Advancing Tobacco Regulation to Protect Children and Families: Updates and New Initiatives from the FDA on the Anniversary of the Tobacco Control Act and FDA’s Comprehensive Plan for Nicotine

By: Scott Gottlieb, M.D., and Mitch Zeller, J.D.

This summer marks nine years since the Family Smoking Prevention and Tobacco Control Act (TCA) was signed into law, and one year since we announced the FDA’s Comprehensive Plan for Tobacco and Nicotine Regulation. This comprehensive plan places nicotine, and the issue of addiction, at the center of the agency’s tobacco regulation efforts. The multi-year roadmap provides a framework for regulating nicotine and tobacco and is designed to reframe the conversation around nicotine and harm reduction.

A principal reason people continue to smoke cigarettes — despite the dangers — is nicotine. Our plan recognizes that nicotine isn’t directly responsible for the morbidity and mortality from tobacco, but creates and sustains addiction to cigarettes. It’s the delivery mechanism for nicotine that’s more directly linked to the product’s dangers. That’s why our plan focuses on minimizing addiction to the most harmful products while encouraging innovation in those products that could provide adult smokers access to nicotine without the harmful consequences of combustion and cigarettes.

Dr. Scott Gottlieb, Commissioner of the U.S. Food and Drug Administration

Scott Gottlieb, M.D., Commissioner of the U.S. Food and Drug Administration

Over the past year, we’ve taken important steps toward fully implementing this plan as part of our overarching goal: a world where cigarettes can no longer create or sustain addiction, and where adults who still seek nicotine could get it from potentially less harmful sources. In implementing this comprehensive plan, we’ve already issued three important advance notices of proposed rulemaking (ANPRMs) that have the potential to reframe the tobacco landscape. These ANPRMs focus on:

  • The potential development of a product standard to lower nicotine in cigarettes to minimally or non-addictive levels – which could make it harder for future generations to become addicted in the first place and could allow more currently addicted smokers to quit more easily or switch to potentially less harmful products. Given their combination of toxicity, addictiveness, prevalence and effect on non-users, it’s clear that to maximize the possible public health benefits of our regulatory tools granted to us under the Tobacco Control Act, we must focus our efforts on the death and disease caused by addiction to combustible cigarettes. We believe this pivotal public health step has the potential to dramatically reduce smoking rates and save millions of lives;
  • The role that flavors – including menthol – play in initiation, use and cessation of tobacco products. Input on these issues will assist in the consideration of the most impactful regulatory options the FDA could pursue to achieve the greatest public health benefit. We’re proceeding in a science-based fashion, building a strong administrative record by securing more information about the potential positives and negatives of flavors in both youth initiation and in getting adult smokers to quit or transition to potentially less harmful products; and,
  • The patterns of use and resulting public health impacts from what are often referred to as “premium” cigars to inform the agency’s regulatory policies.

The public comment periods for all three ANPRMs, which were extended by 30 additional days to allow more time for submissions, have now closed. We are beginning the process of reviewing those comments.

Mitch Zeller, J.D., Director of FDA's Center for Tobacco Products

Mitchell Zeller, J.D., Director of FDA’s Center for Tobacco Products

At the same time, the FDA is also pursuing additional new policies as part of our comprehensive plan as well as our ongoing commitment to improve the efficiency and effectiveness of our tobacco regulatory programs.

Part of these efforts are aimed at making the pathway for developing nicotine replacement therapy (NRT) products more efficient to promote the development of novel NRT products. The agency’s efforts to re-evaluate and modernize its approach to the development and regulation of NRT products is aimed at opening up new pathways for the development of improved products, regulated as new drugs, that demonstrate that they are safe and effective for the purpose of helping smokers quit.

Many of our new efforts, as part of our comprehensive plan, are aimed at using our existing authorities under the TCA to minimize addiction to the most harmful products, principally cigarettes, while encouraging innovation in new products that may offer adults less harmful forms of nicotine delivery.

A key part of achieving these goals is issuing foundational rules and guidances to help industry better understand what is needed to submit product applications. At the same time, we are pursuing new efforts to improve the transparency and efficiency of the premarket review process.

These important foundational steps are a key element of our efforts to advance the pre-market review of tobacco products. This review is one of the most important responsibilities we have. It’s how we can assess new products and their potential impact on the public health. This is our opportunity to determine how a product may positively or negatively affect both non-users and current users. So, it’s crucial that we continually improve in this area and have a transparent and efficient process.

To address these goals, we’re committing to a number of steps, some new, to respond to stakeholders and to make the regulatory process more efficient, predictable, and transparent for industry, while also advancing the agency’s public health mission. Establishing a rigorous, predictable, science-based framework for the premarket review of tobacco products is a key element of our program.

Among the steps that we are pursuing to better achieve these goals:

  • Proposing foundational rules: We all need to be on the same page regarding the basic “rules of the road,” especially when it comes to what’s expected in premarket applications. We’re working to propose new rules to help industry on topics including Substantial Equivalence, Premarket Tobacco Applications, Modified Risk Tobacco Product Applications, and Tobacco Product Manufacturing Practices. We will begin publishing these foundational proposed rules in the coming months. They will lay out a transparent, modern, and science-based framework for manufacturing practices and the development of tobacco product applications that meet the legal requirements.
  • Holding a public meeting on premarket review: Within the next few months, the FDA will hold a public meeting on the premarket application and review process. The goal of the meeting is to solicit comments on our processes and provide a dedicated venue for specific suggestions on how to further improve them. Potential topics for discussion include: how to achieve greater efficiencies in review, while continuing to protect public health; how to review products that are rendered “new” due to changes made to comply with a product standard; and, how to facilitate greater company consultation with the FDA prior to submitting applications.
  • Exploring opportunities for premarket review efficiencies through rulemaking and guidance and new administrative steps to modernize and improve the review process: The FDA is taking additional steps to pursue the shared interest with industry of increased flexibility and efficiencies within the application review process. If carefully developed, rulemaking and guidance efforts in this area could help ensure that our public health standards for premarket review are met while mutually benefiting both the industry and the FDA. For example, an opportunity may exist to allow for faster and cheaper development of products that will benefit public health. In the months ahead, the FDA intends to explore what improvements can be made along these lines within our existing legal authorities. We also plan to advance a comprehensive suite of improvements to the review process, as part of a Regulatory Modernization, to make our program more efficient, transparent, predictable, and efficient. We will unveil these programmatic reforms in advance of our upcoming public meeting.

These programmatic and process improvements are aimed at solidifying the FDA’s regulatory pathways and improving its predictability and transparency. As the FDA advances its regulatory approach to these important public health considerations, it’s critical that we keep in mind a bedrock principle: No kids should be using any tobacco or nicotine-containing products, including e-cigarettes.

Protecting our nation’s youth from the dangers of tobacco products is among the most important responsibilities of the FDA. That is why we recently launched our Youth Tobacco Prevention Plan.

