FDA’s Comprehensive Effort to Advance New Innovations: Initiatives to Modernize for Innovation

By: Scott Gottlieb, M.D.

Our longstanding goal for medical care is to ensure that the right drug or device is delivered to the right patient at the right time. This vision is increasingly possible with the innovative products that are becoming available. Many of these opportunities are enabled by new technology platforms such as digital health, targeted medicines, and regenerative medicine, including cell and gene therapies. These new technologies offer transformative opportunities. But they also challenge the U.S. Food and Drug Administration (FDA) to modernize its approach to evaluating new innovations. In many cases, we’ve had to refashion our regulatory approach to create more modern platforms that are better suited to the efficient evaluation of these advances.

Dr. Scott Gottlieb, Commissioner of the U.S. Food and Drug AdministrationIn short, we’ve had to modernize our overall approach to regulation to effectively advance the kinds of innovations that are becoming available. This includes modernizing how we organize our medical product review programs. These initiatives are part of our comprehensive Medical Innovation Access Plan.

These efforts are strengthened by new authorities and resources made possible by bipartisan legislation like the 21st Century Cures Act, as well as the recent re-authorization of the FDA’s user fee agreements. The actions that we’re taking have additional support from the President’s Fiscal Year 2019 budget. Together, these efforts will enable the FDA to fund the creation of a cross-cutting data enterprise for the generation of evidence, and a more modern and integrated approach to the evaluation of this information, to make sure that our regulatory decisions are as flexible and sophisticated as the science driving these advances.

And we’re not doing it alone.

We’re working closely with our public and private sector partners to better meet shared public health goals and address cross-cutting scientific and technical challenges, while making regulatory decisions more transparent and predictable for all stakeholders. My recent written testimony on how the FDA is implementing the 21st Century Cures Act contains an overarching picture of the agency’s many activities related to our new policies aimed at advancing innovative products.

I’d like to use this opportunity to reflect on how the FDA is creating a new operating system for innovation by modernizing clinical trials, streamlining the FDA’s organization and processes to advance regulatory science, and expanding the FDA’s capacity to analyze complex real-world data streams to detect early safety and efficacy signals. And to describe the new policies we plan to announce to advance these goals. These mutually reinforcing efforts will help the FDA meet its mission of promoting and protecting public health, and they will help unlock the full public health potential of America’s public and private investments in medical research.

Modernizing Clinical Trials for Drugs and Devices

Prospectively randomized, placebo controlled clinical trials are often the most powerful tool that we have for answering fundamental questions about the safety and efficacy of new medical products. But greater efficiency is needed, as clinical trials are becoming more costly and complex to administer. Moreover, many of the new products that we’re being asked to evaluate aren’t easily evaluated using these traditional approaches. At the same time, new technologies and sources of data and analysis make better approaches possible.

Added complexity can not only make medical product development more uncertain, expensive, and time consuming; overly complex trials and unnecessary data collection can deter patient enrollment, exhaust investigators, and delay completion of studies so long that their findings aren’t relevant. They can also discourage the development of second and third-to-market innovations, meaning that first-in-class drugs enjoy monopolies for longer periods of time. This can reduce competition that lowers prices, and limit therapeutic diversity.

The FDA is working across its medical product centers, in collaboration with the Clinical Trials Transformation Initiative (CTTI) and the Medical Device Innovation Consortium (MDIC), to facilitate innovative trial designs and patient-centered endpoints for drugs and medical devices that can make clinical trials more efficient. These approaches can also be more rigorous. Developing more efficient strategies for generating critical evidence relating to the safety and efficacy of drugs and devices in specific populations (for instance, through seamless trial designs, and the use of master protocols and basket trials) can help make the clinical development process more efficient. It can enable investigators to learn more about a product’s efficacy and safety, and help regulators and sponsors detect efficacy and safety signals earlier in the development process.

Lowering the cost and time needed to conduct trials can promote market competition, help check drug prices, and bring patients innovative medical products earlier. These approaches can lower costs by making it more economical for second or third- in- class products to compete with first entrants. Right now, when it comes to drugs targeted to unmet needs, we’re seeing a trend where second and third-to-market competition is taking longer to reach patients. There are complex reasons for this. But one is the difficulty of conducting traditional clinical trials in settings where there is an available therapy, but still significant unmet medical need – for instance, in some rare diseases.

We studied these trends. A new FDA analysis considers the number of drugs or biologics that CDER has approved in the same class. They’re drugs that use the same mechanism to produce a physiological change in the same or related condition. We found that new competition isn’t entering the market as quickly for these drugs. In other words, when a novel sole source drug wins approval it faces no competition from other drugs in the same class. Follow-on drugs and biologics to compete with the first-in-class have been arriving more slowly.

Here are some results from the data we reviewed. We plan to publish the full analysis soon.

For non-orphan pharmaceuticals, which treat conditions affecting larger patient populations, 41 percent of the first-in-class products approved between the years of 1991 and 2000 had at least one competitor in the same class within five years. This rate dropped sharply over the next decade. For the years from 2001 to 2010, for the same kind of cohort of medicines – first-in-class products that were approved to treat patients with prevalent conditions – only 18 percent of these drugs had a within-class competitor after five years. Another way of interpreting the data is to describe the lag in any competition.  For the older classes, where the first-in-class was approved in 1991 to 2000, nearly a quarter had a competitor within two years. For the cohort where the first-in-class was approved in 2001 to 2010, it took an additional five years for there to be nearly as much competition. By year seven, competition still lagged the previous cohort, with only 22 percent of classes having any competitor. We see similar patterns in most rare disease treatments.

