Comments on: FDA issues Complete Response Letter to Fibrocell Science BLA http://regenmedguru.com/fda-issues-complete-response-letter-to-fibrocell-science-bla The Knowledge Portal to Help Regenerative Medicine Products Reach the Clinic Sooner Sun, 05 Jun 2011 16:36:47 +0000 hourly 1 http://wordpress.org/?v=3.2.1 By: Darin http://regenmedguru.com/fda-issues-complete-response-letter-to-fibrocell-science-bla/comment-page-1#comment-41 Darin Tue, 09 Feb 2010 19:50:14 +0000 http://regenmedguru.com/?p=147#comment-41 Hi Nancy: Thank you for sharing your insights. I know you have a wealth of experience in this area. My experience as a consultant is that developers frequently fail to challenge assumptions and most importantly verify the validity of their assumptions with regulators. I think many hesitate to ask regulators questions, not just those concerning safety, because they are rightly concerned that the response from the regulators can cost them both time and money. However, as you know, asking regulators questions which are put in context with relevant data, can frequently result in a reasonable and thoughtful response. Whether this occurred with Fibrocell/Isolagen is not known. Hopefully, others working with cell-based regenerative medicine product can take some lessons from this. Thanks again for sharing your perspective. --Darin Hi Nancy:

Thank you for sharing your insights. I know you have a wealth of experience in this area. My experience as a consultant is that developers frequently fail to challenge assumptions and most importantly verify the validity of their assumptions with regulators. I think many hesitate to ask regulators questions, not just those concerning safety, because they are rightly concerned that the response from the regulators can cost them both time and money.
However, as you know, asking regulators questions which are put in context with relevant data, can frequently result in a reasonable and thoughtful response. Whether this occurred with Fibrocell/Isolagen is not known. Hopefully, others working with cell-based regenerative medicine product can take some lessons from this.

Thanks again for sharing your perspective.

–Darin

]]> By: Nancy Parenteau http://regenmedguru.com/fda-issues-complete-response-letter-to-fibrocell-science-bla/comment-page-1#comment-40 Nancy Parenteau Mon, 08 Feb 2010 15:01:37 +0000 http://regenmedguru.com/?p=147#comment-40 What I don't quite understand is why companies continue to miss fundamentals in their cell therapy submissions. The autologous fibroblast product should be fairly straightforward. Perhaps one problem is that companies don't realize that process validation and quality assurance can be as or more challenging when you are working with patient samples. Genzyme certainly learned this with Carticel and they also set the BLA bar for autologous therapies high. The perception that autologous cells are inherently safer biologically, is just that, perception. Once you get passed the possibility of disease transmission, immune reaction with the use of allogeneic cells, the possibility of something untoward happening during the in vitro process is the same. In fact, one might argue that the inability to rigorously test autologous samples for biological stability, could put be a risk not present in the use of allogeneic cell banks. Of course, how much these things are issues depends on the process. Patient histories may be important as well. I agree that delay because of the request for biopsy data and CMC concerns could be significant. At the end of the day, sponsors do have to take a proactive role in supporting every aspect of their product before the eleventh hour. As you point out, meeting with the FDA can be an important part of that. What I don’t quite understand is why companies continue to miss fundamentals in their cell therapy submissions. The autologous fibroblast product should be fairly straightforward. Perhaps one problem is that companies don’t realize that process validation and quality assurance can be as or more challenging when you are working with patient samples. Genzyme certainly learned this with Carticel and they also set the BLA bar for autologous therapies high.

The perception that autologous cells are inherently safer biologically, is just that, perception. Once you get passed the possibility of disease transmission, immune reaction with the use of allogeneic cells, the possibility of something untoward happening during the in vitro process is the same. In fact, one might argue that the inability to rigorously test autologous samples for biological stability, could put be a risk not present in the use of allogeneic cell banks. Of course, how much these things are issues depends on the process. Patient histories may be important as well.

I agree that delay because of the request for biopsy data and CMC concerns could be significant. At the end of the day, sponsors do have to take a proactive role in supporting every aspect of their product before the eleventh hour. As you point out, meeting with the FDA can be an important part of that.

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