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Today’s Topic: Clinical Considerations for Studies of Devices Intended to 

Treat Opioid Use Disorder

September 14, 2023
Clinical Considerations for Studies of Devices 

Intended to Treat Opioid Use Disorder

Sergio M. de del Castillo, RAC - Devices 

(he/him; they/them) 

Associate Director for Policy 

Office of Neurological and Physical Medicine Devices (OHT5) 

Office of Product Evaluation and Quality (OPEQ)

Center for Devices and Radiological Health 

U.S. Food and Drug Administration
Draft Guidance

• Clinical Considerations for Studies of Devices 

Intended to Treat Opioid Use Disorder 

– www.fda.gov/regulatory-information/search-fdaguidance-documents/clinical-considerationsstudies-devices-intended-treat-opioid-use-disorder

3
Learning Objectives

• Describe the challenges in designing pivotal Opioid Use 

Disorder (“OUD”) device studies 

• Identify the purpose and scope of the draft guidance 

• List the important design features to consider for the 

design of pivotal studies of devices intended to treat 

OUD (“OUD device studies”)

4
The Complexity of OUD

5
Opioid Epidemic and OUD

• The opioid crisis is a public health emergency (PHE) 

• PHE most recently renewed on March 31, 2023

• FDA is committed to addressing this PHE: 

• Part of FDA Overdose Prevention Framework

6
Stigma

Reluctance to 

seek treatment

Relapse Chronicity

Long-term 

recovery

7

OUD
Purpose and Scope of Guidance

8
Purpose

• Provides recommendations for the design of pivotal 

clinical studies for devices intended to treat OUD and 

used to support future marketing authorization

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Scope

• Only devices intended to treat OUD 

• Excluded: 

– Early feasibility/preliminary studies 

– Devices intended to detect opioid use 

– Devices intended to diagnose/determine risk of developing OUD 

– Devices intended to treat pain 

– Combination products

10
Recommendations for Pivotal OUD Device 

Studies

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General Principles

• Well - controlled studies: 

– 21 CFR 860.7 (Determination of safety and effectiveness) 

– See “Design Considerations for Pivotal Clinical 

Investigations for Medical Devices” 

• Discussion through Q - Submission (Q - Sub) 

– See “Requests for Feedback and Meetings for Medical 

Device Submissions: The Q-Submission Program” 

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Clinical Study Design Recommendations

Patient Population 

& Treatment 

Assignment

Recording 

Medication Use

Monitoring Drug 

Use

Study Length and 

Evaluation

Participant 

Retention & 

Missing Data

Clinical Outcomes

13
Patient Population and Treatment Assignment

• Defined, representative study population 

– See “Collection of Race and Ethnicity Data in Clinical Trials” 

• Use a sham control to minimize bias and uncertainty

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Recording Medication Use

• Need to mitigate inaccurate reports of drug use 

• Record baseline medications 

– Prescribed drug(s) 

– Dose 

– Duration 

– Timing of drug use 

– Medication changes 

15
Monitoring Drug Use

• Self - reports and objective measures, such as: 

– Random and schedule urine tests 

– Drug levels in blood or saliva 

– Use of drug detection technologies 

• Quantitative methods take precedence over selfreported information

16
Study Length and Evaluation

• Minimum of 6 months treatment duration

• Include procedures to account for: 

– Participant retention 

– Missing data

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Participant Retention and Missing Data

• Maximizing study participation is critical 

• Missing data introduces a high degree of uncertainty 

in study results 

• Need methods/procedures to address, detect, and 

correct missing data

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Clinical Outcomes

• Generate valid scientific evidence 

• Demonstrate clinically significant benefits 

• Primary outcomes 

– Main source of effectiveness evidence 

• Secondary outcomes 

– Help confirm or explain primary outcomes 

• Consistent with the Center for Drug Evaluation and Research (CDER) 

guidance 

– “Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for 

Treatment Guidance for Industry”

19
OUD 

Device 

Study

Change in 

Drug Use 

Pattern

Change in 

OUD Disease 

Status

Reduction in 

Adverse 

Outcomes of 

OUD PatientReported 

Outcomes

Other 

Outcome 

Measures

20
Resources

Slide 

Number

Cited Resource URL

12 21 CFR 860.7 – Determination of 

safety and effectiveness

www.ecfr.gov/current/title-21/chapter-I/subchapter-H/part860/subpart-A/section-860.7

12 Design Considerations for Pivotal 

Clinical Investigations for Medical 

Devices

www.fda.gov/regulatory-information/search-fda-guidancedocuments/design-considerations-pivotal-clinical-investigationsmedical-devices

12 Requests for Feedback and Meetings 

for Medial Device Submission: The QSubmission Program

www.fda.gov/regulatory-information/search-fda-guidancedocuments/requests-feedback-and-meetings-medical-devicesubmissions-q-submission-program

14 Collection of Race and Ethnicity Data 

in Clinical Trials

www.fda.gov/regulatory-information/search-fda-guidancedocuments/collection-race-and-ethnicity-data-clinical-trials

19 Opioid Use Disorder: Endpoints for 

Demonstrating Effectiveness of Drugs 

for Treatment Guidance for Industry

www.fda.gov/regulatory-information/search-fda-guidancedocuments/opioid-use-disorder-endpoints-demonstratingeffectiveness-drugs-treatment-guidance-industry

21
A Note about Draft Guidances

• You may comment on any guidance at any time 

– see 21 CFR 10.115(g)(5) 

• Please submit comments on draft guidance before 

closure date 

– to ensures that FDA considers your comment on a draft 

guidance before we work on final guidance 

• Draft guidance is not for implementation

22
Submit Comments to Dockets by: 

October 26, 2023

• Draft Guidance: Clinical Considerations for Studies of 

Devices Intended to Treat Opioid Use Disorder 

– Docket: FDA-2023-D-0466

– www.regulations.gov/docket/FDA-2023-D-0466

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Summary

• Spur innovative options to combat opioid epidemic 

• Provide recommendations for OUD device studies: 

– Generate valid scientific evidence from well - controlled studies 

– Demonstrate clinically meaningful benefits 

– Provide reasonable assurance of safety and effectiveness 

• Complexity of OUD necessitates: 

– Collaboration with FDA (Q - Submission process) 

– Discussion of alternative study designs and methods 

24

Additional Panelists

John Marler, MD 

Deputy Office Director 

Office of Health Technology 5 

(Neurological & Physical Medicine Devices) 

Office of Product Evaluation and Quality

Allen Chiu, MD 

Medical Officer 

Division of Health Technology 5B 

(Neuromodulation & Rehabilitation Devices) 

Office of Health Technology 5 

(Neurological & Physical Medicine Devices) 

Office of Product Evaluation and Quality

Megha Reddy, MBA 

Regulatory Advisor 

Regulation, Policy, and Guidance Staff 

Immediate Office 

Office of Product Evaluation and Quality

Center for Devices and Radiological Health 

U.S. Food and Drug Administration

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Let’s Take Your Questions

• To Ask a Question: 

1. Raise your hand in Zoom 

2. Moderator will announce your name and invite you to ask your question 

3. Unmute yourself when prompted in Zoom to ask your question 

• When Asking a Question: 

• Ask one question only 

• Keep question short 

• No questions about specific submissions 

• After Question is Answered: 

• Mute yourself and lower your hand 

• If you have more questions - raise your hand again

27
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