Drugs: Ultra-rare Diseases

Luciana Berger Excerpts

Tuesday 16th June 2015 Westminster Hall

[Luciana Berger (Liverpool, Wavertree) (Lab/Co-op)]

It is, as ever, a pleasure to speak under your chairmanship, Mr Crausby. I, too, commend the hon. Member for Leeds North West (Greg Mulholland) on securing this important debate on access to medicines for ultra-rare diseases and on his contribution to this debate. He has pursued the issue doggedly through debates and questions, and it is right that he has been allotted time to bring these matters to the attention of the Government today.

I commend the UK parliamentary outreach team for hosting the online debate on this issue yesterday on Twitter, using #RDdebate. The public have had an opportunity to contribute to and inform this debate, and that is valuable. I am aware that many are watching us this afternoon. I also welcome the Minister.

When viewed collectively, it is more than apparent that rare diseases are simply not that rare. One in 17 people will be affected by a rare disease at some point in their life, which means that some 3.5 million people in the UK have a rare disease. About 75% of rare diseases affect children and almost one in three rare disease patients will die before their fifth birthday. These are sobering statistics and it is clear that more must be done.

In June 2009, the previous Labour Government adopted the Council of the European Union’s recommendation on action in the field of rare diseases, which recommended that member states should establish and implement plans or strategies for rare diseases. Following the work set out under the Labour Government, the coalition published the UK strategy in November 2013, and 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. While each of the publications is a step in the right direction, so much more needs to be done, as many have said this afternoon. The health reforms of the Health and Social Care Act 2012, which was introduced by the coalition, have seen patients and professionals left to navigate a labyrinth to access particular medicines that in many cases have already been approved and received licences.

We have heard already about tuberous sclerosis complex. It is a rare genetic condition that is estimated to affect 1 million people worldwide. Those with the condition develop non-cancerous tumours, often in the brain, eyes, heart, kidney, skin and lungs. Often, TSC patients are at risk of complications, and surgical removal of the tumours is not always an option. It can have a massive and often severe impact on a person’s quality of life. We have heard about a drug called Everolimus that has been developed to treat some tumours associated with TSC; it has been granted market authorisation by the European Medicines Agency. However, despite being licensed in the UK 28 months ago, it has not been appraised by NICE. It is only available through the NHS on an individual basis or through the cancer drugs fund, resulting in significant inequalities in patient access.

Another example we have heard about throughout this debate, and on other occasions in recent weeks in the House, is Duchenne muscular dystrophy, which leaves many patients wheelchair-dependent by the age of 12. The drug Translarna received conditional approval in the EU in August 2014 for the treatment of DMD. However, almost a year on, too many boys who could benefit are still awaiting a decision on funding from NHS England. I commend my hon. Friend the Member for North Tyneside (Mary Glindon), who has done so much to raise awareness of the issue on behalf of her constituents. As we have heard, many Members from all parts of the House also have constituents who are affected, and the hon. Member for Romsey and Southampton North (Caroline Nokes) made a passionate contribution. Each day of delay sees the boys come closer to losing the ability to walk, by which point they would no longer be eligible for the drug.

Countries across Europe have already approved the drug. The UK has taken a leading role in clinical trials for Translarna, but we are lagging behind other European countries in the delivery of the drug to patients. Will the Minister tell us why we have fallen so far behind? I understand that NHS England is set to take a decision on funding shortly. We often hear the word “shortly”, so will he provide a further update and clarify and qualify what “shortly” actually means?

The system to approve prescription is confusing and frankly chaotic. There are seven pathways through which drugs for rare diseases can be evaluated and made available to patients. I will not go through every one of them, but it is clear that there is no clarity in the process to decide on which pathway a particular drug will be put. In particular, owing to a lack of clarity and transparency in the process, information on how or why one medicine evaluation approach or access route is selected over another is simply not available. Will the Minister outline the steps the Government will take to clarify the process, to speed up decisions and to make those decisions more open, so that patients can better understand the process?

I have specific questions for the Minister on two of the pathways: the highly specialised technology evaluation programme, which is administered by NICE, and Evaluation through Commissioning, which is administered by NHS England. There is significant concern that they could limit access to medicines for people with rare diseases. There are widely held concerns that the process in the highly specialised technology evaluation programme, introduced following the 2012 Act to appraise medicines for rare diseases, is too opaque and that the topic selection process is out of date. Does he have any plans to work with NICE to update the selection criteria for the pathway, as they do not take into account conditions defined by genetics, biomarkers or differences in clinical presentations?

Do the Government have any plans to increase the resources available to NICE to evaluate drugs through the highly specialised technology evaluation programme route, given that it is only resourced for three drugs appraisals a year, despite the European Medicines Agency licensing more than four times that amount? Finally, it is essential that patient groups have input on the process by which the drugs upon which patients rely are appraised, but a consultation on the programme has not yet been announced. When does the Minister expect that to take place?

Evaluation through Commissioning is a specialised commissioning pathway to conduct pilots to collect data to inform the decision-making process on funding for specialised commissioning proposals. It is more than a year since Commissioning through Evaluation was expanded, and a few months since it was rebranded as Evaluation through Commissioning. Will the Minister update members on the progress the process has made in expanding patient access to drugs? As I understand it, no medicine has been selected for the programme. When does he expect that to change? Will he update the House on the effectiveness of the early access to medicines scheme to date?

There are more than 6,000 rare conditions. A disease can be described as rare, but having a rare disease is clearly anything but. Improving access to medicines and treatments for the 3.5 million people affected by rare diseases is crucial in improving their quality of life. We have heard moving personal stories from Members from all parts of the House in this debate. I was looking on Twitter at some of the contributions made by members of the public in the past 24 hours. One tweeter said:

“If I could go on the #vimizim I could start to work and pay taxes for others to get hold of the drugs they need”.

That is just one contribution among many, and I ask the Minister to reflect on them after the debate. Many issues need addressing to improve the system of medicine appraisals, and we have touched on just a few today. I hope the Minister will take on board what Members have said, and I look forward to his reply.