FDA 2015: A Look Back (and Ahead) – Part 2: Medical Product Safety and Oversight

By: Stephen M. Ostroff, M.D.

In my first look back on FDA’s 2015 accomplishments, I focused on our achievements in medical product innovation and our constant drive to make safe, effective and innovative products available. Because FDA’s responsibility covers the entire life cycle of products, in this second year-end blog post, I will review FDA’s impact on medical product safety and oversight.

Acting FDA Commissioner, Stephen Ostroff, M.D.Responding to Ebola

In a world where disease knows no borders FDA’s response to the Ebola epidemic in West Africa demonstrates how we use our scientific expertise and regulatory authorities to the fullest extent possible to address a tragic public health crisis of global impact. Our response involved collaborating with partners across government, pharmaceutical and diagnostic companies, international organizations like the World Health Organization, and our international regulatory counterparts. We played a key role in expediting the availability of diagnostic tests and investigational therapeutics and vaccines, as well as investigating fraudulent products marketed to diagnose, prevent and treat Ebola. And many FDA commissioned corps officers of the U.S. Public Health Service served on the front lines, deployed in a humanitarian mission to provide care to patients at the Monrovia Medical Unit in Liberia, one of the West African nations that were hard hit by the outbreak.

Addressing Transmission of Infections from Duodenoscopes

This year we took steps to help protect the public from the risk of transmitted infections, including antibiotic-resistant infections, from duodenoscopes. Duodenoscopes are complex devices used during endoscopic retrograde cholangiopancreatography (ERCP), a potentially life-saving procedure to diagnose and treat blockages in the pancreas and bile ducts. In the United States, duodenoscopes are used in more than 500,000 ERCP procedures each year.

Last February, the FDA issued a safety communication to raise awareness about the risk of transmitted infections from duodenoscopes, after it determined that the design of these devices may impede effective reprocessing, even when the manufacturer’s reprocessing instructions are followed correctly. Reports also indicated that some healthcare facilities may not have adequately followed the manufacturer’s reprocessing instructions. To address these concerns, the FDA has been working with the device manufacturers to ensure that the reprocessing instructions for their duodenoscopes are put through the most rigorous testing. The Agency held a public advisory committee meeting in May to discuss the scientific challenges, and it incorporated recommendations for enhancing the safety margin of reprocessing duodenoscopes into a safety communication in August. Also in August, FDA issued Warning Letters to all three duodenoscope manufacturers citing violations found during recent inspections. In October, the FDA ordered the manufacturers to develop postmarket surveillance studies of how the devices are reprocessed in real-world clinical settings.

Our foremost concern is protecting patients, and we are committed to taking steps to assure that duodenoscopes – and all reprocessed medical devices — are safe to use.

Compounding

We continue to respond effectively to the 2012 outbreak of fungal meningitis linked to contaminated compounded drugs. We are implementing the Drug Quality and Security Act and continuing our inspection and enforcement efforts at compounding facilities nationwide. To that end we have issued numerous policy documents regarding compounding and related activities to provide guidance to industry as we implement the new law. We’ve also held meetings with stakeholders, including pharmacy, physician, and consumer groups, and we have continued our active and successful collaborations with state governments.

Addressing the Opioid Abuse Crisis

Over the last year, we’ve been very focused on the growing epidemic of opioid abuse and addiction and its devastating impact on public health. This focus has required us to strike a delicate balance: ensuring medical treatments are available for patients who are in pain, while addressing the often tragic consequences of abuse and misuse, which all too often overwhelm individuals, families, friends and communities. Our approach is multi-pronged, from encouraging scientific investigation to improving the training of practitioners who prescribe these powerful medicines.

We believe it is vitally important to encourage the development of abuse-deterrent formulations of opioids and to support options for medication-assisted treatment of opioid-dependence. Final guidance for industry regarding the development of abuse-deterrent formulations was issued in April and several abuse-deterrent products have been approved. We are also making strides to treat the consequences of overdoses. In November, FDA approved the first nasal spray version of naloxone hydrochloride, to provide a route of delivery in addition to injection for this life-saving medication that can stop or reverse an opioid overdose. And we are working with our federal partners to improve access to naloxone.

