I graduated as a medical doctor and specialised in clinical pharmacology/ clinical research. Patient care and science have always been the guiding stars for my career choices and have led me to work  in small, mediumand big pharma with a focus on patient centricity and innovation. I have held diverse roles in R&D, medical affairs, business and general management.

Before joining Alexion I was CEO of two early stage biotechs in the UK and venture partners in a global investment firm. Alexion fits my ideal profile as a science-driven and patient centric organisation. It is in the company’s DNA. Learning from patients and co-creating solutions with them, and for them, is what we do every day.

Looking back, each role took me on a journey, starting from great science to the ultimate goal: the challenge of providing effective and safe therapy for patients. This value chain is made of interconnected, inter-dependant factors, all essential to the innovation ecosystem in health care.

The achievements from Europe’s SMEs within advancements for rare disease: A vital part of the ecosystem

The innovation cycle in biopharmaceuticals requires the coexistence and collaboration of multiple organizations at any given time. No one organization can deliver this successfully on its own, no matter how big and sophisticated it is.

The R&D process requires more focus and specialization to explore new avenues and technologies to address the unmet medical needs not already met by existing approaches.  In rare diseases this is taken to the extreme – we research diseases which affect unbelievably small patient populations – often fewer than 1,000 across Europe.

Early stage SMEs are exactly the organizations that are much more likely to succeed in this. They are often spin-offs created by scientists and researchers focused on a very specific problem and a disruptive scientific hypothesis. They channel their entire energy on trying to solve this problem through research and early development.  Some fail, but conclusive research always helps advance the science.

Some succeed and often, they need a bigger partner to complete the product development, regulatory approval and commercialization at scale.  To me, SMEs are an indispensable element of the ecosystem for rare disease innovation. According to the recent research, there are several hundred SMEs focused on rare diseases in Europe. These exist only because of the legislation we have in Europe that created the framework and the incentives for this to happen.

OMP legislation under the spotlight: How does it enable innovation from Europe’s research strengths in rare diseases?

The EU OMP Regulation is a key element of the rare disease innovation ecosystem in Europe. It has changed the Orphan Drug landscape in two main ways:  First, it sets up the regulatory pathway for orphan drugs, including the OD definition, criteria, requirements and a dedicated process and governance and second, it establishes the incentive system for OD manufacturers to mobilize R&D investments.

It is a great opportunity as the legislation is being revised to include learnings from the past years of experience and refine/improve this framework to reinforce its two pillars: regulatory OD designation and incentives for R&D investments to accelerate the innovation for rare diseases.

The voice of EuropaBio: Ensuring that biotechnology plays its full role in advanced therapies and rare diseases

EuropaBio has an important role to play, in representing the voice of the biotech SME community and how they contribute to innovative science and biotechnology development in Europe.

Those small yet innovative organizations could never afford resources and time to shape the bigger policy landscape at the European level, meanwhile their perspective is very important. As we discussed earlier, biotech SMEs are the innovation trailblazers and EuropaBio is perfectly positioned to make their voice heard and respected in Europe.