The American Chamber of Horrors

By: Vanessa Burrows, Ph.D., Suzanne Junod, Ph.D., and John Swann, Ph.D.

In the early 20th century, Americans were inundated with ineffective and dangerous drugs, as well as adulterated and deceptively packaged foods.

A cosmetic eyelash and eyebrow dye called Lash Lure, for example, which promised women that it would help them “radiate personality,” in fact contained a poison that caused ulceration of the corneas and degeneration of the eyeballs. An elixir called Banbar claimed to cure diabetes as an alternative to insulin, but actually provided no real treatment and caused harm to those patients who substituted this for effective insulin therapy. Food producers short-changed consumers by substituting cheaper ingredients. Some products labeled as peanut butter, for instance, were filled with lard and contained just a trace of peanuts, and some products marketed as “jellies” had no fruit in them at all.  Unscrupulous vendors even sold products to farmers, falsely promising they could treat sick animals – in at least one case, a product called Lee’s Gizzard Capsules killed an entire flock of turkeys instead of curing them.

Although the FDA sought to remove these unsafe and misleading products from commerce, it was severely limited in its efforts by the 1906 Pure Food and Drugs Act.  That law laid the cornerstone for the modern FDA and marked a monumental shift in the use of government powers to enhance consumer protection by requiring that foods and drugs bear truthful labeling statements and meet certain standards for purity and strength.

Over time, however, the shortcomings of the Pure Food and Drugs Act became apparent, as it failed to take into account the extraordinary changes in industries, products, markets, and advertising tactics. Dangerous drugs were a particular problem. As long as a drug met the law’s labeling requirements, the agency did not have the authority to remove even clearly dangerous products such as radium water and drugs with poisonous ingredients from the market because legal action against a drug product required a finding of fraud. If a drug’s maker could convince a court that he truly believed his own therapeutic claims, he won his case. In addition, the law provided no authority over cosmetics or medical devices, and did not specifically authorize standards for foods, which limited the agency’s ability to take action on behalf of consumers.

A popular book of the day, “100,000,000 Guinea Pigs: Dangers in Everyday Food, Drugs, and Cosmetics,” claimed that consumers were being used as guinea pigs in a giant experiment by food companies and makers of patent medicines, with the authors blaming the FDA for failing to act. But the critics failed to acknowledge the limits of the agency’s authority under the law at the time.

In an effort to inform the public about the 1906 law’s shortcomings, the FDA’s Chief Education Officer, Ruth deForest Lamb, and its Chief Inspector, George Larrick, led the creation of an influential traveling exhibit in 1933 to highlight about 100 dangerous, deceptive, or worthless products that the FDA lacked authority to remove from the market.

The exhibition was put on display at events like the 1933 World’s Fair in Chicago, at state fairs, and on Capitol Hill. It was so shocking that it was dubbed the “American Chamber of Horrors” by a reporter who accompanied First Lady Eleanor Roosevelt to view the exhibit. Lamb also adapted the exhibit into a 1936 book in which she explained, “All of these tragedies…have happened, not because Government officials are incompetent or callous, but because they have no real power to prevent them.”

The exhibit, which was viewed by millions, was an enormous success, helping promote greater awareness and understanding about the FDA’s role in protecting the public and the need for greater consumer protection and the limitations on its power to do so. To this end, it played an important role in moving Congress to enact a stronger food and drug law – the 1938 Food, Drug, and Cosmetic Act.

The 1938 law, which has been amended many times and remains the law of the land today, brought cosmetics and medical devices under the FDA’s authority, and required that drugs be labeled with adequate directions for safe use. It also mandated pre-market approval of all new drugs, such that a manufacturer would have to prove to the FDA that a drug was safe before it could be sold. And it prohibited false therapeutic claims for drugs. The Act also corrected abuses in food packaging and quality, and it mandated legally enforceable food standards. It formally authorized factory inspections, and added injunctions to the agency’s enforcement tools. In short, it gave the FDA many of the means it has today to protect the American public.

Many of the products from the original Chamber of Horrors exhibit are in the FDA’s permanent collection, and, to commemorate the 80th anniversary of the 1938 law, they are part of a special display currently on exhibit at the FDA. The objects provide a compelling visual record of how far science has brought us from the worthless and dangerous elixirs, foods, and other consumer products of the early 20th century, as well as underscoring the essential role the FDA today plays in protecting and promoting American health.

