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  • Adverse Event

    An adverse event is any undesirable occurrence on a particular study. For clinical trials of Investigational Medicinal Products (IMPs), an adverse event is defined by the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) as any unfavourable and unintended sign, symptom or disease temporally associated with the use of an IMP, whether or not related to that product. For other research that does not use IMPs, it may be any unfavourable and unintended sign, symptom or disease temporally associated with participation in the research project.  The term has also been used to describe unfortunate events that may have serious consequences for the research project, e.g. breaking down of a freezer and loss of tissue samples.

  • Adverse Event Reporting

    System for reporting notifications of adverse events to a regulatory body.

  • Adverse Reaction

    Untoward or unintended response directly related to an Investigational Medicinal Product.

  • Allogenic

    Where donor and recipient are different individuals.

  • ATMP (Advanced Therapy Medicinal Product)

    Advanced Therapy Medicinal Products (Advanced Therapies or ATMP's) are innovative, regenerative therapies which combine aspects of medicine, cell biology, science and engineering for the purpose of regenerating, repairing or replacing damaged tissue/cells. The use of this technology has already led to the development of products that are used clinically for the treatment of burns or ulcers and cartilage repair systems used in the treatment of early arthritis. More complex products are currently being developed for the treatment of heart disease and other degenerative conditions. In the EU, ATMPs are regulated through Regulation (EC) No. 1394/2007 and include any of the following medicinal products for human use: (1) a gene therapy medicinal product, (2) a somatic cell therapy medicinal product, (3) a tissue engineered product, as defined in the aforementioned Regulation, or (4) a combined advanced therapy medicinal product where the cells are in combination with a medical device.

  • ATMP Regulation

    Advanced Therapy Medicinal Products (Advanced Therapies or ATMPs) are innovative, regenerative therapies which combine aspects of medicine, cell biology, science and engineering. ATMPs include any of the following medicinal products for human use: (1) a gene therapy medicinal product, (2) a somatic cell therapy medicinal product, or (3) a tissue engineered product as defined in the Regulation (EC) No. 1394/2007, or (4) a combined advanced therapy medicinal product where the cells are in combination with a medical device. Whereas somatic cell therapy medicinal products are intended to treat human disease through pharmacological, immunological or metabolic means, tissue engineered products are intended for the regenerating, repairing or replacing of human tissue. The use of this technology has already led to the development of products that are used clinically for the treatment of burns or ulcers and cartilage repair systems used in the treatment of early arthritis.

    Regulation (EC) No 1394/2007 on Advanced Therapy Medicinal Products entered into force on 30 December 2007 and applied from 30 December 2008. The Regulation has been fully implemented in the UK. Market approval for ATMPs is through the centralised procedure administered by the EMA. Specific guidance is available from the EMA via the link below. This includes information about the procedures in place for obtaining scientific advice and information on the committee for advanced therapies (CAT) as well as advice about seeking product classification. For further information, visit the MHRA or EMA websites.

    Under the ATMP Regulation, products which are prepared on a non-routine basis and used within the same Member State in a hospital in accordance with a medical prescription for an individual patient will be exempt from the Regulation. Exempted products need to comply with manufacturing, quality and pharmacovigilance standards which are defined at national level. The MHRA is the competent authority for ATMPs which are prepared and used under the hospital exemption in the UK. Please visit the MHRA site for further detail about the arrangements that apply under the UK’s hospital exemption scheme.

  • Autologous

    Where donor and recipient are the same individual.

  • Blastocyst

    A blastocyst is an embryo that has developed for five to six days after fertilisation, consisting of about 150 cells organised into an outer layer of cells and a cluster of cells inside known as the Inner Cell Mass.

  • CAT (Committee on Advanced Therapies)

    A Committee for Advanced Therapies (CAT) has been established within the European Medicines Agency (EMA) to assess the quality, safety and efficacy of ATMPs and follow scientific developments in the field. The CAT was established in accordance with the ATMP Regulation and is a multidisciplinary committee bringing together a wide range of expertise from within the EU.

