The FDA also states that the statutory requirements of safety and efficacy will be applied...but with flexibility.
Quote: "To A Degree, Flexibility Is Part of FDA Regulation Paradigm (21 CFR 314 (APPLICATIONS FOR FDA APPROVAL TO MARKET A NEW DRUG)
Regulations allow for flexibility and judgment in applying the standards, and the FDA has a solid record of appropriately applying regulatory flexibility.
Flexibility
“While the statutory standards apply to all drugs… the many kinds of drugs… and wide range of uses for those drugs demand flexibility in applying the standards.
Thus FDA is required to exercise its scientific judgment to determine the kind and quantity of data and information an applicant is required to provide for a particular drug to meet the statutory standards.” (21 CFR 314.105 Approval of an Application)
How Much Evidence Is Enough to Obtain FDA Approval?
FDA may consider “data from one adequate and well-controlled clinical investigation and confirmatory evidence” to constitute substantial evidence."
Hopefully, I've established the fact that the FDA is going to be very flexible when Anavex submits data from the two trials (P2 N=25 and P3 N=36...which totals 61 patients).
Furthermore, the two trials mentioned above are NOT the only data to prove safety. I'm assuming that Anavex will also submit a safety record that goes back seven years. The safety record is the #1 concern for the FDA before anything else.
Side note: I just noticed that the Doc posted his reservations about the adult Rett approval too. What he doesn't address is the 7-year safety record that Anavex will submit vs. other rare disease drug approvals. Again, safety is the #1 concern for the FDA before any drug is approved. (We got this!)
Lastly, the FDA will be meeting with the International Rett Syndrome Foundation on March 11thto discuss new drug approvals. What are the chances that the Rett Foundation will be promoting (demanding!) Anavex's drug for approval?
"The International Rett Syndrome Foundation and the Rett Syndrome Research Trust are hosting an Externally-Led Patient-Focused Drug Development meeting and invite you to attend. By signing up you will receive the link to a public meeting with the US Food and Drug Administration and other stakeholders including drug developers, where you will be able to share the impact of Rett syndrome on your child and family, your perspective on possible treatments, and how symptom improvements would affect your and your child’s quality of life. The FDA is especially interested in hearing from parents, caregivers, and family members from the United States."
One other note: Does anyone know if Anavex will be applying for drug approval in Australia and England with the same data? If so, that too may have some sway with the FDA as well.
Excellence delay ensures Avatar results will receive the go ahead nod from FDA…no point in waiting another 6 months + just to confirm it works and it’s safe.
It’s a rare disease and nothing is available. People are suffering now. To wait for delayed Excellence is cruel!!!
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