Category Archives: Regulatory Science

FDA’s Criminal Enforcement Priorities Protecting Public Health

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By: John Roth

Although perhaps not widely known, FDA’s Office of Criminal Investigations (OCI) is an integral part of FDA’s mission to protect the public’s health.  FDA’s team of top-flight federal agents – who have the same type of arrest authority as other federal law enforcement agents – gives the FDA unique fact-finding tools and provides for strong, industry-wide deterrence. Their work is different from, but enhances, the regulatory work typically conducted by inspectors and investigators that makes up the bulk of FDA’s field operations.

OCI focuses its efforts on threats to the public health in four areas: we investigate criminals who can’t be reached by the rest of FDA, particularly in the area of counterfeit and unapproved medical products; we move against public health problems when our ordinary regulatory tools aren’t the best option; we seek criminal penalties against conduct in which the harm to the public is so grievous that a criminal response is appropriate; and we investigate lying to the FDA and other behavior that deprives the FDA of its ability to regulate. We have a variety of tools at our disposal to accomplish our mission, including felony charges under the Federal Food, Drug, and Cosmetic Act, misdemeanor prosecutions of responsible corporate officers, and prosecutions for fraud, money laundering and obstruction of justice.

It’s a big job.  FDA regulation affects more than 20 cents of every consumer dollar spent on products in the U.S. And the fact is, some conduct by individuals and entities is beyond the reach of FDA’s typical administrative and civil enforcement tools. This conduct includes rogue internet pharmacies, purveyors in grey and black market unapproved medicines, and counterfeiters, both in the United States and overseas. Individuals intent on defrauding an unsuspecting public are often part of a larger organized criminal enterprise and don’t respond to the normal FDA regulatory process. They are engaged in a crime, and as a result the FDA treats it as a crime, and that’s where OCI steps in. 

The case of Shengyang Zhou is a good example. In January 2010, the FDA became aware of counterfeit versions of the popular weight loss drug Alli being distributed in the United States, and warned consumers about its dangers. The counterfeit product had entered the legitimate supply chain lacking the active ingredient contained in the genuine Alli and, especially alarming, contained undeclared amounts of the drug sibutramine, which is no longer sold in the United States because of concerns over its association with increased heart attacks and strokes. OCI agents had initiated a criminal investigation after intercepting unapproved dietary supplements laced with sibutramine and phenolphthalein that had been shipped from China to an individual in Colorado. This seizure ultimately led to OCI agents developing an undercover operation against a Chinese counterfeiter, Shengyang “Tom” Zhou. Posing as buyers, OCI Special Agents met with Zhou in Bangkok and negotiated a sale of counterfeit drugs to be shipped to the United States. During the undercover operation, Zhou bragged that he had a factory in southwest China that could make thousands of boxes of weight-loss products per day. Zhou was ultimately arrested, pled guilty, and was sentenced to 87 months’ imprisonment.  Evidence presented at Zhou’s sentencing included a physician who had unsuspectingly taken the counterfeit Alli and suffered a stroke.

Admittedly, not all our work is this interesting and dramatic. But this is a good example of the truly remarkable skills – from technological and scientific, to the intangible personal skills of working undercover overseas – that our agents bring to the task. In a future blog, I’ll explain our work when ordinary regulation is insufficient and when we move against people who lie to the FDA, which prevents the Agency from doing its regulatory job. Some may not be as colorful as the example above – but all will show the key role of OCI in furthering the FDA mission in protecting the public health. 

John Roth is the Director of FDA’s Office of Criminal Investigations

 

 

The Science of Abuse-Deterrence – Progress Toward Creating Safer Opioids

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By: Douglas C. Throckmorton, M.D.

Douglas C. Throckmorton, M.D.Since the 1990’s, extended-release and high-potency opioids have been on the U.S. market, providing a new option to help the treatment of pain. Unfortunately, while these pain relievers often have provided tremendous relief for many, their abuse has reached epidemic levels in the U.S., with devastating consequences to families and patients.

Over the years, FDA has worked diligently with many partners to address this crisis, while also working to ensure that patients in pain have appropriate access to opioid pain relievers.

