#OrkambiNow?

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.