Translarna Benefits Don’t ‘Justify the Current Cost’: NICE Draft Guidance

Trial data show therapy boosts dystrophin production, preserves motor function

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The decision by the U.K.'s National Institute for Health and Care Excellence to reject coverage for Translarna to be covered to treat Duchenne muscular dystrophy is illustrated in this thumb's down graphic.

The U.K.’s National Institute for Health and Care Excellence (NICE) has published new draft guidance that does not recommend Translarna (ataluren) be covered to treat Duchenne muscular dystrophy (DMD) caused by nonsense mutations.

According to NICE, although there’s evidence Translarna can benefit people with DMD, it’s not worth its substantial cost. Translarna is sold by PTC Therapeutics at a list price of £220,000 (about $245,000 USD) per patient a year.

“We know that today’s provisional decision not to recommend [Translarna] will be disappointing for people with this devastating condition and to the people who care for them,” Helen Knight, interim director for medicines evaluation at NICE, said in a press release. “We will continue to work closely with the company and NHS England to try to resolve the issues identified by the committee in this draft guidance.”

Knight said those with an interest in the issue should submit their comments to the agency. NICE’s full draft guidance is available online and members of the public can submit their comments via its website.

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Translarna Preserved Walking 5-plus Years in DMD Boys

DMD is caused by mutations in the gene that provides instruction for making a protein called dystrophin that’s important for maintaining muscle health.

Translarna is designed to treat DMD caused by nonsense mutations, a specific type of genetic change that introduces a “stop” signal in the middle of the gene. Sort of like sticking a period in the middle of a sentence, this signal results in a truncated version of the dystrophin protein being produced that doesn’t function correctly. Translarna helps cells “read through” this early signal to produce a full-length dystrophin protein.

Benefits vs. cost-benefit

Data from clinical trials have demonstrated that treatment with Translarna can boost dystrophin production and preserve motor function in people with DMD caused by nonsense mutations.

The therapy has been available in the U.K. through a managed access agreement (MAA), which allows patients to obtain it while more data is being collected. About 60 children with DMD have received Translarna under MAA so far, according to NICE.

For the new draft guidance, a NICE committee reviewed data collected through the MAA, as well as other clinical and real-world data, on Translarna’s use. According to the guidance, there’s solid evidence Translarna can help preserve DMD patients’ ability to walk and their upper body strength, and improve outcomes even in patients who can’t walk, though its effects on the disease’s later stages or survival remain unclear.

“The Committee heard from and took into account the testimonies of patient experts who described how [Translarna] provided hope to people with DMD and their caregivers because it slowed down disease progression and allowed caregivers more time to adjust to different stages of the disease,” Knight said. “They also heard that one of the most important aspects of managing DMD is maintaining their child’s ability to walk because this means they can continue to go to school independently and participate more fully in social and sporting activities with family and friends.

“However, after considering all the available evidence, and the opinions of the clinical and patient experts, the committee concluded that the benefits provided by [Translarna] over standard treatment do not justify the current cost of [Translarna],” Knight said.

Under an agreement between NHS England and PTC therapeutics, patients treated in MAA will continue to have access to Translarna as long as their clinicians deem them to be benefiting from it, even if NICE ultimately does not recommend the therapy for coverage. MAA ends in January and patients diagnosed up to that point are allowed to start and continue treatment.