Statement from FDA Commissioner Scott Gottlieb, M.D., on the approval of a new formulation of buprenorphine and FDA’s efforts to promote more widespread innovation and access to opioid addiction treatments

FDA Statement

• For Immediate Release: November 30, 2017

With the approval today of a monthly formulation of the drug buprenorphine for the treatment of opioid use disorder, patients have access to a new and longer-acting option for the treatment of opioid addiction. Millions of Americans are suffering from addiction to opioid drugs, and millions more are worried that the overdose epidemic could claim the lives of a friend or loved one. We need immediate actions to help those suffering from an opioid use disorder transition to lives of sobriety.

An urgent need is broad access to high-quality, effective treatment. The President’s Commission on Combating Drug Addiction and the Opioid Crisis found that treatment of opioid addiction that includes medications “has been associated with reduced overdose deaths, retention of persons in treatment, decreased heroin use, reduced relapse, and prevention of the spread of infectious disease.”

Medication-assisted treatment (MAT) for opioid addiction uses drugs to stabilize brain chemistry, reduce or block the euphoric effects of opioids, relieve physiological cravings and normalize body functions. There are three drugs approved by the FDA for the treatment of opioid addiction: buprenorphine, methadone and naltrexone. All three of these treatments have been demonstrated to be safe and effective in combination with counseling and psychosocial support. Everyone who seeks treatment for an opioid use disorder should be offered access to all three options. This allows providers to work with patients to select the treatment best suited to a patient’s individual needs.

The FDA plays a critical part in the use of medications for opioid addiction treatment, first and foremost, through its role in the approval of new drugs. Given the scope of the epidemic, we believe our efforts should go beyond the approval of these treatments. We believe that in light of the agency’s public health mandate, and the gravity of this crisis, the FDA also should encourage wider, appropriate use of these safe and effective medications. The agency is committed to taking new steps to advance each of these efforts.

Promoting Development of New Treatments

The FDA is taking steps to facilitate the development of new medications for the treatment of opioid use disorder and new formulations of the existing drugs that could have attributes that are better tailored to patient needs. At the same time, the FDA is taking a more active role in supporting wider adoption of these drugs as part of comprehensive treatment programs, working in collaboration with other health agencies.

As part of our effort to support the development of new formulations of existing MAT, and new kinds of drugs altogether, the FDA intends to issue two guidance documents. The first will detail the agency’s current guidelines for the approval of depot formulations of buprenorphine for opioid dependence. The second will expand on these guidelines to describe how to establish additional endpoints that may help facilitate the development of better treatments.

As part of these guidance documents, the FDA intends to expand on two new principles that we believe can help make the pathway for developing new MAT more efficient.

The new guidance will create a framework for linking exposure levels to the safety and benefit of new, longer-acting formulations of existing drugs. We believe that the agency’s substantial investments in modeling and simulation techniques to support these therapeutic effects can contribute to the development of this new framework for the approval of improved formulations of existing treatments.

Second, the FDA intends to provide a framework for the development of novel clinical endpoints that can form the basis of product approvals. For example, one endpoint that’s relevant to the treatment of addiction is the craving that fuels continued drug use. Craving is an endpoint that the FDA has included in labeling for smoking cessation products, and the agency intends to provide assistance to develop a validated measurement of “craving” or “urge to use” illicit opioids to complement other endpoints and to determine how it supports the goal of sustained abstinence.

Promoting Wider Adoption of Effective Therapies

Unfortunately, far too few people who are addicted to opioids are offered an adequate chance for treatment that uses medications. In part, this is because private insurance coverage for treatment with medications is often inadequate. Even among those who can access some sort of treatment, it’s often prohibitively difficult to access FDA-approved addiction medications. While more states are adopting broader coverage owing to new legislation and resources, among public insurance plans there are still a number of states that are not covering all three FDA-approved addiction medications.

