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

 

Spring Unified Agenda: FDA’s Anticipated Upcoming Regulatory Work

By: Scott Gottlieb, M.D.

Today, the federal government published the Spring 2018 “Unified Agenda of Federal Regulatory and Deregulatory Actions” (Unified Agenda), which provides federal agencies with the opportunity to update the American public on our government’s regulatory priorities.

Dr. Scott GottliebFor its part, the U.S. Food and Drug Administration (FDA) continues to make swift progress on our regulatory agenda, which reflects the key strategic priorities of the FDA and the Administration. Our regulatory agenda reflects our adherence to science based decision making and our commitment to our mission to protect and promote public health.

I provided a detailed overview of many of our proposed regulations for 2018 around the release of the Unified Agenda last fall – most of which we continue to take forward. I’d like to take this opportunity to highlight for you some of FDA’s new contributions to the Spring Unified Agenda.

Addressing the Nicotine Addiction Crisis

Smoking remains the leading cause of preventable death and disease. And too many young people are still being initiated on tobacco products, and becoming addicted to nicotine.

We’ve taken steps to address the morbidity and mortality associated with tobacco through the comprehensive plan that we announced last summer. We’re considering regulating the nicotine levels in combustible cigarettes, to render cigarettes minimally or non-addictive.

At the same time, we’re continuing to advance our framework for how we’ll regulate both novel nicotine delivery products, such as e-cigarettes, and traditional tobacco products. One goal of our efforts is to encourage innovation of less harmful products. We will ensure that all tobacco products, whatever their nicotine content or delivery mechanism, are put through an appropriate series of regulatory gates to maximize any public health benefits and minimize harms.

To that end, we will be proposing a new regulation to establish product standards for electronic nicotine delivery systems or ENDS. The proposed standard will, among other things, address the levels of toxicants and impurities found in nicotine, propylene glycol, and vegetable glycerin e-liquid, as these toxicants and impurities can cause death or other adverse health effects.

As part of our comprehensive plan, we’re also working hard to prevent access to products we believe are adulterated or misbranded. We recently joined with the Federal Trade Commission to issue 13 joint warning letters to companies that misleadingly labeled or advertised nicotine-containing e-liquids as kid-friendly food products (juice boxes, candies, and cookies).

As part of our comprehensive approach, we’ll also be proposing new regulations to establish requirements for the administrative detention of tobacco products encountered during an inspection that an officer or employee believes to be adulterated or misbranded. These steps will allow us to more effectively block the distribution and use of products that are ultimately found to be violative, including products that are misbranded because their labeling or advertising causes them to resemble kid-friendly foods.

Modernizing and Harmonizing Standards

As part of our efforts to continue to ensure efficiency of existing regulations, we will be taking another step to modernize medical device regulation, by proposing a new regulation to replace certain aspects of existing Quality System regulations with specifications of an international consensus standard for medical device manufactures (ISO). This rule, if finalized, will harmonize domestic and international requirements and modernize the regulation to make it more efficient for manufacturers of medical devices seeking to sell their products globally, while also continuing to ensure they adhere to high, internationally-accepted quality systems.

Enhancing Clinical Trial Processes

The Spring Unified Agenda also will propose rules to support the clinical trial process, for instance regarding the requirements for cooperative research. We’re proposing a new rule that would, in most cases, allow any institution located in the U.S. that is participating in a multisite cooperative research to be able to rely on approval from a single institutional review board.

We also will be issuing a proposed rule to update the agency’s investigational new drug application regulations to define and clarify the roles and responsibilities of the various persons engaged in clinical investigations to enhance protection of the rights, safety, and welfare of subjects and better ensure the integrity of clinical trials.

In addition to the new proposed regulations I’m highlighting here, FDA will continue to pursue a multitude of other important rules across the Agency, such as taking forward our compounding policy priorities and advancing food and drug safety initiatives. Moreover, we continue to remove outdated rules or reconsider proposed rules in light of our evolving policy priorities. I want to note, however, that some previously identified regulations that weren’t included in this Unified Agenda may still remain FDA priorities. Just because you don’t see them here, doesn’t mean that we don’t intend to continue advancing some prior policy proposals.

While we continue to have a robust regulatory agenda for the coming year, regulation is only one way in which we can foster our mission and improve public health. We’ll continue to tackle many additional priority areas through guidance documents and other policy efforts. These areas will include efforts to reduce the cost of drugs by encouraging competition – including in biosimilars; spurring innovation across medical products; battling obesity through our various nutrition initiatives; and, continuing to attack the opioid addiction crisis facing our country.

I look forward to keeping you updated as we progress toward these goals.

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

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

Mission Possible: Moving the Needle Forward to Advance Health Equity

By: CAPT Richardae Araojo

Every April our country observes National Minority Health Month to spotlight what we’re doing to eliminate health disparities among minority populations. A health disparity is a particular type of health difference that is closely linked with social and economic disadvantage, discrimination, or exclusion. We strive for what we call health equity―the attainment of the highest level of health for all people―by enlisting a range of approaches to remove the social and economic obstacles to health faced by racial and ethnic groups.

CAPT AraojoAs the Director of FDA’s Office of Minority Health (OMH), I lead cross-agency efforts with my team to protect, promote, and advance the public health of our country’s most vulnerable and underrepresented populations. OMH does this in many ways. For example, we:

  • Conduct and fund research on diseases that disproportionately affect minorities, like HIV/AIDS, diabetes, and heart disease.
  • Work to diversify the public health workforce by training principal investigators and pharmacists from diverse backgrounds, such as African Americans, Hispanics, American Indians/Alaska Natives, and Asian Americans and other Pacific Islanders, who can relate to research volunteers and patients from underserved communities. Research shows that people want their health professionals to look like them, so a workforce that reflects the demographics of the community it serves is vital.
  • Help minorities make better informed health decisions by creating culturally and linguistically tailored health education materials for use across different social media platforms.
  • Engage with minority-serving institutions of higher learning to protect and improve the health of the populations they serve.
  • Serve as a voice for those in need by encouraging all our constituents to participate in the work that we do. One example is the inaugural FDA Rural Health Symposium, a cross-Agency effort among OMH, the Office of Health and Constituent Affairs, and the Center for Tobacco Products, with participation from other FDA product centers. The symposium offered stakeholders from rural and tribal communities a forum to discuss how we can work together to address rural health challenges that range from the opioid crisis and tobacco use among youth to the need for telemedicine.

