Batten Disease

Sharon Hodgson Excerpts

Monday 22nd July 2019 Commons Chamber

[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.

[Mrs Sharon Hodgson (Washington and Sunderland West) (Lab)]

Thank you, Mr Speaker, for granting this urgent question. I thank and congratulate the hon. Member for North East Somerset (Mr Rees-Mogg) for securing it following his Adjournment debate last week. I do not doubt that he would have preferred the Minister to have come before the House voluntarily, rather than being forced to come here today for his urgent question.

Time and again, we come to this place to talk about a drug and its benefits to patients, only to be told that no matter how good it is, people cannot access it on the NHS. Among all the politics, there are people, including children like Max, who are suffering. No parent wants to hear a critical diagnosis for their child who has not yet really experienced childhood, let alone reached adulthood.

As we have heard, Brineura, a drug made available by BioMarin, could stop the progression of Batten disease. An assessment by NICE has found that Brineura could provide 30 extra years of good-quality life to patients. But, as has become expected when we discuss drugs for rare diseases in this place, Brineura is not available for patients on the NHS. NICE confirmed earlier this year that it was unable to recommend the use of Brineura on the NHS because of cost-effectiveness. The drug costs over £500,000 per person for each year’s treatment. BioMarin has another drug for rare diseases—Kuvan, for patients with phenylketonuria, or PKU. PKU patients do not have access to Kuvan, because it is also deemed not to be cost-effective. Does the Minister agree that the NICE appraisal process is just not fit for purpose when it comes to assessing the suitability of drugs and treatments for rare diseases?

Access to Brineura would help to give patients and families their child back, and it would allow them to enjoy time with their child and treasure special moments with them. As time ticks on without access to the drug, parents will witness their child’s condition deteriorate. No parent wants to see that, so we really need an appraisal process that captures rare diseases effectively.

Will the Minister step in and personally urge BioMarin, NHS England and NICE to meet and come to an agreement? Families do not want just warm words from the Minister; they want and need access to medicines now. I hope that this urgent question will result in real change in how we address rare diseases.

[Seema Kennedy]

In answer to my hon. Friend the Member for North East Somerset (Mr Rees-Mogg), I urged BioMarin to get back around the table with NHSE and NICE and come to a fair and reasonable price. NICE has already approved drugs for 75% of rare diseases through its technology appraisal programme, including drugs for idiopathic pulmonary fibrosis and neuroblastoma. NICE’s process and review methods are constantly reviewed, and they are internationally respected. NICE knows that it has to keep up to date with developments in science, medicine and healthcare. There is a periodic review going on at the moment, and that includes extensive engagement with stakeholders.