Voluntary Accreditation in Cell Therapy: Working Toward Global Standards

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Voluntary Accreditation in Cell Therapy Working
Toward Global Standards

• CF LeMaistreEJ ShpallAlvaro UrbanoPeter Wernet

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Are Global Standards Needed?

• International transport of hematopoietic
  progenitor cells
• Patient safety
• Optimize outcomes

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NMDPOrigin of HPC
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Are Global Standards Needed?

• International transport of hematopoietic
  progenitor cells
• Patient safety
• Optimize outcomes
• Protect research and development
• Protect availability of new products
• Need for a tiered, risk-based approach

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How Best to Realize Global Standards?
Unique national standards with mandatory
accreditation
International standards developed by experts
with voluntary accreditation
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JACIE
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Foundation for the Accreditation of Cellular
Therapy
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Foundation for the Accreditation of Cellular
Therapy

• Co-founded by
• American Society for Blood and Marrow Transplant
• International Society For Cellular Therapy
• Purpose
• Establish standards for quality medical and
  laboratory practice
• Implement voluntary inspection accreditation
  program

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FACT Major Goals

• To promote quality patient care and quality
  laboratory performance
• a valid accreditation process for HPC transplant
  must review both
• Continue development of rapidly evolving field
  where major therapeutic advances continue to be
  made

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FACT History

• 1994
• ISHAGE (ISCT) Laboratory Standards drafted
• ASBMT Clinical Standards drafted
• 1995
• Standards merged
• Public comment and review
• 1996
• FAHCT is incorporated
• FAHCT Standards published
• 6/97 Inspection and Accreditation Program began
• 3/98 First North American programs accredited by
  FAHCT

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Joint Accreditation Committee EBMT-EuroISHAGE
(JACIE)

• Established 1999
• Adopted FACT standards 1999
• Joint inspector training workshops in 2000, 2001,
  2002
• Joint review of 2002 FACT standards

• Countries participating
• AustriaBelgium DenmarkFinland
  FranceItalyThe NetherlandsNorwaySpainSweden
  SwitzerlandUnited Kingdom

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Other International Relationships

• Australia
• Japan
• Israel
• Mexico

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FACT History

• 2000 Joint Standards for Umbilical Cord Blood
  Banking
• 2002 Inspection program started

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FACT History 2002
FAHCT becomes FACT
FACT Standards Second Edition Published
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2004 FACT Board of Directors

• C. Fred LeMaistre, MD President
• Elizabeth J. Shpall, MD Past-President
• Richard Champlin, MD Vice President
• Adrian Gee, PhD Secretary
• Frank Smith, MD Treasurer
• Kenneth Cornetta, MD Linda Kelley, PhD
• Neal Flomenberg, MD Joseph Antin, MD
• Shelley Heimfeld, PhD Donna Wall, MD
• Helen Heslop, MD Phyllis Warkentin, MD

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FACT ORGANIZATION
ASBMT
ISCT
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FACT Inspected Facilities

• Demonstrated commitment to quality
• Support international standards of quality in hpc
  transplant
• Are critical to improving the process

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FACT Inspectors

• Volunteers
• Expert in their field
• Trained to perform inspections
• Role is to
• Evaluate compliance with standards
• Represent the quality and goals of FACT
• Suggest improvements in the process

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Fact Board
Standards
Quality Assurance
Accreditation
Education Training
Bylaws
Nominating
Finance
Regulatory Relationships
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FACT Standards

• New standards drafted by standards comm. chaired
  by Dr. Linda Kelley
• Review and revision
• Board of directors.
• Legal review.
• Published for member/public comment
• Comments review/revisions made
• Adopted by board and published
• Effective 90 days from publication

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FACT Standards Scope

• Cover all phases of collection, processing and
  administration of hematopoietic progenitors
  
• Unique in depth and breadth of inspection offered
• Require all clinical, collection and processing
  facilities
• to evaluate and report clinical outcomes.
• to have a Quality Management Program in place

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FDA
FACT
Adapted from Gastineau, BMT 33777,2004
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FACT Process Improvement

• FACT Quality Assurance Committee Chaired by Dr.
  Adrian Gee
• Investigates complaints and performance issues
• Inspector handbook/ new training workshops
• Recommend new processes and procedures
• Accreditation timelines
• Central Office review
• Communication with inspectors

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FACT Accreditation Timeline
APPLICATION
12m
Checklist submission
3m
On-site Inspection
3m
Board Response
6m (11m if reinspection)
ACCREDITATION
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FACT Inspection Outcomes 128 Accredited Programs

• Initial Outcome No. of programs
• No deficiencies 3
• Minor deficiencies 105
• (written response required)
• Major deficiencies 20
• (reinspection required)
• TOTAL Accredited 128

