Drugs: Ultra-rare Diseases

Gerald Howarth Excerpts

Tuesday 16th June 2015 Westminster Hall

[Greg Mulholland]

That is a powerful suggestion, as is using the surplus from the tariffs that drug companies are expected to pay to form part of a fund. There certainly needs to be an overhaul.

[Sir Gerald Howarth (Aldershot) (Con)]

Will the hon. Gentleman give way?

[Greg Mulholland]

I will give way, but I am conscious that I have not gone through the process yet.

[Sir Gerald Howarth]

We are all extremely grateful that the hon. Gentleman has been so generous in giving way. Like him, I was at Downing Street last week, supporting my constituent Harry Barnley, who suffers from Duchenne muscular dystrophy. The headquarters of the Batten Disease Family Association are in Farnborough in my constituency. The hon. Member for North Tyneside (Mary Glindon) referred to ring fencing. Part of the problem is that there is a very small number of these cases and they are very expensive to treat. I wonder whether we should either ring-fence some funding or introduce a surcharge on prescription charges generally paid by the public, so that the funding issue is taken out of it. There are two issues: the clinical issue and the funding. If we remove the funding issue, we can concentrate on the clinical issue.

[Greg Mulholland]

I thank the hon. Gentleman for his contribution. I am sure the Minister will want to consider that in his drive for an appropriate system.

After NHS England suspended the use of the scorecard on 2 December, a meeting of the NHS England clinical priorities advisory group on 15 December was called off. That is when we started campaigning for an interim process while NHS England went back to the drawing board. NHS England refused to do that, which I am sorry to say left all these families in the dark, with no idea what would happen next or in what timescale. NHS England then launched a consultation on 27 January, with a new process for deciding which drugs to fund that closed on 27 April. We still have not heard the decision. We have been told that there may be a decision on 25 June, although that has not been confirmed in writing. I hope that the Minister will give confirmation today.

Linked to that are the recent NICE recommendations, and particularly those on Vimizim. Even though we were clearly told by NHS England that its decision on 25 June would not be dependent on NICE, it now says that it will not approve Vimizim because NICE will not do so in the short term. The whole thing is a fiasco and an embarrassment. I understand the Minister’s argument that we cannot have political interference. However, the Secretary of State for Health made clear when he appeared before the Public Administration Committee in the previous Parliament that he accepts that the buck stops with him. When things are wrong and when bureaucrats are failing, it comes to his desk and to the Life Sciences Minister’s desk. I urge the Minister to take that up.

I pay tribute to the MPS Society for its amazing campaigning, and particularly to the chief executive Christine Lavery, whose son Simon had Morquio and died in 1982 aged just seven. Her passion and her colleagues’ passion have inspired me and others, and we will continue to work with them. The enzyme replacement therapy produced by BioMarin, Vimizim, is currently supplied on a free trial by BioMarin to 34 patients around the country out of a total of 88 patients, so more people with Morquio are not getting Vimizim than are.

The list price for Vimizim is £395,000 per person per year. In October, BioMarin proposed a fixed-term arrangement with NHS England to supply the drug at a lower price for a number of years. After BioMarin’s offer in October, NHS England did not even reply, despite repeated follow-ups, forcing BioMarin to announce in February that it would cease to supply the drug after 11 May; that date was then extended to 25 June. Having heard nothing, BioMarin said that it would have to withdraw the drug.