Vanessa Burrows, Ph.D., Suzanne Junod, Ph.D., and John Swann, Ph.D., are FDA Historians

FDA’s New Efforts to Advance Biotechnology Innovation

By: Scott Gottlieb, M.D., and Anna Abram

Scientific advances in biotechnology, such as genome editing and synthetic biology, hold enormous potential to improve human and animal health, animal welfare, and food security. And researchers and companies based in the United States helped pioneer these technologies. They position the U.S. as a global leader of this rapidly growing and highly promising field.

Dr. Scott Gottlieb

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

To advance this progress, it’s key that the FDA adopt a regulatory approach to these technologies that’s as innovative and nimble as the opportunities that we’re tasked with evaluating.

FDA is committed to helping ensure the safety of biotechnology products, while also facilitating innovation by applying a risk-based regulatory approach that provides developers with regulatory clarity and predictability and maintains public confidence in our regulatory system.

And we’re taking some new steps to advance these goals. We know that products enabled by new techniques of biotechnology have the potential to significantly enhance public health.

For instance, these new methods can be used to alter animals to minimize or prevent their ability to spread human disease. Genome editing in animals and plants also can be used to produce human drugs, devices, or biologics, including tissues or organs for xenotransplantation. Scientists are also exploring editing the genomes of animals with the goal of improving the health and welfare of food producing animals and public health, for example by reducing their susceptibility to diseases like novel influenzas and resistance to zoonotic or foreign animal diseases.

Similar and equally beneficial applications of genome editing are currently being explored in food crops. These include our ability to develop disease-resistant plants and plants with increased resistance to environmental stress. Such advances can have many advantages to consumers, including better yields, more product variety, and healthier nutrient profiles.

Anna Abram

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

We believe the FDA is uniquely positioned — with the expertise, experience, credibility and trusted scientific framework — to advance innovation and support the development of products with immense potential for public benefit. And we’re fully committed to these goals.

The breadth of FDA’s statutory authorities and regulatory framework allows us to comprehensively review the potential impacts of products on both human and animal health. For example, for genetically engineered animals, FDA evaluates not only the safety of food or drug products derived from that animal, but also the effect of the genetic alteration on the health of the animal. FDA has decades of experience successfully evaluating products of complex technologies, such as recombinant DNA-derived plant foods, medicines made with nanotechnology, and cellular and gene therapy products.

Moreover, because of the wide spectrum of products that we regulate, and the in-depth scientific and policy engagement that the agency has with innovators and counterpart regulatory agencies around the world, FDA can help facilitate the progression of research and development. For example, we’re focused on the timely transition of technologies from animal research models to products intended for use in humans. As our knowledge of genome editing applications increases over different product areas, we expect to build on those even greater synergies and increase our understanding to help with assessments of risks to human and animal health.

FDA will continue to apply a risk-based framework grounded in sound science to evaluate products of plant and animal biotechnology, and our framework will continue to evolve as science advances and experience with these technologies grows. We also look forward to working with stakeholders to help understand current scientific information and describe challenges and gaps in regulatory science that are important for our regulatory decision-making. We’re also going to take new steps to help developers understand their responsibility to ensure product safety and we’ll identify ways to help reduce unnecessary regulatory burden and undue barriers to bring potential beneficial products to commercialization while ensuring their safety.

Protecting and promoting public health is our mission and we’re taking steps to help ensure the safety (and as applicable, effectiveness) of products that can benefit patients and consumers, while supporting innovation and sustaining public confidence.

To help advance these goals, in early May, FDA formed a new Biotech Working Group. This Working Group is comprised of representatives from multiple FDA centers and offices. In the coming months, we’ll release an Action Plan that lays out the steps we intend to take to ensure that we have a flexible regulatory framework for evaluating the safety of products that also supports plant and animal biotechnology innovation.

Our actions will focus on three key areas:

First, advancing and protecting public and animal health by promoting innovation through an efficient and predictable science- and risk-based regulatory framework; second, strengthening public outreach and communication through strong, effective and transparent engagement with stakeholders; and third, increasing engagement with domestic and international partners through coordinated and collaborative actions to support regulatory alignment and efficiency.

