Orkambi and Cystic Fibrosis

James Frith Excerpts

Monday 19th March 2018 Westminster Hall

[James Frith (Bury North) (Lab)]

It is a pleasure to serve under your chairmanship, Mr Evans. My constituent Graham Little got in touch with me. His wife Claire has cystic fibrosis. Until that moment, I was not aware of the potential life-changing drug available to sufferers of cystic fibrosis. Graham and his father-in-law Brad raised with me the status of the precision medicine Orkambi. The result of today’s debate could make the difference and give them decades more with their wife and daughter, Claire, and give her decades more with her children. I cannot imagine how that must feel.

We can either observe the steady death caused by cystic fibrosis—it has been compared to drowning—or reach for the life jacket, which is primed, ready for use and in all sizes. Our country has the largest number of cystic fibrosis patients anywhere in the world, so we ask the Government what they are going to do for our cystic fibrosis sufferers. If they will not make Orkambi available, what are they going to do? The transformative, life-prolonging impact of the drug is tantamount to life-saving. Used early enough, it can ensure that people fulfil their potential. It can prolong life and lung capacity, enabling those with the disease to contribute to the economy and wider society, as well as providing extra decades with family and loved ones.

The Government should consider the case for a commissioning body for rare diseases. It is not right that the judgment criteria used to determine funding on globally common diseases are universally applied when the uncommon traits of a rare disease bring untypical symptoms and a different economic argument. We risk overlooking the incredible efficacy of the drugs now available.

The power in numbers is not much solace to a cancer sufferer, but such is the profile of cancer treatment that we are all assured of the progress being made there. The same cannot be said for the rare diseases argument, however. We have a job to do in making the case for rare diseases. I hope the Minister will listen to the case being made for a medicine and treatment decision-making body for rare diseases, as well as consider how we bring NICE into the modern age, 20 years after it was established.

[Liz Kendall]

My hon. Friend will know that the Government review NICE once every three years. Does he agree that the next review, which will start in July this year, must look seriously at how NICE makes its decisions? Does he also agree that the results of that review should come out quickly? Last time, it took about a year and a half.

[James Frith]

I completely agree with my hon. Friend. We need to see far more nimble-footed decision making by the decision-making body, as per my call for a specific dedicated body for rare diseases. Parents in my constituency make the case about the plight of those with rare diseases. There is a constant sense of those enduring rare diseases being overlooked.

We come to the remaining agents at the table. Just this weekend, NHS England has refused to agree to the deal that Vertex proposed last month. I do not think the wording of the refusal was constructive, but I am not surprised that the NHS was unwilling to sign up in principle to the deal, which amounts to a futures prospectus from Vertex off the back of a sure thing with Orkambi. Patient groups are at the table, and I commend the work that the Cystic Fibrosis Trust is doing on behalf of CF sufferers.

We need compromise and urgency from all. Nobody should expect Vertex to surrender its drugs, but neither should we accept a failure to reach an agreed price. I say to Vertex that the pipeline might need to be for another time. When I met Vertex, I cautioned that its “portfolio of drugs” approach may be cost-prohibitive to a decision, and that appears to have been the case. We need Orkambi for patients today. It is not the time to test the innovative decision making of NHS England. Vertex should take in good faith that a fair price to the world’s largest population of cystic fibrosis sufferers will set it up well for future developments. A price somewhere between what it was offering in its recent pipeline deal and a single purchase deal is where we are now. I ask Vertex to please stay at the table. We keep in mind its proposal of having Orkambi readily available, swiftly and easily, for all. We urge focus on enabling that. The rest will surely follow.

At the table, there is a ministerial chair that needs filling by a Minister who is willing to lean in to the debate and signal their support for prescribing Orkambi on the NHS; willing to lead and lean in to the deal-making part of the job to transform lives; willing to look at the costs and to help with the price; and willing to align the political will to the possibility of life for those with cystic fibrosis. Treatment for cystic fibrosis has moved from science fiction to science fact, so the Minister is required to act and step in. The Government have a choice to make. They can ignore our arguments, or, having heard them, choose to change their mind. In changing their mind, they can change lives and lifetimes. I urge the Minister to do that and make real-life change for cystic fibrosis sufferers a reality. He would have support from Members across the House, as has been demonstrated by the magnificent strength of feeling in the contributions and arguments made today.

--- Later in debate ---

[Steve Brine]

I thank 163 of my own constituents who signed the petition and who have been in touch with me, and one who has been to my constituency surgery. I may be the Health Minister responding to the debate, but I am also a constituency MP and a parent of two young children.

I pay tribute to the Cystic Fibrosis Trust, which does a tremendous job on behalf of all our constituents. I know we are not meant to address the Gallery. I will not do so, but if I did I would thank those who have made the trip in the snow and ice, probably not all from SW1, to come to Westminster today. It is a credit to them that they have made time to do that.

I recognise the progress that Vertex has made to date in its mission—I believe it is a mission—to discover ultimately a cure for cystic fibrosis, and recognise the treatments that it brings to the market. I know Vertex understands the importance of the UK market, and NHS England as a customer, to its future and to that mission.

This petition is calling on the British Government—that would be me—

“to call for a resolution to ongoing negotiations between Vertex Pharmaceuticals, NHS England and NICE as a matter of the utmost urgency. It is essential that a fair and sustainable agreement is found.”