In the spirit of this year’s theme for National Minority Health Month, Partnering for Health Equity, I’d like to share a couple of other ways we’ve been partnering with private- and public-sector organizations to further equity on all fronts.

Getting culturally sensitive messages out to minority communities

My office conducts robust communications and outreach activities to share research and information on FDA-regulated products and to promote public health. For instance, Asian Americans, African Americans, and Latinos have lower immunization rates for adult vaccinations like herpes zoster, whooping cough, hepatitis B, and influenza. To better understand these disparities, OMH is supporting a study to assess the impact of advertising and promotional labeling as it relates to vaccine health disparities. OMH has message-tested FDA’s communications with consumer panels, among others, and we’re using the information from this research to shape FDA’s health education materials and outreach to minority communities.

Ensuring minority representation in clinical trials

Ensuring minority representation in clinical trials is crucial to improving minority health because we need to understand how different racial and ethnic groups respond to medical products before they are approved for use in the broad population. To that end, FDA developed guidance for industry and FDA staff. This guidance provides recommendations on using a standardized approach for collecting and reporting race and ethnicity data used to support marketing applications for FDA-regulated medical products.

OMH also works collaboratively with organizations whose mission includes encouraging more minorities to participate in clinical trials. We’ve partnered with the Veteran Health Administration’s Office of Health Equity to launch two videos featuring veterans talking about why diverse representation is so important. These veterans will also appear in the first installment of our new podcast series on health equity and disparities to share their experiences as participants in clinical trials.

Another important partnership involves our newly formed memorandum of understanding (MOU) with Yale University. Under this MOU, we’ll be working to advance scientific collaborations, outreach, and educational initiatives. Especially exciting is the cultural ambassador’s program, which will engage community members to get more involved in clinical research.

In sum, to create a world where health equity is a reality for all we must involve all stakeholders in new ways of thinking and working. And that requires the kind of teamwork, partnerships, and collaboration across disciplines, experiences, and sectors that I’ve shared with you here.

Visit www.fda.gov/minorityhealth for more information on FDA’s Office of Minority Health, and follow us on Twitter @FDAOMH for updates.

CAPT Richardae Araojo is FDA’s Associate Commissioner for Minority Health

Reflections on a Landmark Year for Medical Product Innovation and Public Health Advances and Looking Ahead to Policy in 2018

By: Scott Gottlieb, M.D.

Dr. Scott GottliebAs we look ahead to 2018, I’d like to take a moment to reflect on an inspiring year of advances in both medicine and public health for FDA — from groundbreaking medical products brought to market this year, to a record number of generic drug approvals that will promote competition, and to the agency’s ongoing efforts to advance policies that promote safe and effective product innovation, and keep Americans safe from food-related illnesses.

Today, new medical breakthroughs are profoundly altering how we view and treat disease in ways that seemed inconceivable just years ago. In this modern medical setting, FDA is evaluating all aspects of its policies to make sure we’re protecting consumers, while promoting beneficial innovation that has the potential to effectively treat disease for human and animal patients, and improve public health.

A Record Year for New Innovation

As scientific understanding of disease advances and the practice of medicine becomes more tailored to individual patient needs, we also are modernizing how we work with innovators throughout the development process to bring products to patients more efficiently, using the best available science.

For example, FDA recently coordinated the approval of a novel diagnostic device that can detect hundreds of genetic mutations in a single test with the Centers for Medicare & Medicaid Services’ proposed coverage of the test, thereby facilitating earlier access to this innovative product.

Also, in the rapidly advancing field of individualized medicine, the Agency advanced new draft guidance that addresses better ways to develop treatments that address the underlying molecular changes (e.g., genetic mutations) that often cause or contribute to diseases. This includes uncommon molecular changes that are present in only a small subset of patients. The guidance proposes an approach for drug developers to enroll patients into clinical trials for targeted therapies based on the identification of rare mutations,  when reasonable scientific evidence suggests the drug could be effective in patients with these genomic findings. The new guidance discusses the evidence needed to demonstrate effectiveness for a variety of molecular subsets within a particular disease. The framework could lead to more consistent development and approval of targeted therapies for patients who are likely to benefit from them.

This past August we also saw the practical advent of a whole new way to treat disease with the approval of the first gene therapy product in the United States. We have since approved two more gene therapy medicines. Innovations like these are creating a turning point in the treatment of serious illnesses. With this technology also comes greater potential to cure intractable and inherited diseases.

2017 saw a number of other similar, historic milestones with regard to new innovation. This collective progress reflects a fundamental shift in science that’s enabling us to attack more diseases with novel platforms. We’re increasingly able to identify patient benefit earlier in the development process because of the ability to better target medicines to the underlying mechanisms of disease. At the same time, in many cases these identical tools also allow us to surface safety issues earlier and more effectively.

pie chart of approvals

FDA approved a modern record number (56) of novel drugs and biologics in 2017.

Owing in part to these advances, FDA approved a modern record number (56) of novel drugs and biologics in 2017. Of these 56 novel approvals this past year, 46 were new molecular entities approved by our Center for Drug Evaluation and Research – of which 28 were approved using one or more of FDA’s expedited review programs. Ten of these 56 novel approvals were biological therapeutics that were approved by our Center for Biologics Evaluation and Research. We also had a record number of drugs with orphan indications approved. At the same time we eliminated the entire backlog of pending orphan drug designation requests. We also broke records, with the highest number of generic drugs approved in a single month multiple times in 2017, and we recorded the highest annual total of generic drug approvals (1,027) in the agency’s history. We believe that, if current trends continue, we’ll exceed this record number of generic drug approvals in 2018.