The Working Group’s efforts are well underway and we’ll be providing more details soon.

Finally, we’ll continue the work we began to modernize the regulatory system for biotechnology, including the effort in 2015 with USDA and EPA to ensure preparedness of federal regulatory agencies for future products of biotechnology; as well as the implementation of the 2018 recommendations of the Interagency Task Force on Agriculture and Rural Prosperity. We’ll also continue to build on our Formal Agreement with the USDA, which commits the FDA and USDA to better align and enhance our efforts to develop regulatory approaches to biotechnology.

We’re committed to all of these goals, and we look forward to working with the Interagency Task Force and sharing more of our important work with our stakeholders going forward.

FDA is taking concrete and proactive steps to help ensure the safety of plant and animal biotechnology products, while promoting innovation and enhancing public and market confidence in FDA’s regulation of these products at home and abroad. We recognize the tremendous opportunities offered by this new technology. We’re committed to developing a framework that allows these innovations to safely advance, to fulfill the potential envisioned by those who are pioneering these approaches, and to inspire public confidence in these methods.

The advance of these technologies holds significant public health promise. Unlocking their full potential and competitiveness depends on the trust we build now and in the years to come.

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

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

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

FDA to Expedite Release of Recall Information

By: Douglas Stearn, J.D.

When FDA identifies that a product it regulates violates the law, it protects the public by working with the manufacturer and distributors to facilitate the product’s recall (i.e., removal from the marketplace or product correction). Among other actions, FDA assures that the public is warned when products present the most significant public hazards, including those recalls associated with an outbreak.

Douglas StearnNow, as part of a larger effort to increase transparency, empower consumers, and enhance public health, the FDA is working to alert the public sooner whenever a product has been recalled.

The public’s primary source for recall information is FDA’s weekly, web-based Enforcement Report. Historically, only recalls that have already been classified into one of three categories based on the severity of the hazard have been listed in the report. Through classification, FDA indicates the relative degree of health hazard presented by the recalled product. This enables consumers to better understand the severity of the problem posed by a recalled product so they can take appropriate action. FDA also provides guidance to companies on their recall strategies, taking into account the seriousness of the hazard presented by the recalled product.

However, recall classifications can sometimes take weeks – or even months when FDA needs to conduct a complex evaluation. Such analysis can involve determining whether any diseases or injuries have already occurred, the likelihood that a hazard might occur, or whether vulnerable segments of the population, such as children, are more at risk.

FDA has decided that the public would benefit by having recall information about FDA-regulated products as soon as possible, even though further evaluation remains to be done. Moving forward, FDA will include “not-yet-classified” recalls of human drugs, foods, and veterinary products in the weekly Enforcement Report, even while classification work is still ongoing.

As an example of how this will work, FDA recently posted a “not-yet-classified” recall of a certain lot of animal feed that contained monensin, which can be toxic to cattle at certain levels and could result in cardiovascular illness or death to cattle.

Posting “not-yet-classified” recalls will not affect current FDA protocols for working with companies to ensure that they quickly alert entities in the supply chain as soon as they have identified a problem with their marketed product. Also, FDA will continue to monitor the recalling company’s actions to correct or remove products held by retailers, pharmacies, grocery stores, and hospitals.

In addition to adding “not-yet-classified” recalls of human drugs, food, and veterinary products to the Enforcement Report, FDA recently began posting early summaries of correction or removal actions involving serious problems with medical devices in the Medical Device Recalls Database. FDA has also enhanced the website’s search capabilities. Returning to the site after viewing “not-yet-classified” recalls is recommended because more information is often made available once a recall has been classified.

RECALL CLASSIFICATIONS
Classification Risk Definition Examples
Class I Highest Reasonable probability that use/exposure will cause serious adverse health consequences or death. Defective pacemaker, food contaminated with salmonella, defective anesthesia products
Class II Intermediate Use/exposure may cause temporary or medically reversible adverse health consequences, remote probability of serious adverse health consequences. Failure to declare the presence of monosodium glutamate (MSG) in a food product
Class III Lowest Use/exposure is not likely to cause adverse health consequences. Undeclared colors on food product label, sub-potent dandruff shampoo

The public should recognize that recalls are almost always voluntary. Sometimes a company discovers a problem and recalls a product, while at other times a company acts after the FDA raises concerns. Whether FDA or the company discovers the problem, in every case FDA oversees a company’s recall strategy and assesses the adequacy of the recall.

