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

@FDAOMH

 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

FDA Celebrates 30 Years of Advancing Health Equity

By: Jonca Bull, M.D.

April is Minority Health Month! I am proud to say that FDA’s Office of Minority Health (OMH), in collaboration with  the Department of Health and Human Service’s Office of Minority Health, is celebrating this year’s theme: “30 Years of Advancing Health Equity, The Heckler Report: A Force for Ending Health Disparities in America.” For us at FDA, this year also marks the 5th anniversary of OMH, which serves as the principal advisor to the Commissioner on minority health and health disparities.

Jonca BullThe Heckler Report was a major, ground breaking document that transformed HHS’s views and actions on minority health. For the first time in history, representatives from each agency convened to talk about minority health and, more importantly, put forth recommendations to achieve health equity. Findings illustrated huge disparities between African Americans and other minorities compared to the population at large for key health indicators, such as life expectancy and infant mortality. Key recommendations relevant to FDA’s mission centered around health information and education, cooperative efforts (inside and outside of the government), health professions development, data development, and developing a research agenda.

Let’s stroll down memory lane and recap FDA’s activities that resulted from the Heckler report.

Health Information and Education 

FDA has developed numerous outreach activities to improve consumer education and access to health information by utilizing the best cultural and linguistic practices to reach diverse minority populations. Hosting symposiums and webinars, participating in conferences, exhibiting in health fairs, and creating consumer educational materials are just some of the activities FDA has carried out to raise awareness and educate the public. Most recently, OMH has created a social media presence on Twitter and Pinterest, and maintains an active listserve with a quarterly newsletter. One of our most successful outreach campaigns has been the “Heart Health Toolkit” for American Heart Month, which reached over 6,000 people in February.

Our most recent consumer outreach occurred on March 25th via a webinar on how the public can respond to requests for comments on regulatory proposals and public health issues by using FDA dockets.

Cooperative Efforts/Health Professions Development

OMH embraces the notion that protecting the public’s health cannot be done in isolation. We have focused on four areas to improve stakeholder relations:

  • Work with Industry to increase diversity in clinical trials;
  • Work with minority serving institutions and organizations to implement strategies and programs to improve regulatory science (specific to minorities);
  • Provide platforms for stakeholders to become informed and involved about our work; and,
  • Host and promote mentoring programs to encourage minorities to stay in scientific and academic careers.

Data Development and Research Agenda

We have a robust research agenda that focuses on advancing regulatory science related to eliminating health disparities. The agenda consists of various intramural and extramural grant programs, giving preference to minority-serving institutions. FDA also promotes and funds research that aims to increase the quantity, and improve the quality, of data on minorities, and to make these efforts transparent to the public.

In short: FDA has been and will continue to be committed to narrowing the health disparities gap. OMH will continue our legacy of creating culturally and linguistically tailored tools, materials, and resources for minority communities to increase their awareness and understanding of FDA’s mission and of the products that FDA regulates, increase their participation in clinical trials, and increase diversity in the workforce. This ensures better representation in the workforce, and most importantly: better health for all minorities!

More information about specific programs can be found on our website.

The Heckler Report can be found at: http://collections.nlm.nih.gov/catalog/nlm:nlmuid-8602912-mvset.

Jonca Bull, M.D., is Director of FDA’s Office of Minority Health

Turning the Tide on Ebola

By: Calvin W. Edwards, CAPT, U.S. Public Health Service Commissioned Corps

Before accepting their agreements to work in Liberia in a mobile hospital for Ebola patients, I emailed each of the 68 Commissioned Corps officers of the U.S. Public Health Service that they would face danger, needed to accept certain risks, and that they would have to perform under extremely austere conditions.

Capt. Edwards

Calvin W. Edwards, CAPT, U.S. Public Health Service Commissioned Corps

Turns out, if anything, I may have underestimated the hardship.

Initially we lived 12 to a room in two-bedroom cabanas that were intended for four people and had ”septic” issues (sewage backups and overflowing toilets) to boot. We ate MRE’s (Meals Ready to Eat) intended for troops in combat. Six weeks into the two-month deployment, we were moved into tents next to the mobile hospital, about 20 officers per tent.

We worked 12-hour shifts in 90 degree heat and 90 percent humidity, often wearing head-to-toe heavy layers of plastic gear that left us soaked in sweat within minutes. A single slip in protocol could expose us to a deadly virus as we worked with vomiting patients who could defecate up to three gallons of diarrhea a day and could quickly dehydrate.

