Cystic Fibrosis Drugs: Orkambi

Seema Kennedy Excerpts

Monday 10th June 2019 Westminster Hall

[The Parliamentary Under-Secretary of State for Health and Social Care (Seema Kennedy)]

It is always a particular pleasure to serve under your chairmanship, Mr Hanson. I thank my hon. Friend the Member for Sutton and Cheam (Paul Scully) for opening the debate on behalf of the Petitions Committee. I pay tribute to the more than 100,000 people who signed the petition, and I thank all right hon. and hon. Members who have spoken in the debate; I am sure that they will be rushing back for the wind-ups.

I have been very touched by the stories that we have heard today and the compassion shown by my hon. Friend and all hon. Members in speaking about cystic fibrosis and its physical effects, emotional effects and effects on mental health for those who live with it and for their families. It is a debilitating condition, and I know how absolutely desperate sufferers and families are for access to treatments.

I recognise the great work undertaken by the Cystic Fibrosis Trust and its strong voice in supporting families and bringing cystic fibrosis to the attention of parliamentarians. I also pay tribute to my young constituent Lucy Baxter, who was on “BBC Breakfast” this morning and who lives with cystic fibrosis. She spoke to me very soon after I became a Member of Parliament and is an absolute inspiration to me and to the whole cystic fibrosis community.

Today’s debate has been heartfelt and passionate. The stories that we have heard clearly make the case that Orkambi and other drugs for people with cystic fibrosis should be available on the NHS at a price that is fair and affordable. The Government and I share that view. As the Chair of the Health and Social Care Committee, the hon. Member for Totnes (Dr Wollaston), set out so clearly, we must remember that the NHS must use its budget fairly for the good of all patients. That is why we rightly have a system whereby experts, not politicians, determine the fair price for medicines, based on robust evidence. That system has helped many thousands of patients to benefit from rapid access to effective new medicines.

[Karl Turner]

Forgive me, but I genuinely do not know the answer to this—I find it completely confusing. If the Republic of Ireland and Scotland can get an interim agreement, why cannot we sort this out for patients here in England?

[Seema Kennedy]

I will talk about the interim measures, but I think the more important thing that we need to grip is having a permanent solution for everybody living with cystic fibrosis.

Throughout the negotiations, which are rightly being led by NHS England, the Government have been crystal clear that Vertex must re-engage with the NICE process. I know that hon. Members have questions about that process, and I will try to address some of the points that have been raised. The hon. Member for York Central (Rachael Maskell) raised more detailed points for me to consider; if I do not address them, I will write to her with more detail, but there are some points about the NICE process that I will address later in my remarks.

[Jim Shannon]

Would it be possible for the Minister to give her Department’s and her own opinion on the buyers’ group that is seeking to buy similar drugs from Argentina for cystic fibrosis? Have the Government given that possibility any consideration?

[Seema Kennedy]

I shall turn later in my speech to that specific avenue that some sufferers have gone down.

[Dr Wollaston]

The Minister referred to the importance of Vertex engaging with NICE. Does she share my concern that when I wrote to Vertex and NICE about the failure to make progress, Vertex assured me that it had contributed “substantial new evidence” on the three products in question, yet I subsequently heard from NICE that it had received only

“an overview of the clinical evidence”,

rather than genuine engagement? Will she join me in calling on Vertex to properly engage with the process, so that we can get the full evidence base on which to make these decisions?

[Seema Kennedy]

I agree with the Chair of the Health and Social Care Committee and urge Vertex to re-engage with the NICE process. To date, unfortunately, it has continued to refuse to accept the process or has suggested unacceptable conditions on the NICE value assessment of its product, which would render the outcome meaningless. That comes despite NHS England’s latest proposals offering to reimburse Vertex ahead of a positive NICE recommendation, which for a deal of this size is unprecedented; agreeing to implement real-world data collection, as the Committee has called for, to help Vertex to demonstrate the value of its medicines; and offering significantly increased prices in comparison with their offer last July.

