Opioid Use Disorders: HHS Needs Measures to Assess the Effectiveness of Efforts to Expand Access to Medication-Assisted Treatment

The misuse of prescription opioid pain relievers and illicit opioids, such as heroin, has contributed to increases in overdose deaths. According to the most recent Centers for Disease Control and Prevention data, in 2015 over 52,000 people died of drug overdose deaths, and about 63 percent of them involved an opioid. For those who are addicted to or misuse opioids, MAT has been shown to be an effective treatment.

GAO was asked to review HHS and other efforts related to MAT for opioid use disorders. This report (1) describes HHS's key efforts to expand access to MAT, (2) examines HHS's evaluation, if any, of its efforts to expand access to MAT, and (3) describes efforts by selected stakeholders (states, private health insurers, and national associations) to expand access to MAT. GAO gathered information from HHS officials as well as a non-generalizable selection of 15 stakeholders selected based on their MAT expansion activities, among other factors. GAO also assessed HHS's evaluation plans using internal control standards for defining objectives and evaluating results.

In an effort to reduce the prevalence of opioid misuse and the fatalities associated with it, the Department of Health and Human Services (HHS) established a goal to expand access to medication-assisted treatment (MAT). MAT is an approach that combines behavioral therapy and the use of certain medications, such as methadone and buprenorphine. HHS has implemented five key efforts since 2015 that focus on expanding access to MAT for opioid use disorders—four grant programs that focus on expanding access to MAT in various settings (including rural primary care practices and health centers) and regulatory changes that expand treatment capacity by increasing patient limits for buprenorphine prescribers and allowing nurse practitioners and physician assistants to prescribe buprenorphine.

Some of the grant awards were made in 2015, while others were made as recently as May 2017. (See figure.) As of August 2017, efforts under all the grant programs were ongoing. Grant recipients can use funding to undertake a range of activities, such as hiring and training providers and supporting treatments involving MAT. In addition, certain providers and grant recipients are required to develop plans for preventing MAT medications from being diverted for nonmedical purposes.

HHS officials told GAO that as of August 2017, the department was in the process of finalizing its plans to evaluate its efforts to address the opioid epidemic. In September 2016, HHS awarded a contract to conduct the evaluation. HHS officials told GAO that they are still working with the contractor to finalize the evaluation approach and that it will focus on whether HHS's efforts to address the opioid epidemic have been implemented as intended. HHS officials said that in the future, HHS may also evaluate whether, or to what extent, its efforts have been effective in expanding access to MAT, in addition to evaluating implementation.

While HHS has some of the information that could be used in a future evaluation of the effectiveness of its efforts to expand access to MAT, it has not adopted specific performance measures with targets specifying the magnitude of the increases HHS hopes to achieve through its efforts to expand access to MAT, and by when. For example, HHS has not established a long-term target specifying the percentage increase in the number of prescriptions for buprenorphine HHS would like to achieve, which would help to show whether efforts by HHS and others are resulting in a sufficient number of prescriptions for MAT medications. HHS has also not chosen a specific method of measuring treatment capacity or established targets associated with it, which would help determine whether a sufficient number of providers are becoming available to evaluate and treat patients who may benefit from MAT.

Without specifying these performance measures and associated targets, HHS will not have an effective means to determine whether its efforts are helping to expand access to MAT or whether new approaches are needed. Gauging this progress is particularly important given the large gap identified nationwide between the total number of individuals who could benefit from MAT and the limited number who can currently access it based on provider availability.

In addition, GAO also found that as of August 2017, HHS had not finalized its approach for its planned evaluation activities, including timeframes. Without timeframes for the evaluation's activities, HHS increases the risk that the evaluation will not be completed as expeditiously as possible.

In addition to HHS efforts to expand access to MAT, officials from selected states, private health insurers, and national associations reported using several efforts to expand patients' access to MAT for opioid use disorders. For example, several stakeholders provided GAO with the following examples of their efforts:

States. State health officials from all five selected states have implemented or are planning approaches that focus on integrating the use of MAT into primary care, such as by providing services for centralized intake and initial management of patients or through telehealth that connects patients in rural areas with addiction specialists in a different location.

Private health insurers. Three private health insurers reported removing prior authorization requirements for MAT medications so patients can avoid a waiting period before receiving the medications.

National associations. Officials told GAO that they are conducting outreach and training for their members and developing tools and resource guides. For example, one association developed a road map with strategies that state policymakers can use to address the opioid epidemic, including strategies for reducing the stigma associated with MAT through educating the public and potential providers.

GAO recommends that HHS take two actions: (1) establish performance measures with targets related to expanding access to MAT, and (2) establish timeframes for its evaluation of its efforts to expand access to MAT. HHS concurred with both recommendations.

The misuse of and addiction to prescription opioid pain relievers and illicit  opioids, such as heroin, contributes to significant morbidity and mortality  in the United States. Substance Abuse and Mental Health Services  Administration (SAMHSA) data show that in 2014 almost 2.3 million  people aged 12 years and older misused or were dependent on opioids,  up from almost 1.7 million in 2005. According to the most recent Centers  for Disease Control and Prevention data, in 2015 there were over 52,000  drug overdose deaths in the United States, and about 63 percent of these  deaths involved an opioid.

