Robert Meadowcroft Chief Executive Findacure Conference Our - - PowerPoint PPT Presentation

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May 01, 2023 259 likes •390 views

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

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Robert Meadowcroft Chief Executive

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Findacure Conference

Our Journey: The Translarna Milestone

12 June 2015

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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

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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

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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

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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

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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

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Meeting the PM outside No 10 Presentation of the petition, met the PM

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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

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Time “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

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