One important step towards the goal of creating safer opioids, and one that is a high public health priority for FDA, is to encourage the development of formulations of these drugs that deter their abuse.

Today we’ve announced two actions that signal progress in the battle against the abuse of opioid drugs. First, we approved updated labeling for Purdue Pharma’s reformulated version of OxyContin extended-release (ER) tablets. The new labeling describes the product’s abuse-deterrent properties. These physical and chemical properties make it more difficult to crush, break, or dissolve the tablets. These properties are expected to make abuse by injection difficult and to reduce abuse by snorting. This is the first time we have approved such language in opioid drug labeling, and we made this determination after carefully reviewing the available science.

Our other action today was determining that the original formulation of OxyContin ER, which Purdue Pharma stopped shipping in August 2010, was removed from the market for reasons of safety or effectiveness. This finding is important because it means that FDA will not accept or approve any generic forms of the original OxyContin ER.

In making these decisions, FDA was focused on the relatively-new science of abuse deterrence in which the analytical, clinical, and statistical methods for evaluating these technologies are rapidly evolving. Our decision was grounded in the best available science. To guide drug development in this new field, FDA also issued a draft guidance for industry in January, announcing a flexible, adaptive approach to encourage the development of abuse-deterrent opioids. We believe such products have promise to help reduce prescription drug abuse and improve public health.

In the guidance, we describe four categories of abuse deterrence studies and lay out the kinds of labeling language we would consider approving, depending on the scientific data available. And because the science of abuse deterrence is evolving, we’re encouraging feedback and further development in this area, to help speed progress.

Moving forward, FDA will review every application on its own merits, based on applicable scientific and legal standards, and encourage an ongoing dialogue with manufacturers as they consider developing abuse deterrent opioid analgesic products. In addition to scientific rigor, flexibility is essential to encourage innovation, and, in essence, we’ll let manufacturers know where we want them to go, but not prescribe how exactly to get there. Our general goal, overall, is to encourage development of abuse-deterrent opioid products.

Today’s actions are significant components in a much larger effort to address prescription opioid abuse. We will continue to engage with the many groups active in this area – advocacy organizations, patients and family members, Congress, healthcare providers, and other federal government partners. Reducing the tragic toll of opioid abuse in the U.S. depends on this vital collaboration.

Douglas C. Throckmorton, M.D., is FDA’s Deputy Director for Regulatory Programs in the Center for Drug Evaluation and Research.

Proactive Inspections Further Highlight Need for New Authorities for Pharmacy Compounding

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By: Margaret A. Hamburg, M.D.

Ever since the recent fungal meningitis outbreak tragically took so many lives and injured so many others, we have been very clear that we all must act aggressively to help make sure nothing like this ever happens again. Last month I wrote in FDAVoice about the legislation and resources we believe are urgently needed to effectively oversee the evolving compounding pharmacy industry.

Margaret Hamburg, M.D.Above all, patient safety is our greatest priority. In addition to working with Congress and other stakeholders on legislation, we also have been very focused on evaluating our current surveillance and enforcement approach to compounding pharmacies and are using our existing, yet limited, authorities more effectively to protect consumers.

FDA has continued to conduct for-cause inspections upon receiving reports or complaints about serious adverse events related to drugs made by compounding pharmacies, or when states have requested our assistance.

And in coordination with state officials from around the country, we have just recently completed proactive inspections of 31 firms that are known to have produced sterile drugs in the past. We identified these firms using a risk-based model that included factors such as serious adverse event reports, historical inspection data, and reports of product quality problems. The inspections specifically focused on each firm’s processing of sterile drugs to identify firms that may pose a higher risk of producing contaminated sterile products. A summary of these inspections can be found on our website.

During the course of both our proactive and reactive inspections over the past few months, we observed concerning sterility practices, inappropriate conditions for sterile processing and other practices that create risk of contamination. As of this week, we have issued to all but one pharmacy that we inspected an inspection observation report (called an FDA Form 483, or just a “483”) that lists objectionable conditions observed at the facilities. The one firm not receiving a FDA Form 483 was not producing sterile drugs.  

