ORGANIZED BY: Standards Coordinating Body Taskforce

Standards Coordinating Body For Cellular/Gene and Regenerative Therapies

SCB  MISSION  

Establish  a  Public-­‐Private  Partnership  to  support  the  development,  

dissemina7on,  educa7on  and  implementa7on  of  standards  and  other  related  deliverables  for  the  cellular/

gene  &  regenera7ve  medicine  industries  

To  work  with  na7onal  &  global  consensus  standards  organiza7ons  such  as  ISO  and  ASTM  to  

support  the  development  of  na7onal  and    interna7onal    standards  for  the  cellular/gene  &  

regenera7ve  medicine  industries  

To  serve  as  a  source  of  knowledge  and  experience  to  enable  more  efficient  and  

successful  clinical  and  commercial  development  of  cellular/gene  &  

regenera7ve  medicine  therapies  through  coordina7ng  and  priori7zing  

development  of  na7onal/interna7onal  standards  for  areas  including  measurement  assurance  

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Standards Coordinating Body (SCB)

SCB: Relevant Entities

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FDA and EMA (CAT) – Regulators of cellular/gene & regenerative therapies

NIST – Federal government organization supporting measurements and standards development

ISO, ASTM – Consensus standards development organizations (SDOs)

Large pharma and biotech – Developers/co-developers and/or enablers of developers of cellular/gene & regenerative therapies

Small start-up biotech companies – Developers of cellular/gene & regenerative therapies

ISCT, FACT, PACT, ASGCT, ARM, AABB, TERMIS, CTMC, ISSCR – Professional societies, trade organizations, and consortia in the cellular/gene & regenerative therapies

Academic (e.g. inventors)

Repositories – ATCC

CMOs, CROs and tool providers

NIST  Laboratory  Programs  

SDO  Leadership  

Contribu;ons  

SCB  Ac;vity  

•  Convene experts on standards

•  Recommend projects to address emerging needs

•  Create and maintain comprehensive standards information resource

•  ISO/TC 276 and ASTM F04 consensus activities

•  Convening of National and International meetings

•  Measurement assurance underpin standards development

•  Innovative measurement sciences; enable tools, methods, reference materials, and data

•  Increased industry collaborations

Synergizing Efforts For Standards Development

SCB/Non-gov’t Consortium

NIST

MOU

FDA

DoD

TBD

Internal and external interactions to support SCB efforts/projects; Reference Materials;

Protocols; inter-lab data generation

ECC

SCB staff

Project-specific

CRADAs

Proposed SCB-NIST PPP Model

Project-Oriented Working Groups:

•  To be defined by SCB Work Plan

SCB Partnership with other US Federal Agencies

A Memorandum of Understanding (MOU) memorializes the joint efforts of SCB and NIST, to include activities such as:

•  Potential NIST roles: •  Standard development; •  Workshop planning; •  Review/tasking of research

priority areas; and •  Establishing laboratory program

for research activities with SCB members and other organizations

•  Other US Fed Agency relationships/roles to be defined

•  Will set up official relationships with relevant international agencies and related organizations

 

SCB/Non-gov’t Consortium

NIST

MOU

FDA

DoDTBD

Project-specific

CRADAsSCB

Members

Agency X

SCB  contribu1ons  

• Iden;fy  and  convene  experts  to  priori;ze  and  provide  exper;se  and  knowledge  to  measurement  and  standards  needs  for  cellular/gene  and  regenera;ve  therapies  

• Perform  gap  analysis  to  iden;fy  emerging  needs  for  standards  

• Recommend  projects  to  address  project  requirements  

• Organize  SCB/NIST  workshops  to  discuss  priori;es  for  standards  and  associated  technical  challenges  (logis;cs/financial)  

• Create  and  maintain  comprehensive  standards  informa;on  resource  

• Develop  web  content  with  informa;on  on  standards,  services,  and  products    

• Par;cipate  with  advise  NIST  in  ISOTC  276  US  TAG  and  ASTMF04  

Where SCB Proposed to Fit into Existing cSDO Efforts

Industry  iden;fied  standards  needs;  

coordinate  and  priori;ze  based  on  expert  

consensus  and  support  sufficiency  

Development  of  standards  within  

recognized  Consensus  Standards  Organiza;ons  in  collabora;on  with  NIST  (e.g.  ISO,  ASTM)  

Educa;on  on  development  and  u;lity  of  standards,  

etc.,  including  poten;al  for  use  in  

regulatory  review;  Db  of  successfully  u;lized  

standards    

SCB  ac'v

i'es  

upstream

 of  cSD

O  

SCB  ac'v

i'es  

downstream  of  

cSDO

 

ü  Senator Baldwin introduced S.2443 ‘‘Advancing Standards in Regenerative Medicine Act’’.

ü  Support for the establishment of a Standards Coordinating Body in regenerative medicine and advanced therapies.