Consider first-in-class orphan drugs and biologics for non-cancer indications. For drugs approved between 1991 and 2000, 26 percent had at least a competitor within five years. The comparable rate for the 2001 to 2010 cohort was 13 percent. These trends mean that costlier, branded drugs may enjoy longer periods without facing competition from products in the same class. This may increase their pricing power. For orphan drugs, where conducting clinical trials can be difficult, these periods can sometimes extend long after patents and other exclusivities lapse.

We’re taking steps to facilitate more efficient clinical development programs. The Center for Devices and Radiological Health’s (CDRH’s) work with MDIC, for example, is improving efficiency in trial site contracting, first in patient studies, and Institutional Review Board (IRB) approval. These are three of the costliest factors in device trials and can pose barriers to developing innovative products. Similarly, the FDA has advanced efforts to modernize clinical trials by pioneering Master Clinical Trial Protocols (MAPs) such as basket, umbrella, and platform trials. These approaches can increase trial efficiency and lower costs.

MAPs move away from one-drug, one-disease trials. They involve one or more interventions in multiple diseases or a single disease with multiple interventions, each targeting a biomarker-defined population or disease subtype. A key feature of master protocols is the use of a common clinical trial infrastructure to streamline trial logistics, improve data quality, and facilitate data collection and sharing.

In the coming weeks, we’ll be issuing additional guidance on MAPs and efficient trial design strategies to help expedite the development of oncology drugs and devices. We’ll also be issuing guidance on the use of adaptive trial designs, and innovative endpoints like minimal residual disease in hematologic cancers. We recently issued draft guidance on the use of placebos in randomized trials in oncology. Advances in care, and trial design, can make it unethical and infeasible in some circumstances to use placebo controls in cancer trials. At the same time, the FDA is advancing the development of natural history models for rare diseases. These models may obviate the need for placebo arms in some trials by allowing researchers to replicate the behavior of patients who otherwise are left untreated.

As part of this effort, we’re also launching a complex innovative designs (CID) pilot meeting program to facilitate the advancement and use of novel clinical trial designs. The CID pilot will offer medical product developers an early opportunity to meet with FDA experts in all relevant disciplines from the agency’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) to discuss regulatory approaches to novel trial designs.

Medical devices present a different set of technical challenges and opportunities compared to drugs. But we’re employing the same principles to facilitate the agile development and review of innovative devices. For example, in the FDA’s Breakthrough Devices Draft Guidance, we proposed the use of “sprints” in which the sponsor of a breakthrough device identifies a regulatory challenge they need to solve. We then work interactively with the sponsor to address that challenge within a short timeframe — often just a few weeks. These early interactions have resulted in the development of flexible clinical study designs for certain breakthrough devices and in more FDA review team support and senior management engagement earlier in the development and review process. All of these steps are intended to enable the FDA to evaluate, and the sponsor to develop, innovative devices more efficiently. The FDA has granted 72 breakthrough device designation requests and, as of June 1, 2018, has approved or cleared six breakthrough devices.

As part of these efforts, CDRH continues to apply the “least burdensome” approach to all activities – exceeding what has been mandated in statute – related to medical device regulation. This concept will ensure that regulators and sponsors align on the minimum amount of information necessary to adequately address a relevant regulatory question or issue through the most efficient manner at the right time. This culture helps to further reduce the time and cost required to develop and market safe and effective new devices.

Together, the FDA’s Breakthrough Device program, least burdensome principles, and acceptance of greater uncertainty in appropriate circumstances are already making a dramatic difference in the health of millions of American patients. Just some examples of products that have come to market as a result of CDRH’s streamlined approaches include: an innovative device for  transcatheter aortic valve replacement (TAVR), the “artificial pancreas” (and subsequent expansion of approval to include individuals aged 7 to 13), the world’s smallest heart valve for newborns, first blood test in the world to evaluate mild traumatic brain injury, the first breakthrough-designated next generation sequencing (NGS) based IVD to detect cancer mutations in 324 genes, the first artificial iris in the United States, and the first mobile medical app to help treat substance abuse disorders.

Modernizing FDA’s Organization and Breaking Down Outdated Silos

Building on the FDA’s success in standing up the Oncology Center of Excellence, we’re also modernizing our organizational structure, flattening our review process, and breaking down review silos between different scientific disciplines that are important components of the medical product review process. The goal is to enable FDA review teams to be more disease focused, more integrated across the disciplines involved in drug review, and better able to evaluate and analyze data from agile clinical trials through a more structured approach to data review.

For instance, CDER has proposed an important series of new steps to modernize the organization and functions of CDER’s Office of New Drugs. Part of this involved structural changes. Other elements are aimed at process improvements that make the review process more predictable, consistent, and structured. The idea is to make the review of data more structurally consistent and improve the productivity of our clinical staff. This effort is starting with how we can more carefully and rigorously evaluate safety.

