Ramblings of a CEO
Why do we do what we do?
Well there are many reasons that we work in the rare disease space; money, travel, career, passion, belief that we can make a difference. I recently had some training on health economics and it was a reminder that despite the reasons we all do this there is a money vs benefit-to-life equation. Rare disease treatments are expensive but does that mean that they should be excluded just because of cost or on the premise of needing more long term data? How do you do that with such small numbers and with treatments that are groundbreaking new science?
No matter all the valid reasons for doing what we do, we need to work more collaboratively to counter the increasing barriers being put in the way of these new treatment options.
This is a light hearted reminder to everyone that these treatments are worth the uphill struggle. My two sons with MPS II having fun and able to do so because of the dedication and determination of all of the medical and pharma professionals together with the patient organisations. Treatments do make a difference and looking at this video tell me how do you put a cost vs quality-of-life argument against two boys who obviously just want to live!!