To tackle the treatment gap, the FDA is taking several steps. First, the FDA will convene experts and stakeholders to discuss the evidence of treatment benefits at the population level – such as studies that show community wide reductions in overdose following expansion of access to therapy. While the evidence base demonstrating the benefits of MAT is already substantial, the agency is especially interested in research that can lead to additional label indications that could, in turn, help support more widespread insurance coverage. The protocol the FDA is developing is intended to support a claim that availability of MAT at the point of care ‒ when a patient presents with overdose ‒ could lead to reduction in death over a population.

Such an effort would be a first for the FDA.

We believe such a finding would reflect the urgency of not only expanding coverage, but also assuring access to this life-saving treatment.

For example, it is astonishing that very few criminal justice systems routinely offer treatment that includes options for the three medications for all those in need who are detained. As a result, at the very moment when the criminal justice system could be dramatically lowering the risk of overdose, it is creating the conditions of reduced tolerance to opioids that substantially raises the risk of death upon release. The President’s Commission correctly recommended that this failure come to an end.

At the same time, the FDA is re-evaluating the current labels on the approved treatments to make sure they reflect the available science. One area of interest is duration of use. Clinical evidence shows that people may need treatment with medications for long periods of time to achieve a sustained recovery. Some may even need a lifetime of treatment. The FDA recently revised the labels of buprenorphine products to reflect this fact. Similarly, we are evaluating the labeling of the other two medications.

Finally, to support more widespread adoption of MAT, the FDA will continue to take steps to address the unfortunate stigma that’s sometimes associated with use of these products. It’s part of our public health mandate to promote appropriate use of therapies.

Misunderstanding around the profile of these products ‒ even among many in the medical and addiction fields ‒ enables stigma to attach to their use. These stigmas can serve to keep patients who are seeking a life of sobriety from reaching their goal. In this case, in the setting of a public health crisis, it is imperative that the FDA take a more active role in challenging these conventions around medication-assisted therapy.

The stigma reflects a view some have; that a patient is still suffering from addiction even when they’re in full recovery, just because they require medication to treat their illness. This attitude stems from a key misunderstanding of the difference between a physical dependence and an addiction. Because of the biology of the human body, everyone who uses a meaningful dose of opioids for a modest length of time develops a physical dependence. This means that there are withdrawal symptoms after the use stops. Even a cancer patient requiring long-term treatment with opioids for the adequate treatment of metastatic pain develops a physical dependence to the opioid medication. A physical dependence to an opioid drug is very different than being addicted such a medication.

Addiction requires the continued use of opioids despite harmful consequences on someone’s life. Addiction involves a psychological preoccupation to obtain and use opioids above and beyond a physical dependence. But someone who is physically dependent on opioids as a result of the treatment of pain but who is not craving the drugs is not addicted. The same principle applies to drugs used to treat opioid addiction that are replacement therapy. Someone who requires long-term treatment for opioid addiction with medications ‒ including those that are partial or complete opioid agonists and can create a physical dependence ‒ is not addicted to those medications.

The approval today is one step toward more options for the treatment of those addicted to opioids. The FDA is committed to taking steps to limit exposure to prescription opioids through measures to make sure prescribing is appropriate and carefully reflects evidence-based clinical considerations. We believe the FDA has a significant role to play in reducing the rate of new addiction through its efforts to impact current prescribing. We welcome additional product development aimed at creating new treatments for opioid addiction. Such therapies may qualify for the agency’s expedited review programs.

But with so many people currently addicted to opioids, the agency is devoting equal vigor to efforts to support the availability of lifesaving treatment to those most in need today. This includes steps to encourage the development of new forms of MAT, and better formulations of the existing, approved drugs. The more widespread adoption of MAT, coupled with relevant social, medical and psychological services has the highest probability of being the most effective of all available treatments for opioid addiction.

The FDA, an agency within the U.S. Department of Health and Human Services, promotes and protects the public health by, among other things, assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.