NICE has given PTC Therapeutics extra time to hash out a cost-effective price plan with NHS England before it will allow a final yes for funding through the country’s state-run healthcare system.
Just over two weeks ago NICE recommended that PTC’s ($PTCT) Duchenne muscular dystrophy treatment Translarna (ataluren) be funded for those with the disease caused by a nonsense mutation who are aged over 5 and still have the ability to walk.
NICE estimates the average cost (as it is dosed according to weight) to be around £220,256 ($319,000) per patient, making it one of the most expensive medicines it has ever assessed. This is why the drug is not being looked at under its normal HTA processes but rather under its new, ever-evolving highly specialized technology (HST) review.
This does away with the normal limits that will see it reject a new medicine that has a QALY of more than £30,000--and allows it to essentially pass through much more expensive drugs for rare or ultrarare diseases.
The HST remit has been bestowed upon the watchdog in the last few years by the British government, and NICE is still sorting out just how it should work under its auspices.
A NICE spokesman told FierceBiotech that it hasn’t used QALY for Translarna--as it would likely have rejected it outright--but is rather using a “wide-range” of considerations, including the impact of quality of life for patients and their carers, as well as the effect on NHS England’s budget (as it comes directly from its coffers).
“What we are looking for is a reasonable cost when assessed next to these factors,” the NICE spokesman said.
He added that as it is early days in its HST remit, it was still finding its feet when it came to timing and assessing cost-effectiveness. NICE has decided to give PTC until next week (11 May) to come up with a new “reasonable” price with NHS England and have that approved by the institute.
This is an unusual move in the normal run of things, as both PTC and NHS England were meant to have agreed upon a confidential agreement to cut the overall cost of the drug weeks ago. Seemingly NICE has had little choice but to allow them both more time to decide just how much of a discount should be made at the point of purchase.
Sir Andrew Dillon, chief executive of NICE, said: “We have allowed this extra time with the expectation that an agreement can be reached. The NICE committee was clear that ataluren has the potential to offer benefits to children with this cruel disease and their families and considered that it should be made available. However, because of its high cost, a financial agreement must be reached between NHS England and the company. We have granted this extra time in order for that to happen. It provides an opportunity for NHS England and the company to act quickly to resolve any outstanding issues on price so the guidance can be published.”
NICE told FierceBiotech that a price had not previously been reached, despite recommending the drug for use in England last month, and coming even though it appeared NHS England, NICE and PTC had reached a deal on costs. NICE does not usually say yes in its normal draft guidance unless it has a firm idea of what discounts will be made.
NICE did in fact knock the drug back last year, but lobbying pressure from parents and some sections of the media--as well as the all-important promise of a price cut--has helped it make a U-turn.
Should an agreement be reached, which NICE is confident it will, the drug will be made available on the NHS within three months to the 50 patients eligible for the treatment. If however no agreement has been reached in that time, NICE will advise the Department of Health that it is unable to publish the guidance. A decision is expected next month.
Due to the uncertainty around the long-term benefits of the drug, NICE’s committee also recommended that Translarna be made available under a “managed access” scheme to be agreed between the company and NHS England.
Under the scheme, patients will receive treatment while additional data from the 50 patients is collected. The scheme will be in place for 5 years, after which point NICE will review its guidance. If funded, eligible patients will remain on the drug until they lose the ability to walk unaided and treatment should be stopped no later than 6 months after this point.
In another highly unusual move, patients will also be expected to sign an agreement which makes it clear that if NICE does not recommend the drug when the guidance is reviewed at the end of 5 years, NHS England funding for Translarna will no longer be available for any patient and treatment will stop.
Duchenne muscular dystrophy caused by nonsense mutation causes progressive muscle wasting and is usually fatal by age 30. Children gradually become weaker and are often dependent on a wheelchair by the age of 12.
The drug has proven controversial after being rejected by FDA--with some analysts saying it’s 50-50 as to whether the European regulator will yank its approval.
This is because PTC has never actually produced statistically significant data to support the use of the drug for DMD cases spurred by nonsense mutations--about 15% of the total patient group. Their Phase II failed, and the Phase III flop came despite the fact that investigators designed it specifically to zero in on the positive aspects of the midstage study.
NICE has however been told that research suggested PTC’s drug could delay the use of a wheelchair by up to 7 years. In its guidance, released this morning, NICE said: “Ataluren works by allowing the body to read over the mutation in the DNA and continue to produce dystrophin. It is unclear how long it remains effective.”
Scotland’s form of NICE recently rejected the drug as being too costly.
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