Excellence in Rare Diseases and Orphan Drugs

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

Sanofi Genzyme showed real precision marketing pinpoint of understanding. They took a creative analytical approach and had real, demonstrable outcomes. We liked seeing the re-work of the data and the smart working.