This Roadmap Tool sets out the key steps and activities in the end-to-end pathway for Advanced Therapy Medicinal Products (ATMPs) in England from non-clinical research through to treatment provision & monitoring
The AAC is bringing together industry, government, regulators, patients and the NHS to remove barriers and accelerate the introduction of ground-breaking new treatments and diagnostics which can transform care, including ATMPs
This exemplar guideline suggests roles and responsibilities between CAR T cell referring centres and treatment centres. It also outlines common late adverse effects that may occur when patients have returned to their referring centre.
E-learning on the NHS e-learning platform e-learning for healthcare (registration required) giving the user a refresher of standard clinical trial studies and explains the additional features which impact on the design of trials for investigational advanced therapy medicinal products (ATIMPs) and how they can be addressed. Roles and responsibilities, governance and sources of support for ATIMP clinical trials are also outlined.
A guidance checklist for NHS staff in preparation for ATMP starting material procurement site qualification. This document would be best utilised when beginning to set up a service for procurement of a new cell or tissue type but could be used as a checklist for existing cell or tissue type procurement
An example SOP from University Hospitals Bristol and Weston NHS Foundation Trust to provide a safe and effective framework by which lymphodepletion can be performed in the outpatient setting, prior to admission for CAR-T Cell infusion.
This document summarises results from the safety testing conducted by Alcyomics of two of the exemplar products; tolerogenic dendritic cells and Anti-SARS-Cov-2 T cell product. A full report is expected to be published later on in 2022 in formal journal.
A guidance document providing an oversight of the purposes and contents of a Quality Technical Agreement (QTA) for use between advanced therapy clinical trial Sponsor and Participating Organisation. The document set outs a proposed standardised format for advanced therapy clinical trial QTA with example roles and responsibilities.
Transplantation of insulin-producing cells purified from a donor pancreas is established within the NHS. This is restricted to people with life-threatening complications of type 1 diabetes as liberation from injected insulin cannot yet be guaranteed and life-long immunosuppression is needed. The potential for harnessing regenerative medicine and tissue engineering to deliver truly transformative outcomes is discussed.
Hospital pharmacists are likely to be faced with CAR-T as licensed medicines in the near future but should also be prepared to support commercial and academic clinical trials of novel CAR-T and this briefing document aims to address all scenarios.
An overview of points to consider and SOPs required when establishing a new pathway for the collection of tissues to isolate tumour infiltrating lymphocytes (TIL). Relevant for both research and clinical tissue collection.
This document produced by the Pan UK Pharmacy Working Group for ATMPs provides guidance on the management of acute CAR-T cell toxicities to help inform the development of local guidelines and to standardise the management of acute CAR-T cell toxicities across the UK. This is a consensus guideline developed with input from the pharmacists and consultants from the first and second wave CAR-T centres.
The guideline describes specific aspects of pharmacovigilance, risk management planning, safety and efficacy follow-up of authorised ATMPs, as well as some aspects of clinical follow-up of patients treated with such products.
This publication reports on a study to assess institutional readiness (IR) of NHS sites that form part of the Northern Alliance Advanced Therapy Treatment Centre to deliver advanced therapy medicinal products, and discusses the development of an assessment tool to support self-assessment of IR in healthcare institutions
Ex vivo gene therapy medicinal products are ATMPs, which require pharmacy oversight, even when being handled elsewhere within a healthcare organisation. This document produced by the Pan UK Pharmacy Working Group for ATMPs outlines the key areas where chief pharmacists should focus pharmaceutical expertise prior to an organisation implementing any ex vivo (cell based) Gene Therapy Medicinal Product (GTMP)
Gene therapies for the eye often lead the field of advanced therapeutics, introducing exciting new technologies and techniques to the clinic. Topics include the first FDA approved gene therapy (Luxturna), gene editing trials for inherited vision loss, and how a protein that helps algae move is being repurposed into vision saving technology.
A Pharmacy Working Group for ATMPs resource. Pharmacy is often the first port of call when an organisation wishes to use a gene therapy and needs to provide pragmatic guidance to facilitate the introduction of these innovative products. This document is designed to give practical advice to centres wishing to undertake gene therapy trials and need to understand the requirements and recommendations for governance and, in particular to have in place a Genetic Modification Safety Committee. The document also gives practical advice for centres to help them understand operational requirements including deciding the optimal location for preparation.
