presented by Nora D. Volkow, M.D., Director, National Institute on Drug Abuse National Institutes of Health Department of Health and Human Services
Testimony Before the House Subcommittee on Labor-HHS-Education Appropriations
Mr. Chairman and Members of the Committee:
I am pleased to present the President’s Fiscal Year (FY) 2017 budget request for the National Institute on Drug Abuse (NIDA) of the National Institutes of Health (NIH).
Drug Use and Addiction Research Priorities
As a part of NIH, the Nation’s premier biomedical research agency, NIDA’s mission is to advance science on the causes and consequences of drug use and addiction and to apply that knowledge to improve individual and public health. Over the last four decades, NIDA-supported research has revolutionized our understanding of drug use and addiction, transforming our understanding of the biological, social, and environmental factors that contribute to substance use disorders (SUD), and driving the development of improved strategies to prevent and treat SUDs.
Drug use and addiction represent major public health challenges in our Nation. In 2014 alone, over 47,000 Americans died as a result of an unintentional drug overdose, and the past few years have seen rapid increases in babies born with neonatal abstinence syndrome (NAS), an unprecedented local outbreak of HIV, an increasing prevalence of hepatitis C (HCV), and new synthetic drugs flooding the market. Despite these challenges, this is a time of great opportunities for addiction research. The last few years have seen tremendous advances in technologies with research applications – from gene sequencing and manipulation, to higher resolution brain imaging technologies, to mobile health tools and electronic health records. In 2015, NIDA released an updated strategic plan focused on leveraging these recent scientific and technological advances to re-envision what research can accomplish over the next five years.
The NIDA Strategic Plan for 2016-2020 outlines our broad goals across basic science, prevention, treatment, and public health and identifies four priority focus areas that we believe present unique opportunities to be leveraged over the next five years including:*
Understanding the complex interactions of biological, behavioral, and environmental factors influencing drug use trajectories. Capitalizing on emerging technologies and discoveries to facilitate integration and analysis of diverse data sources, including genomic, behavioral, neurobiological, environmental, and other data associated with drug use and addiction.
Accelerating the development of treatments for SUDs. Translating basic knowledge of the molecular pathways and brain circuits involved in SUDs to develop new therapeutics for SUDs and leveraging existing safety profiles and pharmacology data to lower development costs and shorten the timeline for obtaining FDA approval.
Addressing real-world complexities including comorbidities and poly-drug use. Conducting research to better understand the barriers to successful and sustainable implementation of evidence-based practices and developing implementation strategies that effectively overcome these barriers to ensure that all populations benefit from the Nation’s investments in scientific discoveries.
Advancing bi-directional translation from basic to clinical and applied research. Fostering stronger collaborations across basic and clinical researchers to integrate and coordinate human and animal research on addiction across the trajectory from initiation to recovery.
Within the strategic priorities discussed above, NIDA research is addressing ongoing national public health priorities including the epidemic of opioid overdoses, the impact of adolescent drug use on brain development, and the impact of tobacco use on health.
Addressing the Opioid Overdose Epidemic. In 2015, both President Obama and the Secretary of the Department of Health and Human Services launched initiatives to address the complex problem of prescription opioid and heroin abuse in this country. Both initiatives are focused on improving opioid prescribing practices, expanding dissemination of the opioid overdose reversal drug naloxone, and increasing access to medication assisted treatment (MAT) to treat opioid use disorders. NIDA-supported research contributes to both opioid initiatives through support for research on alternative treatments for pain with reduced potential for abuse; treatment of opioid use disorder; and development of lay-friendly naloxone formulations.
Development of alternative pain treatments with reduced abuse potential. While many strategies are currently being utilized to reverse the opioid overdose epidemic, there remains a pressing need to develop more effective treatments for chronic pain with reduced abuse potential. NIDA, in partnership with the NIH Pain Consortium, funds research to foster development of new pain treatments with reduced potential for abuse. Funded grants range from neurobiology, genetics/epigenetics, and molecular biology research that drives early stage drug target discovery, to therapeutics development including preclinical safety and efficacy testing and early phase human trials, to health services research. For example, NIDA research is supporting:
Testing of new compounds that exhibit novel properties as a result of their combined activity on two different opioid receptors (i.e., mu and delta). Preclinical animal studies show that these compounds can induce strong analgesia without producing tolerance or dependence.
Development of new non-opioid medications for severe pain including compounds that work through the endocannabinoid system and others that modulate members of the transient receptor potential (TRP) ion channel family.
Exploration of combinatorial approaches that utilize both opioid and non-opioid systems to minimize the dose of opioids needed for pain control.
Development of non-pharmacological strategies for pain treatment including transcranial magnetic and direct current stimulation of the brain, electrical deep brain stimulation, peripheral nerve stimulation, stem cell transplants, and integrative health approaches that consider the biopsychosocial nature of pain.