We look at the marketplace for tobacco products today and see increasing concern from parents, educators, and health professionals about the alarming youth use of tobacco products like JUUL and other e-cigarettes. Our mission at the FDA is to protect the public’s health, and we want to assure the public we’re using all of our tools and authorities to quickly tackle this public health threat. We will not allow our efforts to give manufacturers time to file premarket applications with FDA — that are informed by the foundational rules and guidance that we’re now advancing — to become a back door for allowing products with high levels of nicotine to cause a new generation of kids to get addicted to nicotine and hooked on tobacco products.

Our Youth Tobacco Prevention Plan reflects our commitment to address these risks. Congress gave us many powerful authorities, including enforcement, product standards, premarket review, sales and promotion restrictions, and public education. We’ll use every tool available to protect our nation’s kids.

We’ve already announced several vigorous enforcement actions and education efforts aimed at addressing youth use of nicotine, and e-cigarettes in particular. More such actions are imminent. Among the steps we’ve already taken are: sending warning letters to companies for selling e-liquids resembling juice boxes, candies and cookies; sending warning letters to retailers for selling JUUL e-cigarettes to underage youth; working with eBay to remove Internet listings for JUUL, and with other e-cigarette manufacturers to help the FDA better understand the youth appeal of these products; and, creating the  first e-cigarette public education ad, with a full-scale advertising campaign to begin this fall.

These are important first steps. But we still need to do more to address use of tobacco products by kids. That’s why we’re working to quickly advance the following three new initiatives:

  • Expediting action on flavors: The issue of flavors, including flavored e-cigarettes and e-liquids, is at the forefront of any discussion of youth use. However, some flavored tobacco products may also play a role in helping some adults quit smoking cigarettes. Now that the comment period has closed, we intend to expedite the review and analysis of the comments so that we can leverage the information into policy as quickly as possible, should the science support further action.
  • Developing an e-cigarette product standard: The FDA has also begun exploring a product standard for e-cigarettes to help address existing concerns. As part of the standard, the agency will consider, among other things, levels of toxicants and impurities in propylene glycol, glycerin, and nicotine in e-liquids. While the process for establishing a product standard takes time, we recognize the urgency in setting some minimum, common sense standards, and will work to address this on an accelerated timeline.
  • Exploring ways to accelerate enforcement: We’re also looking at ways that the FDA can act even more efficiently when we become aware of violations affecting youth use of e-cigarettes, such as illegal product marketing to youth. We need to be faster and more agile when we identify new risks. We’ve also become aware of reports that some companies may be marketing new products that were introduced after the FDA’s compliance period and have not gone through premarket review. These products are being marketed both in violation of the law and outside of the FDA’s announced compliance policies. We take these reports very seriously. Companies should know that the FDA is watching and we will take swift action wherever appropriate. We are evaluating new ways to strengthen our partnership with sister agencies, including the Federal Trade Commission. We will also announce a robust series of additional enforcement actions in the coming months.

We have made great strides since we first unveiled our Comprehensive Plan for Tobacco and Nicotine Regulation last year. The components of this program we have outlined are intended to work as an integrated package of reform. We will pursue all of these policies simultaneously. Each is interconnected. Each element supports the others. We intend to achieve all of the elements.

We remain on track to meet our ambitious goals. But there is still much work to be done.

The staff at the FDA’s Center for Tobacco Products is working hard to use our available tools to protect Americans from the harms of being addicted to tobacco products. And today, we’re committing to redoubling our efforts. Too many kids are still starting to use tobacco products and getting addicted. And, too many adults are still struggling to quit or to switch to less harmful options. To reduce the disease and death caused by tobacco use, the FDA will do everything within our power to help all ages.

Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration 

Mitch Zeller, J.D., is Director of the FDA’s Center for Tobacco Products

 Follow Commissioner Gottlieb on Twitter @SGottliebFDA

Follow the FDA Center for Tobacco Products on Twitter  https://twitter.com/FDATobacco

 

Advancing Policies to Promote Safe, Effective MedTech Innovation

By: Scott Gottlieb, M.D.

Advanced technologies, biomaterials and digital tools are offering us fundamentally better ways to develop new medical devices to advance human health. To build on these innovations and ensure patients have access to safe and effective advances in medical care, it’s crucial that FDA modernize its policy framework for the review of medical devices. We must provide more opportunity to enhance traditional products with new advances, and to use these same innovations to develop fundamentally novel devices altogether. This requires FDA to implement reforms that make the review process more benefit-risk based; allow us to efficiently adopt more modern tools for evaluating safety and benefits of new products; and to actively embed in our review patient-centric measures of risk and benefit.

Dr. Scott Gottlieb

Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration

We must do this all while taking steps to strengthen FDA’s gold standard for safety and effectiveness.

FDA already has taken steps to modernize its review pathways in recent years as device technologies have advanced. But there’s more we must do.  Early in the coming year, FDA’s Center for Devices and Radiological Health (CDRH) intends to advance several important new regulatory policy initiatives to further modernize the medical devices program and continue to foster new medtech innovation.

Background on the Current Framework

The Medical Device Amendments of  1976 set forth that manufacturers of new, moderate-risk devices – generally Class II and certain Class I devices – must demonstrate substantial equivalence to a similar device legally marketed in the U.S. in order for a new device to be cleared by FDA for use in patient care.  Under this construct, older devices that function as the comparators are referred to as predicate devices. This process is generally known as the 510(k) pathway. It’s designed to assess the safety and effectiveness of medical devices whose risks are well understood.

FDA’s regulatory process has remained largely unchanged since it was first implemented 40 years ago. As a consequence, there are an increasing number of cases where this basic framework isn’t well-suited to reflect the innovation that we see today in certain technologies, and how we must evaluate those technologies..

FDA’s existing framework also fails to realize the full potential of the FDA’s consensus standards program, which was established through the Food and Drug Administration Modernization Act of 1997, and will be refined and expanded as a result of provisions in the 21st Century Cures Act of 2016.

Through this program, CDRH has already recognized more than 1,200 national and international standards wholly or in part. CDRH typically incorporates these standards in guidance documents that provide recommendations on testing and performance standards. These guidance documents provide innovators with predictable, consistent, and least burdensome approaches for demonstrating that a new technology is safe, effective, and functions properly for its intended use.

Yet despite manufacturers being able to demonstrate that they meet these consensus standards, a manufacturer is required to demonstrate that a device is substantially equivalent to an existing, legally marketed device. But the predicate devices that new products must compare themselves to are sometimes 40 years old. FDA recognizes that such direct comparison testing creates burdens for 510(k) applicants, especially when many new devices are designed in novel ways, using more advanced technologies. It’s sometimes hard to identify sufficient, appropriate predicate devices in order to conduct testing. This can create an obstacle to certain kinds of innovation and lead to inefficiency in the review process with few, if any, benefits to patient safety. In fact, at times, it can make it less efficient for FDA to assure the safety of the device.