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FACT Inspection Outcomes 33 Renewal Accredited
Programs

• Initial Outcome No. of programs
• No deficiencies 1
• Minor deficiencies 32
• (written response required)
• Major deficiencies 0
• (reinspection required)
• TOTAL Renewal Accredited 33

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FACT AccreditationApril 2004

• HPC Facilities Registered 221
• Facilities in progress 93
• HPC Facilities Accredited 128
• Renewal Accreditations 33
• Cord Blood Banks registered 33
• CBB in progress 26
• CBB inspected 7
• CBB Accredited 3

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Entities Requesting Information About FACT
Certification

• States
• MA, KY, OK (Medicaid), Minnesota (DHS)
• Cooperative Groups
• CTN, SWOG, ECOG, COG
• Payers
• Aetna, Allianz AG (Lifetrack), BC/BS, First
  Health, Humana, Pacificare, URN

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SURVEY OF IBMTR/ABMTR U.S. TRANSPLANT CENTER
RESOURCES
International Bone Marrow Transplant Registry
Autologous Blood and Marrow Transplant Registry
TCR03_1.ppt
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SURVIVAL AFTER BMT FOR PATIENTS IN EARLY
STAGE- by FACT Accreditation -
Accreditation
Pending accreditation
No accreditation
PROBABILITY,
0
2
4
6
8
12
10
MONTHS
TCR03_45.ppt
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Why is FACT Successful?

• Voluntary, expert inspectors

• Facilities committed to quality

• Motivated technical staff

• Committed Leadership

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JACIE
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Voluntary Accreditation in Cell TherapyWorking
Toward Global StandardsCord Blood Banking and
Transplantation

• May 8, 2004

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The International NETCORD Foundation High
Quality Cord Blood Banking
Peter Horn, M.D. Holger Gressmann, M.Sc. Peter
Wernet, M.D. on behalf of the Board of
DirectorsM. Contreras (London), W. Fibbe
(Leiden), J. Garcia (Barcelona), E. Gluckman/V.
Rocha (Paris), P. Rebulla (Milano), P. Rubinstein
(New York), E.J. Shpall (Houston), T. Takahashi
(Tokyo) and P. Wernet (Düsseldorf)
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NETCORD - a banking organisation
One bad apple spoils the whole bunch. -
Geoffrey Chaucer, 1343-1400 Father of
English Literature
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The International NETCORD Foundation
Düsseldorf
Santiago de Compostela
Madrid
Paris (France Cord)
Mannheim
Milano (GraceNet)
Barcelona
London
Jerusalem
Tokyo
Leuven
Tianjin
Leiden
Prague
Beijing
Sydney (AusCord)
New York City
Helsinki
Malaga
Tel Hashomer
Liège
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The International NETCORD Foundation
21 Participating CB Banks (from 14
Countries) gt83,000 CB units available 2,672 CB
units used for transplantation 187 Transplant
Centers registered
 
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NETCORD offers

• High quality standard by international
  accreditation (NETCORD/FACT)
• Validation of Cord Blood Inventories
• Unified search and allocation system for CB
  units (Virtual Office) incl. real-time matching
  and electronic administration of searches
• Collection of Follow-up data through EUROCORD

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NETCORD Inventory and Use
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How to become a NETCORD Member

• adopt NETCORD/FACT Standards for the collection,
  processing, testing, banking, selection and
  release
• accreditation by national authorities and
  NETCORD/FACT
• in place for gt3 years, gt1,000 units stored
• at least 5 units supplied for transplant

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Benefits and Obligations NETCORD Members

• submit quarterly reports on transplant
  activities
• attend at least one annual meeting of NETCORD
• communicate all search requests
• joint inventory and real-time searches (Virtual
  Office)
• Interested? Contact us www.netcord.org

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The NETCORD Virtual Office
Things should be made as simple as possible -
but not simpler. - Albert Einstein,
1879-1955
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The NETCORD Virtual Office
www.netcord.org
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Registered Transplant Centres in the VO
187
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Patients entered in the VO
2848
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Overcoming Allocation Conflicts

• CB searches for more patients
• Low CB/Patient ratio
• MORE CONFLICTS
• CB searches for adults
• Competition with many patients
• Only a subset of CB useable
• MORE CONFLICTS
• Increasing Inventory
• LESS CONFLICTS
• BETTER UNITS

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CB Allocation System Requirements

• Advantage of possible fast allocation
• Need for Speed
• Communication between TC and CBB
• Long reservation times limits availability of CB
• Comparability of data

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The real-time matching program