We’re implementing a more standardized, efficient, and comprehensive process for review of drug safety. This new process will leverage staff expertise in data analytics to develop more standardized approaches and templates for how we evaluate safety data as part of new drug applications. This process fully leverages the standard datasets that must be submitted in drug applications. It also brings in added quantitative and programming expertise in the conduct of safety analyses to support the medical team’s efforts. As part of this effort, we’re looking to make the review process more integrated, multi-disciplinary, and problem-focused; and to develop a review document that reflects this multi-disciplinary, problem-focused approach. By enhancing efficiency and providing greater support for the application review, we intend to “front load” this process. This approach should result in more time during the review cycle for key discussions, such as on labeling and on post-market requirements and commitments. These new processes should align well with our ongoing efforts to base our regulatory decisions on an informed assessment of the benefit-risk balance – by providing a deeper understanding of the risks, along with a comprehensive assessment of benefit, incorporating the patient’s perspectives and preferences.

These new approaches will bring added efficiency to our processes and improve our internal productivity. One benefit will be reducing routine administrative burdens on our new drug staff, elevating the role of our scientists and medical officers to take on even more thought leadership in their fields. We’ll use the productivity gained to channel more of the intellectual resources of our clinical staff into thought leadership activities that help advance the principles of regulation. As part of this effort, for example, we’re considering creating many new therapeutic-specific divisions that’ll have more ability to engage in discrete areas of medicine. The goal is to make sure that the drug review divisions are therapeutically focused to promote efficient review and provide greater scientific leadership to academic, industry and patient groups. The Office of New Drugs modernization will give our subject matter experts more time, better analytic tools, and more knowledge management support to advance the clinical and regulatory principles we rely on to evaluate the safety and efficacy of innovative products

This should allow the FDA to issue many more product-specific guidance documents. We plan to develop hundreds of new clinical guidance documents and make sure they stay up-to-date to reflect the latest science. We’ve already issued nearly 100 guidance documents in 2018 alone. Another goal is to allow the FDA’s staff to engage with stakeholders on new technologies like continuous manufacturing of drugs and biological products through the FDA’s Emerging Technology Program, designed to help industry implement innovative technologies that can improve product quality.

The FDA’s Device Center is undertaking a similar modernization of its approach. CDRH has explored, piloted, and developed implementation plans that will help CDRH improve information sharing, decision making, and work efficiency by instituting a Total Product Life Cycle (TPLC) approach to many of the core medical device review activities. TPLC will also enable CDRH experts to leverage their knowledge of pre- and postmarket information to optimize regulatory decision-making. Efforts underway at the FDA’s Device Center share a similar goal with the OND reform. The aim of FDA’s TPLC approach is to ensure not only that devices meet the gold standard for getting to market, but also that they continue to meet this standard as we get more data about devices and learn more about their benefit-risk profile in real world clinical settings.

Harnessing Real World Evidence              

As part of these efforts, the FDA is also actively working to evaluate the use of real-world evidence (RWE) to support regulatory decisions. This includes data captured from sources such as electronic health records, registries, and claims and billing data. Real world evidence can help answer questions that are relevant to broader patient populations or treatment settings where information may not be captured through traditional clinical trials. We are expanding our ability to use RWE for post-marketing safety surveillance, and exploring its potential to help support expanded label indications.

FDARA provided important funding to evaluate how RWE can be generated, and its potential use in product evaluation. The funding included significant new resources to enhance the FDA’s Sentinel system. To date, Sentinel has been used to assess safety. The FDA is now supporting the first randomized prospective intervention trial that makes use of information in the Sentinel system. To take one practical new example of this application, the IMPACT-Afib trial will test an educational intervention to address the important public health problem of underuse of effective medications to reduce the risk of stroke in patients with atrial fibrillation. This proof-of-concept trial can serve as a prototype for future RWE trials. At the same time, in another proof of concept study, the FDA is also funding a project to examine whether real world evidence that’s generated using observational data can replicate the results of approximately 30 randomized controlled clinical trials for drugs.

CDRH has also made one of its top priorities the development of a system of active surveillance for medical devices by building out the National Evaluation System for Health Technology (NEST). The goal is for this to ultimately help drive the development of safer, more effective devices, and timelier patient access to those devices. It will also increase the value and use of real-world evidence to support the needs of multiple stakeholders in our health care system, including the detection of emerging safety signals. NEST may also eventually be used to facilitate reimbursement (the Centers for Medicare and Medicaid Services serves on the NEST Governing Committee) as improved data collection can help encourage coverage with evidence development (CED).

FDA’s Role in Curating Standards for Novel Technologies

The agency’s role in curating standards for medical technologies can help advance innovation in areas that may lack consensus standards now. One example is through software-based platforms that are playing an increasingly central role in managing patient health. These tools can help more patients gain more control over their own health.

These software tools are becoming more sophisticated, enabling a broader set of opportunities. Artificial intelligence (AI), for example, holds enormous promise for the future of medicine. We’re actively developing a new regulatory framework to promote innovation in this space and support the use of AI-based technologies. So, as we establish and apply our Pre-Cert program – where we will focus on a firm’s underlying quality in assuring software products meet safety and effectiveness standards – we’ll consider how to account for one of the greatest benefits of machine learning – that it can continue to learn and improve as it is used.

