Seminar PC3: Hospital pharmacists involved in APTM and in Risk Assessment

Linked to EAHP Statements

Section 1 – Introductory Statements and Governance: Statements 1.2, 1.4
Section 3 – Production and compounding: Statement 3.3, 3.4, 3.5, 3.6
Section 4 – Clinical Pharmacy Services: Statements 4.6, 4.7
Section 5 – Patient Safety and Quality Assurance: Statements 5.1, 5.2, 5.3, 5.4, 5.5, 5.6, 5.7, 5.8, 5.9, 5.10, 5.11
Section 6 – Education and Research: Statements 6.1, 6.2, 6.3, 6.4, 6.5

ACPE UAN: 0475-0000-18-017-L04-P. A knowledge based activity.

Abstract

A medicine which is based on genes, cells or engineered tissues, meets the scientific criteria for defining Advanced Therapy Medicinal Products (ATMPs). New challenges are arising from differences in terms of donation, procurement, testing, clinical trials, and GMP requirements as compared to current medicines. Therefore, novel analytical and technological equipment and methodologies rarely seen previously in hospital pharmacies will need to be installed, qualified, instructed and safely run. Communication-lines with professionals such as cell biologists, biosafety officers and responsible persons for tissue and cell procurement need to be established. This might be feasible in the course of the general technical and electronical revolution in which the younger generations grow up. Nevertheless, adaptations to educational pharmacy curricula and specialisation must be implemented. This seminar will outline the provisional future and importance of this class of medicines and how hospital pharmacists should manage supply and use of ATMPs.

Working in a highly-regulated environment such as a hospital pharmacy comprises risk assessments and calculations. Errors and incidences can arise at every step from production to procurement, storage, distribution, and clinical trials. GMP, GCP, CAPA (corrective and preventive action), are designed to steer processes in a way that errors are prevented and, if occurring, are effectively corrected. Qualified persons (QP) must calculate risks, which might have a negative impact on the product quality and which are linked to their human resources, fixed and mobile infrastructure, equipment, processes in force in manufacturing, cleaning, disinfection, analytical, storage and distribution steps. The effectiveness of coping strategies must be proven by the QPs to inspectorates. The FMEA (failure mode and effect analysis) is a tool to effectively assess risks arising from such processes. It rates and ranks the risks by calculating the risk score as the product of its probability of occurrence, its importance in terms of danger, and its detection chances.

Learning objectives

After the session, the participant should be able to:

• Describe the scope of ATMP regulations;
• Outline the challenges of ATMPs for hospital pharmacists;
• Make out opportunities and threats of ATMPs;
• Anticipate future research and development as related to ATMPs;
• Describe the GMP-requirements on risk analysis;
• Outline challenges and limit risks as related to corrective and preventive actions (CAPA);
• Relate the number of patient cases with the importance of the risk;
• Conclude on the minimal risk prevention activities to be performed by different types of production units;
• Implement a suitable risk prevention activity in his/her own hospital; and
• Perform a FMEA for the assortment issued from own production and reconstitution.

Educational need addressed

Short half-life times of scientific knowledge are a challenge for professionals in practice. Human genome sequencing and recent developments of omics-based methods (genomics, proteomics, metabolomics, etc) as well as cell- and tissue engineering options have brought new insights in (patho-)biochemical and pharmacological mechanisms. With the transfer of this knowledge into practice, numbers of new opportunities as well as challenges arise from production, preparation, use, and risk management. To keep the pace and to be able to deal with therapeutic entities such as Advanced Therapy Medicinal Products (ATMPs), hospital pharmacists need regular update and upgrade on new pharmacotherapy options, technologies, and risks. Hospital pharmacists need to know how to identify and calculate risks arising from their specific scale of production according to GMP and PIC/s guidelines.

Keywords: ATMPs, gene therapy, cell therapy, engineered tissues, reconstitution, quality control, risk calculation, FMEA, quality assurance, qualified person, GMP, risk, PIC/s, FMEA