• Input of high or low resolution HLA A, B and DR
• Data transfer and matching start immediately
• NMDP codes are matched according to search
  determinants
• Status of matching displayed via Web interface
• Match results available in lt1 minute

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The NETCORD Virtual Office
1. Performing a match run and output of matching
results 2. Selecting of cord blood units by
TC 3. Preparing and submitting CB unit request
by TC 4. Continued administration of search
process
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Accreditation by NETCORD/FACT
Not everything that can be counted counts and
not everything that counts can be counted.
- Albert Einstein, 1879-1955
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Potential Advantages of Cord Blood

• Procurement non-invasive
• Expanded donor pool minorities targeted
• Immediate availability (can be shipped overnite)
• Requires less stringent HLA matching (4/6, 5/6)
• Less GVHD in children

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130,000 cord blood units reported to Bone Marrow
Donor Worldwide in 34 CB banks 2003
2.500 3.000 UCBT performed
E. Gluckman
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Benefits of Cord Bank Banking

• Provides a source of hematopoietic progenitor
  cell support for patients who have no donors
• Ability to target collections for minorities with
  unique HLA types (Hispanics, African Americans,
  Asians)
• Provides a surplus of cord units for research
  (small or infected units)
• Stem cell plasticity
• Specific tissue repair (heart, pancreas, neural)
• Gene transduction for therapy

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Cord Blood BankingDifficulties for the
Transplant Centers

• Knowing the quality of the cord unit
• Is it the right cord unit?
• Does it have sufficient cells?
• Will it engraft?
• Solution
• Cord Bank Inspection and Accreditation

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NETCORD/FACT Accreditation Process
NETCORD/FACT standards developed in consensus
by representatives of NETCORD, FACT, and
individual members of ISCT and ASBMT
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Required Standards Cover

• CORD BLOOD BANK STANDARDS including quality
  management of staff and supplies.
• CORD BLOOD DONOR AND COLLECTION STANDARDS
  including cord blood collection sites
• CORD BLOOD PROCESSING STANDARDS including
  processing of cord blood and cryopreservation
• SELECTION, RELEASE AND SHIPPING OF CB UNITS
  including transport of cryopreserved units to the
  transplant facility

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NETCORD/FACT Standards Adopted by

• ASBMT American Society for Blood and Marrow
  Transplantation
• ISCT International Society for Cellular Therapy
• JACIE Joint Accrediation Committee of ISCT-Europe
  and EBMT
• EBMT European Group for Blood and Marrow
  Transplantion
• CBMTG Canadian Blood and Marrow Transplant Group
• ONT Organización Nacional de Trasplantes of Spain
• WMDA World Marrow Donor Association

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NETCORD-FACT CBB Inspector Qualifications

• Member ISCT, Euro-ISHAGE, ASBMT, EBMT or NETCORD
• Completed inspector training, test, and
  continuing education
• CBB Collection Inspector
• MD, PhD or RN/Med. Tech with gt 1 year as CBB
  Supervisor
• CBB Laboratory Inspector
• MD, PhD or Med. Tech with gt 5 years as
  Supervisor,
• Medical Director or Laboratory Director of a
  hematopoietic
• cell processing laboratory

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NETCORD/FACT - Accreditation process
Accreditation
For each individual cord blood bank Review of
documents by FACT
Date for on-site inspection of the cord blood bank
Review/Approval by NETCORD Board
Review/Approval by FACT Board
Team of NETCORD/FACTinspectors evaluate CB bank
Evaluation
Review by FACT-NETCORD CB Subcommittee
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FACT-NETCORD INSPECTIONSPotential Outcomes

• No deficiencies or variances
• Accreditation 3 years
• Some significant deficiencies
• Written response documenting corrections
• Responses evaluated staff /Chairman
• Review by CBB Subcommittee, Boards
• Accreditation

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FACT-NETCORD INSPECTIONSPotential Outcomes

• Major and Systemic deficiencies
• Written documentation of corrections
• Focused reinspection of specific area(s)
• Review by CBB Subcommittee with recommendations
  to Boards
• Review by FACT and NETCORD Boards
• Non-accreditation
• Reapplication

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NETCORD/FACT - Accreditation completed
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FACT-Netcord AccreditationApril 2004
Cord Blood Banks registered 33 CBB
in progress 23 CBB inspected
7 CBB Accredited 3 New York,
London, Duesseldorf
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The important thing is not to stop questioning.
- Albert Einstein, 1879-1955
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Harmonization of Standards for Bone Marrow
Transplants in EuropeAn EBMT ISCT (Euro)
Proposal
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JACIE ACCREDITATION PROGRAM

• OBJECTIVES
• Improve the quality of clinical practice of
  centres dealing with haemopoietic progenitor cell
  transplantation
• Establish an international program of
  accreditation with common standards