  • Cell Bank

    A cell bank is an in vitro repository for cells and cell lines. Typically, a single aliquot from the master cell bank is cultured to prepare a working cell bank.

  • Cell Bank System

    A system whereby successive batches of a product are manufactured using in vitro cultured cells derived from the same master cell bank in order to produce batches of consistent quality.

  • Cell Culture

    A cell culture is a population of cells that is grown and maintained in vitro.

  • Cell Expansion

    A method used to increase in the number of cells in culture through repeated passage as cells proliferate.

  • Clinical Trials Regulations

    The Medicines for Human Use (Clinical Trials) Regulations 2004 ('The Clinical Trials Regulations') implement the European Clinical Trials Directive into national legislation in the UK. The regulations define a clinical trial, how research on the safety and efficacy of medicinal product in human participants should be conducted and criteria for ethics review. In the UK, no clinical trial of an IMP can begin until the approval of both the MHRA and a REC has been granted. For further detail, click here.

  • Combined advanced therapy medicinal product

    A combined advanced therapy medicinal product, as defined in Article 2 1 (d) of the ATMP Regulation refers to an advanced therapy medicinal product that fulfils the following conditions: (a) it must incorporate, as an integral part of the product, one or more medical devices within the meaning of Article 1 (2) 9a) of Directive 93/42/EEC or one or more active implantable medical devices within the meaning of Article 1 (2) (c) of Directive 90/385/EEC, and (b) its cellular or tissue part must contain viable cells or tissues , or (c) its cellular or tissue part containing non-viable cells or tissues must be liable to act upon the human body with action that can be considered as primary to that of the devices referred to.

  • Contained Use Regulations

    The Genetically Modified Organism (Contained Use) Regulations [The 'Contained Use Regulations' or simply GMO (CU) Regulations] provide for human health and safety and environmental protection from genetically modified micro-organisms in contained use (as opposed to 'deliberate release'), and additionally the human health and safety from genetically modified plants and animals (GMOs). The key requirement of the GMO (CU) Regulations is to assess the risks of all activities and to make sure that any necessary controls are put in place. The GMO (CU) Regulations provide a framework for making these judgments, and place clear legal obligations on people who work with GMOs. For more information, click here.

  • Culture Medium

    Nutrient supply used to support cell maintenance in vitro.

  • Differentiation

    The development of specialised types of cells from less specialised ones.

  • EMA (European Medicines Agency)

    The European Medicines Agency is responsible for the scientific evaluation of applications for European marketing authorisation for medicinal products (centralised procedure). Under the centralised procedure, companies submit one single marketing authorisation application to the EMA, which cover the whole of the European Economic Area. Its main responsibility is the protection and promotion of public and animal health, through the evaluation and supervision of medicines for human and veterinary use across the European Economic Area.

  • Embryonic Stem Cell

    Embryonic stem cells (ESCs) can be derived by isolation and subsequent culture of cells from the inner cell mass, part of early embryo. The inner cell mass is isolated at the blastocyst stage (4-6 days post conception) and subsequently cultured in the laboratory under specific conditions favouring the development of an ESC line. Derivation of a bona fide ESC line is determined by testing for the presence of the key characteristics of ESCs, namely pluripotency and the capacity for continuous self-renewal. A cell is pluripotent if it has the potential to develop into derivatives of all three embryonic germ layers: ectoderm, endoderm and mesoderm. Under the right culture conditions ESC lines can maintain pluripotency and the ability to self-renew indefinitely and are thus valuable as a potentially unlimited source of cells.

  • EudraCT

    EudraCT is a central database of all clinical trials in the European Union. All clinical trials must be registered with EudraCT. 

  • Ex Vivo

    Outside of the living body.

  • Feeder Cell

    Feeder cells are support cells used in co-culture systems to sustain or enhance the viability and desired characteristics of other cells. Embryonic stem cells are sometimes cultured using mitotically inactivated (non-proliferating) mouse or human fibroblast feeder cells.

