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.
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.
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).
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.
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.
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.
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
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.
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.
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 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.
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
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.
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 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.
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
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
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.
This document 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.
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
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.
Ex vivo gene therapy medicinal products are ATMPs, which require pharmacy oversight, even when being handled elsewhere within a healthcare organisation. This document 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)
The purpose of this document 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.
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
The purpose of this document 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
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
The aim of the Pan UK Pharmacy Working Group for ATMPs in producing this document is to clarify the regulatory perspective regarding out of specification ATMPs, and to provide guidance to NHS organisations which are provider sites for ATMPs, about governance in the event of an out of specification ATMP being supplied for one of their patients.
The cellular therapy and transplant programme modified existing and created a number of documents in order to deliver advanced therapies and comply with JACIE’s immune effector cell standards (edition 6.01)
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 to document useful medication restriction information for CAR-T Cell therapy.
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 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.
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.
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.
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
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
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.
This document 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
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