We know that to support the widespread adoption of AI tools, we need patients and providers to understand the connection between decision-making in traditional health care settings and the use of these advanced technologies. One specific area that we’re exploring with stakeholders is how we can benchmark the performance of AI technologies in the field of radiogenomics, where AI algorithms can be taught to correlate features on a PET or MRI scan with the genomic features of tumors. This provides an opportunity to improve patient prognosis, identify early response to treatment, or develop novel imaging biomarkers that could be used to triage high risk patients who may need more frequent screening.

Toward these goals, the FDA is exploring the use of a neutral third party collect large annotated imaging data sets, for example highly annotated radiology scans used in a variety of clinical trials for specific disease indications, for purposes of understanding the performance of a novel AI algorithm for a proposed indication. Such a capability would enable a transparent benchmarking system for AI algorithm’s performance, and help providers and payors compare AI systems with the best human standard of care.

The FDA is also one of many stakeholders deeply interested in advancing the assessment and quantification of symptom and functional outcomes in cancer patients through clinical outcome assessments (COAs). COAs, in layman’s terms, are measures that describe or reflect how a patient feels, functions, or survives. Several technological advances hold promise to revolutionize how we can capture patient-centered clinical outcomes in controlled trial and real-world settings. One traditional COA is a survey that collects patient reported outcomes (PROs) through a questionnaire.

Electronic capture of PRO data (ePRO) is also becoming standard, providing a rich pipeline of structured clinical data. In addition to ePRO, mobile wearable technologies can complement traditional PRO surveys by generating objective, continuous activity and physiologic data. Obtaining reliable wearable device data on activity level, coupled with direct patient report on their ability to carry out important day to day activities, can provide information on physical function that is directly relevant and important to the quality of life of cancer patients.

Medical products are becoming increasingly sophisticated. The advent of advanced computing and systems biology will continue to help make health care more personalized, while connected technologies break down barriers between clinical research and real-world patient care. New platforms like targeted medicine, cell and gene therapy, and regenerative medicine hold more curative opportunities.

To facilitate these opportunities, and help make sure these innovations are able to improve public health, we’ve undertaken a comprehensive effort to make sure that our organization and policies are as modern as the technologies we’re being asked to evaluate, and that we’re able to efficiently advance safe, effective new innovations.

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

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

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

 

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

Taking New Steps to Meet the Challenges of Rare Diseases — FDA Marks the 11th Rare Disease Day

By: Scott Gottlieb, M.D.

Today 30 million people in the United States – or one out of every 10 Americans – lives with at least one of more than 7,000 rare diseases. These conditions include rare cancers to inherited metabolic disease. And tragically, half of those affected by rare diseases are children.

Dr. Scott GottliebThis week, the U.S. observes the last day of February as Rare Disease Day to raise awareness about rare diseases and their impact on patient’s lives. Unfortunately, finding treatments for these conditions does not become easier or less costly with the rarity of a disease.

In many cases, developing a treatment for a rare disease can be especially hard and present unique challenges. Each success is the end of a long uphill climb. It requires the concerted efforts of many stakeholders, including scientists, product developers, regulators, policy makers, and of course, the energy and organization from patient advocacy groups.

For FDA, Rare Disease Day offers an opportunity to take measure of the progress we’ve made to help people affected by rare disease; and evaluate what more we can do to meet our commitment to advance the needs of patients with rare diseases and their families.

Thirty-five years ago there were few drugs and biologics for rare diseases and even fewer devices. Enacting the Orphan Drug Act in 1983 with its financial incentives and other inducements was an important start to enabling more investment and development of treatments targeted to rare diseases. Also important was legislation passed in 1990, creating a rare disease path for medical devices; known as the Humanitarian Device Exemption (HDE).

Rare Disease Treatment GraphSince 1983, we’ve seen significant progress in treating rare diseases. FDA has approved more than 650 therapies for rare indications. This includes new molecular entities and biologics, as well as new rare disease indications for drugs approved for another indication. We’ve also seen progress in the development of devices for rare diseases. Since 1990, the FDA has approved 72 medical devices for an orphan indication under the agency’s HDE program.

In recent years, the increasing emphasis on personalized medicine, including genetically targeted drug development, has enabled even more opportunities to develop treatments aimed at rare diseases. As a result, during the past five years, the number of requests to have a drug designated as serving an orphan population has steadily increased. In 2017, there were over 700 requests for designation. This was more than double the number of requests received in 2012. Last year we also saw 80 treatments approved by FDA for rare indications, the highest number ever.

FDA’s orphan drug program focuses its efforts on the full range of rare diseases, including relatively more rare or ultra-orphan diseases. In 2010, Miles Braun and other FDA researchers used data from 1983-2008 to show that there’s substantial effort with regard to the rarest diseases. The categories with the most orphan drug designations and the most approvals had very low prevalence levels. New analysis of more recent data shows this trend has been maintained. This experience suggests that the orphan drug program may continue to grow in importance as medicine becomes increasingly personalized, and better able to target the underlying molecular and genetic basis of even very uncommon disorders.