  • GCP (Good Clinical Practice)

    Good Clinical Practice (GCP) describes the ethical and scientific quality standard for designing, conducting and reporting trials that involve the participation of human subjects. The International Conference on Harmonisation (ICH) standardised practice across Europe, Japan and the USA. For Clinical Trials involving Investigational Medicinal Products, it is a legal requirement that the trial is run to the principles of GCP.
  • Gene Therapy (Gene Therapy Medicinal Product)

    Gene therapy is broadly defined as the deliberate introduction of genetic material into human somatic cells. Commission Directive 2009/120/EC of 14 September 2009 amending Directive 2001/83/EC of the European Parliament and of the Council on the Community code relating to medicinal products for human use as regards advanced therapy medicinal products specifies that "Gene therapy medicinal product means a biological medicinal product which has the following characteristics: (a) it contains an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence; (b) its therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence. Gene therapy medicinal products shall not include vaccines against infectious diseases."

  • GLP (Good Laboratory Practice)

    Good Laboratory Practice (GLP) embodies a set of principles that provides a framework within which laboratory studies are planned, performed, monitored, recorded, reported and archived. These studies are undertaken to generate data by which the hazards and risks to users, consumers and third parties, including the environment, can be assessed for pharmaceuticals, agrochemicals, veterinary medicines, industrial chemicals, cosmetics, food and feed additives and biocides. GLP helps assure regulatory authorities that the data submitted are a true reflection of the results obtained during the study and can therefore be relied upon when making risk/safety assessments. 
  • GM (Genetic Modification)

    The term 'Genetic Modification' has been formally established under 'Contained Use Regulations'. Here, a Genetically Modified Organism is defined as an organism (with the exception of humans) in which the genetic material has been altered in a way that does not occur naturally by mating and/or natural recombination using recombinant nucleic acid techniques involving the formation of new combinations of genetic material by the insertion of nucleic acid molecules, produced by whatever means outside an organism, into any virus, bacterial plasmid or other vector system and their incorporation into a host organism in which they do not naturally occur but in which they are capable of continued propagation. This is a broad definition and further information on what is covered by the GM Regulations can be found in A guide to the Genetically Modified Organisms (Contained Use) Regulations 2000 L29 (Third edition) HSE Books 2000 ISBN 978 0 7176 1758 6.

    There has been recent interest in the construction of novel biological systems (sometimes termed Synthetic Biology) and some of these approaches may also be covered by the legislation.

    According to the route maps generated via this Tool Kit, and purely for illustrative purposes, human stem cell lines are generated before genetic modification. However, in the case of induced Pluripotent Stem (iPS) cells, genetic modification may take place prior to any stem cell line being generated. Regardless, any centre undertaking GM activities may need to notify the HSE. For individual activities, notification is only required where the activity presents a risk to human health or the environment. For further information, click here.

  • GMP (Good Manufacturing Practice)

    Good Manufacturing Practice is that part of quality assurance which ensures that medicinal products are consistently produced and controlled to the quality standards appropriate to their intended use and as required by the marketing authorisation (MA) or product specification. GMP is concerned with both production and quality control.

  • Growth Factor

    Growth factors are proteins capable of stimulating cellular proliferation and differentiation. Cell culture medium is often supplemented with specific growth factors in order to achieve a desired outcome.

  • GTAC (Gene Therapy Advisory Committee)

    The Gene Therapy Advisory Committee (GTAC) is a Research Ethics Committee within the National Research Ethics Service and is recognised by the UK Ethics Committee Authority. Under the Clinical Trials Regulations, all applications to conduct clinical trials of investigational medicinal products for gene therapy must be mde to GTAC (though GTAC may transfer an application to another REC where the trial is of low risk). GTAC also has UK-wide responsibility for ethical review of clinical trials involving other ATMPs or cell therapies derived from stem cell lines.

  • HFEA (Human Fertilisation & Embryology Authority)

    The Human Fertilisation and Embryology Authority is the UK's independent regulator overseeing the use of gametes and embryos in fertility treatment and research. The HFEA licenses fertility clinics and centres carrying out in vitro fertilisation (IVF), other assisted conception procedures and human embryo research.

