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Approved Research

Supporting development of more successful medicines by leveraging the relationship between genotype and phenotype to explore efficacy, safety and indication expansion.

Principal Investigator: Dr Martin Armstrong
Approved Research ID: 44411
Approval date: August 7th 2020

Lay summary

The aim of this project is to develop drugs that are more likely to work well (efficient), less likely to cause side effects (safer) and to identify the people that will benefit most from those drugs (targeted). By combining the genetic, phenotypic and biomarker data from the UK Biobank, UCB will gain a deeper biological insight into the effects of genetic variants on diseases (PheWAS studies) and explore the relationships between genetic variants, clinical outcomes and biomarkers (Mendelian Randomisation). 

Exploring associations between genes and multiple phenotypes in a hypothesis free manner allows us to identify when genes are having multiple effects, identifying other diseases that might be targeted with drugs acting on that gene, what some of the side effects might be if affect the activity of a gene and what markers we might look at to help us measure these effects (called biomarkers). This can lead to better targets for the prevention of multiple conditions and complications. This approach identification of genes that are actually causing the conditions. The project will also allow the application, and development, of new analytical approaches. These will also support more work of this nature.

The outcomes of this work will support decision making and project prioritization within UCB, supporting reduced attrition rates and promoting efficient use of resources. This could then lead to more efficient clinical study designs. Ultimately the outcome of this will be drugs that are more likely to succeed, developed and delivered in a more efficient manner which will promote cost savings. This work will also add to the scientific understanding of the diseases and outcomes studied. This information will be returned to the UK Biobank with an ultimate intent to publish, promoting scientific advances.

UCB intends to use this approach across their project portfolio, which is dynamic and constantly evolving. In this respect UCB would like long term access to the data, as well as future data enhancements. In this first instance, we would consider access for a defined period of time, for example 3 years, with the ability to discuss prolongation after that time.

The development of more efficient, safer and targeted drugs would have a public health impact. Less obvious benefits are likely which support a sustainable healthcare system. Quicker, cheaper studies and less drug attrition all contribute to reduced drug development costs and timelines, which permeate through to savings for health care providers.