Despite these successes, we recognize that thousands of rare diseases still have no approved treatments. Indeed, FDA’s recent needs assessment survey, done in collaboration with the NIH’s National Center for Advancing Translational Sciences, identified a major public health need for innovative medical devices to care for children and adults with rare diseases.

FDA is committed to doing its part to facilitate continued progress toward more treatments and even potential cures for rare diseases. New scientific opportunities enabled by advances in cell and gene therapy hold out more opportunities to develop these potential cures. With efficient regulation, proper incentives for product development, and the continued support of patients, providers, and innovators; we’re more able to pursue these opportunities than ever before.

In June, I announced FDA’s Orphan Drug Designation Modernization Plan. Our aim was to create a more efficient, scientifically advanced, predictable, and modern approach to the approval of safe and effective treatments for rare diseases. Since then, we’ve eliminated the backlog of orphan drug designation requests. In addition, we’re fully implementing a 90-day timetable for processing new designation requests. We also established an FDA Orphan Products Council to further address scientific and regulatory challenges pertaining to orphan products.

Through our long-standing Orphan Products Grants Program we recently provided $17 million in funding to directly support 15 new clinical trials on products for rare diseases and to fund natural history studies for the first time. These four natural history studies, and an additional two studies funded through an NCATs partnership, could provide key information about how rare diseases develop and progress. This information can be vital for product development.

Of note, I also recently communicated our desire to expand upon these efforts to help foster investment and innovation in, and medical product development for, rare diseases by developing clinical trial networks to create an understanding of the natural history (such as individual patient experiences and progression of symptoms) and clinical outcomes of rare diseases. FDA’s 2019 budget includes a request for resources to make additional investments in rare disease natural history models. It’s clear more work can be done to advance these efforts.

Today I’m pleased to announce several new actions FDA is taking as part of our ongoing commitment to support and expedite the development of rare disease products. They include:

  • A new pilot for more efficient orphan designation requests, including a new fillable form that will make the submission process easier for sponsors to complete designation requests; and make it more efficient for FDA to review. This also includes an on-line tutorial to guide sponsors through the submission process. We’ve also developed a new inter-center consult process to streamline and standardize our communications process.
  • We are entering into a new Memorandum of Understanding with the National Organization for Rare Disorders to conduct outreach with our new Patient Affairs Staff on ways to enhance the incorporation of patient experience into regulatory discussions. As part of this process, we’re planning a joint series of pilot listening sessions on rare diseases. We recognize that early and iterative engagement can improve clinical and regulatory understanding of diseases and conditions; provide a common understanding of the most urgent patient needs; and inform drug development programs.
  • We’re planning a public meeting that will help us prepare for the changing landscape of orphan drug development posed by the growth in targeted therapies and molecularly defined diseases. At an upcoming meeting, we’ll seek input on complex scientific and regulatory issues related to cancer drugs and biologics that target a tumor’s specific genetic features rather than its location in the body (i.e., tissue agnostic approvals). We’ll also consider the appropriate application of orphan incentives to this new paradigm of drug development, and how we apply designations to these indications.

To provide the public with a more complete discussion of the scope of FDA’s rare disease activities, we’ve also created a new, enhanced web page that features videos from our three center directors plus other materials. I invite you to take a look.

Over the course of 2018 we’ll continue our efforts to increase the consistency and efficiency of our reviews of rare disease products. We remain committed to supporting rare disease research on diagnostics, therapies, and potential cures. We’ll also continue to evaluate how to best support investment in rare diseases; and to encourage the development of drugs that target rare, unmet patient needs. A lot of devastating and rare conditions still lack approved therapy. During this Rare Disease Week, it’s gratifying to review the steps we’ve taken, and to commit to more progress in the future, and making sure that our framework supports the needs of patients.

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

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

Many “Firsts” for CDER’s 2017 Drug Approvals Reflect Innovation and Enhanced Patient Care

By: Janet Woodcock, M.D.

In 2017, FDA’s Center for Drug Evaluation and Research (CDER) approved many new drugs never before marketed in the United States, known as “novel” drugs, to help improve people’s health.

Janet WoodcockNovel drugs often represent innovative therapies for advancing patient care. 2017 was no exception. We approved new treatments for patients with rare diseases such as Batten disease, Chagas disease, and hemophilia A with inhibitors. We also approved new cancer therapies, new antibiotics, and new therapies for patients with multiple sclerosis, Parkinson’s disease, tardive dyskinesia, Duchenne muscular dystrophy, and amyotrophic lateral sclerosis (often called Lou Gehrig’s disease), among many others.

For the past six years, we have summarized our novel drug approvals in an annual report. This year, we expanded the report beyond novel approvals to show a wide range of other drug therapy approvals that help improve health. For example, we approved many new uses for drugs already on the market, extending their benefits and expanding their reach into new populations, such as children. We have also approved new dosage forms for drugs already available. These are the kinds of actions, separate from approving a novel drug, that can also provide important medical value.

