New JAMA Study Confirms Importance of Trial Design for FDA Approval

Posted by Cytel Consulting

Apr 24, 2014 11:00:00 AM

Here at Cytel we firmly believe that if you don’t get the design of a clinical program right, then nothing else matters. A study recently published by the Journal of the American Medical Association, once again confirms the vital importance of proper trial design for the timely approval of a new drug. The article reports that nearly half of all submitted NME applications fail upon first submission, and only half of those that fail are eventually approved. The median approval delay is a costly 435 days. At least 53.6% of the trials eventually approved would have benefitted from improved study design: 24 of the unsuccessful first-time applications (15.9%) resulted from uncertainties related to dose selection; 20 of the rejections (13.2%) were due to choice of study end points that failed to display a clinically meaningful effect; 20 of the rejections (13.2%) were a consequence of inconsistent results when different end points were tested; and 17 (11.3%) demonstrated inconsistent results when different trials or study sites were compared. Moreover, 89 (58.9 %) showed inadequate efficacy, raising a question about whether the dose had been selected properly.

Cytel has biostatisticians with broad experience in clinical trial and clinical development program design, including adaptive designs and designs for efficient dose-finding. We are eager to collaborate on your clinical programs to enhance their probabilities of success.

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Sacks LV, Shamsuddin HH, et al., 'Scientific and Regulatory Reasons for Delay and Denial of FDA Approval of Initial Applications for New Drugs, 2000-2012,' JAMA. 2014 311(4):378-384. 

Please note that to access the full article you must click the button on the top right of the JAMA website, which says, ‘Read the current issue for FREE’

 

 

Topics: Cytel Consulting, Trial Design, Efficacy, Regulation, Safety

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