Advanced Therapies NHS Readiness Toolkit

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).

The HTA is the regulator for human tissue and organs, this includes activities using  human tissues or cells used as starting materials in the manufacture of ATMPs

Main pages from the EMA website focussed on ATMPs

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 webinar giving an overview of governance, operational and clinical challenges which need to be overcome in order to implement these novel medicines

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

The purpose of this document is to outline the key areas where chief pharmacists should focus pharmaceutical expertise prior to an organisation implementing any Tissue Engineered Product.

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)

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.

Governance structure organogram for supporting the introduction of ATMPs at the Christie Hospital

Terms of reference for specialist group focussing on the training and education requirements for ATMPs within the Christie Hospital

Terms of reference used for the ATMP board at The Christie Hospital including membership of the group

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