2017 generics approvals

Both full approvals and tentative approvals, which do not allow the applicant to market the generic drug product and postpones the final approval until all patents/exclusivity issues have expired.

Our science-based and patient-centered regulatory approach also extends to medical devices, where we’ve focused on a life-cycle approach to product development. This has allowed us to streamline clinical development protocols without compromising on our commitment to rely on rigorous evidence. By carefully considering when clinical data can be better gathered through post-market, as opposed to pre-market, studies, patients are waiting less time to access some breakthrough devices without conceding one bit FDA’s gold standard for demonstrating reasonable assurance of device safety and effectiveness.

Chart of device approvals

In 2017, the agency approved a record number of novel devices — 95. This was more than four times the number of novel devices that received market approval in 2009.

Our commitment to applying the “least burdensome standard” for generating information critical for device approval was strengthened and advanced by provisions in the 21st Century Cures Act. This policy approach is a hallmark of our efforts to help innovators generate high quality evidence that can support marketing approval as efficiently as possible. Our embrace of these principles has resulted in remarkable advances in access for patients. In 2017, the agency approved a record number of novel devices — 95. This was more than four times the number of novel devices that received market approval in 2009.

Modernizing FDA’s Regulatory Programs

These new advances also present new challenges. At FDA, we’re being confronted with the need to regulate highly novel areas of science like gene therapy, targeted medicine, cell-based regenerative medicine, and digital health; where our traditional approaches to product regulation may not be as well suited. To meet these new challenges, we’re taking a fresh look at how we can adapt our customary approaches to regulation. We need to make sure that we’re allowing beneficial new technologies to advance, while continuing to protect consumers as part of our product review processes.

To promote these efforts, we advanced a new policy framework allowing certain diagnostic tests to undergo review by accredited third parties. This new framework will reduce the burden on test developers and streamline the regulatory assessment of these types of innovative products. This approach more readily accommodates the highly iterative nature of these technologies, where tests often undergo routine modifications to improve their precision and clinical utility.

Over the past summer, we also launched a pilot program exploring a new way of regulating digital health devices so that these fast-evolving technologies can similarly undergo the rapid product evolution that’s the hallmark of software tools like medical apps, while FDA maintains the ability to make sure that these digital health tools are being reliably produced. We followed these actions with a suite of guidances that clarify how we intend to regulate certain digital health technologies in a way that encourages innovation.

More broadly, we provided more clarity for manufacturers of low- to moderate-risk medical devices which will reduce unnecessary submissions to FDA for minor modifications that could not significantly affect device safety or effectiveness. As a result, patients will benefit from upgraded products more quickly.

This effort to properly match our policies to the unique attributes of the new technologies we’re being asked to review was also evident in new steps we took across other programs; from our comprehensive policy on regenerative medicine aimed at spurring safe and effective innovation in these potentially transformative products, to our draft guidance for manufacturers of 3D printed medical devices.

We know that the public health benefits derived from our efforts to modernize our regulatory approaches are not confined to the pre-market review process. Advances in our post-market tools and policies can yield meaningful advances for patients in the form of safer products, better information to guide medical decisions, and more opportunity to more efficiently move products to market – if we can have confidence in our post-market oversight. This is why we’re always looking for ways to reform and improve this oversight, and advance the ways that we share this information with patients and providers.

For example, last fall we launched a new searchable database to better inform patients and health care professionals of adverse events reported with drug and biologic products. We’ll be taking other steps soon to improve on the ways that we share important clinical information with patients and providers. These goals also include new efforts to step up our post-market oversight of potentially risky products, and warn consumers earlier of potential problems we find. As an example, we’ve taken decisive action to protect the public from risky stem cell products offered by unscrupulous clinics. We’ll pursue similar actions in 2018.

In 2017, we also took new steps to warn companies making false claims that their unapproved products can treat or cure life-threatening diseases; we advanced a new draft guidance describing FDA’s approach to regulating homeopathic products based on the risk they can pose to consumers; and we took steps to alert the public to the dangers of other unproven and untested products, such as certain body-building products, contaminated dietary supplements and kratom. Among other efforts, we also took new steps to facilitate faster patient access to needed compounded medicines, while protecting the public from poorly compounded drugs. There will be additional enforcement steps in 2018. And we continue to promote work that will enable FDA to use real world data to better inform our regulatory decision-making.

Promoting Drug Competition

Many say that FDA has no role in drug pricing, but I disagree. While we don’t have the authority to regulate prices, we do have the authority — and the responsibility — to ensure that the agency’s policies are not impeding competition that could ultimately be a check to rising drug prices and patient access.

Our role as gatekeeper of cost-effective, high-quality generic drug products is a foundational part of fostering human and animal drug competition. We’re advancing new ways that FDA can help enable patients to get access to more affordable medications. We shared some of the steps the agency is taking with our launch in June 2017 of the Drug Competition Action Plan — from prioritizing our review of generic drug applications, to working to stop companies from finding loopholes in the system that delay the entry of generic drugs to market, to making substantial progress on the generic drug review backlog, to ensuring that low cost drugs get to the patients who can benefit from their effectiveness and more affordable price.

New Steps to Combat Addiction

I’ve noted many times that among my highest priorities as Commissioner is addressing addiction crises facing the nation, principally with respect to nicotine and opioids. In 2017 we announced new plans for how we address these crises. In July, we announced a comprehensive plan that proposes to lower nicotine in combustible cigarettes to minimally or non-addictive levels. At the same time, we took new steps to enable development of innovative delivery systems that could be potentially less harmful than cigarettes for adults who still want to get access to satisfying levels of nicotine. As part of that plan, we formed a new Nicotine Steering Committee. It’s charged with modernizing FDA’s approach to development and regulation of nicotine replacement therapy products that can help smokers quit and stay quit

FDA also unveiled new actions to confront the staggering human and economic toll created by opioid abuse and addiction, starting with my first major action as commissioner to establish an Opioids Policy Steering Committee. Under the leadership of this committee, FDA is reevaluating how drugs that are already on the market are used, both for legitimate purposes and misuse and abuse.

