PAA, A New Dawn , FDA ODD Approval and Entering Phase II MND Trial, page-380

TY@Nothofagus

Compassionate use is available outside of clinical trials via the Expanded Access pathway.

https://www.fda.gov/news-events/public-health-focus/expanded-access

Pretty sure this has been covered a few times, but there are a lot of new eyes on these threads now, so an expanded answer may be helpful …

Expanded Access may be appropriate when all the following apply:

• Patient has a serious or immediately life-threatening disease or condition.
• There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
• Patient enrollment in a clinical trial is not possible.
• Potential patient benefit justifies the potential risks of treatment.
• Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

When seeking expanded access to an investigational medical product, it is critical that the patient and his/her licensed physician consider all possible risks.
Here’s the process…

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What are the roles and responsibilities?

https://www.fda.gov/news-events/expanded-access/expanded-access-information-industry
Patient
Consults with licensed physician to explore and decide about alternative options.
Licensed Physician
Agrees to oversee the patient's treatment and works with industry (e.g., medical product developer), files paperwork with FDA and IRB (for many expanded access request types), and is responsible for patient care and reporting.
Industry (Company)
Willing to provide the investigational medical product and either submits the expanded access request to FDA, allows the FDA to cross-reference their IND (for drugs and biologics) or IDE (medical devices) on behalf of the expanded access sponsor-investigator through the use of a letter of authorization, or provides the necessary investigational medical product information for the sponsor-investigator to submit to support an expanded access request.
Investigational Review Board (IRB)
Reviews expanded access protocol and consent to ensure that the patient is informed about the nature of the treatment.
FDA
Reviews the expanded access request and determines if the treatment may proceed.

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Note: Investigational drugs, biologics or medical devices have not yet been approved or cleared by FDA and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.

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• So it’s not something the FDA initiate, or respond to a company request — it’s a patient-physician initiated process, and yes, imho they certainly would consider it.

hth,
Iceps personal aside -- the Antarctic Beech is a favourite of mine -- was that a forum name inspiration?