The Orkambi row explained

The Orkambi row still isn’t resolved. Who is to blame for this mess?

It’s a long story. Friction between pharma and NICE and NHS England is par for the course, as one side will always aim to maximise expenditure and the other minimise it. But the Orkambi row does stand out. One key factor is that back in 2013, Vertex managed to get a very favourable pricing deal for its first CF drug, Kalydeco. Specialised commissioning head John Stewart says this was because NHSE was new then and just finding its feet. He says it has overpaid for Kalydeco by £400m over five years – not a ‘fair and reasonable price’. This, along with a prolonged budget squeeze, are factors informing NHSE’s ‘hard ball’ tactics on a portfolio deal for Kalydeco, Orkambi and subsequent CF therapies.

From Vertex’s side, the company may have assumed Orkambi could get a deal similar to Kalydeco, which did not go through the NICE process. This means Vertex was unprepared for NICE’s rejection and a price per QALY score ten times higher than NICE’s acceptable threshold.

Vertex was also under pressure to maximise revenues, as England is one of the biggest global CF markets, and Vertex struggled to turn an overall profit until 2018.

How good is Orkambi compared to Vertex’s other drugs?

This is a key point – Orkambi’s data shows it is less effective than Kalydeco at improving lung function, but it is licensed to treat a larger proportion of the CF population – 40-50% compared to 5%. It is also slightly more expensive. That’s a far less favourable proposition in health economic and budget impact terms; Vertex argues that Orkambi will help pave the way for more effective pipeline treatments for even more of the CF population – but that’s not how NICE works.

Is Vertex really an ‘extreme outlier’ in terms of its behaviour?

NICE agrees with NHSE on this one. Its chief executive Sir Andrew Dillon – an ‘iron hand in a velvet glove’ sort of character – told the health select committee that Vertex was “unique in my 20-year experience of working at NICE in demonstrating so little flexibility in their expectations”.

Is the UK an extreme outlier in terms of market access for Orkambi?

The long stalemate certainly puts the UK far behind many comparable countries regarding Orkambi access. Germany granted very rapid approval after launch, and Ireland – which has the highest per capita incidence of CF – agreed a portfolio deal in April 2017. That’s not to say there haven’t been major rows elsewhere in the world.

France agreed to a managed access programme that allowed rapid access tied to later value assessment, and also includes a budget cap of €30m with much lower payment rates beyond that.

But in February 2018 a disagreement on this deal led Vertex to threaten to deprive France of trials of its new triple therapy. An outcry against this threat led the company to back down. Canada also recently refused reimbursement and Spain is caught in a similar stand- off to the UK with Vertex over pricing.

What does Vertex’s CEO Jeff Leiden say?

“We are at a pivotal moment for the global CF community. Within a few years, with continued investment and research, we hope to offer life- changing treatments for 90% of people with CF.” He points to 20 years of risky investment, with nearly 70% of profits reinvested in the last five years. “Our work in CF is not done and we need to continue to invest at very high levels if we are to achieve our goal of developing such medicines for the 90% of CF patients we think we can help.”

How influential have patient campaigners been?

They’ve played a huge role, led by the Cystic Fibrosis Trust, and also through individuals and groups on social media who are CF patients themselves or parents of children with CF. They’ve kept the pressure on two successive health secretaries, and have won the support of many backbench MPs to keep the issue in the spotlight. Campaigners tend to apportion blame equally to the government and Vertex, although one group, Just Treatment, points the finger more firmly at the pharma company. It has called for a Crown Use compulsory licensing of Orkambi, but this always looked like an outside bet.