The committee will recommend new policy steps to address this crisis. FDA also is taking immediate action where needed, as we did with FDA’s first-of-its-kind request to remove a currently marketed opioid pain drug from sale due to the public health consequences associated with the product’s abuse and misuse. We’ve also worked to identify ways to decrease exposure to opioids, prevent new addiction, and support the treatment of those with opioid use disorder; for example, through new Risk Evaluation and Mitigation Strategy requirements for makers of immediate-release opioids, and requiring labeling changes to add important clarifying information regarding the use of medication-assisted treatments for patients suffering from opioid use disorder. We’re continuing to pursue other creative ways to address the crisis, such as leveraging different forms of packaging, storage and disposal of opioid medications.

Protecting and Empowering Consumers

It’s not only medical products and policies where FDA can innovate to better serve the public – we’ve also made much progress in the implementation of the Food Safety Modernization Act, which was designed to keep the American public safe from food-related illness. Implementing the most comprehensive food safety reform in 70 years requires a massive commitment from federal, state and local governments to food producers, farmers and other stakeholders that are working to protect the public health in new and innovative ways. That’s why in July we announced more than $30 million in funding for states to help implement new produce safety requirements. We also launched an innovative software tool called the Food Safety Plan Builder that assists food manufacturers in creating a food safety plan to help prevent foodborne contamination and ultimately protect public health. And we’re looking at other ways to empower farmers and producers to ensure the law’s modernized requirements are effectively fulfilled.

I also believe in empowering people to make better choices. This is reflected in our continued efforts to pursue the practical implementation of the menu-labeling rule. We listened to public feedback and have proposed practical solutions to make it easier for industry to meet obligations in these important public health endeavors, while ensuring restaurant patrons have access to the nutrition information they need.

Empowerment also is critical for patients facing life-threatening or debilitating illness. This year we held our first-ever patient engagement advisory committee meeting. This is a pioneering effort that seeks to strengthen our engagement with patients and secure the patient voice in our regulatory decision-making.

We also understand that in many serious diseases, patients want earlier access to experimental treatments. We’ve taken new steps to improve the expanded access resources we have to serve patients, including enhancing our online Expanded Access Navigator Tool and simplifying the process for approving a patient’s request for access to an investigational treatment. Last month, as part of our commitment to expediting drug development for rare diseases, we issued draft guidance that describes a possible new approach for companies to collaborate and test multiple drug products in the same clinical trials for a specific ultra-rare pediatric disease, thereby reducing the number of patients that need to be treated with placebo. This framework can be applied more widely to other ultra-rare diseases.

In everything we do at FDA, our top priority is to protect the public health. Perhaps nowhere was this more evident in 2017 than in the areas of the United States that were impacted by last year’s hurricanes.

The devastation caused by Hurricanes Harvey, Maria and Irma brought to public view some of the critical work FDA does in overseeing the safety of the food and medical products. We worked around the clock to ensure that farmers in Texas and Florida could safely handle their crops affected by flooding. We remain deeply committed to the recovery in Puerto Rico and that island’s long-term success. We worked closely with drug and medical device manufacturers in Puerto Rico to take steps to address potential and apparent shortages of medical products that resulted from the devastation left by Hurricane Maria.

Although we’re seeing progress in Puerto Rico owing to the hard work of federal and local authorities — and primarily because of the resilience of the American people who are affected — our work and commitment to hurricane victims and patients in need of critical medical products will continue into 2018.

Improving our Stewardship of Vital Drugs

Antimicrobial resistance continues to be a major public health challenge. Addressing this problem requires adoption of a “one health” approach that involves new efforts to use antibiotics more responsibly in both human and animal medicine. To better manage antibiotic usage in food animals, in 2017 FDA completed its implementation of a groundbreaking Guidance for Industry (GFI #213). This new guidance document eliminates the use of medically important antimicrobials for food production purposes and brings the remaining therapeutic uses of antibiotics in animals under veterinary oversight.

A total of 292 approved drug applications were impacted by this new guidance; with 84 drug applications withdrawn, 93 applications for oral dosage form products intended for use in water converted from over-the counter (OTC) to prescription, and another 115 applications for products intended for use in feed converted from OTC to Veterinary Feed Directive. This is a major accomplishment. It represents a milestone in our efforts to promote judicious use of antibiotics in animal health. We will take additional steps in 2018 to build on these successes, and improve stewardship over medically important antibiotics.

A look ahead to 2018

When I look back at my eight months as Commissioner, since coming aboard at FDA in May, I’m humbled by the many accomplishments of the agency’s dedicated professional staff.

We’ve achieved a great deal in 2017. We’re committed to making even more progress this year. 2018 holds promise in even more areas where the agency will take steps to advance beneficial innovation by adopting new measures to make sure our processes are efficient, human and animal products are safe, and practical solutions are implemented that protect and promote the public health. The launch of our Unified Agenda highlights some of our priorities. These include advancing biosimilar policies, modernizing how we advance over-the-counter products, and better informing women about health issues and risk factors.

Nobody innovates in a silo. Advancements in medicine, biotechnology, food science, and the whole of public health are possible only because of the collaboration of the public health community. FDA is in a unique position to bring together stakeholders from across the sector — patients, industry, academics, providers, other government agencies — to ensure innovation translates into successful outcomes that protect and benefit the public. That’s what drives us at FDA. It’s what we’ll pursue in the year ahead.

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

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

Looking ahead: Some of FDA’s major policy goals for 2018

By: Scott Gottlieb, M.D.

Twice a year the federal government publishes the “Unified Agenda of Federal Regulatory and Deregulatory Actions” (Unified Agenda), which provides the American public with insight into regulations under development or review throughout the federal government. For the U.S. Food and Drug Administration (FDA), it gives us an opportunity to outline some of our efforts to modernize our approach to our work and improve our efficiency, while fulfilling our mandate to protect and promote the public health and uphold FDA’s gold standard for regulatory decision-making. While many of FDA’s policies are advanced through guidance documents and other proposals, this annual list of proposed regulations provides one element of our policy agenda.

