Excellence in Rare Diseases and Orphan Drugs

Summary of work

In 2018, Sanofi acquired Ablynx, a small biotechnological company focused on the development of nanobody technology, the first being Cablivi for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP). This rare, acute and life-threatening disease currently has a treatment landscape which has remained unchanged for many years. With no heritage in rare blood disorders, the Sanofi team, with limited resources, worked to build relationships with clinicians, payers and patient organisations, to develop advocacy for Cablivi and raise awareness of this catastrophic disease.

The team needed to gain insights, identify data gaps and put projects in place, working against the clock to build a robust economic model in what would be an extremely challenging health technology assessment (HTA). The rarity of aTTP meant little data was available, and acute nature of episodes meant proving cost-effectiveness would be our biggest hurdle. There was a clear clinical need and strong advocacy for the drug, so the team worked cross-functionally to find innovative ways to collect data and engage stakeholders to ensure that every patient experiencing an acute episode of aTTP would have access to Cablivi.

Judges' comments

Sanofi Genzyme’s submission was a great market access project. The judges really liked the earthquake imagery, which made it particularly memorable.  The commitment and drive from the cross functional team was impressive.