One of the hottest subjects in the world of stem cells this year has been the REGROW RESPOND and at a larger level the movement for changes within FDA oversight of investigational regenerative medicine therapies (see my past posts on it here ), so the related provisions in the 21st Century Cures Act are usually stirring much discussion today. The bill is known as Cures for short and you can read the newest draft here .
Cures may get the vote in Congress as early as tomorrow so this is a large week for the stem cell field and the life technology more generally in the U. S. See areas 3033-3036 of the bill as those are on regenerative medication.
I’ m reaching out to a number of stakeholders for perspectives. Earlier today I interviewed Michael Werner (MW), the Executive Director of the Alliance for Regenerative Medicine (ARM) on Cures’ potential impact on the originate cell field and I have posted that interview beneath.
Note that ARM opposed REGROW, but facilitates Cures. I will post my own perspectives on the potential dangers and benefits of Cures for the stem cell field plus patients in a separate piece most likely later today.
What are the key reasons that PROVIDE supports the Cures Act regenerative medicine provisions?
MW: Four things spring to mind: first, there has been a lot of talk about Japan and other countries which have taken steps to support this field and now with Treatments, the United States does, too. It sends a clear signal in order to researchers and commercial developers that regenerative medicine is really a critical technology that will be supported. Second, it optimizes the particular pathway without reducing FDA standards or authority. 3rd, it also provides for resources and flexibility in this area. Fourth, the particular inclusion of the standards section directing the FDA to do business with stakeholders is very important. The lack of standards has been identified as an barrier by all key stakeholders.
Will there be effects on IND/BLA requirements from Cures?
MW: I don’ t believe this legislation would change those specifically. The issue is to make certain that all stakeholders work together on standards development and that the particular FDA is part of the process. Cures does that.
What about Cures’ pre-approval (meaning the meaning of an “ advanced regenerative therapy” ) versus post-approval requirements?
MW: I think that will FDA will be comfortable with the scientific basis of regardless of whether something meets the definition of an advanced regenerative therapy. Furthermore, the patient’ s condition has to be serious or life-threatening. It was written pretty carefully.
What does “ real-world evidence” mean and is the particular FDA on board with that?
MW: It means using sources like patient registries and other information collected outside the formal clinical trial process. ARM a new series of conversations with FDA about the use of real world proof and when it makes sense to include it as part of the review process. In the event that Cures passes, the agency still has to approve the usage of real world evidence in specific circumstances and it can state “ yes” or “ no . ”
Do you think it’ ll pass? Obama indication it?
MW: I think it’ ll pass, and I believe if it does, that Chief executive Obama will sign it.