This guidance summarises the authorisation process for Genetically Modified Organisms (GMO) as Investigational Medicinal Products (IMP) for contained use (CU) within the United Kingdom (UK). These submissions are unique to GMOs, in addition to Clinical Trial Authorisation (CTA) and Ethics
Guidance produced by the Pan UK Pharmacy Working Group for ATMPs, providing definitions of oversight and supervision and clarifies where each are required when implementing Advanced Therapy Medicinal Products
HSE (Health and Safety Executive) guidance to help organisations to choose the most appropriate regulatory procedure, particularly those wishing to undertake studies involving the administration to humans of substances based upon genetically modified microorganisms (GMMs)
A Pan UK Pharmacy Working Group for ATMPs resource.
Some medicines, including many ATMPs, achieve greatest stability at ultra-low temperatures. Therefore the use of -80°C and below is commonplace to achieve optimal product shelf-life. As the usage of ATMPs is increasing, Pharmacy departments are becoming required to receive shipments and to store products at ultra-low temperatures
An example SOP from University Hospitals Bristol and Weston NHS Foundation Trust to provide a framework by which high-dose melphalan and autologous stem cell rescue (autologous stem cell transplant, ASCT) can be performed in the outpatient setting
Horizon Scanning is the systematic process of identifying new medicines or technologies in development. This information is useful to clinical trials sponsors, ATMP developers and manufacturers, organisations aspiring to hold ATMP marketing authorisations, as well as to NHS group members and beyond.
The Specialist Pharmacy Service produces a range of information to support managed entry of new drugs into the NHS, to assist organisations in developing medicines management policies and to inform prescribing decisions when a product has been launched. The service provides ways to track the development of drugs from when key trials start until they launch in the UK.
When applying for HRA Approval you will need to check what supporting documents you will need to provide well in advance of when you expect to submit your application. This will avoid unnecessary delays to your application. This page details the required supporting documentation.
The training passport is designed for all pharmacists providing pharmaceutical care to HSCT patients including those undergoing autologous and allogeneic transplant, patients admitted to the HSCT unit with post-transplant complications and patients attending post-transplant out-patient clinics
The purpose of document produced by the Pan UK Pharmacy Working Group for ATMPs is to outline the key areas where chief pharmacists should focus pharmaceutical expertise prior to an organisation implementing any in vivo (virus based) Gene Therapy Medicinal Product (GTMP) e.g. IMP for Clinical Trials or Marketed ATMPs.
E-learning on the NHS e-learning platform e-learning for healthcare (registration required) giving the user an overview of the requirements for labelling human tissues and cells for use as starting materials for advanced therapy medicinal products using the ISBT 128 system.
The objectives of this SOP are to ensure products are correctly and consistently labelled to ensure the positive identification of each harvest and facilitate cell chain to track from donation to reinfusion. For products transferred to areas outside the Clinical Apheresis Unit, and those reinfused within the transplant programme.
There are a number of restrictions in medicines allowed (and wash-out periods recommended) for patients on CAR-T Cell therapy at each stage of the pathway (apheresis, lymphodepletion, CAR-T infusion). This guidance has been produced by the Pan UK Pharmacy Working Group for ATMPs to document useful medication restriction information for CAR-T Cell therapy.
A training needs package and competency guide for healthcare and academic professionals involved in the delivery and direct care of patients receiving advanced therapy treatment; it provides information and signposting to external resources for independent training, learning and further reading on a number of key areas relevant to these medicines.
The web-based interactive Costing Tool (iCT) provides a framework for transparent cost display and calculation to support swift local site budget negotiations when planning commercial trials in the NHS.
The NIHR Clinical Trial Routemap is an interactive colour-coded tool to help navigate through the legal and good practice arrangements surrounding setting up and managing a Clinical Trial of an Investigational Medicinal Product (CTIMP). The routemap distinguishes between legal and good practice requirements, and indicates which aspects of these are relevant to wider clinical research in general. It includes an overview of trial practices, along with more detailed information available at ‘stations’ along the route.
This webinar to showcases the Pan UK Pharmacy Working Group for ATMP’s pragmatic advice which clarifies the regulatory perspective regarding OOS ATMPs, and provides guidance to NHS organisations which are provider sites for ATMPs, about governance in the event of an OOS ATMP being supplied for one of their patients.
