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#OrkambiNow?

After a long standoff over CF treatment Orkambi, is a deal imminent?

Orkambi

Photo credit: Cystic Fibrosis Trust

The long-running row over access to Vertex’s cystic fibrosis drug Orkambi has been one of the worst episodes for access to new medicines in the UK, and has reflected badly on the pharma company, budget holder NHS England and cost effectiveness watchdog NICE.

The row boiled over into accusations being traded publicly – Vertex calling the health service budget holder’s stance ‘outrageous’ last summer and NHS England’s head of specialised medicines commissioning John Stewart in January calling the pharma company an ‘extreme outlier in terms of both its pricing and behaviour”.

In the middle, of course, are the cystic fibrosis patients, many of them young children, and their families. People with this genetically inherited disease are vulnerable to potentially deadly lung infections, and currently half the people in the UK with CF die before the age of 31.

For them and their families, the row is all the more agonising as Orkambi, which has transformed lives for some patients, is available in other countries, and yet just out of reach for them.

Now after three years of an impasse, there looks to be light at the end of the tunnel. But some big questions remain: could this delay have been avoided? Can any one side be blamed? And perhaps most importantly, what needs to be done to avoid a similar breakdown in relations and the system occurring again?

The lack of progress since Orkambi gained EU approval in 2015 has been agonising for CF patients and their families, with the two sides unable to agree a pricing deal for the treatment, which has a UK list price of £105,000 ($139,000) for a year’s treatment.

The US company, based in Cambridge, Massachusetts is a pioneer of groundbreaking ‘precision medicine’ drugs against cystic fibrosis Kalydeco (launched in 2012) and Orkambi (2015) and now Symkevi (2018).

These are first drugs to tackle the underlying mechanisms of the disease, which are acknowledged by clinicians and healthcare payers as a major step forward in treating the disease.

While the company has been lauded for its breakthroughs, at the same time it also stands accused of ‘price gouging’ and exploiting its monopoly on CF treatments. This has led to renewed scrutiny of pharma’s profit motive, and in response a more militant patient activist group in the UK, Just Treatment, has bee formed, calling for the UK government to issue a compulsory licensing of Orkambi.

But NHS England and NICE have also been accused of inflexibility and presiding over a ‘broken’ market access system, with a confusing and complicated array of multiple routes to market, which often fail to capture the full value of a medicine, and don’t allow risk and reward to be shared between the NHS and pharma.

#OrkambiNow?

A breakthrough now appears in sight after the two sides were called in by the House of Commons health select committee to explain the standoff last month. Vertex CEO Jeff Leiden flew to London to give his side of the story, alongside his counterparts from NHS England and NICE, and market access talks have now resumed.

A market access deal with Scotland’s government has provided new impetus to the talks in England, with the hope that both sides can climb down and reach agreement soon.

The impact of this long delay has been very real. While it is impossible to say for sure, it look likely that in three years of waiting for Orkambi, people have died from the disease who might have lived had they received the drug.

Nick Medhurst, is head of policy and public affairs at the Cystic Fibrosis Trust, and is a veteran of the battle for access, having been in the post since around the time of Kalydeco’s arrival in 2012.

He says that because Vertex has been operating a named patient/ compassionate use scheme, there has been an opportunity for around 600 CF patients in the UK to receive Orkambi.

Stressing that it is not possible to back up this ‘what if’ with hard data, he nevertheless he believes that access could have saved lives in that period.

“I’m certain that there are people who have died who would be with us today if the UK had been able to find an access solution. For example in Germany, from the time of European licensing, adults and children of 12 and over were able to access Orkambi if they had the right mutations.”

Looking to the future, Vertex and other companies will be developing more potent treatments, including gene therapies which could one day cure patients of this terrible disease.

The hope is that both sides can come to a longer-term agreement which will help avoid similar clashes over the next drug in the company’s portfolio, Symkevi, as well as a late-stage triple therapy.

