The new article provides an overview of the main points highlighted by the authority in terms of the applicable regulatory requirements and also outlines the aspects to be taken into consideration when following the applicable rules.

The Food and Drug Administration (FDA or the Agency), the US regulating authority in the sphere of healthcare products, has published a draft guidance document dedicated to clinical considerations for studies of devices intended to treat opioid use disorder. Once finalized, the document will provide additional clarifications about the applicable regulatory requirements set forth under the existing legal framework as well as recommendations to be followed by medical device manufacturers and other parties involved in order to ensure compliance. At the same time, provisions of the guidance are non-binding in their legal nature, and they are not intended to introduce new rules or impose new obligations. Moreover, the authority explicitly states that an alternative approach could be applied, provided such an approach is in line with the underlying legislation and has been agreed upon with the authority in advance.

Regulatory Background

The authority acknowledges the significance of the opioid overdose crisis affecting patients in the US. As a part of mitigation measures, the authority takes additional steps intended to decrease unnecessary exposure to opioids by preventing new cases and providing support and treatment to  existing ones. The present document is dedicated to the regulatory requirements for medical devices intended to treat opioid use disorder (OUD). In particular, the document provides recommendations with respect to pivotal clinical studies related to such devices, conducted in order to collect the necessary clinical evidence supporting the claims made by the manufacturer. As explained by the authority, these recommendations are applicable to the design and development of clinical studies to provide a reasonable assurance of safety and effectiveness for a device intended to treat OUD; they may also be helpful for the evaluation of other types of valid scientific evidence demonstrating clinically significant results that may be used to provide a reasonable assurance of safety and effectiveness. The document describes in detail the approach to be applied in order to collect sufficient evidence demonstrating the safety and proper performance of the device in question when used for its intended purpose. It is also important to mention that the authority reserves the right to make changes to the guidance and recommendations provided therein, should this be reasonably necessary to reflect corresponding amendments to the underlying legislation or due to new information becoming available to the authority. 

In accordance with the existing legislation, an OUD stands for a problematic pattern of opioid use leading to clinically significant impairment or distress…occurring within a 12-month period. As further explained by the FDA, it causes significant long-term impairment of physical, emotional, social, or cognitive function. The OUD could be a result of the improper use of medicines containing opioids or the use of illicit drugs. 

The authority further explains that there are numerous challenges related to the design of studies involving OUD medical devices due to the complexity of the disorder itself. The most common issues associated with such studies are those related to inaccurate reports, missing data, and prolonged observation reasonably necessary to assess the long-term effect of the treatment the device in question provides. This guidance is intended to assist the parties involved in developing a proper study design, addressing the abovementioned issues, and mitigating their potential impact on the accuracy and reliability of the study results, as well as the safety of patients.


According to the document, its scope covers pivotal clinical studies addressing matters related to the safety and performance of OUD devices when used for their intended purpose in the context of marketing submissions and evidence used to support the claims made by medical device manufacturers. The document describes the main principles to be taken into consideration by medical device manufacturers and study sponsors when assessing the actual safety and effectiveness of the products subject to investigation. It is important to mention that, in general, the authority provides recommendations for pivotal studies; however, the recommendations contained in the document could apply to other study types. At the same time, the scope of the document does not cover early feasibility and other preliminary studies. The guidance does not cover aspects related to the following products:

  • Diagnostic tests for the detection of opioid use 
  • Devices intended to diagnose or help determine the risk of developing OUD;
  • Devices intended to treat pain; and 
  • Combination products.

Clinical Study Design: Basics

The document further outlines the key points to be taken into consideration by the parties responsible for clinical studies and provides recommendations to be followed in order to ensure the accuracy and reliability of study results. In this respect, the authority emphasizes the importance of ensuring that the studies are well-controlled and that all matters related to potential bias, placebo effects, or other issues affecting the results are addressed. According to the guidance, pivotal OUD device studies used to support marketing submissions should have a well-defined study population, appropriately monitor drug use, control bias, and include an appropriate follow-up period, study participant retention plans, and a data analysis plan. The authority also mentions that the study participants (subjects) involved should be representative of the target population for the device. 

In summary, the present document provides an overview of the regulatory requirements for OUD medical devices as well as recommendations to be considered with respect to the design of clinical studies associated with them. The authority also outlines the key points to be taken into consideration by medical device manufacturers and study sponsors in order to ensure potential issues are duly addressed.

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