Transmedcon Ahmedabad, Gujarat November 14-16, 2014

Transcription

1 FACT Accreditation for Cord Blood Banks and Cellular Therapy Nadim Mahmud, MD, PhD Associate Professor of Medicine Division of Hematology/Oncology Director, Hospital Stem Cell Laboratory Blood & Marrow Transplant Program University of Illinois at Chicago Chicago, IL Transmedcon 2014 Ahmedabad, Gujarat November 14-16, 2014

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4 Rescue of an Irradiated Mouse by a transfusion of Bone Marrow Cells QuickTime and a Photo - JPEG decompressor are needed to see this picture. CFU-S

5 Transplants by Cell Source (NMDP)

6 Dr. E. Donnall Thomas Nobel Prize in Medicine 1990 for Bone Marrow Transplantation

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8 Stem Cell (HPC) Transplant Program 1. Collection of HPC 2. Processing of HPC Product characterization cell number (assays: CD34, CFU, etc.) cell viability (trypan blue, 7AAD/PI), RBC depletion Cryopreservation Storage Release/disposal of HPC Transportation of HPC 3. Administration Fresh infusion Thaw and infusion (administration) HPC: Hematopoietic Progenitor Cells: bone marrow, peripheral blood, umbilical cord blood

9 Activities of a Stem Cell Processing Lab Plasma depletion Cell Count /Cell viability (Tryplan Blue, or Flow Cytometry based: Propidium iodide or 7AAD) RBC depletion BM products (BY starch-gravity (major ABO mismatch) CD34 enumeration CFU (colony forming unit), In vivo repopulation unit (engraftment, chimerism) Cryopreservation Storage Release Disposal Records QM Plan

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11 361 vs 351 Products 361 Products: 1. Minimal manipulation 2. Intended for homologous use 3. Not combined with another article 4. No systemic effect and not dependent on metabolic activity of living cells except if for autologous use, use in a first degree or second-degree blood relative or for reproductive use 351 Products A product containing or derived from HCT/P falls outside the four criteria-a request for designation to TRG (Tissue Reference Group). Biologic/ Medical Devices Non homologous use Manipulation considered to be more than minimal. Autologous chondrocytes expanded in vitro for repair of cartilage defects Allogeneic hematopoietic stem/progenitor cells and cord blood Genetically modified cell therapy products

12 Core cgtp (current Good Tissue Practice) *Facilities *Environmental Control *Equipment *Supplies *Recovery *Processing and Process control *Labeling controls *Receipt, predistribution, shipment and distribution *Donor eligibility determination: Donor screening, testing etc.

13 Risk management methodologies and tools Wilson D, Kurz J. Risk management in tissue and cell processing. In: Fehily D, Brubaker S, Kearney J, Wolfinbarger L, editors. Tissue and Cell Processing. 1 ed. West Sussex: Wiley-Blackwell; p. 31.

14 3 Indicators for Quality Management Program 1. Level of Activity 2. Process (How the system works) 3. Outcome

15 Purtill D et al. Blood Nov 2014 FACT Accreditated CB Bank-Better Cell Survival

16 What is FACT? The Foundation for the Accreditation of Cellular Therapy Collaborative organization Activities conducted by peers Builds consensus in cellular therapy Supports facilities seeking accreditation Promotes quality patient care and valid research results through Standards The premiere accrediting body focusing on cellular therapy and cord blood banking

17 The Goals of the FACT Community Promote quality patient care and laboratory practices Improve treatment outcomes Foster continued development of the field Pursued through three core services: Standards Accreditation Education

18 FACT s Beginnings A perceived need existed for: Professional Standards across the fields of Hematology/Oncology and cellular therapy Collaboration between clinicians and laboratory scientists Physicians (ASBMT) and laboratory scientists (ISCT) merged their respective Standards in Formed FACT to establish voluntary accreditation program Initial goals of FACT: Improve patient care and laboratory practice Minimize burden of regulatory oversight Advance the field of cellular therapy

