NICE calls for more data, price transparency for Duchenne’s drug

A new treatment to treat the rare wasting disease Duchenne's muscular dystrophy (DMD) has been rejected by NICE in preliminary guidance.

England's health cost effectiveness watchdog says Translarna (ataluren) could help slow the progress of the disease, but has demanded more evidence of its effectiveness from pharma company PCT Therapeutics – as well as justification for its high cost.

DMD is a rare disease which kills most sufferers before the age of 30. There are currently no approved drugs which can halt it.

Translarna is licensed to treat a sub-group who have a mutation (known as a nonsense mutation) in the dystrophin gene. Around 8-13 boys are born with the condition each year in the UK.

The drug is the first ever drug approved to treat DMD, gaining European approval in August 2014. It has been available in several EU countries since last year, and Scotland has just approved its use in one boy via an individual funding request (IFR).

However, as with all new rare disease treatments, Translarna carries a high price: NICE estimates the drug would cost, based on the average patient, £220,256 per patient per year.

This price makes it considerably cheaper than the last rare disease drug given preliminary approval by NICE - BioMarin's Vimizim, which costs £395,000 – but nevertheless it wants to see more evidence to support its use and reimbursement.

Professor Carole Longson, NICE Health Technology Evaluation Centre Director, said: "We are disappointed not to be able to recommend ataluren in this draft guidance. DMD is one of the most common and severe forms of muscular dystrophy. It reduces life expectancy and causes debilitating symptoms that severely affect the quality of life of people with the condition, and their families and carers."

NICE said, after its consultations with expert clinicians and patients, its committee agreed that the drug represents an important development in treating the disease, and could potentially prolong the time before children have to use a wheelchair, compared with best supportive care.

This is particularly important for the children and their families, as it could help them to attend school and engage in normal daily activities for longer.

NICE stated that it wants to see results of a confirmatory study of ataluren, which are due shortly. It will review the data, along with further clarification of the size of benefit ataluren provides for patients, carers and family members – as well as "further justification" of the cost of the drug.

Charities working in the field have expressed their disappointment at the draft decision, but remain hopeful that a 'yes' can be arrived at in the final decision.

Action Duchenne chief executive Paul Lenihan said: "I am hugely disappointed that this draft guidance was unable to deliver the direction to fund Translarna, but I am heartened by the language and direction given within it. It is clear that with the NICE's Health Technology Evaluation Centre Director stating her disappointment there was an appetite to grant such approval."

Lenihan said he believes that the demands for confirmatory data and an explanation on pricing shows NICE wants to ultimately direct NHS England to fund the drug.

However he is realistic about the timings of the next steps, predicting PTC will need extra time to complete the analysis of its data and submit it to NICE, which would mean that NICE's next planned meeting on 17 November would have to be postponed.

Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK, said the news was a 'bitter blow' for everyone awaiting the news, and called on NICE and the pharma company to arrive at a solution as quickly as possible.

"PTC Therapeutics needs to work with NICE and NHS England to find an urgent solution to approving this breakthrough therapy. We will not give up on these boys who are eligible for Translarna – they must have access to this life-changing drug."

The issue was thrown into the spotlight earlier this week when Prime Minister David Cameron discussed the case in the House of Commons. He said politicians should not have a say in the final decision on drug funding, but did call for more pressure on pharma companies to lower their prices.

PCT Therapeutics has not issued a statement in response to NICE's draft guidance.