How valuable are these other approvals? Let’s look at an answer in terms of “firsts.” There are many. For instance, there was the first approval of a drug to treat liver cancer in almost a decade, and the first approval of a drug to treat sickle cell disease in almost 20 years. Other firsts for approvals in 2017 will benefit certain patients affected by a wide range of medical conditions, including:

  • Giant cell arteritis, a dangerous condition that results in inflammation of blood vessels;
  • Cytokine release syndrome – a condition related to a reaction caused by a treatment called chimeric antigen receptor (CAR) T cell therapy;
  • Chronic graft versus host disease after a bone marrow transplant;
  • A type of blood cancer called marginal zone lymphoma;
  • Eosinophilic granulomatosis with polyangiitis, a rare autoimmune disease that causes vasculitis, an inflammation in the wall of blood vessels of the body; and,
  • Erdheim-Chester Disease, a rare cancer of the blood.

Other firsts for CDER’s drug therapy approvals that are not novel drug approvals include the first:

  • Biosimilars to treat certain cancers;
  • Immediate-release opioid product with properties intended to deter abuse;
  • Once-monthly injectable buprenorphine product to help patients struggling with opioid addiction;
  • Cancer treatment based on a genetic feature of the cancer rather than the location of the body where the tumor originated;
  • Treatment to help prevent recurrence of renal cell carcinoma (kidney cancer);
  • Complete regimen to treat HIV-1 that contains only two drugs, neither a nucleoside reverse transcriptase inhibitor, which can be detrimental to a patient’s kidneys, bones, and heart;
  • Drug approved in the United States with a sensor embedded in the pill that enables a patient to create an electronic record that the medication was taken; and,
  • Short-acting “follow-on” insulin product to treat patients with diabetes.

Our report Advancing Health through Innovation: New Drug Approvals and Other Drug Therapy Advances of 2017 tells the story of these and other important drug therapy approvals that occurred last year. The report also shows the variety of regulatory methods we used to efficiently execute our review and approval of novel drugs, while always prioritizing safety over speed. For example, CDER used at least one “expedited” development and review method for 61 percent (28) of the 46 novel drug approvals of 2017.

In part, as a result of CDER’s efficient methods, 78 percent (36 of 46) of the novel drugs approved in 2017 were approved in the United States before any other country, and 100 percent were approved within their targeted user fee date for application review, as per the goals of the Prescription Drug User Fee Act.

The increasingly vital role patients play in drug development and approval is very important to new drug regulation. CDER collaborates with a wide range of patient advocates and patients to ensure that patients’ perspectives are considered when the agency reviews development plans and drug applications that meet previously unmet needs. We also work with stakeholders in manufacturing, scientific, and medical organizations across the globe to help advance the science needed to develop and evaluate new drug therapies. Our report highlights some of the many public-private partnerships and consortia that CDER leads or participates in to support innovation and improved public health.

Finally, the 2017 report describes CDER’s response to the devastation inflicted by the 2017 hurricane season, working with hospitals and pharmaceutical manufacturers to limit drug shortages and keep those affected safe.

We hope our new report provides a deeper understanding of the many ways CDER works to support innovation and access to medications for improved public health.

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

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

Communication, Breaking Down Walls, and a Huge Step Forward for People with Type 1 Diabetes

By: Courtney Lias, Ph.D., and Stayce Beck, Ph.D., M.P.H.

At FDA’s Center for Devices and Radiological Health (CDRH) we recognize that medical innovators with a novel technology can get lost in navigating the regulatory landscape if there is no well-worn path to follow.

Without such guideposts, even well-intentioned scientists and businesses can make incorrect assumptions that can cost a lot of time and money, such as assuming FDA only will accept certain types of rigidly-defined data. Such assumptions inevitably delay patient access to important devices.

We’ve learned that to promote innovation we must break down walls and open lines of communication, an approach we used with great success recently in approving an automated insulin dosing system, commonly known as an “artificial pancreas” device.

Artificial Pancreas Diagram

Example of an Artificial Pancreas System
1. Glucose Sensor & Transmitter: Detects person’s blood glucose level and transmits that information to the glucose monitor and insulin pump.
2. Control Algorithm: Calculates dose and sends instructions to the insulin pump. This software can run on any number of devices, including directly on the insulin pump, or remotely from a laptop computer or a smart phone.
3. Continuous Glucose Monitor & Insulin Pump: Displays the person’s blood glucose levels and, based on calculations made by the control algorithm, administers the correct dose of insulin.
4. It is necessary to periodically calibrate the Continuous Glucose Monitor (3.) using a Blood Glucose Device.

Several FDA guidance documents and approvals had already laid the groundwork for the device, approved in 2016. But in years past, the diabetes and medical device communities commonly believed that FDA would never approve a device that does all of the thinking about when and how much insulin should be used without human input.

Companies had assumed that FDA would take an overly cautious approach to the study and development of this type of device which would delay its availability for U.S. patients.

To correct these assumptions and open a line of communication between FDA and the diabetes community, we developed a proactive approach with patients, their caregivers, device developers, academia, and the many doctors and scientists who have devoted their careers to developing automated insulin dosing systems.

Starting in 2012, FDA’s artificial pancreas team worked to better understand the daily struggles of living with type 1 diabetes by reaching out to patients and their caregivers. We heard from parents of young children with diabetes who often had to wake up multiple times every night to check their children’s blood sugar to ensure that it had not gone too high or too low in a way that can be dangerous.

