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Approved research

Development and application of phenome-wide Mendelian randomization methods for drug discovery and development

Principal Investigator: Dr Alexander Blocker
Approved Research ID: 44424
Approval date: February 4th 2019

Lay summary

Genetic and other information collected in the UK Biobank program will lead to a better understanding of the relationship between genetics and outcomes such as disease and its response to treatment. Our goal is to apply that information to improve health outcomes, including for residents of the United Kingdom. To take advantage of increasingly diverse sources of genetic associations, the field requires methods that can provide valid inferences even when some standard assumptions are violated. Refinement of these methods requires deep and broad phenotyping data at an individual level. The UK Biobank is an ideal resource for our research, and we plan to use the data to improve causal inference with genetic data, leading to more reliable associations and more efficient drug development, particularly in the understanding of on-target effects. The potential impact on public health follows from the incrementally better understanding of human health and disease that might result from this effort, together with its application to reduce harm and improve outcomes when developing new treatments. A secondary benefit is that treatments developed in this manner might be more targeted and therefore more cost-effective. We aim to improve and develop robust methods for understanding the relationship between genetic targets and health-related outcomes. The methods and the resulting catalog of causal effect estimates will be published and made broadly available to the community over the next 3 years.