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NHS England says yes to Roche's Hemlibra

But stalls on funding for Actelion's Uptravi

Hemlibra

Roche’s new haemophilia A treatment Hemlibra has gained a valuable market access approval from NHS England.

The decision is just one of a slew of 'clinical commissioning policy' decisions NHS England announced on Friday, with 16 separate rulings on specialised medicines and other interventions unveiled.

While NICE is England’s best known market access gatekeeper, NHS England is in direct control of specialised commissioning, and negotiates directly on pricing and reimbursement on a select number of specialised and high cost medicines.

Hemlibra gained European approval in February this year for haemophilia A patients with inhibitors, antibodies which mean patients no longer respond to standard Factor VIII therapy.

Hemlibra is expected to become a blockbuster worldwide, especially as it has also now shown major benefits in all haemophilia A patients, cutting bleeds by more than two thirds compared to standard factor VIII therapy.  This broader use will have to wait for a later reimbursement decision in England, however.

Roche UK welcomed the breakthrough in reimbursement for patients with inhibitors in England, one of Europe’s most important markets.

“NHS England’s decision is a great step forward for people with haemophilia A,” said Liz Carroll from The Haemophilia Society. “The development of inhibitors is a very serious and challenging complication of haemophilia treatment and there has been an urgent need for new options. This news means people with haemophilia A who have inhibitors will now have access on the NHS to a new treatment that could improve their quality of life.”

Around 20% of people with severe haemophilia A develop inhibitors to factor VIII replacement therapy – the current standard of care, which means the treatment becomes ineffective. Bypassing agents are used in patients who develop inhibitors and fail immune tolerance induction therapy (ITI).

Brigitte Nolet, Rare Diseases Lead at Roche UK said, “We are delighted that NHS England has agreed to reimburse emicizumab, which has been shown in clinical trials to significantly reduce bleeds in people with haemophilia A who have inhibitors compared to on-demand factor VIII bypassing agents.  This announcement is credit to the collaboration between NHS England and the Clinical Reference Groups which has ensured access to emicizumab for patients with inhibitors with limited treatment options.”

The date that patients will be able to access Hemlibra is expected to be announced shortly.

Hemlibra’s price in the US is $482,000 for the first year and $448,000 thereafter, though prices in Europe will be considerably lower.

As price negotiations between pharma and NHS England are strictly confidential, there is no knowing what price the two sides agreed, but it is safe to assume Roche had to offer a hefty discount to its UK list price.  The highest dose vial of the drug 150 mg/ml, 1 vial of 1 ml  costs £12,076.50.

There are an estimated 5,930 patients registered with Haemophilia A in the UK, of which 230 have factor VIII inhibitors. The majority of patients (71%) with inhibitors had severe haemophilia.

NHS England's controversial priority system

Hemlibra's market access was agreed outside the normal framework of NHS England's specialised commissioning, which ranks treatments and procedures 1-5, with 1 the highest priority. One generic treatment, teriparatide, has been listed as a Level 1 priority for men with osteoporosis.

NHS England says there is "sufficient funding available" for treatments ranked at levels one to three. it says three treatments ranked in either Level 4 or 5 can be considered in the next prioritisation round in November 2018 and if successful, could be funded in the 2019/20 financial year.

The system is very unpopular with many pharma companies, who say its workings remain less than transparent, and presents very few opportunities for assessment of new treatments.

Among the treatments and interventions listed in the latest review at 'Level 2' were Shire’s Obizur (susoctocog alfa) for patients with acquired haemophilia A (a rare form of the disease) and Sobi’s Kineret (anakinra) for periodic fevers and autoinflammatory diseases. Amryt Pharma’s Lojuxta (lomitapide) for homozygous familial hypercholesterolaemia (HoFH) was ranked as a Level 3.

One product which was recognised as effective, but ranked as a Level 4 priority - and therefore not funded on this occasion - was Actelion’s pulmonary arterial hypertension (PAH) treatment Uptravi (selexipag).

Actelion have hit out at the ruling, saying it will create a disparity in the UK, as patients in Scotland and Wales already having access to the treatment.

Selexipag 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.

Robin

“This disappointing decision now creates a grave disparity in access to selexipag which has been shown to be both clinically and cost-effective for use on the NHS in both Scotland and Wales,” commented Robin Bhattacherjee, General Manager, Actelion Pharmaceuticals UK & Ireland (pictured).

He added that NHS England’s medicine review process “works against patients with rare diseases such as PAH, and in effect, sees important treatments and subsequently patients de-prioritised.“

He concluded: “We ask that NHS England share our urgency and ensure this much-needed medicine is made available for patients and their families as quickly as possible.”

Read all 16 of NHS England's Clinical Commissioning Policies issued on 6 July here

Article by
Andrew McConaghie

9th July 2018

From: Marketing

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