These parents were worried that their children might not wake up the next day, and were willing to use a device that would help, even if that device is not perfect. These parents and patients provided valuable insight into the risks they were willing to take and helped us understand that even if this device increases risks in certain areas, it might decrease them in others. Some were willing to accept a slight increase in risk of long-term health effect due to high blood sugar, if the device provided greater assurance that their child would make it through each night safely.

We also developed productive relationships with key academic investigators and thought leaders who knew they could call us up and talk about any issues or concerns they might have about the required clinical trials, their design, or the trials themselves.

Much early stage work had already occurred that laid the foundation for an artificial pancreas, including the previous clearance of insulin pumps made by several manufacturers, approvals of interactive glucose monitors that transmit blood glucose readings to the pump, and the development of investigational algorithms that determine insulin doses based on blood sugar readings and other considerations, such as carbohydrate intake.

Lias and Beck image

Courtney Lias, Ph.D., (left) Director, Division of Chemistry and Toxicology Devices; and Stayce Beck, Ph.D., Chief of the Diabetes Diagnostics Branch, at CDRH. Lias, Beck, and the center’s Artificial Pancreas team received the 2017 Samuel J. Heyman Service to America Medal for Management Excellence for developing the first hybrid closed loop system to treat type 1 diabetes three years earlier than expected.

FDA’s artificial pancreas team met monthly for three years prior to approval with the manufacturer, Medtronic, to develop an efficient study design to evaluate their “closed looped system,” a significant step toward a truly artificial pancreas device. That least burdensome clinical research approach balanced pre-market and post-market data collection by allowing Medtronic to do a small, focused pre-market study to support approval of the device, followed by a larger post-market study to gather additional real-world information about use of the device.

We understood their device design and their clinical study approach before they even started the process of developing a medical device for market.

These meetings and relationships helped the artificial pancreas team understand the challenges and questions faced by all involved. It also gave the FDA team an opportunity to provide input on what factors would be critical during review of the product.

The results speak for themselves. FDA’s approval of Medtronic’s Minimed 670G hybrid closed loop system, the first FDA-approved device intended to automatically monitor glucose and provide appropriate basal insulin doses in people with type 1 diabetes came three years earlier than anticipated by the company and was a first-in-the-world approval.

We know that this effort is only the tip of the iceberg; much more work remains to be completed. FDA continues to engage the diabetes community, academic investigators, and industry to advance this and other novel device technologies. In the diabetes community, the walls are coming down, and we are excited to see the advances for patients that will result.

Courtney Lias, Ph.D., is Director, Division of Chemistry and Toxicology Devices, at the Center for Devices and Radiological Health; Stayce Beck, Ph.D., is Chief of the Diabetes Diagnostics Branch at the center. Lias, Beck, and the CDRH Artificial Pancreas team were awarded the 2017 Samuel J. Heyman Service to America Medal for Management Excellence for their work in the  approval of Medtronic’s Minimed 670G hybrid system.

New FDA/EMA rare diseases and patient engagement clusters underway

By: Jonathan Goldsmith, M.D., FACP, and Sandy Kweder, M.D., RADM (Ret.) US Public Health Service

Drug development and approval happens across the globe and we at FDA strive to collaborate with other countries and international regulatory agencies to ensure public health. One of our most valuable collaborators is the European Medicines Agency (EMA) — our counterpart agency for drug regulation in Europe that coordinates a network of 4,500 scientists and evaluates and supervises medicines for more than 500 million people in 31 countries.

Dr. Jonathan Goldsmith

Jonathan C. Goldsmith, M.D., FACP, FDA’s Associate Director Rare Diseases Program, Center for Drug Evaluation and Research, Office of New Drugs

For more than a decade, FDA and EMA scientists have collaborated to help solve some of our biggest challenges. We work with them in groups called “clusters.” The first cluster was initiated in 2004. Since then clusters have been formed to focus on treatments for children; establish effective measures for the development and use of biosimilar medications as cost effective alternatives to brand name biologic drugs; evaluate new treatments for patients with cancer; set standards to help develop medicines personalized to a patient’s genetic makeup, and much more. Both agencies have benefited from this joint work. The EMA summarizes these and our other clusters on its website.

We are excited about the initiation of our most recent cluster activity with our EMA colleagues. Just last month we established a cluster that will work to advance treatments for patients with rare diseases. This cluster’s primary goal is for FDA and EMA scientists to share valuable information about their work and to collaborate on certain review aspects of rare disease drug development programs. FDA’s core members of the cluster include experts from FDA’s Center for Drug Evaluation and Research’s Rare Diseases Program, the Office of Pediatric Therapeutics, the Center for Biologics Evaluation and Research’s director’s office, and the Office of Orphan Products Development, but other experts will be engaged on specific topic areas as the cluster evolves. Among many other important activities, our agencies will collaborate on:

  • Identification and validation of trial end points;
  • Potential trial designs when only small populations of patients are available for testing the safety and effectiveness of prospective new therapies;
  • Ways to apply flexibility in evaluation of drug development programs;
  • Expediting the review and approval of drugs to treat rare diseases to bring new drugs to patients in need as soon as possible.
Sandra Kweder

