The U.K.'s drug cost watchdog has opted not to back GW Pharmaceuticals PLC's cannabis-based drug for the treatment of difficult to control epilepsies in its draft guidance but said it will work with the company to address the issues raised, including cost.
GW Pharma makes Epidiolex, known as Epidyolex in Europe, to treat rare childhood epilepsy conditions Dravet and Lennox-Gastaut syndromes in combination with clobazam. The cannabidiol therapy was the first marijuana-based medicine to be approved by the U.S. Food and Drug Administration in 2018.
Britain's GW Pharma also brought the first medical cannabis drug to European countries in 2010 with Sativex, which treats spasticity in patients with multiple sclerosis.
Epidyolex received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use in July 2019, which is a recommendation for approval by the European Commission. However, the Aug. 23 draft guidance from the U.K.'s National Institute for Health and Care Excellence did not recommend the drug due to uncertainty about Epidyolex's long-term effectiveness, as GW Pharma's clinical trials for Epidyolex lasted "only" 14 weeks.
In addition, NICE said in an Aug. 23 press release that it was "not convinced about the way the company had modeled the effect on people living longer or having a better quality of life." NICE advises the state-run National Health Service on which medicines represent value for money.
NICE said GW Pharma's economic models did not accurately reflect Dravet syndrome and LGS and their effects on patients and families — for instance, accounting only for the main types of seizures associated with the conditions. GW Pharma's models also did not "explore scenarios that allow for the effectiveness of cannabidiol to diminish over time," NICE said.
Nevertheless, NICE acknowledged that the clinical data GW Pharma has provided shows Epidyolex with clobazam reduces seizures, and patients and caregivers have noted the importance of Epidyolex's benefits.
"We are committed to working with the company to resolve the economic modelling issues identified by the committee, and to help them understand what they may need to do to mitigate the cost of cannabidiol to the [National Health Service]," Meindert Boysen, director of NICE's Centre for Health Technology Evaluation, said in the release.
The draft recommendations are open to public comment until Sept. 16, and NICE will publish final guidance on Epidyolex in November.
About 600 people in England have Dravet syndrome, and approximately 1 in 5 die from the condition, mostly before the age of 10, according to NICE. Lennox-Gastaut syndrome affects about 4,000 people in England, 5% of whom die prematurely from the disease.
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