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Under the new proposals, drug companies will do what trials they can in infected people, but can run more safety tests in healthy people, and prove efficacy through tests on bugs in laboratories.
We also feel the risk of the regulator refusing approval is low, based on (1) the fact the regulator effectively designed the trial for GW; (2) the regulator has previously stated that Sativex is safe, and today's trial was needed only to prove efficacy; and (3) the fact that today's trial is so clearly positive".
Moreover, a multicenter RCT would be feasible and would require 280 patients to prove efficacy.
With traditional drugs, clinical trials tend to include individuals whose cancer is at an advanced stage to prove efficacy.
The most direct method to prove efficacy is to compare an active treatment vs. no treatment or placebo control.
In addition, I'm told the startup has recently completed a Randomized Control Trial, the same type of trial drug companies use to prove efficacy of their products, and will publish the results later this year.
Similar(17)
Sorafenib has proven efficacy in metastatic renal cell carcinoma (mRCC).
The first-generation (nicardipine) agents have proven efficacy against hypertension.
Citicoline is the only neuroprotectant that has proved efficacy in patients with moderate to severe stroke.
The Flutter Valve (Varioraw SARL, Scandipharm Inc, Birmingham, AL) has proven efficacy in hypersecretive spontaneously ventilated patients.
Calcium phosphates (CaPs) biomaterials including biphasic calcium phosphate (BCP) ceramics have proven efficacy in numerous clinical indications.
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