ü  Language included in current draft of 21st Century Cures Legislationü  FY 2017 Appropriations report language directs NIST to work with

stakeholders to develop strategic plan to establish SCB

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Supporting Legislation

SCB PPP Utility Summary

• SCB PPP is intended to bring together a diverse set of stakeholders to address standards development issues for innovative emerging technologies

• While each stakeholder will maintain its own mission focus, collaboration allows for synergy in the pre-and pro-competitive space, and provides benefits to private sector, gov’t and patients

•  Intent is to produce outcomes that are facilitating innovation and improved product development in advanced therapy development as well as support/speed regulatory decision-making

• SCB coordination and support of newly initiated and ongoing standards projects in conjunction with consensus Standards Development Organizations (SDOs) will facilitate more industry –relevant standards and other deliverables will be developed and implemented in a timely manner.

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Proposed Major Near-Term Milestones for SCB*Establish/select charter members to define objectives

and select org. structure  

Develop MOU/CRADA or other framework to engage federal entity – NIST, and launch official partnership

Identify/hire leadership and staff to manage SCB

Establish initial operating budget to include private sector contributions and public grant funding

Finalize short-term project plans

Define long-term funding model to sustain/grow operations

Determine mechanism for international participation

*  Not  in  chronological  order  

•  Chair: Jessica Carmen, Lonza •  Co-Chair: Morrie Ruffin, ARM

•  Establish official SCB entity •  Establish membership requirements •  Create operating principles and by-laws •  Collaborate with steering committee to develop proposed governance

structure –  Board of Directors/Oversight Committee –  Officers –  Composition guidelines for Board of Director

•  Open to public

Governance Structure

•  Co-Chairs: Morrie Ruffin, ARM, Claudia Zylberberg, Akron Biotech •  Participants: Elena Kfoury, Axiogenesis and Sven Kili, GSK

Economic Model and Budget

Sponsorship  

Grants  

Dues  and  member  

contribu;ons  

Federal  and  state  funding  support  –  appropria;ons  etc.  

User  fees  

Other  in-­‐kind  support  

Note : Participation will be open to public, activities transparent, and deliverables will be open source

Work Plan •  Chair: Michael Mendicino, Mesoblast •  Co-Chair: Krish Roy, Cell Manufacturing Consortium

•  Establish official SCB work plan by a team of all stakeholders across all product areas, with both international and Federal Agency representation

•  Discuss/share draft short- and long-term topics with wider SC and finalize •  Designate 1-2? champions for each topic from the work plan WG to develop

details with input from the work plan WG •  Prioritize based on budget and resources as per economic WG •  Determine how best to disseminate work plan to stakeholders and

disseminate/gather participant organizations/individuals •  Work with Federal Liaison WG on logistics of NIST, FDA and other Agency

interactions/activities with regards to work plan and ISO activities

NIST/FDA Liaison •  Chair: Jiwen Zhang, GE Healthcare •  Co-Chair: Wen Bo Wang, CDI

•  Establish working relationships with NIST, FDA, and other agencies –  Coordinate activities related to standards development, dissemination,

and implementation –  Develop mechanisms and activities for potential use of consensus

standards in regulatory process •  Assign point-of-contacts:

–  SCB/NIST: Jiwen Zhang/Sheng Lin-Gibson –  SCB/FDA: Michael Mendicino/FDA PoC TBD

•  SCB-FDA interactions proposed to be established via SCB and FDA point-of-contact. Specific mechanisms will depend on FDA feedback on what is feasible for FDA

–  Other agencies and PoC to be identified

ISO/TC  276:  Biotechnology  Secretariat:  DIN  

22  par;cipa;ng  countries  13  observing  countries  

Terms  and  Defini;on  

Biobanking  and  Bioresources    

Analy;cal  Methods  

Bioprocessing  

Data  Processing  and  Integra;on                      

US  TAG  276:  Biotechnology  Secretariat:  NIST  >  40  Organiza;ons                                              >  100  members  and  

observers    

Terms  and  Defini;on  

Biobanking  and  Bioresources    

Analy;cal              Methods            

Bioprocessing  

Data  Processing  and  Integra;on                      

Germany

USA

France

Japan

Germany

C Allocca/NIST

S. Lin-Gibson/NIST

N. Ramirez/CAP

J. Zhang/GE Healthcare

Vacant/SLG acting

Current Biotechnology Standard Activities

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Claudia  Zylberberg,  Akron  

Morrie  Ruffin,  ARM  

Alicia  Sukup,  ASGCT  

Elena  Kfoury,  Axiogenesis  

Knut  Niss,  Biogen  

Tom  Novak,  CDI  

Roy  Krishnendu,  Cell  Therapy  Manufacturing  Consor;um  

Jiwen  Zhang,  GE  Healthcare  

Sven  Kili,  GSK  

Ohad  Karneli,  ISCT  

Francis  Meacle,  Johnson  &  Johnson  

Jessica  Carmen,  Lonza  

SCB Steering Committee Members and AdvisorsMichael  Mendicino,  Mesoblast  

Sheng  Lin-­‐Gibson,  NIST  

Anne  Plant,  NIST  

Eric  Roos,  ThermoFisher  

Richard  Snyder,  Brammer  Bio  

Jason  Wertheim,  Northwestern  

 Advisory  Posi'ons:  

Michael  Werner,  ARM  

Sowmya  Viswanathan,  CCRM  

Damian  Marshall,  Cell  Therapy  Catapult  

Honeyeh  Zube,  NIST  

Fouad  Atouf,  USP