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Pink Sheet – Senate Gene Editing Hearing: Trust In US FDA On Display

Pink Sheet – Senate Gene Editing Hearing: Trust In US FDA On Display

Executive Summary

Discussion on cutting edge medical technology demonstrates the strong reputation FDA has built as a regulator. Witnesses in the gene editing field said FDA is doing a good job – and Congress is likely to leave it at that.


The US FDA’s early approach to regulation of gene editing is “robust” and sufficient to maintain adequate oversight without stifling innovation, academicians and gene editing sponsors testified at a Nov. 14 hearing by the Senate HELP Committee.


The endorsement for FDA’s early regulatory actions in the area came from representatives of three key stakeholder communities: research, bioethics and commercialization.


Editas Medicine Inc. CEO Katrine Bosley stated that FDA has the appropriate authority to oversee gene editing and is exercising it “well and thoughtfully.” She was even more effusive in her prepared testimony in her praise for FDA’s early engagement with the developing industry and the creation of a good atmosphere for commercial development – including the high standards which help to set boundaries for the industry.


Matthew Porteus, Associate Professor of Pediatrics at Stanford University, echoed the sentiments, saying FDA has continued to work well and support researchers like himself. Bioethicist Jeffrey Kahn (Johns Hopkins Berman Institute) added support for FDA’s oversight as “robust.”


The regulatory role of FDA is no longer a hypothetical issue: the hearing came one day after the announcement of the first dosing of a Hunter Syndrome patient with an in vivo gene-editing therapy from Sangamo Therapeutics Inc.


While reports on that event formed the background for the hearing, the event was not specifically discussed by the committee members or witnesses. Still, there is a sense that the new technology is advancing very rapidly from science fiction to (potential) medical practice. (Also see “Gene Editing: A Powerful, Growing Modality In Regenerative Medicine” – Pink Sheet, 27 Nov, 2017.)


It is a testament to FDA’s current reputation with lawmakers that the hearing featured neither calls for the agency to ease up and let innovation flourish – nor to crack down and protect vulnerable patients (and the public) from unproven technology.

FDA Touts Internal Research

FDA itself was not a participant in the hearing. However, during a same day appearance at the Prevision Policy/Friends of Cancer Research Biopharma Congress, Center for Biologics Evaluation & Research Director Peter Marks highlighted the rapid advance of the gene-editing field several times as an example of the challenges FDA must be on top of in the modern regulatory era.


Marks especially highlighted the important played by CBER’s internal research programs in helping keep abreast of the field. “When I first came to the center several years ago, I did not strike me as how important that was,” Marks said. “With the advent of each successive problem that we have had to deal with in a regulatory manner – three-parent babies, CRISPR-Cas9 and single nucleotide gene editing – it has been wonderful to have a group of scientists that do regulatory work but they are also in the field and are able to keep up at the leading edge that is really advancing.”


Marks also used the hype around the field as an example of why the watchdog role of FDA remains critical, noting that the agency has seen “do it yourself” gene-editing kits advertised. “I don’t recommend you go home today and order a CRISPR genome editing kit,” he said. “I do think there’s still a world for regulators out there,” he said. Things “can get very dangerous with molecular genetics.” (Indeed, FDA subsequently issued a notice warning consumers to avoid “do it yourself” gene therapy kits.)


“Like everything else in life there is some balance,” Marks said. “Probably a regulatory sweet spot where we facilitate innovation, may shift so it’s a little on the earlier side and more data on the back side in the real-world setting.”

Effusive Praise For FDA From A Biotech CEO

The Senate hearing suggests that, for now, at least, FDA has found the “sweet spot” with gene editing.


Committee members, starting with the Chair, Lamar Alexander, R-Tenn., duly asked the witnesses if regulatory obstacles exist that are inhibiting research. In general, however, they heard only praise for FDA and its approach to date.


Indeed, Editas CEO Bosley’s remarks were the opposite of the typical tone of comments from regulated industry in a hearing about regulation. “In our experience, the agency’s leaders and scientific reviewers have also demonstrated a strong commitment to understanding the latest breakthroughs and to improving their regulatory science,” she said. “I commend the FDA in particular for their outreach to leading academic and industry experts in genome editing.


“To date, the agency has been forward-looking and thoughtful in starting early conversations about how they plan to integrate oversight of genome editing into their existing regulatory framework,” Bosley said. “As the field of genome editing continues to advance in the years ahead, these kinds of early, constructive, and collaborative engagements will be invaluable in keeping all parties aligned and focused on delivering important medicines to patients.”


The praise for FDA’s approach comes on top of good planning and preliminary policy formation by the agency and scientific community to prepare the public for the fast-developing gene-editing field.


The work by the National Academies of Science, Engineering and Medicine to develop a consensus on gene-editing research in 2016 formed foundation for the testimony on adequate controls. Kahn described the NASEM project as “a recipe for very tight control to allow the benefits to go forward in cases where there really is no other way to achieve a therapy for a particular disease.


Kahn noted the attempt to bring global entities into the discussion around the NASEM project. The NASEM Consensus Committee was a year-long process of partnership between the National Academies in the US, the UK Royal Academy, and the Chinese Academy of Sciences.


Kahn supported the need for finding a balance between having “no rules” and completely stifling innovation. He referred to a decision in Canada to make it a criminal offense to use gene editing tools that could lead to heritable alterations as stifling innovation. Kahn described the United Kingdom’s “permissive regime with very tight controls,” which he deemed the correct approach. “A much smarter approach to policy,” Kahn said “is strict control to allow careful, responsible science to go forward in ways that are controlled and within our borders.”


Several senators picked up on the theme of regulatory and global competitiveness. Sen. Tim Kaine, D-Va., expressed interest in how lawmakers should think about gene editing as a regulatory issue by maintaining “tight control” without driving innovators away.


Senator Tim Scott, R-S.C., supported U.S. leadership in setting the precedents for gene-editing use. “Some nation will set the ethical boundaries for this conversation going forward. It would certainly be helpful for the United States of America to establish those boundaries to a large extent.”…