Replies to post #267806 on Anavex Life Sciences Corp (AVXL)

[rayovacAAA]

WHAT IS DIFFERENT ABOUT APPLYING FOR RIGHT TO TRY VS. APPLYING FOR SAS APPROVAL?????????

I know we have "right to try" in the U.S., but I wish the FDA would come out like Australia to have the opportunity for anyone who is suffering to try 2-73!!

http://righttotry.org/rtt-faq/

Who qualifies for Right to Try?

To be eligible for Right to Try, a patient must meet the following conditions:

Be diagnosed with a life-threatening disease or condition;

Have exhausted approved treatment options;

Be unable to participate in a clinical trial involving the eligible investigational drug, as certified by a doctor, who is in good standing with her licensing organization and will not be compensated directly by the manufacturer for so certifying; and

Give written informed consent regarding the risks associated with taking the investigational treatment.

What drugs or treatments qualify for Right to Try?

The treatments available under the law must meet the following conditions:

Have completed an FDA-approved Phase 1 clinical trial;

Be in an active clinical trial intended to form the basis of an application for approval or be the subject of an application for approval that has been filed with the FDA; and

Be in ongoing active development or production and not discontinued by the manufacturer or placed on clinical hold.

[Doc328]

There are actually two less steps in the US than in Australia. There are only 3 parties involved in the US (patient/doctor/pharmaceutical company) instead of 4 parties in AU (patient/doctor/Government/pharmaceutical company).

I wonder if Anavex has received requests. Many companies don't participate in early access schemes like RTT and SAS because risk of deaths for non-trial patients (even if not related to the study drug) muddies the water for approval.

In US:
1. Patient (and/or caregiver) and doctor agree that trying a drug is worth the potential risk
2. Patient/caregiver sends a letter to the drug company
3. Drug company agrees or does not agree to provide a drug for free or for a price without profit

In Australia:
1. Patient (and/or caregiver) and doctor agree that trying a drug is worth the potential risk
2. Patient/caregiver send in the SAS Cat B 2 page application to the TGA
3. The TGA either agrees or does not agree to allow the access.
4. Patient/caregiver or doctor (not clear who requests) sends a letter to the drug company
5. Drug company agrees or does not agree to provide a drug for free or for a price

In US, a phase 1 must have been performed and patient is ineligible for an existing trial. The FDA plays no role in the RTT process as long as phase 1 has been performed.

In AU, a phase 1 is not necessary. Point is moot as A273 has phase 1 (and also 2a data)