Dr. Scott GottliebPatients and consumers across our country depend on us to regulate products in a predictable, efficient, science-based manner. We also serve the public health by efficiently advancing innovations and therapies that improve patient care, enhance choice and provide competition; by aggressively taking action against serious threats to public health, such as opioid addiction and addiction to the nicotine in cigarettes; by empowering patients, consumers and healthcare providers with accurate and up-to-date information; and by recognizing when scientific innovations warrant new, more flexible regulatory approaches in order to make sure advances in care can reach patients. In addition to these goals, we must continually adapt our regulations to enhance efficiency, improve our effectiveness, and update old and out-of-date requirements.

FDA’s contributions to the Fall 2017 Unified Agenda address a number of these areas of policymaking under way at the agency, and are directly aligned with our key priorities:

Addressing the Nicotine Addiction Crisis

To reduce the morbidity and mortality associated with combusting tobacco, we are proposing meaningful actions to advance our new, comprehensive approach to nicotine and the regulation of combustible cigarettes. These efforts include an Advance Notice of Proposed Rulemaking asking critical questions related to our pursuit of regulation that would result in a targeted reduction of the nicotine levels in combustible cigarettes to eliminate or dramatically reduce their addictive value. At the same time, FDA is taking new steps to facilitate innovation in products that can deliver satisfying levels of nicotine to adults who want or need such access without the same health risks associated with combustible tobacco.

As part of this plan, FDA will also be issuing an Advanced Notice of Proposed Rulemaking to look at how to best regulate flavors in tobacco products to limit their appeal to youth, while considering the potential role that some flavors may play in helping users transition away from combustible products. Further, FDA will be issuing an Advance Notice of Proposed Rulemaking to solicit information that may inform regulatory actions FDA might take with respect to premium cigars, asking certain questions related to how we might define and regulate “premium cigars,” taking into consideration the health effects of these products and their patterns of use.

Advancing Drug Safety

FDA will issue several regulations on drug compounding to help ensure the quality of medicines that patients need. We want to make sure that outsourcing facilities clearly understand which drugs they may compound and allow these firms to adopt more efficient, streamlined manufacturing standards, while ensuring they observe necessary safety and quality measures.

Focusing on the safety of prescription drugs, FDA is also pursuing a proposed rule to establish national standards for the licensing of prescription drug wholesale distributors and third-party logistics providers, as part of track-and-trace requirements. By establishing national standards for all State and Federal licenses issued to key parts of the supply chain, these regulations will allow for the effective and efficient distribution of prescription drugs throughout the U.S.

Promoting Food Safety

FDA continues to take steps to improve its oversight of food safety. To address critical issues related to the overall safety of the food we eat, FDA intends to propose a rule on lab accreditation, which would establish a program to accredit labs to do food safety testing and to require that these accredited labs be used in certain situations.

Additionally, in the Unified Agenda, FDA committed to pursuing a rulemaking that will clarify registration requirements for food facilities to better align how facilities and farms that perform similar activities are treated under the preventive controls rules and the produce safety rule.

Empowering Consumers

Many of our agenda submissions are part of a broader effort to empower consumers and patients to make more informed and effective health decisions and ensure they have appropriate autonomy over their choices, while continuing to ensure the products they consume and use are safe and effective. Consumers tell us that they want this information. We also know that consumers who have access to more diverse, safe and effective options – and who have improved information about those choices – make better, more cost-effective decisions. 

  • Providing Better Information on Drugs: We have included a rulemaking that proposes a new type of patient medication document that would help ensure that patients have access to clear, concise, and useful written information about their prescription drugs or biologics, delivered in a consistent and easily understood format, each time they receive a medication from the pharmacy. We want to give patients the ability to make high value decisions about the medicines they take, and help them use drugs safely and effectively.
  • Broadening Access to Nonprescription Drugs: We are considering innovative action in the nonprescription drug area to expand the scope of drug products that can be made available to consumers without a prescription. We will be proposing to allow certain innovative approaches for demonstrating that a drug product can be used safely and effectively in a nonprescription setting. This will allow some drugs that would otherwise require a prescription to be marketed without a prescription through the use of innovative technologies and other conditions that will ensure appropriate self-selection and/or appropriate actual use of the nonprescription drug product by consumers. Examples of such conditions could include use of self-selection questions on a mobile medical app prior to permitting access to the drug, or other innovative technologies to improve safety. Through use of these types of additional conditions, we hope to create a new paradigm of drug safety with greater flexibility that will benefit patients and public health. We are committed to advancing this new framework to enable a potentially broader selection of nonprescription products for consumers, empowering them to self-treat more common conditions and chronic conditions. This also could help lower costs by increasing the availability of products that would otherwise be available only by prescription.

Modernizing Standards

Importantly, we also are working to ensure efficiency of existing regulations – a key focus of the Unified Agenda – by making sure that our standards are clearly defined, that they advance our public health goals and help promote the protection of consumers, and achieve these goals in an efficient way that does not place unnecessary burdens on those we regulate. We also want to ensure that our standards and regulations are modern and reflect the latest science, and have not become outdated, obsolete or otherwise not applicable to the current environment.

  • Harmonizing Global Standards: We will be updating FDA’s requirements for accepting foreign clinical data used to bring new medical devices to market. While helping to ensure the quality and integrity of clinical trial data and the protection of study participants, this rule should also reduce the burden on industry because it will harmonize with the standards currently used in drug regulation.
  • Modernizing Mammography Standards: We will be proposing a rule to modernize mammography quality standards that will improve women’s health. Our aim is to recognize advances in technology and help to ensure women get the most relevant, up-to-date information about their breast density, which is now recognized as a risk factor for breast cancer. This information can help doctors and patients make more informed decisions about further imaging.
  • Embracing Electronic Submissions: We will propose a new framework that will allow FDA and product developers to take greater advantage of the efficiency of electronic, rather than paper, submissions for devices and veterinary drugs.
  • Removing Outdated Rules: We will propose to remove an outdated inspection provision for biologics and outdated drug sterilization requirements to remove barriers to the use of certain sterilization techniques.

