Genervon and the FDA have been engaging in an interesting p.r. war, lobbing statements overhead while people with ALS and their caregivers are stuck in the trenches.
Genervon has been obvious (and, therefore, not very effective) in trying to make a very public appeal for FDA Accelerated Approval of GM604 for ALS.
The FDA has been very nuanced in making statements to cast aspersions on Genervon. I'm pretty sure this recent public statement cost the American taxpayer a small fortune http://www.fda.gov/Drugs/DrugSafety/ucm443242.htm
And the media know an interesting story when they see one.
So who is using whom here? I don't know. I don't really care.
We need to take a time-out from this p.r. circus and listen to the adults in the room -- the people with ALS and their caregivers.
This is about much more than Genervon. This is about a failed process for testing and approving therapies for ALS.
Fact: A number of years ago an FDA representative explained to some ALS advocates that the FDA can't just do things. They need to react to things that are brought to them, and, frankly, the FDA doesn't get much for ALS beyond a few traditional Phase 1,2,3 trials.
Fact: Genervon took them something different -- a request to use the Accelerated Approval path that was established for the FDA some years ago.
Fact: People with ALS and their caregivers have asked repeatedly for the chance to assume more risk in order to try investigational therapies and to advance the science more quickly. The FDA and our ALS organizations have nodded but delivered nothing to let that happen.
Fact: We don't know if GM604 will work, but its safety profile to-date does not put the fear-of-God in most people with ALS.
Fact: Traditional Phase 2 trials are not big enough to be definitive. They are not designed to look for efficacy, yet for a disease like ALS, every trial is being scrutinized for some sign of efficacy.
Fact: Traditional Phase 3 trials are dreadfully expensive; therefore, "promising" drug candidates sit on shelves while people with ALS die.
We have a failed process. It takes too damned long.
We have people with ALS and caregivers who are demanding that the word urgency finally be given some teeth in the fight against ALS. They are the ones who need to be heard in this conversation. They are smart. They are thoughtful. They are facing a fast and difficult path to death. They understand good science. They have eyes wide open about risk. They know p.r. volleys when they see them, too.
Genervon has pushed the boundaries of the same old same old with their request for Accelerated Approval. They have upset those who seem to believe that the existing Phase 1,2,3 trial model is the only way to do good science.
Listen to the adults in the room -- those with ALS and their caregivers. They are smarter than any of the p.r. wonks realize.
You will have a chance to listen to some of them in person http://www.meetup.com/ALS-Faster-Drug-Approval/events/221529700/ .
They are the ones acting like adults here. They need to be heard. And this is about much more than GM604.
FDA can (and it fact is limited by law) to communicating any concerns or questions it has regarding the information submitted by Genervon, to Genervon. Further, the FDA is in complete control of the regulatory path and availability to patients of GM604. Genervon appears to be following the FDA’s process in good faith, and there is nothing improper about their request that FDA consider Genervon’s request for the agency’s position on the eligibility of GM604 to be considered for Accelerated Approval. A decision to accept an application for Accelerated Approval is made solely by the FDA.
ReplyDeleteIt is important for all to know that FDA is prohibited by law and regulation from publicly discussing its interactions with companies developing new medicines. The FDA is also strictly prohibited from releasing any information submitted by a company developing a drug within the FDA’s process, except under a small set of special circumstances that arise later in the development process, and that do not apply in this case. It seems that these restrictions on the agency also would prohibit the FDA from trying to force a company to release confidential data to the public by posting a request for that release on its website. The campaign supporting AAP for GM604 has communicated with the FDA regarding their post and asked them to clarify that Genervon has no obligation to comply with their request. The FDA has refused, leaving Genervon and the ALS community to try to deal with FDA’s irresponsible mistake on our own.
This appears to be a powerful government agency playing games with a promising drug for ALS for its own unstated purposes in a manner that may violate its own laws and regulations, and it is unacceptable.
Genervon is NOT following the FDA process in good faith. They have to supply the data they have and complete the appropriate applications/paperwork, which they have not done. What is it exactly that the ALS community is asking for the FDA to do...? They have nothing to approve yet.
ReplyDeleteActually anonymous, Genervon HAS followed the FDA process. It asked the FDA for guidance as to how to apply for accelerated approval and if the FDA would consider taking it's application for AAP based on surrogate endpoints. This is the proper procedure used by ALL companies interested in applying for AAP, to ask for guidance. It is the first step, always. The FDA's response was to demand Genervon PUBLICLY release all it's trail data and study results. This goes against the FDA's own regulatory guidelines and is possibly illegal. It was not Genervon who started the GM604 demand for AP. It was a single patient in the trial who saw significant results in his swallowing capacity and asked Genervon to approach the FDA about AAP for the peptide. And it has caught fire in the ALS community. The FDA is trying to throw a small drug company who has offered ALS patients a glimmer of hope under the bus to take the pressure of of their monumental lack of action on behalf of ALS patients. They are confused as to why their normally complacent lab rats are getting uppity about their own fate and they don't like it, one bit. The FDA is at fault, not Genervon.
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