For those who misuse or are addicted to opioids—a condition known as  opioid use disorder—research shows that medication-assisted treatment  (MAT) is an effective treatment. MAT—which combines behavioral  therapy and the use of certain medications (methadone, buprenorphine,  and naltrexone)—has been shown to reduce opioid use and to increase  treatment retention compared with other treatments. Treatment can  occur as part of federally regulated opioid treatment programs (OTP) or in  other settings, such as office-based settings, within certain restrictions.

While MAT has been shown to be effective, several reports have also  highlighted that accessing MAT services may be challenging for many.  For example, in a 2016 report we identified several factors that affect  access to MAT, including lack of insurance coverage and an insufficient  number of existing treatment programs or practicing physicians offering  MAT. Similarly, a 2016 Surgeon General’s report on Alcohol, Drugs, and  Health noted barriers to accessing treatment, such as the cost of care  and challenges accessing providers who offer treatment services. In part  because of these challenges, it is likely that only a portion of the  individuals who need MAT are receiving it. The 2016 Surgeon General’s  report and a 2015 study on MAT treatment capacity have both highlighted  this “treatment gap.” The 2015 study, for instance, found that the  availability of MAT has not kept pace with the incidence of opioid use  disorders in the United States, as more are in need of the treatment than  can currently access it—a treatment gap estimated to be nearly 1 million  people in 2012.

In March 2015, the Department of Health and Human Services (HHS)  established the HHS Opioid Initiative, which aimed to decrease (1) opioid  overdoses and overall overdose mortality, and (2) the prevalence of  opioid use disorders. Led by the Office of the Assistant Secretary for  Planning and Evaluation (ASPE) in coordination with the Immediate  Office of the Secretary, the HHS Opioid Initiative had three priorities  related to opioid use disorders, one of which was to expand access to  MAT. Although the HHS Opioid Initiative ended in January 2017, HHS  activities related to improving access to treatment for opioid use disorders  have continued under the new administration’s five-point Opioid Strategy,  which was announced in April 2017. Several agencies within HHS, such  as SAMHSA and the Health Resources and Services Administration  (HRSA), are overseeing various efforts to expand access to MAT, with  ASPE responsible for leading efforts to collectively evaluate the results of  these efforts.

You requested that we provide information on HHS and other efforts to  expand access to MAT for opioid use disorders. In this report, we  1.  describe HHS’s key efforts to expand access to MAT;  2.  examine HHS’s evaluation, if any, of its key efforts to expand access  3.  describe efforts by selected stakeholders (states, private health  insurers, and national associations) to expand access to MAT.

To describe HHS’s key efforts to expand access to MAT, we reviewed  HHS documents that describe the HHS Opioid Initiative and the activities  associated with it, such as HHS press releases, the ASPE Issue Brief on  the Opioid Initiative, and HHS funding announcements for grants to  expand access to MAT. We also reviewed HHS’s April 2017  announcement of the Opioid Strategy, as well as relevant federal laws  and regulations relating to MAT. In addition, we interviewed officials at  relevant federal agencies, such as ASPE, SAMHSA, HRSA, the Agency  for Healthcare Research and Quality (AHRQ), and the Food and Drug  Administration (FDA) with knowledge of MAT expansion efforts within  HHS. We specifically asked the officials to identify their key efforts to  expand access to MAT and to confirm the key efforts that we identified  independently through our document reviews. Our review focused on  those efforts implemented from fiscal year 2015 through August 2017. We  also inquired about the extent to which diversion control plans— procedures that reduce the possibility that controlled substances will be  transferred or used illicitly—are included in HHS’s efforts to expand  access to MAT, and we reviewed relevant documents.

To examine any HHS evaluation of its key efforts to expand access to  MAT, we reviewed relevant documentation that describes HHS’s  evaluation plans to assess MAT expansion efforts. These documents  included a 2016 report by Mathematica Policy Research that describes a  proposed evaluation approach developed under contract with HHS and  the solicitation for a contractor to conduct the evaluation. We assessed  these plans using criteria for defining objectives and evaluating results in  federal internal control standards. We also interviewed ASPE officials  about their evaluation approach and the status of the evaluation.

To describe efforts by selected stakeholders to expand access to MAT,  we first identified relevant stakeholder organizations that represent states,  private health insurance plans, and national associations. Through our  interviews with HHS officials, background interviews with officials from  organizations, such as the America’s Health Insurance Plans and Blue  Cross Blue Shield Association, and background research, we identified a  non-generalizable selection of 15 stakeholders in the following groups— states, private health insurance plans, and national associations—to  obtain information about their efforts to expand access to MAT.  Specifically, we gathered information from officials at (1) state health  departments and behavioral health services agencies in five states:  Rhode Island, Maryland, Washington, West Virginia, and Indiana; (2)  seven private health insurance plans; and (3) three national associations  that represent providers, government officials, and public health officials:  the American Society of Addiction Medicine, the National Governors  Association, and the Association of State and Territorial Health Officials.  In addition to identifying and selecting stakeholders with efforts to expand  access to MAT for opioid use disorder based on background interviews  and research, we considered (1) geographic diversity and Centers for  Disease Control and Prevention data on states with high opioid death  rates when selecting the five states; and (2) health insurers with large  market share when selecting the seven private health insurers. We also  asked several stakeholders about their views on the diversion of MAT  medications as part of their efforts to expand access to MAT, and we  reviewed relevant documents.

