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Regulatory Update, November 2016

Tuesday, November 1, 2016

Proposed Changes to NIH Guide­lines for Human Stem Cell Research

In the August 5, 2016 Federal Register, the NIH requested public comment on a proposal to amend Section IV and Section V of the NIH Guidelines for Human Stem Cell Research and on the proposed scope of certain human-animal chimera research that will be considered internally by an NIH steering committee to provide program­matic input to the director of the relevant NIH Institute(s) or Center(s) or equivalent NIH officials responsible for funding deci­sions. The comment period was extremely brief, ending on September 6, 2016.

Since the NIH Guidelines were issued in 2009, growing knowledge and advance­ment of stem cell biology has created new research opportunities. Some scientists are exploring strategies for growing human tis­sue and organs in animals through the in­troduction of human pluripotent cells into early stage embryos of non-human verte­brate animals. These experimental designs raise questions regarding where the human cells might go in the developing animal and how they might function, such as whether the human cells might contribute to the central nervous system and affect the cog­nition of the animal.

In 2015, while considering these issues, the NIH issued a funding moratorium. It also held a workshop with experts on No­vember 6, 2015, to review the state of the sci­ence and discuss animal welfare issues. The workshop illustrated that while there are significant challenges to creating chimeric models, there is clear interest and potential in producing animal models with human tissues or organs for studying human devel­opment, disease pathology and eventually organ transplantation.

The NIH has decided to establish a steer­ing committee to provide input to the rel­evant NIH Institute(s) or Center(s) on hu­man-animal chimera research proposals. It will consider factors such as (1) the charac­teristics of the human cells to be introduced (including potency and any modifications of those cells); (2) characteristics of the recipi­ent animal (e.g., species, stage of develop­ment, and any modifications that affect lo­cation or function of human cells); (3) other data relevant to the likely effects on the ani­mal (e.g., changes in cognition, behavior or physical appearance); (4) planned monitor­ing (including animal welfare assessments); and (5) any staging of proposed research (e.g., assessing the outcome of a particular experiment before conducting a further experiment). This internal programmatic work will be conducted independent of, and in addition to, the usual peer review proce­dures for research at the NIH.

The NIH also proposes to revise the Guidelines to expand the existing prohi­bition on introducing human pluripotent stem cells into blastocyst stage nonhuman primate embryos to include pre-blastocyst stage nonhuman primate embryos; and to expand the prohibition on research involv­ing the breeding of animals where the intro­duction of human embryonic stem cells or human induced pluripotent stem cells may contribute to the germ line to include any human cells that may result in the forma­tion of human gametes. Interested readers should see the notice for a detailed summa­ry of the proposed changes for which com­ments are solicited.

FDA Final Guidance on Medical De­vice Clinical Study Adaptive Designs

In the July 27, 2016 Federal Register, the FDA announced the availability of a final guidance document titled “Adaptive De­signs for Medical Device Clinical Studies.” This guidance provides sponsors with guid­ance on how to plan and implement adap­tive designs for clinical studies when used in medical device development programs. An adaptive design for a medical device clinical study is defined as a clinical trial design that allows for prospectively planned modifications based on accumulating study data without undermining the trial’s integ­rity and validity. Adaptive designs, when properly implemented, can reduce resource requirements and/or increase the chance of study success.

The document provides guidance on how to plan and implement adaptive designs for clinical studies when used in medical de­vice development programs. It addresses adaptive designs for medical device clini­cal trials and is applicable to all medical device marketing submissions, including investigational device exemption (IDE) submissions. This guidance can be applied throughout the clinical development pro­gram of a medical device, from feasibil­ity studies to pivotal clinical trials. It does not apply to clinical studies of combination products or co-development of a pharma­ceutical product with an unapproved diag­nostic test.

Interested parties may submit electronic or written comments on final guidance at any time. FDA may consider those com­ments for future revisions. Submit electron­ic comments at http://www.regulations.gov/. Submit written comments to the Di­vision of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fish­ers Lane,

Room 1061, Rockville, MD 20852. Iden­tify comments with Docket No. FDA-2015-D-1439. The FDA also has instructions on how to submit confidential information. If those instructions are not followed, any submitted confidential information will be made public.


The Regulatory Update is excerpted from Re­search Practitioner, Volume 17, Number 05, September-October 2016.

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