  • Hospital Exemption

    Under the Regulation on ATMPs, products which are prepared on a non-routine basis and used within the same Member State in a hospital in accordance with a medical prescription for an individual patient will be exempt from the Regulation. Exempted products are required to comply with manufacturing, quality and pharmacovigilance standards which are defined at national level. Information about the arrangements that apply under the UK’s hospital exemption scheme has been published on the MHRA’s website. For further detail, click here.

  • HSE (Health & Safety Executive)

    The Health and Safety Executive operates and enforces legislation in Great Britain that aims to control the risks to human health and the environment arising from activities involving GMOs in containment under the Genetically Modified Organisms (Contained Use) Regulations 2000. Containment means that physical, chemical or biological barriers are used to limit contact between GMOs and humans or the environment. Contained Use facilities therefore include; laboratories, animal houses, plant growth rooms and glasshouses, industrial fermenters used for large scale production of enzymes or therapeutics, and facilities to contain genetically modified farm animals.

  • HTA (Human Tissue Authority)

    The Human Tissue Authority (HTA) is the UK's Competent Authority under the Quality and Safety Regulations and licenses the procurement, testing, processing, storage, distribution and import/export of tissues and cells for human application. 

  • Human Application

    In relation to tissues and cells, means use on or in a human recipient, including use in extracorporeal applications, but not including use for autologous graft (tissues and cells removed from and appliedin the same person within the same surgical procedure).

  • ICM (Inner Cell Mass)

    The inner cell mass (ICM) is a cluster of cells within the developing blastocyst from which all adult somatic cells arise.in vivo and from which embryonic stem cells can be derived in vitro.

  • IMP (Investigational Medicinal Product)

    Investigational Medicinal Product (IMP) means a pharmaceutical form of an active substance or placebo being tested, or to be tested, or used, or to be used, as a reference in a clinical trial, and includes a medicinal product which has a marketingauthorization but is, for the purposes of the trial(a) used or assembled (formulated or packaged) in a way different from the form of the product authorised under the authorization, (b) used for an indication not included in the summary of product characteristics under the authorization for that product, or (c) used to gain further information about the form of that product as authorised under the authorization. Treatments derived from stem cells can be considered to be medicinal products or investigational medicinal products, if they are being presented as exerting principally a pharmacological, immunological or metabolic action.

  • In vitro

    Laboratory research taking place outside of the body

  • In vivo

    Within the living body

  • Informed Consent

    Voluntary consent given by an individual ahead of participation in a study and only after being informed of the purpose, methods, procedures, as well as the potential risks and benefits of participation.

  • iPS (induced Pluripotent Stem) Cell

    Induced pluripotent stem (iPS) cells are generated by reprogramming differentiated cells to a pluripotent stem cell state. Reprogramming refers to a resetting of cell fate control mechanisms and is not fully understood. Human iPS cells were first generated by Shinya Yamanaka’s research group in 2007 using integrating retroviruses. Since then, reprogramming has been achieved using numerous cell types and techniques, including methods that do not directly involve genetic modification techniques. According to the route maps generated using this Tool Kit, and purely for illustrative purposes, human stem cell lines are generated before genetic modification. However, in the case of iPS cells, genetic modification may take place prior to any stem cell line being generated. Regardless, any centre undertaking GM activities may need to notify the HSE. For individual activities, notification is only required where the activity presents a risk to human health or the environment. For further information, click here.

  • IRAS (Integrated Research Application System)

    Integrated Research Application System is a single system for applying for the permissions and approvals required for health and care research in the UK.