We conducted this performance audit from October 2016 to October 2017  in accordance with generally accepted government auditing standards.  Those standards require that we plan and perform the audit to obtain  sufficient, appropriate evidence to provide a reasonable basis for our  findings and conclusions based on our audit objectives. We believe that  the evidence obtained provides a reasonable basis for our findings and  conclusions based on our audit objectives.

Health care practitioners prescribe opioid medications to treat pain and  sometimes for other health problems, such as severe coughing. Opioid  medications are available as immediate or extended release and in  different forms, such as a pill, liquid, or a patch worn on the skin. Opioids  slow down some processes of the body, such as breathing and heartbeat,  by binding with certain receptors in the body.

Over time, the body becomes tolerant to opioids, which means that larger  doses of opioid medications are needed to achieve the same effect.  People may use opioids in a manner other than as prescribed—that is,  they can be misused. Because opioids are highly addictive substances,  they can pose serious risks when they are misused, which can lead to  addiction and death. Symptoms of an opioid use disorder include a strong  desire for opioids, the inability to control or reduce use, and continued use  despite interference with major obligations or social functioning. Another  concern associated with prescribed opioids is the potential for diversion  for illegal purposes, such as nonmedical use or financial gain.

Research has shown that MAT—which combines behavioral therapy and  the use of certain medications (methadone, buprenorphine, and  naltrexone)—can be more effective in reducing opioid use and increasing  retention (i.e., reducing dropouts) compared to abstinence based  treatment—that is when patients are treated without medication. Three  medications are currently approved by FDA for use in MAT for opioid use  disorders—methadone, buprenorphine, and naltrexone.

Methadone: Methadone is a full opioid agonist, meaning it binds to  and activates opioid receptors to help prevent withdrawal symptoms  and reduce drug cravings. It has a long history of use for the  treatment of opioid dependence in adults. Methadone suppresses  withdrawal symptoms during detoxification therapy, which involves  stabilizing patients who are addicted to opioids by withdrawing them in  a controlled manner. Methadone also controls the craving for opioids  during maintenance therapy, which is ongoing therapy meant to  prevent relapse and increase treatment retention. Methadone can be  administered to patients as an oral solution or in tablet form.

Buprenorphine: Buprenorphine is a partial opioid agonist, meaning it  binds to opioid receptors and activates them, but not to the same  degree as full opioid agonists. It reduces or eliminates opioid  withdrawal symptoms, including drug cravings. It can be used for  detoxification treatment and maintenance therapy. It is available for  MAT for opioid use disorder in tablet form for sublingual (under the  tongue) administration, in film form for sublingual or buccal (inside the  cheek) administration, and as a subdermal (under the skin) implant.

Naltrexone: Naltrexone is an opioid antagonist, meaning it binds to  opioid receptors but does not activate them. It is used for relapse  prevention following complete detoxification from opioids. Naltrexone  prevents opioid drugs from binding to and activating opioid receptors,  thus blocking the euphoria the user would normally feel. It also results  in withdrawal symptoms if recent opioid use has occurred. It can be  taken daily in an oral tablet form or as a once-monthly injection given  in a doctor’s office.

Two of the three medications used to treat opioid use disorders— methadone and buprenorphine—are drugs that carry a potential for  misuse. Under the Controlled Substances Act (CSA), treatment involving  these medications can take place in certain authorized settings: as part of  federally regulated OTPs or in other settings, such as a physician’s office,  within certain restrictions.

OTPs. OTPs provide MAT, including methadone and buprenorphine,  for people diagnosed with an opioid use disorder. Methadone may  generally only be administered or dispensed within an OTP, as  prescriptions for methadone cannot be issued when used for opioid  use disorder treatment. Buprenorphine may be administered or  dispensed within an OTP, or may also be prescribed by a qualifying  practitioner who has received a waiver from SAMHSA. Naltrexone is  not a controlled substance and can be used in OTPs and other  settings.

Office-Based and Other Settings. Under a Drug Addiction Treatment  Act of 2000 (DATA 2000) waiver, practitioners may prescribe  buprenorphine to up to 30 patients in the first year of their waiver, 100  patients in the second year, and up to 275 patients in the third year.

Practitioners at the 275-patient level must meet additional  qualifications and requirements. Naltrexone does not have similar  restrictions.

HHS Uses Grant Programs and Other Efforts to Expand Access to MAT for Opioid Use Disorders

HHS has implemented five key efforts from 2015 through August 2017  that focus on expanding access to MAT for opioid use disorders. Four of  these are grant programs, including programs focused on health centers  or primary care practices in rural areas.