As noted on some of these 483s, select FDA observations during the inspections include:  unidentified black particles floating in vials of supposedly sterile medicine; rust and mold in “clean rooms” where sterile injectable medications were produced; technicians handling supposedly sterile products with bare hands; and employees wearing non-sterile lab coats.

In part, due to FDA’s inspectional observations noted during the inspections, several firms have voluntarily recalled their products, and others temporarily suspended production of their products intended to be sterile while they correct deficiencies found by the investigators.

It may surprise some people to know that, even in light of the recent tragic events, some of the firms we inspect still challenge our authority to conduct full inspections of their facilities. Our inspections have been delayed or the inspectors were denied full access to records at some of these facilities. At least four of our recent inspections were delayed by such resistance; and in two other instances, we had to get administrative warrants from the courts, resulting in U.S. marshals accompanying FDA inspectors back to the firms so FDA could complete the inspection. These challenges and others highlight the need for clearer authorities for FDA to efficiently protect public health.

Additionally, these inspectional observations reveal that there continues to be reason for concern about sterility deficiencies and other problems in some compounding pharmacies across the country – problems that could potentially affect the health of patients. To carry out this proactive inspection effort, FDA had to shift resources from other areas, and this is not a sustainable approach for the longer term. 

We are committed to working with the states, industry and Congress to put the necessary protections in place, but we need to act quickly. And we will continue to work tirelessly on this issue. Patients deserve nothing less.

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

FDA’s International Food Safety Capacity-Building Plan: Improving Food Safety Protections Around the World

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By Julie Moss, Ph.D., R.D., and Katherine Bond, Sc.D.

Our increasingly globalized world means that more and more of the food we eat is imported. Congress recognized this fact of modern life by passing the FDA Food Safety Modernization Act (FSMA), landmark legislation signed into law in 2011 that shifts the emphasis from responding to food safety incidents to preventing them.

Julie Moss

In that spirit, Congress saw the importance of improving the capacity for food safety protections. It directed FDA to develop a comprehensive plan to expand the technical, scientific and regulatory food safety capacity of foreign governments and their respective food industries in countries that export foods to the United States.

FDA has met that challenge, recently releasing its International Food Safety Capacity-Building Plan, which provides a strategic framework intended to guide FDA’s capacity-building efforts over the next five years. FSMA marks the first time that Congress has charged FDA with comprehensively addressing the building of international food safety capacity, a development that has been very exciting for us.

While this mandate is new, we have successfully supported food safety capacity-building efforts and conducted training programs for many years. The agency has participated in global, multilateral food safety programs, including work with the World Health Organization, train-the-trainer programs (in areas such as good agriculture practices), various seminars and web postings, and collaborations with other U.S.government agencies, among other efforts. However, FSMA allows us to take this work to a whole new level.

For example, we are working with foreign governments to see if we can exchange more scientific and technical information, such as outbreak and inspection data. This will open up communication channels and promote collaboration with those governments. We are also working to support both the acceptance of laboratory methods across the international community as appropriate and the exchange of information on current and new laboratory methods. This helps ensure that the data obtained from different domestic and international laboratories is of good quality and can be easily compared.   

We have developed a plan that incorporates many of FSMA’s main principles, including comprehensive prevention, risk-based resource allocation, and partnering. The plan’s key goals are:

  • Goal 1: Ensure efficiency across the Foods and Veterinary Medicine Program
  • Goal 2: Increase effectiveness through evidence-based decision making
  • Goal 3: Support the exchange of information between FDA and other foreign government agencies or other entities
  • Goal 4: Enhance technical assistance and capacity-building in food safety

    Katherine Bond

Overall, the plan charts a direction for how we will prioritize our capacity-building efforts based on risk, how we will link capacity-building efforts to their impact on public health, and how we will work in partnership with counterpart authorities, industry and other organizations to achieve lasting food safety results. Through this plan, countries that export to the United States will have an opportunity to learn about our food safety capacity-building priorities and see the breadth of the efforts that we are pursuing.   