While we cannot solve this complex problem alone, we remain committed to making the best use of our regulatory authorities and working with our partners both in and outside government to reduce the risks associated with opioids. To continue to achieve that, we have been engaging in a comprehensive review of our many current activities related to opioids and identifying which measures can and should be strengthened and what further measures are needed to address this crisis during 2016.

Ensuring the safety of the medical products we regulate requires us to manage a wide-range of issues across multiple scientific disciplines; and to employ scientists with the knowledge to solve today’s complex regulatory challenges. The last year brought many challenges, and just as many solutions.

In my final post, I will address some of our accomplishments in the area of food, tobacco product regulation, and antimicrobial resistance.

Stephen M. Ostroff, M.D., is Acting Commissioner of Food and Drugs

What We Mean When We Talk About Data

By: Robert M. Califf, M.D. and Rachel Sherman, M.D., M.P.H.

Robert M. Califf, M.D., MACC, FDA's Commissioner of Food and Drugs

Robert M. Califf, M.D., Commissioner of the U.S. Food and Drug Administration

Medical care and biomedical research are in the midst of a data revolution. Networked systems, electronic health records, electronic insurance claims databases, social media, patient registries, and smartphones and other personal devices together comprise an immense new set of sources for data about health and healthcare. In addition, these “real-world” sources can provide data about patients in the setting of their environments—whether at home or at work—and in the social context of their lives. Many researchers are eager to tap into these streams in order to provide more accurate and nuanced answers to questions about patient health and the safety and effectiveness of medical products—and to do so quickly, efficiently, and at a lower cost than has previously been possible.

But before we can realize the dramatic potential of the healthcare data revolution, a number of practical, logistical, and scientific challenges must be overcome. And one of the first that must be tackled is the issue of terminology.

Defining Terms

Although “data,” “information,” and “evidence” are often used as if they were interchangeable terms, they are not. Data are best understood as raw measurements of some thing or process. By themselves they are meaningless; only when we add critical context about what is being measured and how do they become information. That information can then be analyzed and combined to yield evidence, which in turn, can be used to guide decision-making. In other words, it’s not enough merely to have data, even very large amounts of it. What we need, ultimately, is evidence that can be applied to answering scientific and clinical questions.

So far, so good. But what do we mean when we talk about “real-world data” or “real-world evidence”?

Rachel Sherman

Rachel Sherman, M.D., M.P.H., FDA’s Associate Deputy Commissioner for Medical Products and Tobacco.

Clinical research often takes place in highly controlled settings that may not reflect the day-to-day realities of typical patient care or the life of a patient outside of the medical care system. Further, those who enroll in clinical trials are carefully selected according to criteria that may exclude many patients, especially those who have other diseases, are taking other drugs, or cannot travel to the investigation site. In other words, the data gathered from such studies may not actually depict the “real world” that many patients and care providers will experience—and this could lead to important limitations in our understanding of the effectiveness and safety of medical treatments. Clinicians and patients must be able to relate the results of clinical trials—studies that are done in controlled environments with certain patient populations excluded and which may therefore be challenging to generalize—to their own professional and personal experiences. It seems straightforward, then, to think that studies including a much fuller and more diverse range of individuals and clinical circumstances could ultimately lead to better scientific evidence for application to decisions about use of medical products and healthcare decisions.

But “real-world evidence” has its own issues that must be understood and dealt with carefully. First of all, the vague term “real-world” may imply a closer relationship with the truth—that the real-world measurement is preferable to one taken in a controlled environment. For example, is “real-world” blood pressure data gathered from an individual’s personal device or health app better (e.g., more reliable and accurate) than a blood pressure measurement from a doctor’s office? It could be, because a patient’s blood pressure might be uncharacteristically elevated during a visit to the physician. But at the same time, do we know enough about the data gathered from the patient’s personal device—how accurate is it? Is the patient taking their own blood pressure correctly? What other factors might be affecting it?—to use it for generating evidence? Already we are being reminded of the complexities of potentially relying on data that were gathered for purposes other than the ones for which they were originally intended.

In most cases “real-world evidence” is thought of as reflecting data already collected, i.e., epidemiologic or cohort data that researchers review and analyze retrospectively. Also of interest is whether randomized trials can be conducted in these “real-world” environments. In considering comparisons of treatments, one must always consider the possibility that the treatments were not assigned randomly, but reflected some relevant patient characteristic. This is, of course, the reason for doing randomized clinical trials.