The Payoff

But when the first patient emerged with a clean bill of health, the hardships were forgotten.

A Liberian physician’s assistant, a man in his 30s, was the first to walk out with a certificate documenting that two tests showed him to be virus-free.

Ebola Survivor

U.S. Public Health Service officers celebrate as a Liberian man adds his handprint to a “survivors wall.” Each patient who overcame Ebola after treatment at the USPHS mobile hospital outside Monrovia was given a set of clothes and essentials and invited to mark their recovery with a handprint.

His family arrived like a chorus, singing, praising God, thanking America, jubilantly crying, and shouting, “He lived!”

This ex-patient became the first person to dip his hand in yellow paint and make an imprint on our “survivors’ wall.” Although total numbers have not yet been released, there were more handprints to come, and more patients survived than died.

Each patient who walked out confirmed that we were correct deciding to cross an ocean to help stop the spread of Ebola. To see a once desperately sick person recover and go home, knowing we had a part in that, was more satisfying than I can describe.

We were the first group of Public Health Service officers to move to the tents that were set up as a hospital in Liberia. Our group, which I commanded, included 24 women and 45 men. I had three weeks’ notice before flying out; some in our group had as little as five days to decide and be deployed.

Since our return, two more groups of Public Health Service officers have been deployed to the hospital built in a remote area about 1.5 hours from downtown Monrovia. The final group is still there.

The Mission

Our mission was to treat health care workers who contracted the Ebola virus. Before our arrival health care workers were leaving in droves, and the volunteer pool was drying up. The creation of our treatment facility helped turn the tide.

Liberia Compound

An aerial photo shows the mobile medical compound where Public Health Service Officers treat health care workers who contract Ebola.

The Liberian Ministry of Health told us that some health care workers were waiting until we had set up the facility before agreeing to come to Liberia. The fact we had a small part in increasing the pool of workers treating desperate patients was awesome. How often in our lives do we get a chance to do something so big?

On the other hand, one of our greatest stresses was how little we could do to help defeat the virus. Often the most we could do was keep people clean and hydrated while hoping their bodies would develop antibodies.

It was especially hard emotionally when we lost a patient at night. Because Ebola patients are most infective right after they succumb to the disease, we could not risk moving them over the dimly-lighted pathway to the morgue.

The Public Health Service officers were the only people working in the hospital. They performed every job, even those most unpleasant, and did it regardless of rank, exalted skills, or letters after their names. Yet all came away happy about the role they played.

For my part, I am both humbled and intensely proud of these men and women — of their skills, dedication, selflessness, ruggedness and resiliency. I wish all Americans could share some measure of my joy.

CAPT Edwards is a Public Health Service officer who serves in FDA’s Office of Regulatory Affairs. The U.S. Public Health Service Commissioned Corps is made up of 6,500 public health professionals who provide leadership and clinical service roles within the federal government agencies to which they are assigned.

Rare Diseases at FDA: A Successful Year for Orphan Products

By: Gayatri R. Rao, M.D., J.D.

2014 was a strong year for rare disease product development at FDA. It was also a year of significant firsts.

Dr. Gayatri RaoIn recognition of Rare Disease Day, February 28th, we want to reflect on the progress we have made thus far as we renew our commitment to rare disease patients. A rare disease is generally defined as a disease which affects fewer than 200,000 Americans a year. At FDA, the commitment to increase access to diagnostics and treatments to change the day-to-day reality of those living with rare diseases began over 30 years ago with the passage of the Orphan Drug Act.That commitment has steadily increased since then.

In 2014, we received our highest number to date of new requests for orphan drug designation. We received over 440 requests while just 7 years ago, we received less than half of that. We designated and approved more orphan drugs in 2014 than we had in previous years – nearly 300 drugs were designated and 48 were approved, including both novel and repurposed drugs. In 2014, 41% of all novel new drugs approved by the Center for Drug Evaluation and Research were for the treatment of rare diseases. Many of these orphan drug approvals were new and innovative, including Sylvant, to treat Castleman’s disease, which results in excessive lymph node growth, and Impavido, to treat forms of the tropical disease, leishmaniasis.

