The UK’s GW Pharma has insisted there will be no repeat of the controversy seen with cystic fibrosis drugs after NICE said “no” to regular NHS funding for its Epidyolex for rare forms of childhood epilepsy.
In first draft guidance NICE did not recommend Epidyolex (cannabidiol) with clobazam for treating Dravet and Lennox-Gastaut syndromes, forms of epilepsy that begin in early childhood but last for a lifetime and are difficult to control.
Both organisations have said they will work together to resolve issues that have emerged in an early assessment of the drug, saying that discussions are already under way to get the drug funded by the NHS.
Even though a NICE committee noted clinical trial evidence that the drug reduces the number and main types of seizures, the cost-effectiveness body rejected funding because the longer-term effectiveness is uncertain.
Its committee also had concerns over the economic model proposed by GW Pharma, saying it was unconvinced it reflected the underlying conditions, nor did the company explore scenarios allowing for the effectiveness of cannabidiol to diminish over time as often seen with other antiepileptic drugs.
The committee concluded that the models may not capture all aspects of severe treatment-resistant epilepsy that are important to people with these conditions, and to their carers and families.
For example, only the effects on health-related quality of life of reducing the number of main types of seizure are modelled, and not the effects of reducing other types of seizures.
But a company spokesperson said it is “hopeful” NICE will recommend the medicine, which is being reviewed early before it has been granted a European licence under a new process being trialled by NICE.
The spokesperson told pharmaphorum that the committee has only assessed the economic modelling and has not talked about price yet, adding the firm has been “reassured” by NICE’s overtures so far.
There is already a meeting arranged between NICE and GW Pharma for technical discussions and a positive recommendation could come before or shortly after European approval expected later this year, the spokesperson said.
The spokesperson conceded that the issues were in part caused by the decision to treat Epidyolex as a regular drug instead of as one to treat an ultra-rare condition that could be assessed using a specialist commissioning pathway.
As was the case with Orkambi for cystic fibrosis, the number of patients affected was slightly too large to be considered using the pathway giving more leeway in terms of price, and the kind of clinical measures acceptable in assessments.
Meindert Boysen, director of NICE Centre for Health Technology Evaluation, said: “Based on the evidence presented to it, the committee could not recommend cannabidiol with clobazam as an effective use of NHS resources.
“However, 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 NHS. Patients, carers and their families deserve no less.”
The FDA approved the drug in June under the brand name of Epidiolex in the US in Lennox-Gastaut and Dravet syndromes – and European approval looks almost certain in the coming weeks as the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion at the end of last month.
Positive CHMP opinions are passed on to the European Commission, which nearly always gives a marketing authorisation within a few months.
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