Since the start of 2015, Action Duchenne campaigned tirelessly for Translarna (ataluren) to be made available, exerting maximum external pressure upon NHS England, the National Institute of Health & Care Excellence, and the Scottish Medicines Consortium whilst working within their evaluation processes.
Thanks to the campaigning and work by Action Duchenne and the community, this treatment is now available on the NHS. Read what Action Duchenne did to achieve this and the timeline from the campaign.
February 2016
Translarna is the first ever treatment capable of addressing the underlying causes of Duchenne muscular dystrophy for people with a nonsense mutation. Despite receiving funding approval in nations all over Europe, we are still awaiting a final and determinative funding decision on the use of Translarna in the UK. In the face of the inadequacies and inertia experienced in the evaluation of Translarna, we have also sought to ensure that UK commissioning processes are efficient, equitable and transparent so that future Duchenne treatments can be quickly and fairly appraised.
Campaign Timeline:
2015
January 1: Translarna Petition – Action Duchenne founder launches petition to fund to first treatment for Duchenne Muscular Dystrophy
January 14: Westminster Lobby and Petition Submission – The UK Duchenne community stands united in demanding the immediate availability and full funding of Translarna by NHS England
February 11 : Vigil for Translarna – The Duchenne community takes their demands to NHS England
March 1: Letters to Key Figures – Action Duchenne keep up the pressure on parliamentarians and NHS England alike
March 11: #FundOurDrugsNow – Action Duchenne and the MPS Society collaborate with Greg Mulholland MP to submit a letter to no.10 and organise rally
March 24: No Guarantees: NHS England confirm there is still no guaranteed date for a funding decision
March 26-28: Wilfully Misled: George Freeman MP invites charities to prove NHS England are wilfully misleading parliamentarians and the public alike. Action Duchenne oblige
April 1: #fundingNOTfoolishness: Action Duchenne co-organise rally to highlight the human cost of NHS England’s dysfunctional and intransigent conduct
April 8: Minister replies: Minister replicates much of the deliberate misguidance we are used to from NHS England
April 14: 90 day consultation: Action Duchenne publish feedback on NHS England consultation establishing the parameters within which Translarna will be evaluated for funding approval
April 23: 30 day consultation: Action Duchenne publish feedback on NHS England consultation outlining the therapeutic benefit of Translarna and the clinical findings that have engendered a clinical commissioning policy
April 30: More misguidance. More delay: CPAG meeting pushed back from May to June
May 5: Sir Bruce Keogh makes u-turn on IFR route: Door potentially opened for interim funding!
May 14: Action Duchenne submit formal complaint to NHS England: Failing to provide clear, accurate and thorough information
June 9: Clinician makes Individual Patient Funding Request for Translarna: Reiteration of advice to all families to engage in dialogue with their clinician
June 11: APG on Translarna follows submission of letters to No. 10: Eleventh hour appeal to David Cameron for his support ahead of NHS England’s funding decision
June 18: Northern Ireland and Scotland meetings on Translarna: The different nationwide contexts discussed
July 1: A message from our CEO on the pending Translarna decision: A thank you to all of the Duchenne and other rare disease charities for their unstinting efforts over the previous 12-months
July 2: Duchenne community failed by NHS England: Funding decision is deferred to NICE
July 7: The fight goes on: Action Duchenne in parliamentary session to discuss existing access barriers to medicines
July 9: Action Duchenne meet senior NHS figures: Scrutiny in parliament over ongoing policy failures and the rationale behind Translarna decision
July 15: CEO Update on Translarna: Action Duchenne in parliament 3 times in 10 days
July 20: Freedom of Information Requests: Action Duchenne contact HM Treasury and the Department of Health for crucial information
July 28: Injustice in Writing: NHS England offer official explanation for denying Translarna funding
August 4: Future decisions to rest on flawed process?:NHS Response to 90 day public consultation is woefully inadequate
August 11: PM Replies to Parents and Patients on Translarna: Cameron refuses to intervene and direct NHS England to approve funding
September 7: A Gross Misuse of funds?: We continue investigations into use of PPRS payments by NHS England
