Significant Efforts Underway to Lay the Foundation for Standards Development in Regenerative Medicine Therapies

U.S. Food and Drug Administration selects Nexight Group, LLC to lead a contract to Recommend Practices for Standards for Regenerative Medicine Therapies

Washington, D.C. (October 25, 2017) – On September 27, 2017, the U.S. Food and Drug Administration awarded a one-year contract to Nexight Group to engage with experts to recommend processes and outline a strategic plan for developing standards in regenerative medicine and advanced therapies. The Standards Coordinating Body for Gene, Cell, and Regenerative Medicines and Cell-based Drug Discovery (SCB) has partnered with Nexight to provide subject matter technical support for this effort. This work will create a foundation that allows industry, regulatory authorities, and other stakeholders to reduce barriers to regenerative medicine research and product development and accelerate the market readiness of these life-changing medical treatments.

The 21st Century Cures Act, signed into law on December 2016, directs the U.S. Food and Drug Administration, National Institutes of Standards and Technology, and industry stakeholders to “coordinate and prioritize the development of standards and consensus definition of terms... [that] support, through regulatory predictability, the development, evaluation, and review of regenerative medicine therapies and regenerative advanced therapies.”[1] Jiwen Zhang, SCB’s President, commented, “This provision in the Act is consistent with the mission of the SCB to improve product development, manufacturing, and analytical techniques to help bring innovative regenerative medicine products to market to help patients. Therefore, SCB is pleased to have the opportunity to partner with Nexight in implementation of this provision.”

Regenerative medicine therapies present unique challenges related to product testing, scientific protocols, product quality and specifications, performance characteristics, and compliance criteria. Thus, advancing the development and use of standards has the potential to:

  • Transform product development by inspiring innovation and focusing expertise where it is needed most;
  • Contribute to regulatory predictability so that investment and research is catalyzed and safe therapies are brought to market in a timely manner; and
  • Facilitate the overall development of promising therapies that can cure or treat many conditions and diseases that are not currently easily treatable through conventional means.

Ross Brindle, Nexight Group’s Chief Executive Officer said, “Nexight Group is honored to be selected by FDA to conduct this important work. Engaging the regenerative medicine scientific community to inform standards development has great potential to get novel, life-changing therapies to patients who need them both quickly and safely.”

Between now and September 2018, Nexight Group and the SCB will work together to engage the community to gain a clear picture of the current regenerative medicine standards landscape and recommend an efficient standards development process that incorporates stakeholder input. As part of this work, the Nexight-SCB team will:

  • Develop a landscape report that outlines business needs, gaps in standards, and promising therapy areas for standards application in regenerative medicine therapies.
  • Host three webinars for community stakeholders, including researchers, product developers, academic institutions, clinicians, professional societies, and government agencies on standards development. Stand by for information on the first of the webinars in December 2017.
  • Recommend processes and criteria for identifying and prioritizing standards that will have a high impact on the quality and safety of regenerative medicine therapies, and identify those factors that affect the feasibility and cost-benefit of certain standards.

With Nexight Group’s proven processes for engaging diverse experts in discussions about gaps and needed solutions to advance scientific fields, and the technical acumen and industry involvement of the SCB, the Nexight-SCB team provides both an unparalleled understanding of the work required and the highly developed technical approach necessary to successfully support this promising industry. While working with key regenerative medicine stakeholders throughout this project, we aim to help lay the foundation for a compliance environment that encourages growth and innovation in a safe and consistent manner.

The Nexight-SCB team will be engaging with community members throughout the project to provide input and validate perspectives. If you are not already engaged in this field as a member of the SCB, please contact kchappell@nexightgroup.com and agetz@regenmedscb.org to be included in the outreach. 


About the Standards Coordinating Body

The Standards Coordinating Body (SCB) is a non-profit consortium of non-government stakeholders that operates through public-private partnerships with government agencies, regulatory bodies, and other government organizations involved in establishing consensus standards for regenerative medicine and other advanced therapy products. The SCB’s members include industry representatives, professional societies, and government and academic entities working together to support standards development. The SCB’s mission is to efficiently and effectively support and improve the cost, time, and resources used in regenerative medicine product development by creating a more harmonized compliance environment. For more information on the SCB, visit standardscoordinatingbody.org.

