4:54 PM
Feb 23, 2018
 |  BioCentury  |  Regulation

Herculean task for DMD

How Duchenne UK’s HERCULES consortium hopes to improve access to DMD therapies

Driven by the delayed reimbursement decision for Europe’s only approved DMD therapy, the patient advocacy group Duchenne UK has brought together seven companies in the space to create a set of shared tools that should expedite HTA evaluations for all of their therapies.

Project HERCULES was launched in January with Pfizer Inc., PTC Therapeutics Inc., Roche, Sarepta Therapeutics Inc., Solid Biosciences Inc., Summit Therapeutics plc and Wave Life Sciences Ltd. Steering Committee members include CureDuchenne and Parent Project Muscular Dystrophy (PPMD).

Duchenne UK co-founder and co-CEO Emily Crossley told BioCentury the impetus for the consortium arose out of the mounting frustration of parents of children with Duchenne muscular dystrophy (DMD) during NICE’s health technology assessment (HTA) of Translarna ataluren from PTC.

The small molecule received conditional approval in Europe in August 2014, but NICE didn’t recommend its use on the NHS until July 2016, nearly two years later.

Crossley noted the delay left some DMD boys without therapeutic options while their disease continued to worsen.

“You had families in the UK who were eligible for the drug when it was approved, who then watched their children come off their feet and stop walking in the time it took for them to discuss how to get it reimbursed,” Crossley said.

Translarna was not a straightforward case, as the drug’s efficacy data weren’t overwhelming. But HERCULES project director Josie Godfrey told BioCentury the review was also hindered by factors that had nothing to do with the molecule itself.

“There was...

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