FDA Response To "Compassionate Use"

[Magnum]

I've tried this along with my doctor before, while drugs were in Phase 2 of trials and was denied by Vertex. However, maybe now that they are in their final phase, they may relent.. It may be worth a try for those of you who's backs are up against the wall and need the meds ASAP. At any rate, below is the response from the FDA...

Mag


Thank you for your message to the Division of Drug Information at the Center for Drug Evaluation and Research (CDER), within the Food and Drug Administration (FDA).

We sympathize with your situation and appreciate the time that you have taken to contact us.

Outside of a clinical trial, the only mechanism for a patient to legally obtain an unapproved drug is through a Physician Request for an Individual Patient IND (Investigational New Drug) application under Expanded Access for Non-emergency or Emergency Use ( commonly referred to as  "compassionate use"):  http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/ucm107434.htm

Please note that the physician must be willing to undertake the necessary responsibility and adhere to the FDA's regulatory requirements, but the manufacturer must also be willing to make the drug available for the individual patient IND. The physician should first ensure that the manufacturer of the unapproved drug is willing to provide the drug. If the manufacturer agrees to provide the drug, the physician should submit an IND to the appropriate review division.

If the physician needs further general direction, he can reach this office at 1-301-796-3400 or 888-INFO-FDA.

Best regards,
Drug Information BLS
Division of Drug Information
Center for Drug Evaluation and Research
Food and Drug Administration

[Magnum]

I have a contact at Vertex and will call Monday and post the response. I will ask if they will now consider a "compassionate use" release of the drug for those in dire need since they are in their final testing / clinical trials phase. Wish me and you luck...

Mag

[willing]

thanks for posting - I wonder whether this path is or is not related to the expanded-early-access they're considering.

[Marcia2202]

I hope that they will give it to you this time!!!

Wishing you the best of luck my friend.

Marcia

[22hamilton]

Thanks Magnum I know that compassionate use was permitted for HIV so why not HEP C also,I know that my hospital has good ties with a lot of drug companies and ny consultant will more than likely apply as soon as he is able. I will post on here as soon as I get something to report.

[nygirl7]

Well Magnum it looks like ever so slowly and surely you are getting closer.  Just goes to show what you can really accomplish when you don't give up.

As always hoping for the best for you and that you get them SOONER than anybody!  :)