Targeted Capacity Expansion: Medication Assisted Treatment –  Prescription Drug and Opioid Addiction (MAT-PDOA). This grant  program is administered by SAMHSA and provides funding to states  to increase their capacity to provide MAT and recovery support  services to individuals with opioid use disorders. Grant recipients  are expected to identify a minimum of two high-risk communities  within the state and partner with local government or community- based organizations to address the MAT-related treatment needs in  these communities. Among other things, recipients are to use  outreach and other engagement activities to increase participation in  and access to MAT for diverse populations at risk for opioid use  disorders. In August 2015, SAMHSA awarded 3-year grants to 11  states, under which each of the states will receive up to $1 million in  each grant year. In September 2016, SAMHSA awarded 11 additional  3-year grants to other states. Total funding is expected to be up to  $66 million for all 22 grants. SAMHSA announced the availability of up  to 5 additional 3-year grants for fiscal year 2017. Applications for  these grants of up to $2 million per year were due in July 2017 and as  of August 2017 they had not been awarded.

Substance Abuse Service Expansion Supplement to Health  Centers. This grant program is administered by HRSA and provides  funds for existing health centers to improve and expand their delivery  of substance abuse services, including services with a specific focus  on MAT for opioid use disorders in underserved populations. Health  centers that receive these grants are required to increase the number  of patients with health center-funded access to MAT for opioid use or  for other substance abuse disorders treatment by adding at least one  full-time substance abuse provider and supporting new or enhanced  existing substance abuse services. HRSA awarded 2-year grants in  March 2016 to 271 health centers. According to HRSA documents,  total funding could be up to $200 million for all grants over 2 years.  HRSA announced the availability of another set of grants to health  centers for fiscal year 2017. Applications for these grants were due in  July 2017, and as of August 2017 they had not been awarded.

Increasing Access to Medication-Assisted Treatment in Rural  Primary Care Practices. This grant program is administered by  AHRQ and funds demonstration research projects that aim to expand  access to MAT for opioid use disorders in primary care practices in  rural areas of the United States. Grant recipients are expected to  recruit and engage primary care providers and their practices, provide  training, and support physicians and their practices in initiating  treatment. The program also identifies and tests strategies for  overcoming the challenges associated with implementing MAT in  primary care settings and creates training and other resources for  implementing MAT. AHRQ awarded these 3-year grants of up to $1  million per year to four recipients—the recipients are teams of state  health departments, academic health centers, local community  organizations, physicians, and others—with project start dates of  September 30, 2016. According to AHRQ documents, total funding is  expected to be up to $12 million for the four grants over 3 years.

State Targeted Response to the Opioid Crisis Grants (Opioid  STR). This grant program is administered by SAMHSA and provides  funding to states and others to increase access to treatment services  for opioid use disorders, including MAT; reduce unmet treatment  needs; and reduce opioid overdose deaths. Grant recipients are  expected to implement or expand access to evidence-based  practices, particularly the use of MAT, and to report on the number of  people who receive opioid use disorder treatment, the number of  providers implementing MAT, and the number of providers trained to  use MAT. SAMHSA awarded 2-year grants starting in May 2017 to 50  states, the District of Columbia, four U.S. territories and the free  associated states of Micronesia and Palau. According to SAMHSA  documents, total funding could be up to $970 million for all grants over  2 years.

Figure 1 displays the implementation timeframes, the number of grants,  and funding levels for the four HHS grant programs related to MAT. As  the figure shows, some of these awards were made in fiscal year 2015,  while others were made as recently as May 2017. As of August 2017,  these efforts were ongoing.

In addition to these four grant programs, HHS’s fifth key effort increases  treatment capacity by expanding the waivers that practitioners may  receive to prescribe buprenorphine. Specifically, SAMHSA issued a  regulation that became effective August 8, 2016 increasing the number of  patients that eligible practitioners can treat with buprenorphine outside of  an OTP (e.g., in an office-based setting). Previously, qualified  practitioners could request approval to treat up to 30 patients at a time,  and after 1 year the limit could increase to 100 patients at a time upon  SAMHSA approval. The new regulation expanded access to MAT by  allowing eligible practitioners who have had waivers to prescribe  buprenorphine to 100 patients for at least 1 year to request approval to  treat up to 275 patients thereafter. Similarly, SAMHSA has implemented  provisions of the Comprehensive Addiction and Recovery Act of 2016  (CARA) that expanded the types of practitioners who can receive a  waiver to prescribe buprenorphine in an office-based setting to include  qualifying nurse practitioners and physician assistants. CARA generally  requires that these nurse practitioners and physician assistants complete  24 hours of training to be eligible for a waiver. According to HHS  documents, as of early 2017, nurse practitioners and physician assistants  who have completed this training could request a waiver from SAMHSA  to treat up to 30 patients at a time.

In addition to its five key efforts focused specifically on expanding access  to MAT for opioid use disorders, HHS has other efforts with broader  focuses, such as treating multiple types of substance abuse. While these  efforts are not specifically focused on expanding access to MAT for opioid  use disorders, they may result in expanded access to MAT. For example,  CMS has approved section 1115 Medicaid demonstration projects to  allow states to undertake comprehensive reforms of their delivery of  substance abuse services, including provisions to enhance the use of  MAT for opioid use disorders. In July 2015, CMS issued a state  Medicaid Director letter informing states that they may seek approval of  section 1115 demonstrations to undertake comprehensive substance use  service reforms. According to CMS, all participating states are using the  demonstration authority to develop a full continuum of care for individuals  with substance abuse disorders, including coverage of short-term  residential treatment services not otherwise covered by Medicaid.

