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Most orphan drugs submitted for regulatory approval are based on RCTs that follow generally accepted rules and guidance.
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There was no significant relationship between annual cost and year of approval for drugs approved for treatment of acute lymphoblastic leukaemia (p=0.38); however, exclusion of the most recently approved drug of which approval was based on a historical perspective (Xaluprine) resulted in a significant relationship being observed (p=0.01).
At most, he can approve positions taken by management and this approval is based on scanty facts, not on time-consuming analysis.
This controversy emanates from approval being based on a single randomized controlled trial (RCT) (PROWESS) and particularly on one subgroup analysis.
The FDAs approval was based on several recent studies.
Owing to limited clinical data, the approval was based on eteplirsen's effect on dystrophin expression.
The approval was based on three separate studies that involved about 1,000 patients with CML.
However, its approval is based on an environmental study the US Forest Service conducted more than 25 years ago, in 1986.
The approval was based on data from a nearly 18,000-patient study, called Jupiter, financed by AstraZeneca.
That approval was based on a 45-person trial that lacked a control group for comparing how well stents worked.
The FDA has said its approval was "based on sound science and a comprehensive review" and that regulators are confident the genetically altered fish is safe to eat.
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