NHS England proposes managed access agreement for Vertex's cystic fibrosis drugs

Country : UK

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LONDON, 24 May (APM) - England's National Health Service (NHS) has made an improved bid to try and gain access to Vertex's cystic fibrosis drugs Orkambi and Symkevi after months of tough negotiations that seemed to have stalled.
NHS England is proposing a two-year managed access arrangement for both drugs, with a requirement for the collection of real-world data on patient outcomes, according to a document published on Thursday.
The plans were outlined in a letter from John Stewart, NHS England's national director, specialised commissioning, to Sarah Wollaston, the chair of the House of Common's Health and Social Care Select Committee, which last year launched an inquiry into why UK patients with CF do not have access to Orkambi (ivacaftor+lumacaftor) despite it being available in several European countries.
Orkambi was approved in Europe at the end of 2015 to treat CF patients who have the F508del mutation in both copies of the CFTR gene.
It was intended as a successor to Kalydeco (ivacaftor), which is approved in a smaller population of patients and is available in the England and Wales after being recommended by health technology assessment (HTA) body NICE. However, Orkambi was turned down by NICE, which deemed it not to be a cost-effective use of NHS resources in 2016 at a list price of £104,000 per patient per year (APMHE 47016).
NHS England later offered an all-encompassing £108 million a-year over five years for all of its cystic fibrosis medicines, including Kalydeco, Symkevi (tezacaftor+ivacaftor) and future treatments such as a triple combination therapy (APMHE 61254).
However, this was rejected by Vertex, after which the UK's Health and Social Care Committee held a meeting in March where representatives from Vertex, NICE and NHS England were questioned on the lack of access for patients (APMHE 62224).
Vertex and NHS England have since had further discussions leading to the new proposal, which includes a two-year managed access period for Orkambi and newer drug Symkevi where further data is collected that could then inform a further appraisal of both products by NICE, alongside any other newly-available evidence.
Stewart did not give specific financial details in the letter, saying this information is "commercially sensitive and confidential", but he did say the interim funding period offers a "significant increase in the per patient per year price for each medicine" compared to the earlier offer.
He added that at the end of the managed access period, prices could then either be adjusted upwards further or downwards depending on the NICE committee’s conclusions.
And if NICE concludes that the interim price had been set too high then the NHS would receive a rebate for the difference.
Stewart also referenced NHS England and NICE's recent deals to provided access for two other drugs that had previously been rejected: Roche's Ocrevus (ocrelizumab) for primary progressive multiple sclerosis and Biogen's Spinraza (nusinersen) for spinal muscular atrophy (APMHE 62923, APMHE 62984).
"This illustrates that where companies are willing to engage with NICE and price responsibly then deals can be reached that are of benefit to patients, taxpayers and industry," he said.
Vertex wrote a separate letter to the Health and Social Care Select Committee saying it will review the new proposal at a meeting next week.
An agreement is still far from certain, however, with the company saying: "We note however that their offer reflects the same annual spend on our CF medicines as their July 2018 offer."
It added: "As we have said previously, any agreement must recognise the significant value of these medicines and the substantial investment made in developing them, and it cannot jeopardise Vertex’s ability to deliver on its mission of developing future innovations for the treatment of CF and other rare diseases."



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