For example, an insulin biosimilar could be approved based on a study in people with type 1 diabetes, but upon approval, it would receive approval for use in all patient populations for which the reference product has been approved (e.g., type 2 diabetes, pediatrics).
One contentious provision, for example, directs the FDA to assess whether new drug uses, or indications, could be approved based on "real-world evidence", such as observational studies.
The article shows how value of information analysis can be used to inform a range of policy questions including whether a new technology should be approved based on existing evidence, whether it should be approved but additional research conducted or whether approval should be withheld until the additional evidence becomes available.
It is also telling companies when a drug is unlikely to be approved based on current data.
Essentially, the FDA's scientific staff, as well as an external advisory committee, questioned the efficacy of eteplirsen and, despite its benign safety profile, recommended that it not be approved based on the extant clinical trial data.
The commission also wanted the cell towers to be approved after a gap in coverage was established, but because that process is lengthy and expensive, staff recommended that the cell towers be approved based on aesthetics, land-use compatibility and code compliance.
The ophthalmic drugs in our study cohort were approved based on a median of two pivotal trials, compared with a median of one pivotal trial per device approval.
The modeling is approved based on a large number of tests on 304 stainless steel and is compared with SWT and FS models.
Keytruda was approved based on what was essentially an extra-large Phase 1 trial involving 173 participants who all received the drug, with no control group.
The result is a "predicate creep," the report said, in which devices are approved based on information from older devices that often bear little resemblance to each other.