A Pan UK Pharmacy Working Group for ATMPs resource to clarify the regulatory perspective regarding Out of Specification (OOS) ATMPs, and to provide guidance to NHS organisations which are provider sites for ATMPs, about governance in the event of an OOS ATMP being supplied for one of their patients.
This guidance produced by the Pan UK Pharmacy Working Group for ATMPs describes the legal basis for associated activity and provides guidance for Pharmacy clinical trial sites who may need to outsource these services. The information provided will guide Pharmacy trial sites in establishing appropriate partnerships and understand the regulatory position with regard to the activities contracted.
Initially set up to facilitate the onboarding of Wave 3 CAR-T centres in England, this template Technical Agreement produced by the Pan UK Pharmacy Working Group for ATMPs is suitable for adaptation by NHS Pharmacies, when there is a requirement to outsource any aspect of cell or tissue based ATMP handling for ATMPs which hold a marketing authorisation.
The Pan UK Pharmacy Working Group (PWG) for ATMPs was established in 2018 to act as an expert and informed body to support the activities of UK Pharmacies to facilitate ATMP usage. This is a link to their homepage on the specialist pharmacy service webiste
A presentation of the findings from the CPROR team’s recent publication “Patient and public perspectives on cell and gene therapies: a systematic review” and the CPROR’s ongoing work to develop a an evidence-based toolkit to facilitate the design of patient-focused educational resources.
This review highlights the need for appropriate patient and public education on the various aspects of cell and gene therapies. Patient and public perceptions of these therapies, alongside evidence of clinical and cost-effectiveness, will be central to their uptake and use.
A Pharmacy Working Group of ATMPs resource. CAR-T treatments require governance and management by Chief Pharmacists. They present risks which Chief Pharmacists should ensure are minimised. Whilst collaboration with expert cellular product handling colleagues will be key operationally, it may present challenges for Pharmacy to embed this new working relationship
The purpose of this guidance produced by the Pan UK Pharmacy Working Group of ATMPs is to outline the responsibilities of pharmacists where preparation of advanced therapy medicinal products (ATMPs) holding marketing authorisations is required prior to administration in hospitals. Full compliance with the summary of product characteristics (SmPC) requirements is always recommended. This guidance clarifies where activities require the oversight of a pharmacist and distinguishes circumstances in which preparation under the supervision of a pharmacist would be a legal requirement. It provides definitions and distinction between the use of these terms.
To describe the procedure for managing CTPs used as ATMP which are manufactured outside The Christie NHS Foundation Trust, received, stored and issued by The Stem Cell Laboratory at The Christie Pathology Partnership which has been prescribed by an appropriate prescriber
As Gene Therapy Medicinal Products (GTMPs) and Gene Therapy Investigational Medicinal Products (GTIMPs) are Advanced Therapy Medicinal Products (ATMPs), it is important that organisations have a defined governance process in place for their approval and implementation. This document gives an example of a Trust Policy for approval of GTMPs/GTIMPs by a Trust Genetic Modification Safety Committee (GMSC).
This poster presentation is the product of a short live working group of ATMP industry partners and captures the non-label (not appearing on the product label) data produced during an exemplar apheresis process. The poster aims to demonstrate the ability for standardisation of data transfer from NHS to manufacturers.
E-learning on the NHS e-learning platform e-learning for healthcare (registration required) giving the user an overview of the procedural requirements for the procurement of human tissues and cells but does not discuss in depth the consent and ethics requirements
Developed by the CRUK Manchester Institute, this SOP describes the procedure for performing Quanterix SP-X ELISAs (Formerly known as Aushon Ciraplex and referred to simply as multiplex in this document). (Enzyme Linked ImmunoSorbant Assay) using the Quanterix SP-X imaging system (Formerly known as Aushon Cirascan).
This guidance from the Pan UK Pharmacy Working Group for ATMPs has been written in collaboration with the HTA and MHRA. It clarifies the regulatory requirements for healthcare organisations who are procuring (harvesting) patient’s own cells or tissues to be used as starting materials in the manufacture of an ATMP where the subsequent ATMP manufacture occurs outside of the EU. In the event of a no deal EU exit, it will also be relevant for ATMPs manufactured in the EU
This module introduces nitrogen vapour phase dry shippers and dry ice shipments, explains the hazards of using these low temperature transport vessels and describes how they can be used and stored safely.