Better Terms for Complex Subjects

There is little doubt that the new sources of data now being opened to researchers, clinicians, and patients hold enormous potential for improving the quality, safety, and efficiency of medical care. But as we work to understand both the promise and pitfalls of far-reaching technological changes, we need a more functional vocabulary for talking about these complex subjects, one that allows us to think about data, information, and evidence in ways that capture multiple dimensions of quality and fitness for purpose (e.g., for appropriate use in regulatory decision making). The incorporation of “real-world evidence”—that is, evidence derived from data gathered from actual patient experiences, in all their diversity— in many ways represents an important step toward a fundamentally better understanding of states of disease and health. As we begin to adapt “real-world data” into our processes for creating scientific evidence, and as we begin to recognize and effectively address their challenges, we are likely to find that the quality of the answers we receive will depend in large part on whether we can frame the questions in a meaningful way.

Robert M. Califf, M.D., previously FDA’s Deputy Commissioner for Medical Products and Tobacco, became FDA’s Commissioner of Food and Drugs on Feb. 25, 2016.

Rachel Sherman, M.D., M.P.H., is FDA’s Associate Deputy Commissioner for Medical Products and Tobacco.

Want to Help the FDA? Become a Consumer Representative on an FDA Advisory Committee

By: CAPT Dornette Spell-LeSane, M.S.N., M.H.A., A.N.P.-B.C.

Have you ever wanted to be part of the food and drug regulatory process? Do you have a history of public interest or a passion for consumer advocacy? Do you have experience analyzing scientific data?

Dornette Spell-LeSaneIf you answered “Yes,” here’s your opportunity to become an advocate for consumers! The Food and Drug Administration continually seeks input from consumers on scientific and medical issues by including Consumer Representatives on Agency advisory committees.

Participation as a Consumer Representative requires a modest time commitment. Travel expenses are paid and representatives receive reasonable compensation.

Consumer Representatives serve as Special Government Employees on a committee for up to a four-year term. Committees meet 1-3 times annually for 1-2 days. All meetings are held in the Washington, D.C. area. Members receive per-diem and travel expenses and are paid at a GS-15/10 hourly rate for the days attending a meeting.

Consumer Representatives provide the perspective of consumers to advisory committees and do not represent their own personal expertise. Their role is to:

  • Represent the consumer perspective on issues and actions before the advisory committee;
  • Serve as a liaison between the committee and interested consumers, associations, coalitions, and consumer organizations; and,
  • Facilitate dialogue with the advisory committees on scientific issues that affect consumers.

To apply you must:

(1) Be an active participant in independent consumer- or community-based organizations or have a history of advocating for the public’s interest; and

(2) Demonstrate an ability to analyze scientific data and critique research design.

FDA’s Consumer Representatives are grass-roots advocates, organizers, policy makers, or leaders of organizations with an interest in a specific subject matter. We encourage all meeting the criteria to apply.

Individuals applying to be a Consumer Representative may nominate themselves or be nominated by an organization. Applications for membership are reviewed and individuals are selected for final nomination by their peers. The Consumer Nominating Organizations, called CNOs, whose objectives are to promote, encourage, and contribute to the advancement of consumer education and to the resolution of consumer problems, receive ballots and rank the nominated candidates. The individual with the highest number of votes is forwarded for final nomination and generally will be the consumer representative for the committee.

FDA utilizes a total of 50 advisory committees and panels to provide independent advice to the agency on a range of complex scientific and policy issues, and they are an important part of the agency’s decision-making processes.

Here is a list of our current and upcoming vacancies on FDA advisory committees:

Medical Device Panels:

Anesthesiology and Respiratory Therapy

Circulatory System

Molecular and Clinical Genetics

Dental Products

Hematology & Pathology

General and Plastic Surgery

Neurological

General Hospital and Personal Use

Ophthalmic

Drugs Products Advisory Committees:

Dermatologic and Ophthalmic

Pharmaceutical Science & Clinical Pharmacology

Biological Products Advisory Committees:

Vaccines & Related Biological Products

Other:

Science Advisory Board to the Food and Drug Administration

Science Advisory Board to National Center for Toxicological Research (NCTR)

CAPT Dornette Spell-LeSane, M.S.N., M.H.A., A.N.P.-B.C., is Deputy Director of FDA’s Advisory Committee Oversight and Management Staff

FDA Invests in Innovative Ways to Communicate to Hispanics

By: Gloria Sánchez-Contreras, M.A.

