Introduction
Advanced Therapy Medicinal Product (ATMP) are a rapidly growing category of complex, innovative treatments—including gene therapies, somatic cell therapies and tissue-engineered products—that pose specific challenges to the design and conduct of preclinical and clinical trials. For instance, manufacturing requirements, short shelf life and eventual biosafety considerations of an investigational ATMP require tight controls on logistical arrangements for transportation, storage and administration. Additionally, risks associated with potential long-term effects of an ATMP may necessitate ongoing monitoring of clinical trial participants for many years after therapy is completed. And while the general principles of the International Council for Harmonization of Technical Requirements for Pharmaceuticals (ICH) Good Clinical Practices (GCP) apply to clinical trials with ATMPs, in some cases additional ATMP-adapted GCP must be followed. Additional measures may also be necessary, such as ensuring traceability for ATMPs that contain cells or tissues of human origin, training on upstream intervention of subjects and/or administration procedures, and others. Regulatory guidance on the conduct of clinical trials involving ATMPs is regularly issued. This paper explores the implications of recent guidance on ATMP development and identifies some key areas that sponsors should keep in mind to mitigate risks in the ATMP development process.
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