Excellence in Innovation

Summary of work

Mucopolysaccharidosis Type I (MPS I) has an overall prevalence of 1:100,000¹ meaning that any one paediatrician is unlikely to see a patient during his/her career. It is a progressive, debilitating, life- threatening disease which, if diagnosed early with a simple urine test, can be effectively managed at specialist centres to give patients better outcomes. We needed a new approach, without an in-house sales team, to raise awareness of MPS I. Analysis of RWD, conducted in line with Information Governance (IG) rules, outlined MPS I prevalence at a suitable regional level and a separate segmentation exercise provided priority lists of paediatric specialities who may potentially see MPS I children in their clinics. To execute the final part of the campaign, a third-party remote detailing agency contacted physicians, from the priority lists provided, to educate them on the condition, give information on how to test and diagnose MPS I and refer suspected cases to specialist treatment centres for follow-up and management.

1. Laraway S, Breen C, Mercer J et al Molecular Genetics and Metabolism 2013;109:315-316

Judges' comments

Making the Connection uses an innovative approach to understanding real world data. Sanofi Genzyme were able to demonstrate evidence of stakeholder specific information and the evolution of the target audience and content.