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  1. 1. 1 HTA Breakoutsessions Breakout session 1 Policy development at the HTA Kate Rolfvondenbaumen Regulation Manager This session will answer the questions of how and why policies are developed at the HTA. It will cover the entire policy development process from building the rationale, creation and finally implementation of the policy. Breakout session 2 Consent – the regulatory requirements Emyr Harries Regulation Manager This workshop will cover the expanded roles, responsibilities and obligations with regards to the Quality and Safety Regulations and the HT Act. Breakout session 3 Serious adverse events and reactions Christiane Niederlaender Regulation Manager Reporting and managing serious adverse events and reactions is a key responsibility for the DI. But what happens to the information we receive? This workshop will answer that question and help to increase your understanding of the hows and whys of reporting SAEARs.
  2. 2. 2 Invited Speakers Breakout Impact of regulation on ocular tissue banking Professor W. John Armitage CTS Bristol Eye Bank, University of Bristol There are at least 3000 ocular tissue transplants every year in the UK, with corneal transplants accounting for the great majority of these procedures. The CTS Eye Banks in Bristol and Manchester supply tissue for up to 90% of these transplants with eye banks at Moorfields and East Grinstead also supplying tissue principally for their own surgeons. By the late 1980s, the Corneal Transplant Service was providing a centrally co-ordinated delivery of ocular tissue through the former UK Transplant Service (now NHSBT Organ Donation and Transplantation, ODT) for patients throughout the UK. This organizational structure provided a means of setting and implementing standards for eye donor selection, retrieval, storage, tissue assessment, allocation and distribution. There has since been a marked increase in standards for critical processing areas in eye banks and related environmental monitoring. Policies and processes are fully documented and mechanisms have been established for the reporting and investigation of serious adverse events and reactions. This process began under the voluntary DH Code of Practice for Tissue Banks and was expanded and formalized into statutory requirements through the EU Tissues and Cells Directive, transposed into UK law by the Quality and Safety Regulations. One advantage in the area of corneal transplantation is the routine collection of clinical follow-up data through ODT for up to five years postoperatively. These data are stored on the National Transplant Database (NTxD) along with follow-up data from all organ transplants. Not only does this provide robust donor-recipient traceability, but these data help to identify adverse events and reactions. Moreover, eye bank processes can be validated through corneal transplant outcome, and the outcome of new surgical techniques and new methods of tissue preparation in eye banks can be properly evaluated. The impact of regulation has been beneficial in that policies and procedures are now more formalized and the statutory process of biennial inspection by the Competent Authority is helpful and informative. These inspections and the overall regulatory environment also serve to raise the level of confidence that surgeons and their patients have in the quality and safety of tissue provided by eye and other tissue banks. The financial and workload implications for eye banks should, however, be acknowledged. So far as future developments are concerned, eye banks are already moving towards cell-based therapies and advanced therapy medicinal products (ATMP). Currently, these include the ex vivo expansion of limbal
  3. 3. 3 stem cell sheets for the treatment of ocular surface disease; but manipulation of corneas either for the purposes of immunomodulation or to increase corneal endothelial cell density, or expansion and application of retinal progenitor cells for the treatment of retinal disease will present new challenges for regulation by multiple agencies, such as the Human Tissue Authority (HTA) and the Medicines and Healthcare products Regulatory Agency (MHRA). Multiple activities, one licence - a personal perspective Alistair Cranston Designated Individual, Birmingham Children’s Hospital NHS Foundation Trust In this discussion I should like to describe my personal experiences as Designated Individual for my Trust. This started as a relatively low profile role that was managerially convenient for the Trust and that did not require a great deal of my time to discharge. As my experience has grown, and particularly since the introduction of procurement regulation in 2008, a great deal more involvement and time has been necessary. I will discuss the issues both personally, as DI, and for the organisation that HTA Licensing has exposed and some of the steps I have taken to meet these challenges. I am sure that others will have had similar experiences and I hope that the discussion will allow us to share problems and solutions that will be mutually of interest and benefit. When HTA regulation was introduced for the “Human Application” sector in 2006 there was one small and relatively self contained activity that fell under the conditions of the Human Tissue Act, The Trust’s Heart Valve Bank. As Clinical Director for cardiac services within the Trust and also as an anaesthetist with a predominantly