Fibrodysplasia Ossificans Progressiva

Will Quince Excerpts

Thursday 7th September 2023 Commons Chamber

[The Minister for Health and Secondary Care (Will Quince)]

Let me start by thanking my right hon. Friend the Member for Hemel Hempstead (Sir Mike Penning) for securing this important debate on FOP and for his powerful and compelling contribution. As he eloquently and articulately set out, FOP is an incredibly rare and truly devastating condition. He is a champion for people living with it and for their families, and I thank him for that. It is impossible not to be moved when reading about this currently incurable condition and when seeing pictures such as those he sent me about it. I am sure I speak for the whole House when I say that hearing about Lexi Robins and her parents, Alex and David, greatly moved me. I can only imagine what they must be going through, and I send my heartfelt sympathies to them and their entire family.

I also wish to express my admiration for Chris and Helen Bedford-Gay, who established the charity FOP Friends, after their son, Oliver, was sadly diagnosed in 2009. I understand from my right hon. Friend that they have raised more than £700,000 since then to fund research into treatment and to support families impacted by FOP. I am sure that many, many families are incredibly grateful for all their efforts.

At the outset, let me confirm that I am happy to join my right hon. Friend in meeting the Science Minister on this issue. We already work hand in glove on matters of research, and the House would expect nothing less. It would be remiss of me not to also welcome the hon. Member for Bristol South (Karin Smyth) to her place and to congratulate her on her promotion on the Front Bench. I also thank the hon. Member for Blaydon (Liz Twist) and my friend the hon. Member for Strangford (Jim Shannon) for their constructive and powerful contributions to this afternoon’s debate. I will endeavour, with your permission, Mr Deputy Speaker, to give as full and comprehensive a response as possible, given that I have far longer than any Minister usually has to respond to a Backbench Business Committee debate.

Let me kick off with a question asked by the hon. Member for Blaydon about the national genomic test directory. I scribbled those words down at pace as she was speaking. I will check and write to her specifically to confirm this, but my understanding is that FOP is a form of skeletal dysplasia, which is in the national genomic test directory. I will confirm that in writing once I have checked with officials on my return to the Department.

I want to assure the House and, of course, all families affected by rare diseases and conditions—I note my right hon. Friend’s point about FOP being a condition, but “condition and disease” is a phrase that the Department and clinicians often use—that we certainly recognise and understand the challenges they face. These diseases and conditions have wide-ranging impacts, not just on sufferers’ physical health, but on their mental health and on their involvement in the community, as he set out. That is why it is crucial that those affected by this condition receive the best possible care, from swift diagnosis to treatment, to improve their quality of life as diseases and conditions, sadly, progress.

To do that, it is vital that the UK harnesses our research prowess. Today’s debate has largely focused on the power of research and how it is often the key to unlocking so much. Our universities, scientific institutions and healthcare system very much lead the world in this space—we often say that we are world leading in lots of areas, but research is certainly one area where we are. They have enormous potential to develop solutions to improve the lives of people living with rare conditions, including FOP. Let me be clear that the Government are committed to funding this research.

In July, the Medical Research Council and the National Institute for Health and Care Research announced £14 million of investment to launch the UK rare diseases research platform. That is bringing together expertise from across the UK to foster new and innovative treatments for rare diseases and conditions, and to accelerate work to improve diagnosis. What is more, the Government are committed to funding groundbreaking research through nearly £790 million of new investment, again through the National Institute for Health and Care Research’s biomedical research centres. There are 20 centres, all of which research rare conditions and bring together academics and clinicians. Their role is to translate scientific breakthroughs into potential new treatments, diagnostics and technologies.

[Liz Twist]

I thank the Minister for his comments about the UK rare disease funding. It is very welcome, and I am particularly pleased to see that some of those centres are in Newcastle, in the north-east. That is really important, as I will discuss in my Adjournment debate later. Will he talk specifically about research into this condition, which is what the families will want to hear?

[Will Quince]

The hon. Lady pre-empts my next sentence, but let me give way to my right hon. Friend before I continue.

[Sir Mike Penning]

I will await the next sentence with interest. I have been to Oxford and met many of the scientists who are carrying out the research, but bidding for this sort of funding is enormously complicated. It takes forever and a day to fill in the bids—a lot of time, and time is money for a scientific facility—and will not help with FOP because it does not fit into the categories that the Minister refers to. It is so rare that we have to bid for funding across Europe or with the Americans. Is there any way that we could simplify the process, so that researchers could know earlier whether they have a chance of getting the funding or if they are wasting their time?

--- Later in debate ---

[Will Quince]

I thank my right hon. Friend for his question. Given the complexity of the issue he raises, it might be helpful if I respond in greater depth at our meeting, where I will invite Professor Lucy Chappell, who heads up the NIHR, to explain that process. She is always looking at how we can improve routes to research and make it as easy as possible for researchers to come forward with fundable bids that the NIHR is then in a position to fund.

The hon. Member for Blaydon pre-empted my next sentence, which was going to be on FOP specifically. I am pleased to say that the Government are funding dedicated research into FOP. Since 2010, the Medical Research Council has funded three projects relevant to FOP. I will ensure that right hon. and hon. Members get further detail about exactly what the Government have funded.

In the last five years, the NIHR has supported seven studies specifically into FOP. As my right hon. Friend the Member for Hemel Hempstead said, the biomedical research centre in Oxford has supported the STOPFOP clinical trial that is exploring the safety and efficacy of using the new drug Saracatinib, to which the hon. Member for Strangford referred, to treat sufferers of FOP. Both the National Institute for Health and Care Research and the Medical Research Council welcome funding applications into any aspect of human health.

