Alex’s Wish attends Health & Technology Workshop

September 16, 2016 12:21 pm

We were delighted to attend a workshop organised by Duchenne UK this week in London.

The workshop was designed to understand the Health Technology Assessment (HTA) process in England, and to give an overview of health economics and the process of new drug approval and how the Duchenne community can come together to bring new drugs to market as quickly as possible.

The attendees of the workshop included NICE (The National Institute for Health and Care Excellence) who make decisions based on evidence based data on which drugs to bring to the market, TREAT-NMD, various charities working on bringing new treatments for children/young adults with Duchenne Muscular Dystrophy, drug companies and parents to share patients perspectives and what is important at every stage of the disease from initial diagnosis, right the way to the final stages.

It was incredibly informative and we hope that this will help the Duchenne community learn from what we know to date with bringing the first ever drug to market Translarna and ensure when new drugs need to be assessed, the drug companies and patients and their families present evidence in way that support the approval process. Time is ticking for families who are living with Duchenne and by working together pro-actively will ensure drugs are brought to market a quickly as possible.

We would like to thank Duchenne UK for inviting us along to this workshop and look forward to future meetings.

About NICE

The National Institute for Health and Care Excellence (NICE) provides national guidance and advice to improve health and social care. For more details visit


TREAT-NMD is a network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Since its launch in January 2007 the network’s focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide.  For more information visit

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