Drugs: Ultra-rare Diseases

Cheryl Gillan Excerpts

Tuesday 16th June 2015 Westminster Hall

[Greg Mulholland]

I hope that we get answers today and a real promise of intervention from the Minister.

[Mrs Cheryl Gillan (Chesham and Amersham) (Con)]

I pay tribute to the hon. Gentleman’s work on this issue. I joined this campaign because of Archie Hill, a constituent of mine aged 10 who has Duchenne muscular dystrophy. No matter what the Minister says about drugs such as Translarna and the process that the hon. Gentleman is about to outline, which has been disgraceful, that drug is available in other European countries and we have still not cleared it for patients in the UK.

[Greg Mulholland]

Indeed. It was a pleasure to meet the right hon. Lady’s constituent, Archie, and his parents. These young people are inspiring us to campaign. She is absolutely right. We are debating the European Union Referendum Bill today in the Chamber. Other EU countries, and some non-EU countries, regard these treatments as effective and affordable, yet we do not.

I will fast-forward from the scrapping of the previous body to October 2014, when NHS England came out with the scorecard system. That is despite one of the clinicians involved, Dr Chris Hendriksz, saying on 22 October in an email:

“I would suggest the scoring is not used at all for decision making this round and I would rather have people acknowledging that they are making random decisions than to try and give some credibility to a process that was deeply flawed.”

That is from one of the senior clinicians.

NHS England none the less went ahead with the scorecard system to decide which funding should be prioritised. Suzanne Mallah and her 10-year-old boy Kamal, who has Morquio and is another inspiring young person whom I have been delighted to meet, saw that that was not only haphazard but discriminatory. With the help of the MPS Society, they threatened legal action on 28 November against NHS England on the basis that the scorecard was clearly discriminatory, that there was no policy explaining it and that there had been no public consultation on its use. Just one week after that, on 2 December, NHS England announced that it was suspending use of the scorecard because the MPS Society and Kamal were right and it was wrong.

--- Later in debate ---

[Mrs Cheryl Gillan (Chesham and Amersham) (Con)]

I pay tribute to the hon. Member for Leeds North West (Greg Mulholland) for raising this issue and for his untiring work. I agree with my hon. Friend the Member for Romsey and Southampton North (Caroline Nokes) and the hon. Member for Strangford (Jim Shannon), who made many of the points I hoped to make.

I am grateful to have a short time to raise the case of my constituent Archie Hill and his parents’ tireless campaigning to get access to Translarna for him. It is inspirational to see how this family, and many others, have campaigned for their children. I can imagine nothing worse than watching one’s child slowly lose their mobility, knowing that their life expectancy will not be as great as ours might be.

Translarna is available in other European countries. As we have heard, it is available in Greece, which is not in the best economic health. Only recently, in Germany, the Federal Joint Committee determined that it provides a benefit for ambulatory patients aged five years and above with the nonsense mutation. The rise in the PTC Therapeutics share price on the back of that announcement shows that the company is well placed, and its drug is being recognised right around the world.

There is an irony here. If the decision coming down the track goes against making Translarna available to the patients who deserve it so much, the question arises as to whether this is about cost. The decision will almost definitely be made on a cost basis. Day by day, I see millions being spent in my constituency on High Speed 2 when we cannot spend £150,000 to keep a 10-year-old boy ambulatory and enjoying his life. We must question where a Government’s priorities are, when there are such people in front of us and we see the pie-in-the-sky projects that Governments of all complexions sometimes choose to pursue.

The point I really want to make is that if the decision is against providing the drug—bear in mind the failed processes that it has gone through—the Government have a golden opportunity to rescue the dish from the fire. I do not think it will necessarily fall out of the frying pan. On 8 July the Chancellor of the Exchequer will deliver his emergency Budget. We have previously created a cancer drugs fund, so that expensive drugs could be available to save lives. Will the Minister have conversations with the Treasury to see whether the Chancellor will on 8 July announce an access fund for drugs for rare conditions? If Translarna was one of the drugs on the list, it would be available in time for Archie Hill and the other children we have heard about today. To me, the awful thing is that time is running out. I do not think that letting time run out for those children would be the mark of a civilised Government, when the cost involved is small compared with some other expenditures that Governments make.