Improving Treatment of Opioid Use Disorders. An estimated 1.9 million people in the United States suffered from SUDs related to prescription opioid pain medicines in 2014, and 586,000 suffered from a heroin use disorder. Despite the availability of evidence-based pharmacotherapies for opioid use disorders, including methadone, buprenorphine, and extended-release naltrexone, we have a significant and ongoing treatment gap in our Nation with more than a million persons unable to access care and less than 40 percent of those being treated for opioid addiction receiving these medications. NIDA supports a broad portfolio of research to improve treatments for opioid use disorders (OUDs) ranging from basic research to identify novel targets for new therapeutics to preclinical and early clinical trials of new or improved therapeutics; to implementation science to improve the dissemination and effective use of evidence based practices.
For example, NIDA research supported the development of a buprenorphine implant (Probuphine), a novel formulation that provides stable round-the-clock dosing for six months. Buprenorphine is one of three FDA approved medications that has been shown to be a safe and highly effective treatment for opioid addiction. This new formulation delivers a steady dose of buprenorphine for six months, an innovation that improves the efficacy and acceptance of buprenorphine maintenance treatment by: 1) removing the need to take a daily pill, promoting continuous patient adherence; 2) preventing diversion of the drug; and 3) eliminating the risk of accidental ingestion by children. FDA accepted a resubmission of the Probuphine New Drug Application (NDA) that includes results from a Phase III double-blind clinical study in September of 2015. Agency action is expected by May 27, 2016.
Development of Lay-Friendly Formulations of Naloxone. The opioid overdose-reversal drug naloxone can rapidly restore normal respiration to a person who has stopped breathing as a result of overdose from heroin or prescription opioids. Naloxone is widely used by emergency medical personnel and some first responders and a growing number of communities have established overdose education and naloxone distribution programs that issue naloxone directly to opioid users and potential bystanders. As of 2014, more than 152,000 naloxone kits had been distributed to laypersons, and more than 26,000 overdoses had been reversed since 1996.
In late 2015, FDA approved a user-friendly intranasal formulation that was developed through a NIDA partnership with Lightlake Therapeutics, Inc. (a partner of Adapt Pharma Limited). This formulation can easily be used by potential bystanders (laypeople) and is an important advance beyond the improvised nasal devices (consisting of a syringe of injectable naloxone attached to an atomizer) that have been used in recent years.
NIDA also continues to support implementation science to develop strategies to improve the dissemination and sustainable implementation of naloxone distribution programs; to increase co-prescribing of naloxone to patients receiving opioid medications; to test strategies for teaching high risk individuals strategies for managing overdose and mitigating risk; and to test the efficacy of pharmacy based naloxone access polices.
Basic Research on Brain Development and Drug Use Trajectories. NIDA supports a robust portfolio of basic research to understand the mechanisms that underlie the transition from drug use to addiction and the brain changes that characterize SUDs. One illustrative example of this type of research is the Adolescent Brain Cognitive Development (ABCD) study, a landmark study led by NIDA in partnership with NIAAA, NCI, and other NIH partners that officially got underway in September 2015 with 13 grants. Researchers will recruit approximately 10,000 children at age 9 or 10 – before initiation of drug use – and collect detailed neuroimaging, genetic, behavioral, environmental, and other health data at periodic intervals over the course of a decade. This study will examine how biology and environment (including drug use) interact and relate to developmental outcomes such as physical health, mental health, and life achievements including academic success. This study will allow us to answer questions such as how does marihuana use during adolescence affect the development of the human brain and how does this subsequently influence behavior. Such information is crucial to guide prevention efforts.
Research on Tobacco Use and Health. Smoking is the leading preventable cause of disease, disability, and death in the United States. Over 480,000 Americans die each year from smoking and second hand smoke exposure. NIDA partners with FDA’s Center for Tobacco Products on the Tobacco Regulatory Science Program (TRSP) to support research on the impact of tobacco-related policies on population health and on the Population Assessment of Tobacco and Health (PATH) Study, a national longitudinal study of tobacco use and how it affects the health of people in the United States. One recent study evaluated the effects of smoking cigarettes that contained different levels of nicotine among current smokers and found that the average number of cigarettes smoked per day was lower after six-weeks in participants randomly assigned to cigarettes containing lower amounts of nicotine. Compared with the normal (control) cigarettes, the cigarettes with lower levels of nicotine decreased exposure to and dependence on nicotine and also reduced craving during abstinence from smoking.
Ongoing studies are examining how low-nicotine cigarettes affect smoking behavior in vulnerable populations including women of childbearing age or pregnant women, individuals with comorbid SUDs, and individuals with comorbid serious mental illness. Each of these populations is at increased risk for tobacco use and dependence or tobacco related adverse health outcomes. Yet despite these serious vulnerabilities, these populations have not typically been included in tobacco regulatory studies.
Drug use and addiction are complex conditions. The FY 2017 budget request will allow NIDA to support cutting-edge research that leverages the most powerful technologies and latest emerging opportunities to expand our understanding of drug use and addiction to advance prevention and treatment and improve public health.