The development of new drugs is big business, costing up to £850 million to get a single treatment to the point it can be sold and used in the clinic. A new study published in the International Journal of Epidemiology has found that genetic research in large-scale prospective biobank studies may significantly improve the drug development pipeline, thereby reducing costs.

New treatments are discovered by exploring biological pathways that cause disease, but can be modified by drugs, and the route to a successful drug is littered with those that have fallen by the wayside at various points during development. However, a study of one such unsuccessful drug has pointed to a way that could reduce costs. 

Researchers at the University of Oxford and the Chinese Academy of Medical Sciences joined forces with Glaxo SmithKline (GSK) to explore whether the disappointing results of previous research into cardiovascular disease could have been predicted by using a genetic variant that mimics the drug effect.

People with a non-functioning variant have lower levels of Lp-PLA2 so the idea is to see if these people have a lower risk of cardiovascular disease than those with functioning variants. In a study of over 90,000 participants in the prospective China Kadoorie Biobank (CKB) study, the association between this genetic variant and a range of cardiovascular and non-cardiovascular diseases was explored. This method is termed ‘Mendelian randomization’ because it relies on the way the play of chance determines the genetic variants inherited at conception, and allows nature to mimic randomised controlled trials. 

The researchers found that people with a non-functioning genetic variant were not at lower risk of developing cardiovascular (and other non-cardiovascular) diseases – upholding the trial findings. Compared with the many steps in the traditional pathway to drug development (at a cost of several billion dollars) the availability of large scale biobanks make the cost of carrying out Mendelian randomization analyses trivial. This methodology is likely to be much more widely used to examine the causal nature of biological pathways involved in diseases before mounting large-scale trials in the future. Medicine applicants should consider how this step will help with drug trialling in future, and Experimental Psychology applicants should explore the benefits of studying failed research to provide better, more scientifically rigorous methodologies for experiments.

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