About Nexight Group

Nexight Group, LLC is a small business based in Silver Spring, Maryland, with extensive experience engaging stakeholders, establishing new collaborative organizations, developing roadmaps and strategic plans, establishing new business processes, and supporting technical education and outreach efforts for experts in diverse industries, including energy, materials and manufacturing, infrastructure security and resilience, and health and development. In collaboration with Georgia Tech and the Georgia Research Alliance, Nexight Group led the coordination and development of Achieving Large-Scale, Cost-Effective, Reproducible Manufacturing of High-Quality Cells: A Technology Roadmap to 2025, a roadmap that aims to facilitate widespread access to life-changing cell therapies, engineered tissues, medical devices, and drug discovery and testing platforms through collaborative research and development. For more information on Nexight Group, visit nexightgroup.com

 [1] Public Law 114-255

CRISPR Standards

With the ever-expanding use of CRISPR technology, the development of standards to quantitatively benchmark on- and off-target activity needs to keep pace.

NATURE METHODS / EDITORIAL

Nature Methods 14, 541 (2017) | doi:10.1038/nmeth.4328
Published online 30 May 2017

 

Newly Created Standards Coordinating Body Seeks to Advance Development and Establishment of Regenerative Medicine Standards

WASHINGTON, DC--(Marketwired - Jan 19, 2017) - The international Standards Coordinating Body for regenerative medicines (SCB), in coordination with the Alliance for Regenerative Medicine (ARM), today announced its formal launch as a public-private partnership for coordinating, prioritizing and supporting standards that advances process, measurement and analytical techniques to support the global availability of products across the gene and gene-modified cell therapy, cell therapy, cell-based drug discovery, tissue engineering and biomaterials sectors.

"The SCB's long-term mission is to efficiently and effectively support sector standards development to accelerate product development and scalability, and streamline regulatory submission review and approval," said Dr. Robert A. Preti, Chairman of ARM; President of PCT, a Caladrius Company; and CTO & SVP of Manufacturing & Technical Operations at Caladrius Biosciences.

The SCB brings together product developers, tools and service providers, professional societies, government entities and academic centers for the purpose of supporting standards development through coordination, prioritization, resource compilation, inter-laboratory data generation, joint-participation in consensus Standards Development Organization (SDO) and other standards setting organization activities, and education and implementation of standards.

"Cell and gene therapy product developers and other companies in the regenerative medicine sector have been working to establish meaningful standards for the past 20 years," said Jessica Carmen, SCB Officer and Board Member and Director of Business Development, Cellular Therapies for MaxCyte. "Creation and launch of the SCB is a major milestone toward that effort."

The SCB builds upon Section 3036 of the 21st Century Cures Act, which calls for the coordination and prioritization of standards creation to support the development, evaluation and review of regenerative medicine and regenerative advance therapies.

"Standards development and technological advancements, including pre-standards activities, will support a more uniform product development and regulatory environment," added Michael Mendicino, Ph.D., Chair of the SCB Board of Directors and Owner of Hybrid Concepts International, LLC. "This will also assist in future international harmonization efforts."

In support of these efforts, the SCB aims to build a coalition that will include partnerships with federal agencies, international standards and other organizations, industry and other public and private sector entities.

On September 19, 2016, the SCB announced its first public-private partnership via a Memorandum of Understanding with the National Institute of Standards and Technology's (NIST) Material Measurement Laboratory (MML).

Through this partnership, NIST also holds an advisory position on the SCB Board of Directors. Other SCB launch Board members include representatives from: Akron Biotech, Axiogenesis, Biogen, Cellular Dynamic International, GE Healthcare, GSK, Editas, MaxCyte, AxoGen, Histogenics and Brammer Bio; academic institutions such as Northwestern University, AABB, the Cell Therapy Manufacturing Consortium and Wyss Zurich; and other stakeholders including ARM, ASGCT and ISCT.

