Improve Treatments for Opioid Misuse Disorder and Addiction

More than 2 million Americans have opioid use disorders. Millions more misuse opioids, taking opioid medications longer or in higher doses than prescribed. Through the NIH HEAL (Helping to End Addiction Long-term) Initiative, NIH will support research to prevent and treat opioid misuse and addiction to help people with substance use disorders achieve and maintain a meaningful and sustained recovery.

Precision Prevention

Problem: Doctors cannot predict which patients will misuse opioid medications. Improved understanding of the transition from opioid use as prescribed, to misuse, to addiction could enable prevention strategies tailored to individual needs.

Approach: Advance the scientific understanding of addiction:

  • Enhance understanding of vulnerability to addiction through the analysis of longitudinal data of patients prescribed opioid medication.
  • Comprehensively study genomic variation that is predisposing to opioid use disorder (OUD) to be able to predict which individuals are most likely to develop OUD.  

New Treatments for Addiction

Problem: Despite multiple FDA-approved therapies for OUD, adherence to therapy is low, and more than 50% of patients receiving Medication Assisted Treatment (MAT) relapse within six months.

Approach: Develop flexible and complementary treatment options for opioid use disorder to reduce drug use, prevent overdoses and support recovery:

  • Expand treatment options for MAT through the development of new formulations and combinations of effective treatments.
  • Develop novel immunotherapies (vaccines and/or monoclonal antibodies) using comprehensive knowledge of the immune system, to neutralize opioids and prevent relapse when provided to patients in combination with MAT.
  • Develop additional endpoints for studies of treatment for opioid addiction, taking into account the patient experience and needs for long-term recovery.

Overdose Prevention and Reversal

Problem: More than 42,000 Americans died from opioid overdose in 2016, and this number is increasing with the rise of powerful synthetic opioids. Effective overdose reversal agents are available, but multiple doses can be required to reverse overdose from synthetic opioids. In addition, most overdoses occur when individuals are alone and unobserved by family members and first responders.

Approach: Develop new agents to prevent and reverse opioid overdose that are capable of reversing overdose from highly potent synthetic opioids such as fentanyl and carfentanil:

  • Enhance overdose reversal agents to save lives in the face of increasingly widespread illicit use of synthetic opioids.
  • Develop devices to automatically reverse overdose in high-risk patients.

Enhance Treatments for Mothers and Infants

Problem: Approximately one third of reproductive-aged women fill a prescription for an opioid medication each year, and many pregnant women receive opioid medications. By 2012, nearly 22,000 infants were born with Neonatal Abstinence Syndrome/Neonatal Opioid Withdrawal Syndrome (NOWs) yearly, at a cost of over $1.5 billion in hospital charges nationwide. The best treatment approach for these infants is not known.

Approach: Enhance treatments for pregnant women with an OUD and for newborns with NOWs:

  • Evaluate optimal management strategies for pregnant women with an OUD.
  • Expand the Advancing Clinical Trials in Neonatal Opioid Withdrawl Syndrome (ACT NOW) project and tap into the Environmental Child Health Outcomes and IDeA States Pediatric Clinical Trials Network to learn how to best to treat and manage opioid withdrawal in newborns, and determine long-term consequences on development, and possible management strategies.

Optimize Effective Treatments

Problem: Americans with addiction need help right now and cannot wait for new treatments. More than 2 million Americans have an opioid use disorder, but less than 20% receive appropriate treatment for their disorder.

Approach: Develop and test additional MAT options. Test strategies for translating research into practice, and advance implementation of evidence-based treatments in real world settings:

  • Develop additional options for treatment of addiction, in collaboration with the private sector and federal and state partners.
  • Assess the additive role of social and behavioral interventions to MAT programs.
  • Test the implementation of strategies to treat opioid use disorder through adaptive trials in various community settings.
  • Employ pilot studies for adoption of best practices across multiple healthcare settings, such as primary care practitioners, emergency rooms, state health departments, treatment programs, and the criminal justice system.
  • Launch major demonstration projects in communities highly affected by the opioid crisis.

This page last reviewed on April 5, 2018