Speech by Scott Gottlieb, M.D.
Commissioner of Food and Drugs
National Press Club, Washington, DC
November 3, 2017
(Remarks as prepared for delivery)
Thank you for the opportunity to be here today.
I want to start by extending my apologies for cancelling my earlier appearance at the Press Club. I had to travel to Puerto Rico that day on urgent business, to assess the impact of hurricane Maria on our FDA facility in San Juan, our staff, and the people of Puerto Rico.
When I arrived at the FDA’s facility in San Juan, I witnessed the emotion of the assembled FDA staff, as one of the FDA team members, who had not been heard from since the storm, showed up for our meeting.
Her FDA colleagues had feared the worst. They were overcome to see she was unharmed. She had been tending to her own destroyed home and displaced family. It was her first contact with the FDA team.
The destruction is profound. Despair is widespread.
The stories I heard from FDA’s team made the ongoing hardships clear. Yet they’ve all stood their post. They’ve been working day and night to help get Puerto Rico’s medical product manufacturing restarted.
Even as their own homes were destroyed and most of the island remained without power, our team was able to make 130 firm visits, to help manufacturing sites get restarted. In 113 of the visits, they were able to make contact with the firm, and in 99 cases, the firms were operational. This is a monumental task given the logistical challenges they face in moving around the Island, and the personal challenges they face at home. I’m proud of their effort, and deeply moved by their dedication.
I want to focus my remarks today on some of the efforts we have underway at FDA relating to medical product development.
I’ve been at FDA for six months now as the agency’s Commissioner. But, as some of you know, I’m not new to the FDA.
This is my third time serving at the agency. I held three different positions at FDA during a span of almost 15 years.
In between my roles at FDA, I worked in the private sector. The chance to see FDA’s work from both inside and outside the agency has shaped my approach to my current role -- and shaped my perspective and understanding of what I think inspires FDA’s unique mission.
From this vantage point as Commissioner, I can tell you with certainty that FDA is a mission-driven organization motivated by a unique esprit de corps. There’s a shared sense of public health mission that animates the agency’s work. It might sound quaint in some quarters to say that your job is to protect and promote the public health. But at FDA, people voice this call to duty without a hint of irony.
It’s this spirit of mission that inspires us.
When people want to know about the agency they often ask how we achieve our mission. And most go directly to try and understand the “what.” What is it that we do? But that’s the wrong question to ask.
The right question is: why does FDA do what it does?
I want to focus my remarks today on the “why;” -- “why” FDA does the work we do to describe the heart of our mission.
To understand FDA is to understand “why” we do what we do.
But to answer the why, I’d start by asking: “why have an FDA at all?”
We have an FDA to help make it easier for people to be a parent or caretaker, and to improve their lives.
The FDA exists to empower people to make choices and decisions about their own health and the health of their families.
To give people access to safe and effective technologies that can provide them with meaningful choices when they face a serious illness.
To offer hope that they can cure an acute disease, or more reasonably manage a chronic one. And to protect them from potential harms
That “why” also describes the foundation of American public health: Simply put, our mission, and the mission of public health, is to help people live up to their full potential. That “why” is to advance the health of our nation; and this influence is central to us flourishing.
The question of why we do what we do is central to the organizational and policy reforms we’re undertaking at FDA, for instance when it comes to our medical product review programs. I want to highlight one particular idea today. It brings to life a broader change that’s underway in our organizational approach to new medical product review.
We’re changing how we organize ourselves as part of the medical product review process, and moving away from a structure that had people working in discrete organizational units that often operated as independent entities rather than an integrated team that functioned together to share best practices and knowledge.
Instead, we’re evolving toward a more team-based approach. This approach will integrate people from different disciplines – and across different stages of the life cycle of a product from the pre- and post-market phases -- who are working toward a common public health goal.
In most cases, that immediate goal will be the review of a new product application. But the ultimate goal is to facilitate a fluid and dynamic team environment that fosters a deep understanding of these products, across the full continuum of the pre-market and post-market phases.
We’ve already started down the path of enabling this organizational approach in the creation of our Oncology Center for Excellence. The OCE brings together cross-center teams to work together to examine products to treat cancer. So rather than focusing on the primary mechanism of action, or on the kind of product platform being used, teams are grouped based on their deep understanding of the disease.
Similar changes are underway in our drug and device review programs.
I believe these changes will elevate the role of our clinical and scientific experts to take a more universal view of the products they evaluate – a role where they take more stewardship of products over their entire life cycle -- from the initial product application, to its review by FDA, to the approval and safe use of a product by patients and providers.
