If you are not familiar with edaravone (Radicut or Radicava), here is some important background:
MT Pharma recently filed a New Drug Application (NDA) with the US FDA, asking for a "priority review." That priority review would have been the FDA's commitment to review the application in six months rather than ten. For some reason, the FDA declined the priority request and now we can't expect its decision until June, 2017. That's four extra months for people with a disease that kills 500 Americans every month.
So now we wait while people die. Or maybe we don't.
Normally I'm not a fan of Expanded Access Programs (EAPs) that the FDA allows for drugs that are not yet approved. They can be terribly unattractive for drug developers, especially if their entire market for a brand new drug is an unmet-need disease like ALS. They can be secretive programs where data are lost. The right solution is to get drugs to market approval faster (where everybody has access and payers are paying),
Maybe in this case we shouldn't let the perfect accelerated approval concept get in the way of some possible benefits for the 4000 Americans with ALS who will die before the FDA review is completed and for the 4000 Americans who are about to be diagnosed with ALS in that same period.
Why is this case different?
- We believe that MT Pharma America may be amenable to doing some kind of Expanded Access Program in the United States.
- The product has already been approved and marketed for ALS in Japan since 2015.
- The product had been approved and marketed for stroke in Japan since 2001.
- The product has a good safety profile so far.
- If people with ALS who might participate in an EAP would voluntarily self-report data, perhaps at a place like patientslikeme.com, all might learn more about the drug and potentially which subgroups of people with ALS it may help.
There has already been talk of medical tourism for people with ALS who have the substantial means to go to Japan. There have already been shadowy internet schemes suggested to buy the product in other countries. Neither of these paths of desperation is the right answer for Americans with ALS. A A transparent and legitimate EAP would make so much more sense.
An EAP wouldn't be easy. This is an infusion product and the process won't be cheap. But ALS isn't easy, and it is anything but cheap.
If you have any interest at all in seeing MT Pharma America provide an Expanded Access Program for edaravone, they need to hear from you. If nobody asks, they will have no reason to go down that path. Here is the contact information: 1-888-292-0058 (typo in phone number corrected Nov. 3, 2:30pm EDT... I apologize for the error) or email firstname.lastname@example.org
Some grass-roots ALS advocates started calling yesterday. MT Pharma America has contractors fielding phone calls. They will ask your name, phone number, email address for followup, and relationship to one with ALS. Please take a few minutes and call or email them to let them know the importance that providing early access through an EAP could mean to the thousands of people with ALS who have no great options today. And thank them for their consideration.
It's worth asking. We certainly can't demand it. This case may be different enough that an EAP might be possible for the drug developer to offer, could give people with ALS an option, and might even advance some scientific understanding more quickly.
Please consider and call today. This could be a viable lifeline for people with ALS.