How Science and Strategic Collaboration Led to a New, “Personalized” Cystic Fibrosis Treatment for Some Patients

By: Janet Woodcock, M.D.

Targeting a drug for small subgroups of patients is a new way to find effective therapies. This is often called personalized medicine, and it’s one of today’s most promising areas of new drug development.

Last year, FDA approved two important targeted medicines: Xalkori (crizotinib), a lung cancer drug that targets tumors with the abnormal ALK gene, and Zelboraf (vemurafenib), a drug to treat malignant melanomas that have a certain gene mutation. Both drugs were approved with companion diagnostic tests to identify if patients have a susceptible tumor.

Today, the FDA approved Kalydeco (ivacaftor) to treat a specific subgroup of patients with cystic fibrosis (CF). Cystic fibrosis is an inherited genetic disease that affects a person’s lungs and other organs and may lead to an early death.

Janet Woodcock, M.D.What makes the availability of Kalydeco even more unique is that the drug’s developer, Vertex Pharmaceuticals, teamed up with the Cystic Fibrosis Foundation to develop and study the drug.

This success story began in 1989 when a team of researchers, including Francis Collins, now the director of the National Institutes of Health, discovered the gene that is involved in cystic fibrosis. This gene, known as CFTR, plays an important role in producing a protein that regulates the flow of salt and water out of the cells that line the cavities of the body. There are a number of different mutations that can cause the CFTR gene to produce a defective protein. This results in lung congestion and digestive problems.

Kalydeco targets a gene mutation that only occurs in about 4 percent of CF patients. Before using this medicine, doctors will test CF patients to determine whether they have this mutation (many CF patients have already been tested to understand what caused their CF).  If the patient is a match, the drug may provide substantial benefits including improved lung function and weight gain.

Patients have played an important role in how new drugs are developed and studied since the HIV/AIDS activists in the 1980s and 1990s. But what the Cystic Fibrosis Foundation pioneered is a new form of patient power that some have called venture philanthropy. The Foundation helped with a portion of the drug’s development costs, provided researchers with useful insights about the CF patient population and helped in the recruitment of study participants – contributions that were critical to quickly bringing the innovative new therapy to patients.

The unique and mutually beneficial partnership that led to the approval of this new therapy for some CF patients serves as a great model for future drug development and patient group collaboration moving forward.

Here’s to innovation and continued cooperation and progress for patients!

Janet Woodcock, M.D., is the Director for FDA’s Center for Drug Evaluation and Research

Skin Deep: New Skin Cancer Therapies Offer Progress for Patients

By: Patricia Keegan, M.D.

Skin cancer is the most common form of cancer in the United States and rates of new cases continue to increase, especially in younger populations. Some of that increase may be attributed to greater public awareness about the risks of skin cancer, resulting in more patients inspecting their skin and talking with their doctors about an appropriate diagnosis. 

There are three main types of skin cancer: basal cell carcinoma (BCC), squamous cell carcinoma (SCC), and melanoma. Melanoma is much less common than basal cell and squamous cell skin cancers but it is far more dangerous – it’s the leading cause of death from skin disease and is emerging as one of the most frequently diagnosed forms of cancer. In 2011, there were an estimated 70,230 new cases and 8,790 deaths from melanoma in the United States.  

But last year represented a tremendous leap forward for patients living with late-stage (metastatic) melanoma. For the first time in FDA’s history, we approved two novel therapies that extended the lives of patients (overall survival) with late-stage melanoma. Cancers that spread are frequently more difficult to treat and associated with poor outcomes for patients.

The two treatments, Yervoy (ipilimumab) and Zelboraf (vemurafenib), are not only unique because of their ability to extend a person’s life, but also recognized for the role they play in attacking the cancer itself.

Yervoy blocks a molecule known as cytotoxic T-lymphocyte antigen or CTLA-4 and works with a person’s immune system to recognize, target, and attack cells in melanoma tumors.

