Approval on Gold Standard, from extension!!! ANAVEX®2-73. In the study population, when participants with these variants (approximately 20 percent) were excluded, the remaining study participants (approximately 80 percent) showed improved scores on gold-standard tests of cognition (MMSE) and activities of daily living (ADCS-ADL) (p<0.05).
Including participants with milder disease (baseline MMSE ≥20) and excluding those with a SIGMAR1 variant resulted in an average improvement of +1.7 MMSE and +3.9 ADCS-ADL at week 57 compared to baseline.
To approve a new anticancer drug, the US Food and Drug Administration often requires randomized trials. However, several oncology drugs have been approved on the basis of objective end points without a randomized trial. We reviewed the long-term safety and efficacy of such agents.
The United States Food and Drug Administration has several mandates for developing oncology treatments, including the approval of claims made about the use of a particular drug.1 To approve a new product, the US Food and Drug Administration requires adequate and well-controlled trials in support of marketing claims, in addition to proof of efficacy and safety. Randomized clinical trials demonstrating a statistically significant improvement in survival are considered the “gold standard” for approval of anticancer drugs by the US Food and Drug Administration.2 However, using the randomized trial approach, several oncology drugs have been US Food and Drug Administration-approved on the basis of relatively small differences (ie, weeks to 2 to 3 months of survival or relapse-free survival).3–12
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