  • Licence

    Before a medicine can be sold in the UK, a number of licences are essential. The companies that are involved in all stages of the manufacture and distribution of the product need to have licences (manufacturers and wholesale dealers licences). New products which are still in development also need a licence before they can be tested on human subjects (clinical trial authorisations). In addition, the product itself must have a licence called a Marketing Authorisation (formerly called a Product Licence). In the case of ATMPs, all marketing authorisations for product licensing, or marketing authorisations, takes place at a European level via the EMA. Medicines legislation (specifically The Medicines for Human Use (Marketing Authorisations Etc.) Regulations 1994/SI 3144) requires that medicinal products are licensed before they are marketed in the UK. There can be exceptions to this, however. See the Glossary terms 'Hospital Exemption' and 'Specials Legislation'.

  • MA (Marketing Authorisation)

    Medicines, which meet the standards of safety, quality and efficacy, are granted a marketing authorisation (previously a 'product licence'), which is normally necessary before they can be prescribed or sold.

  • MCB (Master Cell Bank)

    A culture of fully characterised cells distributed into containers in a single operation, processed together in such a manner asto ensure uniformity of cellular content of containers and stored in such a manner as to ensure stability.

  • Medicines Legislation

    The Medicines Act 1968 governs the manufacture and supply of medicine in the UK.  The legislation is due to be consolidated later in 2012. There are three categories: (1) Prescription Only drugs can be sold by a pharmacist if prescribed by a doctor, (2) Pharmacy medicines may be sold by a pharmacist without prescription, and (3)General sales list medicines may be sold without a prescription in any shop. The current relevant leglislation is given in European Directive 2001/83/EC relating to medicinal products for human use, amended by Directives 2002/98/EC, 2003/63/EC, 2004/24/EC and 2004/27/EC. For further information, click here. For medicines derived from stem cells (somatic stem cell medicinal products), derived from gene therapy (gene therapy medicinal products) or engineered from tissue (tissue engineered products), or in combination with a medical device, the 'ATMP Regulation' applies. For further information, click here.

  • MHRA (Medicines and Healthcare products Regulatory Agency)

    The Medicines and Healthcare products Regulatory Agency (MHRA) is a statutary agency charged with ensuring that medicines and medical devices work and are acceptably safe.

  • MP (Medicinal Product)

    Article 1 of Directive 2001/83/EC as amended defines a "medicinal product" as: "Any substance or combination of substances presented as having properties for treating or preventing disease in human beings; Any substance or combination of substances which may be used in or administered to human beings either with a view to restoring, corecting or modifying physiological functions by exerting a pharmacological, immunological or metabolic action, or to making a medical diagnosis". Treatments derived from stem cells can be considered to be medicinal products or investigational medicinal products, if they are being presented as exerting principally a pharmacological, immunological or metabolic action.

  • MRC (Medical Research Council)

    The Medical Research Council (MRC) is a publicly-funded organisation dedicated to improving human health and supports research across the entire spectrum of medical sciences, in universities and hospitals, in its own units and institutes in the UK, as well as in Africa.

  • NRES (National Research Ethics Service)

    The National Research Ethics Service is part of the Health Research Authority in England. It has a mission to protect the rights, safety, dignity and well-being of research participants; and to facilitate and promote ethical research that is of potential benefit to participants, science and society. NRES includes Research Ethics Committees established in England. Click here for further information.

  • Pluripotent

    A cell is pluripotent if it has the potential to develop into derivatives of all three embryonic germ layers: ectoderm, endoderm and mesoderm.

  • Post-marketing Surveillance

    Regulation (EC) No 1394/2007 of the European Parliament and of the Council on Advanced Therapy medicinal products introduces additional provisions to those laid down in Directive2001/83/EC and Regulation (EC) 726/2004. Article 14 (4) of Regulation (EC) No 1394/2007 specifically requests the European Medicines Agency to draw up detailed guidelines relating to the post authorisation follow-up of efficacy and adverse reactions, and risk management. The EMA has recently issued this guideline to meet this request and to complement existing guidelines in the area.

