The FDA held a two-day stakeholder meeting on September 12 and 13 to review four draft Guidances the Agency wants to finalize. In this post, and several upcoming posts, I will review the comments of individuals who wanted to have their voices heard at the Workshop. The speakers were to comment on the following four draft Guidances (in the order they were issued):
Same Surgical Procedure Exception(opens in a new tab) (October, 2014)
Minimal Manipulation(opens in a new tab) (December, 2014)
Adipose Tissue Uses and Abuses(opens in a new tab) (December, 2014)
Homologous Use(opens in a new tab) (October, 2015)
Each of the draft Guidances refer to a law known as 21 CFR 1271, which was published in the Federal Register on January 19, 2001. So, the law of the land in dealing with certain types of human-derived materials useful for regenerative medicine has been on the books since 2001, with a modification in 2005.
This is what the FDA published as a description of the Workshop (in part):
The purpose of the public hearing is to obtain comments on the four draft guidance documents relating to the regulation of human cells, tissues, and cellular and tissue-based products (HCT/Ps) from a broad group of stakeholders, including tissue establishments, biological and device product manufacturers, health care professionals, clinicians, biomedical researchers, and the public. For example, FDA would like comments on the scope of the four draft guidances, including the particular topics covered, the particular questions posed, whether there are additional issues for which guidance would be helpful, and whether FDA’s recommendations for each topic are sufficiently clear and consistent within and across documents to provide meaningful guidance to stakeholders. In addition, FDA welcomes comments that will enhance the usefulness and clarity of these documents. FDA recommends that comments exclude discussion of products that do not meet the definition of an HCT/P, such as platelet rich plasma and other blood products. FDA will consider information it obtains from the public hearing in the finalization of the four draft guidance documents.
The video casts of the two-day Workshop are available here(opens in a new tab). Eventually, the FDA will post transcripts as well.
Despite the overwhelming requests for an opportunity to speak during the Workshop, the FDA decided to allocate a 5-minute block of time for a speaker of a single entity (e.g., a company or an academic institution) and an 8-minute block of time for presenters of multiple stakeholder organizations (e.g., the International Society of Cell Therapy). Turned out there were more than 90 speaking slots allocated, and each of the videocasts last longer than four hours.
On Day One of the Workshop, we learned that the FDA participants on the panel constituted an informal team, and that the “rules of evidence” didn’t apply. I thought it was just going to be a sharing of opinions, with a brief Q&A after each segment of presenters, but apparently, the FDA wanted everyone to know that what was being said by Agency personnel was not official, so please don’t get the impression that their comments could be used against the FDA in a future legal matter.
It has been a challenge for me to settle on an approach to summarizing the great diversity of comments, but one thing I won’t do is give a brief summary of the comments of each speaker, since there were just too many speakers. However, I think it would be useful to present an overview of the themes that emerged from the speakers, in order to highlight perceived issues associated with the draft Guidances.
Keep in mind, the FDA held the Workshop to gather comments from the speakers about the utility of the draft Guidances themselves as they move the process forward to finalize the language in the Guidances. For example, some of the FDA panel members asked specific speakers for examples of what they had commented on, which would allow the FDA to better address the concerns expressed. And then there were the comments from some of the speakers that essentially indicated that the FDA would destroy the field of regenerative medicine and cripple innovation by industry if the Guidances were ever issued. I am not sure how the FDA will handle those comments as they move toward issuing the final Guidances.
A fair number of speakers included in their remarks specific recommendations on adjustments to language of the draft Guidances. For example, the Chief Biotechnology Officer and Head of Scientific Strategy and Policy at J&J, Jay Siegel, indicated that the FDA needs to update its polices, based on the lessons they have learned through the years. Specifically, he said that Section 1271.15(b) (covered by the draft Guidance on the same surgical procedure exception) needs to be broaden, since surgical facilities already operate in a way that minimizes the threat of communicable diseases—the main reason the Federal Government established the FDA in the first place. The speaker believes that minimally manipulated HCT/Ps when made in surgical facilities already have sufficient safeguards in place, such that additional scrutiny by the FDA is redundant. And likewise, for more than minimally manipulated HCT/Ps, Mr. Siegel suggests that the FDA should consider other regulatory pathways that apparently don’t involve forcing multi-billion dollar corporations like J&J to pony up tens of millions of dollars to demonstrate the safety and efficacy of what the FDA considers to be biologic drugs. Now, that is what I call a reform, but I am not sure who will benefit the most from the suggested changes.
The other problem highlighted by Mr. Siegel’s comments is that he speaks only about one type of facility where translational cell therapy could occur: the surgical facility. He states that this type of facility already is accredited, which means that they operate to prevent the spread of infectious agents. While I am sure J&J would never associate itself with an operator of a strip mall-based treatment center, translational cell therapy certainly will be offered in such non-surgical facilities. Consequently, the position that Mr. Siegel takes—different rules for different folks/facilities—would lead to an enforcement nightmare.
I will continue with my review of the highlights of the presentations in the next post.
