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Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products Proceedings of a Workshop (2019) / Chapter Skim
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6 Exploring Variability and Its Impact on Product Regulation and Outcomes
Pages 71-78

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From page 71...
... has several programs to help expedite product development and review, including the Regenerative Medicine Advanced Therapy designation and the INTERACT program. Early interaction with FDA is encouraged.
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From page 72...
... In this session, speakers explored objective metrics and reliable approaches to interpreting the outcomes of clinical trials for regenerative engineering therapies. Speakers also considered how variability in regenerative engineering products can affect the regulatory approval pathway.
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From page 73...
... Christman confirmed that there is endogenous infiltration and increased cardiac muscle, but she said that the type of study proposed by the participant has not been done for the heart. An analogous material derived from porcine skeletal muscle was studied in a mouse model for its ability to deliver askeletal myoblasts, she said, and the data from it suggested that the material increased cell survival over simply injecting cells in liquid suspension.
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From page 74...
... . The affected pathways included cell death, hypertrophy, immunomodulatory response, metabolic processes, blood vessel development, and heart development.
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From page 75...
... . VARIABILITY OF REGENERATIVE ENGINEERING PRODUCTS AND THE REGULATORY APPROVAL PATHWAY A broad variety of regenerative medicine products exist, Marks said, from bioengineered skin, blood vessels, and bladders to CAR T cells.
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From page 76...
... He said that much of the downstream time and effort could be saved if everyone manufacturing CAR T cells at academic institutions could agree on "early phase manufacturing processes that were commensurate with being transferred to commercial scale processes." Referring to the molecular cloning laboratory manual first published in 1982 by Tom Maniatis, he suggested that a similar core resource is needed for regenerative medicine. There are also challenges in the clinical development of complex therapies and in working with small patient populations.
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From page 77...
... In closing, Marks said, "FDA is committed to advancing the development and evaluation of regenerative medicine products." The agency wants to help developers move through the process as quickly as possible, while still ensuring safety and efficacy, he said, and he encouraged product developers to reach out to the agency for early discussions about their products. FDA can, for example, help individualize product development, overcome manufacturing limitations, and provide input on novel endpoints and innovative trial designs.
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Key Terms

  • medicine products
  • regenerative engineering
  • regulatory approval
  • engineering products
  • clinical evidence

  • approval pathway
  • heart failure
  • clinical trials
  • advanced therapy
  • regulatory pathway

  • clinical development
  • medicine therapies
  • immune response
  • interact program
  • complex mechanism

  • address unmet
  • patient populations
  • increased cardiac
  • receiving ventrigel
  • animals receiving

  • expedited programs
  • accelerated approval
  • rmat designation
  • cardiac muscle
  • failure patients

  • therapy designation
  • injectable myocardial
  • extracellular matrix
  • matrix hydrogel
  • myocardial infarction


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