The Food and Drug Administration (FDA) has provided potential pathways for patients who are suffering from serious or life-threatening diseases or conditions to access an investigational product for treatment outside clinical trials. There are separate pathways for investigational medical devices and investigational drugs/biologics. All expanded access pathways require both the IRB and Regulatory Affairs to be involved early on as the IRB may need to approve the study prior to use and a submission to the FDA may be needed prior to use. Please see the FAQs below for more detail.
What are the investigational medical device expanded access pathways?
Expanded access(also known as "compassionate use") is a potential pathway for patients with a serious or life-threatening disease or condition to access an investigational medical device that has not been approved or cleared by the FDA for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available through one of three mechanisms:
- Emergency Use
- Compassionate Use (or Individual Patient/Small Group Access)
- Treatment Investigational Device Exemption (IDE)
Please contact the Regulatory Affairs and Clinical team early in your design process so that the team can identify what controls are needed and provide a path on how to comply with them.
What are the investigational drug/biologic expanded access pathways?
Expanded access(also known as "compassionate use") is a potential pathway for patients with a serious or life-threatening disease or condition to access a new drug or biologic that has not been approved or cleared by the FDA for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available through one of three mechanisms:
- Individual Patient Access
- Intermediate-Size Patient Population Access
- Expanded Access for Widespread Use
Depending on the scenario, all three mechanisms can be submitted to the FDA as either a Treatment IND or a Treatment Protocol. Please contact the Regulatory Affairs and Clinical team early in your design process so that the team can identify what controls are needed and provide a path on how to comply with them.
When should I get Regulatory Affairs involved?
It is highly recommended to engage with the Regulatory Affairs and Clinical Trials team early on in the study design process. The Regulatory Affairs and Clinical Trials team can identify possible challenges that may need to be addressed prior to submitting funding proposals and/or submitting to the IRB for review. Thees challenges can both delay the start of the study and add unexpected costs.
How does Regulatory Affairs support the expanded access process?
The Regulatory Affairs and Clinical Trials team will help the study team identify an appropriate third party firm to meet comply with what is required. What is required may vary on the risk of the product and how the product interacts with the patient. Please contact the Regulatory Affairs and Clinical team early in your design process so that the team can identify what controls are needed and provide a path on how to comply with them.