Cystic Fibrosis

Andrew Gwynne Excerpts

Tuesday 8th December 2015 Westminster Hall

[Andrew Gwynne (Denton and Reddish) (Lab)]

It is a pleasure to be back, having spent four and a half weeks in cold, wet Oldham, running the Labour party by-election campaign. I pay tribute to my fellow shadow Ministers for standing in for me in numerous Westminster Hall debates. It is good to be back, and it is good to see you in the Chair, Sir Edward.

I pay tribute to my hon. Friend the Member for Dudley North (Ian Austin) for making sure that this important debate could take place. He is right that we need to make sure that access to pharmaceuticals is one of the most important policy areas. With the results of the accelerated access review coming out in the new year, the effectiveness of NICE is very much on the agenda.

I also pay tribute to the right hon. Member for Chesham and Amersham (Mrs Gillan), who spoke powerfully about not only Archie, but Duchenne muscular dystrophy, which is a terrible disease, and she is right that we need to do much more to make sure drugs are available to treat it. I hope the case she made for Translarna will not fall on deaf ears with Ministers, because such drugs can make a big difference to the quality of life of children such as Archie. The right hon. Lady has put that case very powerfully in her question to the Prime Minister, and in her contribution today.

In June 2009, the previous Labour Government adopted the European Council recommendation on action in the field of rare diseases, which recommended that member states should establish and implement plans and strategies for rare diseases. Following on from work set out before the 2010 election, the coalition Government published the UK strategy in November 2013. NHS England published its statement of intent with regard to the UK strategy in February last year.

Since then, we have had the five-year forward view, which reaffirms NHS England’s commitment to achieving better outcomes for people with rare diseases, and when the Minister concludes the debate, I am sure he will give us more detail about how he sees the points made by my hon. Friends the Members for Dudley North and for York Central (Rachael Maskell), the hon. Member for Strangford (Jim Shannon) and the hon. Member for Linlithgow and East Falkirk (Martyn Day), who leads for the SNP on these matters, and the right hon. Member for Chesham and Amersham. Rare diseases are a crucial part of the five-year forward view, and given that the UK leads in life sciences, there is no reason why we cannot start to push the boundaries on what is achievable in respect of the drugs available for rare diseases.

The problem is that, although each of the publications I mentioned set out some laudable intentions, the actions arising from those publications have been baby steps in comparison with what we actually need. Changes resulting from the Health and Social Care Act 2012 have left patients and professionals to navigate a labyrinth when accessing medicines that, in many cases, have already been approved or have received licences. That really should not be happening.

Before the last election, the Opposition said unambiguously that we would reform NICE from top to bottom to remove the requirement to enforce competition rules and to ensure that access to medicine was decided on the basis of a medical justification, balanced with consideration of how much money we had available. I think we all now agree that NICE needs some reform. The current appraisal system makes decisions based on 24 weeks of clinical trials data, but that understates the efficacy of drugs that provide long-term stabilisation of a condition.

Other Members have spoken of the frustration cystic fibrosis patients have felt at not being able to access new treatments because those will not be approved given the way NICE appraises them. As my hon. Friend the Member for York Central said, the NICE system is not set up to assess precision medicines, and the issue extends well beyond cystic fibrosis to other rare diseases. Members have spoken powerfully about cystic fibrosis, and what we have heard about could be an excellent platform for testing new ways of doing things, and that could, indeed, also be the case with muscular dystrophy. The appraisal system for innovative medicines needs to be overhauled and to be adapted to include personalised medicines. Until the system is prepared to look at the value of new medicines over time, instead of looking for more rapid effects, it will not be suitable for its purpose.

I have some specific questions for the Minister. I am concerned that the highly specialised technologies evaluation programme could limit access to medicines for people with rare diseases. There are widely held concerns that the process, which was introduced after the 2012 Act to appraise medicines for rare diseases, is opaque and that the topic selection process is out of date. Does the Minister have plans to work with NICE to update the selection criteria for the pathway? Am I right that the process does not take into account conditions defined by genetics, biomarkers or differences in clinical presentation? Will he look again at that? Will he, as other hon. Members have asked, meet representatives of Muscular Dystrophy UK and the Cystic Fibrosis Trust to discuss those matters?

I accept that there has been huge investment in life sciences in the UK, but the current system, which encourages investment in technology and does not facilitate patient access to it, is unsustainable and wrong. Without such reform as we have discussed, funding for research on the relevant medicines could dry up and we could lose crucial shots at tackling a lot of rare diseases once and for all. The accelerated access review is a timely opportunity to take a careful look at how people get access to the kinds of medicines that might change their lives. It would be a tragedy if we threw that opportunity away.

I do not doubt for a moment that the Minister is fully behind every Member taking part in the debate in wanting to expedite the availability of the drugs in question, and I am keen to hear more about how the Government plan to do that. I commend Members for speaking so powerfully in a consensual cross-party manner in today’s debate and at other times when we work in Parliament to promote such causes. I hope that the Minister will answer all the questions that have been asked, and offer a glimmer of hope for those people who seek access to such drugs.