Al's Comment:

 This is completely unacceptable to the brain tumor community. The FDA is taking it upon itself to use it's regulatory flexibility to change the way accelerated approval works. The article hints that this will add more than 3 1/2 years to the process of getting approval.   The cause of this change seems to be a mistake they made with approving a drug for Alzheimer’s disease.   There are a few drugs that should have already received accelerated approval for brain tumors and I am being told the FDA told these companies they couldn't even apply yet, and that they want a final phase 3 placebo controlled trial fully enrolled before the FDA would accept an application for accelerated approval. This will kill thousands of patients needlessly and reduce the number of treatments available to us.  This is the same FDA that told me they do not have the regulatory flexibility to grant accelerated approval to a treatment that has been shown to work well in phase 3 trial, but only in certain subgroups that made sense.  They wanted a repeat of the phase 3 randomized trial in those  subgroups, which makes perfect sense from a scientific viewpoint but wound up making the company go out of business, forcing us to lose the treatment forever, and probably killing 1,000- 2,000 brain tumor patients a year needlessly.   

I am working on a fix. I invite other foundations who are also interested in this to contact me.  My original plan was the promising pathway act, which would create a new conditional approval pathway to allow treatments to get approved after a phase 2 trial, but require all patients who use the drugs approved under this pathway to participate in a virtual trial until efficacy was proven, or failure was shown.   That bill is still in Congress - so it has a chance - but I have thought of another possible way forward. Change accelerated approval to require all patients who use drugs approved by the pathway to participate in a virtual trial, instead of requiring the confirmatory trial. This would go on until we either proved the drug works or not.  This would give us more knowledge as we learn from every patient.  


Posted on: 11/22/2022

The accelerated approval pathway is evolving in plain sight, with or without congressional help

 


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