My right hon. Friend the Member for Hemel Hempstead rightly made a point about the challenges of designing trials for rare diseases. The efficacy and mechanism evaluation programme that is funded by the NIHR and the MRC may suit researchers who are interested in FOP, as it looks to attract studies with novel methodology designs that can deliver results efficiently and quickly.

My right hon. Friend and others rightly referred to Horizon Europe. I know that, ideally, he wanted the Minister of State for Science, Research and Innovation to respond to this debate, but I reiterate that I work incredibly closely with that Minister because I have responsibility for all research funded by the National Institute for Health and Care Research and through the Department. Moreover, the NHS is a vehicle for so much of the research that happens.

As my right hon. Friend pointed out, Horizon Europe is a valuable platform for researchers from across the continent to come together to tackle shared challenges, especially in areas of this nature where the number of patients affected, or certainly diagnosed, is very small. It has been an important source of funding for research into rare diseases and conditions, including the STOPFOP study. As my right hon. Friend alluded to, we have been negotiating the UK’s continued involvement in the programme. I am delighted to say that, such is the timeliness of this debate, the Prime Minister this morning announced the successful conclusion of those negotiations with a bespoke deal in our national interest, as the Secretary of State for Science, Innovation and Technology set out in a statement to the Commons earlier today.

Let me respond to my right hon. Friend’s specific point about how quickly research can be stood up. As of today, UK researchers can apply for grants and bid to take part in projects under the Horizon Europe programme, and they can do so with certainty that the UK will be participating as a fully associated member for the remaining life of the programme to 2027.

[Sir Mike Penning]

This is a learning process for all of us, and it is not just about FOP. As I understand it, Horizon asks for bids on certain aspects. A bidder can only bid if they fit into those criteria and then they have to wait for up to two years to run the project. The FOP project had to stop because of covid. To start all over again would be very difficult. I wonder whether the Minister will join me in that meeting to see whether we can slot into the process, rather than start all over again.

[Will Quince]

I thank my right hon. Friend for his intervention. Again, he has pre-empted what I was about to say next. I listened intently to his question to the Secretary of State during the statement earlier today, where he secured a meeting with the Minister of State for Science, Research and Innovation. Of course, I would be very happy to join him at that meeting. I share with him the view that if there is an opportunity for that research to be continued, we certainly do not want to see it start from scratch. At the moment, my understanding is that that research relates only to adults and my right hon. Friend, understandably and rightly, would like it to be extended to children.

Let me turn to the steps that the Government are taking to tackle rare diseases and conditions more broadly. In 2021, we published the UK rare diseases framework, which embodies our commitment to building a brighter future for people living with rare diseases and conditions. The framework identified four key priority areas: helping patients get a faster diagnosis; increasing awareness of rare diseases among healthcare professionals, which is something that my right hon. Friend is particularly passionate about; delivering more co-ordinated care; and, alongside that, improving access to specialist care, treatment and drugs. Since then, as was alluded to by the hon. Member for Inverclyde (Ronnie Cowan), all four nations of the United Kingdom have published rare disease action plans to suit their healthcare systems, and we very much work—and have to work—in collaboration on that.

In England, we published our second rare disease action plan on 28 February this year, which also marks International Rare Diseases Day. In that, we set out 13 new actions to drive improvements across the health system and we are committed to publishing action plans throughout the lifetime of the framework.

I touched on awareness of FOP and rare conditions and diseases more broadly, because my right hon. Friend rightly highlighted how a lack of awareness of FOP in the medical community can lead to under-diagnosis, missed diagnosis and, sadly, unnecessary examinations, which can exacerbate symptoms. That is why improving healthcare professionals’ awareness of rare diseases and conditions is central to the rare diseases framework. I am pleased to inform the House that we are making good progress in that regard. NHS England has developed GeNotes, which is an online resource that puts information about genetic diseases and conditions at the fingertips of healthcare professionals. I am pleased to tell the House that resources dedicated to FOP will be added to GeNotes. That will help more clinicians to diagnose this awful condition, and sufferers will receive the treatment that they need more quickly.

My right hon. Friend has raised newborn screening with me privately. The hon. Member for Bristol South (Karin Smyth) also raised newborn screening for FOP. They are both right that antenatal and newborn screening can be a vital tool in the early diagnosis of rare diseases. National screening programmes are implemented on the advice of the United Kingdom National Screening Committee, which makes independent evidence-based recommendations to Ministers in all four nations. While I understand that there are currently no plans for a national newborn screening programme specifically for FOP, I highlight that proposals for new conditions can be submitted to the National Screening Committee’s annual call for new topics.

[Liz Twist]

On newborn screening, is the Minister aware that there is concern among the rare disease community about the limits of our newborn screening process? This is something that we are speaking about at the moment. We think that there are options for it to be expanded in a positive way.

[Will Quince]

I have heard that from other charities, groups and organisations representing those with rare diseases and conditions. It is right that we respect the independence of the National Screening Committee, which puts forward to Ministers evidence-based decisions, but it is nevertheless important that we listen to organisations that are making valid cases. I am also concerned about the length of time that the National Screening Committee can take to make decisions, review evidence and present recommendations to Ministers. In our meeting, I will happily discuss that issue in greater depth and at greater length.

Let me once again thank my right hon. Friend the Member for Hemel Hempstead for securing today’s hugely important debate, and in particular for securing it on the Floor of the House. I express my heartfelt support for the entire FOP community: those living with the disease, their carers, their families, the clinicians and the charity FOP Friends, which works tirelessly to improve the lives of people affected by this devastating condition. I reiterate my thanks to all Members for shining a light on the condition this afternoon. I very much look forward to our meeting and to discussing this further. I genuinely believe that together we will continue to strive for progress and awareness, and support those living with FOP.