and supporting platform we call precisionFDA.

Taha Kass-Hout

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

Sophisticated, relatively inexpensive technology known as next generation sequencing (NGS) already exists to sequence a person’s genome quickly. Developers and users of NGS tests must then comb these sequences to look for segments that suggest potentially meaningful differences and determine whether those differences provide useful and actionable information about the state of a person’s health, and their future risk of disease, behavior, or treatment choices.

Special features of this technology pose novel regulatory issues for FDA. Most diagnostic tests follow a one test-one disease paradigm that readily fits FDA’s current device review approaches for evaluating a test’s accuracy and clinical interpretation. Because NGS tests may be used in many ways in the clinic and can produce an unprecedented amount of data about a patient, we are working to evaluate whether a better option might simply be requiring each NGS test developer to show that the test meets certain standards for quality. Similarly, to demonstrate a test’s clinical value, we are assessing whether it may be more efficient for developers to refer to evidence in well-curated, validated, and shared databases of mutations instead of independently generating data to support a mutation-disease association.

David Litwack

David Litwack, Ph.D., Policy Advisor, Office of In Vitro Diagnostics and Radiological Health, at FDA’s Center for Devices and Radiological Health.

To begin to realize this new vision, precisionFDA is designed as a crowd-sourced, cloud-based platform to advance the science needed to develop the necessary standards. PrecisionFDA will supply an environment where the community can test, pilot, and validate new approaches. For example, NGS test developers, researchers, and other members of the community can share and cross-validate their tests or results against crowd-sourced reference material in precisionFDA.

Planned for beta release (work in progress) in December 2015, precisionFDA will offer community members access to secure and independent work areas where, at their discretion, their software code or data can either be kept private, or shared with the owner’s choice of collaborators, FDA, or the public. Initially, precisionFDA’s public space will offer a wiki and a set of open source or open access reference genomic data models and analysis tools developed and vetted by standards bodies, such as the National Institute of Standards and Technology (e.g., Genome in a Bottle). We believe precisionFDA will help us advance the science around the accuracy and reproducibility of NGS-based tests, and in doing so, will advance consumer safety. We look forward to continuing to update the community on the development of these new tools.

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

David Litwack, Ph.D., is Policy Advisor, Office of In Vitro Diagnostics and Radiological Health, at FDA’s Center for Devices and Radiological Health.

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: www.fda.gov/minorityhealth

Follow us on Twitter @FDAOMH

More information about the HDEART Workshop can be found here: http://www.pvamu.edu/nursing/hdeart/

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

The Times They Are a Changin’ – And So is FDA

By: Luciana Borio, M.D.

Today, at a public meeting of the FDA Science Board, the agency is releasing our progress report, FDA Science Moving Forward, highlighting advances that FDA has made since the Science Board’s 2007 report FDA Science and Mission at Risk. Also at this meeting, a Science Board subcommittee will present its recommendations on FDA’s regulatory science programs, scientific workforce, and collaborations.

Dr. Lu BorioAs our report FDA Science Moving Forward illustrates, FDA regulatory science programs have made dramatic advances in the last eight years. These advances are critical because, as I’m always reminded, regulatory science underpins virtually every decision we make at FDA.

Some of our early efforts focused on establishing an organizational framework to foster FDA’s vibrant scientific culture, with increasing opportunities for scientific collaborations and training of our staff.

FDA created the Office of the Chief Scientist and appointed a Chief Scientist, who was charged with working internally and externally to provide strategic leadership and advocacy for regulatory science and FDA scientists. Today that office, which I currently lead, has grown to also encompass offices dedicated to scientific professional development, counterterrorism and emerging threats, scientific integrity, toxicological research, women and minority health, as well as regulatory science and innovation.

In October of 2010, we outlined our broad vision for advancing regulatory science, followed by a more specific strategic plan in 2011 for our work within eight regulatory science priorities. Guided by these changes, we have successfully recruited additional top scientific talent to FDA, while strengthening our training programs and professional development opportunities for current staff. Importantly, we have developed new mechanisms and programs to leverage external expertise through a number of new partnerships and collaborations with our stakeholders, including the Centers of Excellence in Regulatory Science and Innovation.

FDA continues to keep pace with new science and technology, found in a growing number of medical product applications submitted for our review. For example, applications involving 3-D printing, devices incorporating nanotechnology and wireless controls, targeted drug therapies, and next generation sequencing technology are now commonplace in our regulatory portfolio.

Who would have imagined more than a century ago when FDA occupied a small office in the Department of Agriculture’s Bureau of Chemistry, that it would become what it is today—a leading regulatory agency with a public health reach that extends across the globe? More inspiring still have been the extraordinary advances in science and technology that have enabled FDA researchers to continually improve our food safety systems and help bring life-saving medical products from bench to bedside.

Although many challenges lie ahead, the progress report we are releasing today shows unequivocally FDA’s strong commitment to letting science guide our work of protecting and promoting the public health.

Luciana Borio, M.D., is FDA’s Acting Chief Scientist

‘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