HER2 Support Group Forums - View Single Post - Help me! Compassionate use exceptions needed for trial drugs NOW

StephN · 06-21-2009, 12:05 PM

Hello all -
This discussion has been rattling around in my head the last fews days as I fight off a vicious cold.

I am wondering why there could NOT be a class of use that is not exacly a Clinical Trial in the usual sense, but after a promising phase 1, a drug could not be open to compassionate use? The answer most likely lies in the clammoring that would happen for that use. If you let in 20 patients, why not 40 or 80??

Anyway, we know now how to administer once the dose is defined in phase 1, and how to gauge efficacy of the drug. If the patient shows improvement, stay on it. This could run parallel to the "official" trials. This "use" would probably have to be in centers where the trial is taking place for access to the drug.

But then the other questions are: which drugs to allow such use? Where are the lines drawn in such a gray area? I am sure other concerns arise, but just as in the trials there would be Informed Consent, and the drug companies are off the hook if the patient wants the drug that badly.

WHO PAYS? That is probably the biggest question, if this would not fall under Compassionate Use.

06-21-2009, 12:05 PM	#26
StephN Senior Member Join Date: Nov 2004 Location: Misty woods of WA State Posts: 4,128	Hello all - This discussion has been rattling around in my head the last fews days as I fight off a vicious cold. I am wondering why there could NOT be a class of use that is not exacly a Clinical Trial in the usual sense, but after a promising phase 1, a drug could not be open to compassionate use? The answer most likely lies in the clammoring that would happen for that use. If you let in 20 patients, why not 40 or 80?? Anyway, we know now how to administer once the dose is defined in phase 1, and how to gauge efficacy of the drug. If the patient shows improvement, stay on it. This could run parallel to the "official" trials. This "use" would probably have to be in centers where the trial is taking place for access to the drug. But then the other questions are: which drugs to allow such use? Where are the lines drawn in such a gray area? I am sure other concerns arise, but just as in the trials there would be Informed Consent, and the drug companies are off the hook if the patient wants the drug that badly. WHO PAYS? That is probably the biggest question, if this would not fall under Compassionate Use. __________________ "When I hear music, I fear no danger. I am invulnerable. I see no foe. I am related to the earliest times, and to the latest." H.D. Thoreau Live in the moment. MY STORY SO FAR ~~~~ Found suspicious lump 9/2000 Lumpectomy, then node dissection and port placement Stage IIB, 8 pos nodes of 18, Grade 3, ER & PR - Adriamycin 12 weekly, taxotere 4 rounds 36 rads - very little burning 3 mos after rads liver full of tumors, Stage IV Jan 2002, one spot on sternum Weekly Taxol, Navelbine, Herceptin for 27 rounds to NED! 2003 & 2004 no active disease - 3 weekly Herceptin + Zometa Jan 2005 two mets to brain - Gamma Knife on Jan 18 All clear until treated cerebellum spot showing activity on Jan 2006 brain MRI & brain PET Brain surgery on Feb 9, 2006 - no cancer, 100% radiation necrosis - tumor was still dying Continue as NED while on Herceptin & quarterly Zometa Fall-2006 - off Zometa - watching one small brain spot (scar?) 2007 - spot/scar in brain stable - finished anticoagulation therapy for clot along my port-a-catheter - 3 angioplasties to unblock vena cava 2008 - Brain and body still NED! Port removed and scans in Dec. Dec 2008 - stop Herceptin - Vaccine Trial at U of W begun in Oct. of 2011 STILL NED everywhere in Feb 2014 - on wing & prayer 7/14 - Started twice yearly Zometa for my bones Jan. 2015 checkup still shows NED 2015 Neuropathy in feet - otherwise all OK - still NED. Same news for 2016 and all of 2017. Nov of 2017 - had small skin cancer removed from my face. Will have Zometa end of Jan. 2018.