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Afinitor and Uptravi gain funding from NHS England

Rare disease treatments navigate tricky market access route

Novartis’ Afinitor in Tuberous Sclerosis Complex (TSC)-related epilepsy patients and Actelion’s pulmonary arterial hypertension (PAH) treatment Uptravi (selexipag) have gained funding from NHS England.

The two specialised, high cost treatments gained funding approval on 22 December from NHS England’s specialised commissioning budget, one of the most difficult routes for market access in the country.

Afinitor (everolimus) will be funded from April 2019 for patients living with Tuberous Sclerosis Complex (TSC)-related epilepsy, where the condition has not responded to standard anti-epilepsy medicines (refractory epilepsy).

TSC is a rare genetic condition affecting 1 in 6,000 people that can lead to growths in various organs of the body, most commonly the brain, eyes, heart, kidney, skin and lungs.

Around 70 people in England are currently prescribed everolimus for TSC-related kidney and brain tumours. NHS England estimates that a further 300 people in England will benefit from treatment with everolimus for TSC-related refractory epilepsy.

Louise Fish, chief executive of charity the TSA Association, said: “We’re delighted that NHS England has decided to fund this life-changing and potentially life-saving treatment from April 2019 onwards. We’ll be working with TSC clinics across England to help them get ready to prescribe this drug to more people who can benefit from it.”

Meanwhile the decision on Uptravi was also welcomed by patient groups and clinicians, some of whom have been fighting to gain access to the drug for three years.

The drug is the first oral medicine targeting the important prostacyclin pathway. Other therapies which target this mechanism can be hard to administer - inhaled iloprost requires the use of a nebuliser up to nine times a day.

If left untreated, life expectancy in PAH patients is poor; average life expectancy for some types of PAH can be two to three years. NHS England will be funding Uptravi in adults with one of the more severe forms of the disease (World Health Organisation [WHO] functional class [FC] III) who remain insufficiently controlled despite receiving oral treatment with two other classes of PAH medicines.

Robin Bhattacherjee, general manager, Actelion Pharmaceuticals UK & Ireland said the decision had brought “relief from a lengthy process” and meant England was now aligned with the rest of the UK.

In July NHS England had ruled the drug to be effective, but ranked it as a low Level 4 priority - and therefore did not clear funded on that occasion. This caused an outcry over the delay until the green light finally came in December.

robin

Actelion's Robin Bhattacherjee

“Selexipag has been licensed for more than two and a half years, and since then Actelion has worked tirelessly and co-operatively with NHS England to enable access to selexipag, meaning equal access for all patients across the UK. Actelion has a long-term goal to enable a normal life for patients with cardiopulmonary disease and to achieve this, it is important that we all work together to ensure timely access to beneficial medicines, so that no patients experience such a long delay in access to treatments.”

The approvals capped a year of some notable market access deals struck between NHS England and the industry, including funding approval for Roche’s Hemlibra in July.

2018 was also notable, however for the long-running and very public, and still unresolved row over cystic fibrosis treatment Orkambi between NHS England and the Department of Health and Social Care on one side, and Vertex on the other.

Article by
Andrew McConaghie

2nd January 2019

From: Healthcare

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