In vivo gene therapies are a type of Advanced Therapy Medicinal Product. Many in vivo gene therapies use an adeno-associated vector (AAV) to deliver the genetically modified material into the cell. These shared care guidelines describe a typical pathway for an in vivo gene therapy utilising an AAV.
This webinar will focusses on genetically modified immune cells as a treatment choice for solid tumours discussing the use of tumour-infiltrating lymphocyte therapy to treat H&N cancer in the context of a clinical trial
The purpose of this document produced by the Pan UK Pharmacy Working Group for ATMPs is to outline the key areas where Chief Pharmacists should focus pharmaceutical expertise prior to an organisation implementing any Somatic Cell Therapy Medicinal Product (sCTMP). This document presents a flow diagram outlining a stepwise approach to implementing processes to prepare and administer sCTMPs. It is followed by checklists which relate to the various steps presented in the diagram
The purpose of this document is to provide clear guidance on the procedures to be followed during the processing of ovarian tumour tissue to establish a) a tumour infiltrating lymphocyte culture; (b) an autologous tumour cell culture; (c) a cryopreserved aliquot of tumour culture digest; (d) subsequent cryopreservation of TIL and autologous tumour cultures for future assays.
This SOP provides the basis for the standardised collection of solid tumour and fluid samples from cancer patients undergoing surgical treatment, where there is excessive tumour tissue available which can be collected without effecting the normal standard of care.
This example SOP considers general principles for the handling and administration of an example in vivo GMO gene therapy in a clinical area which can be adapted to accommodate local procedures or product-specific requirements.
SOP to ensure all nursing and medical staff involved in the infusion of cellular products are aware of their responsibilities, the procedure and any action to be taken in the event of adverse reactions or incident.
A guidance document providing an oversight of the purposes and contents of a Starting Material, Quality Technical Agreement (QTA) for use between customer and supplier. The document set outs a proposed standardised format for Starting Material QTA with example roles and responsibilities.
Prior to receiving CAR-T cell therapy, patients will receive lymphodepletion chemotherapy. CAR-T cell therapy and lymphodepletion regimes can increase the likelihood of certain side effects and infections so supportive medicines are recommended.
These consensus recommendations, prepared by the Clinical Pharmacy subgroup of the Pan UK Pharmacy Working Group for Advanced Therapy Medicinal Products (ATMPs), relate to supportive medicines recommended for adults receiving licensed CAR-T cell therapy.
Some ATMPs contain living cells. These cell-based therapies may be cryopreserved to extend their shelf life, requiring carefully controlled thawing prior to administration. Failure to thaw the product correctly may impair the integrity of the cells (viability and/or function) and the product may be rendered unfit for purpose. This document describes and example procedure for thawing cryopreserved ATMPs using a water bath.
E-learning on the NHS e-learning platform e-learning for healthcare (registration required) giving the user an introduction to the challenges posed when moving advanced therapies around a hospital and how effective teamwork is required
This document produced by the Pan UK Pharmacy Working Group of ATMPs describes the role of Pharmacy in overseeing the governance arrangements and ensuring that Advanced Therapy Medicinal Products (ATMPs) used are of the appropriate quality for their intended use.
The purpose of this document produced by the Pan UK Pharmacy Working Group for ATMPs is to outline the key areas where chief pharmacists should focus pharmaceutical expertise prior to an organisation implementing any Tissue Engineered Product.
The use of immune effector cell therapies can result in significant toxicities that are directly associated with powerful immune-mediated responses. The most frequently observed of these is cytokine release syndrome (CRS). CRS represents a life-threatening situation that requires early recognition of symptoms and signs and prompt intervention. This short training video can be used to support healthcare professionals and others to gain an introduction to CRS presentation, diagnosis and clinical management.
Cryopreservation is now integrated into the manufacturing workflow of approved ATMPs. However, a comprehensive end-to-end solution for large scale ATMP production is still under development. This document provides an overview of the current state of cryopreservation in ATMP workflows by: addressing where cryopreservation fits into the workflow, the rules and regulations, the major challenges of cryopreservation logistics; reviewing the effects of cryopreservation of starting materials on the final product safety and efficacy and how developing technologies are set to help optimize cryopreservation processes for scalable manufacturing.
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