In 2011, the FDA approved Zelboraf, a personalized medicine to treat patients with melanoma whose tumors have the BRAF V600E gene mutation. With the aid of an FDA-approved test, patients can be selected to receive treatment with the drug, which blocks the ability of mutated BRAF to make tumors grow.

Today’s approval of Erivedge (vismodegib) for patients with late-stage or advanced basal cell carcinoma is the latest treatment advance for patients with the most common type of skin cancer, which is usually cured by surgery to remove the cancer. The treatment is the first drug approved for the small fraction of patients whose tumors are not controlled by or who cannot receive curative local therapy.

These important and innovative new therapies are encouraging for the millions of patients currently living with a form of late-stage skin cancer. Their availability today for patients is a testament to the ingenuity and dedication of the academic researchers and companies who studied and developed them; the patients willing participate in the investigational trials; and the FDA staff dedicated to timely scientific reviews with an eye to what is best for patients.

Patricia Keegan, M.D., is Director for the Division of Oncology Products 2 in the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research

Cancer Care in the U.S. – Looking Back, Looking Forward

By: Richard Pazdur, M.D.

2012. A milestone in my professional career marking the 30th anniversary since I finished my medical oncology fellowship and started practicing in the field. Oncology is a much different place today than in the late 1970’s when I was an oncology fellow in Chicago and when I took my first faculty position at Wayne State University in Detroit in 1982.  In 1988, I assumed a faculty position at The University of Texas M.D. Anderson Cancer Center and left Houston in 1999 to join FDA.

I remember a time when the only drug we had to treat advanced kidney cancer was the hormone, Megace… a time when the only drug to treatment metastatic colorectal cancer was 5-fluorouracil… a time when the only drugs to treat chronic myelogenous leukemia (CML) were hydroxyurea and Busulfan… a time when adjuvant therapy in breast cancer was just beginning to be investigated.

Today is a very different picture. Since 2005 FDA has approved seven drugs for advanced kidney cancer. Oncologists now have multiple options for the treatment of metastatic colon cancer, including the combinations of Camptosar (irinotecan), Xeloda (capecitabine), Eloxatin (oxaliplatin), Avastin (bevacizumab), Erbitux (cetuximab), and Vectibix (panitumumab). Patients with CML have been greatly helped with the introduction of tyrosine kinase inhibitors. Adjuvant therapy, both for breast and colorectal cancers, now has an established role in medical oncology practice and we have moved the early use of anti-cancer drugs a step further into the neoadjuvant setting in breast cancer.

There have been many advances during this 30-year period, including the development of curative therapies in childhood malignancies and great strides in supportive care products. I did not intend to prioritize these advances and I apologize for missing the one you feel most important.

“Cancer” is a multitude of diseases and our current classification of cancer based on organ location (“breast cancer”, “colon cancer”, lung cancer”) will be transformed based on the “molecular fingerprints” of an individual’s tumor. The increased understanding of cancer biology will have profound positive implications for all stakeholders with the development of drugs with greater efficacy and less toxicity.  

The future therapeutic advances in oncology will be based on our enhanced understanding of the biology of cancers. The identification of specific mutations in the BRAF gene in melanoma and mutations in BCR/ABL genes led to the development of therapeutic breakthroughs in melanomas and CML, respectively. Herceptin (trastuzumab) for the treatment of HER-2 positive breast cancer and the anti-CD-20 antibody, Rituxan (rituximab), to treat B-cell lymphomas are examples of using enhanced understanding of cancer biology to discover and develop targeted therapies.  In 2011, FDA approved two drugs – Xalkori (crizotinib) and Zelboraf (vemurafenib) – with concurrent approvals of in vitro diagnostic tests for patient selection providing a clear regulatory pathway for co-development of a drug and a diagnostic test. This year we also saw the approval of Jakafi (ruxolitinib), the first drug approved for myelofibrosis, whose development was based on our understanding of the JAK 1 and 2 pathways in the disease.

