Orkambi and Cystic Fibrosis

Debate between Crispin Blunt and Bob Stewart

Monday 19th March 2018 Westminster Hall

[Bob Stewart]

On my good friend’s excellent point, I wonder how much it costs to put a child in hospital for a month. I am pretty sure it is not far off £100,000.

[Crispin Blunt]

My hon. Friend leads me to my next point. How do we wish to value better and longer life for those with cystic fibrosis? How do we wish to value the savings to the NHS of reducing the 9,500 hospital admissions and the 100,000-plus hospital bed days a year? How do we wish to value the societal and economic benefits of helping young people grow up to play a fuller part in their education and employment, and the benefits to their families and care givers? How do we wish to value and reward the risk and innovation that goes into researching and developing breakthrough medicines?

NICE applies rigid health economics methodologies through its standard technology appraisal, which is designed not for rare diseases and specialised services but for primary care medicines that treat large populations with well-known diseases. Are we content for NICE to apply a threshold for valuing life that has not changed since it was established in 1999, even though healthcare inflation has almost tripled what we spend on healthcare?

My right hon. Friend the Member for Chingford and Woodford Green (Mr Duncan Smith) referred to social impact bonds and payment by results. I do not envy those who have to make those evaluations, such as the Minister and NICE. The costs fall on NICE and the health service budget, and the benefits are often felt elsewhere, not least in patients’ quality of life. Our society and the Government have to become more sophisticated about early investment. We must be able to measure the savings that come from having fewer hospital admissions and from the greater contribution to society that people who suffer cystic fibrosis will make if we improve their quality of life and reduce the degradation of their lungs.

Does the Minister accept that, although NICE has a specific evaluation process for highly specialised technologies for ultra-rare diseases, it is missing a framework for other rare diseases and precision medicines that treat sub-groups of rare diseases? He will no doubt point to initiatives such as the accelerated access review and the sector deal for the life sciences industry. I welcome those schemes in so far as they aim to address some of the access challenges, but they count for little if there is not a willingness to find innovative and flexible approaches to introducing innovation in the NHS. If we get this right, the UK has huge opportunities better to serve NHS patients and attract industry investment in clinical trials.

Vertex, the manufacturer of Orkambi, recently proposed a portfolio arrangement to NHS England, as we have heard from many colleagues, whereby all its current and future cystic fibrosis medicines could be made available to eligible patients at a fixed cost to the NHS, irrespective of the number of patients treated. Vertex wants to work with NHS England and NICE to put a long-term arrangement in place, as it has already done in Ireland. As the company introduces new medicines and line extensions, patients will get rapid access to the most suitable products for them upon regulatory approval, and the NHS will have budget certainty. Vertex expects to develop therapies that will treat 90% of cystic fibrosis patients within seven years.

In the light of the company’s proposal and the strength of feeling expressed in the petition and by colleagues in this debate, will the Minister look at mandating NHS England and NICE to prioritise discussions with Vertex to find an innovative and sustainable funding solution? It gives me hope that the offer that Vertex found unacceptable last Friday included a possible portfolio approach. It failed simply because NICE was not prepared to increase the resources it already pays for existing drugs, not including Orkambi. That plainly meant that the offer could only be unacceptable to Vertex, given that it is proposing new treatments that are going to treat 10 times as many patients as are being treated by the drugs currently available.

This is urgent. As my constituent, Sharon Cranfield, said to me:

“Each day of delay is additional delay of ‘irreversible lung damage’”

for those with treatable cystic fibrosis.