Clinical Trial Risk in Type-2 Diabetes: Importance of Patient History

Emmanuel O. Aiyere1, Jay Silverberg2, Safina Ali3, Jayson L. Parker1

1Department of Biology, University of Toronto at Mississauga, Toronto, ON
2Sunnybrook Health Sciences Centre, Toronto, ON
3Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON, Canada


Purpose. To determine the risk of clinical trial failure for drugs developed for type-2 diabetes.  Methods. Drugs were investigated by reviewing phase I to phase III studies that were conducted between 1998 and February 2013. The clinical trial success rates were calculated and compared to the industry standard. The drugs were classified into GLP-1 receptor agonists, DPP-4 inhibitors, SGLT-2 inhibitors and “Other”. The exclusion criteria for drugs in this study: Drugs that were started in phase I studies prior to January 1998 for this indication and drugs whose primary indications were not for the control of blood glucose levels.  Results. Data was extracted from; there were a total of 131 drug candidates that fit our specified criteria, of which 8 received FDA approval. The cumulative success rate for molecules developed for type-2 diabetes is 10%. Small molecules were more successful than biologics. A strong disparity was observed in phase III, with studies that utilised treatment naïve patients having a 40% success rate, compared to an 83% success rate in patients who have had previous anti-hyperglycemic exposure.  Conclusions. 1 in 10 drugs that enter clinical testing in this disease will be approved. The DPP-4 inhibitor class of drugs had the highest success rate of all drug classes with a 63% cumulative success rate; while treatment naïve patients carried the greatest clinical trial risk.  Keywords: Clinical trials, Type-2 diabetes, Drug development, Clinical trial risk.


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J Pharm Pharm Sci, 17 (3): 393-400, 2014

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