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大概扫了一下，感觉上是现在研究结果不充分，但也没有彻底凉凉，估计星期四开会要讨论后面的几个问题。
Topic for Discussion #1: Protocol MSB-GVHD001 was a single-arm trial designed to determine
if the Day-28 ORR exceeded 45% for pediatric patients with SR-aGVHD grades B-D treated
with remestemcel-L. Although the null rate and hypothesis were prespecified in the SAP, there
were some limitations with regard to how 45% was chosen for the null rate, and it is uncertain as
to whether the data cited for use as historical controls are sufficient to establish the null
hypothesis for the purposes of quantitating a treatment effect in a single-arm trial of a new
therapy for SR-aGVHD in pediatric patients.
Given these limitations, what are the strengths and weaknesses of the study design?
Topic for Discussion #2: The primary endpoint results in MSB-GVHD001 were statistically
significant, the measured response was durable (median 54 days), and the study results were
consistent across subpopulations and secondary efficacy endpoints. However, the results of
Protocols 265 and 280, the two randomized trials, did not provide evidence of a treatment effect
for remestemcel-L in aGVHD even when reanalyzed using the efficacy endpoint of Day-28
ORR. In fact, a treatment effect has not been identified in any of the previous clinical trials
conducted in various disease entities, including: type 1 diabetes mellitus, Crohn’s Disease,
myocardial infarction, or severe chronic obstructive pulmonary disease.
Therefore, how are the results of one positive single-arm trial interpreted in a landscape of
multiple negative clinical trials, including several randomized, controlled trials that failed to
show a treatment effect of remestemcel-L?
Does the fact that Study 265 and Study 280 were conducted over ten years ago impact how they
should be considered in this context?
Is an additional clinical trial in the SR-aGVHD population required for confirmation of the
effectiveness of the product? What trial design trial would be required to provide evidence of
effectiveness in this indication?
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1995年的黑白街拍 (2011-3-4) 胡椒老罗 
发表于 2020-8-11 14:58