En Español

National Hispanic Heritage Month–celebrated annually from September 15 to October 15—gives Americans a great opportunity to celebrate the histories, cultures, and contributions of Hispanic Americans whose roots are in Spain, Mexico, the Caribbean, and Central and South America.

Gloria Sanchez-ContrerasAt FDA, we join in this celebration as we continue to use innovative ways to reach Hispanics as part of our mission to protect the public health. To achieve this goal, FDA uses media strategies that are culturally and linguistically tailored to Hispanics, who, according to research, are avid users of online and social media.

There are 54 million people of Hispanic origin in the United States, making them the nation’s largest ethnic or racial minority group, with 17 percent of the nation’s total population, according to the U.S. Census Bureau. The United States has the second-largest population of Spanish-speaking residents in the world, ahead of Colombia and Spain, and second to Mexico, a recent study by the Instituto Cervantes shows.

These statistics cannot go unnoticed. FDA recognizes the importance of connecting with this growing and diverse segment of our population. Consequently, we have increased our online consumer information in Spanish and developed a variety of bilingual communications strategies to reach and engage all Hispanics.

One of the most important strategies we use is to make sure that messages created for Hispanics speak to them effectively. We consider Hispanics’ informational needs, lifestyles, and cultural health beliefs both when creating new messaging and when translating messaging from English to Spanish.

For example, we know Hispanics respond better when communications are in their primary language – which can be English or Spanish – and when communications use images that relate to them. We do this by employing a bilingual and bicultural team that reviews messaging for cultural competence and adapts translations to ensure they are culturally sensitive and in plain language.

In addition to our English-language communications, we have developed strategies to reach out to Spanish-speaking Hispanics online. Our Consumer Updates and drug safety communications are regularly translated into Spanish. We share Spanish-language information through our social media channels, including Twitter, Facebook, Pinterest, and YouTube.

In addition, we also have a complete Web section in Spanish for consumers (www.FDA.gov/ArticulosConsumidor), a press room (“Comunicados de Prensa”), and a central page (www.FDA.gov/Espanol) that links to a variety of Spanish-language content developed across the Agency’s product centers and offices.

These are exciting times, and it is a privilege to lead some of these efforts for our agency. The Office of External Affairs works diligently across FDA to share important and timely public health news with Latino consumers, stakeholders, media, and community organizations. And during Hispanic Heritage Month—and all the months of the year–we want Hispanics to know that FDA is a trusted source of consumer information.

Gloria Sanchez-Contreras, M.A., is a Bilingual Public Affairs Specialist and the Spanish-Language Communications Lead in FDA’s Office of Media Affairs.

National Preparedness Month: FDA and Access to Medical Countermeasures During Public Health Emergencies

By: Brooke Courtney, J.D., M.P.H.

Just weeks after witnessing the fall of the World Trade Center on Sept. 11, 2001, I was a student volunteer in a New York City hospital emergency department when several people arrived saying they had been exposed to anthrax.

Brooke CourtneyOne had even brought a small plastic bag holding white powder. Around this time, the media was reporting on letters mailed that were laced with white powder confirmed to be Bacillus anthracis, which causes anthrax.

At the hospital, we wondered whether we might become exposed to anthrax and how it could be prevented or treated. We quickly escorted the patients who had been exposed to white powder safely away from others to be examined by physicians.

Fortunately, our patients hadn’t been exposed to anthrax. But the letters contaminated with the agent tragically led to five deaths, and 17 more people became ill. Many others were treated with antibiotics as a precaution.

That year, 2001, was a turning point in our nation’s readiness for public health emergencies, including those that result from deliberate attacks or from natural causes like a disease outbreak. In particular, the U.S. government has invested substantially in medical products required for diagnosis, prevention or treatment of a wide range of threats, including anthrax. FDA is part of that national preparedness.

At FDA, we work to help ensure the availability of safe and effective medical countermeasures (MCMs). These are the medical products, including drugs, vaccines, and in vitro diagnostics (IVDs), to counter chemical, biological, radiological and nuclear (CBRN) threats, including emerging infectious diseases like Ebola.

