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2016-2020 NIDA Strategic Plan

Objective 2.4: Develop and test novel strategies for preventing prescription opioid misuse and addiction

An estimated 100 million U.S. adults suffer from chronic pain.85 Over the last 16 years, there was a threefold increase in opioid prescribing, which was associated with a dramatic rise in opioid use disorders, overdose deaths, and cases of neonatal abstinence syndrome.86,87 This effect was due in part to the limited options for effectively treating chronic pain. As summarized in a recent report from the NIH Pain Consortium, there is a pressing need for more research on the effectiveness and safety of using opioids to treat chronic pain as well as on optimal management and risk mitigation strategies.88

There are some patients who benefit from opioid medications, especially in the context of a broader pain treatment program. However, many other chronic pain patients are inappropriately prescribed opioid medications that may be ineffective or even harmful.88 More research is needed to develop strategies to identify the patients for whom opioids are the most appropriate treatment, to develop new opioid drug formulations with reduced potential for abuse, and to develop more effective non-opioid pain treatment strategies. The U.S. Department of Health and Human Services is in the process of developing a National Pain Strategy89 that outlines priorities in population-level research on pain and recommends specific steps to a) increase the precision of information about chronic pain prevalence overall, for specific types of pain, and in specific population groups; b) develop the capacity to gather information electronically about pain treatments and their usage, costs, effectiveness, and safety; and c) enable tracking changes in pain prevalence, impact, and treatment over time, allowing evaluation of population-level interventions and identification of emerging needs.


  • Develop and test non-opioid medication targets for chronic pain
  • Develop and test adjunctive therapies that can reduce the dose of opioids to control pain
  • Develop and test nonpharmacological pain treatment strategies such as neural stimulation therapies (e.g., transcranial magnetic stimulation, electrical deep brain stimulation), biofeedback, and behavioral treatments
  • Identify and test strategies for improving opioid prescribing and patient management practices to reduce the development of opioid use disorders

Precision Medicine for Pain

Each year, about 100 million American adults experience chronic pain, costing the nation between $560 billion and $635 billion.85 Opioid pain relievers are among the most effective medications for the management of severe pain and are often used to treat chronic pain. However, the benefits of long-term opioid treatment are increasingly being questioned as patients face a significant risk of developing drug tolerance (needing higher doses) and hyperalgesia (increased pain sensitivity). Also, use of these medications can lead to dependence or even addiction, and they may trigger relapse in people who are recovering from SUDs. Over the past two decades, a steep rise in misuse of and addiction to opioid pain relievers has led to a dramatic increase in opioid use disorders as well as an epidemic of overdose deaths. As people with addictions to opioids seek cheaper alternatives, it has also led to a rise in heroin use and related deaths.

NIDA plays a central role in supporting research efforts aimed at developing safe and effective options for managing chronic pain while minimizing the risk of abuse. NIDA pursues this objective through research and development of non-opioid pain medications, abuse-deterrent formulations of existing medications, and user-friendly overdose reversal drug formulations (e.g., intranasal naloxone). NIDA is also committed to educating health care providers and the broader public about the proper use and potential risks of these medications.

NIDA supports a pharmacogenomics program that is developing ways to identify which pain management interventions are most effective for specific chronic pain patients and how to predict which patients might be at higher risk for opioid use disorders. Research has identified a number of genetic markers associated with risk for opioid addiction as well as pain sensitivity, including the µ opioid receptor-1 and cytochrome P450 2D6.90 The ultimate goal of this research is to match pain treatment to a person’s unique DNA profile and prescribe opioid pain relievers only to those who will benefit from them.

These efforts reflect a growing understanding at NIDA and across NIH that approaching health care in a patient-centered way, known as precision medicine, promises to spur significant, potentially transformative advances in clinical care and research.