In a two-part appearance on Biocentury TV starting Sunday, January 29, I discuss what I see as the changing landscape of oncology (check your local listings). One point I discuss is the need for companies to take a more strategic approach to cancer drug development and to begin thinking of their business as a “disease” company vs. a “drug” company. Successful drug development will only occur with an in-depth understanding of basic disease mechanisms. This “disease orientation” will require greater collaboration between pharmaceutical sponsors, the academic community, government agencies, and patient groups.

The following week (February 5), three reviewers from FDA’s oncology office join me for a roundtable discussion about what they see as the future of oncology and what they encounter as practicing oncologists.

I have been fortunate to work with a talented team of oncologists, pharmacists, toxicologists, nurses, safety and public health experts to evaluate cancer drugs. Our staff understands that there are many diverse opinions in the oncology community regarding approval of oncology drugs. The balance between the immediate needs of an individual patient and the assurance of a drug’s safety and efficacy for approval will always exist. When appropriate, we have permitted the use of single patient INDs, exemptions to existing protocols, and expanded access programs to allow patient access to promising, yet unapproved, drugs.

To expedite drug development and our review of applications, FDA has approved oncology drugs on the basis of a single trial and used a variety of clinical trial endpoints — including overall survival (the time a patient lives before death), progression-free survival (the time a patient lives without their cancer progressing), and response rates (tumor size reduction). FDA has taken advantage of regulatory initiatives for serious and life-threatening diseases, such as fast-track, priority review, and accelerated approval.

When aspiring physicians ask me what it takes to become a medical oncologist I tell them – you must be an optimistic person. It’s demonstrated in the care of patients and a vision for the future of cancer care. Thirty years from now, our success won’t be measured by the number of cancer drugs approved, but by the number of patients living and functioning longer and feeling better.

This year also marks my 30th wedding anniversary to an oncology nurse who I met while we were mixing a chemotherapy drug in Chicago. My wife Mary would clobber me if I didn’t acknowledge the role of the dedicated oncology nurses who kept us all in line—past, present, and future.

Richard Pazdur, M.D., is the Director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research

FDA Voice Interviews Stephen Spielberg, MD, PhD

FDA Voice: Dr. Spielberg, thank you for agreeing to let us interview you for FDA Voice.  Can you tell us about your position at FDA and what that entails?

Dr. Spielberg: I am the Deputy Commissioner for Medical Products and Tobacco. In creating this new position, Commissioner Hamburg envisioned that it would “provide high-level coordination and leadership across the Centers for drug, biologics, medical devices, and tobacco products, and support, coordinate and advocate for the work and needs of the Centers.”  In my short time at FDA, I have been working with the Center Directors and their staff to understand shared challenges and opportunities to advance regulatory science and practice across all “human products”.  Together, we have begun to define areas of mutual interest and synergy where we can work together and with external partners in the public and private sectors to bring the best of science to bear on our public health responsibilities, to advance managerial and operations support to optimize our core tasks, and to assure in everything we do that FDA is at the cutting edge of promoting and protecting the public health.

 Stephen Spielberg, MD, PhDFDA Voice: Why did you join FDA?

Dr. Spielberg: Throughout my career, I have been involved with FDA.  In fact, my MD-PhD training at the University of Chicago, particularly in the Department of Pharmacology, was in the context of former UC faculty (Drs. E.M.K. Geiling and Francis Kelsey) who were intimately involved with creation of FDA by their work on elixir of sulfanilamide (Federal Food, Drug, and Cosmetic Act of 1938) and thalidomide (Drug Amendments of 1962). So, in retrospect, I suppose I “grew up” with knowledge and appreciation of FDA.  Over the years, I have served as a member of the Pediatric Subcommittee, the Science Board, rapporteur for ICH E-11, and helped get BPCA and PREA through Congress. At this stage of my career, it is a true honor for me to be able to serve the Agency at a critical time in biomedical science and therapeutics.

FDA Voice: What did you do before joining FDA?

