August 2009 FDA ruling on access to investigational drugs
- link here to read new FDA rules on when patients can access unapproved drugs and whether phamaceutical companies can charge for this access.
"Compassionate use" refers to obtaining a medication that does not have FDA approval yet (possibly when trials have been conducted but approval is pending) or a drug that is in a trial for which the patient does not qualify. If you do not want to enter an actual trial due to personal circumstances, or are unable to enter one due to your medical situation, you can have your doctor apply directly to the drug company that is testing the medication. If you qualify, then you are given the medication directly, under the supervision of your own doctor and the institutional review board (IRB) of his or her clinic or hospital.
IF the requesting physician submits the correct documentation, the approval by the drug manufacturer may only take a few days. However, the FDA must also review the application, requiring more time. Almost all of the delay occurs at the physician and medical institution end, including IRB approval, and at the FDA.
In 2007 one example of a drug with a compassionate use program is nilotinib (Tasigna, formerly known as AMN107) manufactured by Novartis. However, the FDA required this access to be discontinued after nilotinib was approved by the FDA for chronic myelogenous leukemia.
Back in 2004, when Sutent was in trials, someone asked some good questions, that may also apply to today's drugs. Here are Dr. Demetri's answers to her questions:
Q: What criteria determined that you would be a compassionate user rather than a trial patient?
A: I believe that the patients here who are being treated on the SU11248 expanded access program were considered ineligible for the Phase III SU11248 clinical trial based on variety of criteria. Some people simply were too unwell to participate in the trial. Others had some abnormalities in certain screening tests (such as liver or kidney function) which disqualified them from participation in the Phase III trial.
Q: How is your condition being monitored?
A: The follow-up of patients on the SU11248 expanded access trial is a compromise between careful and close observations (which might be inconvenient for patients, especially those who travel from far away) and less-intensive scheduling which might be more convenient for the patient. We are definitely focused on maximizing patient safety, and therefore there is a need for ongoing evaluation at the center which takes responsibility for prescribing this investigational new drug.
Q: Are you on the same dosage as trial patients? Do you have the option of increasing that dosage? Are you on the drug all the time or is there the 2 week wash out period like the trial patients?
A: The patients on the SU11248 expanded access trial are taking the same doses of SU11248 as the Phase III clinical research trial patients, with the caveat that there may be dose reductions if there are any safety concerns. For example, if a patient's liver function is not sufficiently good to allow them to enter the Phase III trial, it might be better for that patient to consider starting the SU11248 expanded access treatment plan with a somewhat lower dose of SU11248, at least to start, to maximize safety. Once the drug is shown to be safe and well tolerated in that individual, the physician may choose to increase the dose to the usual and standard dose. All patients are taking the SU11248 in the same schedule as the Phase III trial patients. At this point, all the experience with SU11248 is based on intermittent dosing.
Q: Are any of the patients currently on the compassionate use program former trial patients who have progressed to the point where they are officially "kicked out" of the trial?
A: We do not have any sense of "kicking out" any patient. There may be patients who have safety issues (where the risk of the SU11248 might be viewed as outweighing the risk of the disease, believe it or not) and in those patients individual investigators might choose to discuss with Pfizer how best to customize the approach to therapy for that individual patient. Such patients might be able to remain on study with some sort of special exemptions or might be better served by moving to the expanded access option.
Q: If a trial patient has surgery on one or two tumors that are progressing, are they permitted to stay in the trial or would they then have to seek compassionate user status?
A:We have typically kept such patients on the trial, although the specific details about how to measure their disease burden and code the data becomes somewhat problematic. We do what is viewed as best for the patient. I hope this helps. George Demetri MD
I am receiving Amn107 on a compasionate use protocal. With compasionate use provided that you are an acceptable candidate you just need to know the contact info for Novartis. You then take this info to your local oncologist or specialist and have them request the medication on your behalf. There are several hurdles to over come. I chose this route because I didn't want to travel. Basically, your local doctor becomes the investigator for the pharmaceutical company.