If a product is believed to pose an immediate, serious hazard, FDA will also act quickly, even before classification is complete, to widely publicize a recall. FDA’s publicity efforts may include press releases, advisories, distribution of alerts to subscribers, and updates to the FDA web site. This outreach has proven effective in bringing exhaustive TV, radio, and newspaper coverage about recalls. Increased coverage has occurred in recent years, for example, with certain brands and batches of medicines, injection pens, and contaminated foods such as smoked fish, peanut butter, spinach, frozen vegetables, and ice cream.

The FDA Consumer Update provides more details about the process. Subscribe to the Enforcement Report mailing list at Enforcement Report email subscription. To comment, e-mail enforcementreports@fda.hhs.gov.

Douglas Stearn, J.D., is FDA’s Director, Office of Enforcement and Import Operations, Office of Regulatory Affairs

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

Talking FSMA in the Land of Cotton and Looking for Middle Ground

By: Stephen Ostroff, M.D.

You may not know that in addition to producing cotton for the fabrics that we use every day, cotton farmers and ginners produce food. After being separated from fibers in a process called ginning, cotton seeds and cotton plant parts are often used to feed livestock. Cotton seeds may also be further processed into cottonseed oil and cottonseed meal for animal food.

Dr. Ostroff discusses the cotton ginning process with Jason Kirk, Supervisor for the Gins and Warehouses Section of the Alabama Department of Agriculture and Industries.

Dr. Ostroff discusses the cotton ginning process with Jason Kirk, Supervisor for the Gins and Warehouses Section of the Alabama Department of Agriculture and Industries.

Because products from ginning enter the food supply, concerns have been raised by the cotton industry about the impact on cotton ginners of new requirements under the FDA Food Safety Modernization Act (FSMA). To better understand these concerns, my colleagues and I traveled to northern Alabama last month to visit a cotton farm, ginning operation and a cotton warehouse.

FDA regulates the use of cotton plant material and seeds in animal food. Traditionally, cotton ginning has fallen under the general food adulteration provisions of the Federal Food, Drug, and Cosmetic Act. Now, certain operations are also subject to the FSMA-mandated preventive controls requirements, which applies to manufacturing facilities rather than farms. Most cotton ginning is done by farms, whose ginning operations don’t fall under the new requirements. But cotton gins that aren’t part of a farm are considered manufacturers subject to the preventive controls requirements, which include a hazard analysis and the implementation of controls to minimize hazards.

Steven Barber, director of FDA’s Human and Animal Food Division, and Jim Davis, Southeast Member Service Representative for the National Cotton Council, discuss cotton growing and harvesting at Isbell Farms. A bale of cotton from the field is in the background.

Steven Barber, director of FDA’s Human and Animal Food Division, and Jim Davis, Southeast Member Service Representative for the National Cotton Council, discuss cotton growing and harvesting at Isbell Farms. A bale of cotton from the field is in the background.

The cotton industry argues that this doesn’t make sense since all cotton ginners are essentially doing the same work. So why would we impose FSMA requirements only on non-farm operations? We have always said that we will work with industry to find solutions when the FSMA rules produce inconsistencies like this one. We have visited food facilities and farms all over the country and overseas to understand what’s working well and what issues may need to be addressed.

And that’s why we went to Alabama, to see the cotton operations first-hand. I was accompanied by Jennifer Erickson in FDA’s Center for Veterinary Medicine and Steven Barber, director of FDA’s Human and Animal Food Division, which covers Alabama. We were also joined by representatives of the National Cotton Council and members of cotton ginning associations, as well as representatives from the Alabama Department of Agriculture and U.S. Rep. Robert Aderholt’s office.

the authors on a cotton harvester

Jennifer Erickson of FDA’s Center for Veterinary Medicine and Dr. Ostroff on a cotton harvester at Isbell Farms. The harvester removes the cotton from the plant in the field and compresses it into round bales wrapped in plastic that will be taken to the cotton gin.