I am here today to do exactly that. It is not a difficult call to agree to. The negotiations must remain constructive and be undertaken with the utmost urgency, for all the reasons we have heard—I will not repeat them all—or, as my hon. Friend the Member for Mid Dorset and North Poole (Michael Tomlinson) said, people living with cystic fibrosis will suffer.

It seems to me that the main ask I have heard from pretty much every hon. Member speaking today is, “Get involved.” I am not the Minister directly responsible. That falls to Lord O’Shaughnessy, the Parliamentary Under-Secretary who sits in the other place, but I can say on his behalf and on mine that we are involved, and you can bet your life that we will continue to be involved. I do not think I can be clearer.

[James Frith]

The Minister says, “You can bet your life” that he will continue to be involved. Any further delay is literally betting lives and the ever-diminishing lung capacity of those who go without Orkambi. What will he commit to do differently from today, and when will Orkambi be prescribed by the NHS?

[Steve Brine]

The hon. Gentleman knows that I cannot give the answer to his when question. I will come to the other bits of his question. His point about the reducing lung function is well made, and has been made by almost everybody. The Government wholeheartedly support efforts to ensure that the precision medicines we have heard about are made widely available to CF patients and other patients. It is true that high-cost precision medicines represent a challenge to the NHS, but they are also a tremendous opportunity to deliver high-quality outcomes through highly specialised treatment. I hope I can go some way to ensuring that people with comparatively rarer conditions such as CF get the same quality, safety and efficacy in medicines as those who have more common conditions, and to doing so in a way that is sustainable for the NHS.

There has been much talk about NICE, which has a difficult job, as the hon. Member for Bury South rightly said, but as we know, England has the second-highest number of cystic fibrosis patients in the world and there is sadly no cure. Current treatments generally target the complications rather than the cause of the disease. Of course, I can appreciate the huge daily burden of treatment for patients and their families, and the uncertainty that they live with. Uncertainty is a huge burden—the hon. Member for Dudley North (Ian Austin) mentioned the mental health burden. It is so important that patient voices are heard during any appraisal process to ensure that that burden is fully understood.

NICE’s technology appraisal programme makes recommendations for the NHS on whether drugs represent an effective use of NHS resources in what we must remember is a publicly funded health system, as many hon. Members have said. There are other parts of the world where, if someone could not afford it or their insurer could not afford it, this would not even be a discussion. That system means that patients can have the confidence that the price paid by the NHS is consistent with the improvement in health outcomes a medicine brings, ensuring fairness as well as the best possible use of funding for patients and the NHS.

Those are very difficult decisions to make, but it is essential that patients are getting the maximum benefit from every pound of our constituents’ money that is spent by the NHS. NICE takes its decision independently of Ministers. The Government rightly have no say in whether a new medicine is recommended for the NHS at the price proposed by the manufacturer. NICE published its final guidance on the use of Orkambi in July 2016 and, as we have heard, did not recommend it for use in treating cystic fibrosis. That, of course, is not the end of the story, and I do not want it to be. I will return to that in a moment—although, ironically in a three-hour debate, time is short for me to give our position.

Since 2013, NHS England has been responsible for securing high-quality outcomes for patients with cystic fibrosis. Six years ago, it agreed to fund Ivacaftor and Kalydeco for cystic fibrosis patients with the relevant genetic mutation. An additional possibility that may further the pool of treatment options for CF in the near future is the double combination therapy branded Symdeko, which has shown positive results and is currently undergoing European Medicines Agency licensing.

More broadly, the number of medicines for cystic fibrosis patients expected within the next three years is promising, with products being developed by a range of manufacturers as well as Vertex. NICE is aware of 31 other technologies by 19 different companies—not Vertex—that are in the pipeline for cystic fibrosis. Clearly, the prices for any new treatment will also be considered by NICE and we must ensure that the arrangements NHS England enters into now do not restrict options for patients to have the best available medicine in the future. The hon. Member for Bury North made that point very competently.

The NHS is in discussions with Vertex about Orkambi. Vertex has approached NHS England with a proposed deal to reduce its prices. While I cannot share the details of this proposal due to their commercially confidential nature—believe you me, I wish I could—I can assure hon. Members that, at the level that Vertex has proposed, the products are still far from cost-effective. Therefore, there is more talking to do.

Last week NHS England made a counter-proposal, which would ensure that the drugs could be used at a price that is cost-effective. I understand that NHS England has agreed to meet with Vertex—I would jolly well hope so, and of course it is not over email—to discuss that counter-proposal further. I have seen the statements made this weekend—I must say I am no fan of Twitter diplomacy in this or any other form of diplomacy. The statements this weekend were not exactly encouraging, but they must not be the end of the story.

While it is not for Ministers to approve, I truly believe this can be a mutually satisfactory arrangement for both parties. I know I speak for my colleague, Lord O’Shaughnessy, when I say that we are both impatient for a breakthrough and are watching the matter like hawks. I get the message loud and clear: the House has said, “Get involved.” The House has that assurance from me.

I thank all hon. Members who have spoken so passionately. They have helped the cause on behalf of their constituents. On Orkambi in particular, I hope above all that hon. Members can see that this is a live issue and that it is work in progress.