Dr. Spielberg: I have had a 35 year career as a pediatrician and clinical pharmacologist, including in academic settings in the US and Canada, as well as in the pharmaceutical industry. My research career has focused on human pharmacogenetics and pharmacogenomics, mechanisms of adverse drug reactions, and pediatric/development pharmacology and pediatric clinical trials. In the years prior to coming to FDA, I was Dean of Dartmouth Medical School, and subsequently headed a new personalized medicine program at Children’s Mercy Hospital in Kansas City, MO, as well as working with the Institute for Pediatric Innovation, a non-profit organization focused on advancing therapeutics for children.

FDA Voice:  What is the favorite part of your job here at FDA?

Dr. Spielberg: This is a remarkable time to be at FDA. Biomedical science is advancing at an incredible rate. We are now beginning to see the impact of genomic and other science in defining the causes of disease, and in the discovery and development of new therapies. Medicine now is at a place that I could barely have imagined when I began my career, but we have the age old challenges of how to skillfully and wisely use the knowledge we have at any time to advance the health of individual patients and of all patients we serve. At such a time, what could be more challenging and satisfying than being here at FDA and having the opportunity to advance the promotion and protection of the public health.

FDA Voice:  If you could tell the American public one thing that you think they don’t know about what your office does to directly benefit them, what would it be?

Dr. Spielberg: Every day, I am impressed by the outstanding, dedicated, hard working people here at FDA. Their focus on our public health mission is remarkable, and it is an honor to work with them.

FDA Voice: Dr. Spielberg, thank you so much for your time!

Stephen Spielberg, MD, PhD, is Deputy Commissioner for Medical Products and Tobacco

What Happens Behind the Scenes before You Receive Your Flu Vaccine

By: Maureen Hess, MPH, RD

You probably think about influenza once a year — during the winter months when flu season rolls in as it does every year. But, for FDA, it’s a year-round initiative; we are on the front lines of making sure there is an adequate supply of safe and effective vaccine every year. And even though the current flu season is just getting started, we are already making preparations for next year.

Woman getting flu shotVaccination is the single best way to prevent influenza. Influenza is a contagious respiratory disease caused by a number of different influenza viruses, infecting the nose, throat and lungs. Even if you are healthy, you can pass the influenza virus to someone else one day before symptoms begin, and, you can continue to infect others up to five days after getting sick. Therefore, it’s possible for a healthy person to unknowingly spread the virus.

Preparing for influenza season each year is a time-critical, highly orchestrated, collaborative effort by FDA, the Centers for Disease Control and Prevention (CDC), the World Health Organization (WHO), vaccine manufacturers, and the public health community.

FDA is at the center of the process. Working with the WHO and CDC to review disease surveillance and laboratory data, and with the input of our advisory committee, we select the three influenza strains for the vaccine that will be made available in the United States. Once the strains have been selected, work continues to get vaccine manufactured and distributed well before influenza season starts. We inspect the manufacturing facilities and we prepare and provide the reagents used by vaccine manufacturers to verify the vaccine’s identity and strength.

FDA also evaluates each manufacturer’s vaccine each year for approval and conducts lot release, that is, we perform certain tests and review the results of the manufacturers’ tests on the vaccines prior to vaccine distribution. And, we continue to monitor the safety of the vaccines once they are distributed and used to vaccinate the public. The manufacturing demands are tremendous, especially since a new vaccine must be  manufactured each year. And, this new vaccine contains not just one but three new vaccine components each year, specifically, the three strains of influenza virus within the seasonal vaccine: one influenza A (H3N2) virus, one influenza A (H1N1) virus, and one influenza B virus.

Why is FDA focused on influenza? Influenza seasons are unpredictable and present a serious public health issue. Vaccination is one of the most important ways to prevent influenza. Influenza can cause various symptoms, which may include fever, cough, sore throat, headache, body aches and chills and tiredness. And the disease can range from mild to serious: over a 30 year period between 1976 and 2006, estimates of seasonal influenza-associated deaths range from a low of about 3,000 to a high of about 49,000 people.

