Workshop Summary and Materials: Realizing the Benefit of 21st Century Cures through Standards Development

A Workshop Convened by FDA, NIST, SCB, and Nexight Group on March 18 - 19, 2019 at the National Cybersecurity Center of Excellence (NCCoE) in Rockville, MD


This two-day workshop featured updates from Standards Development Organization (SDO) representatives and industry organizations on the benefits of standards and of participating in the standards development processes. Participants also had the opportunity to learn about current regenerative medicine standards efforts that need community support and participate in discussions to support their advancement.

DAY 1 — MATERIALS & session summaries:

Welcome: Collaborations on Standards for Regenerative Medicine TherapiesCelia Witten, Ph.D., M.D., Deputy Director, FDA Center for Biologics Evaluation and Research (CBER) spoke about the close collaboration between CBER and NIST in implementing the 21st Century Cures Act’s provisions for accelerating medical product development in regenerative medicine through the use of standards.

Keynote: Changing the Course of Human Health through Bold Pursuits in Science — Chris Wiwi, Ph.D., CAR-T Technical Commercialization Lead, Celgene Corporation spoke about the unique challenges of moving regenerative medicine products such as the Celgene bb2121 CAR-T product from Phase 1 clinical trials toward commercialization and how standards can promote manufacturing efficiencies to smooth this process.

Building the Foundation for Regenerative Medicine Innovation through StandardsJudith Arcidiacono, M.S., International Regulatory Expert, Standards Liaison, Office of Tissues and Advanced Therapies, FDA/CBER provided definitions of common standards terminology, spoke about the benefit of standards in regenerative medicine, and gave an overview of the standards development process.

The Standards Evolution of Regenerative MedicineSheng Lin-Gibson, Ph.D., Chief Biosystems and Biomaterials Division, NIST provided an overview of the historic landscape of regenerative medicine standards development and also detailed the “who, what, when, where, and why” of standards development.

Panel: “Fireside Chat” – Documentary Standards and Reference Materials — Gordon Gillerman, NIST; Malcolm Moos, FDA; Scott Colburn, FDA; John Elliott, NIST (moderator). Panelists answered questions about documentary standards and reference materials on topics including the biggest misconceptions in standards development, differences between the various types of standards, and how development teams can avoid partial FDA recognition for standards.

Topic: Starting with Success in Mind

  • Case Study: Counting Colony Forming UnitsGeorge Muschler, M.D., Orthopedic Surgeon, Cleveland Clinic discussed his experiences creating an automated colony counting system standard for stem cells in tissue to account for variability in samples.

  • Case Study: OsteoinductivityAlyce Linthurst Jones, Ph.D., Director of Cardiovascular Product Development, LifeNet Health described her experience helping to create an ASTM standard on in vivo evaluation of osteoinductive potential for materials containing demineralized bone.

  • Panel Discussion — Alyce Linthurst Jones, LifeNet Health; George Muschler, Cleveland Clinic; Claudia Zylberberg, Akron Biotech; Chris Wiwi, Celgene (moderator). The panelists answered questions on their experiences working on standards, with topics including hurdles for getting the field to buy into a standard, what can be done early in development to help ease a standard’s adoption for a broad audience, and whether it is necessary to create a new standard when attempting to expand one to other applications.

Topic: Moving from Idea to Innovation

  • Case Study: Viability of Cryopreserved TherapiesBrian J. Hawkins, Ph.D., Chief Technology Officer, Pluristyx described his experience working on a standard for identifying optimal cryopreservation approaches to help manufacturers avoid duplication of work and enhance viability outcomes.

  • Case Study: Microscopy Fluorescence IntensityMichael Halter, Ph.D., Biosystems & Biomaterials Division, NIST discussed his experiences working on a standard for imaging measurement of cell therapy products to help drive adoption of more sophisticated imaging technologies.

  • Panel Discussion — Clare Allocca, National Institute of Standards and Technology; Michael Halter, National Institute of Standards and Technology; Brian Hawkins, Pluristyx; Jessica Carmen, Pullan Consulting (moderator). Panelists addressed questions about their experiences in standards development on topics including how to know when you have an idea for a standard, what to do after you get your standard published, and how to keep up with the pace of technology changes when developing a standard.