In developing this plan, we consulted with foreign governments, industry, non-governmental organizations, certain U.S.officials, academia and other stakeholders. A public meeting was held on June 19, 2012, and comments received were considered in development of the plan [Docket No. FDA-2012-N-0437].

Given the breadth of this work, it is essential that we collaborate with partners to get the work done. We realize we can’t do it alone. Partnerships are important in everything we do, and even more so with capacity building. We also recognize the importance of establishing strong relationships and mutual support among all stakeholders from farm to table. As we begin to implement this new plan, we look forward to sharing ideas and new information with our international counterparts and other interested stakeholders, leveraging resources that will help us improve global food safety.

Julie Moss, Ph.D., R.D., is Deputy Director, International Affairs Staff in FDA’s Center for Food Safety and Applied Nutrition

Katherine Bond, Sc.D., is Director, Office of Strategy, Partnerships and Analytics, in FDA’s Office of International Programs

 

FDA Must Have New Authorities to Regulate Pharmacy Compounding

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By: Margaret A. Hamburg, M.D.

The deadly outbreak of fungal meningitis associated with a compounded medication was a horrible tragedy, and I’ve asked myself many times if and how it could have been prevented. I speak for everyone at FDA when I say that our hearts go out to the many victims, including those still struggling with this devastating infection, and their loved ones.

Margaret Hamburg, M.D.While our investigation of this deadly outbreak has been a top priority, our responsibility at FDA is also to help make sure this doesn’t happen again. We are currently deploying resources to work with states to inspect certain state-licensed pharmacies that produce sterile drug products that we believe may present the highest risk. Over the past two months, we have inspected over 30 facilities and will continue to work to protect public health. 

But our authorities are limited and not the right fit for FDA to provide appropriate and efficient oversight of this growing industry. There should be legislation that establishes appropriate, minimum federal standards for firms that compound sterile drug products in advance of or without a prescription and ship them interstate.  FDA must have clear authority to proactively inspect pharmacies to determine the scope and nature of their operations.  Even during this time of heightened awareness, our inspectors are being delayed in their work or denied full access to records at some of the facilities we are inspecting.

And serious problems at compounding pharmacies continue to occur. Just this week there have been two recalls of sterile compounded and repackaged drug products. In one recall, the presence of floating particles, later identified to be a fungus, were reported in five bags of magnesium sulfate intravenous solution, resulting in a nationwide recall of all sterile drug products produced by the pharmacy. In the other recall, all sterile drug products from a second pharmacy were recalled as a result of reports that five patients were diagnosed with serious eye infections associated with the use of repackaged Avastin. 

There is a legitimate role for traditional pharmacy compounding. Every day, thousands of pharmacists practice traditional pharmacy compounding—mixing a drug in response to a valid prescription for an individual patient’s need. For example, these drug products could be liquids for patients who can’t swallow pills, or they could be made without certain allergens. These operations are licensed and primarily regulated by the states.

However, a new breed of specialty pharmacy compounding has evolved that has outgrown the law, and can pose a threat to the health of the public.  New legislation is needed that specifically addresses such compounding and the now-established safety concerns.

These pharmacies produce medications in advance of or without obtaining a prescription and distribute them across the country. They make drug products that are intended to be sterile and must be made to exacting standards to prevent dangerous contamination. The magnitude and complexity of these operations have outpaced the current patchwork of state laws that differ in prescription requirements and quality control rules.

I firmly believe that new legislation is necessary to help FDA effectively oversee firms engaged in widespread distribution of sterile compounded drug products in advance of or without receiving a prescription. To that end, FDA is working with Congress, states, industry, and all interested stakeholders to develop a basic framework to protect public health. The Senate committee with jurisdiction over this issue has been working hard on a bipartisan basis to craft such a framework, and we are hopeful that their efforts will yield strong legislation for patients across the nation.