New Steps to Reform, Modernize 510(k) Review

To address these challenges, in the first quarter of 2018, FDA intends to publish a draft guidance outlining a voluntary, alternative pathway for demonstrating substantial equivalence. This pathway will allow more flexibility to use more modern criteria as the reference standard, and permit comparisons to standards that more closely approximate the kind of novel technology we’re being asked to evaluate.

Under this new framework, device manufacturers could demonstrate substantial equivalence by meeting objective safety and performance criteria. These can include FDA-recognized standards, FDA-developed guidance documents, or a combination of the two, which embody the safety and performance criteria that new devices could meet to be cleared under a 510(k).  This pathway would be available for pre-specified categories of mature devices – those for which safety and performance criteria that meet or exceed the performance of existing, legally-marketed devices can be identified. It will be outlined in new draft guidance that we are announcing today in the Federal Register and plan to issue in the first quarter of 2018. This approach will also make it easier for FDA to conform its framework for evaluating new products to international consensus standards where such standards exist.

The program will be voluntary, and manufacturers could continue to utilize the existing 510(k) pathways.  It would apply only to devices for which 510(k) clearance is the appropriate pathway to market and would not affect the scope of the current De Novo program for more novel devices that do not have a predicate. But imagine the benefits of a more efficient and transparent pathway for bringing to market well-understood technologies like ultrasound imaging machines, common in vitro diagnostic devices, and blood pressure monitors.

This regulatory innovation holds tremendous promise to further streamline device review for sponsors and FDA and allow new innovations to get to patients more quickly; to allow more advanced technologies to be efficiently incorporated into new devices; and to foster greater confidence in the FDA’s ability to efficiently evaluate safety and benefits of technologies cleared under this pathway – all while maintaining the same gold standard that we apply to existing review processes. By modernizing the benchmarks by which we evaluate the performance characteristics of cleared devices, we believe it will also enable us to put in place more modern criteria for assessing safety.

New Framework to Balance Pre- vs. Post-Market Requirements

For all medical devices that are subject to premarket review – whether through a premarket notification, premarket approval application (PMA), humanitarian device exemption application (HDE), or other type of marketing submission – FDA must determine whether probable benefits to health from use of the device outweigh any probable risk of injury or illness from its use.  As part of that analysis, FDA considers what level of uncertainty is acceptable about the product’s probable benefits and risks before the device is available to the public. To fully discharge any uncertainty, FDA requires post-market follow-up studies. These studies are a way to fully elucidate the long-term performance of new medical products.

But the balance between acceptable uncertainty in the pre-market setting, relative to a product’s benefits and potential risks – and, in turn, how much reliance FDA can place on post-market follow-up studies – has never been objectively defined in one, comprehensive policy framework.

FDA intends to publish a draft guidance, again in early 2018, setting forth factors that the agency may consider when assessing acceptable uncertainty. The guidance will outline how certain issues could be ultimately resolved in the post-market setting, rather than the pre-market setting, to allow patients to gain faster access to potentially life-saving devices, when appropriate. Under this form of more progressive review, FDA will outline how it makes judgments about when it’s appropriate to place greater reliance on post-market data in order to facilitate access to certain innovation, or when the agency needs to rely more on pre-market data collection because of certain issues related to a particular product or how it may be used.

This approach will be outlined in a new draft guidance that relates to the acceptable levels of uncertainty that FDA can accommodate in the pre-market setting relative to the public health benefits, and in turn rely on post-market data to answer certain questions. This balance is already a feature of product review across all of FDA’s programs. The new guidance is an attempt to make these considerations more transparent, consistent, and objectively-defined.

To further assess the impact of pre-market uncertainty, FDA may consider several factors.  One is the extent of the public health need. Factors that may influence these considerations are the seriousness of the illness that the device will treat or diagnose, the size of the population that could benefit from a new innovation, and the benefit-risk profile of alternative therapies or diagnostics (if any exist). Another factor is how likely uncertainty can be resolved pre-market versus the post-market.  FDA might accept greater uncertainty for a device where gathering extensive clinical evidence pre-market would not be feasible given the small patient population that the device is intended to treat.  The likelihood that uncertainty can be resolved by collecting data post-market, such as through the use of registries, is another consideration. FDA must have confidence that it’ll be able to acquire required post-market data.

This approach to evaluating uncertainty could be used in any one of the existing pathways that developers must follow to market new devices – 510(k), De Novo, PMA, or HDE.  That’s because these factors should guide our decisions about patient access to medical devices in any of the regulatory classifications. We’ll use our existing authorities to require more post-market data collection if we determine that’s the best way to resolve appropriate pre-market uncertainty for a specific device.  And in all cases when this approach is taken, we’ll uphold the same standards for safety and effectiveness.

Looking Ahead to Other Device Policy in 2018

Although I’ve highlighted only two upcoming guidance documents related to FDA’s medical device review program, additional important guidance is forthcoming over the next several months, as outlined in FDA’s list of pending device-related guidance documents being published today.  FDA publishes this list annually to provide transparency about our medical device regulatory priorities in the upcoming fiscal year. Several of the additional planned guidance documents, including the draft guidance documents regarding application of least burdensome and the Q-Submission Program, further demonstrate FDA’s commitment to advancing modern regulatory policies that are designed to promote medical device innovation, while continuing to assure the safety and effectiveness of new products.

Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

FDA Takes Important Steps to Stem the Tide of Opioid Misuse and Abuse

By Scott Gottlieb, M.D.

 America is awash in immediate-release (IR) opioids. About 90 percent of all opioid pain medications prescribed – or 160 million prescriptions a year – are for IR formulations like hydrocodone and acetaminophen or oxycodone and acetaminophen combinations. Many people who are currently addicted to opioids became medically addicted. Their first exposure to opioids was from a legal prescription, and for many, that prescription was written for an IR formulation of these drugs. Many addicted patients may then move on to higher dose formulations or more accessible illegal street drugs.

At FDA, we believe it’s necessary to continue to take steps to address both ends of this continuum, the potential gateway to addiction that is often the IR formulations, and the higher dose, extended-release formulations, both of which carry a significant risk of overdose and mortality. We are taking several actions to address these challenges. This week, we issued letters notifying 74 manufacturers of IR opioid analgesics intended for use in the outpatient setting that their drugs will now be subject to a more stringent set of requirements under a Risk Evaluation and Mitigation Strategy (REMS). The REMS requires that training be made available to health care providers who prescribe IR opioids, including training on safe prescribing practices and consideration of non-opioid alternatives.