  • Product Licence

    Before a medicine can be sold in the UK, a number of licences are essential. The companies that are involved in all stages of the manufacture and distribution of the product need to have licences (manufacturers and wholesale dealers licences). New products which are still in development also need a licence before they can be tested on human subjects (clinical trial authorisations). In addition, the product itself must have a licence called a Marketing Authorisation (formerly called a Product Licence). In the case of ATMPs, all marketing authorisations for product licensing, or marketing authorisations, takes place at a European level via the EMA. Medicines legislation (specifically The Medicines for Human Use (Marketing Authorisations Etc.) Regulations 1994/SI 3144) requires that medicinal products are licensed before they are marketed in the UK. There can be exceptions to this, however. See the Glossary terms 'Hospital Exemption' and 'Specials Legislation'.

  • Provenance

    Knowledge of the consent and source of material, cells and reagents used in the derivation of cells.

  • QP (Qualified Person)

    The primary legal responsibility of the Qualified Person (QP) is to certify batches of medicinal products prior to use in a clinical trial (human medicines products only) or prior to release for sale and placing on the market (human and veterinary medicinal products). For more information, click here.

  • Quality and Safety Regulations

    The European Union Tissue and Cells Directives (EUTCD) set out to establish a harmonised approach to the regulation of tissues and cells across Europe. The EUTCD is made up of three Directives, the parent Directive (2004/23/EC) which provides the framework legislation and two technical directives (2006/17/EC and 2006/86/EC), which provide the detailed requirements of the EUTCD. The Directives were fully implemented into UK law on 5 July 2007, via the Human Tissue (Quality and Safety for Human Application) Regulations 2007 (The ‘Quality and Safety Regulations'). For further information, click here.

  • REC (Research Ethics Committee)

    Research Ethics Committees have been established throughout the UK for many years with the purpose of safeguarding the rights, dignity and welfare of people participating in research in the NHS (and outside the NHS where ethical review is required by law). Potential research participants at any NHS organisation in the UK will come under the protection of a REC. The REC is entirely independent of the researcher and the organisations funding and hosting the research. RECs are recognised by the United Kingdom Ethics Committee Authority (UKECA) for the review of clinical trials of investigational medicinal products (CTIMPs), in accordance with The Medicines for Human Use (Clinical Trials) Regulations 2004, for the class of research and geographical area indicated. Recognised RECs can also review non-CTIMP research. There are three types of recognised RECs: Type 1 RECs-recognised for review of phase I Clinical Trials of Investigational Medicinal Products (CTIMPs) in healthy volunteers only; Type 2 RECs-recognised for review of Clinical Trials of Investigational Medicinal Products (other than phase I trials in healthy volunteers) taking place within a single domain (new applications are no longer allocated to Type 2 RECs but their recognition remains relevant for review of substantial amendments to ongoing trials); Type 3 RECs -recognised for review of Clinical Trials of Investigational Medicinal Products (other than phase I trials in healthy volunteers) taking place in more than one domain anywhere in the UK. GTAC acts as the specialist REC for UK clinical trials involving either gene therapy or cell therapies derived from stem cell lines.

  • SAE/SAR (Serious Adverse Event/Serious Adverse Reaction)

    For Medicinal Products, Serious adverse event or serious adverse drug reaction or unexpected serious adverse reaction is:

    Any adverse event, adverse reaction or unexpected adverse reaction, respectively, that (a) results in death, (b) is life-threatening, (c) requires hospitalisation or prolongation of existing hospitalisation, (d) results in persistent or significant disability or incapacity, or (e) consists of a congenital anomaly or birth defect.

    A Sponsor shall ensure that all relevant information about a suspected unexpected serious adverse reaction (SUSAR) which occurs during the course of a clinical trial in the United Kingdom and is fatal or life-threatening is reported as soon as possible to the MHRA, the competent authorities of any EEA State, other than the United Kingdom, in which the trial is being conducted, and the relevantEthics Committee. This needs to be done not later than seven days after the Sponsor was first aware of the reaction. Any additional relevant information should be sent within eight days of the report.

    A Sponsor shall ensure that a suspected unexpected serious adverse reaction (SUSAR) which is not fatal or life-threatening is reported as soon as possible, and in any event not later that 15 days after the Sponsor is first aware of the reaction, to the MHRA, the competent authorities of any EEA State, other than the United Kingdom, in whichthe trial is being conducted and the relevant Ethics Committee.