Breakout Session TOPIC: Kicking Off and Advancing Development

  • Cell Characterization: Two groups discussed the biggest challenges and most important techniques to standardize to increase the effectiveness of cell characterization. Their findings will inform and accelerate the SCB cell characterization standards advancement project.

  • Rapid Microbial Test Methods. Two groups convened to discuss current standard development efforts for rapid microbial test methods (RMTMs). The discussion in these groups will help support and accelerate the SCB-coordinated RMTM standards advancement projects with in-development ASTM International and ISO standards.

How to Get InvolvedRichard McFarland, Ph.D., M.D., SCB President outlined the different ways that participants can get involved in the work of SCB.

Day 2 — MATERIALS & Session Summaries

Keynote: Why Standards are Critical for the Transfer of Novel Technologies from Academia to Clinical UseBarbara Boyan, Ph.D., Dean, College of Engineering, Virginia Commonwealth University (VCU) spoke about the challenges of translating new technologies from academia to clinical use and the critical importance of standards for developing safe and high-quality products.

Panel: Common Questions About Standards, Demystified — Anne Caldas, ANSI; Malcolm Moos, FDA; Wen Bo Wang, Fate Therapeutics; Barbara Boyan, VCU College of Engineering (moderator). Panelists answered audience questions on topics including how to navigate the complex standards landscape, how to incentivize standards work in academia, and how to succeed in international standards development.

The Importance of Reference MaterialsJohn Elliott Jr., Ph.D., Cell Systems Science Group Leader, NIST spoke on the value of reference materials for ensuring confidence in the accuracy, value, and certainty of measurements with high variability.

Topic: Reference Materials

  • Case Study: Human Adenovirus 5 (Ad5)Keith Carson, M.B.A., Founder and Content Chair, ISBiotech spoke about the effort to develop a viral gene vector reference material to help to determine non-toxic dose amounts appropriate for use in clinical trials.

  • Case Study: CD34+ Cell Enumeration StandardKevin Carrick, Ph.D., Director of Global Biologics, U.S. Pharmacopeia (USP) spoke about the effort to develop a reference standard for CD34+ cell enumeration that can be used to assess reagents and ensure correct gating for data acquisition and analysis.

  • Panel Discussion — Kevin Carrick, USP; Keith Carson, ISBiotech; Jean Qiu, Nexcelom Bioscience LLC; John Elliott, NIST; Sowmya Viswanathan, University Health Network; Sumona Sarkar, NIST (moderator). Panelists addressed questions on topics including the most useful applications for reference materials, common barriers to developing reference materials, and how to balance developing materials made for specific purposes with developing materials that have more flexibility across the field.

Breakout Session TOPIC: Priority Standards Advancement Projects

Workshop participants convened into a breakout groups to discuss four potential high-priority standards topics to help SCB determine how standards advancement efforts should move forward and which stakeholders to involve in these efforts.

  • Cell Viability: The group found that cell viability standards would help enable viability assay comparisons, clarify the best ones to use, and indicate when and when not to use assays, as well as allowing SDOs to establish criteria for correct testing and reducing the burden of FDA product reviews.

  • Chain of Custody and Chain of Identity: The group decided that more reliable COC and COI processes would mean manufacturers can quickly and precisely identify and resolve failures in the supply chain, to ensure patients receive their intended therapies and that these therapies were developed and handled correctly.

  • Characterization of Scaffold Materials: The group determined that comparability, lab-to-lab testing validation, and guidance on what to measure where the most impactful benefits of this standard, but the risk of creating too-specific of a standard or a standard that would increase the regulatory burden need to be considered. 

  • Viral Vector Gene Quantification: The group identified potential impacts to stakeholders, including better consistency in dose-determining assays for industry and improved scientific rigor and reproducibility for academia.

Workshop Closing RemarksRichard McFarland, Ph.D., M.D., SCB President