2014 was also a year of firsts for rare disease product development:

There were firsts in device development. For example, the Center for Biologics Evaluation and Research approved its first device through the Humanitarian Device Exemption (HDE) pathway. This device, CliniMACS CD34 Reagent System, helps to mitigate potentially serious immune reactions associated with stem cell transplantation in patients with acute myeloid leukemia.

FDA produced in 2014 its first agency-wide blueprint to accelerate the development of therapies for pediatric rare diseases – a report and strategic plan outlining how to address issues for developing products for this population.

2014 saw the issuance of the first rare pediatric disease priority review voucher for the treatment of mucopolysaccharidosis type IVA (Morquio A syndrome), a rare lysosomal storage disease which affects about 1000 patients in the United States and can lead to debilitating and life-threatening abnormalities of bones, joints and the heart.

In recognition of Rare Disease Day 2015, the international rare disease community is coming together to pay tribute to the millions of individuals impacted by rare diseases all over the world. Through the solidarity and commitment of many stakeholders – patients and families, healthcare professionals, researchers, companies, and policy makers – the awareness of the daily challenges that are unique to each rare disease and the efforts to create solutions has risen exponentially in the past several decades. As members of the rare disease community, we are proud of our collective accomplishments but remain acutely aware of how much more there is still to be done. Given how 2015 is already shaping up, we expect that by working together, we will continue to make great strides in developing much needed products for the millions of patients living with rare diseases.

Gayatri R. Rao, M.D., J.D., is FDA’s Director for The Office of Orphan Products Development

Shedding some light on FDA’s review of sunscreen ingredients and the Sunscreen Innovation Act

By: Theresa M. Michele, M.D.

With recent record snowfalls in many parts of the country, the use of sunscreens may not have been on many people’s minds. But here at FDA, sunscreens have been a front-and-center issue.

Theresa Michele, M.D.On November 26, 2014, Congress enacted the Sunscreen Innovation Act (SIA) that provides a new process for the review of safety and effectiveness of nonprescription sunscreen active ingredients. Among other things, the SIA creates timelines for FDA review.

Before the law was enacted we followed the regulatory process known as the Time and Extent Applications process, or TEA process for sunscreen active ingredients. This regulatory process provides, among other things, a mechanism for sponsors to request that FDA evaluate active ingredients that are used in over-the-counter (OTC) drug products, particularly those marketed in other countries. The TEA process can be summarized in two basic steps. Step 1 is FDA’s determination of eligibility, made upon a showing that the ingredient has been marketed over-the-counter in one or more countries for a material time and extent. Step 2 is FDA’s evaluation of the data to determine whether the ingredient is generally recognized as safe and effective (GRASE) for its intended use in an OTC drug product as described in the relevant regulation. If, after review of the data, FDA ultimately finds the ingredient to be GRASE for its intended OTC use, the ingredient may enter the U.S. marketplace. There were eight TEAs for sunscreen ingredients submitted to FDA before the SIA went into effect.

On January 7, we met the first requirement of the SIA. In doing so, we announced our tentative determinations that six of these ingredients are not GRASE for use in sunscreens because we need more data from the manufacturers to help establish the safety and effectiveness of these products.

Today, we completed another requirement by taking initial action on the last two pending ingredients, ecamsule and enzacamene. We tentatively determined, as we had with the other six ingredients, that we need more data to decide if these ingredients are, in fact, GRASE for use in OTC sunscreen products. Information about the SIA and our recent actions under the law are available on our new web page for this topic.

At this time there is not enough generally available data to determine whether any of the ingredients under review meet FDA’s safety and effectiveness standards.

We know our careful actions to seek more information may be disappointing to some who would like to see additional sunscreen products on the market immediately, but I’d like to take this opportunity to clarify some misconceptions about the SIA and the process for making sunscreen ingredients available for use in OTC products marketed without individual premarket review in the U.S.:

