State-of-the-art knowledge on the regulation of advanced therapy medicinal products.
Patcharaphun KidpunWarit RuanglertboonRapeepun ChalongsukPublished in: Personalized medicine (2022)
Advanced therapy medicinal products (ATMPs) constitute therapeutic agents based on obtained cells, tissues or genes representing a novel treatment opportunity in medicine. In addition, ATMPs are administered into the cells or tissues of humans from the patient's own cells, donors, or genetically modified cells. Recently, the field of developing ATMPs has become a point of attention due to the clinical efficacy expected in defeating incurable diseases such as cancers and neurodegenerative disorders. Currently, there are two modes regarding the distribution of ATMPs. First, ATMPs that might be legally authorized for marketing. Second, the patients are able to access unapproved ATMPs through the hospital exemption (HE) or clinical practice program or through the compassionate use and expanded access program. The aim of this review is to discuss state-of-the-art knowledge on the regulation of ATMPs and provide regulatory recommendations.
Keyphrases
- induced apoptosis
- cell cycle arrest
- clinical practice
- healthcare
- end stage renal disease
- gene expression
- chronic kidney disease
- endoplasmic reticulum stress
- emergency department
- stem cells
- signaling pathway
- cell death
- quality improvement
- ejection fraction
- cell proliferation
- newly diagnosed
- peritoneal dialysis
- replacement therapy
- combination therapy
- adverse drug