Vaccines to prevent seasonal influenza have a long and successful track record of safety and effectiveness in the United States. The vaccine won’t give you the flu, and not only should it protect you from getting sick, it can also make your illness milder if you are exposed to a different influenza virus strain that’s not included in the vaccine.

Have you gotten your flu vaccine? It’s not too late!

Maureen Hess, MPH, RD, is Health Science Advisor, Office of Vaccines Research and Review, in FDA’s Center for Biologics Evaluation and & Research

Don’t Worry About Your Orange Juice

By: Michael R. Taylor, J.D.

You may have heard something on the news about low levels of a pesticide found in some orange juice products that are a blend of domestic and imported orange juice.  Here are three reasons you do not need to worry about the safety of that glass of orange juice you are about to enjoy.  

First, in the United States, we have a public health goal to fully protect people from harmful pesticide residues in the food we consume, and we have a system in place to do just that. Before the Environmental Protection Agency (EPA) sets “tolerances,” or the maximum level of pesticide residue allowed on a food, it first must determine that the pesticide residue level is safe in food.  It is then FDA’s responsibility to ensure that those tolerances are not exceeded on the foods it regulates.  If a tolerance has not been set or there is no exemption from a tolerance, companies are responsible for ensuring that their products do not contain pesticide residues.

In this case, FDA recently learned that a company had detected very low levels of the pesticide carbendazim in its own and its competitor’s orange juice products.  While carbendazim is used in Brazil and some other countries to protect against a type of mold that grows on orange trees, it is not used in the U.S., there is no EPA tolerance set for it and it is an unlawful pesticide residue.  It is also important to note that three quarters of the juice consumed in the U.S. is from oranges that are grown in the U.S. The implicated orange juice from Brazil accounts only for a much smaller proportion.

Second, FDA is testing orange juice products being imported into the United States and will not permit their entry into the U.S. until our testing and analysis confirms that the orange juice product complies with our laws.  This means that the orange juice products entering the country from Brazil or anywhere else are safe for consumers.  It also serves as a reminder to other food companies not to allow illegal pesticide residues on their products entering the United States.

Third, FDA immediately consulted with EPA, which conducted a risk assessment and found that the levels being reported for orange juice products already in the United States were far below any level that would pose a safety concern.  For that reason, FDA determined that a recall of orange juice already on the market is not warranted.  However, FDA is testing orange juice products for carbendazim in the U.S. and if we identify orange juice with carbendazim at levels that present a public health risk, we will alert consumers and take the necessary action to ensure that it is removed from the market.

We recognize that from time to time problems in our food safety system occur, particularly with increased globalization.  With limited resources, we obviously cannot be everywhere at once.  That’s why we target our efforts on those products that pose the greatest risk and act promptly when problems are brought to our attention.  In this case, FDA and EPA quickly assessed the health risks and made a decision that protects consumers without creating unnecessary disruptions to orange juice products already on the market. 

What does this mean for consumers?  Continue to enjoy your orange juice.

 Michael Taylor is Deputy Commissioner for Foods at the FDA


Committees, Conflicts and Everything in Between

 By Jill Hartzler Warner, J.D.

One of the strengths of this agency is the breadth of experience and diversity of opinion that exists among the more than 11,000 employees who work on FDA’s behalf around the world.

As with any organization that relies on science to guide its decision-making, robust scientific debate is expected and encouraged. In circumstances where agency staff seeks additional input because of such things as emerging research, the complexity of a new technology, or conflicting scientific data, FDA may turn to outside experts to provide important insights that would help inform our decision-making.

We often ask these experts to advise us on a range of scientific questions – including whether the safety of a drug is properly characterized for patients or if a new device would provide meaningful benefit for patients.

Health care professionals and patients should know that FDA ultimately makes the final decision on all regulatory activities. However, our advisory committee experts, which include patients, provide invaluable insights to the agency on how the broader public will approach a particular product or issue.

There has been a lot of public discussion in recent years about how we engage these outside experts through our advisory committee process. Typically, that discussion centers on how we evaluate committee members’ financial interests and prior involvement with regulated industry.

