The rare disease challenge: two decades since Europe passed legislation to develop and use orphan drugs, are we succeeding?

The final Special Report of 2017 comes from Scott Pescatore, the Europe region general manager for rare diseases at Novartis Oncology, and is an overview of how progress has been made, and how more can be achieved, in the orphan medicines space.

When you consider there are more than 7,000 known rare diseases that affect one million people across the world, it’s hard to believe that less than 20 years ago, only eight treatments were approved in Europe. Fortunately, things have improved over the past two decades. Following the introduction of European Orphan Drug legislation in 2000, we have seen more than 100 rare disease treatments reach patients.

So, 20 years on, should we be happy with the progress made to-date? And should we be confident about what the future holds?