Excellence in Professional Education Programmes

Summary of work

Niemann Pick type C (NP-C) is a rare disease in which the movement and storage of lipids in the body stop working. There is no cure; treatment aims at slowing disease progression. On average, patients wait 5-6 years for a diagnosis, as symptoms are difficult to identify. Yet rapid diagnosis and initiation of treatment are crucial to improving patients’ and families’ lives.

‘Think Again. Think NP-C’ is an international professional education campaign created by the patient association the International Niemann-Pick Disease Alliance (INPDA) and Actelion. The campaign aims to increase NP-C understanding and recognition among clinicians and increase NP-C diagnoses worldwide, by encouraging doctors to make the connection with NP-C when they see certain symptoms in their practice and refer suspected patients for follow-up/testing.

Expert guidance was sought from practising clinicians across Europe to ensure the campaign was meaningful for target clinical audiences. Various materials were created for local adaptation, highlighting symptoms clinicians should look out for.

17 countries have committed to rolling-out the campaign locally, including nearly 80% of INPDA member groups. Of the countries that have launched so far, 3 have reported a significant increase in diagnoses and 6 countries have reported an increase in referrals/screening.

Judges' comments

A simple yet impressive multi-channel programme with clear objectives, broad reach and outcomes that really resonated with people, supported by strong stakeholder feedback. What is equally impressive is that this was market tested and they seem to have taken on board valuable learnings to ensure the programme will evolve.