Link to EAHP Statements

• Section 1 - Introductory Statements and Governance: Statement - 1.1
• Section 3 - Production and Compounding: Statements - 3.3, 3.4
• Section 5 - Patient Safety and Quality Assurance: Statements - 5.2, 5.4, 5.6
• Section 6 - Education and Research: Statement - 6.4

ACPE UAN: 0475-0000-23-008-L04-P - A knowledge-based activity  

Abstract

With the release of Regulation (EC) No 1394/2007, a new framework for gene and cell therapy medicinal products and tissue-engineered products was established in the European Union. Advanced therapy medicinal products (ATMPs) are a dynamic and current topic for healthcare systems, with new products progressing to market at an increasing rate.

Advanced therapy medicinal products (ATMPs) are medicines for human use that are based on genes, tissues or cells. They offer groundbreaking new opportunities for the treatment of disease and injury. ATMPs can be classified into three main types: 

• gene therapy medicines: these contain genes that lead to a therapeutic, prophylactic or diagnostic effect. They work by inserting 'recombinant' genes into the body, usually to treat a variety of diseases, including genetic disorders, cancer or long-term diseases. A recombinant gene is a stretch of DNA that is created in the laboratory, bringing together DNA from different sources;
• somatic-cell therapy medicines: these contain cells or tissues that have been manipulated to change their biological characteristics or cells or tissues not intended to be used for the same essential functions in the body. They can be used to cure, diagnose or prevent diseases;
• tissue-engineered medicines: these contain cells or tissues that have been modified so they can be used to repair, regenerate or replace human tissue.

In addition, some ATMPs may contain one or more medical devices as an integral part of the medicine, which are referred to as combined ATMPs. An example of this is cells embedded in a biodegradable matrix or scaffold.

ATMPs are medicines and so by definition, they fall under the responsibility of the hospital pharmacist. The hospital pharmacist should therefore be involved in procurement, production in the hospital, reconstitution, quality control and logistics. The European Association of Hospital Pharmacist (EAHP) requires in its Position Paper on Pharmacy Preparations and Compounding that the management of ATMPs, as licensed medications, remains the responsibility of the hospital pharmacist. Pharmacists play an important role in the safe implementation of ATMPs. Wherever possible pharmacist advocate that medicines are issued to the clinical area in a ready-to-administer presentation. Some ATMPs arrive ready to administer but some require a preparation step to make them ready to administer to patients. The seminar will discuss the optimal location in which any preparation should be undertaken. It will discuss that this may vary depending on the type of ATMP and will ask whether pharmacy training is currently sufficient to safely handle cell or tissue-based products. Where preparation occurs in areas other than pharmacy aseptic suites it will discuss the oversight role of pharmacists who ensures, from a governance perspective, that the medicine is being handled in line with the Summary of Medicinal Product Characteristics or the Clinical Trial Protocol.

Learning objectives

After the session, participants should be able to:

• Explain the therapeutic, prophylactic and diagnostic effect of ATMPs
• Describe Regulatory Framework for ATMPs in Europe
• Apply the pharmacy-related aspects to implementation of ATMPs for use in clinical practice

Educational need addressed

To learn and understand the history, regulatory aspects, current clinical evidence, therapeutic, prophylactic and diagnostic indications, safety aspects of ATMPs and the role of the pharmacist in safe implementation of ATMPs.

Keywords: Advanced therapy medicinal products (ATMPs), regulatory aspects, efficacy, safety, implementation, hospital pharmacy