"We are delighted to be a founding member of this extremely important effort," said Elena Kfoury, President of the SCB Board of Directors and General Manager (U.S.) for Axiogenesis. "The regenerative medicine and regenerative advanced therapies sector is rapidly maturing and the development of standards serves as a significant step forward in ensuring timely clinical development, commercial availability and ensuing market adoption of these novel treatments."

The SCB, established as a 501c3 organization, was originated by the Alliance for Regenerative Medicine's Science and Technology Committee.

About The Standards Coordinating Body
The Standards Coordinating Body for regenerative medicines (SCB) is a multi-stakeholder consortium representing varied interests across the gene and gene-modified cell therapy, cell therapy, cell-based drug discovery, tissue engineering and biomaterials sectors. The SCB is a non-profit organization working to support the development, dissemination, education and implementation of relevant standards and related deliverables via public-private partnerships and ongoing work with national and international consensus standards and other organizations. The SCB will serve as a source of information, knowledge, experience and data collection related to process, material and reference standards to enable more efficient and successful development, manufacture and testing of advanced therapies and cell-based drug discovery. To learn more about the SCB, visit http://www.regenmedscb.org.

About The Alliance for Regenerative Medicine
The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 250 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

SCB and NIST to Work Together to Advance Development and Establishment of Industry-Wide Standards

Standards Coordinating Body for Regenerative Medicines and the National Institute of Standards and Technology Enter into a Memorandum of Understanding to Advance Development and Establishment of Industry-Wide Standards

WASHINGTON, DC, September 19, 2016 – The Standards Coordinating Body for Gene, Cell and Regenerative Medicines and Cell-based Drug Discovery (SCB) announced today it has entered into a memorandum of understanding (MOU) with the National Institute of Standards and Technology’s (NIST) Material Measurement Laboratory (MML) to advance the development of industry-wide standards for cell therapy, gene therapy, regenerative medicine and cell-based drug discovery.

This MOU signifies the first step toward establishing an SCB-NIST public-private partnership. The SCB aims to build a coalition that will include other partnerships with federal agencies and international organizations. The initiative to establish the SCB originated from the Alliance for Regenerative Medicine’s (ARM) Science and Technology Committee.

Signed by Laurie Locascio, MML director, and Morrie Ruffin, ARM’s managing director and a founding director of the SCB, the MOU describes the first formal collaboration between SCB and NIST.

“The SCB and NIST are united in our dual missions to enable more efficient and successful clinical and commercial development for cell and gene therapies, regenerative medicine and cell-based drug discovery,” said Morrie Ruffin, ARM’s managing director. “Widespread coordination, establishment and implementation of standards for these products is essential to driving our field forward and speeding patient access to these potentially transformative therapies.”   

According to the terms of the MOU, the SCB and NIST will work together to advance the field by collaborating on and contributing to the development of relevant consensus standards and among other activities, improving confidence in measurements of gene-based and cellular systems and their manufacturing processes by engaging key stakeholders to better understand and address industry priorities.

NIST is a non-regulatory federal agency within the U.S. Department of Commerce whose mission is to promote U.S. innovation and industrial competitiveness by advancing measurement science, standards and technology. The SCB will work with NIST’s MML, which serves as the national reference laboratory for measurements in the chemical, biological and material sciences. NIST engages in ongoing activities of standards development organization leadership and developing capabilities in measurement science.

The SCB, a multi-stakeholder consortium representing varied interests across this sector, is a non-profit organization working to support the development, dissemination, education and implementation of relevant standards and related deliverables via public-private partnerships and ongoing work with national and international consensus standards organizations. The SCB will serve as a source of information, knowledge, experience and data collection related to process, material and reference standards to enable more efficient and successful development, manufacture and testing of advanced therapies and cell-based drug discovery. 

For more information about ARM, please visit www.alliancerm.org. For more information about NIST, please visit www.nist.gov. For more information about the SCB, please visit www.regenmedscb.org

About The Alliance for Regenerative Medicine
The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 250 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org.