In 2010, FDA launched an agency-wide effort, the Medical Countermeasures Initiative (MCMi), to advance and coordinate the challenging, ongoing MCM development and emergency use work that was occurring in FDA’s product centers and other offices and with other federal partners. Our most recent program update details many of FDA’s MCM achievements since that time, including important, exciting product approvals and regulatory science advances.

At the foundation of FDA’s MCM efforts is a legal and regulatory framework strengthened by Congress after 2001 with the enactment of several MCM-related laws. For example, FDA now has the authority:

  • When the Secretary of HHS declares that the circumstances justify such an authorization, to authorize the use of unapproved MCMs and unapproved uses of approved MCMs under Emergency Use Authorizations (EUAs) during or in preparation for an emergency, and,
  • For approved MCMs, to authorize emergency dispensing by stakeholders, waive certain manufacturing requirements, and extend the useful life of product held in state and local stockpiles.

As an example of our legal authorities in action, we’ve issued multiple EUAs to facilitate access to uncleared IVDs to support disease detection and diagnosis during the H1N1 influenza pandemic and for H7N9 influenza, Middle East Respiratory Syndrome coronavirus (MERS-CoV), and Ebola virus that emerged in West Africa in 2014.

Today, our nation is far more prepared than at the time of the anthrax attacks with flexible emergency legal authorities, critical MCMs stockpiled or under development, and enhanced knowledge about how to prevent or treat threats. But, as the recent Ebola epidemic and MERS outbreak show, threats both known and unknown continue to evolve or emerge and require our constant attention and vigilance.

September is National Preparedness Month. And while FDA and other agencies work hard every day to help prepare the nation for potential threats, everyone can be involved in disaster readiness. As we approach the end of Preparedness Month, here are a few things you can do now:

  • Become familiar with disasters that might occur where you live; plans for your community, workplace or school; and what HHS is doing. You can also download a variety of free disaster apps.
  • Make and test a family plan (e.g., communicating during an emergency).
  • Make an emergency kit of supplies, including medical products, you’ll need for at least three days.

Brooke Courtney, J.D., M.P.H., is Senior Regulatory Counsel in FDA’s Office of Counterterrorism and Emerging Threats.

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

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

Stroke Awareness Month: What’s New in Stroke Therapies?

By: Jovonni R. Spinner, M.P.H., C.H.E.S.

Stroke is the leading cause of severe disability, and the fifth leading cause of death for all Americans. The burden is worse in minority communities; minorities have higher stroke risks, strokes at an earlier age, and more severe strokes. For example, African-Americans are twice as likely to die from a stroke compared to Whites.

Jovonni SpinnerOften this is because people do not know the warning signs (e.g., sudden numbness, confusion, or loss of balance), or the risk factors that lead to stroke, like high blood pressure, diabetes, and an irregular heart rhythm (atrial fibrillation, or AF). Some minority groups also suffer disproportionately because of cultural and language barriers- which can lead to a delay in treatment or not seeking treatment at all.

Aspirin Therapy: Who should use it?

Although there is broad agreement about the benefits of aspirin in secondary prevention of stroke, (the use of aspirin in people who have already had a stroke) there has been debate in the scientific community about the benefits and risk of using aspirin for primary stroke prevention, i.e., in people without a prior stroke. The Food and Drug Administration has not recommended that use.

To help dispel myths and provide accurate information, we have issued consumer and provider friendly guidance on the appropriate use of aspirin therapy.

Here is the latest evidence on who should and should not use aspirin for stroke prevention.

Primary prevention: In patients who have never had a stroke, aspirin therapy can increase their risk for bleeding in the stomach and brain and a reduction in strokes with aspirin has not been established.

Secondary Prevention: In patients who have already had an ischemic stroke, which happens when a blood vessel that supplies blood to the brain becomes blocked by a blood clot; aspirin therapy has been shown to decrease the risk of having a subsequent event. In general, the benefits may outweigh the risks for these patients.

Aspirin is, of course, readily available in drug stores and grocery stores. Before using it, however, patients should discuss with their healthcare providers whether aspirin therapy is the right course of action for stroke prevention.

