Advanced Therapy Medicinal Products (ATMPs) are a category of innovative medical products that includes gene therapies, cell therapies, and tissue-engineered products. These therapies are designed to treat or potentially cure diseases by using advanced technologies to manipulate the patient’s genes, cells, or tissues. Comparing ATMPs to other complex products, such as traditional small molecule drugs and biologics, reveals several key differences:
Mechanism of Action:
- ATMPs: ATMPs work by directly altering the patient’s genes, cells, or tissues to achieve therapeutic effects. For example, gene therapies replace or modify malfunctioning genes, cell therapies involve the transplantation of functional cells, and tissue-engineered products replace damaged tissues.
- Other Complex Products: Traditional small molecule drugs and biologics usually work through interactions with specific molecules, receptors, or pathways in the body to achieve therapeutic effects.
- ATMPs: Manufacturing ATMPs involves complex processes such as genetic modification, cell culture, and tissue engineering. These processes are often individualized for each patient, making scalability and consistency challenging.
- Other Complex Products: Small molecule drugs and many biologics are typically produced through well-established manufacturing processes, allowing for greater scalability and consistent quality.
- ATMPs: Many ATMPs are designed to be personalized for each patient, as they often involve the use of the patient’s own cells or genetic material to create the therapy.
- Other Complex Products: Traditional drugs and biologics are generally not personalized and are designed for broader populations.
- ATMPs: ATMPs have the potential to provide curative or long-lasting effects for certain diseases by addressing underlying genetic or cellular defects.
- Other Complex Products: Traditional drugs often provide symptomatic relief or manage diseases without necessarily addressing the root cause.
- ATMPs: Regulatory authorities have developed specific pathways for the approval and regulation of ATMPs due to their unique nature. These pathways consider the complexity of the therapies and the need for balancing innovation with patient safety.
- Other Complex Products: Traditional drugs and biologics follow well-established regulatory pathways based on their respective characteristics.
Clinical Trials and Development:
- ATMPs: Clinical trials for ATMPs often involve small patient populations and require longer follow-up periods to assess long-term safety and efficacy.
- Other Complex Products: Clinical trials for traditional drugs and biologics may involve larger patient populations and shorter follow-up periods in comparison.
Safety and Risks:
- ATMPs: The use of ATMPs carries unique safety considerations, such as the potential for unintended genetic modifications, immune responses, and unpredictable cellular behaviour.
- Other Complex Products: Traditional drugs and biologics have their own safety considerations, but they are generally more understood due to their longer history of use.
QP Release of ATMPs
- Due to ATMPs being live cells the shelf life is days rather than months / years like traditional medicines. To administer the product to a patient before the product expires a provisional QP Release / Parametric Release is required as final Sterility testing can take up to 14 days. The final QP release certificate is generated only when the QP receives confirmation that all tests have passed.
“Parametric Release and Sterilisation Parametric release is based on evidence of successful validation of the manufacturing process and review of the documentation on process monitoring during manufacturing, without direct measurement of quality attributes.
It can be used as an operational alternative to end product testing for the drug product in certain cases when approved by the competent authority. Sterility testing for terminally sterilized drug products is one example.
(Parametric release is referred to in the European Pharmacopoeia monograph ”Methods of preparation of sterile products”9 . This states ”When a fully validated terminal sterilisation method by steam, dry heat or ionising radiation is used, parametric release, that is the release of a batch of sterilised items based on process data rather than on the basis of submitting a sample of the items to sterility testing, may be carried out, subject to the approval of the competent authority.”)
In summary, ATMPs stand out from other complex products due to their mechanism of action, manufacturing complexity, personalisation, treatment approach, regulatory pathway, clinical trial characteristics, and safety profile. These differences reflect the innovative nature of ATMPs and the challenges associated with harnessing advanced technologies for therapeutic purposes.
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