The 21st Century Cures Act has some important provisions that will impact the stem cell and regenerative medicine industry. What is driving these efforts is a widespread feeling that the status quo of regenerative medicine oversight isn’ capital t working very well.
I’ meters talking with key stakeholders and still trying to sort out the thoughts on the stem cell provisions in Cures. Exactly what would it mean? Cures’ language on stem cellular material is nuanced and its provisions also are not so black and white since REGROW was in my view.
You can see the interview with ARM’ s Executive Director Michael Werner on Cures here . ARM facilitates Cures. ISSCR issued a statement today on Cures, which is upbeat but tensions the importance of data. The statement is not a clear thumbs up or even down. Earlier this year, both ARM and ISSCR opposed REGROW.
Overall, there are both potential risks plus benefits to Cures’ stem cell provisions, which I talk about below. Note that this post is focused primarily on the stem cell-related provisions of Cures. There is ambiguity in terms of how Treatments will affect NIH overall or have broader outcomes for your life sciences.
Potential upsides in order to Cures for stem cell research. A single possible benefit of the Cures regenerative medicine provisions is the fact that some high quality stem cell therapies that ultimately is going to be proven safe and effective would likely get to patients more quickly and assist patients sooner. That’ s a big deal. For biotech the main plus would be that they will have to spend less money to get remedies further in the trial pipeline. Since we as an industry need stem cell biotechs to be successful in order for all of us jointly to help the most patients, the benefit for biotechs would likely produce some benefit for the field. Another possible plus right here with Cures is the focus in the language on the requirement for standards for regenerative medicine, which have been lacking in the field.
Possible risks. A major worry is that some stem cell therapies that are not looking forward to primetime may move forward anyway into patients and possibly harm them, depending on how the Cures language translates into real changes in oversight. The “ real world evidence” supply in Cures is causing some unease as it might be at the expense of the gold standard requirement of data through RCTs. There is also uncertainty over the stem cell products provisions in Cures, which some view as not really strong enough. Finally, my impression is that too much of exactly what in the way of data that would today be required at the pre-approval degree, would be shifted to post-approval under Cures, which could considerably increase risks for patients. ISSCR places weight to the need for rigorous data as you can see from well this a part of their statement:
“ Of extremely important concern is that the streamlined process for FDA review plus approval outlined in the bill should not compromise the demanding review of scientific and clinical evidence required to ensure the particular safety and efficacy of regenerative therapies”
I strongly agree with this statement.
What qualifies as an advanced regenerative therapy? One of the most important stem cell-related provisions in Remedies is the third criterion for definition of a product as an sophisticated regenerative therapy that qualifies for acceleration:
“ preliminary clinical evidence indicates that the medication has the potential to address unmet medical needs intended for such a disease or condition. ”
Who decides on this evidence and how? It’ s not clear. The word “ preliminary” could be an issue here in terms showing how it is interpreted.
Senator Elizabeth Warren is one of the fiercest opponents of Cures general and she specifically weighed in with the girl objections to the stem cell provisions:
“ Warren criticized provisions designed to speed authorization for stem cell therapies. Referring to a major Republican subscriber who would stand to benefit from the provisions, “ This megadonor has poured millions of dollars into Mitch McConnell’ s individual campaign coffers and into his Republican super PAC, and now he wants his reward. So the Cures react offers to sell government favors. ”
Overall gestalt. My feeling right at this moment is that if the 21st Century Cures Respond passes with the current stem cell provisions that it may kick off a high-risk, potentially high-reward regulatory experiment upon regenerative medicine here in the U. S. It’ s i9000 unclear if on the whole there would ultimately be more advantage or harm to patients and the field. We don’ t know the results of experiments in advance, right? Another main factor in how this plays out will be how the FOOD AND DRUG ADMINISTRATION more broadly fares under the new administration.
The bottom line is that for me at this time I can’ t actually say I strongly oppose or support the Remedies stem cell provisions because there is so much uncertainty about how it could all unfold and as discussed above there are potential advantages and risks. My initial impression is that there are considerably more potential risks than potential benefits, but it’ s not very clear cut. What do you think?