Advanced Therapy Medicinal Products (ATMPs) are an exciting new therapeutic approach based on genes, cells, or tissues. They are a rapidly growing area of medicine offering groundbreaking new opportunities for the treatment of disease and injury, with the potential to provide marked and durable responses for a diverse array of serious diseases and disorders.
As with medicines, use of ATMPs is governed and controlled by the Medicines Act 1968 and the Human Medicines Regulations (2012). These regulations give requirements for the legal sale, supply, and administration of all medicines. Other legislation e.g. the Health and Safety Executive (HSE) must also be considered as these products may also be genetically modified.
Due to the specificity and personalisation of these products, these new medicines present several challenges to pharmacists who are the gate keepers for safe, effective, and quality use of medicines.
The challenges faced are:
- The high cost of drugs – This requires the adjustment of a provider of insurance for cover. These medicines can be up to £2.5 million for treatment. Many organisations have had to ensure that their insurance arrangements will cover the full cost of ATMPs.
- The responsibilities and high pressures placed on the pharmacist – This includes the accountable pharmacist for aseptic preparation e.g. of gene therapy medicinal products (GTMP), independent prescribers, training multi-disciplinary teams, and the supervision of preparation steps, including the thawing of human cells done at ward level. Processes classed as preparation include thawing, washing, reconstitution, and loading into a delivery device.
Most sites will complete clinical, technical and regulatory risk assessments, which defines and sets out how the Gene Therapy Medicinal Products (GTMP) will be used at the site, with considerations being made for handling.
Regarding the pharmacist role and responsibility in the delivery of ATMPs, consideration must be made to the ‘one off’ dosing nature of these products. Patients can only be treated once with these products as the body becomes sensitised after the product is administered. Hence, we must consider the impact of dispensing and preparation errors on:
- The cost of the drug in the event product must be resupplied.
- Lost/last chance for patient to be treated again with the same product.
- Secondary effects (e.g. secondary cancers) this is borne with the Multi-Disciplinary Team.
- ‘Batch of one’ production which is the impact of non-conforming products, ethical considerations for use of out of specification products.
- Personalised and patient specific medicines.
- Time and cells/tissues required to produce the dose.
In light of the gravity and implications of any pharmacy errors in delivery of these products, it may be worth getting pharmacists involved in this work and considering if their current arrangements for personal indemnity would be sufficient in this new context. The PDA will be able to advise on this and PDA members are encouraged to contact the PDA Member Support Centre.
By Shingi M Mutamba, Lead Pharmacist AT(I)MP and Accountable Pharmacist at the Pharmacy Advanced Therapy Unit of University Hospital Southampton NHSFT
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