Dr. Scott GottliebFDA also will soon issue a final guidance document that will assist potential applicants who plan to develop, and submit to FDA, an application to seek approval of a generic version of abuse-deterrent formulations (ADFs) of opioid drugs. Most of the currently approved opioids with labeling describing abuse-deterrent properties are extended release/long-acting (ER/LA) formulations of opioids. These drugs are generally formulated to be more resistant to the sort of manipulation that would otherwise make them amenable to snorting and/or injecting. Addicted patients who start by using the IR drugs will sometimes migrate onto the ER/LA formulations, and then try to manipulate those higher-dose formulations in ways that can provide a more immediate “high” through injection or snorting. But there are currently only brand name ADF formulations. These steps that FDA is taking are aimed at addressing each end of the spectrum of abuse and addiction.

With respect to the new REMS measures to address the safer use of the IR opioid pain medications, these short-acting drugs will now be subject to the same regulatory requirements as the ER/LA opioid analgesic formulations. Since 2012, manufacturers of ER/LA opioid analgesics have been subject to a REMS, which requires, as its primary component, that training be made available to prescribers of those products. To meet this requirement, the sponsors of the ER/LA opioid analgesics have been providing unrestricted grants to accredited continuing education providers for the development of education courses for health care professionals based on content outlined by FDA, which the agency calls the “Blueprint.” FDA is now extending these REMS requirements to the IR manufacturers.

While some of the ER/LA manufacturers also make IR opioids, today’s action will greatly expand the number of products covered by the REMS. The existing REMS currently includes 64 ER/LA opioid analgesic products. Once the action is finalized, an additional 277 IR opioid analgesics will be subject to these REMS requirements.

In addition to expanding the REMS to include IR products, FDA is modifying the content of the educational “Blueprint” required under the REMS. The agency is adding content on pain management, including non-opioid alternatives. This includes principles related to the acute and chronic pain management; non-pharmacologic treatments for pain; and pharmacologic treatments for pain (both non-opioid analgesic and opioid analgesic). The revised Blueprint will also cover information about the safe use of opioids, and basic information about addiction medicine and opioid use disorders.

For the first time, this training will also be made available to other health care professionals who are involved in the management of patients with pain, including nurses and pharmacists, which is in addition to prescribers of opioid analgesics. FDA believes that all health care professionals involved in the management of patients with pain should be educated about the safe use of opioids so that when they write or dispense a prescription for an opioid analgesic, or monitor patients receiving an opioid analgesic, they can help ensure that the product is properly indicated for the patient and used under appropriate clinical care.

FDA’s new Opioid Policy Steering Committee is also considering whether there are circumstances when FDA should require some form of mandatory education for health care professionals, and how the agency would pursue such a goal. The agency’s purpose is to reduce overall exposure to opioids by making certain that prescribing doctors are properly informed about appropriate prescribing recommendations, that providers understand how to identify the risk of abuse in individual patients, and know how to get addicted patients into treatment. In fact, today, the agency issued a public notice to solicit input on a detailed series of questions related to these goals. FDA has also been scheduling meetings with provider organizations and sponsors engaged in dispensing drugs – including health systems and pharmacy chains, in an effort to solicit additional input on new strategies.

Sending out the manufacturer notification letters is the first step in extending the REMS to the IR drugs. This process could take about a year to finalize. The modified REMS will continue to include a requirement for patient Medication Guides, patient-counseling documents, and plans for assessing the effectiveness of the revised REMS. The crisis of opioid addiction is a public health tragedy of enormous proportions. By putting in place safety measures for IR opioid analgesics, and creating a more robust path to converting the high dose opioids to formulations that are more resistant to manipulation, we are addressing both ends of this crisis. Our hope is that we can help prevent new patients from becoming addicted, and keep some individuals from experiencing the serious adverse effects associated with these medications.

Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

Patient Reps – Bringing the Voice of Patients to FDA

By: Jack Kalavritinos

At FDA we never lose sight of the fact that the work we do in evaluating and approving new medical products is done to benefit patients.

Increasingly, that means taking into account the views and expertise of patients and their caregivers, because they provide a unique voice and perspective and know best what they are living with on a day-to-day basis. Earlier this month, for instance, we announced the creation of the first advisory committee made up solely of patients and caregivers, who will provide advice on complex issues related to medical devices.

Another way we incorporate the patient viewpoint is through FDA’s Patient Representative Program. This program brings patients – and their caregivers – and the extraordinary breadth of knowledge and personal experience in more than 300 diseases and conditions they possess, directly into the regulatory medical product development and review process. They serve on 47 FDA Advisory Committees and panels to advise on drugs, devices and biologics currently being considered for approval or clearance. They also serve as a consultant for the review divisions (doctors and scientists who review data to determine whether the medical product’s benefits outweigh the potential risks), and as presenters at FDA meetings and workshops on disease-specific or regulatory and health policy issues.

Jack Kalavritinos , FDA’s Associate Commissioner for External Affairs

Members of FDA’s Patient Representative Program together with Jack Kalavritinos, Associate Commissioner for External Affairs, during a recent training session in suburban Washington, D.C.

Every year our team at FDA’s Office of Health and Constituent Affairs brings new FDA patient representatives to the Washington, D.C. area, for training and orientation. They receive briefings on everything from medical product review policies and clinical trials to the life cycles of drugs, biologics, and devices, and even a brief primer on statistical analysis.

Without a doubt, the most moving moments of the recently completed two-day workshop  were when the patient representatives told their own stories, how they are making a difference in so many lives as advocates for their own patient communities, and explaining why they had decided to make the commitment to become patient reps. One was more compelling than the next. Here are some of their stories:

  • A woman with fibromyalgia who said she joined the program “to give a voice to those who suffer with chronic, life-altering conditions.”  She believes her training in qualitative research, combined with her personal experience with her disease, will help her listen to and understand patient experiences and to “sort out the ‘noise.’”
  •  A caregiver whose husband survived for three years after being diagnosed with glioblastoma multiforme, a form of brain cancer. During that period, he received four surgeries and took 15 different chemotherapy drugs. She became a patient advocate as a result of her experience so she could be involved in the process of finding new treatments for “this daunting disease.” She also works with other caregivers to help them cope with the diagnosis. As she explained, “Being able to talk to someone who has experienced this same disease helps to reduce their level of anxiety and some of the unknowns that accompany this diagnosis.”
  • A patient with schizoaffective disorder who talked about how her disease first appeared when she was in college, when the voices she would hear interfered with her ability to learn and function. But with proper treatment, and incredible discipline and support, she was able to learn to control her disease and not let it take over her life. Today, she is a mental health therapist who works to combat stereotypes that prevent psychiatric patients from getting the help they need, when they need it. Her goal has been “to put a face to those of us who struggle with psychosis, but yet are seen as being ‘functionally well.’”
  • A father and uncle to two women with Friedreich’s ataxia, a life-shortening genetic mitochondrial nerve disorder that has no treatment. He has watched as his daughter, now 31, has become a quadriplegic. As he said with incredible honesty and pain, “she will die soon.” But he also has turned his pain into action. In addition to assisting his daughter and niece, he also has lent his energy to helping others with the disease and to generating attention and resources for finding a treatment. He became a patient representative, in part, he explained, so that he “could be at FDA on the day a treatment needs his yes or no vote.”