19 FACT s Collaborations JACIE: Joint Accreditation Committee of ISCT-Europe & EBMT Adopted first & second editions of FACT Cellular Therapy Standards; Joint collaboration third edition - international Standards Accreditation: similar but separate NetCord: Collaborate on development of Cord Blood Banking Standards Cord Blood Bank accreditation conducted by FACT worldwide

20 Who leads FACT? Cellular therapy experts! Activities performed by volunteers actively engaged in cellular therapy and cord blood banking Ensures FACT initiatives and services meet your needs Accredited programs are active participants in FACT activities and committees No bureaucrats or non-cellular therapy experts set requirements or perform inspections Supported by staff dedicated to assisting programs seeking accreditation

21 What services does FACT provide? Sets minimum standards for quality cellular therapy transplant programs and cord blood banks Accredits facilities based on the standards Educates the field about FACT requirements and cellular therapy principles Advocates the advancement of cellular therapy worldwide

22 The Threshold for Excellence in Cellular Therapy Elevates your position as a quality organization Informs patients, payers, governments, funding organizations, and clinical trials groups that your organization meets or exceeds the standards set by leaders in the field

23 The Supportive Network of Peers Collegial Helping Peers Dynamic and flexible to adapt to evolving needs Updated guidance information as needed Assistance throughout the accreditation process and between cycles

24 The Most Beneficial Accreditation Serves as a resource for patients deciding where to receive healthcare Used as a criterion by international clients seeking quality cellular therapy products Demonstrates to clinical trial sponsors that the facility meets requirements published by the experts Facilitates the establishment of quality management and process control to minimize liabilities

25 The Trusted Accrediting Body Societies Registries Regulators Public

26 The Global Standard in Cellular Therapy STANDARDS

27 International Standards Uniquely address clinical and laboratory aspects of HCT and entire spectrum of CBB Evidence-based Standards Written by world-renown experts in the field, filling a variety of professional roles Clinicians, laboratory scientists, cord blood bankers, technologists, quality experts Each edition published for public comment FACT Standards are not best practices Define minimum requirements Allows flexibility in methods/processes to meet requirements

28 Standards Committee International representation Cellular Therapy Committee currently consists of members from 13 different countries Cord Blood Committee currently consists of members from 10 different countries World-renowned experts Variety of professional roles Clinicians, scientists, technologists, quality experts, etc.

29 Advantage of FACT Standards Cornerstone of FACT accreditation program Clear list of requirements cellular therapy programs and cord blood banks must meet Accreditation activities are based upon the Standards Requirements that focus on both clinical aspects and laboratory practices

30 Minimum Requirements = Flexibility FACT Standards are not best practices Outline only minimum requirements Allows flexibility of how to meet requirements Quote from Accreditation Manual, which provides explanation of the Standards and examples of compliance: This is not an exhaustive list of possible ways to meet the Standards, and the intent is to provide examples only since there are many effective mechanisms by which to achieve compliance...

31 Example of Different Methods to Comply with a Standard Standard: B1.2 The Clinical Program shall use cell collection and processing facilities that meet FACT- JACIE Standards with respect to their interactions with the Clinical Program. Ways in which this requirement could be met: Clinical program is integrated with collection and processing facilities Clinical program contracts services with external collection and processing facilities that are FACTaccredited

32 Scope of Cellular Therapy Standards Hematopoietic progenitor cells (HPC) and Mononuclear cells (MNC) Collection of cells from marrow and peripheral blood Cellular processing Clinical administration of HPCs and MNCs from marrow, peripheral blood, and placenta/umbilical cord blood Requires a comprehensive Quality Management Program

33 Scope of Cord Blood Standards Cord Blood (CB) Donor: recruitment, screening, testing, and consent Product: collection, transport, processing, storage, search and matching Requires a comprehensive Quality Management Program Common Deficiencies Quality Management; Audits Policies & Procedures

34 Cord Blood Standards - Common Citations Quality Management Audits Policies & Procedures Donor Informed Consent Labeling Version control, verification, validation Cord Blood Collection Procedure Improper labeling, adverse events not recorded Cord Blood Unit Testing Microbial culture assays not validated