Drug Trials Snapshot: Savaysa

On another note, In January 2015, FDA approved Savaysa, a drug used to reduce the risk of stroke in patients with AF, a type of abnormal heart rhythm. This is a blood thinning medication similar to several other recently approved anti-coagulants and an older drug, warfarin. All of these drugs reduce the chance of stroke in patients with this condition by more than 50%. But note, that for patients with kidneys that work really well, Savaysa did not work as well as warfarin.

More than 21,000 people with AF participated in the Savaysa clinical trial.  Clinical trial data, which are made available from the “Drug Trials Snapshot”, showed a large stroke reduction and no meaningful differences by sex, race (Whites versus Asians), or age (greater than 75 years) for the drug’s performance or side effects (e.g., major bleeding), a finding that is also true for the other anti-coagulants. Other minority groups were under-represented in this trial, so data are not available for those groups.

The Drug Snapshot is part of FDA’s transparency initiative that displays the clinical trial data analyzed by subgroup (e.g., sex, race, and age). This is an important initiative because it provides information on clinical trial participation among varying groups.

Here at FDA, we strive to make data transparent and easily accessible to our stakeholders. The Office of Minority Health is leading FDA’s efforts to encourage diversity of participants in clinical trials and assess possible differences in effects among varying groups. We know that demographic subgroups (e.g., minorities, women) can respond differently to medications and clinical trial participants should reflect the populations that will most likely use these products.

Visit our website or follow us on Twitter to find out more information about our research programs, outreach, and communications.

www.fda.gov/minorityhealth

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

FDA Teams With National Forum to Reduce Deaths from Heart Disease: Program is first of its kind

By: Heidi C. Marchand, Pharm.D.

In the U.S., only about 1 in every 4 prescriptions is taken as directed by a health care provider – a problem that costs our nation more than 125,000 lives a year. Millions of Americans with heart disease – the nation’s No. 1 killer – are especially vulnerable.

Heidi MarchandTo stem that tide, FDA has teamed with the nonprofit National Forum for Heart Disease and Stroke Prevention to advance the cause of a heart-healthy and stroke-free society.

FDA’s Office of Health and Constituent Affairs has signed a Memorandum of Understanding with the National Forum to promote and increase the use of health knowledge, skills and practices by the public in their daily lives. The five-year agreement is a first-of-its-kind cooperative public education program to reduce the burdens of heart disease and stroke.

Heart disease, which kills 1 in 4 Americans, can be managed. To prevent heart attacks, transient ischemic attacks and other cardiac events, doctors prescribe medications and lifestyle therapies (e.g. heart-healthy diets). Because medication is not readily adhered to – and neither are lifestyle treatments – millions of people suffer from preventable cardiac episodes. As a nation, lack of medication adherence (which can be as simple as not getting a prescription filled or refilled) costs more than $100 billion annually in excess hospitalizations.

To confront this problem, FDA is taking the lead in support of Million Hearts®, a national initiative of the Department of Health and Human Services to prevent 1 million heart attacks and strokes by 2017. A key partner in that mission is the National Forum, whose members include more than 80 U.S. and international organizations representing public, private, health care, advocacy, academic, policy and community sectors.

Together we will:

  • Explore, demonstrate and evaluate innovative health promotion concepts.
  • Exchange information on nutrition, heart disease, and ways to increase the number of patients who take their medication and/or therapy.
  • Identify and systematize best practices in behavior modification education.
  • Develop concepts for community-based interventions.

Our goals are clear: create recommendations to improve compliance with prescribed medical therapies and implement the recommendations to improve the lives of patients living with heart disease.

FDA’s Dr. Helene Clayton-Jeter and Dr. Fortunato “Fred” Senatore are leading a diverse team in identifying strategies to help patients take their medicines as directed and follow the advice of their doctors.

Concurrently, the National Forum will recruit a Therapy Adherence Steering Committee, made up of experts and stakeholders from physician and nursing groups, pharmacy (retail/system), behavioral health, consumer/patient groups and others invested in complying with medical therapy.

We’ll then jointly develop action plans for high-probability, high-yield strategies to promote heart health by helping ensure that patients take their medicines and adopt healthier lifestyles. Our plan is to complete all steps in the next several years.

We cannot fix this problem overnight. But by addressing it strategically, we can move forward and improve the odds of preventing and surviving heart disease and stroke among Americans.

Heidi Marchand, PharmD, is Assistant Commissioner in FDA’s Office of Health and Constituent Affairs