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

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

‘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

Improving Access to Medical Devices: FDA Uses Existing Clinical Data to Reduce Premarket Data Needs

By: Ben Fisher, Ph.D.

At the FDA, we recognize the value of encouraging medical device innovation. We recently have acted to reduce the time and cost of clinical trials while maintaining patient protections. By doing so, FDA is helping to ensure that manufacturers will be more likely to conduct their clinical studies in the U.S., and patients in this country will have earlier access to innovation.

Ben FisherOne way the FDA can reduce the time and cost of a clinical trial is to determine if publicly available clinical data for medical devices with which we have considerable experience can be leveraged to develop a less burdensome clinical trial design. For example, take the case of global endometrial ablation (GEA) devices, used to treat heavy menstrual bleeding by applying heat or extreme cold to the inner lining of the uterus.

Since 1997, the FDA has approved five GEA devices based on the results of randomized clinical trials (RCTs) of 250-350 women in each trial. The participants were assigned to a group that received treatment with a new GEA investigational device or to a control group treated with rollerball ablation, an older, well-known technology for treating heavy menstrual bleeding.

Each of the RCTs shared similar study and control populations, study design, and endpoints. Those characteristics, combined with the consistent performance of the roller ball ablation device (the control device) across the RCTs, prompted the FDA to assess whether we could leverage the RCT data to help support a less burdensome clinical trial design for future premarket approval applications for GEA devices.

With input from industry and members of the FDA Obstetrics and Gynecology Devices Advisory Panel, the FDA was able to apply a statistical analysis model, called an objective performance criterion (OPC), to determine the minimum acceptable success rate for demonstrating device effectiveness. The FDA will post detailed information on how we developed this OPC on our website soon.

The FDA’s development of an OPC means that less burdensome clinical trial designs without a control group may be appropriate for clinical studies of GEA devices, resulting in studies that require fewer subjects, thereby reducing the length and cost of such clinical trials compared with RCTs.

In addition, development of an OPC may help encourage subjects to enroll in these clinical trials since all study subjects would undergo treatment with the investigational device.

The FDA has established a strategic priority of strengthening the clinical trial enterprise. This includes finding ways to streamline clinical trials so that fewer resources are required to bring a new device to the market.

Through strengthening the clinical trial enterprise, we hope to encourage manufacturers to study new and important medical devices in the U.S., helping us fulfill our vision of providing patients with high-quality, safe and effective medical devices of public health importance first in the world.

Ben Fisher, Ph.D., is FDA’s Director, Division of Reproductive, Gastro-Renal, and Urological Devices, in the Office of Device Evaluation at the Center for Devices and Radiological Health

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

Meeting Face-to-face Makes All the Difference

By: Heidi C. Marchand, Pharm.D.

While to many, the cherry blossoms in Washington, D.C., signal spring, for my office the season means bountiful opportunities to meet with groups in town for meetings and conventions in our capital city.

Heidi MarchandPatient and health professional advocacy groups that are some of FDA’s key stakeholders come to FDA Headquarters in nearby Maryland —or we go downtown to their meeting sites—for a mutual exchange of information that often has a profound influence on how we do our jobs protecting and promoting the public health.

So far, we have had informative discussions with groups as varied as the American Association of Nurse Anesthetists, the American Academy of Pediatrics, the American Celiac Disease Alliance, the ALS (Amyotrophic lateral sclerosis) Association, and Parent Project Muscular Dystrophy.

Because we are part of the Office of the Commissioner, we’re familiar with the agency across its various centers and are ideally positioned to connect stakeholders with the experts best suited to answer questions and offer assistance.

We hear from individuals on the front lines—parents of patients with heartbreaking childhood diseases, nurses who witness firsthand the consequences of a medical device that fails to work properly, patients who want to know where and how they can participate in clinical trials.

Many are experts in their area of advocacy—they’ve had to be—and their insights are invaluable.

Putting What We Learn To Good Use

For example, as we developed a rule, mandated by Congress, to define the term “gluten-free” for voluntary use in food labeling, we not only opened the proposed regulation up for public comment on two separate occasions, but we also conducted listening sessions with groups representing people with celiac disease, who must avoid consuming gluten but want a diverse and nutritious diet. They talked about the difficulties they face in trying to identify foods that won’t endanger their health, shared information about their understanding of challenges facing the food industry, and discussed the science that underlies this issue. This information helped us to ensure that the final rule was responsive to their needs. Now people with celiac disease can be assured that if they see “gluten-free” on food labels, that term has a specific, nationally uniform (and federally enforceable) definition.

Of course, our outreach efforts extend beyond these meetings. Our staff keeps in close touch with patient and health professional advocacy groups throughout the year, and through our FDA Patient Network website where we provide information on public meetings, current FDA draft guidances, clinical trials, and drug and device approvals. In addition, our patient newsletter keeps our stakeholders apprised of this and other important work FDA is doing.

But there’s nothing like meeting face-to-face across a table.

We listen to what our constituents have to say, we take it to heart, and we share it with our colleagues. What we learn through these conversations informs our work. It becomes part and parcel of the regulations we put into place to promote and protect the public health.

Heidi C. Marchand, Pharm,D., is Assistant Commissioner in FDA’s Office of Health and Constituent Affairs