Updated FDA Guidelines for Drug Development for Opioid Use Disorders


With the rise in opioid use disorders due to the COVID pandemic, the agency is re-examining drug development goals.

COVID-19 has received all the attention this year, but, sadly, that doesn’t mean other health crises are gone, including in the area of ​​pain management. Some are getting worse. Social disruption, increased stress from the pandemic and resulting economic crisis, and difficulties accessing routine medical care have increased the incidence of opioid use disorders (OUD) in many parts of the United States.1.2

In an editorial titled “An Epidemic in the Midst of a Pandemic: Opioid Use Disorder and COVID-19”, Alexander et al. disorders, which – already vulnerable and marginalized – rely heavily on face-to-face health care delivery.2 In addition to the need for access to care, the need for better drugs to treat TOU is becoming increasingly urgent.

To close the gap, the FDA announced last fall a final orientation for the development of drugs to treat TOU. “As we fight the COVID-19 crisis, we are also focusing on the other health crises facing our country, including the current crisis of substance use and overdose disorders that our country is battling. … One way to do this is to explore new ways to measure the effectiveness of treatments as we seek to help people with opioid use disorders, said FDA Commissioner Stephen M Hahn, MD, when announcing the updated guidelines.3

The final guide is only slightly modified from the draft version and builds on a separate but related final guide for industry outlining the test design related to the deposit study buprenorphine products. The Buprenorphine Product Directive was published in February 2019.

Test design recommendations around the OUD

The final guidance addresses issues of selecting study populations, particularly advising when patients currently consuming opioids are or are not suitable for inclusion, and clarifies when different trial designs are best used. The FDA encourages researchers to assess outcomes such as overall mortality and overdose mortality, the need for emergency medical intervention, and infection or reinfection with hepatitis C. The paper also details how the changes in drug use patterns can be assessed and used as evaluation criteria.

Extended endpoints for searching on OUD results

In addition, the guide encourages developers to go beyond using a simple reduction in drug use behavior as an endpoint and to look at outcome measures that are important to clinicians, patients, and patients. patient families and communities.

It is also recommended to obtain reports from patients and family members to determine the most concerning symptoms of TOU. Specifically, the FDA suggests developing a patient-reported outcome instrument to assess changes such as improvement in sleep or mood, and states that a similar instrument could also be useful for measuring the intensity of the urge to take medication. The guide offers specific suggestions for developing patient-reported outcome instruments, such as determining how much change in a given patient-reported outcome represents a clinical benefit and how long that change needs to be sustained to maintain. this advantage.

Other outcome measures are also discussed. These include a reduction in hospitalizations, an increase in the ability to return to work, school or other activities. The FDA recognizes that evaluating these results will likely require larger trials than those typically subject to market approval. However, the agency encourages the collection of this data, noting that “the use of these results as endpoints for clinical trials could provide the basis for inclusion in the labeling approved by the FDA.”

The directive specifies, however, that “retention in treatment is not recommended as a stand-alone endpoint. Many features of trial design can produce incentives to stay on treatment without accumulating clinical benefit. “4 Should new endpoints be used in drug development trials, the agency strongly encourages developers to discuss these plans early in the drug development process.

The guide also states that when selecting evaluation criteria to support the effectiveness of a particular treatment, developers give due consideration to the risk-benefit equation. If there is a serious risk of adverse events or potential abuse, the demonstrations of the benefits should be convincing.

Overall, as the pandemic creates many challenges for the treatment of patients suffering from opioid dependence, these new directions for OUD drug development offer hope for better treatments to come.

Last update on: December 11, 2020

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