cardiac practice it seemed that I was well placed to act as DI. This proved to be the case and the role seemed relatively easy to manage within the context of my full-time clinical role. The first HTA inspection in 2007 gave me relatively few headaches and the few conditions imposed were not difficult to address. Following the introduction of procurement regulation in 2008, additional activities were added to our licence. (Procurement of Peripheral Blood Stem Cells and Bone Marrow Cells and Skin procurement from burned patients). My familiarity with the haematology and burns services was less than that with cardiac procedures but it appeared that there would be no particular difficulty in continuing to act as DI for the additional activities involved. Clinically this proved to be the case, but from a management and process point of view these services were very much less
  4. 4. 4 cohesive than the self contained Heart Valve Bank. The 2009 HTA inspection was a rude awakening and imposed a large number of conditions on our licence, almost exclusively related to the procurement activity. I will discuss how I have attempted to deal with these issues and how I have had to re-evaluate the role of DI since then. I hope that we might be able to explore some of the challenges that multiple applications within one licence produce and share potential solutions. ChondroCelect©, the first Advanced Therapy Medicinal Product approved by the EMA Gil Beyen CEO of TiGenix NV. TiGenix NV (NYSE Euronext: TIG) is a biomedical company that focuses on ‘Regenerating Motion’. The company is exploiting the power of Regenerative Medicine to develop durable treatments, validated through controlled clinical trials, for damaged and diseased skeletal tissues. In October 2009, the company’s lead product ChondroCelect, received Marketing Authorization as the first Advanced Therapy Medicinal Product (EU/1/09/563/001). The product is indicated for the repair of single symptomatic cartilage defects of the femoral condyle of the knee in adults. Demonstration of efficacy is based on a randomized controlled trial evaluating the efficacy of ChondroCelect in patients with lesions between 1 and 5 cm². In this trial ChondroCelect demonstrated a) superior structural tissue regeneration after 12 months, and b) clinical benefit over microfracture at 36 months. The main challenges encountered during the development and registration of ChondroCelect were:  Managing non-clinical expectations: finding the relevant preclinical model to demonstrate safety and efficacy in (large) animal models.  Meeting CMC requirements: ensuring reliable and consistent quality manufacturing, starting from autologous cells with inherent variability.  Meeting clinical requirements: demonstrating safety and clinically meaningful efficacy in a randomized controlled clinical trial.
  5. 5. 5 The company is now in the process of launching the product in selected European markets. We've got life... in the bag! Terie Duffy Midwife National Cord Blood Coordinator, Kingscord This session will focus on the consent, training and collecting processes with emphasis on safety and how various regulations have impacted on them. Stemming vision loss using stem cells - seeing is believing Professor Pete Coffey Director of the London Project to Cure Blindness and Professor of Cellular Therapy and Visual Sciences The aim of the London Project to Cure Blindness is to develop a stem cell therapy for the majority of all types of age-related macular degeneration. Professor Coffey and his team are working on the use of human embryonic stem cells for age-related macular degeneration and retinitis pigmentosa and the development of a cell based therapy for currently untreatable age-related macular degeneration (dry AMD). Fit for purpose; the SaBTO microbiological testing guidelines Professor Richard Tedder Professor of Medical Virology at University College London, Member of the Advisory Committee on the Safety of Blood Tissues and Organs and representative of the UK Public Health Agency This session will focus on the newly drafted SaBTO testing guidelines and the implications for establishments in the human application sector.
  6. 6. 6 Plenary sessions Emerging trends & their impact on human health Luc Noel Coordinator of Clinical Procedures, Essential Health Technologies, World Health Organisation (WHO) Dr Noel will speak on the subject of WHO projects and issues relating to organ transplantation and the use of tissue and cells, both, in the developed and developing nations. Harmonising the approach to regulation of tissues & cells in Europe Deirdre F. Fehily Italian National transplant Centre and technical coordinator of EUSTITE (European Union Standards and Training in the Inspection of Tissue Establishments) Ms Fehily will speak on the subject of European Union projects relating to tissue vigilance and the EUSTITE programme of training inspectors who inspect tissue establishments. The road to harmonization is not an impossible journey Scott A Brubaker Chief Policy Officer, American Association of Tissue Banks Mr Brubaker will speak on the subject of cross border regulation differences and their implication. Summary of HTA Activity in the Human Application Sector Imogen Swann Head of Regulation, Human Application Sector, Human Tissue Authority Ms Swann will provide an update on HTA activities, developments and plans with specific reference to the Human Application sector.