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Excellence in Rare Diseases or Orphan Drugs

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Winner

Transforming Care in Hereditary Angioedema

by Takeda UK
with support from Makara Health


Summary of work

Hereditary angioedema (HAE) is a rare genetic disorder characterised by unpredictable episodes of painful swelling – if swellings affect the throat, they can be fatal. HAE has an impact on all aspects of life and is often misdiagnosed. There are an estimated 1,300 patients with HAE in the UK.

Research suggests patients ‘put up with’ relatively frequent attacks, accepting the disease burden as unavoidable, with some HCPs viewing attacks as ‘part of life’ for patients.

Takeda UK wanted to transform the standard of care – raising expectations for diagnosis, treatment and management.

Takeda has a comprehensive HAE portfolio with established medications Firazyr and Cinryze▼ and a recently launched, fortnightly prophylactic, Takhzyro▼, with the potential to reduce the number of attacks to zero.

A multichannel campaign was developed to engage immunology specialists, framed by the HAE Exchange Virtual Education Programme. The focus was on articulating the value Takhzyro can deliver to patients and examples of success were shared to provide early evidence. Patients were also supported by a host of non-branded educational resources developed in collaboration with HAE UK.

Additionally, in response to COVID-19 lockdown, Takeda introduced what it believes is a first-of-its-kind ‘at-home initiation’ treatment initiative.

Judges' comments

Transforming Care in Hereditary Angioedema demonstrated clear evidence of a mindset shift in HCPs and potential changes in behaviour. It clearly sets out the potential for better clinical and health outcomes with a nice emphasis on execution and engagement.


Finalists

Transforming the Rare Disease Sales Call - by Sanofi Genzyme
with support from Redline Strategic

Introducing the First Treatment Option for Paediatric and Adult Patients with Primary HLH - by Sobi, Inc. (North America)
with support from W2O Group


Key dates

Entry deadline 26 August 2021
Extended entry deadline 2 September 2021*
*additional fee applies
Judging Day 1 October 2021
Company of the Year Interviews 22 October 2021
PMEA Event 24 November 2021