Excellence in Rare Diseases and Orphan Drugs

Summary of work

Rare diseases often don’t get the same recognition as other conditions. In Pompe disease, there are very few specialist treatment centres and very few families affected. So why would any pharmaceutical company reach out to them? It takes a brave and forward-thinking company to respond to unmet needs in this community. Sanofi Genzyme believed that even if one person benefits, it was prepared to invest that time.

Often, ‘patient involvement’ manifests as involving patients in commercial activities that the company wants to do. This project was completely the opposite. Aurora and Sanofi Genzyme worked with the audience in a true collaboration that identified the challenges and drove development of solutions.

This project may only reach a small number of people, but for those it reaches it means the world. This pack means that each child with IOPD can have the best experiences no matter how long or short their life is. It means they can make friends, learn about the world and, for a short while, put their own stamp on it.

This approach should inspire other companies, not just in rare diseases, to involve patients more deeply and support their broader unmet needs.

Judges' comments

The Infantile Onset Pompe Disease project is an impressive school pack supporting patients’ needs. It has clear multifunctional working with admirable execution. It is a struggle to educate schools on healthcare needs and they have achieved this.