In addition, FDA has programs to help expedite development and to  provide for faster review of marketing applications for certain drugs.  According to FDA, it has conducted expedited reviews of Suboxone  (buprenorphine and naloxone sublingual film), Vivitrol (extended release  naltrexone injection) and Probuphine (buprenorphine subdermal  implant).

According to some federal officials and other stakeholders that we  interviewed, as part of efforts to expand access to MAT for opioid use  disorder, steps are being taken to prevent the possibility that the MAT  medications could, in some cases, be diverted for illicit use, misuse, or for  purposes not intended by a prescriber. For example, OTPs and  practitioners who request and receive a waiver to prescribe  buprenorphine to treat up to 275 patients outside of an OTP setting are  required under federal regulations to maintain a diversion control plan.  In addition, the MAT-PDOA grant program explicitly requires grant  recipients to implement a diversion control plan, though the other grant  programs do not have similar additional requirements. (See appendix I for  an overview of the diversion control plan requirements for OTPs and the  practitioners who prescribe buprenorphine outside of an OTP.) The 2016  Surgeon General’s report on Alcohol, Drugs, and Health noted that  decades of research have shown that the benefits of MAT greatly  outweigh the risks associated with diversion, and that withholding these  medications greatly increases the risk of relapse to illicit opioid use and  overdose death.

HHS is Finalizing Its Approach for Evaluating MAT Expansion Efforts but Lacks Performance Measures with Targets and Implementation Timeframes

HHS officials told us that as of August 2017, the department is in the  process of finalizing its approach for evaluating the implementation of its  agencies’ collective efforts to address the opioid epidemic that were  undertaken as part of the HHS Opioid Initiative and will continue under  the new administration’s Opioid Strategy. HHS officials provided a draft of  the evaluation’s schedule. According to the officials, the evaluation will  include, but not be limited to, efforts to expand access to MAT. In  September 2016, HHS awarded a 2-year contract to Research Triangle  Institute International (RTI) to evaluate HHS agencies’ collective efforts.  HHS officials told us that they are still working with RTI to finalize the  evaluation approach given new leadership priorities. Specifically, in April  2017, the new Secretary of HHS announced a revised strategy for  addressing the opioid epidemic that will continue to address access to  MAT for opioid use disorders but also include additional priority areas.  According to HHS officials, to be responsive to the new priorities, the  evaluation will focus initially on whether HHS’s efforts have been  implemented as intended, and officials expect the evaluation to also  provide information on any challenges HHS has faced in implementing  these efforts.

According to HHS officials, while the evaluation of MAT expansion efforts  will use information from several sources, they have not yet determined  exactly which information will be used or how it will be used. This  information may include, for example, results from a separate, planned  evaluation of one of the grant programs, Opioid STR, as well as other  information HHS agencies collect as part of their ongoing monitoring  efforts for each of their individual MAT grant programs. While the  reporting requirements vary across the four MAT grant programs, the  grantees provide HHS with information related to expanding access to  MAT. Specifically,

Targeted Capacity Expansion: Medication Assisted Treatment –  Prescription Drug and Opioid Addiction (MAT-PDOA): Every 6  months, grant recipients are expected to submit progress reports to  SAMHSA on the planned and actual number of patients treated, as  well as information on other performance measures.

Increasing Access to Medication-Assisted Treatment in Rural  Primary Care Practices: Grant recipients are expected to submit  quarterly progress reports to AHRQ with various information, such as  information on the number of physicians who have been certified to  prescribe buprenorphine and the number of primary care practices  successfully initiating the delivery of MAT services as a result of the  grant project.

Substance Abuse Service Expansion Supplement to Health  Centers: Health centers that received these grants were expected to  submit quarterly progress reports to HRSA through the second  quarter of 2017 on the number of physicians who have obtained a  DATA 2000 waiver and the number of patients who received MAT  from these physicians. Health centers must now report these data  elements in their annual performance reporting along with information  on the number of certified nurse practitioners and physician assistants  who have received a DATA 2000 waiver.

State Targeted Response to the Opioid Crisis Grants (Opioid  STR): Every 6 months, grant recipients are expected to submit  progress reports to SAMHSA on the number of individuals who  receive opioid use disorder treatment, the number who receive opioid  use disorder recovery services, and the number of providers  implementing MAT, among other measures.

While HHS’s evaluation will focus on whether HHS’s efforts have been  implemented as intended, officials told us that in the future an evaluation  may also focus on the effectiveness of these efforts, including the  effectiveness of efforts to expand access to MAT. Doing so would be  consistent with federal standards for internal control, which call for  agencies to evaluate results. HHS has some of the information that  could be used in a future evaluation of the effectiveness of its efforts to  expand access to MAT. In particular, an HHS document describing the  department’s fiscal year 2016 – 2017 goals identifies expanding MAT  access as an important strategy for the success of HHS’s longer-term  goal of reducing opioid use disorders and opioid overdoses. In addition,  HHS has identified three potential ways to measure access to MAT:  the number of prescriptions for MAT medications,  the treatment capacity of practitioners who are authorized to prescribe  buprenorphine for opioid use disorders through a DATA 2000 waiver,  and  the treatment capacity of OTPs certified to administer methadone and  other medications.

