One of the many upsides of this job is that I am able to attend events and congresses that invariably move and motivate me. Why? Because there is one thing in common to healthcare, therapies, disease management, scientific progress, healthcare system opportunities and challenges, digitisation of healthcare and that is the patient. Whatever event I attend always has one or more stakeholders explaining their quest to meet patient needs and/or one or more patients or careers telling their story, their experience and giving their invaluable point of view on all of the above.

One such event was the recent European Conference on Rare Diseases I attended partly held over last weekend. 900 people committed to furthering results in the agenda of research, development, approval and access to orphan medicinal products and therapies gathered over 3 days to discuss the various facets of each element together. Patients, their representatives, their families and carers, national and supranational institutions, industry and academics filled a packed agenda of plenaries and parallel workshops on everything from Structuring the research and diagnostic landscape to Global Rare Equity.

Among the many moving life stories of patients and their parents, one that struck me in particular was the story of 24 year old patient Maria, a patient with a life threatening condition called Mukopolysaccharidosis type IVA who when diagnosed aged 3 and a half had not been expected to survive (watch her story from 1’ 24 at www.youtube.com/watch?v=2LJs0IUIy8A). Maria who lives in Austria explained how her life changed from when the therapy became available in terms of her mobility and ability to participate more actively in day to day activities. She also illustrated her journey as she went from being part of a Clinical trial in London having to travel there for a week a month and being unable to work, to having to continue the trial in Germany (a shorter train ride away) to celebrating when the product was finally approved, as she was finally able to take the treatment at home in Austria where a nurse would come once a week. Maria’s mother’s comment was that this was the best possible solution as she no longer needed to plan the gruesome trips and just needed to open the front door. Maria herself now holds a job and enjoys as normal a life as possible living with her condition. I was thinking of the difference in quality of life for Maria and her family as well as of the incredible impact that an approved product can have on a healthcare system. From monthly trips to London by plane, to the same to Germany for Maria and her Mother, including stays in hospital and other associated costs, to the cost of a healthcare worker visiting Maria once a week at her home in Austria.  And this doesn’t even take into consideration the difference between the care, and related personal and financial implications, of worse that would have to be administered to Maria had there not been a therapy in development. 

So our quest as EFPIA to take the conversation about therapies and healthcare to the next level continues. We can see the power of innovation, we witness our members develop these innovative solutions to tackle unmet medical needs and improve on existing treatment options and we acknowledge the challenge of introducing these products into financially strained healthcare systems. We are convinced that with constructive dialogue across the healthcare system (current silos) supported by the proper tools to measure gains brought about by new treatments as well as any existing waste, we can optimise healthcare systems and find ways of investing in new innovative treatments to allow the lives of all patients to be transformed for the better.