This first use of a genetic therapy for Duchenne has been approved by the EMA and other European countries have already agreed to fund the use of Translarna. In Scotland one young person, Cormac Fegan, has been granted funding for Translarna.
Patients, patient organisations and UK clinicians have all recently presented a strong case to NICE for NHS England to fund this new medicine. Families have lobbied the Government and MP's have raised the issue with the Prime Minister this week at PMQ's.
The decision making process for Translarna has been blighted for months by cancelled meetings and a lack of urgency despite the knowledge that delay in delivering medicines like Translarna affects the quality of life and possibly the life expectancy of young people living with DMD.
The current decision by NICE, to be further reviewed in November 2015, seems to be based upon a perceived lack of positive data that would make this drug cost effective. In other words NICE seem to be arguing that it is too expensive and does not cure or significantly reverse the severe muscle wasting associated with the condition over and above current treatments like steroids.
So what does the published data from PTC show? Is the decision made by NICE politically driven by austerity and budget constraints? How are the NHS in England going to manage the delivery of new experimental drugs for conditions like DMD?
This week PTC have published new data relating to a Phase 3 clinical trial that follows data already presented to NICE and the EMA from a Phase 2b trial.
The overall ITT is not statistically significant for the 228 patients in the 48 week trial. But the sub group pre specified that walked for between 300-400m at baseline showed 47m benefit that is highly significant. There would seem to be a window of opportunity in administering Translarna for boys who are still ambulant and can walk more than 300m in 6 mins. Of course this does not mean that other boys could not benefit (see data below for the North Star analysis) but administration at this stage appears to be most effective.
This shows graphically how this group maintained 6MWT vs placebo. The drug seemed much less effective for boys able to walk <350m at baseline but still better than placebo. These Phase 3 results seem to replicate the previously submitted data from the phase 2b trial.
PTC have also used the North Star Ambulatory Assessment as a secondary endpoint. Families in the UK will be familiar with this assessment undertaken by expert physiotherapists at our Neuromuscular Centres. These assessments are wide ranging standardised measurements of muscle function and relate directly to the natural progression of the condition for individual patients. Boys showed significantly improved scores in both the overall group and more significantly for the 300-400m base line group.
NICE, however, are still prevaricating on making a final decision despite having access to similar data from the 2b trial and the conditional approval from the EMA. At several meetings that I have personally attended with NHS England and NICE the argument comes up again and again about the cost of this drug and the Governments budget for specialised medicines supposedly a paltry £340m. Translarna is a high cost drug. We have no information on the price to the NHS but figures from £100- 200K pa have been suggested. However the Government have had plenty of time to make plans for funding high cost drugs for rare diseases. The Charity Action Duchenne has lobbied Governments on this issue since at least 2003. The APPG Muscular Dystrophy comprehensive report for example was published back in 2013 but little has been done to plan for funding drugs like Translarna or the exon skipping drugs now going before the FDA in the US. The Government still has its head in the sand.
So the crisis of NHS funding in general stems from political choices. I don't remember there being much debate about bailing out banks to the tune of £500bn following the 2008 crash. However the support from families and the wider public is palpable with over 25,000 signatures from a recent petition shows real public support for funding drugs for rare diseases like Duchenne in the UK.
The data so far published by PTC shows a real window of opportunity for boys to take Translarna. The drug would appear to be very safe and not have the significant side effects of cortiocsteroids the current drug of choice. The delays are criminal and can not be justified on the basis of cost. Boys will be losing ambulation and will miss this important window to get maximum benefit from this drug. NICE and NHS England have a duty of care to ensure these young people have access to medicines that could improve the length and quality of their lives.
Fund Translarna............... and the Government must do much much more to help to support the efforts of families, Duchenne Charities, our Neuromuscular Centres of excellence to continue to monitor and improve treatments so that one day we will see a complete cure for Duchenne Muscular Dystrophy.
79 is hosted by Nick Catlin
Nick Catlin has a son Saul who has Duchenne Muscular Dystropy. He is a founding member and previous CEO of the Charity Action Duchenne. Nick now works with young people with Duchenne and their families at Decipha assessing for Special Education Needs and advising on reviewing Education Health and Care Plans.
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