- Speech by
Leadership RoleCommissioner of Food and Drugs - Food and Drug Administration
Remarks by Scott Gottlieb, M.D.
Commissioner of Food and Drugs
Silver Spring, MD
Remarks as prepared for delivery before the FDA meeting on Data and Methods for Evaluating the Impact of Opioid Formulations with Properties Designed to Deter Abuse in the Post Market Setting: A Scientific Discussion of Present and Future Capabilities
I want to thank you for coming today, to discuss how we can improve the science around evaluating the impact of opioid formulations that might be less prone to manipulation, misuse, and abuse. We’re grateful for the chance to discuss how we have been approaching these evaluations. This scientific discussion will inform our development of an effective and efficient regulatory framework, so that we can facilitate the continued development of these formulation advances.
I especially want to thank my FDA colleagues who are here today. I know you have worked hard on these issues for many years.
Opioid addiction and the resulting overdoses and deaths are an enormous national crisis. The men and women of FDA are working to help address this epidemic; at the same time, we continue to make sure that properly indicated patients, who are suffering from pain conditions, have appropriate access to medicines. This crisis is, in my view, the toughest public health challenge that we face at FDA.
I’ve asked my FDA colleagues to take a fresh look at what more we can do to confront this challenge and change the trajectory of the epidemic of addition afflicting our nation. We need to make sure we strike a careful balance between access and safety while taking more vigorous steps to combat the epidemic. I’m immensely grateful for the dedication of the professional staff at FDA in pursuing these goals, and the efforts of our experts, who work every day on these issues.
There are many elements to the work FDA is doing to confront this epidemic. Today, I want to highlight three of the clinical and policy areas that I’ve asked my FDA colleagues to take a fresh look at since I’ve arrived at the agency.
The first is how we combat the crisis of new addiction.
This relates to people who will be exposed to opioids in the clinical setting, through a prescribed treatment, and then go on to become addicted to these drugs. To reduce the rate of new addiction, we need to decrease overall exposure to opioids. We need to make sure that only appropriately indicated patients are prescribed opioids, and that the prescriptions are for durations and doses that properly match the clinical reason for which the drug is being prescribed in the first place.
Given what we already know about the scope of current prescribing, and the subsequent patterns of abuse, it’s clear that there should be fewer prescriptions being written for opioids. When opioid prescriptions are written, they should be done so for shorter durations of use. I believe there are still too many thirty-day prescriptions being written for conditions like dental procedures or minor surgery, which should require very short-term use, if they require an opioid prescription at all. Therefore, we are exploring whether FDA should take additional steps to make sure that general prescribing, and the number of opioid doses that an individual patient can be dispensed is more closely tailored to the medical indication.
The second area I’ve asked my colleagues to examine is how we balance benefit and risk when it comes to scheduled drugs, or controlled substances. In particular, how do we look at benefit and risk not only in the labeled indication for opioid drugs, but also evaluate the individual and societal risks associated with their illicit use?
The question is this: What more can we do, and do we have the right regulatory tools, policies and science for assessing the overall risk associated with the illicit use of these drugs? This means carefully re-evaluating not only how we make decisions to approve new opioid drugs, but also how we continually assess their safety after approval. It also means carefully evaluating the framework we use for deciding when to revise labeling to better manage how these products are used, or make a decision to request that a marketed opioid drug should be withdrawn.
FDA has a clear legal and public health mandate to consider the safety of opioid drugs in terms of the risks and benefits of the labeled uses, as well as the risks associated with intentional or illicit misuse or abuse of the drugs. This regulatory principle is especially true when it comes to opioids, where intentional misuse or abuse is both too common, and associated with tragic outcomes. As an integral part of our efforts to address this epidemic, we’re exploring how this safety mandate can be further defined in support of our commitments to stem the tide of addiction.
To pursue these policies, we plan to continue to have public dialogue like this meeting. We need to make sure we have the scientific and policy base to implement effective new measures to achieve these and other public health goals. We’ll also continue to take more vigorous actions to address this crisis. Its enormous scope implores us to take aggressive actions. This is a public health emergency. We are treating it that way and we’ll continue to take assertive new steps.
The third area on which I’ve asked my colleagues to focus is improving prescriber training. Among the questions I’ve asked are these: whether the content of existing programs is appropriate to ensure that prescribing doctors are properly informed about appropriate prescribing recommendations, that prescribers understand how to identify the risk of abuse in individual patients, and know how to get addicted patients into treatment? And are there circumstances under which FDA should require some form of mandatory education for health care professionals.
As we continue to pursue a broad range of new steps to more forcefully address this public health crisis, I want to close by highlighting three new actions that we’re taking now, and announcing today, starting with additional steps on training.
First, we know that most of the exposure to opioids isn’t from extended release or long-acting formulations, which include most of the abuse deterrent formulations we’re discussing today. Most of the exposure to opioid drugs comes from the immediate release formulations like hydrocodone and acetaminophen or oxycodone and acetaminophen combinations. America is simply awash in immediate release opioid products. In fact, about 90 percent of all opioid prescriptions in the U.S. are written for the immediate release formulations of these drugs.
