Using Standards in the Development of Regenerative ......Judith Arcidiacono, M.S. International...
Transcript of Using Standards in the Development of Regenerative ......Judith Arcidiacono, M.S. International...
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Using Standards in the Development of Regenerative Medicine Products
Judith Arcidiacono, M.S.
International Regulatory Expert and Standards Liaison
Office of Tissues and Advanced Therapies
CBER/FDA
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Types of Standards
Reference materials Documentary
standards
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Documentary Standards
• Performance characteristics
• Testing methodology
• Manufacturing practices
• Scientific protocols
• Ingredient specifications
• Data standards
• Terminology/Nomenclature
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Physical Standards/Reference Materials
• Highly characterized reagents that are distributed to assure consistency, quality and safety.
• ISO Guide 35 Reference Materials: Material, sufficiently homogeneous and stable with reference to specified properties, which has been established to be fit for its intended use in measurement
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Standards Development Process
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Key Principles in Consensus Standards Development
1. Standards respond to a need in the market
2. Standards are based on global expert opinion
3. Standards are developed through a multi-stakeholder process
4. Standards are based on a consensus
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When should a standard be developed?
• Does the base of scientific knowledge on the subject support the development of standardized approaches to methods, testing, etc. ?
• Is there consensus among the scientific community that the proposed approaches are appropriate to address the need for standardization?
NEED
KNOWLEDGE
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Other Options When Standardization is not Achievable
• Standard guides
• Terminology
• Practices
• Guidelines
• Publically Available Specifications (PAS)
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Considerations When Drafting a Standard
• What is the scope of the standard?
• What is the purpose of the standard?
• Who will likely use the standard?
• What are the possible intended/unintended consequences of the standard?
• How does the proposed standard affect existing work?
• Are there similar efforts to develop a specific standard in other standards venues?
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Considerations for Developing Reference Materials
• What is the intended use of the material?
• What is the state of the methodology for which the RM will be used? (standardized/validated?)
• Who will be likely use the standard?
• How will inter-laboratory testing be conducted?
• What are the possible intended/unintended consequences of the reference material?
• How does the proposed reference material affect existing work?
• Are there similar efforts to develop a specific standard in other standards venues?
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FDA Standards Activities for Regenerative Medicine Products
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Standards Activities for Regenerative Medicine Products
• Staff act as liaisons to Standards Development Organization (SDO) technical committees
– FDA researchers, reviewers, and policy makers
• FDA/OCTGT Standards Working Group
• Workshop sponsorship and participation
• Laboratory Programs
– Inter-lab round-robin testing (e.g., ISO cell counting, ASTM Demineralized Bone Matrix standard)
– Collaborations with NIST (e.g., flow cytometry, viability)
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FDA Standards Workshop
Synergizing Efforts in Standards Development for Cellular Therapies and Regenerative Medicine Products (March 31, 2014) sponsored by FDA/CBER/OCTGT http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/WorkshopsMeetingsConferences/ucm364114.htm
• Inform stakeholders on: 1) The role that Federal Agencies play in standards
development;
2) the different types of standards that can be useful in these types of products;
3) identify organizations that are developing standards for such products.
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Workshop Agenda
1. Government Overview – FDA/CBER, FDA/CDRH, NIH, NIST, NSF
2. Standards Development Organizations – ATCC/ANSI, ASTM, ISO
3. Professional Societies – AABB, ISCT/ARM, TERMIS, USP
4. Case Studies on standards development and standards use in product development
5. Panel Discussion
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Outcome: Factors Important for Standards Development
in the RM Space
• Awareness of existing standards- (e.g., mechanism to identify existing standards, a mechanism to allow widespread commenting on draft standards under development by various Standards Development Organizations (SDOs))
• A mechanism to identify the need for specific standards
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Factors (Continued)
• Education on standards development and
standards use at scientific conferences, society meetings and universities
• Coordination between groups interested in standards development to prevent duplication of efforts
• Funding support and scientific interest in developing the needed standards
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Standards Use to Support Regulatory Applications Submitted
to the U.S. FDA
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Types of Standards that Can be used to Support a Regulatory Application
• Voluntary Consensus Standards (ISO, ASTMi, etc.)
• Harmonization Standards
o WHO:
• Requirements, Recommendations, and Guidelines
• Physical Standards for manufacture and control of biological products
o ICH
• Standards on Quality, Safety and Efficacy
• Pharmacopeial Standards (USP, Eur. Ph., JP)
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OTAT Contact Information
Judith Arcidiacono, M.S. International Regulatory Expert, Standards Liaison [email protected] 240-402-8250
Regulatory Questions: Contact the Regulatory Management Staff in OTAT at [email protected] or [email protected] 240-402-8361
OCTGT Learn Webinar Series: http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/ucm232821.htm
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