Hi Mag1 and Betty,
Although I've never applied for an FDA compassionate use ruling, I can give you some basic information about it.
The FDA sometimes allows people with serious and life-threatening illnesses to take an investigational new drug (IND) while it is still being tested in a clinical trial. Typically, however, drugs allowed for this use have already have shown promise and proven safety.
Here's what the FDA site says about the "compassionate use" ruling for individual patients:
"If enough is known about the drug's safety, and there is some clinical evidence of effectiveness, FDA may allow a patient to become his or her own study. This so-called single-patient IND, or compassionate use IND, virtually ensures that any patient can get access to any investigational new drug.
"Although FDA's requirements for a single-patient IND are relatively simple, setting up this kind of access for an individual patient is not. First of all, the company must be willing to provide the new drug to the patient. This can be expensive and time consuming for the company since, in addition to providing the drug, the company needs to track shipments of the drug, create special instructions for its use, and create a way of collecting safety data and a mechanism for tracking outcomes for each patient. Second, the patient must give informed consent, understanding that the drug is not approved and may cause side effects from mild to fatal. Third, the patient's physician must be willing to take responsibility for treating the patient and agree to collect information about the effects of the drug.
"Companies sometimes say that they cannot make the drug available to a patient because FDA won't allow it, but that is rarely true. FDA only denies access when there is evidence that the risk of using the experimental drug clearly outweighs any potential benefit to the patient."
Mag1, you don't say what drug you are applying for or why. If I understand the FDA information correctly, I think they usually only approve compassionate use for patients with life-threatening illnesses - but I could be wrong. From what this info said, it sounds like the hardest and most time-consuming part of the process is getting the pharmaceutical company and the doctor to agree to do the necessary tracking and record keeping.
If you're willing to share, I'd be interested to know more about your request and whether you have begun the process yet.
Wishing you the best,
Karen
Karen Lee Richards
ChronicPainConnection Expert
