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Remarks from FDA Commissioner Scott Gottlieb, M.D., as prepared for oral testimony before the House Committee on Energy and Commerce Hearing, “Federal Efforts to Combat the Opioid Crisis: A Status Update on CARA and Other Initiatives

Chairman Walden and Ranking Member Pallone, thank you for the opportunity to testify today before the full committee.

The epidemic of opioid addiction that’s devastating our nation is the biggest crisis facing public health officials, FDA included.

As this crisis grew, many of us didn’t recognize the consequence of this threat. We missed opportunities to stem its spread. So we find ourselves at a tragic crossroad.

We have a crisis of such massive proportion that the actions we need to take are going to be hard.

We’ll need to touch clinical practice in ways that may make certain parties uncomfortable. This may include such steps as restrictions on prescribing, or mandatory education on providers. Long ago, we ran out of straightforward options.

At FDA, we’re working across the full scope of our regulatory obligations to impact this crisis.

That means updating and extending the risk management plans and educational requirements that we impose on sponsors as a condition of a product’s approval.

It means doubling our efforts to promote the development of new, less addictive pain remedies; as well as opioids that are harder to manipulate and abuse.

It means updating our risk-benefit framework to take measure of the risks associated with misuse and abuse of opioids; and using this information to inform our decisions -- including recommending that products be withdrawn from the market.

These steps and others are needed to prevent new addiction.

But given the scale of the epidemic, with millions of Americans already affected, prevention is not enough.

We must also help those who are suffering from addiction by expanding access to lifesaving treatment.

I’d like to announce three new steps towards this goal today.

First, FDA will issue guidance for product developers as a way to promote the development of new addiction treatments.

As part of this guidance, we’ll clearly lay out our interest in the development and use of novel, non-abstinence-based endpoints as part of product development. We’ll also aim to make it easier to develop new products that address a fuller range of the symptoms of addiction such as craving.

Second, FDA will take steps promote the more widespread use of existing, safe and effective, FDA approved therapies to help combat addiction. There are several FDA approved treatments. All of these treatments work in combination with counseling and psychosocial support. Everyone who seeks treatment deserves the opportunity to be offered all three options as a way to allow patients and providers to select the treatment best suited to the needs of each individual patient.

How do we know that medications work?

To start, the FDA approval process requires that all products demonstrate that they are safe and effective prior to approval.

The evidence base then grows significantly after approval.

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 insurance coverage for treatment with medications is often inadequate.

To tackle the treatment gap, FDA is planning to 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. We will also discuss the potential for further research to assess the value of these medications in the context of today’s overdose epidemic.

There’s a wealth of information supporting the use of these medications. We’re focusing on the data in the drug labeling that can help drive broader and appropriate prescribing. So one concept that FDA is actively pursuing is the research necessary to support a label indication for medication-assisted treatment for everyone who presents with an overdose, based on data showing a reduction in death at a broader population-level. Such an effort would be a first for FDA. We believe that granting such an indication in labeling can help promote more widespread use of, and coverage for these treatments.

Why is it important to promote the value of these treatments at the population level? Here’s an example. Recently, the Commonwealth of Massachusetts found a greater than 50 percent reduction in the risk of death from overdose among individuals treated with methadone or buprenorphine after a nonfatal overdose. These kinds of data have immense implications for insurers and policymakers in deciding how to adopt these treatments.

A common question that arises with treatment is the proper duration of medication therapy. 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. Recognizing this, the FDA is revising the labels of these medical products to reflect this fact.

Now I know this may make some people uncomfortable. That’s why the third step I’m announcing today is that FDA will join efforts to break the stigma associated with medications used for addiction treatment. This means taking a more active role in speaking out about the proper use of these drugs.

It’s part of our existing public health mandate to promote the appropriate use of medicine. Misunderstanding around the profile of these products enables stigma to attach to their use.

This stigma serves to keep many Americans who are seeking a life of sobriety from reaching their goal. In this case, in the setting of a public health crisis, we need to take a more active role in challenging these conventions around medical 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 reveals a flawed interpretation of science. It stems from a key misunderstanding many of us have of the difference between a physical dependence and an addiction.

Because of the biology of the human body, everyone who uses opioids for any length of time develops a physical dependence—meaning there are withdrawal symptoms after the use stops. Even a cancer patient requiring long-term treatment for the adequate treatment of metastatic pain develops a physical dependence to the opioid medication.

That’s very different than being addicted.

Addiction requires the continued use of opioids despite harmful consequences. Addiction involves a psychological craving above and beyond a physical dependence.

Someone who neglects his family, has trouble holding a job, or commits crimes to obtain opioids has an addiction.

But someone who is physically dependent on opioids as a result of the treatment of pain but who is not craving more or harming themselves or others is not addicted.

The same principle applies to medications used to treat opioid addiction. Someone who requires long-term treatment for opioid addiction with medications – including those that cause a physical dependence --- is not addicted to those medications.

Here’s the bottom line:

We should not consider people who hold jobs, re-engage with their families, and regain control over their lives through treatment that uses medications to be addicted.

Rather, we should consider them to be role models in the fight against the opioid epidemic.

Committee members, we need to embrace long-term treatment with proven therapies to address this crisis. At FDA, we will step up our efforts to do our part to promote these goals.

I look forward to discussing these issues with the committee. And I’m grateful for the opportunity to be here.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by 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.