Looking to the Future

FDA serves Americans by delivering on the critical mission of protecting and promoting the public health. The more than 70 actions we have identified, as part of the Fall 2017 Unified Agenda, will help us even better deliver on this mission. But regulation is only one way in which we can foster our mission and improve American health.

Over the next year, we will also tackle many additional priority areas through guidance documents and other policy efforts. These areas will include efforts to reduce the cost of drugs by encouraging competition, spur innovation across medical products, give consumers access to clear and consistent nutrition information, create greater regulatory efficiencies in bringing products to market, and put a dent in the opioid addiction crisis facing our country.

Further, just because a previously identified regulation does not appear on this Unified Agenda submission does not necessarily mean the agency does not consider it a priority or will not continue to consider it moving forward. Look for additional information about the many initiatives identified in the Fall 2017 Unified Agenda as we advance all of these goals.

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

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

Advancing Medicinal Nicotine Replacement Therapies as New Drugs – A new step in FDA’s comprehensive approach to tobacco and nicotine

By: Scott Gottlieb, M.D., Janet Woodcock, M.D., Mitchell Zeller, J.D.

As the leading cause of preventable disease and death in the United States, tobacco causes more than 480,000 deaths every year. To address this devastation, earlier this year FDA announced a new regulatory plan to lower this burden of tobacco-related disease and death. The plan takes a comprehensive approach to nicotine and tobacco, including an initiative to lower nicotine in cigarettes to minimally addictive or non-addictive levels. Aimed at shifting the trajectory of tobacco-related disease and death, the agency’s approach recognizes that nicotine is delivered through products posing a continuum of risk. This ranges from combusted cigarettes at one end, to nicotine replacement therapy (NRT) products – designed to safely reduce withdrawal symptoms, including nicotine craving, associated with quitting smoking – at the other.

Dr. Scott Gottlieb

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

Today, FDA is taking an additional step in our new, comprehensive approach to the regulation of nicotine and tobacco. We are announcing the formation of a new Nicotine Steering Committee that will be charged with re-evaluating and modernizing FDA’s approach to development and regulation of nicotine replacement therapy products that help smokers quit. These products are typically sold as over-the-counter drugs in the form of gums, patches and lozenges.

The primary focus of the new Committee will be on these therapeutic nicotine products for combustible tobacco product cessation. This reflects the need to critically examine the evolving science behind FDA’s evaluation of the safety and efficacy of NRTs. Key topics will be the types of safety and efficacy studies we require and the way these products are used and labeled.

The aim is to make sure FDA has the right policies in place to enable the development of product innovations that have the potential to be more helpful in helping smokers quit combustible cigarettes and maintain abstinence. This could include changes to the labeling and indications for existing products or a new product that might deliver nicotine at different rates, or through different delivery mechanisms entirely. To enable innovation, FDA might contemplate additional approaches to developing these products, including new clinical trial endpoints.

Janet Woodcock

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

This new Committee will include Senior Agency Leadership from the Center for Tobacco Products, the Center for Drug Evaluation and Research, and the Office of the Commissioner. While one significant focus will be on NRT, the mandate of this committee will be to address the FDA’s overall approach to nicotine. It will create a forum for developing and implementing nicotine policy and regulation to address the public health crisis of tobacco usage in this country.

Among adult smokers in the U.S., around 70% report that they want to quit. Nearly half try to quit each year, and few succeed. Many treatment-seeking smokers often use medications such as NRTs. FDA has approved nasal spray and inhaler NRTs for prescription-only use; and gum, transdermal patch and lozenge formulations for over-the-counter (OTC) sale. In addition, FDA has also approved  pharmacological therapies to aid in smoking cessation.

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

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

FDA has found these therapeutic products to be safe and effective in helping smokers quit. Using approved NRTs is generally considered to double the likelihood of a successful quit attempt, with variations between products. While most existing NRTs have been approved for over 20 years, novel forms of nicotine delivery are being developed based on new technologies and innovations. These may develop into FDA-approved therapies that help more smokers quit.

When it comes to the discussion around transitioning current smokers to safer alternatives for the delivery of nicotine, a lot of public debate, to date, has been placed on the potential for modified risk tobacco products like electronic nicotine delivery systems and e-cigarettes. FDA also sees compelling opportunities to explore additional opportunities for the development of new and improved products that can be sold as new drugs, typically as over-the-counter pharmaceuticals.

If there are new kinds of NRTs — with different characteristics or routes of delivery – that can offer additional opportunities for smokers to quit combustible tobacco, we want to explore what steps we can take using our own regulatory policies to enable these opportunities, while making sure these products are demonstrated to be safe and effective for their intended use.

We’ll be joined on the Nicotine Steering Committee by Rachel Sherman, M.D., M.P.H., Principal Deputy Commissioner, who will chair the Committee; Grail Sipes, J.D., Director, Office of Regulatory Policy, CDER; Melissa A. Robb, B.S.N., M.S., Associate Commissioner for Clinical Policy, Office of Medical Products and Tobacco (OMPT); Allison Hoffman, Ph.D., Associate Director for Health Science, OMPT; and Priscilla Callahan-Lyon, M.D., Deputy Director, Division of Individual Health Science, CTP.

Public participation will be central to the Committee’s discussions. As the Committee’s first action, we’ve scheduled a public hearing on Jan. 26, 2018, to consider FDA’s Approach to Evaluating Nicotine Replacement Therapies. We encourage those interested to  join the discussion on this important topic, including stakeholders from the public health community, researchers, health care professionals, manufacturers, and industry and professional organizations. FDA believes it is critical to obtain input from the public on how evolving science could influence the agency’s approach to evaluating the safety and effectiveness of NRT products.

