New Talks Aimed at Breaking Deadlock on Cystic Fibrosis Drug

Peter Russell

March 13, 2019

The Chairman of pharmaceutical company Vertex, Dr Jeffrey Leiden, held talks on Monday with the Secretary of State for Health and Social Care, Matt Hancock, in an attempt to break the deadlock over patient access in England to its combination cystic fibrosis (CF) drug lumacaftor-ivacaftor (Orkambi).

Continuing obstacles to making lumacaftor-ivacaftor, and the company's other CF medicines, available for routine NHS use has become subject to a parliamentary inquiry.

Dr Leiden, and key figures from the National Institute for Health and Care Excellence (NICE) and NHS England, were questioned by MPs on the Health and Social Care Committee last week over the failure to agree a pricing structure since July 2016.

The impasse, in which both sides blame each other, has been the source of frustration to patients and family members, with several demonstrating outside parliament while the committee was in session.

Lumacaftor-ivacaftor is licenced to treat CF in people 12 years and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which around 50% of people with CF in the UK have.

An estimated 2834 people in England could benefit from treatment with the systemic protein modulator.

Rejected on Cost-Effectiveness Grounds

NICE rejected lumacaftor-ivacaftor for routine NHS use in England on the grounds that its £104,000 per year list price tag was not a cost-effective use of resources.

The Scottish Medicines Consortium also rejected lumacaftor-ivacaftor for an estimated 243 people in Scotland on the grounds of cost-effectiveness and lack of long-term data.

Sir Andrew Dillon, chief executive of NICE, explained to MPs that it could typically approve treatments up to £30,000 per quality-adjusted life-year (QALY). "Our assessment of Orkambi was that it was somewhere around £350,000 per QALY," he said, "so it gives you some indication of just how far away, just the size of the mismatch, between the additional benefits that Orkambi brings and what the company wanted the NHS to pay for it."

NHS England confirmed reports that it was prepared to offer Vertex around £500 million over the next 5 years, or £1 billion over 10 years, to also include access to its existing CF treatment portfolio of ivacaftor (Kalydeco), the new combination drug ivacaftor/tezacaftor (Symkevi), and any upcoming treatments.

"In this case we are offering upfront money to Vertex in order to make some progress here," said John Stewart, national director, specialised commissioning at NHS England. "We recognise that patients have been waiting a very long time for some of these drugs."

NHS England and NICE were challenged by the committee over whether their price negotiation with the manufacture over lumacaftor-ivacaftor reflected an attempt to recuperate some of the money spent by the NHS on ivacaftor, which they now regarded as being an overly generous settlement.

In his evidence, Dr Leiden, chairman, president, and chief executive officer at Vertex, said the company had successfully negotiated reimbursement agreements in 17 countries, including Ireland and Australia.

He insisted that England had been offered "the best price in the world" for the drug, reflective of the country's high prevalence of CF.

Assessment Process 'Out-of-date'

Dr Leiden blamed a health technology assessment process which was a quarter of a century old which he suggested was no longer suited to measuring the cost-effectiveness of modern medicines.

"The policy has not caught up with these advances in science," he said, "so as these new precision medicines are made which have fundamentally different properties, because they treat the underlying cause of the disease – they extend life by decades – these older assessments, which were actually excellent for looking at drugs that had short-term benefits, aren't well suited to these kinds of drugs."

The Health and Social Care Committee is expected to produce its recommendations in the next few months. However, yesterday's meeting between Dr Leiden, Mr Hancock, and representatives of NHS England and NICE, suggested a renewed willingness to reach a deal.

A press statement from Vertex expressed gratitude to Mr Hancock for his "leadership" and said the company expected to hold further talks with NHS England and NICE next week.

After the meeting, David Ramsden, chief executive at the Cystic Fibrosis Trust said it was "important that all parties keep urgent focus on [the] needs of people with cystic fibrosis".


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