In the new framework, FDA believes that certain high-risk sterile compounding facilities should be subject to federal oversight to ensure that the compounding of sterile drug products at those facilities can be done without putting patients at undue risk, including: 

  • requiring compliance with federal quality standards that are appropriate for the compounding of riskier products and exposure of larger numbers of patients,
  • requiring federal registration of the compounding facilities that will be subject to federal quality standards so FDA knows where they are and what drug products they are making, and
  • requiring these higher-risk compounding pharmacies to report to FDA serious adverse reactions to their drugs of which they become aware so that we can act before potential problems get out of hand. 

And for all pharmacy compounding, FDA believes certain basic protections should be in place. These include: 

  • clear authority to examine a pharmacy’s records to more quickly locate the cause of an outbreak or other violations of the law, and
  • prohibiting compounding of the most complex and highest risk products—drugs and biologics that should only be made for patients by an FDA-registered drug manufacturer under an approved new drug application. The manufacturer will have demonstrated in this application that the product is safe and effective and can be safely made according to the highest quality standards.  

FDA supports and would like to explore with Congress several other ideas, such as requiring compounded drug products to have clear label statements identifying the nature and source of the product. The labeling statements would provide prescribers and consumers with valuable information about the products they are using or taking so that they can make informed judgments about their use. Of course, funding will be necessary to support the inspections and other oversight activities outlined in this framework.  We look forward to working with Congress to explore funding mechanisms, which could include registration or other fees, as Congress has authorized and FDA has successfully implemented in other settings. 

Protecting Americans from unsafe and contaminated drugs is not just an important responsibility for FDA—it is part of our core mission. We must be able to identify dangerous practices before they result in actual harm and, when necessary, intervene to minimize the damage and prevent such tragedies in the future.

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

Keeping Up With Mobile App Innovations

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By: Christy Foreman

A smart phone that can perform an electrocardiogram (ECG)—measuring the electrical activity of a person’s heart to determine whether he or she is having a heart attack—is in my opinion an extremely smart phone. That is just one example of how mobile medical applications are transforming health care.

As we testified today before Congress, FDA has no intention of stifling innovation in this exciting and rapidly growing field. The fact is, only a fraction of mobile apps would require FDA review. However, when a mobile app is doing the job of a medical device that requires FDA clearance or approval, it’s only logical that both should be governed by the same rules. These are the small percentage of mobile apps that pose a risk of serious illness or death to patients. With these considerations in mind, FDA in coming weeks will be issuing a final guidance document that will help companies determine whether their product will require FDA clearance or approval.

They would be limited to mobile apps that meet the definition of device and are intended for use: 

  • to transform a mobile device into a medical device already regulated by FDA
  • as an accessory to a medical device already regulated by FDA

In addition to the smart phone that performs an ECG, other examples include a mobile medical app that controls the delivery of insulin; another that acts as a stethoscope; a mobile medical app that takes patient-specific information and provides a clinician with radiation dosage calculations, and mobile medical apps that allow doctors to view X-rays or other imaging on smart phones and tablets.

These examples show why FDA has a public health concern about the potential consequences of a malfunctioning mobile medical app. 

FDA’s Center for Devices and Radiological Health has been reviewing mobile medical apps for more than 10 years and in that period we have reviewed about 100 applications and each review has taken about 60 days to complete. We’re confident that the center has the expertise to continue the timely review of the small number of submissions we expect to receive from mobile app developers.

Our final guidance will be informed by some 130 public comments, most of which were overwhelmingly supportive of our risk-based, narrowly-focused approach proposed in the draft guidance. Once the guidance is released, we are confident that the public will see that it represents a careful balance between the need to encourage innovative technology with our mission of providing reasonable assurance that medical products are safe and effective.

Christy L. Foreman is Director, Office of Device Evaluation, at FDA’s Center for Devices and Radiological Health

Students Bring Energy and Good Ideas to the Table

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By: Jesse Goodman, M.D., M.P.H.

Graduate students who won a faculty-judged “America’s Got Regulatory Science Talent” competition at the University of Maryland recently filled my office with some of their ideas and energy.

Their winning ideas, and their commitment and excitement, bode well for the future of the sciences involved in ensuring that medical products are safe and effective. Perhaps above all, the problems that inspired their ideas provided powerful reminders that consumers should be engaged by health professionals and involved in their own care, including understanding the names and purposes of any medicines they are taking.