Our experts are our clinical and scientific officers. They must have a stewardship over the products they evaluate that extends throughout the entire product lifecycle. That’s their commitment to public health.
The connection between the products we regulate, and the lives we seek to improve over time, is what first brought many of us to FDA.
The benefit that people ultimately derive from a new product after it’s approved, and the risks they might encounter in the ordinary routine of clinical medicine, is our shared responsibility and obligation.
It’s the outcome that expresses why we do our work. And so the same commitments that stir our efforts before a product is approved for use are equally important after it’s made more widely available.
We need each of our medical and scientific experts to have more opportunity to extend their expertise, and leave their mark, over the full duration of a product’s life cycle rather than just one stage.
Our new organizational models will work toward this goal, by engaging more of our experts in each phase of product review.
Part of our effort to modernize the structure of our review teams is as much a cultural change as it is an organizational one.
I’ll focus on these changes first as they relate to devices, where this modernization is embodied by the creation of a “Total Product Life Cycle Office.” This new structure consolidates many of the current aspects of product review into a new, team-based approach.
Our clinical and scientific staff is comprised of some of the leading experts in their fields. To maximize their effectiveness and efficiency, and fully leverage and integrate their knowledge and expertise into product review, we’re changing from an individual-centric approach to a team approach. It’s key that our organizational structure supports that purpose. But that’s not always the case today.
Instead, the current organization often fosters intellectual and managerial siloes. It splits pre-market and post-market functions into separate offices that don’t talk to one another as much as they should. It places staff into a hierarchical structure and management chain.
This makes it more difficult to share information and to hand off work between the offices – for example, between our compliance officers and our pre-market experts. Often expert input across different parts of the review function is sought through consults rather than an ongoing dialogue offered as part of an integrated review team.
One of the key purposes of our new approach is to make information sharing easier. Reviewers, compliance officers, and other experts will look at the product’s total life cycle rather than different staff looking at different devices at different stages of their development and commercialization. Regulatory oversight will span the continuum of pre-market and post market functions and product evolution.
The aim is to make sure that the people with expertise in how a product works can also inform those who are monitoring its continued performance after it’s approved for use by patients and vice versa.
This means combining the medical device Office of Compliance, Office of Surveillance and Biometrics, and Office of Device Evaluation into a new Total Product Life Cycle Super Office. The device program will be organized around new teams that break down the branches that created the current structure and facilitated the silo culture.
The new configuration will eventually comprise six different device specific offices and each of these offices will be responsible for pre-market review, post market surveillance, and device and manufacturing quality and compliance.
We’re also pursuing similar organizational changes when it comes to new drugs. These modernizations have the same public health goals as those embodied in the new efforts related to medical devices.
Under the leadership of Dr. Janet Woodcock, the Office of New Drugs is evaluating a series of structural changes to address how new science is changing the nature of how drugs are developed.
Our Center for Drug Evaluation and Research is piloting the creation of one common, shared review memorandum. This will ensure early, cross-disciplinary interaction among scientists and clinicians who have specialized knowledge in disease that inform product review. These interactions have become more critical because fields such as genomics, human factors analysis, advanced modeling, immunology and others have become integral parts of the drug review process. A single review memorandum will also be much more accessible to the biomedical research community.
At the same time, we’re evaluating the creation of more disease-specific offices as part of a more modern approach to the Office of New Drugs. The goals are to provide stakeholders with a single point of contact, and to allow synergies and surge capacity within offices.
The broader community often measures FDA’s productivity by its adherence to review goals. These are the timelines that are embodied by our deadlines negotiated as part of our user fee agreements.
These are important metrics for measuring our organizational efficiency and we intend to hit these commitments. But goal dates aren’t always good approximates for our public health impact. Our impact can best be measured by the completeness and the quality of our clinical and scientific work before and after a new product is approved; by the safe and effective medical technology that we help facilitate; and how good we are at advancing products that also help advance peoples’ health.
A central tenet of these new team-based approaches is to increase cross-disciplinary collaboration. The goal is to make sure decision-making at every stage of a product’s review is more fully informed by scientists and clinicians with very discrete and deep areas of expertise.
This gets me back to the why of our mission.
It isn’t simply to meet a user fee goal, or to approve more novel products. It’s to make sure we’re having a meaningful impact in people’s health, and positively impacting their lives. The impact of our work is becoming especially palpable as we see more products coming to market that have transformative and even curative effects on vexing diseases.
