Action Duchenne
Epicentre
41 West Street
London
E11 4LJ
Mr Richard Jeavons
Specialised Commissioning
4N26 Quarry House
Quarry Hill
Leeds
LS2 7UE 6 November 2015
Dear Mr Jeavons.
We the undersigned are writing to you concerning the National Institute for Health & Care Excellence’s (NICE) ongoing Highly Specialised Technology evaluation of Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene. We would be grateful if assurances could be provided on the response of NHS England, in the context of national commissioning, to NICE guidance on the use of Ataluren.
Ataluren is currently scheduled to be subject to a Second Evaluation Committee meeting on the 17 November 2015. This meeting will consider further evidence on the clinically meaningful benefit of Ataluren, in addition to responses to NICE’s evaluation consultation document on their draft guidance on this technology, before rendering a final evaluation determination (FED). We have today written to Sir Andrew Dillon requesting that any positive recommendation, reached within the FED, be immediately used as the basis for NICE guidance on using Ataluren in the context of national commissioning by NHS England. Similarly, we implore you to undertake all necessary measures to ensure funds can be immediately released by NHS England if a positive recommendation on the use of Ataluren is reached. This will assist in accelerating the availability of Ataluren to those patients eligible to benefit.
The importance of accelerating clinical access to this treatment cannot be understated. The Duchenne community has already been subjected to significant delay in awaiting a final and determinative decision on Ataluren, allowing severe and irredeemable muscle damage to our boys to continue unabated. In light of the irreversible and degenerative nature of Duchenne, we therefore demand that you make every effort to expedite NHS England’s response to NICE guidance.
Yours sincerely,
The Delegates and Attendees of the Action Duchenne International Conference 2015
Marie Devlin
John Devlin
Manoj Thakrar
Sejal Thakrar
Sharan Price
David Price
Anayansi Chadwick
Stephen Chadwick
Joanne Chadwick
Claire Francis
Elaine Froggatt
Helen Goodenough
Jo McSherrie
Nick Crossley
Emily Crossley
Sue Barnley
Tom Barnley
Lesley Wegg
Adele Cheetham
Shaun Rowlands
Claire O'Hanlon
Aidan O'Hanlon
Phillip Grannum
Nicola Grannum
Parampreet Singh
Simranjit Kaur
Linda Hawthorn
Mary Down
Raj Mann
Jaspal Mann
Richard Amphlett
Andy Hallam
Katherine Wedell
Stuart White
Angela Culhane
Ian Hornett
Penny Rowntree
Kevin Rowntree
Janet Bloor
James Ker-Lindsay
Biljana Radonjic Ker-Lindsay
Joanne Clements-Lewis
Mark Lewis
Susan Doyle
Clare Louise Gibbons
Colin Gibbons
Lucy Arnott
Trevor Cook
Helen Kemp
Rosalind Stopps
Gareth Hegarty
Divyesh Popat
Seema Shah
Andreas Kalogeropoulos
Xenia Kalogeropoulou
Dawn Howell
Kevin Howell
Mark Silverman
John Miller
Brenda Silverman
Warren Silverman
Farzana Khan
Mr Khan
Vivien Field
Anthony Doran
Moira Doran
Trevor Stringer
Robert Laid
Nisha Laid
Godfrey Adams
Jackie Purdy
Dirk Fair
Yvonne Grant
Michelle Young
Alasdair Robertson
Robyn Pete
Tina Johnson
Anoushka Kubaj
Gary Ford
Bernie Mooney
Padraig O Longaigh
Roma Mulvey
Tony Levene
Rene Brectan
Jana Haberlova
Angela Russell
Paul Roberts
Dimitrios Athanasiou
Omaira Gill
Graham Wynne
Tracy Levene
Nicky Willis
Maria Chatzopoulou
Malkit Ram
Bimlajit Kaur
Tyran Hawthorn
Óskar Reykdalsson
Benjamin James
Ryan O'Leary
Mithun Soul
Farhan Mian
Sam Bailey
William Lister
Wesley Gray
Iain Clarke
Helen Keenan
Aaron Revel
Paul Lenihan
Diana Ribeiro
Kelly Ferguson
Owen Callaghan
Paul Bown
Rujeena Khanum
Angela Stringer
Ravi Mehta
Jake Mousley
Kwame Gyebi
Polly Jones
Pauline Sharratt
Jashma Kaira
Beloslava Kormeva
Naomi Rune
Greg Holland
Miguel Cazorla
Masoud Borafia
Paul Fuller
Jessica Gillard
Lana Hylands
Helen Dorman
Alice Wicks
Laura Hunt
David Hickman
Maxine Murray
Yongxue Mei
Sam Allard
Pegah Bakhshi
Stephanie Akhurst
Dean Jose
Maxine Jose
Rebecca Moore
Petra Palmgren Lindwall
Magnus Lindwall
Levi De Bilde
Wayne Olsen
Georgina Olsen
Connor Medford Hawkins
Shelley Simmonds
Geoff Simmonds
Sharon Kitcher
Terry Kitcher
Mandy Dailey
Jak Knowles
Samantha Power
Robert McDonald
Kim Down
Kate Barklie
Kathy Tampin
Wendy Bridge
Mandy Knott
Claire Griggs
Zoey Towey
John Porter
Susan J Ward
Guðjón Reykdal Óskarsson
Bryndís Guðjónsdóttir
Priyanka Thapa
Linda Morrissey
Doron Rosenfeld
Miguel Moutinho
Adalberto Moutinho
Kostantinos Chalkos
Sophie Williams
Els De Geyter
Marijke Meuleman
Alex Smith
Maria Fries
Jean-François Briand
Ricky Andreis
Marisol Montolio
Eleanor Howell
Roger Douglas
Ronald Howell
Sylvia Howell
Glenda Edwards
Gareth Edwards
Valeria Lovato
Magdalena Novakova
Alexandra Johnson
Andrew Johnson
Nikolaos Evangelou
Chrysoula Tzavella
Annie Kennedy
Ketna Popat
Clair Kelly
Sheli Rodney
Nicoletta Madia
Mariateresa Moscato
Fernanda De Angelis
Klair Bayley
Paul Ackroyd
Dalbir Singh
Panayiotis Voskos
Charalambos Papadopoulos
Sophie Schipper
Heather Smith
Mujtaba Chohan
Penny Bailey
Martin Lister
Charlotte Lister
Grigoris Skourtopoulos
Aimilia Palavou
Eleana Kokkini
Christina Ioanna Pyrgianaki
Georgia Dragoumanaki
Charalampos Gourdoumisas
John Gorman
Isabela Tudorache
Marina Katsalouli
Mike Chapman
Arati Kreibich
Ozge Ozkaya
Marita Pohlschmidt
Anne Jeffers
Paula Naughton
Harpreet Kaur
Andrea Lukács
Wayne Ebanks
Vicky Ebanks
David Kemp
Marie Keymist
Sue Blackman
Steve Blackman
Sharon Scutt
Chris Scutt
Tanya Carroll
Neil Carroll
Sally Bassett
Adele Chapman
Penny Southall
Daniel Southall
Jack Ebanks
Aaron Ebanks
Joseph Zerafa
Sean Blackman
Tommy Scutt
Lloyd Carroll
Adam Bassett
Saul Catlin
Venetia James
Harry Luxton
Wayne Gray
SEARCHING FOR A CURE – IMPROVING LIVES
Action Duchenne Ltd
Tel: 0208 5569955 Fax: 0208 5569272
www.actionduchenne.org
Patron Helena Bonham Carter Registered Charity Nos: 1101971, SCO43852