  • The law does not change FDA’s standard for general recognition of safety and effectiveness. The SIA requires strict deadlines for FDA to take action on these ingredients, but it does not relax the FDA’s scientific standards for evaluating the ingredient’s safety and effectiveness, or our need for adequate data on which to base such determinations.
  • The law does not provide FDA with additional resources. Recognizing the public health importance of sunscreen use, the FDA is proceeding as quickly as practicable to meet the requirements of the legislation. To assist in this process and to reduce the negative impact on other work, FDA is requesting funds for implementation of the SIA as part of the President’s fiscal year (FY) 2016 budget.
  • The SIA does not guarantee that products with additional sunscreen ingredients will be on the market in a specified timeframe. Because additional data are needed for each of the eight sunscreen ingredients, timelines for FDA actions are triggered by industry’s submission of required data.
  • There is apparent confusion as to why ingredients that have been on the market for years in other countries cannot be used in the U.S. without further review by FDA. While information on marketing history in other countries is helpful, what we can learn from it is limited. For example, such information doesn’t tell us anything about the long-term effects from use of the ingredient or how much is absorbed. Because of the widespread daily use of sunscreen products by a broad population, including babies and pregnant women, FDA has proposed data requirements that will allow us to determine that sunscreen ingredients are generally recognized as safe and effective. These data requirements were unanimously supported by a panel of scientific experts at a recent public Advisory Committee meeting on sunscreens.

We cannot achieve success in bringing additional sunscreens to market on our own. FDA is committed to doing our best to meet the new statutory deadlines, and we will be transparent in our process and progress. Successful implementation of the SIA will require a cooperative effort with industry and other stakeholders. We look forward to continuing this important work.

Theresa M. Michele, M.D., is the Director of the Division of Nonprescription Drug Products in FDA’s Center for Drug Evaluation and Research’s Office of New Drugs

The Meaning of Wearing Red

By: Margaret A. Hamburg, M.D.

Last night I had the pleasure of attending the annual Woman’s Day Red Dress awards ceremony in New York City. The event is one of the highlights of American Heart Month, and it was created by that magazine to educate Americans about, and help fight, heart disease, which has become the number one killer of women. Many are surprised to learn that while breast cancer is the cause of death of one in every 31 American women, one of every three women dies of heart disease. So I found it particularly meaningful, both as a doctor and a woman, to be honored for FDA’s work to improve women’s cardiovascular health.

Commissioner Hamburg at Event

FDA Commissioner Margaret A. Hamburg, M.D., at the Woman’s Day Red Dress awards ceremony in New York City

One of our efforts toward this end that was cited by the magazine was the proposal to update the Nutrition Facts Label. The proposed updates would more prominently highlight calorie and serving size information, inform consumers about “added sugars,” update the daily values for nutrients, and ensure that the serving size requirements reflect the amounts of food people actually consume. They would encourage consumers to use the label to take note of foods high in sodium, saturated fat, and trans fat, which can increase the risk of coronary heart disease.

We also published final rules on restaurant menu and vending machine labeling. Calorie information is the key component of these requirements, and obesity is associated with a range of heart disease related problems. The new rules also require that other nutrition information, such as sodium, is provided upon the consumer’s request. High sodium intake can increase blood pressure, a major risk factor for heart disease. As with the nutrition facts label, these menu labeling requirements will give consumers nutrition information they need to be able to make healthy food choices for themselves and their families.

Another part of FDA that matters for cardiovascular health is our Center for Tobacco Products. Though its work is designed to protect the health of all Americans, it has special significance for women who, sadly, are catching up to men in the prevalence of tobacco-related diseases.

In the last 50 years, a woman’s risk of dying from smoking has more than tripled, and is now equal to that for men – not what we desire when we talk about equality. The more than 20 million women in the U.S. who smoke cigarettes are at risk not just for heart attacks, lung cancer, and strokes, but also emphysema and other serious chronic illnesses such as diabetes.

Our actions on smoking and nutrition have been complemented by the work of the Office of Women’s Health. Its outreach initiatives have helped provide women with tips and resources they can use to make better heart health decisions for themselves and their families. This Office has also supported research on treatment of heart disease in women.

FDA’s responsibilities also include reviewing, approving, and helping advance new and innovative medical products to diagnose, treat and prevent heart disease, including life-saving medical devices such as artificial hearts, stents, and heart valves, essential tests like echocardiograms, and important drugs for hypertension, lowering cholesterol and treating other aspects of cardiovascular disease.

Over the years, FDA’s support of women’s health has grown thanks to scientific advances, changes in society, and improvements in the agency itself. We will continue to promote these goals, not just in the area of cardiovascular health, but in women’s health more generally.

Of course, we can’t do it alone. And that’s why I sincerely welcome such events as the National Wear Red Day and Woman’s Day’s red dress awards. They help focus our nation’s attention and energy on the fight against women’s heart disease to which we, at FDA, are fully committed.

Margaret A. Hamburg, M.D., is the Commissioner of Food and Drugs