Estimating Probabilities of Success of Clinical Trials for Vaccines and Other Anti-Infective Therapeutics

Chi Heem Wong; Kien Wei Siah; Andrew W Lo

is the (which was not peer-reviewed) The copyright holder for this preprint From Figure 5a , we can see that the overall PoS across all industry-sponsored drug development programs treating infectious diseases is 16.3% (SE: 0.7%). The PoS12, PoS23, and PoS3A are 65.0% (SE: 0.8%), 64.3% (SE: 1.0%), and 51.1% (SE: 1.6%), respectively. Based on our data, the highest success rates for industry-sponsored non-vaccine development programs have been for smallpox (100.0%, SE: 0.0%), CMV infection (31.8%, SE: 7.0%), and onychomycosis (29.8%, SE: 6.7%). There are currently no non-vaccine therapies approved for rotavirus, SARS, rabies, Ebola, West Nile Virus (WNV), Marburg, yellow fever, chikungunya, MERS, monkeypox, or norovirus. With the exception of norovirus and MERS, these diseases without any vaccine are predominantly prevalent in nonindustrialized nations, and thus represent neglected diseases. It is also interesting that for the latter eight diseases, even the PoS12 is low. Since phase 1 trials in the development of anti-infective therapies focus primarily on safety, understanding the pharmacokinetics of the compound, and maximum tolerable dose levels, it can be inferred that the drugs tested are either of high toxicity or lack the necessary characteristics required for optimal absorption, distribution, metabolism, and excretion (ODME), or perhaps failed to advance due to financial constraints.