In addition, HHS has data that could be useful for tracking progress in  these areas (see table 1).

However, HHS has not adopted specific performance measures with  targets specifying the magnitude of the increases HHS hopes to achieve  through its efforts to expand access to MAT, and by when. For example,  HHS has not established a long-term target specifying the percentage  increase in the number of prescriptions for buprenorphine HHS would like  to achieve, which would help to show whether efforts by HHS and others  are resulting in sufficient progress in increasing prescriptions for this MAT  medication. HHS has also not chosen a specific method of measuring  treatment capacity or established targets associated with it, which would  help to show whether a sufficient number of providers are becoming  available to evaluate and treat patients who may benefit from MAT.

Without specifying these performance measures and associated targets,  HHS will not have an effective means to determine whether its efforts are  helping to expand access to MAT. The lack of such performance  measures with associated targets is inconsistent with federal internal  control standards that specify that management should define objectives  and evaluate results. According to these standards, using performance  information such as performance measures can help agencies monitor  results and determine progress in meeting program goals. In the context  of HHS’s efforts to expand access to MAT, establishing appropriate  performance measures with associated targets would allow HHS to  determine whether its efforts are making sufficient progress or whether  they need to be improved. Gauging this progress is particularly important,  given the large nationwide MAT treatment gap identified in 2015 between  the total number of individuals who could benefit from MAT and the  limited number who can access it based on provider availability. This gap  was estimated at nearly 1 million people as of 2012, and according to  HHS officials and other stakeholders, lack of providers continues to be a  challenge. Until HHS establishes performance measures with  associated targets for the factors related to access to MAT, the  department will be unable to evaluate its progress expanding access to  MAT for opioid use disorders.

In addition, as of August 2017, HHS has not finalized its approach for the  planned evaluation activities, including timeframes. ASPE officials said  that timeframes for a finalized evaluation approach had not been  established because they were still working with RTI to finalize the  evaluation approach given the new leadership priorities. When we spoke  with the officials, they provided us with a draft evaluation schedule that  covered the contract period ending September 2018. As of October 2017,  HHS had not provided a finalized evaluation approach or schedule.  Federal internal controls call for management to establish and operate  monitoring activities and evaluate results. Without an implementation  timeframe for the evaluation’s activities, HHS increases the risk that its  evaluation of its agencies’ efforts will not be completed as expeditiously  as possible, including an evaluation of HHS’s efforts to expand access to  MAT.

Selected Stakeholders Reported Using Outreach, Training, and Other Efforts to Help Expand Access to Medication- Assisted Treatment for Opioid Use Disorders

Officials from selected state health departments and behavioral health  agencies, private health insurers, and national associations reported  using several different efforts to help expand patients’ access to MAT for  opioid use disorders. All of the stakeholders we interviewed reported  conducting outreach efforts to communicate information about the  importance of MAT and how to access it, or providing training to educate  providers on prescribing MAT medications.

Efforts by states. State health officials we spoke to described several  planned or ongoing efforts to expand access to MAT, some of which are  supported by federal funding, including federal grant programs. Officials  from all five selected states told us that they are offering outreach to and  training for providers to help expand access to MAT. For example,  several state officials told us that they are promoting training to (1)  encourage physicians to obtain authorization (DATA 2000 waivers) to  prescribe buprenorphine and (2) encourage physicians with waivers to  treat patients up to their patient limit or to request a higher patient limit.

According to the stakeholders, all five selected states have implemented  or are planning to implement a health care delivery model or approach  that will expand access to MAT. Specifically, these models or  approaches focus on integrating the use of MAT into primary care  settings. For example, health officials from three states described use of a  hub-and-spoke model. This model generally involves centralized intake  and initial management of patients at a “hub” (e.g., an OTP) and then  connecting these patients to community providers at “spokes” (e.g.,  primary care clinics) for ongoing care, with ongoing support provided by  the hub as needed. Additionally, officials from two states described  offering remote MAT-related consultations through telehealth that  connects patients in rural areas with addiction specialists. According to  a 2017 Healthcare Fraud Prevention and Partnership whitepaper,  telehealth expands the reach of the addiction professional workforce and  the existing pool of MAT providers, and it supports remote forms of  behavioral therapy to make trained professionals more accessible to  those in underserved or isolated communities.

Officials from three states described focusing their MAT expansion efforts  in various settings, such as in the criminal justice setting and emergency  room departments. State health officials from four of the five states told us  that programs in their states are using peer specialists (individuals who  have successfully recovered from substance abuse disorders) in  emergency rooms and other settings to engage with addicted patients  and refer them to addiction specialists or behavioral health counselors.  Officials from the selected states said that some of these and other efforts  are funded through federal sources, such as MAT expansion grants  awarded by SAMHSA, or with state funds to the extent they are available.

Efforts by private health insurers. Officials from private health insurers  reported that they are expanding access to MAT through outreach or  training for providers and through the following three efforts:

Eliminating the need for prior authorization to prescribe MAT  medications. Officials from three insurers reported removing prior  authorization requirements for MAT medications, thereby making it  easier for patients to access needed MAT medications more readily,  rather than undergoing a waiting period for approval to receive the  medications. Other private health insurers told us that they continue to  require prior authorization, intended for safety reasons and to reduce  drug misuse, and officials from one insurer told us that they will allow  a patient to access a limited amount of MAT medications for a period  of 24 to 72 hours while making a determination about the appropriate  treatment services for the patient.

