1. What inspired your dedication to the rare disease landscape?
My dedication to the rare disease landscape stems from a deep conviction that every patient, regardless of the rarity of their condition, deserves access to appropriate care, treatment, and support. After many years working in non-orphan pharma sector, I was offered the chance to work for a first drug dedicated to a very limited population and have understood that this would need a different approach. I was motivated to see how I could help.
2. In your view, what are the critical elements needed to drive meaningful change in this domain?
Driving meaningful change in the rare disease landscape requires many things but if I was to choose only 2 I would say collaboration and speed. First, collaboration between stakeholders—patients, researchers, policymakers, Health authorities and industry—is essential. Each of these stakeholders has its own agenda, which is normal, but we all have the same enemy: we want to defeat the disease. And you know, the enemy of my enemy is my friend….. Second, the Sense of urgency: most of the time, the therapeutic options at hand are not yet optimal. This means that we need to go full speed as the patients need it
3. What motivated your involvement as a founding member of BDI?
AS you may know, BlueDil was first founded in France, based on the working principles described above and operating as a consultancy firm dedicated to support stakeholders involved in the battle against rare diseases. Adding “international” to BlueDil, was driven by the willingness to expand our scope of work in Europe. We have decided to ally our local expertise to bring international impact. This was also, and that is very important for me, the opportunity to work with partners, friends and colleagues from all over Europe. For instance, I have known Kamlesh, Leonardo and Elena for almost 20 years. We have the experience to work together, and I know their high level of expertise. It is good to be part of such a team.
4. How does BDI position itself strategically to advance rare disease initiatives across Europe?
BDI is an expert consultancy firm in the field of orphan and ultra orphan diseases. In a nutshell, our mission is to help our customers to bring new therapeutic options to the treating physician and ultimately to the patients. We consider ourselves as one of the very few in this area that provide a pan European coordination conjugated with a high level of national expertise in the execution of our missions.