The “why” of our work is deeply expressed in a lot of other areas of our portfolio. The most prominent, I believe, are FDA’s efforts to impact America’s crises of addiction. This goal is very clearly embodied in our new initiative on the regulation of tobacco and nicotine.
The nicotine in cigarettes doesn’t directly cause tobacco related cancer, lung disease, or heart disease. But the powerfully addictive nature of the delivery of nicotine in combustible cigarettes makes tobacco use the leading cause of preventable death in the United States.
So we’re putting nicotine at the center of our regulatory strategy. We’re taking steps to render combustible cigarettes minimally or non-addictive. This could prevent future generations of kids from becoming addicted to cigarettes, the deadliest form of nicotine delivery. We’ve said our goal is to issue an advanced notice of proposed rulemaking related to the regulation of nicotine before the end of this year.
At the same time, we’re putting through an appropriate series of regulatory gates, new technology that’s emerging that could deliver nicotine to those adults who still want or need satisfying levels of this drug, but that can enable them to get that nicotine in a way that may pose far less risk than smoking combustible cigarettes.
We need to make sure these new products, like electronic nicotine delivery systems, are properly regulated. For example, if they claim the product modifies the risk to users, they must prove that they can significantly reduce that risk.
Our job is to make sure that, in fact, they do this -- prior to marketing. That’s what we intend to do. We believe this balanced approach can sharply reduce rates of smoking combustible cigarettes by rendering combustible cigarettes minimally or non-addictive, while also allowing products that can deliver nicotine more safely to continue to advance.
The expected public health impacts from the reduction in tobacco-related disease over time will be enormous. We’ll soon publish our detailed modeling that quantifies these expected benefits.
We’re also focused on another devastating addiction crisis in America: The addiction to opioids. As you know, this is a top priority of the administration and, as such, the FDA has an important role to play in every aspect of this crisis. But two of our key obligations are FDA’s influence on the rate of new addiction; and our impact on the availability and use of treatments that can help people live lives of sobriety.
We know that many people who become addicted to opioids will become medically addicted. And their first exposure will be through a lawful prescription. For most people, that first prescription will be for an immediate release formulation of an opioid drug.
Science based evidence shows that the key to reducing new addiction is to reduce exposure to opioid drugs in the clinical setting. This means making sure that fewer prescriptions are written, and shorter durations of doses are dispensed. We recently released a Federal Register notice that will begin a process at FDA to evaluate, and perhaps implement, steps reduce exposure to opioids through our influence on prescribers. Some of the steps we’re evaluating are how we require doctors to be educated, our role in regulating how products are packaged, and how doses are dispensed based on indication; among other influences we believe we can have on bending the rate of new addiction.
As another part of our work to address the opioid epidemic, we’re reconsidering how we address risk and benefit to make sure we’re taking appropriate measure of the risk associated with misuse and abuse of opioid drugs, both as part of our pre- and post-market review.
As one part of this effort, we requested earlier this year that Endo Pharmaceuticals withdraw its reformulated Opana ER from the market, based on our analysis of the risks associated with that drug’s illicit use.
I recently have seen media reports stating that Endo is participating in a re-launch of the old version of Opana ER. This is the version of that drug that Endo had previously withdrawn from the market when it launched its reformulated version of Opana -- because that older version didn’t purport to have abuse deterrent features.
I don’t want to speak about our regulatory intentions with respect to any specific drug. But I do want to address oxymorphone products more generally.
FDA previously commissioned a study to formally evaluate whether oxymorphone, an active ingredient in certain opioid drugs, has qualities that make it more likely to be abused than other Schedule II opioids, including through illicit routes of administration such as snorting and injection. I’m announcing that study for the first time. If the scientific results of this study demonstrate that this ingredient has qualities that make it more likely to be abused, FDA would consider taking regulatory actions that could limit patient exposure to oxymorphone.
In closing, the aim of the organizational and policy changes I discussed today is to empower the scientific and clinical experts at FDA; with a primary objective in mind. It’s to more fully engage – and inspire – their work -- day in and day out -- to solve similar public health challenges.
Our goal is to make it simpler for our scientists and physicians to pursue and accomplish these goals. By working as teams, by sharing different expertise, we’ll be more closely aligned as an organization around a common ambition -- to enable people to have more opportunities to use diets and novel medical products to improve their lives.
That’s the “why” of our work: It’s the common thread that links our shared goals. And it’s the principle that underlies our public health mission.
Thank you for the opportunity to join you today.