Jane Kalinina and Neha Patel, who are both pursuing degrees in law and pharmacy, saw the need for better patient awareness and understanding while working in pharmacies.

“Patients regularly called and asked, ‘What am I taking this for?’” said Kalinina. Citing a typical example, she described a patient who had been confusing her hypertension and diabetes medicines, and thus had been taking the wrong amount of each.

Such problems, they noted, are particularly problematic among the elderly, who often take multiple medicines and may see multiple doctors who may not always be aware what their colleagues have already prescribed.

Kalinina and Patel’s idea to help potentially better inform patients include the name of the condition a prescription medicine treats right on the bottle. In researching this idea, they learned that the State of California recently implemented just such an approach, with plans to evaluate potential benefits due in 2014. Kalinina and Patel plan to track this program and its evaluation and, if benefits are confirmed, hope to see the approach adopted nationally. Their idea won third place in the competition sponsored by the University of Maryland’s Center of Excellence in Regulatory Science and Innovation (M-CERSI).

Second place was awarded to five doctoral students in Pharmaceutical Health Services Research: Patience Moyo, Mehmet Burcu, Sarah Dutcher, Xinyi Ng and Dinci Pennap. Their proposal was aimed at reducing the risk of harmful drug interactions through enhancing links among doctors, health systems and pharmacists, so that pharmacists have access to complete records of medicines being prescribed.

First place went to Curtis Gallagher, a doctoral student in molecular medicine at the University of Maryland, Baltimore, who researched “untapped opportunities” to facilitate reporting and monitoring of potential drug side effects and other unanticipated problems.

He noted that about 150 million Americans take at least one prescription. Based on personal experience and a 100-person survey, he found most people did not know about MedWatch, the FDA program where negative events can be reported and are tracked for trends, or found reporting too time consuming. His suggestion was for FDA to develop easier-to-use pull down menus of potential side effects, based on a drug’s FDA labeling, and allow consumers to file through FDA or through other websites consumers typically find when doing a computer search for information on a drug or condition. His ideas are being discussed at FDA.

As our health care system works to develop and refine many new approaches to enhancing the safe use of medications, this advice to consumers remains critical: know what you are taking, and why.

Jesse L. Goodman, M.D., M.P.H., is FDA’s chief scientist. In addition to being responsible for FDA’s cross-cutting scientific and public health efforts, he also provides strategic leadership for FDA’s regulatory science and innovation initiatives.

A Key FDA Resource for Industry and the Public: Working with the FDA Office of the Ombudsman

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By: Andrew Moss and Laurie Lenkel

Like many Federal agencies, FDA has a robust ombudsman program that addresses concerns and complaints from regulated industry and the public. At FDA, most product evaluation centers house their own ombudsman staff that address center specific issues. The FDA Office of the Ombudsman, as part of the Office of the Commissioner, provides this function for the agency as a whole.   

Click Image to Download this brochure (PDF 1021 KB)

While some think of an ombudsman as a type of court of last resort or legal adviser, the FDA Office of the Ombudsman rather acts primarily as a counselor or informal mediator. An ombudsman may be called upon by interested parties to provide guidance and assistance at any stage in a dispute, complaint, or other problem that relates to the work of the agency—not only when a matter reaches an impasse. Addressing problems early can often aid in their resolution.

The FDA Office of the Ombudsman employs some basic guiding principles that allow it to serve in this mediating role: 

  • Neutrality—we engage in matters free from bias and independently from the agency components involved; 
  • Transparency—we strive to be as clear and open as possible about the steps we are taking to provide assistance and about what we can and cannot do to help; 
  • Confidentiality— we maintain the confidentiality of all information provided consistent with applicable laws and regulations.

