Professional success and personal highlight: what it means to bring a medicine to the people that need it

According to industry research, around 90% of medicines tested in clinical trials do not make it to market. While we should aim to increase drug development efficiency, this high failure rate comes from trying hard to innovate and create transformative medicines. It also means that essential research and development standards are being applied to ensure patient safety is prioritised and the potential efficacy and risk profile of investigational medicines is being rigorously and thoroughly tested. However, for the many scientists and researchers focusing on developing such medicines - through test after test, often for eight to ten years for a single treatment – the frequent risk of failure is part of their daily work and can often feel relentless.

The odds of success

For one medicine to get through discovery, pre-clinical testing and then be successful in phase I, phase II and phase III clinical trials requires an average of 7,000,874 hours of work, 6,587 experiments and 423 researchers. This is why developing a drug that makes it to approval is a precious achievement, because the odds are always against us. These odds can be stacked even higher when we are trying to develop treatments that have a new mechanism of action, or which target new disease pathways, because this often requires new research at the very edge of scientific discovery. However, these innovations are essential to provide additional value for people living with disease or illness, and for society in removing the burden of disease.

One area that has seen a lot of innovation and research recently is ophthalmology. Vascular endothelial growth factor (VEGF) is a key protein involved in the development and progression of retinal conditions. While VEGF inhibitors revolutionised the management of certain retinal conditions, they have reached what we call an ‘efficacy ceiling’ – meaning that, while they improve vision, there is a limit to the vision gains that people can achieve. Furthermore, these treatments require frequent injections into the eye, which can result in a high treatment burden for people with these conditions. For 15 years, our talented and dedicated team has been working hard, through trial and error, to provide alternative treatment options with new mechanisms of action for people with eye diseases. Finally, our perseverance has paid off and we are starting to see the impact of our work in the real world.

The people behind a new medicine

The teams that are working hard to develop a single investigational medicine put their heart and soul into their work, keeping faith that it might one day have the potential to benefit the people who need it. Each time they encounter a stumbling block, they show impressive problem solving, strategic re-evaluation and resilience. At Roche, our teams make use of cutting-edge science and smart innovations, to learn from and overcome the challenges they face. I find that, each time a medicine reaches the next stage of testing, there is a collective sense of nervousness and anticipation, as we wait to find out if the molecule or medicine really does have what it takes to go all the way. The process takes a lot of patience, a lot of tolerance to failure and a lot of tolerance to risk. I am always in awe of the unwavering commitment I see in the people driving investigational medicines through the development lifecycle.

And it’s not just the researchers, but also the vast teams of people around the world who play a crucial role in the journey of a medicine from initial discovery through to becoming available to the people who need it. This includes safety and regulatory experts, manufacturing and logistical teams, and medical and commercial colleagues. It means that, by the time a medicine reaches approval, so many of our employees will have helped to get it there. Outside of the pharmaceutical industry there are also many different groups of people involved. In ophthalmology in the last 20 years for example, our teams have liaised and worked with those involved in over 100 investor-led studies in 33 different ophthalmology disease areas, and more than 30 clinical trials enrolling 22,000 people globally who live with retinal conditions.

One of the things I love about the drug discovery and development process is the number of individuals who come together to make science work for the benefit of people in need, in order to change lives on a global scale. When a new medicine has overcome the odds, the hurdles and the barriers, to be approved for use in the people it was designed for, the pride, relief and joy felt by all is significant. It means those years of hard work have resulted in success, and it helps remotivate everyone to remain focused on finding the next medicine that will make a difference to the lives of people living with vision loss.

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