Rare Diseases and Orphan Drugs

Summary of work

MDS is a group of haematological malignancies that occur when blood cells remain in an immature or ‘blast’ stage within the bone marrow and never develop into mature cells capable of performing their necessary functions.

The average age of diagnosis for an MDS patient is 73. Patients with higher-risk MDS have a median survival of approximately six to 12 months. They must often rely on blood transfusions to manage symptoms of anaemia and extreme fatigue, which may result in life-threatening toxicity.

In this elderly population with limited alternative treatment options VIDAZA® trebles the two-year overall survival of patients over 75 years old. However market research suggested that this benefit was not being realised in practice, as the majority of VIDAZA® use was in the 65-75 year old population.

Further market research indicated that clinicians were concerned about treating elderly patients with co-morbidities and the challenges of keeping older patients on therapy.

Celgene developed a ‘75 not out!’ campaign focused on patients 75 and over. As a result of the change in focus, market share increased substantially in this patient group and most importantly hundreds of elderly MDS patients trebled their chances of surviving past the two-year mark.

Judges' comments

75 Not Out had a powerful communications message that tackled ageism head on, engaging stakeholders well and demonstrating a strong focus on a particular patient segment. It’s an important initiative to help change perceptions about treating the elderly.