    Under the European Union Tissue and Cells Directive, the HTA has set up a system for tissue establishments to report serious adverse events and reactions (http://www.hta.gov.uk/licensing/adverse_event_and_reaction_reporting.cfm). For these purposes the following definitions apply:

    Serious Adverse Event (SAE) ‘serious adverse event' means any untoward occurrence associated with the procurement, testing, processing, storage and distribution of tissues and cells that might lead to the transmission of a communicable disease, to death or life-threatening, disabling or incapacitating conditions for patientsor which might result in, or prolong, hospitalisation or morbidity.

    Serious Adverse Reaction (SAR) means an unintended response, including a communicable disease, in the donor or in the recipient associated with the procurement or human application of tissues and cells that is fatal, life threatening, disabling, incapacitating or which results in, or prolongs, hospitalisation or morbidity.

  • Somatic Cell

    A somatic cell is a cell derived from any of the three embryonic germ layers: ectoderm, endoderm or mesoderm. Gametes (ova and sperm) and cells making up extraembryonic tissues are not somatic cells.

  • Somatic Cell Therapy Medicinal Product

    'Somatic cell therapy medicinal product' means a biological medicinal product which has the following characteristics: (a) contains or consists of cells or tissues that have been subject to substantial manipulation so that biological characteristics, physiological functions or structural properties relevant for the intended clinical use have been altered, or of cells or tissues that are not intended to be used for the same essential function(s) in the recipient and the donor; (b) is presented as having properties for, or is used in or administered to human beings with a view to treating, preventing or diagnosing a disease through the pharmacological, immunological or metabolic action of its cells or tissues.

    Cells or tissues shall be considered ‘engineered' if they fulfil at least one of the following conditions:

    (1) the cells or tissues have been subject to substantial manipulation, so that biological characteristics, physiological functions or structural properties relevant for the intended regeneration, repair or replacement are achieved. The following manipulations, in particular, are not be considered as substantial manipulations: cutting, grinding, shaping, centrifugation, soaking in antibiotic or antimicrobial solutions, sterilization, irradiation, cell separation, concentration or purification, filtering, lyophilization, freezing, cryopreservation, vitrification.

    (2) the cells or tissues are not intended to be used for the same essential function or functions in the recipient as in the donor.

  • Somatic Stem Cell (or Adult Stem Cell)

    The term ‘somatic stem cell’ is preferred as this type of cell exists not only in the adult body but also in specific tissues during development, including foetal stages. Unlike pluripotent stem cells, somatic stem cells are typically multipotent, meaning that they can differentiate into a limited number of cell types depending on the tissue in which they reside. In vivo, somatic stem cells replace and replenish cells that are lost by depletion or damage in their tissues of origin. Somatic stem cells can be isolated from a variety of tissues, including bone marrow, blood, eye, brain, fat, cartilage and skeletal muscle.

  • Specials Legislation

    Medicines legislation (specifically The Medicines for Human Use (Marketing Authorisations Etc.) Regulations 1994/SI 3144) requires that medicinal products are licensed before they are marketed in the UK. However, some patients may have special clinical needs that cannot be met by licensed medicinal products. So that these special clinical needs may be met, the law allows manufacture and supply of unlicensed medicinal products (commonly known as 'specials') subject to certain conditions. The conditions are that there is a bona fide unsolicited order, the product is formulated in accordance with the requirement of a doctor or dentist registered in the UK, and the product is for use by their individual patients on their direct personal responsibility. If a 'special' is manufactured in the UK, the manufacturer must hold a manufacturer's (specials) licence issued by the MHRA. There are also specific requirements that apply to ‘specials’ that are imported in to the UK.

  • Steering Committee for the UK Stem Cell Bank and for the Use of Stem Cell Lines

    The Steering Committee for the UK Bank and for the Use of Stem Cell Lines must satisfy itself that human embryonic stem cell (hESC) lines used in the UK have been ethically sourced with informed donor consents; are to be used for valuable and ethical research projects reflecting the requirements of the HFEA regulations. The Committee seeks to approve all research with undifferentiated human embryonic stem cells.