FDA’s approach to the regulation of nicotine is a key element of its efforts to address crises of addiction that are impacting families. The nicotine in cigarettes doesn’t directly cause tobacco-related cancer, lung disease, or heart disease. But the powerfully addictive nature of the delivery of nicotine in combustible cigarettes makes tobacco use the leading cause of preventable death in the United States. So FDA is putting nicotine at the center of our regulatory strategy.

We’re taking steps to render combustible cigarettes minimally- or non-addictive. This could prevent future generations of kids from becoming addicted to cigarettes, the deadliest form of nicotine delivery. At the same time, FDA is committed to the proper development of products that can allow adults who still need or want to enjoy satisfying levels of nicotine to get it through products that don’t have all of the risks associated with the combustion of tobacco. Our goal is to enable greater use of safe and effective options to help those who are addicted to nicotine get the help they need to quit combustible cigarettes altogether.

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

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

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

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

“This is Our Watch”: FDA Supports Retailers in Keeping Tobacco Out of Youth’s Hands

By: Mitch Zeller, J.D.

Mitch Zeller, J.D., Director of FDA's Center for Tobacco ProductsEach day in the United States, nearly 2,500 youth under age 18 smoke their first cigarette and more than 400 become daily cigarette smokers. In fact, more teenage boys light up a cigar each day (about 1,300) for the first time than light up a cigarette (about 1,100). Tobacco retailers – including store owners, managers, and clerks—have an important responsibility to protect teenagers from tobacco-related disease and death by not selling tobacco products to minors.

FDA has created a new retailer education program called “This is Our Watch” to help retailers comply with FDA regulations prohibiting the sale of tobacco products to minors. These regulations require, for example, that retailers check photo ID to verify that a person wanting to purchase a tobacco product is at least 18 years old.

FDA’s “This is Our Watch” program provides free resources to help retail store owners and their employees understand how to comply with these federal requirements. Specifically, packages are being mailed to retailers that contain a store poster, stickers and signs for the register, flyers explaining federal requirements, and a calendar to help verify a person’s age.

Retailers should expect to receive these educational materials in the mail within the coming weeks. And additional copies of the materials can be downloaded or ordered at no cost.

Calendar at retailer check-out counter

FDA has created a new retailer education program called “This is Our Watch” to help retailers comply with FDA regulations prohibiting the sale of tobacco products to minors. These regulations require, for example, that retailers check photo ID to verify that a person wanting to purchase a tobacco product is at least 18 years old.

This information is meant to be shared with store employees – including managers and clerks – and can be placed behind the counter where they are easy to see. The materials can also help retailers maintain positive relationships with their customers by informing patrons of the law and emphasizing the role that retailers play in protecting kids from the harm of tobacco products.

“This is Our Watch,” which updates FDA’s “Break the Chain of Tobacco Addiction” retailer education program launched in 2010, was designed based on input from retailers across the country. We created these resources bearing in mind that tobacco product retailers vary—from convenience stores to grocery stores to specialty shops. These new resources are designed to be clear and direct in helping owners and employees adhere to federal requirements.

The resources cover major federal requirements meant to prevent the sale of tobacco products to minors and save lives. Among these requirements, retailers must:

    • Check photo ID of everyone under age 27 who attempts to purchase any tobacco product.
    • Only sell tobacco products to customers age 18 or older.
      • Retailers must also follow state/territory tobacco laws, even if they are more restrictive. For example, the minimum age is 19 in some states.
    • Never sell tobacco products in a vending machine unless it is an adult-only facility.
      • An adult-only facility is one in which minors are neither present nor permitted to enter at any time.
    • NOT give away free samples of tobacco products, including any of their components or parts, to consumers.

In addition to offering compliance training and education programs such as “This is Our Watch,”  FDA also conducts inspections of retail establishments to monitor for compliance and, when violations are observed, takes action in the form of warning letters, civil money penalties, and no-tobacco-sale orders.

When it comes to preventing youth tobacco use, FDA relies on the retail community to protect their young customers by keeping those under 18 from purchasing tobacco products. More information is available on FDA’s website about federal rules and regulations relevant to the tobacco retailer community. People may also contact the Center for Tobacco Products’ Office of Compliance and Enforcement at 1-877-1373 or CTPCompliance@fda.hhs.gov.

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

FDA’s plan to engage the public in the agency’s new effort to strengthen and modernize FDA’s regulatory framework

By: Anna Abram

Anna AbramWe’re at a moment of extraordinary opportunity to improve the public health. New innovations are giving us fundamentally better ways to address disease. Some of the same technology is providing consumers with a broader selection of foods that can improve peoples’ diets, and products that can expand their choices. At the same time, we also face a lot of new challenges. The steps FDA takes to advance these opportunities, and address uncertainties, will directly impact the lives of families. As part of our commitment to protect and promote the public health, we’re undertaking a comprehensive review of our regulations. Our aim is to ensure that our policies and regulations keep pace with the challenges we face in protecting consumers, and the opportunities we have to improve their lives. As in all our actions, science will remain FDA’s North Star when it comes to our role in devising regulatory policy.

Over the past few months, FDA has announced a number of broad policy efforts to address public health opportunities in areas such as regenerative medicine, tobacco products, and access to affordable medicines. As with everything that we do, this work is rooted in our mission to protect and promote the public health, foster safe and effective innovation that can benefit patients, adopt regulatory approaches that enable the efficient development of new innovations, and provide for a safe, healthy and nutritious food supply. For example, we’re looking at places where FDA’s rules concerning new drugs are being used in ways that may create obstacles to the timely entry of generic competition. We want to make sure our policies aren’t being misused in ways that thwart the competition that Congress intended when it created the modern generic drug framework. We know that vigorous generic competition can help benefit patients by lowering drug costs, which improves access to medicines. It’s one example of where a closer analysis of our existing policies can help make sure our regulations are having their intended purpose.