The FDA Office of the Ombudsman handles inquiries about the resolution of consumer complaints as well as inquiries from regulated industry regarding, among other things, agency action or delays in action, compliance activities, import issues, and actions of FDA field offices. We play an important role in assisting small businesses. Although small businesses are generally subject to the same regulations as any other entity, we can help to draw attention to the special needs and concerns of these companies. If nothing else, we can help small businesses to understand messages from the agency and to better communicate with FDA offices and staff, thereby helping companies to satisfy FDA requirements, which are designed to protect consumers and patients.

The tools we use to assist individuals and companies vary from situation to situation.  Sometimes we help them to better understand actions taken by FDA. At other times we can be helpful in calling attention to and moving forward action that has been delayed.  In many instances, we are able to facilitate a productive meeting between key FDA officials and the interested party to discuss and help move toward resolution of issues of concern. 

A new role for the FDA Office of the Ombudsman is in shepherding the consideration of scientific disputes raised by FDA employees that are not resolved elsewhere and rise to the level of the Commissioner, the head of the FDA. While there are often multiple legitimate ways to view different findings and to make regulatory or policy decisions, FDA is committed to the integrity of the underlying science and a science-based approach to its decision making, and places great value on ensuring that divergent scientific opinions are fully and fairly heard. Any FDA scientist can first raise a scientific dispute or disagreement within the center where they work through well defined processes, with the final arbiter being the Director of that FDA center.  However, if they are ultimately not satisfied that their views have been fully heard and considered by the Center, they can bring the matter to the Office of the Commissioner via the FDA Office of the Ombudsman for review of the process.    

Whatever the issue, question, or problem, the FDA Office of the Ombudsman stands ready to provide guidance and assistance. We are here to help. And if we can’t directly assist you in a given matter, we will identify the FDA component that can. You can contact us anytime at ombuds@oc.fda.gov, or visit us on the web. 

Laurie Lenkel is Ombudsman and Andrew Moss is Deputy Ombudsman in FDA’s Office of the Commissioner

Public Meeting on Food Safety Highlights First Two Proposed Rules

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By: Michael R. Taylor

Last week, we kicked off a series of public meetings—with about 400 people participating—to stimulate dialogue and gain input on FDA’s first two proposed rules to implement the FDA Food Safety Modernization Act. In her opening remarks, Dr. Hamburg noted how her appointment as FDA Commissioner by President Obama, coming on the heels of the illnesses and deaths attributed to contaminated peanut products, helped make food safety a high priority for her and the Administration.

Michael R. Taylor, J.D.It was fitting that we held our first meeting in a USDA auditorium in Washington, D.C., because we have worked closely with USDA and with other government partners at the state and local level, as well as with industry and consumer groups, to get the rules right.  That means holding a lot of meetings to explain the proposed rules, visiting farms and facilities, and opening dockets for comments from interested parties, to receive as much input from the public as possible before we finalize the rules.

The diversity of those who signed up to make public comments at the meeting shows how important food safety is to all of us, from victims of foodborne illness to farmers and food manufacturers. First up was 15-year-old Dana Dziadul, who was three years old when she became ill from Salmonella in cantaloupe. The compelling stories from Dana and other victims of foodborne illness, and their families, remind us all that first and foremost, we have a public health obligation to address the harsh reality that each year, 3,000 people die and more than 100,000 are hospitalized because of foodborne illness. Our first obligation is to do everything we can to reduce these numbers.

We also know that improving food safety helps avoid the disruptions in the marketplace that result from illness outbreaks and recalls of food and maintain consumer confidence in healthful foods, such as the produce that the proposed rules cover. This convergence of interests has made our current food safety initiatives a real community effort, with industry, consumers and government coming together around the common goal of modernizing our food safety system to better prevent problems. We all have a responsibility, and we all have a role, in making and keeping food safe.

The work ahead may sound simple—having the right standards in place that are based on the best information we have on how to prevent hazards, and making sure these standards are met. But we have much yet to accomplish. We have to make sure the standards are flexible enough to address the diversity of operations across the country—from small farms to large facilities, from Maine to California. We also need to be sure FDA is ready to operate under this new, prevention-oriented framework, which will require employee training and ensuring our resources are focused on the greatest risks. And we are committed to providing technical assistance so that industry, especially small operations, can meet the new requirements when they are finalized.