Our first stop was Isbell Farms in Cherokee to see cotton operations at the farm level. Although weather conditions limited what we could see in action, we toured the farm and learned how the cotton is picked and rolled into bales for transport to the gin. Then we visited the Cotton Producers Co-op Gin in Tuscumbia to observe cotton ginning and seed storage. At the gin, the cotton moves through a series of machines that remove plant material before the cotton enters the gin stand, where the cotton fibers are separated from the cotton seed. Throughout the process, the cotton moves in enclosed piping through the machines that clean, gin and bale the material. Once separated from the cotton fiber, the cotton seeds are also moved in enclosed piping to a separate building where they are stored. Lastly, we stopped by the Cotton Producers Co-op Warehouse in Leighton for information on the receiving and shipping of baled cotton fiber.

Before the cotton enters the gin stand, it goes through a process that removes plant material, held here by cotton farmer Sam Spruell, before the fibers are separated from the seeds.

Before the cotton enters the gin stand, it goes through a process that removes plant material, held here by cotton farmer Sam Spruell, before the fibers are separated from the seeds.

It was very beneficial to see the process from field to production — to see the ginning process and how the seeds are separated, handled, and stored. It’s a very sophisticated, high-tech operation.

Throughout the day there were lively discussions about the best path forward, one that would not compromise the public health protections at the heart of FDA’s mission. There are a number of possible options to achieve this goal. In August 2016, we extended the compliance date for cotton ginners now subject to the Preventive Controls for Animal Food rule to give us more time to consider the next steps necessary to achieve this shared goal.

In the meantime, I appreciate the warm Southern hospitality that we were shown in the land of cotton. We also ate some great local food. The people we met clearly love what they do and were very gracious and open in sharing their stories and the pride they take in their work. Some traveled a long way to meet us for a short time, but they wanted to be there. I look forward to continuing these conversations in pursuit of a solution that works for industry while protecting public health. I’m certain that we will arrive at that solution.

Stephen Ostroff, M.D., is FDA’s Deputy Commissioner for Foods and Veterinary Medicine

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:

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

FDA Science: Working at the Speed of Emerging Technologies

By Luciana Borio, M.D.

Let’s face it, we’ve all gotten used to nearly instant access to almost anything.

Today, with a tap of an app, we order a car ride, a book, or pizza for dinner. Need to navigate past traffic in downtown city streets? No problem. There’s an app for that, too.

Some may wonder: Why hasn’t rapid medical product development partaken of this need for speed that has reshaped other sectors of our economy? Well, in many ways, it has.

Innovation is happening extraordinarily fast in the biomedical sciences and at FDA. As FDA’s Acting Chief Scientist responsible for leading and coordinating FDA’s cross-cutting scientific and public health efforts, I see close up that years of scientific research, collaboration, and investment are paying off.

FDA Acting Chief Scientist Lu Borio

FDA Acting Chief Scientist Luciana Borio

When I testified at a congressional hearing recently, my colleague, Dr. Anthony Fauci, director of the National Institute for Allergy and Infectious Diseases, gave a tangible example of what I mean. He said it took his team about three months to begin clinical testing of a Zika vaccine candidate developed from scratch. In 2003, it took the same team 18 months to develop a candidate vaccine to address the SARS outbreak and begin clinical testing of that product.

And in just over two decades, a disease like multiple sclerosis has gone from being untreatable to one for which clinicians are nearly “flummoxed by the options,” according to a headline I saw recently.

There is a reason for this success. In the last several years, scientists have identified and begun using “safety-risk biomarkers.” Rather than those for efficacy, these biomarkers identify which patients are at highest risk for certain adverse events. They have opened up an array of therapeutic options for patients who might do just fine with some treatments that may not otherwise have been developed due to our previous inability to properly assess their risk.

None of these successes would be possible without our FDA product reviewers working at breakneck pace to guide these innovative development programs.

It’s not always fully understood that FDA scientists play an essential role in advancing many biomedical innovations. That’s why we invite the public to participate in a two-day Science Forum at FDA every other year to showcase the agency’s robust scientific research and the important work done by our 11,000 scientists.