Space prevents including every one of their stories, but each of these remarkable individuals offered a compelling history of courage. All are committed to fighting the disease that had so directly affected them, whether as a patient or a caregiver. But, in a comment that could be applied to all of them, one woman noted, she “works hard to not let my identity be defined by my illness.” They do this, remarkably, by turning their focus outward, rather than inward, and using their strength and expertise to the benefit of others.

This understated, but courageous, spirit is echoed in one way or another by each of FDA’s patient representatives. We want to thank each of these individuals for their inspiring commitment — to the FDA, to better health, and for their role in these critical public health efforts.

Jack Kalavritinos is FDA’s Associate Commissioner for External Affairs

Working Together to Reduce the Devastating Effects of Opioid Misuse

By Robert M. Califf, M.D.

The public health crisis of opioid misuse, addiction and overdose is one of the most challenging issues the U.S. Food and Drug Administration has faced during my time as FDA commissioner.  Solving this issue is critical to our future.

The issues cut across every socioeconomic level and geographic boundary. It would be difficult to identify any community in America that has not been touched by friends, family members or colleagues suffering from addiction, and far too often, losing their lives to it. As I leave the agency as part of the presidential transition, I have reflected on what I have seen, how far the nation has come, and the important work that remains for both the public and private sectors.

Robert CaliffI’ve made it a point to see affected communities, first-hand, because interventions and national policy solutions work best when they are well informed by what communities actually need. Just last week, I visited Baltimore to better understand how our cities are being affected – in this case, by an inflow of illicit fentanyl, a synthetic opioid that is about 100 times more potent than many other prescription opioids, and can be deadly on its first use. I have also visited the neonatal intensive care unit, or “NICU” of a Tennessee hospital where babies were screaming and shaking in the pain of withdrawal because they were born with Neonatal Opioid Withdrawal Syndrome. I have met people in West Virginia who did back-breaking work in power plants or in coal mines; after suffering on-the-job injuries they were first afflicted with pain, then by addiction. They got prescription pain relief but, too often, it wasn’t accompanied by the proper support and counseling. In Kentucky, I spoke to spouses and families whose lives have been forever changed by addiction, even as the community rallied together to fight it. And, much closer to home, I have heard personal stories from FDA employees and providers in local health care facilities, whose families and friends are not immune.

We have taken a number of actions at the FDA over the past several years to help reduce the number of people who become addicted, or who ultimately overdose from prescription opioids. We’ve improved product labeling, pushed for prescriber education, and encouraged the development of abuse-deterrent formulations. In addition, we have approved new intranasal and auto-injector forms of naloxone — products to reverse opioid overdoses, which can be administered by laypersons and are, therefore, better available able to save lives.

I’m also proud of the partnerships we have formed with other federal Agencies and the work that has resulted from them. But the latest data, including data from the Centers for Disease Control and Prevention (CDC) remind us that while some progress is being made, there is more to do. For example, it is promising to see that the nationally estimated number of outpatient prescriptions dispensed for Schedule II opioids decreased by 10 percent in 2015 compared to the previous year, according to IMS Health. However, the CDC reports that while the rate of overdose deaths associated with prescription opioid use increased by just 4 percent instead of by 9 percent the previous year, deaths associated with heroin use have skyrocketed. Clearly, more work needs to be done.

As I prepare to turn over the awesome responsibility of FDA commissioner to the next Administration, I feel compelled to point out that public and private sector efforts in this area must be continued and strengthened. In particular, I want to call on the pharmaceutical companies that manufacture and sell these drugs to dig deeper into their expertise and resources to prioritize finding solutions to this public health problem. I have consistently been impressed that the motivation to cure disease and improve quality of life for patients is shared across the spectrum of federal agencies, public health workers, health care providers and scientists within the pharmaceutical industry. However, the financial incentives in the industry can lead to a focus on short-term profits instead of patient well-being.This is the time for both branded and generic drug companies  to go beyond marketing and distribution plans and instead commit their expertise and resources to  confronting  the devastating negative consequences of a class of drugs that brings much needed pain relief, when used appropriately.

Specifically, I urge us all to focus on the following priorities:

  1. Encouraging appropriate prescribing by healthcare practitioners. Too many people become addicted from unnecessary prescriptions for minor pain or injury. And even appropriately prescribed opioids can lead to addiction, so careful monitoring of patients prescribed these powerful drugs is needed. While there are situations where opioids are appropriate, there are also situations where other alternatives can be effective. Therefore, conversations between provider and patient about the pain treatment plan are imperative. If an opioid is appropriate, CDC guidelines and FDA labeling emphasize the need to start on the lowest dose and minimum time necessary, and carefully monitoring patients for signs of addiction and inadequate pain control.
  1. Considering the family as well as the patient. Pain treatment, and use of opioid drugs, will be more successful when the family is involved. It will result in fewer drugs diverted from the medicine chest, fewer babies born addicted to opioids and better treatment of pain. Women who use or abuse opioids, or who are in treatment for opioid addiction, should talk to their health care provider before considering pregnancy.
  1. Finding better ways to treat pain with new medications and with more holistic pain management. It’s time to put more resources into the development of non-opioid, non-addictive medications to help people who are in serious, debilitating pain. We need more research to define the most effective non-medication approaches to pain and how to deliver them in a complex and financially constrained healthcare system.
  1. Improving how companies, professional societies and academics communicate about their activities in this area. I urge companies to commit to transparent and appropriate company communications and to work with government and others in the community to do a better job in educating the medical professionals responsible for treating our nation’s pain, as part of the overarching effort to do everything possible to help prevent addiction. Professional societies and academic medical centers also need to continue their efforts at educating their members and examining their practices to find ways to improve. For example, the education of the next generation of physicians about how best to manage pain is critical.
  1. Finding new ways to curb diversion and misuse of opioids. In addition to our continuing efforts to help support the development of abuse-deterrent formulations, the FDA is exploring potential packaging, storage, delivery, and disposal solutions that companies and other stakeholders might consider that would prevent opioids from being diverted to those without a legitimate prescription for these powerful drugs. I implore companies to conduct research and offer their creative ideas and resources to innovate in this area.
  1. Increasing pragmatic research to better understand how to implement appropriate pain therapy in general and use of opioids in particular. Post-market requirements from FDA that mandate industry-funded studies and recent pragmatic research efforts by the Patient-Centered Outcomes Research Institute and the NIH and Department of Defense are expected to provide important data, but we need more robust evidence to better guide practice. Pain is a vexing issue that seems to fall between the cracks in research funding; we need to keep the pressure on funding entities to move pain to the forefront as a research issue.
  1. Treating addiction as a disease, not as criminal behavior. We have the tools to treat addiction and reverse overdose from opioids and are working to develop more of them. But there’s a lot we don’t know about the drivers for drug abuse, and scientific knowledge will help us make better decisions. This is one reason why we need companies with products on the market to monitor the safety of those drugs and make their data public. We have mandated post-market studies to define major questions about chronic use of opioids, and it is essential that industry fulfills these requirements.

