NICE draft guidance says ‘no’ to cystic fibrosis drug Orkambi
pharmafile | March 23, 2016 | News story | Research and Development, Sales and Marketing | NICE, Orkambi, Vertex, cystic fibrosis
NICE has issued draft guidance not recommending Vertex’s Orkambi (lumacaftor/ivacaftor) for use on the National Health Service (NHS) in England for people with cystic fibrosis (CF) aged 12 and above who have two copies of the F508del mutation.
The decision has been questioned by the company behind the drug, Vertex, which concluded that NICE’s appraisal system was inadequate to judge the value of drugs to treat rare diseases, particularly as the price watchdog had recognised the drug’s potential in treating the condition and reducing hospital stays.
Simon Bedson, the company’s senior vice president and international general manager, comments: “Vertex is pleased that NICE has recognised the significant clinical benefits of Orkambi, and, in particular, that the reductions in pulmonary exacerbations were ‘clinically significant and important for the management of cystic fibrosis.’
“We are not surprised that despite this recognition, NICE has not recommended its use in their draft guidance. Since the beginning of our discussions with NICE, Vertex has made clear our belief that the single technology appraisal process is not appropriate for assessing medicines, such as Orkambi, for rare diseases like cystic fibrosis, as it does not take into account the full benefits these medicines can bring to patients. We are concerned that application of the wrong appraisal process may lead to no access to Orkambi for eligible patients in England, who currently do not have a medicine that treats the underlying cause of their disease.”
NICE, for its part, said its independent appraisal committee had concluded that “the cost of Orkambi was considerably higher than the current standard of care and it could not be considered a cost effective use of NHS resources.” NICE noted that the drug costs £104,000 per year per patient and that this cost would be exacerbated by most patients remaining on currently-available treatments while taking Vertex’s drug.
Cystic fibrosis is a rare, life-threatening genetic disease that causes progressive damage to a number of organs throughout the body. In the UK, approximately half of those with CF die by their late 20s.
Orkambi was approved by the European Medicines Agency (EMA) in November 2015 for use in people with CF ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It was heralded as the first medicine to treat the underlying cause of CF in people with two copies of this mutation.
“Vertex supports the need for a full, fair and robust assessment of ORKAMBI and remains committed to continuing to work with NICE, the NHS in England and the Government to make ORKAMBI available to all eligible patients,” adds Bedson.
Vertex confirmed that it is engaged in health technology appraisals with Scottish, Welsh and Northern Irish regulators, in a bid to hasten access to eligible cystic fibrosis patients throughout.
Meanwhile, The Cystic Fibrosis Trust called on the NHS and Vertex to come to an arrangement to ensure such access.
The Trust has proposed a solution that would see Orkambi provided to all who need it while further evidence is collected on its long-term impact via the UK Cystic Fibrosis Data Registry. The Registry, a database sponsored and managed by the charity, holds health data for 99% of people living with the condition in the UK and is able to track the impact of treatments using real-world data.
Ed Owen, chief executive of the Cystic Fibrosis Trust, says: “This decision by NICE today will come as a blow to thousands of people that desperately need Orkambi. It would be ethically unforgiveable if people with cystic fibrosis were treated like pawns in a bigger battle between the NHS and Vertex over price and longer term impact.
“But there is a solution that requires the NHS in England to work with Vertex to provide access to Orkambi and enable the UK Cystic Fibrosis Data Registry to elucidate the drug’s impact more clearly over time.
“The data that is collected will assist in the discussions on longer-term impact and cost. It is vital that this exciting new and clinically effective therapy is not denied to those that need it on the grounds that further evidence is required to justify its cost.”
The Cystic Fibrosis Trust has written today to UK Life Sciences Minister, George Freeman, urging his intervention to support the charity’s plan, and launched an e-action to galvanise support for their plan and encourages as many people as possible to take part. The final decision from NICE is expected at the end of April.
Joel Levy
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