Just as industry focuses on product development research and academia focuses on the scientific foundation, FDA research concentrates on creating test methods and developing knowledge of processes to ensure that our products are safe and effective or, with tobacco, at least with reduced harm.

I like to think that this year’s Science Forum was better than ever. Over two days, hundreds of participants were treated to 230 scientific posters and some 50 presentations by FDA scientists and others, organized under eight broad categories:

  1. Identification and Evaluation of New Biomarkers;
  2. FDA Response to Urgent Public Health Needs;
  3. Microbiome and Human Health;
  4. Advanced Manufacturing and 3D Printing;
  5. Omics Technologies at FDA;
  6. Patient and Consumer Engagement and Communication;
  7. Computational Modeling and Simulation at FDA; and,
  8. Current Progress in Nanotechnology Research at FDA.

Four poster sessions during the two days augmented the presentations that featured the authors of studies describing the methodology, challenges, and results of their research one-on-one with those at the forum. Among the meaty topics discussed were:

  • The emerging technology of additive manufacturing and medical devices, produced by 3D printing. Bioengineers at FDA’s Center for Devices and Radiological Health have positioned themselves at the forefront of knowledge and research about this cutting-edge manufacturing process, by looking into patient matching, imaging, and phantoms. With our proactive posture, FDA is paving the way for safe and effective innovation that will usher in life-saving advanced treatments for patients.
  • The growing use in medical products of nanomaterials – equal to about one-billionth of a meter – so small that they can’t be seen with a regular microscope. Silver nanoparticles are now used in wound dressing for their antimicrobial properties. And liposomal nanoparticles are used as drug carriers to reduce toxicity and increase circulation time in the blood. Characterizing these complex nanomaterials is challenging. FDA scientists highlighted their analytical methods for characterizing nanomaterials in over-the-counter FDA-regulated products. This will help us with assessing risk, developing industry guidelines for characterizing nanomaterials, postmarket surveillance, and determining shelf life of nanomaterials in consumer products.
  • In the area of food safety, FDA has contributed to enhancing antimicrobial resistance monitoring in a collaborative effort with USDA and the Centers for Disease Control and Prevention. And, genomics studies conducted by FDA scientists have demonstrated that we can use the emerging technology whole genome sequencing as an effective tool for predicting antimicrobial resistance of certain foodborne pathogens.

Not all of our essential research deals with cutting-edge technology. Scientists from FDA’s Center for Tobacco Products (CTP) shared their work on water pipe, or hookah, smoking. Water pipes, a centuries-old method of smoking, are becoming an increasingly common method of tobacco smoking in young adults. A rare and serious lung disease – water pipe-induced acute eosinophilic pneumonia – has been reported among these smokers. One of the forum’s posters described how CTP scientists identified the disease and made physicians aware of it.

And, as a sign of the times, mobile communications also were part of the poster sessions. Healthy Citizen @FDA will be a holistic, citizen-centric mobile platform for FDA to collaborate and communicate with citizens to improve public health outcomes and to receive timely FDA alerts.

Of course, events like these are equally valuable for what happens before and after the formal presentations. From the snippets of conversation I picked up in the hallways, FDA and outside scientists had plenty of opportunity to interact, share ideas, and even discuss potential collaborations.

Those who attended the 2017 Science Forum gained a deeper understanding of the cutting-edge science we do at FDA to protect and promote the public health. And those who missed the Forum have the option of watching the recorded presentations on FDA’s website. We look forward to future opportunities to share more of the exciting advances we’re making with our partners in the scientific community.

Luciana Borio, M.D., is FDA’s Acting Chief Scientist

Mixing Kentucky Spirits with Food Safety to Protect Spent Grains Used to Feed Animals

By: Jenny Murphy, M.S.

“Spent grains” is the general term for the remnants of corn, rye, barley and other grains used to make alcoholic beverages. While they are a byproduct of human-food production, spent grains have a long history of being used as a valuable source of nutrients to feed many animal species. They also have been the subject of ongoing concerns among beverage distillers and brewers over the past few years as the FDA drafted rules that will implement the FDA Food Safety Modernization Act (FSMA).