35 Common Standards for Cellular Therapies Drafted in response to rapid advancement of cellular therapies into clinic, wide variety in quality Based on current FACT Standards for HPC and cord blood banking Includes basic fundamentals applicable to any cell source or therapeutic application Could serve as the basis for a new voluntary accreditation in cellular therapy Discipline-specific or product-specific standards will be added going forward Public comment period ended in July; Board will soon make a decision about how to proceed

36 HCT Cellular Therapy Programs Future Goals Cord Blood Banks FACT Accreditation Regenerative Medicine

37 Elevate Your Position in Cellular Therapy as a FACT Accredited Organization FACT ACCREDITATION

38 FACT s Accreditation Program Voluntary Cellular therapy programs and cord blood banks choose to increase the quality of their processes for the benefit of patients Peer-driven Inspectors are your peers who volunteer to serve Inspectors both help and learn from inspections International Promotes access to matched donors and quality cell products worldwide Accredited cellular therapy programs in 5 countries and expanding Accredited cord blood banks in 16 countries and expanding

39 Accredited Cellular Therapy Programs Accredited as of October 2014: 193 facilities in 6 countries 4

40 Accredited Cord Blood Banks Accredited as of October 2014: 52 banks in 21 countries

41 Why Inspections Are Successful FACT staff are dedicated to assisting organizations Accreditation coordinators work directly with organizations Provide Standards interpretation, reminders, tips, etc. FACT educational sessions explain requirements and provide examples In-person workshops and online events FACT s philosophy is to help programs meet minimum requirements Inspectors work professionally in this field and are vested in everyone s success

42 Balancing Rigor with Support Collegial approach Helpful inspectors Dedicated support Rigorous and comprehensive inspection Consistent, thorough, and objective

43 Inspection is not a Pass/Fail Event Programs and banks are given an opportunity to respond to the inspection report and correct deficiencies Overall goal is to help programs and banks improve to a level of quality deserving of accreditation

44 Language of Inspection and Accreditation Process Applications, standards, and requirements are in English A short, specific list of documents are required to be translated Correspondence with FACT office is in English Every effort made to assign inspectors fluent in applicant s language Applicant may be asked to provide an interpreter

45 Types of FACT Accreditation Complete cellular therapy program (clinical, collection, and processing) Independent collection facility Independent processing facility Cord blood bank

46 Preparing for the Accreditation Process Review eligibility requirements Complete a self-assessment and address deficiencies Attend FACT workshops and webinars View FACT tutorials Contact the FACT office for assistance

47 Learning opportunities for all skill levels in all areas of the world EDUCATION

48 FACT Education Program Wide ranging Events cover a variety of topics including accreditation, quality, and operations Far reaching In-person events conducted throughout the globe Online events allow anyone in the world to view the session All encompassing Sessions are targeted to a wide variety of roles (clinicians, technologists, etc.)

49 Educational Offerings Social Media Facebook LinkedIn Events Workshops Webinars Tutorials (subscribe by creating a profile at factwebsite.org > FACTWeb > Create a new profile) Publications Newsletter Accreditation Manual Website

50 provides details regarding the scope of the Standards and links to regulatory and industry resources. includes general information pertinent to obtaining initial or renewal accreditation, including accreditation timelines, process overview, and fees. lists the benefits and expectations of inspectors and provides directions on how to apply to become an inspector. Lists upcoming educational events and provides links to cellular therapy resources

51 Obtain Free Access to FACT Publications! Create a FACTWeb profile at to download free Standards! lmiller1@unmc.edu to receive a coupon code for free access to educational tutorials and webinars!

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53 THANK YOU

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58 Stem Cell / Bone Marrow Processing Lab

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62 Cultured RBC: Possible Routes to the Clinic Anstee et al. Current Opinion in Hematology May 2012

63 Hierarchy in Hematopoietic System

64 3 Indicators for Quality Management Program 1. Level of Activity 2. Process (How the system works) 3. Outcome Caunday O et al. Bone Marrow Transplantation 2012

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