These two proposed rules we have announced are very important, but we have three additional rules coming to further form our food safety framework. To address the safety of imports, we will soon be proposing a rule that requires that importers based in the United States verify that their overseas supplies are following prevention-based standards that provide the same level of public health protection as those that are in place here. Also addressing imports is a proposed rule on the accreditation of the third party auditors that industry uses to help them determine if food safety standards are being met. The third proposed rule coming addresses preventive controls for animal food, including pet food.  Preventive controls are steps that firms must put in place to control hazards such as microbial contamination that could occur in a food facility.

We will be holding two more public meetings—one in Chicago and one in Portland, Oregon. You can find more information on our web site at fda.gov/fsma. Six meetings in various states across the country also are scheduled. Your comments are important to us, and I encourage you to submit comments at regulations.gov before the May 16th deadline. 

The road ahead is a long one, and it will take years, and sustained commitment, to have our framework fully in place. But, for America’s families and our vibrant food system, the hard work is absolutely worth it!

Michael R. Taylor is FDA’s Deputy Commissioner for Foods and Veterinary Medicine

 

Rare Disorders Without Borders: An International Strategy

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By: Katherine Needleman, Ph.D.

The development of 200 new therapies for rare diseases and diagnostic tests for most rare diseases would alleviate untold suffering. So with great enthusiasm, FDA’s Office of Orphan Products Development (OOPD) has joined a global effort to make those goals a reality by 2020.

These are the goals of the International Rare Disease Research Consortium (IRDiRC),  launched by the European Commission and the U.S. National Institutes of Health in April 2011 to foster collaboration in rare disease research. FDA’s OOPD recently joined IRDiRC’s Executive Committee. When the consortium holds its first major conference in Dublin in April, top experts and researchers from around the world will share information and foster collaborations.

Collaboration is critical. Rare disease resources, including experts, are so limited that we cannot afford to waste a single moment by duplicating work or failing to share promising developments. Products for rare diseases are often called “orphan products” because they generally lack sponsors to develop them.

Some rare diseases affect only a few hundred people, and by FDA’s definition, each one affects no more than 200,000 people in the U.S. Yet taken together, about 7,000 rare diseases afflict about 30 million Americans and a similar number in Europe. In much of the world, they go undiagnosed. Often, they are debilitating or deadly. Many are genetic, so children are often victims.

Today, on Rare Disease Day 2013, the international rare disease community comes together with the theme “Rare Disorders Without Borders,” and FDA continues its commitment to close collaboration with international counterparts, such as the European Medicines Agency.  

FDA is the first regulatory agency to join the consortium’s Executive Committee. In addition, FDA is represented on the consortium’s Therapies Scientific Committee by Marc Walton, M.D., Ph.D., in FDA’s Center for Drug Evaluation and Research. We hope to bring regulatory scientific perspectives that help develop great research ideas into great products that meet regulatory approval standards for safety and effectiveness.

FDA has been working to change the orphan status of rare diseases since 1983, with the passage of the Orphan Drug Act. Since then, FDA has approved over 400 products for the treatment of rare diseases, compared to only 10 developed by industry in the decade before the act.

OOPD’s grant program uses its annual budget of approximately $14 million to fund top scored clinical trials of rare disease treatments. The grants program has been successful, as at least 49 OOPD grants supported clinical trials that contributed to agency approval of these products for rare diseases.

Still, for most rare diseases, there are no treatments, or definitive diagnostic tests.

Recent progress in human genomics and newly emerging sciences increase the prospect for treatments or even cures. A new generation of therapies even shows promise for changing the defective genes that result in some rare diseases. These are among the topics that will be addressed at the upcoming conference.

Our OOPD Director, Gayatri Rao, M.D., J.D., will present FDA’s perspective at the IRDiRC meeting while I will be working on the challenges ahead through the Executive Committee as FDA helps ensure that the energy and expertise brought to the conference is harnessed to bring results to patients.  

The need is great; the time is now.

Katherine Needleman, Ph.D., is director of the Orphan Products Grants Program of FDA’s Office of Orphan Products Development.