Advanced Therapies NHS Readiness Toolkit

Information provided by the European Medicines Agency (EMA) on the European Union (EU) pharmaceutical legislation known as the Clinical Trials Regulation, entered into application on 31 January 2022.

Training and supporting materials provided by the European Medicines Agency (EMA) on how to use the Clinical Trials Information System (CTIS).

Examples and guidance provided to University of Manchester (UoM) personnel, for clinical trial set up (UoM sponsored and hosted trials).

UKCRF Network: Framework for conducting highest risk Phase 1 and Experimental Medicine Studies with NIHR CRFs and wider NHS Acute Trusts

The Health Research Authority’s (HRA) online guidance to share best practice on consent and participant information by providing examples and templates

The Health Research Authority’s online guidance for researchers and ethics committees on consent, and how to prepare materials to support this process.

Guidance on costing a clinical trial provided by NIHR.

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.

UK local information pack for participating NHS/Health and Social Care (HSC) organisations.

Advice on preparing and submitting applications on Integrated Research Application System (IRAS).

For new for new Clinical Trials of Investigational Medicinal Products (CTIMPs) and combined IMP/device trials. A step by step guide on how use use IRAS for a combined review.

Government advice on how to apply for a clinical trial including eligibility, phases, model IMPDs, costs and how to make changes to your application.

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.

Model Clinical Trials Agreement – This model agreement captures all the specific contractual requirements of ATMP commercial clinical trials for implementation in the NHS.

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.

Guidance produced by the Pan UK Pharmacy Working Group for ATMPs regarding the operational implications of the UK exit from the EU for NHS Pharmacy clinical trial sites, including ATiMPs

Advice produced by the Pan UK Pharmacy Working Group for ATMPs for Pharmacy sites on costings for clinical trials of ATMPs, using the NIHR interactive costing tool.

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.

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.

This guidance document presents a consensus view on standardised approaches to mononuclear cell procurement to reduce unnecessary complexity and variation

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)

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

Full job description for a lead clinical trials nurse focussing on advanced therapies

This paper highlights the major themes found in designing and applying flow cytometric analysis for cellular therapies and discusses practical solutions to common problems

This training webinar gives an overview of the preparations required prior to treating patients with ATMPs

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)

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 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

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 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.

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

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 documents accompanies the ATMP model clinical trials agreement and can be use as a guide to allow the proper use of the model agreements.