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Rare Diseases and Orphan Drugs

Back to Results


Winner

IMNOVID® for multiple myeloma

Celgene


Summary of work

The development and implementation of an extensive launch programme focusing on strong stakeholder engagement resulted in patients with relapsed and refractory multiple myeloma who have failed all existing options gaining access to orphan drug Imnovid (pomalidomide) via the Cancer Drugs Fund (CDF) at the time of launch and clinicians understanding the clinical value of pomalidomide, as well as the benefit of actively treating patients. 

Recognising the challenges associated with securing funding, particularly for treatments for rare diseases, Celgene worked closely with external stakeholders to maximise the impact of the submission it put to the CDF and to help prepare for the launch of pomalidomide. Ultimately, this enabled patients to access the first and only licensed medicine approved for use in combination with dexamethasone at this stage of the disease 6-12 months earlier than would be possible through NICE.

Judges comments

The challenge was navigating the Cancer Drugs Fund and orchestrating a plan with a small, focused team – and they rose to it. This has everything – the team provided internal training to ensure the portfolio was being used correctly and through cross-functional teamwork they engaged effectively with customers. Their work led to 212 patients being accepted by the Cancer Drugs Fund and an increase in life expectancy from three to 14 months – an inarguably strong outcome.


Highly commended

Cinryze IFR toolkit - Viropharma with support from Havas Life Medicom