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ACCREDITATION PROGRAM
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JACIE ACCREDITATION PROGRAM
First steps

• Adoption of FACT standards
• Discussion at national level
• Health authorities support

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JACIE ACCREDITATION
Scope

• Clinical Units
• Cell Collection Facilities
• Bone Marrow
• Peripheral Blood
• Cord Blood (NETCORD)
• Cell Processing Facilities
• Manipulation of cell populations
• Cryopreservation
• Infusion

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JACIE ACCREDITATION PROGRAM
Procedure

• Verification of the observance of FACT/JACIE
  Standards
• Examination by JACIE inspectors (Peer- review)
• Certificate

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JACIE ACCREDITATION PROGRAM

• Inspection / Accreditation Process
• 1.- Registration Form
• 2- Accreditation Checklist
• 3.- Initial Inspection
• 4.- Review of Inspection Results
• 5.- Potential Inspection Outcomes
• a) Accreditation granted for 3 years
• b) Deficiencies
• c) Non-Accreditation

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JACIE Online

• Designed an online system to manage
• Applications
• National Representatives information
• Document submission
• Inspections
• Consultants tasks
• Board review
• Renewal
• General JACIE information

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National JACIE Representatives
JACIE Board (I)
President A Urbano Ispizua (EBMT) Vice
President I Slaper-Cortenbach (ISCT)

• D Pamphilon, UK
• A Gratwohl, CH
• M Michallett, F
• N Schmitz, DE
• H Greinix, A
• T Ruutu, FI
• B Bär, NL

• A Rambaldi, I
• M Boogaerts, BE
• C Solano, ES
• N Jacobsen, DK
• G Kvalheim, NO
• P Ljungman, SW

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JACIE Board (II)

• Paediatric Representative, J Cornish
• Nursing Representative, B Quinn
• NETCORD Representative, P Wernett

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JACIE Central Office

• Medical Consultant Diana Samson
• JACIE Officer Eoin McGrath
• Executive Board
• President,
• Vice President,
• Medical Consultant and
• Executive Officer

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Role of JACIE Board (I)

• Updating standards
• Appointment of working committees
• Important modifications to accreditation process
• Defining interaction, policy and lobbying
  strategies with regard to other organisations

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Role of JACIE Board (II)

• Harmonisation of different accreditation
  processes
• Resolution of general conflicts
• Approval of budget and accounts
• Official approval of centres accreditation and
  issue of certification

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  PUBLIC HEALTH PROGRAMME APPLICATION FOR
FUNDING  
  European Commission Health and Consumer
Protection Directorate-General Directorate G
Public Health Financial Cell EUFO 4295 Bâtiment
Jean Monnet Rue Alcide de Gasperi L-2920
LUXEMBOURG
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JACIE Activities

• JACIE central office
• Courses
• Training inspectors
• Implementing Quality programmes
• Educational courses
• JACIE online
• Accreditation process

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Training courses in 2004
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Scheduled inspections in 2004
provisional
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Schedule of inspections 04

• January
• Kantonsspital, Basel
• University Hospital, Bern
• Hôpital cantonal universitaire, Geneva
• March
• St. Anna, Vienna
• April
• Schlieren, Zürich
• Hôpital E.Herriot, Lyon
• St. Louis, Paris
• June
• BRHC, Bristol
• St James', Leeds
• Kantonsspital, St.Gallen
• CHU, Bordeaux
• St. Anna, Vienna (Collection)

• July
• Nottingham City Hospital
• August
• Hammersmith, London
• September
• Kantonsspital, Arrau
• Ospedale San Giovanni, Bellinzona
• Centre Hospitalier Universitaire, Lausanne
• Univ. Children's Hospital, Zürich
• University Hospital,Zürich
• Institut G.Roussy, Paris
• October
• AKH, Vienna
• John Radcliffe Hospital, Oxford
• November

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Feedback from applicants

• a long day but we had a fruitful and fair
  inspection with two experts in SCT. They did an
  excellent job
• we had a thorough inspection and we are thankful
  to the auditors for their skill and seriousness.
  The atmosphere was excellent, there was no
  misunderstanding
• The atmosphere of the audit was very
  constructive, open and clear. .. learned many
  things about quality....after the exit interview,
  we had a little party

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Feedback from inspectors

• ..a terrible lot of work and all the inspectors
  were terribly tired after the audit

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COMMISSION OF THE EUROPEAN COMMUNITIES
DIRECTIVE OF THE EUROPEAN PARLIAMENT AND OF THE
COUNCIL on setting standards of quality and
safetyfor the donation, procurement, testing,
processing, storage, and distribution of human
tissues and cells
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Location of HSCT centres