A lack of understanding about our selection and evaluation process, as well as federally mandated limitations on the type of information we can share publicly, have sometimes resulted in confusion and misunderstanding among the public.

Advisory committees play an important role in FDA’s mandate to promote and protect the public health by allowing the agency to engage independent, technical experts from a variety of disciplines. Our committees are often comprised primarily of individuals working in academia; but also may have representation from private practices, professional and patient groups, regulated industry, and other government agencies.

Before each meeting, FDA screens all advisory committee members who are special government employees or regular government employees for financial conflicts of interest. We ask the members to report to FDA any past and all current involvement or relationships with the products, companies, and issues related to the meeting. Members are required to report all current financial interests that could be affected by the outcome of the advisory committee proceedings and any FDA decision based on the committee’s recommendations.  For example, this might include stocks and investments and consulting arrangements.  In addition, they report involvements that might present an appearance of a conflict, to the best of their knowledge.

FDA carefully reviews each financial interest reported by each potential committee member and determines whether any would disqualify them from participating. If a member has a financial conflict of interest, they may not participate unless a waiver is granted, allowing them to participate. The agency also reviews other interests that may raise an “appearance” of a conflict and has the discretion to have the member not participate if the circumstances would cause a reasonable person with knowledge of the relevant facts to question his or her impartiality in the matter.

When the FDA decides to grant a waiver to a committee member, we post it to FDA’s website before the meeting, along with the type, nature and magnitude of the waived financial interest. If no financial conflict was determined by the agency, the information submitted by the committee members is considered confidential and by law we do not release that information to the public.

We make clear to our committee members that failure to report relevant known financial interests to FDA prior to participation in an FDA advisory committee meeting is illegal and subject to possible criminal penalties.  

I want to specifically address recent reports related to the December 8, 2011 advisory committee meeting that discussed drospirenone-containing oral contraceptives. Based on our review of the members’ reported financial interests, we did not identify any financial interests that would have precluded their participation.

In April 2010, Commissioner Hamburg highlighted a draft guidance for FDA staff and advisory committee members that outlined her desire to create a more consistent and transparent process for disclosing conflict of interest waivers granted by the agency. This is a vision I share.

It is worth noting that since FY2007, the rate of conflict of interest waivers granted by the FDA has been declining sharply. In FY2007, the agency granted waivers for 15 percent of meeting participants; in FY2011, we granted only one percent. How to handle conflicts, while obtaining the best expertise available, is not a challenge unique to FDA. In fact, other government organizations, academic institutions, and peer-reviewed journals are frequently confronted with how to obtain this information from prospective staff and authors as well. Given our interest in this area, we have been working closely with outside stakeholders and theInstituteofMedicineto explore whether a common financial disclosure form or website could be created making it easier for individuals to report their financial interests.

I’m personally excited about what an approach like this could mean for the agency. In its design, this approach could allow individuals to report information in a standard and consistent way, serving as a resource for agencies like FDA looking to receive reliable information while at the same time reducing burden on our experts. We are also exploring the administrative steps in review of conflict of interest screening and evaluating whether the process can be streamlined.   

I want to personally thank each and every one of the past and current members of FDA’s 50 advisory committees and panels. They have been generous with their time and expertise and have played an instrumental role in allowing the agency to fulfill its commitment to protecting the public health.

Jill Hartzler Warner, J.D., is FDA’s Acting Associate Commissioner for Special Medical Programs

What User Fees Can Accomplish

By: Margaret Hamburg, M.D.

There’s an old maxim that you get what you pay for. That’s certainly been the case with FDA’s highly successful drug user fee program. Under this program, industry agreed to pay fees to help fund a portion of FDA’s drug review activities and in exchange, FDA agreed to meet specific performance metrics like reviewing a specific percentage of product submissions within a certain time period. The program was launched in the early 1990s at a time when FDA lacked sufficient staff to perform timely reviews or develop procedures to make the process more rigorous, consistent and predictable. As a result, access to new medicines for U.S. patients lagged behind other countries. User fees meant FDA could hire additional reviewers and support staff even when budgets were tight. The upshot: the agency was able to conduct reviews in a timely fashion, revolutionizing the drug approval process.