I am proud to have been part of the effort that’s changing the tide on this epidemic, but the nation has a long way to go.Government, companies, healthcare systems and healthcare providers all have important roles to play. The most recent data reminds us it’s time to double down on these efforts. While I won’t have the good fortune of leading this fight in an official capacity, I’m proud of the work the FDA and others have done so far. I leave FDA’s efforts to the many leaders at the agency who have been working tirelessly on this issue — and will continue doing so — and look forward to supporting public and private efforts to bring this epidemic to an end.

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

FDA’s Science-based Approach to Genome Edited Products

By Robert M. Califf M.D., and Ritu Nalubola, Ph.D.

Recent scientific advances now make it possible to more efficiently and precisely alter the genome of plants, animals, and microorganisms to produce desired traits. These genome editing technologies are relatively easy to use and can be applied broadly across the medical, food and environmental sectors, with potentially profound beneficial effects on human and animal health. However, there are also potential risks ranging from how the technology affects individual genomes to its potential environmental and ecosystem impacts. Additionally, genome editing has raised fundamental ethical questions about human and animal life.

Genome editing technologies can be used to introduce, remove, or substitute one or more specific nucleotides (letters in the DNA code) at a specific site in the organism’s genome, and is achieved with the use of protein-nucleotide complexes. Several classes of these complexes exist, the most recent discovery is known as CRISPR/Cas9.

Research is currently underway that would use these technologies to:

  • Treat HIV, cancer or rare diseases by genetically altering specific types of cells;
  • Control or alter organisms that carry infectious diseases (for example, mosquitoes that are vectors of viruses/parasites causing dengue fever, Zika or malaria; or mice that transmit bacteria causing Lyme disease);
  • Improve the health and welfare of food producing animals, (for example, hornless cattle, pigs resistant to African swine fever or porcine reproductive and respiratory virus); and
  • Alter specific traits of food plants or fungi (for example, non-browning mushrooms).

Accompanying the enthusiasm about these promising technologies are questions about whether FDA is prepared to ensure the safety of regulated products that use this technology. Providing appropriate and balanced regulatory oversight for applications involving an emerging technology is not a new or unique challenge for FDA, but the potential breadth of applications and the fundamental nature of altering the genome call for the participation of multiple constituencies in considering the most effective regulatory policies to address any potential risks.

Robert Califf

Robert M. Califf, M.D., FDA Commissioner

Maintaining product-specific, risk-based regulation

Genome editing applications are relevant to three main FDA-regulated product classes. The specific regulatory approaches for each of these classes vary, reflecting differences in underlying statutory authorities. FDA is maintaining a product-focused, science-based regulatory policy, in accordance with specific legal standards applicable to each type of product and consistent with overarching U.S. Government policy principles.

Human medical products that apply gene editing to exert their therapeutic effect are regulated under our existing framework for biological products, which include gene therapy products. “Gene editing” here refers to non-heritable situations somatic cell gene therapy only, and not to heritable conditions (germ line gene therapy). The FY16 appropriations bill restricted use of federal funds “in research in which a human embryo is intentionally created or modified to include a heritable genetic modification.” FDA’s Center for Biologics Evaluation and Research (CBER) has a well-established program and policies in place to evaluate gene therapy products. Although different types of gene editing have potential clinical applications, currently only one type of gene editing, zinc finger nuclease- (ZFN) mediated, has been announced by their sponsors as being applied in clinical trials underway in the United States. Proposals for NIH-funded human gene therapy clinical trials are discussed and reviewed for scientific, clinical, and ethical issues by the NIH’s Recombinant DNA Advisory Committee (RAC). The RAC recently discussed (and did not find any objections to) the first clinical protocol to use CRISPR/Cas9-mediated gene editing. The potential for “off-target” effects such as insertions or deletions at unintended genetic loci has been identified by experts in the field as a key concern.

Similarly, FDA’s Center for Food Safety and Applied Nutrition and Center for Veterinary Medicine have in place programs to adequately address foods derived from plants produced using genome editing and animals produced using genome editing. In these two product areas, we are issuing documents to clarify our current thinking and seek scientific information. With respect to foods derived from plants produced using genome editing, FDA has a longstanding program for foods derived from new plant varieties, including those developed by recombinant DNA (rDNA) techniques. We are requesting information on whether human and animal foods derived from genome edited plants pose additional risks compared to those from traditionally bred plants. FDA’s decades of experience providing oversight of foods from new plant varieties, coupled with scientific evidence and data received, will help inform our thinking on risk considerations going forward.

When animals are produced using genome editing, FDA has determined that, unless otherwise excluded, the portion of an animal’s genome that has been intentionally altered, whether mediated by rDNA or modern genome editing technologies, is a drug because it is intended to alter the structure or function of the animal and, thus, subject to regulation under our provisions for new animal drugs. We have updated our existing guidance for genetically engineered animals to include genome editing within its scope, and are issuing it in draft form for public comment. We are also seeking input on whether certain types of genome editing in animals pose low or no significant risk, and we may modify our regulatory approach based on this input.

Our efforts to gather necessary scientific data aside, industry remains responsible for ensuring that its products meet all applicable requirements, including safety standards. FDA has historically made itself available to meet with developers and we encourage them to engage with us to help ensure they meet their statutory and regulatory obligations. And we will continue to provide technical advice and guidance for industry, as necessary.

Collaborating with Federal agencies

The White House Office of Science and Technology Policy (OSTP), FDA, the U.S. Environmental Protection Agency (EPA), and the U.S. Department of Agriculture’s Animal and Plant Health Inspection Service (APHIS) initiated an effort in 2015 to ensure public confidence in the regulatory system for biotechnology products and improve the transparency, predictability, coordination, and, ultimately, efficiency of that system. After reviewing public comment to a docket and holding three public meetings, the agencies produced A National Strategy for Modernizing the Regulatory System for Biotechnology Products, to help ensure that the federal regulatory system is prepared to assess future biotechnology products, issued in September; and, earlier this month, a 2017 Update to the Coordinated Framework for the Regulation of Biotechnology (CF Update), to clarify each agency’s role.

Ritu Nalubola

Ritu Nalubola, Ph.D., Senior Policy Advisor, FDA’s Office of Policy

APHIS is proposing to revise its regulation regarding genetically engineered organisms that may pose plant pest or noxious weed risks. As FDA, APHIS, and EPA formulate policies, there may be differences in approaches, reflecting differences in the scope of their authorities and the types of risks addressed. Under the CF Update, interagency coordination and cooperation will continue, including on appropriate terminology, identification of hazards, and approaches to addressing risks, within the constraints imposed by regulatory paradigms for different product areas.