NICE has a 20-year history and is internationally renowned and independent. Its methods and processes for the development of its guidance have been in place for 20 years, but it recognises that it needs to evolve. It continues to review its procedures to ensure that they remain fit for purpose; it is now undertaking a review of its technology appraisal methods in line with the commitment in the 2019 voluntary scheme, and it encourages all stakeholders to engage. NICE has recommended 75% of the drugs for rare diseases—some of which I will touch on later—that have been assessed through its technology appraisal programme for the eligible patient population.

Last week, the Association of the British Pharmaceutical Industry made it clear that

“NICE is the cornerstone of NHS efforts to ensure the price being charged by a company represents the value being delivered.”

Commenting on the current situation, it said that

“the APBI would always encourage companies to fully engage with NICE at all stages of the process.”

Furthermore, it commented on the current structure of NHS England’s proposed deal with Vertex, saying that

“the structure of the offer represents exactly the sort of flexibility the industry has been calling for, for some time.”

However, Vertex is willing to accept only its own valuation of Orkambi; I draw your attention, Mr Hanson, to comments directed at Vertex by Members from across the House, including those made by the shadow Minister, the hon. Member for Washington and Sunderland West (Mrs Hodgson).

[Jeremy Lefroy (Stafford) (Con)]

I am grateful to the Minister for giving way and I apologise for not being here at the beginning of the debate, because of important statements in the main Chamber.

My constituent William Smith, who is a pupil at Cooper Perry Primary School, has cystic fibrosis. Along with his entire class, he presented me with the facts of his case and with letters to the Government, and they made it quite clear that they expect not only the Government but Vertex to come to a decision on this matter. Is it not absolutely vital that a company such as Vertex should engage with the Government given that the lives and futures of people such as William Smith, my 10-year-old constituent, are at stake?

[Seema Kennedy]

My hon. Friend makes a very good point. Members from across the House have told very moving stories of their constituents, the lives they lead, and the stresses and the strains put on them by the lack of an agreement on this matter. However, other drug companies are developing medications for rare diseases, and agreements have been reached on those. I will turn to them very shortly.

We can look at what happened in Spain earlier this year, when Vertex did not accept the terms of Spain’s health outcome-related proposal. The Spanish proposal, which is similar to the recent NHS England offer, is based on the ongoing collection and interpretation of real world data. Why is that not acceptable to Vertex? I also note that dialogue between Spain and Vertex has been ongoing for three years, which is similar to the situation here in England.

We will never walk away, but Vertex must now agree to engage with NICE and we urge it to accept all the flexibilities that NHS England has put on the table. There is nothing unusual about Vertex that means that this is not the right thing for it to do. Recently, we have seen deals reached as part of the NICE appraisal process, including that for ocrelizumab, which is an innovative multiple sclerosis drug, that for Spinraza, which is for people with spinal muscular atrophy, and that for axicabtagene ciloleucel chimeric antigen receptor t cell, or CAR-T, treatments.

Given that Vertex remains an extreme outlier in both pricing and behaviour, it is no wonder that patients and families have been looking at alternative solutions to secure access to this drug, and we have heard about the buyers’ club. Hon. Members have also talked about Crown use licensing, and the hon. Member for Bristol East (Kerry McCarthy) talked about large-scale clinical trials.

Unless Vertex changes its approach and behaves responsibly, I have a moral obligation to look at these other options. Of course NHS England and NICE will carry on the negotiations, because a negotiated outcome is the desired option. However, I have no alternative but to look at these other options on the table.

[Clive Lewis]

I thank the Minister for giving way. I raised the issue of compulsory licensing, which the Government have within their armoury. Of course it would be a measure of last resort, but given that we are dealing with people’s lives, the quality of their lives and a company that is quite simply being intransigent, and greedy, surely that option should be considered. That would send a message to those pharmaceutical companies—that global pharma industry—that if they are going to be greedy and put people’s lives at risk, despite being made fair offers, this option could be used by our Government.

