Orkambi and Cystic Fibrosis

Crispin Blunt Excerpts

Monday 19th March 2018 Westminster Hall

[Crispin Blunt (Reigate) (Con)]

I join colleagues in congratulating my hon. Friend the Member for Sutton and Cheam (Paul Scully) and the hon. Member for Dudley North (Ian Austin) on promoting this cause and on drawing parliamentary attention to it in an extraordinary way in this debate. I have a constituent, Sharon Cranfield, whose 16-year-old daughter, Jessica, has cystic fibrosis. This is about the grassroots and all those people who are affected. She is but one of those who have done an amazing job in promoting this petition and in campaigning for access to Orkambi for the 3,000-plus people who could benefit from it in England.

Last summer, Jessica was given access to Orkambi on a compassionate use scheme for those with the lowest lung function, and her quality of life has been transformed. The medicine reduces the mucus that builds up in the lungs, causing infection and damage. She is already benefiting from fewer exacerbations, which, before the treatment with Orkambi, caused hospitalisations about four times a year for up to a month at a time, taking her out of school. Judging by stories such as Jessica’s and the growing body of evidence that shows that the medicine is working effectively to keep children and young adults what cystic fibrosis out of hospital, there is a clearly a very strong case for making it available on the NHS.

Although many other countries have moved to reimburse this medicine in their healthcare systems, in the UK Orkambi remains unfunded more than two years since it received its European licence. The UK is falling behind our OECD peers in terms of speed of adoption, overall uptake, and expenditure on the newest medicines, and it is becoming increasingly apparent that the ways in which NICE assesses the cost-effectiveness of medicines are outdated and insufficiently flexible for precision medicines for rare diseases with relatively small patient populations, such as Orkambi.

[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.