Modifying health benefit coverage. Officials from one private health  insurance plan told us that the company is redesigning the benefit  coverage for methadone and has removed member copays. This  effort is intended to make MAT medications more affordable and allow  members who are not able to use buprenorphine to have an  alternative, such as methadone, that is not cost-prohibitive.

Incentivizing providers and health insurance plan members to use  MAT. Officials from four private health insurance plans described  plans to offer incentives to providers or patients to use MAT. For  example, officials from three health plans stated that they are offering  alternative payment models or paying higher rates to providers that  offer MAT, and another private health insurer is offering incentives to  its members who are enrolled in behavioral health programs that  provide access to MAT.

Efforts by national associations. Officials we interviewed from the  national associations—including the American Society of Addiction  Medicine, the National Governors Association, and the Association of  State and Territorial Health Officials—told us that they are helping to  expand access to MAT through outreach and training for their members  and by developing tools and resource guides for their members. An  official from one association told us that it shares federal grant  announcements, including those that are focused on expanding access to  MAT, with its members. Officials from another association said it provides  training to providers on how to appropriately prescribe MAT medications.  In addition, officials from one association told us that they developed an  opioid-related road map that identifies examples of strategies—including  MAT—that state policymakers can use in their ongoing efforts to address  the opioid epidemic. Examples of strategies include reducing the stigma  associated with MAT through educating the public and potential  providers. Another strategy in the road map is changing payment policies  to expand access to MAT services, such as ensuring that Medicaid and  other state health programs adequately cover all MAT medications and  behavioral interventions and encouraging or requiring commercial health  plans to adopt similar policies.

HHS funds grant programs and has taken other steps to expand access  to MAT, which has been shown to be effective in reducing the prevalence  of opioid use disorders and with them, the likelihood of drug overdoses.  HHS’s Opioid Initiative began in 2015, and the grants that support it are  ongoing, so it is likely too early to determine how effective HHS’s efforts  have been in expanding access to MAT and in meeting HHS’s other  priorities related to addressing the opioid epidemic. According to HHS,  access to MAT can be measured in terms of the number of prescriptions  for MAT and by the treatment capacities of OTPs and practitioners who  are authorized to prescribe buprenorphine.

Our review suggests, however, that HHS may not be ready to perform  this evaluation. While HHS told us that it may evaluate the effectiveness  of its efforts in the future, the department has not established  performance measures with targets that would specify the results that  HHS hopes to achieve through its efforts, and by when. Furthermore,  HHS has not established timeframes for the activities that will make up its  planned evaluation of whether HHS’s efforts have been implemented as  intended. Without performance measures with targets and evaluation  timeframes, HHS increases the risk that the evaluation will not be  completed in a timely manner or that HHS will not know whether its MAT- related efforts are successful or whether new approaches are needed.  The evaluation is particularly important, given the hundreds of millions of  dollars HHS has invested in its MAT-related grant programs.

We are making the following two recommendations to HHS.

The Assistant Secretary for Planning and Evaluation should establish  performance measures with targets related to expanding access to MAT  for opioid use disorders. (Recommendation 1)

The Assistant Secretary for Planning and Evaluation should establish  timeframes in its evaluation approach that specify when its evaluation of  efforts to expand access to MAT will be implemented and completed.  (Recommendation 2)

We provided a draft of this report to HHS for review, and HHS provided  written comments, which are reprinted in appendix II. HHS also provided  technical comments, which we incorporated as appropriate. In its written  comments, HHS concurred with both of our recommendations.  Specifically, for our first recommendation to establish performance  measures with targets related to expanding access to MAT for opioid use  disorders, HHS stated that developing such measures is appropriate and  that the department will continue to work to develop robust performance  measures, including measures related to MAT, as part of its overall  Opioid Strategy, which includes the department’s most recent efforts to  address the opioid epidemic. For our second recommendation to  establish timeframes in its evaluation approach that specify when its  evaluation of efforts to expand access to MAT will be implemented and  completed, HHS agreed that timeframes are important to any evaluation.  HHS noted that its evaluation is being conducted under a 2-year contract  that is scheduled to end in September 2018. HHS has also provided us  with a draft evaluation schedule. We clarified in our report, however, that  HHS has not yet provided a finalized approach for the planned evaluation  or a finalized schedule establishing timeframes for the activities that will  make up the evaluation. Until it finalizes its evaluation approach and  establishes related timeframes, HHS increases the risk that it will not  complete its planned evaluation by September 2018.

We are sending copies of this report to the HHS, and appropriate  congressional committees. In addition, the report will be available at no  charge on the GAO website at http://www.gao.gov.

If you or your staff have questions about this report, please contact me at  (202) 512-7114 or curdae@gao.gov. Contact points for our Offices of  Congressional Relations and Public Affairs may be found on the last page  of this report. Major contributors to this report are listed in appendix III.

