Accelerated Access Review

Jim Shannon Excerpts

Tuesday 13th December 2016 Westminster Hall

[Jim Shannon (Strangford) (DUP)]

It is a pleasure to speak in this debate. I congratulate the hon. Member for Dudley North (Ian Austin) on bringing it forward. One of the major issues that I seek to raise with Government, as the Democratic Unionist party’s health spokesperson, is the treatment of rare diseases and cancers.

Cystic fibrosis is a most debilitating life-limiting disease. It is believed that one in every 2,500 babies in the UK is born with cystic fibrosis. It is a disease that affects too many households in our nation and as such one that we must address to the fullest degree and in the best way possible. As a member of the all-party parliamentary group on cystic fibrosis, I have a great interest in this work and noted, with a small amount of hope, what was being labelled as a wonder drug—Orkambi, which was touted as having significantly reduced hospital admissions and slowing the decline in lung function in people with the genetic mutations that it targets. However, we all know that this year NICE was unable to recommend Orkambi, despite acknowledging the drug as an effective treatment for the management of cystic fibrosis. Since then, negotiations between the manufacturer Vertex, the Government and NHS England have reached a deadlock. Orkambi is a precision medicine that treats the underlying genetic cause of cystic fibrosis rather than just the symptoms, and is therefore very important.

Like the hon. Member for Dudley North and others, I would like to quote people from my constituency and from Northern Ireland—one is from my constituency and one is not. I was emailed by a man from Castlederg, the hometown of my mother’s family, regarding the failure of the NHS and NICE to recommend this drug for the prescription list. Although I had read much about the drug, the human aspect was made so clear in his letter:

“With the power to lift so many of the limits cystic fibrosis can place on people with the condition, it’s vital that access is granted without delay.”

I believe that many of us in this Chamber are here to highlight and draw attention to the plight of our constituents who are crying out for the hope that this drug could bring—the difference of quality years of life for someone suffering from cystic fibrosis. My friend from Castlederg also wrote about this example of a young lady who quite clearly needs help:

“I have first hand knowledge of this drug Orkambi because my daughter Rachel who suffers from cystic fibrosis has been on it for over three years now. Rachel took part in clinical trials for two and a half years and it has transformed her life. Her lung functions have risen by 19%”.

These are more than stats—this is about her life and how Orkambi has changed it.

[David Simpson (Upper Bann) (DUP)]

A constituent of mine, Charlene Barr, passed away at the age of 20, just days before she was due to visit this House to campaign for cystic fibrosis drugs. I ask the House to pay tribute to her and her family for the fantastic work they do in Northern Ireland to raise awareness of this issue and of cystic fibrosis.

[Jim Shannon]

My hon. Friend has put his constituent’s name in Hansard as part of this debate, and I believe that is a fitting tribute to her.

My friend from Castlederg also wrote of Rachel:

“Her lung functions have risen by 19%, she has gained a stone in weight and has had very little coughs or colds in this period of time. In CF terms this is massive.”

He said that Rachel was 25 years of age last January and that she

“is currently doing a PhD at University and Orkambi has really given her so much energy and strength to be able to carry out such a big undertaking. Rachel has been very fortunate as Vertex have kept her on Orkambi after the trials because I suppose it would be bad looking on their part if they took her off it...I think that its only right that people that are eligible for this drug should be given the chance to receive it and to prolong their lives for many, many years and maybe even save their lives. The problem is that NICE have told the NHS that it’s too expensive at around £104,000 per year. What price do you put on a person’s life?”

I understand the way things work and I understand well the arguments regarding the likes of pancreatic cancer drugs that could add an extra year to someone’s life versus more money for the research to find a cure, but this drug could make a life such as Rachel’s much better and could help her. The new 96-week data published recently show that Orkambi slows decline in lung function, which is the main cause of death among people with cystic fibrosis, by 42%. The data were unavailable to the NHS, as others have said, but they are available now. We look to the Minister to ensure that the opportunity is available for people to have Orkambi. People who are on Orkambi through the compassionate use programme are beginning to report total transformations in their health, including enough improvement to come off the lung transplant list.

I understand the time restrictions, but I will give one more example. So many people have contacted me, including Martin Keefe, whose beautiful granddaughter, Evie-May, was diagnosed with CF at three weeks old. She is now seven years old. Surely this is the time to begin this treatment, so that she has less irreversible lung damage and can look forward to a longer, healthier life. To be clear, I am not a scientist, a doctor or a researcher, but as an elected representative, I can listen to the difference that these drugs have made and could make to people’s lives—to Rachel’s life, Evie-May’s life and the lives of many others. The research that was not available at the time of the NICE guidelines is now available and it is compelling. With great respect, we are all conveying compelling evidence and information directly to the Minister.

The review is an opportunity to do the right thing by those suffering from this disease, particularly those such as Evie-May and Rachel, who has noticed such a change. It is for those people that I ask the Department of Health to end the stalemate and make a new decision. We look forward to the Minister’s giving them the Christmas present that they want and that we in this House all wish for. I understand the budgetary constraints, but the benefit of the drug appears to outweigh the financial cost. Rachel, Evie-May and others like them, UK-wide, deserve the chance to have the drug.

--- Later in debate ---

[David Mowat]

To be honest, I am not qualified to have an opinion on that. The right hon. Gentleman rightly said that decisions of this sort should not be made by politicians and that there has to be a process around them. It is clear that if NICE is presented by Vertex with new clinical data, or indeed new price data—this is perhaps equally relevant, but we have not really discussed it—a review could be carried out quickly without any need for us to go through the whole process again. There is a precedent for that, and if those data exist and Vertex presents them, they would be looked at. I give my commitment, and certainly that of the Minister responsible for this policy area, that that would be the case and there is no impediment to that. I do not want to raise false hopes by saying that, and I do not think I have done so. The fact that it is not a near miss—it is possibly out by a factor of eight or 10—implies that there is quite a lot of work to do on pricing.

It is worth recapping what other countries have done. Orkambi is available in Germany, although it appears from the data available that its use there is quite mixed, with perhaps no more than one in five eligible people having access to it. In France, the other country in Europe that has authorised it, Vertex has booked no sales yet this year. The picture seems quite mixed in those countries. The countries that have not authorised Orkambi include Scotland.

[Jim Shannon]

In my speech I mentioned a young girl from my constituency, Rachel, who has been on the Orkambi trials and shown exemplary improvements in her health. That is an example we can all point to of where goodness has come out of the drug for those who have had the opportunity to have it, and that is true not only in my constituency but throughout the whole of the United Kingdom of Great Britain and Northern Ireland.

[David Mowat]

There is no dispute that the drug works, and there is no dispute at all that it is life-changing. The issue before us is the extent to which it justifies a price tag of £300 million to £400 million versus other NHS priorities. All I can say on that is that it is right that the decision is not made by politicians, for the reasons given earlier by the right hon. Member for Leigh.

I was discussing the countries that have so far not authorised Orkambi. Neither Scotland nor the Republic of Ireland accepted that it was cost-effective, and it is not used in Scandinavia or Canada either.