To deliver treatments for rare diseases more quickly, companies share how they are adapting their launch strategies and engaging more deeply with physicians and experts.

“The only thing that was impossible was to do nothing.” These are the words of Terry Pirovolakis, CEO of Elpida Therapeutics, and father to Michael who was diagnosed as an infant in 2019 with an ultra-rare neurological condition, SPG50. Despite being told there was no cure, Terry moved mountains over four years to find a breakthrough gene therapy that his son and other affected children all over the world could benefit from.

Michael, Terry, and their family are not alone in facing a long, daunting journey to access life-enhancing therapy for a disease with no cure. In the European Union, less than five percent of rare diseases have at least one approved treatment. It can take up to five years for adults to receive a diagnosis, and half of patients will receive a misdiagnosis first.

By 2030, the goal is to support the development of 1,000 new therapies in Europe. However, rare diseases challenge traditional ways of doing business. Once a medicine is approved, organisations must launch and scale quickly, often across multiple markets. Field medical teams must keep track of complex science at a time when the volume of medical knowledge is doubling every 73 days. They need to identify, engage, and provide education to relevant healthcare professionals (HCPs) and experts even though they’re harder to reach via traditional methods (Veeva Pulse data shows that in-person meetings with physicians have declined in Europe in the last 12 months).