Ashton Steinhagen, Lead Clinical Research Associate for IMARC Research
What happens if you have a site that requests to use an unapproved device that could be life-saving for a patient without an Investigational Device Exemption (IDE)? The device has been through the phases of clinical trials and the Sponsor is working on submitting the premarket approval (PMA). The site in question was part of that clinical trial but now the study and enrollment goal has been met and both the site and study are closed.
According to a response by a consumer safety officer at the FDA, there are two options for compassionate use cases; one with an IDE and one without an IDE. The specific criteria for compassionate use cases states that “the patient has a life-threatening or serious disease or condition; and no generally acceptable alternative treatment for the condition exists.” The following criteria for compassionate use with an IDE, noted from the FDA’s website, are as follows:
- A description of the patient’s condition and the circumstances necessitating treatment;
- A discussion of why alternative 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;
- An identification of any deviations in the approved clinical protocol that may be needed in order to treat the patient;
- The patient protection measures that will be followed:
- A draft of the informed consent document that will be used;
- Clearance from the institution as specified by their policies;
- Concurrence of the IRB chairperson;
- An independent assessment from an uninvolved physician; and
- Authorization from the device manufacturer on the use of the device.
If you are interested in reviewing more details about what is required for compassionate use if the device has an IDE, please visit this blog here.
If there is no IDE, the physician or manufacturer will submit the same information noted above that is applicable and a description of the device. A few of the criteria will not be the same as those with an IDE such as IRB responsibilities and protocol references (bullet point three); however, all other requirements should be completed. Based on the response by the safety officer at the FDA, the patient may not need to sign an informed consent document. Her response indicated that the last bullet point noted, which is in regards to demonstrating appropriate patient protection measures, may point a physician or manufacturer to completing an informed consent document as a safety measure but that it is not necessarily required. Based on the response given, it was strongly suggested that the physician or manufacturer work together with the FDA during this scenario to verify that patient safety measures have been thoroughly addressed. The FDA has provided a resource in such cases and questions and/or concerns can be addressed by contacting CDRHExpandedAccess@fda.hhs.gov for assistance.
If this request is approved by the FDA, the physician should highlight a follow-up schedule for monitoring the patient. Because this would not be an approved device, the follow-up schedule might not follow the typical standard of care timelines the physician might be used to. It would be imperative that the patient’s safety is the focal point of this schedule as unknown adverse events or issues could arise that would need to be addressed.
Normal IDE compassionate use cases would fall under the 30-day review cycle; however, this situation, without an IDE would not follow this timeline. Compassionate use cases without an IDE do not have a timeframe; although, the average length for review was 10 days with a review in as little as the same day of receipt for some cases according to the FDA. Most of the cases get approved as the rationale for use outweighs the risk. In 2015, 170 compassionate use cases without an IDE were submitted with 167 (98.8%) meeting approval.
After implementation, a follow-up report has to be submitted by whoever submitted the original application or compassionate use request. The report should include a summary regarding the patient outcome and any problems occurring as a result of the use of the device.
Unfortunately, for compassionate use cases without an IDE, the guidelines and requirements have grey areas regarding what exact patient safety measures are needed. Have you come across a situation like this? Do you think an informed consent document is needed? Please share your thoughts below.
The opinions expressed in this blog post are the author’s only and do not necessarily reflect those of MassDevice.com or its employees.