PFMD member Russell Wheeler, Leber’s Hereditary Optic Neuropathy Society, talks about his journey to becoming a patient advocate in medicines development:

Leber’s Hereditary Optic Neuropathy (LHON) is a rare genetic condition that we knew nothing about until our 23-year-old son suddenly went blind five years ago. Over the space of two months he went from having perfect sight to effectively having no central vision; he can’t recognise faces, he can’t read and he now uses a computer through auditory means only. The life he had been planning has been completely devastated.

With my son’s diagnosis came the knowledge that the rest of the family from the maternal line also carry the mutation, meaning my wife and my other two children could become blind at any time.

I think the patient voice is occasionally heard in medicines development, but not as consistently and not as forcefully as it ought to be. To give an example, a treatment that was approved last year was clinically trialled about five years ago. It is easy to look at these things with hindsight, but there were a number of flaws in the design of that trial and I believe that if patients had been involved in the design it could have been a much more successful trial.

We need more evidence to demonstrate the value of patient engagement. I believe quite passionately that if patients are embedded in the system that everyone will benefit. Pharma will benefit, clinicians will benefit and of course patients will also benefit.

My ultimate goal and vision is that patient engagement is embedded in every part of the medicines development cycle, from bench to bedside and beyond. Patient engagement needs to be so enmeshed in the system that by the time a medicine comes to the regulatory stage it would be practically impossible to extract the patient perspective even if there were a need or desire to do so.

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