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Looking at the original paper, the author does the analysis looking at breakthrough drug trial design, but doesn't really talk about what...

Looking at the original paper, the author does the analysis looking at breakthrough drug trial design, but doesn't really talk about what it means. Of the 46 drugs approved under breakthrough designation, 25 were for oncology. The FDA often allows for a lot of flexibility with trial sign in oncology due to the high unmet need. The author mentions a lack of trials being double-blinded, having a placebo or active comparator and clinical outcome vs. surrogate marker. All of this is quite common outside of the breakthrough drug designation. Nothing new here. Double-blinding a trial isn't as big a deal when what you are measuring is not impact by observer bias (tumor size). In addition, if you're measuring a surrogate marker (that is an accepted proxy for clinical outcomes), you don't need a comparator arm. I don't think any of these findings should be all that surprising or concerning.

Posted by refurb on Aug 20, 2018, 6:02 PM

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Looking at the original paper, the author does the analysis looking at breakthrough drug trial design, but doesn't really talk about what...

refurbAug 20, 2018, 6:02 PM

Looking at the original paper, the author does the analysis looking at breakthrough drug trial design, but doesn't really talk about what it means. Of the 46 drugs approved under breakthrough designation, 25 were for oncology. The FDA often allows for a lot of flexibility with trial sign in oncology due to the high unmet need. The author mentions a lack of trials being double-blinded, having a placebo or active comparator and clinical outcome vs. surrogate marker. All of this is quite common outside of the breakthrough drug designation. Nothing new here. Double-blinding a trial isn't as big a deal when what you are measuring is not impact by observer bias (tumor size). In addition, if you're measuring a surrogate marker (that is an accepted proxy for clinical outcomes), you don't need a comparator arm. I don't think any of these findings should be all that surprising or concerning.

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