One of the hottest topics in the world of stem cells this year has been the REGROW ACT and at a larger level the movement for changes in 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 stirring much discussion today. The bill is called Cures for short and you can read the latest draft here.
Cures may get a vote in Congress as early as tomorrow so this is a big week for the stem cell field and the life science more generally in the U.S. See sections 3033-3036 of the bill as those are on regenerative medicine.
I’m reaching out to a number of stakeholders for their perspectives. Earlier today I interviewed Michael Werner (MW), the Executive Director of the Alliance for Regenerative Medicine (ARM) on Cures’ potential impact on the stem cell field and I have posted that interview below.
Note that ARM opposed REGROW, but supports Cures. I will post my own perspectives on the potential risks and benefits of Cures for the stem cell field and patients in a separate piece most likely later today.
What are the key reasons that ARM supports the Cures Act regenerative medicine provisions?
MW: Four things come to mind: first, there has been a lot of talk about Japan and other countries that have taken steps to support this field and now with Cures, the United States does, too. It sends a clear signal to researchers and commercial developers that regenerative medicine is a critical technology that will be supported. Second, it optimizes the pathway without reducing FDA standards or authority. Third, it also provides for resources and flexibility in this area. Fourth, the inclusion of the standards section directing the FDA to work with stakeholders is very important. The lack of standards has been identified as an obstacle by all key stakeholders.
Will there be effects on IND/BLA requirements from Cures?
MW: I don’t think this legislation would change those specifically. The issue is to make sure that all stakeholders work together on standards development and that the FDA is part of the process. Cures does that.
What about Cures’ pre-approval (meaning the definition of an “advanced regenerative therapy”) versus post-approval requirements?
MW: I think that FDA will be comfortable with the scientific basis of whether something meets the definition of an advanced regenerative therapy. Also, 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 FDA on board with that?
MW: It means using sources like patient registries and other data collected outside the formal clinical trial process. ARM had a series of conversations with FDA about the use of real world evidence and when it makes sense to include it as part of the review process. If Cures passes, the agency still has to approve the use of real world evidence in specific circumstances and it can say “yes” or “no.”
Do you think it’ll pass? Obama sign it?
MW: I think it’ll pass, and I believe if it does, that President Obama will sign it.