Access to Investigational Devices Outside of a Clinical Trial

Emergency Use, Compassionate Use, and Treatment Use of Investigational Devices

Camille Adair ImageUnder U.S. FDA regulations and guidance[1], an unapproved device may normally only be used for human subjects who meet the inclusion criteria in a clinical trial. However, there are circumstances where the device may be used by the investigator or a doctor outside of the clinical trial, most notably through the emergency use, compassionate use, or treatment use mechanisms.[2]

Emergency use of investigational devices

Under 21 CFR § 56.102(d), emergency use is defined as the use of a test article on a human subject in a life-threatening situation in which no standard acceptable treatment is available and in which there is not sufficient time to obtain IRB approval. The IDE regulations permit deviations from the investigational plan without prior approval when necessary to protect the life or physical well-being of a subject in an emergency.[3]  A single emergency use is exempt from IRB review if the emergency use is reported to the IRB within 5 working days. A second use of the test article must receive IRB review and approval prior to use. [4]

Infant_ICUUnder 21 CFR § 50.23(a), the investigator/physician must obtain informed consent from the subject for an emergency use unless he/she and another physician not otherwise participating in the clinical investigation certify in writing all of the following: (1) the subject is confronted with a life-threatening situation necessitating the use of the investigational article; (2) informed consent cannot be obtained from the subject because of an inability to communicate with, or obtain legally effective informed consent from, the subject; (3) time is not sufficient to obtain consent from the subject’s legally authorized representative; and (4) there is no alternative method of approved or generally recognized therapy that provides an equal or greater likelihood of saving the life of the subject.

In addition to notifying Quorum, the investigator/physician must notify the FDA of the use through submission of an IDE report. If approval by the FDA was not obtained prior to the emergency use, the FDA expects a sponsor to provide notice to FDA within 5 working days.[5]

Emergency Use: Reporting Procedures

Click here for information on Quorum’s reporting procedures for emergency use of test articles.

Compassionate use of investigational devices

med_deviceIn comparison to emergency use, compassionate use allows access for a patient who has a serious (albeit not life-threatening) disease or condition, but does not meet the inclusion criteria for the clinical investigation. To qualify, the treating physician must believe the device may provide a benefit in treating and/or diagnosing his/her disease or condition and the investigational device is the only option available. This provision is typically approved for individual patients, but may be approved for a small group.

Unlike emergency use, prior FDA and IRB approval is needed for compassionate use. A sponsor must submit an IDE supplement under §812.35(a) in order to treat the patient. The IDE supplement should include:

  1. A description of the patient’s condition and the circumstances necessitating treatment;
  2. A discussion of why alternatives therapies are unsatisfactory and why the probable risk of using the investigational device is no greater than the probable risk from the disease or condition;
  3. An identification of any deviations in the approved clinical protocol that may be needed in order to treat the patient; and,
  4. The patient protection measures that will be followed. (Informed consent, concurrence of IRB chairperson, clearance from the institution, independent assessment from uninvolved physician, authorization from IDE sponsor).

FDA approval must be obtained before the physician may treat the patient. Typically, the treating physician is responsible for the following:[6]

  • Obtaining sponsor agreement;
  • Obtain independent assessment from uninvolved physician;
  • Ensure the FDA has approved the use before compassionate use occurs;
  • Obtain IRB review and concurrence with the use;
  • Devise schedule for patient monitoring;[7]
  • Obtain consent from the patient or the legally authorized representative;
  • Obtain clearance from the institution (if necessary);
  • Report any problems as a result of the compassionate use tode the IRB and sponsor (if any problems occur, these must be reported as soon as possible to the reviewing IRB); and,
  • Following the use, write a summary and submit to the sponsor and FDA.[8]

Compassionate Use: IRB Submission Requirements

Quorum Review’s Submission Requirements  for compassionate use investigational devices are available here.

Treatment use of investigational devices?

Under U.S. regulations, the primary intent of a treatment investigational device exemption (treatment IDE) is to provide access to the device for people with a serious or life-threatening condition for which there is no comparable or satisfactory device or other therapy available. The secondary intent is to generate additional information about the device, especially its safety and effectiveness.[9]

Treatment IDEs are granted by the FDA pursuant to 21 CFR §812.36 if the following conditions are satisfied:

  1. The device is intended to treat a serious or immediately life-threatening disease or condition;
  2. There is no comparable or satisfactory alternative device or other therapy available to treat or diagnose that stage of the disease or condition in the intended patient population;
  3. The device is already under investigation for the same use, or trials are complete; and
  4. The Sponsor is actively pursuing marketing approval/clearance of the investigational device with due diligence.

Treatment Use: IRB Submission Requirements

Quorum Review’s Submission Requirements  for treatment use investigational devices are available here.

Read previous  article                                         Read next  article

Institutional-Startup-Ad-700-x-100-3

References


[1] http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevice/InvestigationalDeviceExemptionIDE/ucm051345.htm

[2] In addition to the uses noted above, a patient may receive an unapproved device through emergency research or continued access. See the following website for more information: http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevice/InvestigationalDeviceExemptionIDE/ucm051345.htm

[3] 21 CFR § 812.35(a)

[4] 21 CFR § 56.104(c)

[5] See 21 CFR § 812.150(a)(4); information also available in Information Sheet Guidance for IRBs, Clinical Investigators, and Sponsors, Frequently Asked Questions About Medical Devices, available at: http://www.fda.gov/downloads/RegulatoryInformation/Guidances/UCM127067.pdf

[6] http://www.fda.gov/downloads/Training/CDRHLearn/UCM180888.pdf

[7] The attending physician is responsible for devising an appropriate schedule for monitoring the patient, which should take in to consideration the investigational nature of the device and the specific needs of the patient. The patient should be monitored to detect any possible problems arising from the use of the device.

[8] http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevice/InvestigationalDeviceExemptionIDE/ucm051345.htm#emergencyresearch

[9] 21 CFR § 812.36 (a).

Tags: , , , ,