Access to treatments

Duchenne UK hosts an industry workshop on Economic Modelling for HTA

January 2017

The day was led by Fleur Chandler, who sits on the Patient Advisory board for Duchenne UK. Fleur is a specialist in Value Evidence and Outcomes in the pharmaceutical industry, and also a parent of a son with Duchenne. She was ably assisted by Anthony Hatswell and Professor Ron Akehurst from BresMed, and we are very grateful to them for giving us their time and enormous expertise. 

With a wave of treatments in or entering the clinic, with some already gaining approval, the meeting looked at addressing the next challenge after approval: to get organisations like NICE to pay for these medicines. The aim of the day was to work with companies developing potential treatments for DMD to best deal with the NICE process; by asking how can we truly capture the devastating impact of Duchenne Muscular Dystrophy, that will facilitate the approval and reimbursement of drugs.

We held our first workshop in September to bring together the community and industry to better understand the HTA process .  Following that, Duchenne UK  commissioned Professor Braziers’s team at the School of Health and Related Research in Sheffield to undertake a rapid scoping review of the literature. They presented their findings yesterday. 

Eleven companies developing potential medicines for DMD attended the workshop. 

We are now working with them on a plan for next steps.  

Thank you to everyone who attended.

Published on 27 January 2017

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