Robert Meadowcroft Chief Executive
Findacure Conference Our Journey: The Translarna Milestone 12 June 2015
Case study of Translarna • Translarna targets nonsense mutations affecting between 11-13% of boys with Duchenne muscular dystrophy ie around 80 eligible boys in England • For boys like Michael
Fast access to new drugs • Our report through APPG • Case studies/spokespersons/ media • PTC Therapeutics/clinicians/ NHS commissioners • Work with NICE and NHS England inc CRG
and Translarna • EMA/EC conditional approval – July 2014 • Not on NICE radar for HST evaluation • NHS England to consider by end 2014 • CRG-PoC Board-CPAG-RDAG-SCOG-Board • Judicial Review threat halts process Nov 2014
NHS retreat in face of JR • Threat of judicial review of scoring method • Weeks of confusion and uncertainty • IFR route not available • 90 days consultation period starts 27 Jan 15 • CPAG to meet 24/25 June, Board by 30 June • NICE HST appraisal scoping meeting Feb 15
MDUK actions • 30 PQs/debates & lobby event • PMQ/present petition to No 10 • Health Secretary, with a mum • George Freeman and PTC • Directors of NHS England • Submit letters clinicians/family
Meeting the PM outside No 10 Presentation of the petition, met the PM
If a NO from NHS England? • NICE HST evaluation: draft in 2015; full in 2016 • Push use of IFR route via consultants • Appeal NHS-E decision & return to campaign • Usual targets, emphasis on media/families • Celebrity support to raise profile of the issue
T ime “just to be himself” “By far the greatest emotional challenge is trying to offer support to Kirath as he travels on his journey of self realisation that he is never going to be 'just one of the boys'. His inability to keep up with his friends in the playground, or participate in soft play at parties, or even just climb the stairs at home to go to bed - these are all daily reminders of his growing physical weakness as his muscles continue to degenerate. Translarna would buy precious time for Kirath just to be himself .” Jaspal Mann
Robert Meadowcroft Chief Executive
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