Sandra Kweder, M.D., Rear Admiral (Ret.) US Public Health Service, FDA’s Deputy Director, Europe Office, and Liaison to European Medicines Agency

Our work also builds on another exciting and recent development — a patient engagement cluster formed in June 2016 to incorporate the patient’s involvement and viewpoint in the drug development process. FDA and EMA are interested in understanding patient’s experiences and gaining input on their tolerance for risk and uncertainty, on current therapy and its benefits or shortcomings and on the benefits that patients seek. This cluster, among other valuable efforts, will:

  • Help each agency learn how the other involves patients in their work, and to develop common goals of expanding future engagement activities with patients;
  • Discuss ways for finding patients that can serve as spokespersons for their community;
  • Explore ideas to help train selected patients and advocates to effectively participate in agency activities, and;
  • Develop strategies for reporting the significant impact of patient involvement.

Given the focus of both of these new clusters, we expect they will address new areas of interest and also draw on expertise from all of the other clusters, such as oncology, pediatrics, and orphan diseases, contributing to more advanced and robust collaborations across both of our organizations.

Focusing on patients with rare diseases and working to advance patient input enhances the value of our cluster activities. With our colleagues at the EMA we look forward to accomplishing more than what we can individually.

Jonathan C. Goldsmith, M.D., FACP, FDA’s Associate Director, Rare Diseases Program, Center for Drug Evaluation and Research, Office of New Drugs

Sandra Kweder, M.D., Rear Admiral (Ret.) US Public Health Service, FDA’s Deputy Director, Europe Office, and Liaison to European Medicines Agency

The Unique Voices of Our Patient Representatives

By: Robert M. Califf, M.D., and Heidi C. Marchand, Pharm.D.

We recently met with 21 inspirational patients and patient caregivers who have made the extraordinary commitment to become FDA patient representatives. These volunteers were in Washington to participate in our two-day Patient Representative Workshop so they can receive training that will allow them to help FDA meet its critical responsibility of guiding the development and evaluation of safe and effective medical products.

Robert Califf

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

The patient representative program has existed since 1999 and is integral to fulfilling FDA’s strong commitment to ensure that the needs and choices of patients – as well as their families, caregivers, and advocates – are incorporated in ever greater ways in the work we do.

Patients add context and content to the cutting-edge science and other empirical evidence that is so important in our regulatory decision-making.  Including their perspectives and voices in our work along the entire medical product continuum, from development to review and evaluation to post-market surveillance, offers opportunities to enhance our knowledge of the benefits and risks of medical products. It’s not only smart science; it just makes good sense. We know, for instance, that patients who live with a chronic disease are experts in the tangible effects of that disease and its treatments.

The training that patient representatives receive helps prepare them to serve on FDA advisory committees, meetings and workshops, where they are knowledgeable about what it is like to cope with their disease – including such topics as side effects from treatments and important lifestyle issues. They also provide valuable contributions as consultants to our review staff.

Heidi Marchand

Heidi C. Marchand, Pharm.D., Assistant Commissioner in FDA’s Office of Health and Constituent Affairs

To give you an idea of the unique set of skills and experiences patient representatives bring to their work, consider the stories and experiences we heard at the workshop.

One was an elite world class athlete, who initially thought her pain was muscular in nature before it was diagnosed as a serious blood clot. She has been on a series of different products since then and is now intimately familiar with what it is like to be on anticoagulants – reflecting on both the benefits and risks of taking these medications.

Two of our patient representatives are caregivers who have a personal experience with a rare disease, Batten’s Disease, a fatal, inherited disorder of the nervous system. Sadly, each lost a young son to the disease. But in the face of this tragedy, these two mothers have advocated tirelessly to find a cure for this disease and worked to educate other parents.

Another mother related the story of her daughter who, at age 16, survived two craniotomies to remove a lemon-sized brain tumor. The daughter went on to receive of 48 weeks of chemotherapy and 8 weeks of brain and spine radiation. The daughter is now 33 years old and doing well. And the mother told us how critical it was for her daughter to take an opioid to relieve her pain. This kind of input, from those who have experienced it first hand, is critical to our future decisions.

2016 FDA Patient Representative Group photo

FDA Patient Representatives at the 12th Annual FDA Patient Representative Workshop, hosted by FDA’s Office of Health and Constituent Affairs

The stories that these patient representatives tell are moving. But even more moving – and indeed inspirational – is their commitment to the future. That’s why they were selected – because of their individual involvement with their respective patient communities, their analytical skills, and their ability to maintain an open mind and consider options.

While we will help train them about the nuts and bolts of FDA – such as the various pathways that products take to get to market – it is their personal experience and their ability to understand and to articulate the perspectives, concerns, and experiences of patients – that makes them truly special.

As we continue to evaluate potential treatments and cures for different diseases, we must make sure that patients are more than simply statistics in this equation. They are real people, with names, faces, and, thanks to these patient representatives, important voices who represent an essential piece of the puzzle to be solved.

FDA is committed to looking for new and better ways to integrate the patient voice. Our patient representatives are an important piece of this commitment. They have an extraordinary impact. We thank them for their service and commitment, and look forward to working with them.

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

Heidi C. Marchand, Pharm.D., is Assistant Commissioner in FDA’s Office of Health and Constituent Affairs