Many people who become addicted to opioids will eventually move on to seek higher dose formulations of these drugs or illicit street drugs, which are increasingly the low cost alternatives. But immediate release opioid products may serve as the gateway for patients and non-patients who may continue to use or misuse these products, which could lead to a lot of new addiction. And we all need to work to advance policies that rationalize the prescribing and dispensing of these products.
As one step, we’ve determined that a Risk Evaluation and Mitigation Strategy plan or REMS is necessary for the prescribing of immediate release opioid products. This regulatory tool is needed to ensure that the benefits of how these drugs are prescribed continue to outweigh the risks of misuse, abuse, addiction, overdose, and death. It’s time to take direct action to address the close to 200 million opioid analgesic prescriptions each year that are for the immediate release products.
To this end, FDA intends to update the existing REMS on extended release opioid analgesics, and for the first time, extend these same regulatory requirements to the manufacturers of immediate release opioid analgesic products. To start this process, the relevant letters, detailing the new requirements, will be sent to IR manufacturers in the coming weeks. The new training will be aimed at making sure providers who write prescriptions for the IR opioids are doing so for properly indicated patients, and under appropriate clinical circumstances. This is part of a broader effort to take new steps to make sure providers are properly informed about suitable prescribing and the risks and benefits associated with opioid drugs.
The new REMS will include modifications to the existing Blueprint for prescriber education which describes the content of the education. Under the new REMS, the training will continue to be provided by accredited continuing education providers.
As one part of the education for prescribers of immediate release and ER/LA opioids, FDA will broaden information on pain management, including the principles of acute and chronic pain management; non-pharmacologic treatments for pain; and pharmacologic treatments for pain (both non-opioid analgesic and opioid analgesic). The Blueprint will also enhance the information about the safe use of opioid analgesics, basic elements of addiction medicine, and opioid use disorders.
In addition to training for physician prescribers, the REMS will require that training also be made available to other health care providers involved in the management of patients with pain. This includes nurses and pharmacists. FDA believes that all health care providers involved in the management of pain should be educated about the safe use of opioids. Based on the feedback we’ve received from two public meetings over the past year, we’re actively exploring the question of whether, in the future, there should be mandatory provider education, and how we’d operationalize such a condition. As part of our new Opioid Steering Committee, we’ll be reviewing the data necessary to understand the most effective way to move forward.
We recognize that developing REMS for these widely prescribed products, involving numerous application holders, will present challenges. And we’re sensitive to concerns about the potential burdens they may place on providers. We’re taking these steps in a way that’s mindful of these concerns. We’ve solicited a lot of public input on the issues related to these steps. We’re carefully considering that feedback. We’ll monitor the execution of these new efforts, and adjust them as needed.
A second new action we’re taking is aimed at ensuring the safe use of the abuse deterrent opioid analgesic formulations, which mostly relate to the higher dose, extended release formulations of these medicines. We’re undertaking a new study to better understand prescriber beliefs and attitudes when it comes to these new opioid formulations. We want to know whether the perceptions about the attributes of these drugs match the clinical realities. In particular, we want to know whether we have the right nomenclature for describing the drug features that are expected to make opioids less prone to abuse. Among other steps, we’ll be surveying doctors to better assess how they perceive these terms, and understand the clinical understanding that’s been developing around the ADF products.
I want to make sure that the nomenclature we use to describe and label these products is accurately conveying their properties to those who prescribe and use them. In particular, we want to make sure that the labels and nomenclature enable providers to adequately distinguish between the risk of abuse and the risk of addiction. Through the regulatory lexicon we use to describe these products and their abuse deterrent features in drug labeling, we don’t want to improperly convey a perception that a product that’s resistant to manipulation and abuse is somehow also less prone to fueling addiction, when that’s is simply not true.
The term abuse is defined as the intentional, non-therapeutic use of a drug product or substance, even once, to achieve a desirable psychological or physiological effect. Different abuse-deterrent technologies target various known or expected routes of abuse. But the potential for abuse doesn’t necessarily correlate with the potential for addiction. Patients can still become addicted to opioid products with abuse deterrent features. We need to make sure these different risks are fully understood.
Third and finally, I want to highlight for you today that we’re also continuously re-evaluating the safety of approved opioid products based on post-market information. We’re also focused on how we can augment our post market data collection in these areas, which is one of the reasons we have convened this meeting today. And as we recently did with respect to reformulated Opana ER, when we find that the risks of an opioid outweigh its benefits – including the risks associated with illicit use and deliberate manipulation – we will take action. In some cases, that action could be to request the withdrawal of certain products.
These are just a few of the new steps that we’re taking. Today’s discussion is also a key part of our efforts. It’s an important part of our work to build the scientific base to improve our oversight of opioids, and make sure that we have the right policies to strike a careful balance between risk and benefit in these complex settings.
The crisis of opioid addiction is a public health tragedy of enormous proportions. We need to confront it like any explosive epidemic. We need to work together and take more vigorous steps to address it. But we need to make sure all of our actions are carefully considered with broad stakeholder input, and based on sound science.
FDA is immensely grateful for your efforts, and your willingness to join us today for this scientific discussion. Working together, we’ll aim to stem the tide of individuals becoming addicted to opioids, and misusing and abusing these products, and move those who are currently addicted to opioids into safe and effective treatment. All the while, we will continue to address the needs of patients suffering from pain.