September 8: Clarity required from PM over Translarna: Mr Cameron’s advice seems to contradict the expectations of the Duchenne community
September 18: NICE HST Evaluation Committee Meeting on Translarna: PTC, leading clinicians and patient experts provide evidence to NICE in crucial meeting
October 7: Lifeline thrown to families north of border: Individual Patient Treatment Request approved for Scottish Family
October 8: More Delays, More Apologies: NHS England Delay Response to FOI Request
October 8: Assurances Offered: PM moves to quell fears over recent statement
October 16: NICE refuses to recommend funding for Translarna: Further delay as more information is requested to justify cost
October 16: PTC meet NICEs request for additional info: Phase III confirmatory data released for Translarna
October 22: Action Duchenne maintain leading role in fight for Translarna: Community stands shoulder to shoulder to get the treatment approved
November 3: #ADCONF15 to send unified message on Translarna: Attendees and delegates sign letters to NHS England and NICE
November 5: Action Duchenne feedback to NICE consultation Translarna: Draft guidance thoroughly scrutinised and challenged
November 9: #ADCONF sends unequivocal message to NICE & NHS England: Fund Translarna and give families the extra time they crave and deserve
November 12: Action Duchenne Trustee Kathy Wedell sets out case: 13 Reasons to Fund Translarna
November 18: NICE Second Evaluation Committee Meeting on Translarna: Committee seek to take account of further information
November 20: NHS England pledge to make improvements: Commissioning of Specialised Services to be reorganised
November 22: PTC Therapeutics thanks community for their support: Stuart Peltz write to Action Duchenne
November 27: Action Duchenne launch Translarna strategy in Scotland: Letters written to senior figures within Scottish Health Service
November 30: SMC move to allay fears over Translarna: Reassurances offered over potential delays and centrality of patient voice
December 9: Cause for cautious optimism over NICEs decision on Translarna: Minister George Freeman suggests a Managed Access Agreement may be possible
December 16: Action Duchenne offer guidance in Scotland: Patient Group Submission Form sent to Scottish Medicines Consortium
December 17: Translarna decision delayed until new year: Final guidance to be offered by NICE on January 4
December 24: New Year.. Familiar Feeling: NICE suspend issuing guidance until new information is considered
2016
January 9: Translarna set for PACE process: Parents, Patient Groups and Clinicians to make the case in Scotland.
January 11: Quick and positive outcome still as vital as ever: Final appeal to MPs to levy influence on Translarna.
January 12: Third NICE Meeting announced: Final Evaluation Committee Meeting set for date in February.
January 15: Action Duchenne working with Scottish Family on Translarna: Joint submission being written to inform the decision making of SMC.
January 29: Campaign in Scotland gathers momentum: Community and Cross Party Group unite to maximise pressure for Translarna’s approval.
February 10: Strong Case made at PACE meeting: Parent’s and clinicians make compelling argument for Translarna.
February 16: A Message from our CEO on Translarna.
February 17: Third NICE Evaluation Committee Meeting: Proposed Managed Access Agreement Positively Discussed.
Action Duchenne are in ongoing communications with PTC Therapeutics, NICE & NHS England concerning the time frame and outcome of any decision on Translarna. We are anticipating further direction from NICE shortly and will keep everyone posted with any new information we are able to share.
Until the long term funding of this ground breaking treatment is assured we will continue to work with all relevant partners to ensure a positive and timely outcome.
Be it through working with UK health bodies, utilising parliamentary lobbying fields, or taking an active role in community led activism, Action Duchenne are committed to ensuring every eligible patient can benefit from Translarna as quickly as possible. We’ve been fighting this battle for over a year. Duchenne is a severe degenerative condition. Put simply, we do not have time to wait.
We would like to extend our gratitude to the entire Duchenne community for the support you have provided in fighting for this groundbreaking drug. Be it via lobbies, letters or consultation responses, you have been there with us every step of the way. We know you’ll continue to struggle alongside us to secure the future of Duchenne treatment.