Margaret HamburgSince it became law 20 years ago, the Prescription Drug User Fee Act or PDUFA has produced significant benefits for public health, reversing the drug lag and providing patients faster access to over 1,500 new drugs. As a result of the continued investment of PDUFA resources, the United States now leads the world in first introduction of novel drugs.

PDUFA must be reauthorized every five years and the latest program, known as PDUFA IV expires on Sept. 30. Working collaboratively with industry, patient groups and consumer groups, FDA came up with a set of recommendations for a PDUFA V that Health and Human Services Secretary Kathleen Sebelius is sending to Congress today.

Under these recommendations, fees paid by industry would support continued timely review of critical prescription drugs plus some important enhancements for patients and industry including a commitment to advance the development of drugs for rare diseases, provide for enhanced communication with small companies, and adoption of some cutting-edge approaches to the use of data that will improve drug development times.

PDUFA has been so successful that FDA followed a similar model in developing recommendations for two new user fee programs – one is for generic drugs, copies of brand-name drugs and the other for products that are shown to be “biosimilar to” or “interchangeable with” an FDA-licensed biological product – such as a protein, a vaccine or a blood component.

Both programs would offer a net win for consumers and patients, offering more choice and lower-cost products.

To my way of thinking, these significant proposals headed to Congress today demonstrate what can be achieved when industry, patient groups and FDA put their heads together and work towards a common goal.

Margaret Hamburg, M.D., is Commissioner of the U. S. Food and Drug Administration.

Gulf Seafood is Safe to Eat After Oil Spill

By: Michael R. Taylor, J.D.

This week, the Fish and Wildlife Service will begin a series of public meetings on the draft plan for the restoration of damage to natural resources resulting from the Deepwater Horizon oil spill. It is natural that the discussion of this plan would also raise the question of whether Gulf seafood is safe to eat.  The answer to that question is, yes. Gulf seafood is as safe to eat now as it was before the spill.

When the Deepwater Horizon spill occurred, there was a great deal of concern over the safety of the region’s seafood. Fishing areas were shut down and an extensive sampling program was introduced to ensure the seafood was safe to eat before the fishing areas were re-opened.

When we developed the sampling program, we had to determine which components of the oil were harmful so we could test for their levels in the seafood.  Following internationally recognized standards, we tested for Polycyclic Aromatic Hydrocarbons (PAHs).  They stay in seafood the longest amount of time, and once they are at a safe level any other oil hydrocarbons will be proportionately low, as well.

The FDA, NOAA, EPA, and the Gulf States, discussed and agreed on a level of PAHs that would raise a health concern.  If the seafood we tested was below the level of concern, it would be safe to eat. Over 10,000 seafood specimens were tested.  In most cases, no PAHs were found, and, when they were, the PAH levels in the seafood were 100 to 1,000 times below the levels which would raise a health concern.   The best way to understand how safe Gulf seafood is, is to visualize how much seafood you could eat and still not reach the levels of concern.

Given the low levels of PAHs we found, when we found them at all, someone could eat 63 lbs of peeled shrimp (that’s 1,575 jumbo shrimp); or 5 lbs. of oyster meat (that’s 130 individual oysters); or 9 lbs. of fish (that’s 18 8-ounce fish filets) every day for five years and still not reach the levels of concern.  We feel confident that the levels that were set are safe and protect the health of anyone who eats seafood, including children and pregnant women.

It is also important to understand the process we used to sample the seafood.  A seafood sample might actually go through multiple chemical tests.  The first test was a screening test, and, if the results came within 50% of the levels of concern, a second test was performed that more accurately measured the levels, allowing us to be certain about the safety of the seafood.

