Batten Disease

Debate between Seema Kennedy and Jacob Rees-Mogg

Monday 22nd July 2019 Commons Chamber

[Mr Jacob Rees-Mogg (North East Somerset) (Con)]

(Urgent Question): To ask the Secretary of State to intervene to ensure that funding is provided to treat those suffering from Batten disease.

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

I thank my hon. Friend for his question; he is a great champion for his constituents. We must ensure that all children with Batten disease receive world-class care and support. The Secretary of State has met families of children who suffer from the condition and has seen at first hand how cruel the disease can be. I pay tribute to my constituent Melanie Moffatt, whose amazing care for her daughter Matilda has been truly inspiring.

The whole House will recognise that a key element of providing world-class care is getting access to the most effective new medicines. The National Institute for Health and Care Excellence is the expert body, independent of the Government, that provides authoritative, evidence-based guidance for the NHS on whether new drugs and treatments represent an effective use of resources. If they do, the NHS is obliged to provide funding. In 2013, NICE introduced its highly specialised technologies programme, which supports access to drugs for very rare diseases such as Batten disease, through additional funding—up to £300,000 per quality-adjusted life year. However, companies still need to price their products appropriately and fairly. In this instance, Brineura has not been made available at a price that could be recommended by the NHS. NHS England stands ready to do a deal at a reasonable price, but this has not been possible so far. I urge BioMarin to sit down again with NICE and NHS England, as NICE has not yet published its final guidance, so that a fair and reasonable price can be agreed.

I assure the House that my Department and the NHS are working as hard as possible to improve the broader care and support for patients with rare diseases, including Batten disease. I reassure all Members that the Department is committed to ensuring that all patients with rare diseases have access to world-class medicines, care and support.

[Mr Rees-Mogg]

I thank the Minister for her response. I am grateful that she has not hidden behind sub judice on this occasion. Could she confirm to me that, while NHS England is obliged to follow a positive recommendation from NICE, it has the legal discretion not to follow a negative recommendation and can decide to pay for a drug? In the event that NHS England will not do this, what powers—if any—does the Secretary of State have to ensure that a drug is made available? If that cannot be done, in what way is NHS England accountable to Parliament for the decisions it makes, or is it entirely above accountability?

The point at issue today is that Brineura has now been available for two years and it is available in many other countries at a price that has been agreed between their authorities and BioMarin. We now know that three more children—leaving five altogether in this country, including my constituent Max—who ought to be receiving this drug are not. They suffer from a condition that means that they degenerate relatively quickly, and this drug can stop the decline in their condition. It is therefore urgent that this matter is addressed quickly, rather than continuing to allow time to pass with sick children getting worse. It really is a most important and pressing issue. In instances where the drug companies and NHS England cannot agree, but where other countries have agreed, I wonder whether there could be any system of arbitration to determine what is a fair price, because the development of these drugs is exceptionally expensive.

[Seema Kennedy]

I thank my hon. Friend for his questions. I will attempt to answer all of them.

In terms of governance, no, NICE is not above accountability. Ministers set the framework for NICE, which is a non-departmental body. The reason it was established was to have fairness—so that there was no postcode lottery on access to various drugs. It is important that medical experts and scientists make these decisions rather than politicians. Regular governance meetings are held between the Department and NICE. There is a framework agreement. Where the Secretary of State considers that NICE is failing, or has failed, to discharge its functions or to do so properly, he can direct NICE to discharge functions. If NICE were to fail to comply with the Secretary of State’s direction in those circumstances, he could discharge such functions himself. There is therefore a strong and robust governance system with regard to NICE.

It is not always very helpful to use other jurisdictions as a comparison because we do not know the exact price that has been agreed. In addition, different systems have different healthcare populations and do not necessarily have the equivalent of our national health service.

Turning to access to Brineura, I pay tribute to my hon. Friend and to Max’s family. I know from the very moving testimony by him and by other hon. Members such as the hon. Member for Newcastle upon Tyne North (Catherine McKinnell) and from speaking to my constituent Melanie on numerous occasions that this is an absolutely dreadful disease. That is why we want the NICE process to be able to bring drugs to market as quickly as possible. Drug companies find this drug difficult to develop—that it is very expensive. It is not necessarily a drug that will be paid for by having millions of sufferers globally, and therefore a different system needs to be in place. That is why the bar for QALY is so much higher.

My hon. Friend’s suggestion on arbitration is very interesting, and I will take it away. On NHS England and the negative procedure, yes, in theory we could do that, but it is unlikely if NICE does not recommend a process. Overall, where a drugs company and NICE are unable to come to an agreement—we see this with other medication as well—Ministers urge the company to carry on negotiating to have a fair price, because every pound spent on one drug is a pound that we cannot spend on a drug for another sick person.

NHS (Charitable Trusts Etc) Bill

Debate between Seema Kennedy and Jacob Rees-Mogg

Friday 22nd January 2016 Commons Chamber

[Mr Rees-Mogg]

I am grateful to my hon. Friend for that intervention, but I am afraid that I think that most public consultation is bogus. It is about going through the motions and pretending we are interested in views when the Government, or councils or whatever else, want to get on and do whatever they wish to do anyway. It simply allows opportunities for judicial review to gum up the process. We should be incredibly cautious about chucking public consultation into Bills, because that does not actually achieve anything.

[Seema Kennedy (South Ribble) (Con)]

Does my hon. Friend agree that our constituents would look on agog at a Bill designed to simplify the process requiring a three-year consultation followed by yet another one?

--- Later in debate ---

[Mr Rees-Mogg]

What a pleasure it is to see you taking the Chair, Madam Deputy Speaker. We have been waiting for this happy hour to arrive to help us carry our debates forward.

No, I do not think my hon. Friend is right. When people give money to a charity that is linked to the Government, they are even more concerned that it will be spent well, and they therefore want extra protections to assure them of that.

[Seema Kennedy]

Does my hon. Friend not agree that the entire point of the Bill is to dissociate the charities from the Government and to provide independence, which is what gives them such a great reputation in their local areas?

[Mr Rees-Mogg]

As I said, it is a question is how we get to where we are going from where we are starting. As we make the transition, it is absolutely crucial to ensure that the money is handed over in a way that is properly audited so that people can have confidence in the NHS charities and not feel that there is some kind of sleight of hand or money is being siphoned off.