There is a widely held expectation of clinical advance with the development of gene and cell-based therapies (GCTs). Yet, establishing benefits and risks is highly uncertain. We examine differences in decision-making for GCT approval between jurisdictions by comparing regulatory assessment procedures in the United States (US), European Union (EU) and Japan. A cohort of 18 assessment procedures was analyzed by comparing product characteristics, evidentiary and non-evidentiary factors considered for approval and post-marketing risk management. Product characteristics are very heterogeneous and only three products are marketed in multiple jurisdictions. Almost half of all approved GCTs received an orphan designation. Overall, confirmatory evid...
Gene and cell-based therapies (GCTs) are highly innovative therapies and hold great promise in the t...
In Japan, academic clinical trials do not generally need to be conducted under good clinical practic...
Introduction: Advanced Therapy Medicinal Products (ATMPs) are innovative products receiving increasi...
There is a widely held expectation of clinical advance with the development of gene and cell-based t...
There is a widely held expectation of clinical advance with the development of gene and cell-based t...
Innovative characteristics of gene and cell-based therapy products (GCTs) often hinder standardized ...
Gene and cell-based therapies (GCTs) have the potential to revolutionize healthcare. Recently, genet...
Gene- and cell-based therapies (GCTs) offer potential new treatment options for unmet medical needs....
Gene- and cell-based therapies (GCTs) offer potential new treatment options for unmet medical needs....
Human cells and tissue products belong to a relatively new class of medical products. Therefore, lim...
AbstractHuman cells and tissue products belong to a relatively new class of medical products. Theref...
Currently, the problem of adopting viable human cell-based drugs – biomedical cell products (BCPs) –...
Developers of gene therapy products (GTPs) must adhere to additional regulation beyond that of tradi...
is known and objectives: In vitro companion diagnostic devices (CDx) provide information on pharmaco...
In the last decade, many clinical trials with gene- and cell-based therapies were performed and incr...
Gene and cell-based therapies (GCTs) are highly innovative therapies and hold great promise in the t...
In Japan, academic clinical trials do not generally need to be conducted under good clinical practic...
Introduction: Advanced Therapy Medicinal Products (ATMPs) are innovative products receiving increasi...
There is a widely held expectation of clinical advance with the development of gene and cell-based t...
There is a widely held expectation of clinical advance with the development of gene and cell-based t...
Innovative characteristics of gene and cell-based therapy products (GCTs) often hinder standardized ...
Gene and cell-based therapies (GCTs) have the potential to revolutionize healthcare. Recently, genet...
Gene- and cell-based therapies (GCTs) offer potential new treatment options for unmet medical needs....
Gene- and cell-based therapies (GCTs) offer potential new treatment options for unmet medical needs....
Human cells and tissue products belong to a relatively new class of medical products. Therefore, lim...
AbstractHuman cells and tissue products belong to a relatively new class of medical products. Theref...
Currently, the problem of adopting viable human cell-based drugs – biomedical cell products (BCPs) –...
Developers of gene therapy products (GTPs) must adhere to additional regulation beyond that of tradi...
is known and objectives: In vitro companion diagnostic devices (CDx) provide information on pharmaco...
In the last decade, many clinical trials with gene- and cell-based therapies were performed and incr...
Gene and cell-based therapies (GCTs) are highly innovative therapies and hold great promise in the t...
In Japan, academic clinical trials do not generally need to be conducted under good clinical practic...
Introduction: Advanced Therapy Medicinal Products (ATMPs) are innovative products receiving increasi...