FDA also has a longstanding collaborative relationship with the NIH office that oversees the RAC. FDA serves as a non-voting liaison on the committee, hears the discussions first-hand, and receives the written recommendations.  These recommendations may be considered during our overall review of investigational new drug applications (INDs) submitted to FDA.

Scientific engagement and horizon-scanning

Being ready to evaluate innovative emerging technologies is a top FDA regulatory science priority. FDA is co-sponsoring two studies, conducted by the National Academies of Sciences, Engineering, and Medicine (NASEM). Both are expected to be completed this year. FDA is also conducting its own horizon-scanning through its Emerging Sciences Working Group, an FDA-wide science-based forum, and opened a public docket to receive input on emerging technologies.

Working with international partners

Scientific advances do not adhere to national boundaries and therefore it is critical that we understand the evolving views of our international counterparts. Given the leadership role of the United States in biomedical and biological sciences, we cannot afford to fall behind in this exciting scientific frontier. As expected, international regulatory agencies, too, are currently working in this area. FDA’s CBER is an active member of the International Pharmaceutical Regulators’ Forum (and its Gene Therapy working group), which provides a forum for members to identify and exchange information on issues of mutual concern and undertake targeted regulatory cooperation activities.

Going forward

FDA is committed to fulfilling its mission to safeguard public health, while encouraging innovation and competitiveness. The actions we have taken to date, including release of the CF Update, National Strategy, and FDA’s documents – are steps in a series of ongoing activities. We will continue to collaborate with our federal and international partners, and actively communicate with stakeholders to help ensure confidence in FDA’s regulatory system. However, oversight provided by FDA is one aspect of broader governance necessary for safe and responsible research and development of genome editing applications. Moreover, the expansive scope of intentional genomic alterations using modern genome editing technologies has triggered debate on fundamental ethical and social issues, which will continue to influence public opinion and acceptance of genome editing applications. Even as FDA implements necessary steps for effective regulation to ensure the safety of products, the role of broader, inclusive public discussion involving multiple constituencies (e.g., scientists, developers, bioethicists, and public interest and community groups) to address the larger societal considerations should not be overlooked.

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

Ritu Nalubola, Ph.D., is a Senior Policy Advisor in FDA’s Office of Policy

 

Managing Medical Device Cybersecurity in the Postmarket: At the Crossroads of Cyber-safety and Advancing Technology

By:  Suzanne B. Schwartz, M.D., M.B.A.

Protecting medical devices from ever-shifting cybersecurity threats requires an all-out, lifecycle approach that begins with early product development and extends throughout the product’s lifespan.

Today, we’re pleased to announce that industry now has advice from FDA across this product continuum with the release of a final guidance on the postmarket management of medical device cybersecurity. It joins an earlier final guidance on medical device premarket cybersecurity issued in October 2014.

suzanne-schwartz-new-dec-2016To understand why such guidance is so important for patients, caregivers and the medical device community, we need to take a step back and look at how cybersecurity fits into the medical device ecosystem.

In today’s world of medical devices that are connected to a hospital’s network or even a patient’s own Internet service at home, we see significant technological advances in patient care and, at the same time, an increase in the risk of cybersecurity breaches that could affect a device’s performance and functionality.

The best way to combat these threats is for manufacturers to consider cybersecurity throughout the total product lifecycle of a device. In other words, manufacturers should build in cybersecurity controls when they design and develop the device to assure proper device performance in the face of cyber threats, and then they should continuously monitor and address cybersecurity concerns once the device is on the market and being used by patients.

Today’s postmarket guidance recognizes today’s reality – cybersecurity threats are real, ever-present,  and continuously changing. In fact, hospital networks experience constant attempts of intrusion and attack, which can pose a threat to patient safety. And as hackers become more sophisticated, these cybersecurity risks will evolve.

With this guidance, we now have an outline of steps the FDA recommends manufacturers take to remain vigilant and continually address the cybersecurity risks of marketed medical devices. Central to these recommendations is FDA’s belief that medical device manufacturers should implement a structured and comprehensive program to manage cybersecurity risks. This means manufacturers  should, among other things:

  • Have a way to monitor and detect cybersecurity vulnerabilities in their devices
  • Understand, assess and detect the level of risk a vulnerability poses to patient safety
  • Establish a process for working with cybersecurity researchers and other stakeholders to receive information about potential vulnerabilities (known as a “coordinated vulnerability disclosure policy”)
  • Deploy mitigations (e.g., software patches) to address cybersecurity issues early, before they can be exploited and cause harm

This approach enables manufacturers to focus on continuous quality improvement, which is essential to ensuring the safety and effectiveness of medical devices at all stages in the device’s lifecycle.

In addition, it is paramount for manufacturers and stakeholders across the entire ecosystem to consider applying the National Institute of Standards and Technology’s (NIST) core principles for improving critical infrastructure cybersecurity: to identify, protect, detect, respond and recover. It is only through application of these guiding principles, executed alongside best practices such as coordinated vulnerability disclosure, that will allow us all to navigate this uncharted territory of evolving risks to device security.

This is clearly not the end of what FDA will do to address cybersecurity. We will continue to work with all medical device cybersecurity stakeholders to monitor, identify and address threats, and intend to adjust our guidance or issue new guidance, as needed.

Digital connections power great innovation—and medical device cybersecurity must keep pace with that innovation. The same innovations and features that improve health care can increase cybersecurity risks. This is why we need all stakeholders in the medical device ecosystem to collaborate to simultaneously address innovation and cybersecurity. We’ve made great strides but we know that cybersecurity threats are capable of evolving at the same pace as innovation, and therefore, more work must be done.

Learn More

For more information about medical device cybersecurity, visit the FDA’s Center for Devices and Radiological Health web page.

Suzanne B. Schwartz, M.D., M.B.A., is FDA’s Associate Director for Science and Strategic Partnerships, at the Center for Devices and Radiological Health

FDA Working to Keep the U.S. Blood Supply Safe from Zika and other Emerging Threats

By: Peter Marks, M.D., Ph.D., and Luciana Borio, M.D.

En Español

Each day in the United States, thousands of people receive transfusions of blood or blood products as part of their medical treatment. Although such transfusions can save lives, like most medical treatments they may also carry some risks. Among the most concerning risks is the chance that a patient might get an infection from a transfusion. Some of these risks, like hepatitis and HIV, have been known for some time. Others, like the Zika virus, have emerged more recently.

One of FDA’s top priorities is to help assure that the blood supply is safe for all those who need these potentially life-saving products. This mission is accomplished through close collaboration with other government agencies, such as the Centers for Disease Control and Prevention, and non-governmental partners, such as blood collection establishments and screening test developers.

These coordinated efforts are crucial because blood safety relies on a combination of factors, including understanding the epidemiology of infectious disease transmission and the testing that can be done to detect it.

Our experience with other infectious agents has positioned us well to address Zika virus. We have worked closely with the CDC since the outbreak began to emerge. We have also been in continual contact with blood collection establishments, screening test manufacturers, and local health authorities.

