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The Orkambi row explained

Q&A and timeline

Orkambi

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 (NHSE) 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.

NHS England's 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.

Is Vertex’s price reasonable?

This is the million dollar question, so to speak. Vertex’s UK list price for Orkambi is £104,000 ($136,000) for a year’s treatment. If England were to pay this price for the approximately 2,834 people who are eligible, it would cost around £295m, which would make it one of the country’s biggest drug expenditures.

In confidential talks, Vertex may well have settled for a 50% discount. But NHSE England’s offer of 500m over five years represented a 90% discount, or around £10,000 for a year’s supply of Orkambi.

Was it a mistake for NHS England to make its offer public?

NHS England had seemingly had enough of being blamed for the impasse, and decided to make its offer public last summer. As this was a break from the confidential talks that both sides have always agreed are useful and necessary, it certainly looks like a mistake – not least because Vertex could never accept such a public offer without exposing its price negotiations elsewhere in the world.

To what extent does the row reflect bigger industry/healthcare issues?

Orkambi does reflect wider concerns about rare disease drugs that are assessed via NICE’s single technology appraisal (STA) system. NICE maintains, however, that making exceptions to its QALY system for one set of patients will inevitably mean that others will lose out.

How is this stalemate likely to be resolved?

Both sides need a chance in order to climb down – and the first prerequisite for this will be a return to a confidential pricing deal. Obvious solutions include Vertex producing improved outcomes data for Orkambi (not likely, as it’s looking ahead to triple therapy) or a risk-sharing scheme, which it hasn’t been keen on so far. That leaves a compromise price cut, as part of the larger portfolio deal, as the most likely outcome.

The Orkambi timeline

July 2012: Vertex’s Kalydeco, the first medicine to treat the underlying cause of cystic fibrosis, is approved in Europe.

January 2013: NHS England (NHSE) agrees to fund Kalydeco for all cystic fibrosis patients aged six who have the G551D gene mutation – around 270 people.

November 2015: EU approval for Vertex’s Orkambi, the first medicine to treat the underlying cause of CF in people aged 12 + with two copies of the F508del mutation.

April 2016: NICE’s initial rejection of Orkambi despite evidence of its efficacy, stating cost and lack of long-term evidence about benefits.

May 2016: Orkambi and Kalydeco rejected in Scotland.

June 2016: NICE gives final rejection of Orkambi in England – the Cystic Fibrosis Trust suggests a solution using the UK CF Registry to give people access while allowing data on its efficacy to be collected.

October 2016: Orkambi negotiations between Vertex, the UK government and the NHS get underway.

December 2016: NHSE approves expanded use of Kalydeco to two-to-five-year-olds in England, benefiting around 50 children.

April 2017: Ireland, which has the highest per capita incidence of CF in the world, agrees a ‘portfolio’ deal with Vertex after seven months of negotiation. This covers Kalydeco, Orkamabi and pipeline drug therapies.

June 2017: CF Trust chief executive David Ramsden writes to Vertex and the NHS in all four UK nations, appealing to them to reach an agreement on pricing for Orkambi.

January 2018: Orkambi approved in Europe for 6-11 year olds.

February 2018: Vertex submits portfolio proposal to NHSE, similar to that adopted in Ireland. NHSE responds to Vertex portfolio with a counterproposal and releases a statement. Vertex says it is “astonished and dismayed” by NHSE’s initial response.

April 2018: Vertex meets with NHSE to discuss access to Orkambi and future treatments. This is followed by two more meetings in May and June, but yield no progress.

May 2018: 1,000 letters from children with CF and their families asking for access to Orkambi are handed in to Downing Street.

July 2018: Vertex brands NHSE’s behaviour as “outrageous” and “unconscionable”. The company says it has offered England the best value deal anywhere in the world, employing an independent barrister to verify the claim while still keeping the offer confidential.

July 2018: NHSE goes public with a ‘final offer’ of £500m over five years for Orkambi and Kalydeco, soon-to-be- approved Symdeco and a triple therapy in late-stage testing, without having to wait for NICE appraisals.

August 2018: Vertex is angered by NHSE making its price offer public. It refuses to file Symkevi with NICE, stating that it will only submit its data if NICE updates its appraisal process.

August 2018: Simon Bedson, Vertex’s SVP and general manager, international commercial operations, steps down. Ludovic Fenaux, hired from BMS, is named as his successor.

September 2018: Vertex, NICE and NHSE resume talks after summer impasse.

November 2018: Symkevi approved in Europe (tezacaftor/ivacaftor) for people with CF aged 12 and older who have two copies of F508del or one copy of F508del and one of 14 other mutations.

November: Promising data released from phase 3 trials of pipeline cystic fibrosis triple therapy that could benefit more than 90% of people with CF.

December 2018: Scottish Government agrees a deal with Vertex pending submission of Orkambi and Symkevi to Scottish Medicines Consortium (SMC), with interim access possible through the PACS Tier 2 system.

January 2019: NHSE’s John Stewart calls Vertex “extreme outliers in pricing and behaviour” ahead of the health select committee hearing.
European Commission approves a label extension for Orkambi for two-to-five year-old children with CF who have two copies of the F508del mutation, the most common form of the disease.

February 2019: Patient activist group Just Treatment says Vertex is holding children’s lives to ransom. It urges the government to use Crown use compulsory licensing to gain access to cheaper versions of Orkambi.

March 2019: House of Commons health select committee inquiry into Okambi row hears evidence from Vertex, NHSE, NICE and patient and clinical representatives. Outside the meeting, over 200 CF campaigners are joined by MPs taking part in a demonstration calling for immediate access to Orkambi.

March 2019: Vertex, NHSE and NICE meet with Matt Hancock to recommence discussions on cystic fibrosis medicines.

March 2019: Second meeting between Vertex, NHSE and NICE, with hopes of a breakthrough round the corner.

Article by
Andrew McConaghie

11th April 2019

Article by
Andrew McConaghie

11th April 2019

From: Regulatory

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