Table 4.
Comparison of EACTs with CU programs conducted in the United States and Europe
Japan | United States | Europe | |
---|---|---|---|
Type | Cohort |
Cohort Single patient |
Cohort Single patient (United Kingdom, France) |
System | Performed as a clinical trial |
Separate from clinical trials (Administration within 30 days of application feasible) Emergency use system available |
Separate from clinical trials (Prior application to the regulatory authority for each country) |
Target drugs | Investigational products for which there is high social demand and pivotal clinical trials have been conducted or are ongoing | Investigational products in the United States | Investigational products for which marketing authorization will be requested from the EMEA or those under investigation |
Target patients | Serious or life‐threatening diseases for which there are no alternative treatments |
Serious or life‐threatening diseases for which there are no alternative treatments The expected benefit from the study drug justifies the risk |
Serious or life‐threatening diseases for which there are no alternative treatments |
Period | From the completion of enrollment in pivotal clinical trials until approval |
Cohort: After safety confirmation and preliminary efficacy confirmation until approval Single patient: From before/after clinical trials until approval |
Cohort (Germany): Until 1 year after marketing approval |
Does not adversely affect the development of the investigational product (does not adversely affect the progress of clinical trials).
There are reasonable grounds for not being able to participate in clinical trials.
CU, compassionate use; EACTs, Expanded Access Clinical Trials; EMEA, European Medicines Evaluation Agency.