Jenny Murphy holding spent grains

Jenny Murphy, a consumer safety officer in FDA’s Center for Veterinary Medicine (CVM), with handful of dried spent grain at the Wild Turkey distillery. In background, from left: Shannon Jordre, a consumer safety officer in CVM’s Division of Compliance, Jim Sanders, Distillery Manager at the Wild Turkey Distillery, Steve Barber, director of FDA’s Cincinnati District Office, and Jennifer Erickson, a regulatory policy analyst in CVM’s Office of Surveillance and Compliance.

Those concerns brought an FDA team to the rolling hills of central Kentucky this month to visit distilleries and a craft brewery and to meet members of the distilled spirits and brewers industry. The team was led by Stephen Ostroff, M.D., who was then FDA’s deputy commissioner for foods and veterinary medicine and who is now the acting FDA commissioner. I represented the agency’s Center for Veterinary Medicine (CVM) because of the connection between spent grains and animal health.

Congress included language in FSMA to exempt most alcoholic beverage manufacturing facilities from most of the FSMA requirements. But every day these manufacturers produce tons of spent grain, which are commonly used as animal food rather than being discarded in a landfill. Whether or how spent grains would be regulated under FSMA has been a point of some uncertainty for industry.

Based on the information available at this time, FDA considers the potential animal food safety hazards associated with spent grains from the alcoholic beverage industry to be minimal. The agency has assured these manufacturers that they will only be required to ensure that the byproducts are properly labeled and kept safe from contamination while they are held for distribution if these conditions are met: They continue to follow current good manufacturing practice (CGMP) requirements for human food in the production of their alcoholic beverages, and the resulting spent grains are not further processed. If the grains are further processed, such as by drying, they must follow CGMP requirements, with the flexibility to follow either the human food or animal food CGMPs.

But we have been encountering some confusion among brewers and distillers about these CGMP requirements for human and animal foods and how they would apply to spent-grain products. We wanted to reach a greater level of understanding on both sides, and what better place to do that? Kentucky is, after all, the birthplace of bourbon, according to the Kentucky Distillers Association. Recognized by Congress as a distinctively American product, bourbon is a $3 billion industry in Kentucky that generates 15,400 jobs. Kentucky also has a growing craft beer brewing industry.

containers of wet spent grain

Adam Watson (center), managing member and brewer at Against the Grain Brewery in Louisville, shows the visitors the containers of wet spent grain waiting for pickup outside the brewery.

We were a large group – about 20 participants from FDA’s headquarters and district office, the Kentucky Department of Agriculture, the state Division of Regulatory Services based at the University of Kentucky, and industry, including the Distilled Spirits Council and Brewers Association. Over the course of two days, we visited the Woodford Reserve, Wild Turkey, Jim Beam’s Booker Noe plant and Clermont distilleries, as well as the Against the Grain brewery.

This opportunity for education and outreach was extremely productive for all involved. FDA participants were able to allay many concerns and provide clarity about the framework for the CGMPs that the distillers and brewers are expected to meet for both human and animal food. And the distillers and brewers opened their doors, bringing us into their world in a way that was very informative and instructive. There is no substitute for actually seeing how these beverages are produced and how the spent grains are handled.

Although the basic processes are the same, each of the distillers and brewers we visited approaches its work somewhat differently. When it comes to spent grains, there are some operations that simply hold the grain and others with more elaborate processes to distribute the grain because of the large volume produced. We learned distinctions in terminology as the distillers showed us a variety of spent grain products, including wet and dry grains, syrups and cakes.

FDA engaged in this kind of outreach when the FSMA rules were taking shape. Now that the regulations are becoming a reality, we believe these conversations are just as important to help food producers understand what’s expected and avoid any misunderstandings. For FDA, these exchanges help us better understand what, if any, challenges industry may be facing as they strive to meet these new requirements.

Our next step is to take the knowledge we acquired and the lessons we learned and use them to help shape training for regulators and outreach to this industry in support of our shared commitment to keep both human and animal foods safe.

Click here to view FDA’s flickr album from this month’s visit to Kentucky.

Jenny Murphy, M.S., is a consumer safety officer in FDA’s Center for Veterinary Medicine