[Seema Kennedy]

I thank the hon. Gentleman for his intervention. As my hon. Friend the Member for Sutton and Cheam said in his opening speech, which was very well made, we recognise the importance of British pharmaceutical companies and that companies invest hugely in developing new drugs. However, as the other examples of drugs for rare diseases that I have given show, it is possible to go through the NICE appraisal process and reach an agreement with NHS England. As one hon. Member who is no longer in their place said, this is an offer for a long-term agreement.

Vertex is an outlier, and I would like to put that on the record.

[Mrs Hodgson]

Will the Minister give way on that point?

[Seema Kennedy]

Yes, and I hope that I will be able to remember the hon. Lady’s question.

[Mrs Hodgson]

At this point I should clarify, for the benefit of the campaigners who I have spoken to about the Crown use licensing option, that it is not an immediate solution from their point of view; I understand that it would take at least a couple of years. If an agreement can be reached, there would be an immediate outcome. That is why the campaign is called Orkambi Now; it is about trying to get the drug now. Although the Crown use licensing option would be an option to consider if nothing else can be found, it would not give the sufferers and their families the drugs as quickly as we would like.

[Seema Kennedy]

As always, the shadow Minister makes an excellent point. Crown use licensing is not something that any Government would consider lightly. It is very rarely used in health. It has probably not been used—my officials will correct me if I am wrong—since the 1970s.

The ideal thing is to get a deal, and deals have been done with other pharmaceutical companies; that is the point I want to make. As I have said, Vertex is an outlier in this regard, but that does not mean that I do not have an obligation to look at other options. I will do that.

[Kerry McCarthy]

Will the Minister give way?

[Seema Kennedy]

Can I just make a tiny bit of progress first?

[Kerry McCarthy]

It is on this issue.

[Seema Kennedy]

All right, given that we have more than half an hour left.

[Kerry McCarthy]

I really welcome the fact that the Minister is saying so passionately that she feels she has a moral obligation to act. The question is: when? I say that because Vertex has been in these talks for a very long time now. How much longer will it be given before the Minister decides to look at the other options?

[Seema Kennedy]

Well, I have already said it, so I suppose that means—

[Kerry McCarthy]

When?

[Seema Kennedy]

Now. We will look at other options to consider what other methods we can use. As the hon. Member for Washington and Sunderland West said, Crown use licensing has other risks, so the best option is to get a deal. However, we will look at other methods that might enable families to receive the drugs they need.

Some Members have said that Orkambi is available in other countries and asked, “So why not here?” Although that is true, it is also true that other countries have faced problems in agreeing an acceptable price with Vertex; around 50% of the global cystic fibrosis population is unable to access Orkambi. It is not approved for reimbursement in Spain, which I have touched on, or in Portugal, and it is not used routinely in France. It is also of note that the Canadian equivalent of NICE has rejected the drug, saying that the benefit of Orkambi is small and uncertain.

We do not have sight of specific commercial agreements where Orkambi is approved and we do not have the same population needs, as we know, because of the specific population that the UK has; compared with other countries, the UK has a very high proportion of people with cystic fibrosis. So I am not able to make comparisons with other countries. Cystic fibrosis affects about 10,500 people in the United Kingdom—a far higher figure than in other countries. It represents 12% of the global cystic fibrosis population. The UK is a very important market for Vertex.

I thank all Members who have spoken so passionately here today. In particular, I thank the people who have joined us here in the Gallery; I pay tribute to them for all that they do to support their family members. I also thank those people, such as Lucy Baxter, who work so hard to raise awareness of this issue. I thank members of the Health and Social Care Committee, which is carrying out a very thorough and transparent inquiry into this issue, helping to shine a light on it.

As we have heard in great and moving detail today, cystic fibrosis is a devastating, life-limiting condition, and the bravery of those affected should be an inspiration to us all. Drugs that improve sufferers’ quality of life should be available where appropriate, and I urge Vertex to do everything it can to price its medicines fairly and in a way that reflects the health benefits to patients.

NHS England and NICE will, of course, continue their efforts to reach an agreement with Vertex. Access to treatment for all patients is, and always will be, a priority for this Government. My Department has a moral obligation to look at other options now, and that is what we shall do.