The answer is likely to lie, at least in part, in exploiting the UK’s CF disease registry to provide real-world evidence of the drugs’ effectiveness. This would represent a transition towards an outcomes-based reimbursement system, which is certain to be central to market access for many novel drugs in the future.

An undignified tit-for-tat

It’s difficult to untangle blame for the Orkambi affair, but it’s clear there are wider problems in how pharma and NICE and NHS England engage. However it’s equally evident that the Orkambi case also produced some serious errors of judgement on both sides.

So what of the system? Many observers say that Orkambi was a square peg in the round hole of NICE’s single technology appraisal (STA) system, when it was reviewed back in 2016.

The STA system doesn’t account for the rarity of a disease in the same way NICE’s Highly Specialised Technology (HST) appraisal for ultra- rare diseases can.

On the other hand, the trial data that Vertex submitted showed a relatively modest improvement in ppFEV1 lung function, and wasn’t able to demonstrate that it was linked to longer term health gains. This is despite reports from many patients of a transformative effect on their condition, and evidence that the drug can significantly cut pulmonary exacerbations, and the need for hospitalisation and intravenous antibiotics to treat these.

And what about how negotiations were conducted after NICE’s rejection?

NHS England says Vertex wouldn’t budge on its price for 12 months after NICE’s final ‘no’ and offered nothing new to the process, apart from challenging NICE’s methodology. It is also notable that the Cystic Fibrosis Trust’s suggestion of allowing interim access and use the UK CF registry to provide real- world evidence of the drug’s effectiveness - first suggested in 2016 – has not been taken up by the company.

This seems to reflect a belief that real-world databases aren’t yet robust enough for these purposes - but these will need pharma companies to be pioneers to make them work.

Based on a series of sniping press releases from both sides, it’s evident that negotiations between Vertex and NHS England got off on the wrong foot back in late 2016, with limited face- to-face talks. Fast forward to July 2018, and the row had escalated to the point where Vertex branded NHSE’s behaviour as ‘outrageous’ and ‘unconscionable’. The company said it offered England the best value deal anywhere in the world, employing an independent barrister to verify the claim while still keeping the offer confidential.

Days later, NHS England went public with its offer of £500m over five years for Vertex’s portfolio – a figure which looked large, but worked out at a low £10,000 per patient per year price – comparable to the cost of older and less effective CF medicines such as Pulmozyme. Not only that, making the offer public broke the longstanding golden rule of confidentiality which allows drug companies to cut prices in one country without exposing them to reference pricing in all other markets.

The tit-for-tat continued when Vertex then refused to submit its next CF treatment, Symkevi, to NICE in August, saying it would only do so once NICE’s appraisal system was reformed.

Some of these hot-headed outbursts from both Vertex and NHS England are of course regrettable, but could simply be ‘extreme outliers’ which might well not crop up again. But some of these themes are familiar from other disputes, and leaves the questions of failings in both Vertex’s pricing and value strategy and NICE and NHS England’s methods.

New models urgently needed

It’s widely acknowledged that healthcare systems and the industry need to converge around new models, where healthcare savings and value, when generated by novel medicines, can be fairly, sustainably and rapidly rewarded.

There are signs that a new more powerful and commercially-savvy NHS England (its new director of the commercial medicines division being ex-pharma exec Blake Dark) will help minimise these clashes by building trust and risk-sharing models with pharma.

But Orkambi is not the only orphan disease drug stuck in market access limbo in the England – Biogen’s Spinraza and BioMarin’s Kuvan are both currently suffering similar fates, and rare disease campaigners say a fundamental re-think is needed.

For people with CF and these rare conditions, this vision of a less adversarial and more sophisticated approach to market access really can’t come too soon.

Article by
Andrew McConaghie

Is PME's editor

9th April 2019

Article by
Andrew McConaghie

Is PME's editor

9th April 2019

From: Regulatory

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