As part of my commitment to help oversee the development and implementation of key policy issues, and to help advance these broader policy efforts, I’ve been working closely with FDA Commissioner Scott Gottlieb, M.D., and other senior agency colleagues, to explore ways to modernize our regulations in a manner that will benefit all Americans. To achieve this, we’re not only looking at what new regulations or policies we need in order to be most effective in fulfilling our public health responsibilities. We’re also taking a closer look to see if we need to revise, update, and in some cases eliminate existing regulations to help us better keep pace with scientific advancement and the people that we serve. We need policies that are as modern as the products that we’re being asked to evaluate, and a regulatory framework that uses efficient tools to achieve our vital consumer protection role.

This comprehensive review is a large undertaking given the breadth of our public health mission and the fact that FDA-regulated products account for about 20 cents of every dollar consumers spend each year. It involves the support of FDA’s senior leaders and many of our staff. FDA has long played a vital role in protecting and promoting public health, and since its founding in 1906, the scope and charge of the agency’s work has grown to include many products that Americans rely on every day. Over time, the agency has also assumed an increasingly global footprint. Along the way, we’ve taken many steps to modernize our policies and practices and evaluate our portfolio of regulations to make sure they’re keeping pace with our challenges. But our 100-plus-year history lends itself to a closer examination of the regulations that have guided our work. Some have been in place for decades, and may not reflect the most up-to-date approach to achieving our public health mission. We have a lot of ground to cover. Today, FDA’s regulations comprise more than 4,000 pages in the Code of Federal Regulations. Some regulations may not adequately reflect advances in science, technology or changes in industry practice. Others may be geared toward products and practices that have largely ceased to exist. In a world of increasing challenges and opportunities, we need to be risk-based in everything we do in order to make sure we’re using our resources efficiently. Our goal is to have regulations that reflect modern risks and opportunities, and use the full scope of our authorities to achieve our consumer protection mission.

As part of this process we’re asking ourselves and others to think about how current regulations could be reshaped to achieve our public health objectives through more efficient approaches. We are opening a number of public dockets to solicit feedback from patients, consumers, health providers, caregivers, industry, health groups, academia, as well as state, local and tribal governments, and public health partners. We’re also exploring other opportunities to solicit input from stakeholders on this effort. We believe that engaging both internal and external stakeholders are critical to focusing our attention on where our policies might need updating; to ensure FDA’s work maximizes our public health purpose.

Our approach also aligns our efforts with the Administration-wide goal for federal regulatory reform to improve how government serves the American people. At FDA we take seriously our responsibility to protect and promote the public health. This will be our guiding principle. We’ll use this opportunity to make sure our regulations reflect the new benefits that science and technology offer to advance opportunities for patients to improve their lives, and to strengthen our mandate to protect consumers. We look forward to your input and will continue to communicate our plans as we move forward on this endeavor.

Anna Abram is FDA’s Deputy Commissioner for Policy, Planning, Legislation and Analysis

For more information on how to provide input on FDA’s regulations, please visit:

Tobacco Product Master Files: What Are They and How Could They Benefit You?

By: Mitch Zeller, J.D.

In a bid to help streamline and simplify the tobacco product application process and potentially reduce the associated costs, FDA is encouraging the creation and use of tobacco product master files (TPMFs).

Mitch Zeller, J.D., Director of FDA's Center for Tobacco ProductsTPMFs are voluntary submissions to FDA that typically contain trade secrets and/or confidential commercial information about a tobacco product that the owner does not want to publicly share with others. But this information may be referenced by other parties in preparing tobacco submissions if the TPMF owner grants those other parties a right of reference.

Here’s how TPMFs are designed to work. Once a master file is submitted, anyone interested in referencing that file as part of their own submission may request a letter of authorization from the TPMF owner. If the request is granted, FDA reviewers will be able to access and review non-public information in the TPMF according to the authorization in the course of reviewing the submission, even if that information is not available to the authorized party.

For example, a flavor supplier might submit a TPMF to FDA that contains the full listing of ingredients and composition information of different flavors. The flavor supplier could then grant a customer, for example, an e-cigarette manufacturer, the right to reference the TPMF to support the customer’s premarket application for an e-cigarette that uses the supplier’s flavors. When reviewing the premarket application, FDA would also review ingredient and composition information in the TPMF, which may otherwise be confidential, without that information being shared with the customer.

TPMFs can be mutually beneficial for both file owners and parties authorized to reference the files in their own applications—ultimately resulting in potential cost savings for all parties involved. For TPMF owners, allowing FDA to keep certain information on file can reduce administrative work for the company and the need to resubmit this data for future applications, thus easing the application burden. Additionally, when a TPMF owner grants another entity access to a master file, it enables the authorized party to reference scientific data and analyses in the file that otherwise could be costly and time-consuming to obtain if the party, itself, needed to produce the information.

By filing TPMFs with FDA, owners gain the ability to expand existing customer relationships and to foster new ones while also keeping the owner’s information confidential.

Tobacco product submissions that may reference a TPMF include:

  • Premarket tobacco applications
  • Substantial equivalence reports
  • Modified risk tobacco product applications
  • Request for exemption from substantial equivalence requirements
  • Grandfathered submissions
  • Investigational tobacco product submissions
  • Ingredient listings

There are many ways TPMFs can be used in different types of applications. For instance, a lab could create a TPMF that contains protocols and methods for measuring levels of harmful and potentially harmful constituents (HPHCs) in tobacco products. An authorized manufacturer that used the lab’s services to generate HPHC data for, e.g., a substantial equivalence report could reference the lab’s TPMF in its submission to FDA, helping FDA evaluate HPHCs in its tobacco products.

FDA does not intend to conduct scientific review of a TPMF until the TPMF is referenced in a submission.

To learn more about the many benefits of becoming a TPMF owner, see “Tobacco Product Master Files: Guidance for Industry.” Contact us at ASKCTP@fda.hhs.gov or 1-877-CTP-1373 (1-877-287-1373).We will present a webinar on TPMFs soon, so stay tuned and sign up for email updates.

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

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