This process of double testing is not unlike the screening we go through when entering a court house, for example.  We often pass through a metal detector that screens us for metal objects.  If the detector goes off, the security guard typically will use a metal detecting wand to find the metal object and determine if it can brought into the building.  The seafood screening test would identify something that deserved a second look, and the second, more accurate seafood test would determine just what the PAH level was.  When PAHs were found as a result of the second test they were 100 to 1,000 times below the levels of concern.

A great deal of effort was invested after the Gulf spill so that we could provide an answer to one question:  Is Gulf seafood safe to eat?  Yes, Gulf seafood is safe to eat, and it is safe to eat for everyone.

Michael Taylor is Deputy Commissioner for Foods at FDA

Our Global Imprint in the Fight Against HIV/AIDS

By Edward Cox, M.D., M.P.H.

Since HIV/AIDS first entered the public consciousness in the 1980s, the biology of the disease has both perplexed and astounded us. Although more than 34 million people live with HIV worldwide, in recent years the disease has also served to unite us in a common goal – the search for a cure.

When I joined FDA more than a decade ago, after having spent time as a fellow at the National Institute of Allergy and Infectious Diseases at the National Institutes of Health and in private practice, I could not have imagined the unprecedented level of progress we’d see in the area of drug development for HIV/AIDS. The vast majority of these pivotal breakthroughs have occurred during the last decade and the development programs have often included clinical studies conducted outside of the U.S.

And while our core mission has traditionally focused on protecting the health of Americans while promoting the development of innovative drugs for use here at home, my background as an infectious disease expert allowed me realize early on that FDA would increasingly play an important role in the global public health arena, particularly in areas such as HIV/AIDS, hepatitis C and tuberculosis.

I am particularly proud of our role internationally in supporting the President’s Emergency Plan for AIDS Relief (PEPFAR). Since its inception in 2003, the PEPFAR program continues to serve as a great example of what can be accomplished through collaboration and coordination with our foreign partners.

Since 2004, the agency has worked to ensure that people living with HIV/AIDS worldwide have timely access to safe and effective antiretroviral drugs that have been manufactured by facilities in the U.S. and abroad. As of 2011, FDA has approved more than 137 formulations of drugs and new fixed-dose combination products to treat HIV.

The PEPFAR program itself has directly supported lifesaving antiretroviral treatment for over 3.9 million men, women and children through partnerships with more than 30 countries, including China, Haiti, India, Vietnam and more than a dozen African nations.

And just last week HHS Secretary Kathleen Sebelius and Deborah Autor, FDA’s Deputy Commissioner for Global Regulatory Operations and Policy, met with regulatory officials from India and companies with operations in the region to emphasize the importance of continued dialogue on current and emerging public health issues. Among the many topics of discussion was the ongoing need and value of the PEPFAR program.

Today, FDA continues to actively encourage sponsors worldwide to submit U.S. marketing applications for single entity, fixed dose combination, and co-packaged versions of previously approved antiretroviral therapies. My colleagues in FDA’s Center for Drug Evaluation and Research (CDER) Office of Antimicrobial Products and Office of Generic Drugs stand ready to continue working with companies who are dedicated to providing low-cost therapies to people living with HIV/AIDS around the world.

In December, we commemorated the 23rd anniversary of World AIDS Day and highlighted the 2011 U.S. theme — Leading with Science, Uniting for Action — which Commissioner Hamburg noted was particularly meaningful to FDA given the agency’s role. The success of the PEPFAR program is an excellent example of how science and interaction can quickly translate into meaningful progress for people living with HIV/AIDS.

As a scientist and FDA reviewer, I follow the progress being made when new findings about the disease are released each year. Personally, I can tell you that at FDA we all celebrate the victories that represent progress for patients and express our frustration and disappointment with the setbacks.

While a cure remains elusive for now, we intend to solidify our global imprint by continuing our efforts in support of the PEPFAR program. For our part, FDA will continue encouraging the development of new and effective therapies to treat patients.

More information can be found on “What’s New at FDA in HIV/AIDS.”

Edward Cox is the Director of the Office of Antimicrobial Products in FDA’s Center for Drug Evaluation and Research