When it became apparent early in 2016 that Zika virus could pose a risk to the blood supply in the United States, FDA issued recommendations to reduce the risk of its transmission through blood transfusions by deferring those donors at risk of being infected with Zika virus as well as by screening donated blood in certain areas. Given the large amount of scientific data that is emerging and the rapid spread of the outbreak in the Western hemisphere, FDA understands that it may need to revisit and update this guidance soon to maintain the safety of the blood supply.

As part of the overall response, FDA also collaborated with the HHS Office of the Assistant Secretary for Health to ensure that safe blood was available to those needing a transfusion in Puerto Rico and other areas at risk before a blood screening test for Zika virus became available. The Agency also worked with manufacturers to help speed the development of screening tests to detect Zika virus at the earliest point in time after infection. We also worked with the Biomedical Advanced Research and Development Agency to help facilitate the evaluation of these tests and other measures.

Helped by these and other ongoing collaborations with federal and non-federal partners, FDA is able to pursue its mission of protecting and promoting your health.

Peter Marks, M.D., Ph.D., is Director of FDA’s Center for Biologics Evaluation and Research
Luciana Borio, M.D., is FDA’s Acting Chief Scientist

FDA Working to Keep Patients Well Informed

By: Steve L. Morin R.N., B.S.N.

Steve Morin_2823My job in the Food and Drug Administration’s Office of Health and Constituent Affairs (OHCA) is to serve our nation’s patients in two ways: by listening to their concerns regarding FDA’s policy and decision-making and advocating for them in our agency;  and by informing many patients and patient organizations about FDA’s mission and its work to advance the development, evaluation and approval of new therapeutic products.

This dialogue was formalized and greatly expanded in 2012 when, after a series of listening sessions with many patient advocacy organizations, OHCA created the Patient Network.

Specifically designed for patients, caregivers, patient advocates and disease-specific patient advocacy organizations and the communities that advocate on their behalf, this program serves two goals. It facilitates patient engagement with FDA policy and decision makers, and it educates its audience about the process that brings new medications – both prescription and over-the-counter ­– and medical devices from a concept to the marketplace.

Our Patient Network covers a range of FDA-specific topics and conducts numerous activities that are of interest to patients and patient advocates. One of these activities were webinars with information about upcoming public meetings hosted by FDA.

For example on March 31, 2014, OHCA was pleased to host the first-of-its-kind “LiveChat” with the diabetes community. This online discussion gave patients an opportunity to interact with FDA experts and to better understand a recently released draft guidance dealing with the studies and criteria that FDA recommends be used when submitting premarket notifications (510(k)s) for blood glucose meters.

On September 10, 2014, our Third Annual Patient Network Meeting titled “Under the Microscope: Pediatric Product Development” brought together more than 100 patients, patient advocates, representatives of academia and industry, and FDA leaders. The participants discussed pediatric product development and the ways patient advocates can participate in it.

And on September 17, 2014, our Patient Network webpages were upgraded. The “For Patients” section on FDA’s website is presented in a clear manner with easy-to-use formats. Also, a “For Patients” button is located on our homepage.

We have continued to pursue our goals of informing the public and engaging with patients by building upon the patient-centered webpages and enhancing activities that express our desire to be helpful and transparent. This is our philosophy that has helped the Patient Network evolve to what you see today.

As the Patient Network program continues to grow, I hope to expand it to have more interactive webinars like the “LiveChat” that address specific concerns  of the patient communities. Also, we will continue to make it possible for patients to learn from FDA experts who approve medical products.

The FDA realizes that listening to the “patient voice” and conducting our dialogue is important, and it continues to develop its model for patient involvement through the Patient-Focused Drug Development Meetings and other OHCA sponsored meetings and webinars. We hope patients and those who care for them will join us in that effort, and make it still more helpful in protecting and promoting the public health.

Steve L. Morin, R.N., B.S.N., is a Commander of the United States Public Health Service and the Manager of the Patient Network in FDA’s Office of Health and Constituent Affairs

FDA Uses Web Tool to Better the Odds for Food Safety

By: Ted Elkin

When most people hear the words, “Monte Carlo,” they may think about high-stakes gambling.

We, however, think about reducing the risk in food safety through the use of FDA-iRISK, an innovative Web-based food safety modeling tool developed by the Food and Drug Administration and our partners.

Launched in October 2012, FDA-iRISK uses mathematical logic and Monte Carlo simulation (a computer program named for the gambling mecca) to integrate data and generate results that compare and rank risks of the contamination of foods by various hazards.  Unlike a traditional risk assessment of a single food and a single contaminant, FDA-iRISK allows users to compare multiple hazards – microbial or chemical – in multiple foods.

How does FDA-iRISK work?

Through extensive outreach to and collaboration with partners, we developed built-in templates and other features that allow the user to create real-world (or hypothetical) food safety scenarios.

The user provides the data for seven elements: the food(s), the hazard(s), the population of concern (for instance, elderly or immune-compromised), the production or processing system being used for the food, the consumption patterns, the dose response (what level of exposure will have a health impact), and how the health effects are to be calculated.

This allows the user flexibility, for instance, to look at the impact of potential interventions at various stages of the food production system as well as the populations affected.  And it’s easy to use.

Of particular benefit to the user is FDA-iRISK’s ability to generate reports that measure the health impact of an intervention in terms of the widely used public health metric, DALYs (“Disability-Adjusted Life Years,” meaning years of healthy life lost to illness or death).  This measure lets us know the “bang for the buck” of a particular intervention.

FDA-iRISK is quickly gaining acceptance and use in the food safety community.  As of the middle of May, almost 500 users had established accounts with FDA-iRISK and they came from every continent. Because it is web-based, FDA-iRISK is available to anyone in the world who sets up an account, and it is free to use.

Therefore, the knowledge and sharing power of FDA-iRISK is exponential.  As more users use it and generate reports that are then available to the other users, a more consistent, well documented, systematic, structured and quantitative picture of risk in the food supply will emerge, as well as scenarios for reducing risk.

“Information provided by iRisk can aid in developing global scientific exchanges aimed at maintaining and developing agricultural markets around the world,” according to Jamilah (Fagbene) Cassagnol, an international trade specialist at the U.S. Department of Agriculture.

FDA-iRisk is supported by an exceptional project team. FDA staff members Sherri Dennis, Yuhuan Chen, David Oryang, Regis Pouillot, Karin Hoelzer and Susan Cahill developed the tool in collaboration with Risk Sciences International, RTI International and the Institute of Food Technologists.

Ultimately, for food safety, Monte Carlo shouldn’t mean taking a gamble.  Rather, it’s all about using a quantified, standardized and transparent methodology to better understand what interventions and controls will reduce the risk and improve our public health.

Ted Elkin is Director, Office of Analytics and Outreach, at FDA’s Center for Food Safety and Applied Nutrition.