Quality and Safety of HSCT in Autoimmune Diseases Dr John Snowden Stem Cell Transplant Programme Director Sheffield Teaching Hospitals and University of Sheffield & JACIE Medical Director 1
HSCT is GROWING Bone Marrow Transplantation (2009), 1-17
HSCT is COMPLEX
...and has intrinsic risks
especially where there is pre-existing organ dysfunction
Low risk HSCT most autologous transplantation, TRM <5%
High risk HSCT TRM >10% allogeneic HSCT and some autologous HSCT in autoimmune disease
In healthcare, sometimes things go wrong The main findings are that approximately 43 million adverse events occur each year around the globe and cause a staggering 23 million associated disability-adjusted life years (DALYs, the sum of years of life lost and years lost to disability) 117,808 PER DAY GLOBALLY
Interruptions to operations
JACIE Accreditation A Badge of Quality in HSCT Quality management: An integrated program of quality assessment, assurance, control, and improvement
What is JACIE? Joint Accreditation Committee ISCT & EBMT International Society for Cellular Therapy European Group for Blood & Marrow Transplantation International standards-setting and accreditation entity Focussed on cellular therapy Compatible with EU regulations (2004/23/EC etc.) & US (FDA) Independent of the regulators - established by the practitioners themselves
JACIE s journey 1998 Voluntary initiative by the transplant professionals 2000+ Widespread acceptance among the profession Now External recognition e.g. Regulations, Validation
Improving outcomes of HSCT through external demonstration of: EFFECTIVE Quality Management Systems policies & procedures incidents, SAEs, audits and outcomes analysis and corrective actions EFFECTIVE facilities and staffing wards, outpatients, ITU, apheresis units, laboratories EFFECTIVE teamworking complex teams with different backgrounds and expertise training and competency
5th edition released 2012 6th edition work commenced 2013 Clinical Collection Processing lab
JACIE Office, Barcelona Eoin McGrath Executive Officer Inspection Report Assessors: Carole Charley, Ivana Ferrero Iris Bargallo Accreditations Coordinator ALSO: JACIE Executive Committee (Chair: Alessandro Rambaldi) JACIE Accreditation Committee (Chair: Joerg Halter) JACIE Medical Director (John Snowden) EBMT Board Representative (Anna Sureda)
JACIE INSPECTION TEAM RIYADH, SAUDI ARABIA, 2009
Where is JACIE today? Europe Middle East Since 2000 Facilities accredited: 216 Reaccreditations in progress: 43 Singapore (Jan 2013) Pretoria, South Africa (June 2013)
Take-up of centres reporting all transplants to 2011 EBMT Activity Survey 90-100% 80-89% 70-79% 60-69% 50-59% 40-49% 30-39% 20-29% 10-29% 0-9% Also: Saudi Arabia South Africa
JACIE 55% of European allogeneic transplant centres *Based on 2011 EBMT Activity Survey - centres that have applied, been inspected or been accredited since 2000
Why JACIE? Professional pride Competition with other centres? Improved outcome for patients
Impact on survival 22
Why JACIE? Legal requirement in some countries Health insurance companies may require it Social insurance systems may require it - e.g. UK NHS
JACIE & Health Authorities Regulations Belgium France Switzerland www.jacie.org/about/national-regulations Italy The Netherlands Guidelines European Directorate for the Quality of Medicines & HealthCare Guide to the Safety and quality assurance for the transplantation of organs, tissues and cells, 4th edition (2010) Italy Piano Oncologico Nazionale 2010-2012 cites JACIE accreditation United Kingdom Collaboration with regulators Italy Centro Nazionale de Trapianti (CNT) coordinates inspections of Italian centres with JACIE through GITMO. 24
Clinical trials may require it
Why JACIE? Firms up collaboration between departments, services, registries etc e.g. hospitals and national blood services e.g. unrelated donor collection centres and registries and recipient hospitals (often overseas)
Why JACIE? Production of cell-based medicinal products by hospitals and academic facilities rather than pharmaceutical companies Non-conventional use of cytotoxic and immune-suppressive drugs Complex therapeutic procedure, even when routinely applied Advanced therapeutic medicinal products (ATMPs) will be considered in next edition of standards
BENCHMARKING CENTRES
William Kelvin If you can not measure it, you can not improve it." William Thomson (Lord Kelvin) Determined the correct value of absolute zero as approximately -273.15 Celsius..
Future developments in JACIE
FACT-JACIE INTERNATIONAL STANDARDS FOR CELLULAR THERAPY PRODUCT COLLECTION, PROCESSING, AND ADMINISTRATION 5th edition released March 2012 6th edition work commenced 2013
6th Edition Standards 2013 June - Kick-off Aug-Dec - Sub-committee telecons 2014 Feb - Meeting of Committees at ASBMT Tandem Meeting Apr-Jun - Public consultation Oct - Approve final version 2015 March publish 6th edition
Issues to bring to 6th edition Evolution of cellular therapy - ATMPs Haematological therapies e.g. MSC, dendritic cells Regenerative therapies but many outside realm of haematological cell therapy How will FACT-JACIE standards accommodate ATMPs or products with more than minimal manipulation?
Quality and Safety of HSCT in Autoimmune Diseases For discussion and debate 34
Would disease specific quality standards be. Beneficial for HSCT various ADs? Beneficial for cellular therapy in various ADs? Feasible for various ADs? Agreed across specialities External accreditation National or international For discussion.
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