Appendix I: Diversion Plan Requirements for Opioid Treatment Programs and Practitioners Who Prescribe Buprenorphine

According to the Department of Health and Human Services, a diversion  control plan is a set of documented procedures intended to reduce the  possibility that controlled substances will be transferred or used illicitly.  Opioid treatment programs (OTPs) and practitioners who prescribe  buprenorphine at the highest patient level through a Drug Addiction  Treatment Act of 2000 (DATA 2000) waiver are required to have these  plans. OTPs are programs that may administer or dispense medication- assisted treatment (MAT) for people diagnosed with an opioid use  disorder, including the use of methadone and buprenorphine. In addition,  under a DATA 2000 waiver, practitioners may prescribe buprenorphine  for patients, up to a 30-, 100-, or 275 patient limit.

Diversion Control Plan Requirement for OTPs

An OTP must maintain a current diversion control plan that contains  specific measures to reduce the possibility of diversion of controlled  substances from legitimate treatment use. Per federal guidelines, the  goal of the diversion control plan is to reduce the scope and significance  of diversion and its impact on communities. The guidelines state that  each OTP’s diversion control plan should make every effort to balance  diversion control against the therapeutic needs of the individual patient.  They also state that diversion control plans should address at least four  general areas of concern: program environment, dosing and take-home  medication, prevention of multiple program enrollment, and prescription  medication misuse. The guidelines include details about each of these  areas:

Program environment: Diversion in the program environment can be  deterred and detected by regular surveillance and the monitoring of  areas in and around the program, where opportunities for diversion  may exist. A visible human presence at a program’s location gives  community members the opportunity to approach staff with concerns  and communicates the program’s commitment to assuring a safe  environment and a positive impact on the surrounding community.

Dosing and take-home medication: In the area of dosing and take- home medication, diversion control encompasses careful control of  inventory, attentive patient dosing, and close supervision of take- home medication. Observing a patient take his or her dose and having  each of them drink and speak after dosing are fundamental  components of diversion control. Take-home dosing should be  provided with careful attention to regulatory compliance and the  therapeutic benefit and safety these regulations are meant to  promote.

Prevention of multiple program enrollment: Reasonable measures  should be taken to prevent patients from enrolling in treatment  provided by more than one clinic or individual practitioner. An OTP,  after obtaining patient consent, may contact other OTPs within a  reasonable geographic distance (100 miles) to verify that a patient is  not enrolled in another OTP.

Misuse of prescription medication: The misuse of prescription  medication has become an area of great concern nationally and  impacts diversion control planning at OTPs. All OTP physicians and  other healthcare providers, as permitted, should register to use their  respective state’s prescription drug monitoring program (PDMP) and  query it for each newly admitted patient prior to initiating dosing. The  PDMP should be checked periodically (for example, quarterly) through  the course of each individual’s treatment and, in particular, before  ordering take-home doses as well as at other important clinical  decision points.

Diversion Control and Plans for Practitioners Prescribing Buprenorphine Outside of an OTP

SAMHSA’s best-practice guidelines for using buprenorphine for treating  opioid use disorders include multiple references to diversion, including  monitoring for diversion, storage of this medication to minimize diversion,  and use of formulations that may be less likely to be diverted. Specifically,  the best practices state that, when possible, practitioners should use the  combination buprenorphine/naloxone product, which increases safety and  decreases the likelihood of diversion and misuse. Further, physicians  who request and receive a waiver to prescribe buprenorphine to treat up  to 275 patients outside of an OTP are required to have a diversion control  plan. According to an HHS official, as of July 13, 2017, roughly 3,330 of  the over 39,000 practitioners with a waiver had a 275-patient limit waiver.

The majority of these practitioners, just over 27,000, have a 30-patient  limit.

According to SAMHSA guidance, the diversion plan should contain  specific measures to reduce the possibility of diversion of buprenorphine  from legitimate treatment use and should assign specific responsibilities  of the medical and administrative staff of the practice setting for carrying  out these measures. Further, the guidance states that the plan should  address how:  the environment at the practice setting can prevent onsite diversion;  to prevent diversion with regard to dosing and take-home medication;  and  to prevent patients from receiving a prescription from more than one  practitioner and later diverting some of the prescribed medication.

Appendix II: Comments from the Department of Health and Human Services

Appendix III: GAO Contact and Staff Acknowledgments

Elizabeth H. Curda, Director, (202) 512-7114 or curdae@gao.gov.

In addition to the contact name above, Will Simerl, Assistant Director;  Natalie Herzog, Analyst-in-Charge; La Sherri Bush; and Emily Wilson  made key contributions to this report. Also contributing were Muriel  Brown, Krister Friday, Sandra George, and Christina Ritchie.

Over 52,000 people died of drug overdoses in 2015—and 63% of these involved opioids. For those who are addicted to or misuse opioids, medication-assisted treatment (MAT)—behavioral therapy combined with medication (such as buprenorphine)—can help. However, many people who need it don't have access to it.

The Department of Health and Human Services implemented a number of efforts to improve access to MAT for opioids, such as grant programs for rural primary care settings and allowing additional providers to prescribe buprenorphine. We recommended that HHS 1) set targets to measure improvements in access and 2) establish evaluation timeframes.