The ideal (fastest) path to approval for Bryostatin would be accelerated approval.
From Wikipedia:
The [US FDA] initiated the FDA Accelerated Approval Program in 1992 to allow faster approval of drugs for serious conditions that fill an unmet medical need. The faster approval relies on use of surrogate endpoints.[1] Drug approval typically requires clinical trials with endpoints that demonstrate a clinical benefit, such as increased survival for cancer patients. Drugs with accelerated approval can initially be tested in clinical trials that use a surrogate endpoint, or something that is thought to predict clinical benefit. Surrogate endpoints typically require less time, and in the case of a cancer patient, it is much faster to measure a reduction in tumor size, for example, than overall patient survival.
Drugs approved under the FDA Accelerated Approval Program still need to be tested in clinical trials using endpoints that demonstrate clinical benefit, and those trials are known as phase 4 confirmatory trials. If the drug later proves unable to demonstrate clinical benefit to patients, the FDA may withdraw approval.[2][3]
The example above is for accelerated approval for cancer drugs. Most accelerated approvals involve completing at least 1 phase 3, but there are rare cases of approvals after phase 2.
As it relates to Bryostatin for Alzheimer's, if the phase 2 is well-designed and if the results significantly exceed the 95% confidence interval, I could see the POSSIBILITY of accelerated approval for Bryostatin after the upcoming Phase 2 trial. Alzheimer's disease is a serious condition with an unmet need, and one could view the surrogate endpoint as improvement at 15 weeks (typical phase 3 AD drug trials last at least 6 months). The separate "phase 4" trial, conducted after approval, could prove out the 6 months statistically significant improvement, and may extend up to a year to clear up any lingering concerns about long-term safety.
Assuming the FDA agrees to accelerated approval based on the phase 2, the approval process would still take at least 15 months (6 months to prepare paperwork for filing, 6 months to review and then a PDUFA date is set).
Further, the manufacturing site needs CGMP approval by the FDA, which could take at least 1 year (can be done in parallel with paperwork).
Most drug companies will (wisely) allow ONLY very limited access for compassionate-use, typically for those who have already been in a trial (to continue the drug beyond the end of the trial). Agreeing to compassionate use for those who have NOT been in Bryostatin drug trials can be perilous: What if bad outcomes happen with patients who aren't closely followed and monitored? What if patients aren't seeing the benefits, because the drug isn't being administered correctly in a well-controlled trial?
While I would love to see patients get Bryostatin as quickly as possible, I would not be keen to see widespread use of Bryostatin (compassionate-use) until it is approved. Getting approval of Bryostatin should be the focus.
