FDA continues push for new opioid use disorder treatments
The Food and Drug Administration on Monday issued new scientific recommendations that highlight new ways for drug developers to consider measuring and demonstrating the effectiveness and benefits of new or existing medication-assisted treatment, or MAT, products for the treatment of opioid use disorder, or OUD.
“The Trump Administration is pursuing every opportunity to address our country’s opioid epidemic and support patients struggling with opioid use disorder. This work at Health and Human Services includes placing a special priority on ensuring access to a full range of safe and effective options for medication-assisted treatment,” said HHS secretary Alex Azar, in a press statement. “The evidence is clear: medication-assisted treatment works, and it is a key piece of defeating the drug crisis facing our country. The FDA’s new guidances have the potential to bring new medications to market that are more closely tailored to patient needs and help give Americans facing addiction a better chance at recovery.”
MAT for opioid dependence relies on prescription drugs, including buprenorphine, methadone and naltrexone, to stabilize brain chemistry; reduce or block the euphoric effects of opioids; relieve physiological cravings; and normalize body functions. Regular adherence to MAT helps patients gain control over their use of opioids, without causing the cycle of highs and lows associated with opioid misuse or abuse. MAT, coupled with relevant social, medical and psychological services, is a highly effective treatment for OUD.Patients receiving MAT cut their risk of death from all causes in half, according to the Substance Abuse and Mental Health Services Administration.
“As we seek to help those with an opioid use disorder transition to lives of sobriety, we recognize there’s great interest in new treatment options that result in meaningful outcomes for patients. For example, we must consider new ways to gauge success beyond simply whether a patient in recovery has stopped using opioids, such as reducing relapse overdoses and infectious disease transmission,” FDA commissioner Scott Gottlieb said, in a statement.
“Treatments that can impact these aspects of addiction can be important parts of a comprehensive approach to the treatment of opioid use disorder. This new guidance is an important step in fostering the development of new treatment options that help patients achieve these and other real-world outcomes, by providing a pathway for how innovators can use these clinically relevant measures as part of new drug development programs," Gottlied added.
Clinical trials to evaluate the effectiveness of MAT for the purposes of FDA approval have generally used reduction in drug-taking behavior (drug use patterns) as an endpoint. The new draft guidance, “Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for Medication-Assisted Treatment,” identifies several additional potential clinical endpoints and other outcome measures that drug developers may consider.
The FDA encourages drug sponsors to consider the following ways to evaluate the effect and clinical benefit of MAT: the impact of a new drug on adverse outcomes like mortality (overall mortality or overdose mortality); emergency medical interventions and Hepatitis C seroconversion (the period during which antibodies develop and become detectable); studying the proportion of patients that transition from meeting criteria for being diagnosed with moderate to severe OUD — based on both drug use and its impact on patient wellbeing – at baseline to being considered in remission at the end of the study; and improvements in the ability to resume work, school, or other productive activity.
“The Trump Administration is pursuing every opportunity to address our country’s opioid epidemic and support patients struggling with opioid use disorder. This work at Health and Human Services includes placing a special priority on ensuring access to a full range of safe and effective options for medication-assisted treatment,” said HHS secretary Alex Azar, in a press statement. “The evidence is clear: medication-assisted treatment works, and it is a key piece of defeating the drug crisis facing our country. The FDA’s new guidances have the potential to bring new medications to market that are more closely tailored to patient needs and help give Americans facing addiction a better chance at recovery.”
MAT for opioid dependence relies on prescription drugs, including buprenorphine, methadone and naltrexone, to stabilize brain chemistry; reduce or block the euphoric effects of opioids; relieve physiological cravings; and normalize body functions. Regular adherence to MAT helps patients gain control over their use of opioids, without causing the cycle of highs and lows associated with opioid misuse or abuse. MAT, coupled with relevant social, medical and psychological services, is a highly effective treatment for OUD.Patients receiving MAT cut their risk of death from all causes in half, according to the Substance Abuse and Mental Health Services Administration.
“As we seek to help those with an opioid use disorder transition to lives of sobriety, we recognize there’s great interest in new treatment options that result in meaningful outcomes for patients. For example, we must consider new ways to gauge success beyond simply whether a patient in recovery has stopped using opioids, such as reducing relapse overdoses and infectious disease transmission,” FDA commissioner Scott Gottlieb said, in a statement.
“Treatments that can impact these aspects of addiction can be important parts of a comprehensive approach to the treatment of opioid use disorder. This new guidance is an important step in fostering the development of new treatment options that help patients achieve these and other real-world outcomes, by providing a pathway for how innovators can use these clinically relevant measures as part of new drug development programs," Gottlied added.
Clinical trials to evaluate the effectiveness of MAT for the purposes of FDA approval have generally used reduction in drug-taking behavior (drug use patterns) as an endpoint. The new draft guidance, “Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for Medication-Assisted Treatment,” identifies several additional potential clinical endpoints and other outcome measures that drug developers may consider.
The FDA encourages drug sponsors to consider the following ways to evaluate the effect and clinical benefit of MAT: the impact of a new drug on adverse outcomes like mortality (overall mortality or overdose mortality); emergency medical interventions and Hepatitis C seroconversion (the period during which antibodies develop and become detectable); studying the proportion of patients that transition from meeting criteria for being diagnosed with moderate to severe OUD — based on both drug use and its impact on patient wellbeing – at baseline to being considered in remission at the end of the study; and improvements in the ability to resume work, school, or other productive activity.