  • Stem Cell

    Stem cell is a general term used to describe an unspecialised cell. Somatic stem cells exist in vivo in certain tissues at various stages of development. Other types of stem cells are in vitro phenomena, including embryonic stem cells and induced pluripotent stem cells. Embryonic stem cells are derived from the inner cell mass of a 4-6 day embryo. Induced pluripotent stem cells can be generated by a variety of methods for reprogramming differentiated cells to a stem cell state. Treatments derived from stem cells may be considered to be medicinal products or investigational medicinal products if they are being presented as exerting principally a pharmacological, immunological or metabolic action.

  • Stem Cell Culture

    A stem cell culture is a population of stem cells that is maintained in vitro.

  • Stem Cell Line

    A stem cell line is an established culture of unspecialised cells derived from a single parental cell, or group of parental cells, that can (1) proliferate in vitro for a prolonged period under the appropriate culture conditions and (2) maintain the potential to differentiate into more specialised cell types.

  • SUSAR (Suspected Unexpected Adverse Drug Reaction)

    As defined in Clinical Trials Regulations, a Suspected Unexpected Serious Adverse Reaction (SUSAR) is an unexpected Adverse Reaction that (a) results in death, (b) is life-threatening, (c) requires hospitalisation or prolongation of existing hospitalisation, (d) results in persistent or significant disability or incapacity, or (e) consists of a congenital anomaly or birth defect. A SUSAR which occurs during the course of a clinical trial in the UK must be reported to the MHRA and the appropriate Research Ethics Committee, as well as the competent authorities of any European Economic Area State, other than the UK, in which the trial is being conducted; and not later that 15 days after the sponsor is first aware of the reaction.

  • TEP (Tissue Engineered Product)

    ‘Tissue engineered product' as defined in Article 2 1 (b) of the ATMP Regulation means a product that (a) contains or consists of engineered cells or tissues, and (b) is presented as having properties for, or is used in or administered to human beings with a view to regenerating, repairing or replacing a human tissue. A tissue engineered product may contain cells or tissues of human or animal origin, or both. The cells or tissues may be viable or non-viable. It may also contain additional substances, such as cellular products, bio-molecules, biomaterials, chemical substances, scaffolds or matrices. Products containing or consisting exclusively of non-viable human or animal cells and/or tissues, which do not contain any viable cells or tissues and which do not act principally by pharmacological, immunological or metabolic action, is excluded from this definition.

    Cells or tissues shall be considered ‘engineered' if they fulfil at least one of the following conditions:

    (1) the cells or tissues have been subject to substantial manipulation, so that biological characteristics, physiological functions or structural properties relevant for the intended regeneration, repair or replacement are achieved. The following manipulations, in particular, are not be considered as substantial manipulations: cutting, grinding, shaping, centrifugation, soaking in antibiotic or antimicrobial solutions, sterilization, irradiation, cell separation, concentration or purification, filtering, lyophilization, freezing, cryopreservation, vitrification.

    (2) the cells or tissues are not intended to be used for the same essential function or functions in the recipient as in the donor.

  • Tissue Establishment

    Location where the activities of processing, preservation, storage or distribution of human tissue and cells are undertaken.

  • Traceability

    Ability to account for the whereabouts of donated tissues or cells and their status at all points from initial collection right through to transplantation or disposal.

  • UK Stem Cell Bank

    The UK Stem Cell Bank Provides a repository for human embryonic, foetal and adult stem cell lines as part of UK governance for the use of human embryos for research and therapeutic applications. All UK derived embryonic stem cell lines must be offered for deposit in the Bank through the Steering Committee for the UK stem Cell Bank and for the Use of Stem Cells as a condition of the HFEA license.

  • WCB (Working Cell Bank)

    A culture of cells derived from the master cell bank and intended for use in the preparation or production cell cultures.