Fibrodysplasia Ossificans Progressiva

Andrew Selous Excerpts

Monday 6th December 2021 Westminster Hall

[Andrew Selous (South West Bedfordshire) (Con)]

It is always a pleasure to serve under your chairmanship, Sir Roger. I, too, pay tribute to my hon. Friend the Member for Carshalton and Wallington (Elliot Colburn), my right hon. Friend the Member for Hemel Hempstead (Sir Mike Penning) and all colleagues who have spoken, because party politics comes nowhere near today’s debate. We are all here with one purpose, which is to raise awareness about fibrodysplasia ossificans progressiva, which I will refer to as FOP.

I am not aware that any of my constituents have the condition. However, 584 of my constituents signed the petition, and I took that as a direct instruction from my employers—that is what they are—to be here today. I suspect that it had something to do with my right hon. Friend the Member for Hemel Hempstead, one of my constituency neighbours, being so active on the issue.

Like my colleagues, I am very proud to live in a country in which 111,000 people signed a petition relating to 80 people—children, in the main—that we are aware of, although I absolutely take the point that there might be quite a few more cases that have not been correctly diagnosed. That is humbling. It is worth pausing on that for a moment. We are a nation of 67 million people. One might think that something that has affected only 80 people does not really matter, but it matters hugely. All those individuals matter as individuals. That is what we are talking about today.

Fibrodysplasia ossificans progressiva is a variable and progressive illness. It can lock a person’s jaw. It can make eating, talking and dental care extremely difficult. It can lead to breathing difficulties. I have looked at the photographs that my right hon. Friend the Member for Hemel Hempstead brought to the debate—for those who want to google them, they tell a powerful story.

I was interested to read about the palovarotene trial. I understand that there were 107 participants, which might not sound like a particularly large number. However, as we have said, FOP is a very rare disease, so it is significant. I understand that 62% of the people treated with palovarotene saw a reduction in new heterotopic ossification volume, which seems encouraging. I am not a clinician, so I do not know if that result is high enough to put the drug into widespread use. It certainly seems encouraging to me. I too pay tribute to the researchers at the University of Oxford. As we all know, they have done amazing work on vaccines for the pandemic this year. It is incredible that they are researching FOP as well.

I am pleased that our Government have a rare diseases framework, which was published this January. I read through it to prepare for this debate, and I want to say to the Minister and her Department that I think the framework’s aims are absolutely right. We have already spoken about the four priorities: helping patients get the right diagnosis faster; proper awareness of rare diseases among healthcare professionals; better co-ordination of care; and improving access to specialist care, treatment and drugs. Those all seem absolutely right.

There are five underpinning themes that go along with the framework, the first of which is patient voice. The second is national and international collaboration; we have already heard about the research at the Universities of Oxford and Pennsylvania, and I am sure that researchers from both universities talk to each other and follow each other’s work. There is digital, data and technology, which is so important for that knowledge flow to take place and for people to be aware of the latest research. There is wider policy alignment in how we look after people with FOP. Finally, there is the research that we have been talking about.

I am pleased that the National Institute of Health Research has funded eight studies in this area. However, I have heard that FOP Friends is also funding a great proportion of this research. I have a suggestion for the Minister. I know she will not be able to respond to it now, but I ask her to take it back to the Department and discuss it with the Secretary of State and officials. I understand from the research that I have done for the debate that FOP research would help not only its victims, but people with military and blast injuries, joint replacements, severe burns, sporting injuries, osteoporosis, heart disease, atherosclerosis and chronic anaemia. If we took the smallest proportion—maybe even 0.1%—of the funding for all research into other conditions and earmarked it for FOP, we would provide a significant additional pot of money for FOP research without severely affecting the research into those other conditions. That would be a legitimate transfer of funding, given the benefits that FOP research would have for those other medical conditions. I mention that for the Minister’s consideration. I do not know whether that is feasible, but it could be a short-term way of getting more FOP research when budgets are tight.

The point about medical schools is really important. I have a lot of sympathy for medical students, who have an awful lot to learn in their five or six years at medical school. The seriousness of FOP and the amount of misdiagnosis—we are hearing about amputations and cancer treatment, which, tragically, make FOP worse—show the importance of medical students and doctors of the future knowing about FOP, so that we can get those affected on to the right treatment pathway as soon as possible.

Let us look at whether a little funding from research into related areas could go towards FOP, and ensure that FOP is on the radar of medical schools so that the UK has more than three expert clinicians in the field. We will need to significantly increase that number if we are to do the right thing by the people affected.

--- Later in debate ---

[Maria Caulfield]

Absolutely. I would be very happy to do that. Part of this will probably be the co-ordination of what funding, help and support there is for researchers, and then bringing the researchers together.

I reassure those who signed the petition that the NIHR does not ringfence funds for research. The fund is open to everyone, whether they have one of the most common diseases in the country or one of the rarest. The £1 billion research fund is available to all, and funding applications are available for any aspect of human health. When applications come forward, they are subject to peer review, so research colleagues look at it and judge it, with awards being made on the basis of clinical need—clearly, today we have heard of a clinical need that exists—the value to healthcare services, value for money and scientific quality, so there is no barrier to people applying for the funding.

Since 2010, the Medical Research Council has contributed funding to three projects underpinning relevance to FOP and underlying conditions as well—a total of £6.6 million. Outside those studies, UKRI and NIHR have also looked at supporting musculoskeletal health, which, although not directly FOP-specific, will have relevance to that condition.

[Andrew Selous]

I just want to take the Minister back to a point she made a moment ago about the trials for new drugs being limited to a very small number of people because FOP is a rare disease. I wonder what the solution to that is. Do we try to get people with FOP all the way around the world to participate in a trial? I am not sure how many people would be needed for a trial for it to be validated by the Minister’s Department. There were 107 in the trial that I mentioned, which I presume is too small. I wonder how we overcome that when in each individual country there are only a very small number of people to do the trials on.

[Maria Caulfield]

Absolutely. Just to be clear, it is not the Government who would validate the trials; it would be the scientific community. If it is drug-related, the Medicines and Healthcare products Regulatory Agency would go on to change licences if it found a treatment that was applicable to FOP. In many conditions with such low numbers, often there are global studies, and the funding would not be restricted to a UK-based study. If it was part of a global study, I am sure that that would be acceptable. That is why it would be helpful to meet so that the support and mentorship available to researchers who are thinking of applying for funding could bottom out some of those issues.

I want to reassure colleagues who raised concerns that rare diseases are being pushed up the agenda. The rare diseases framework that was published in January is the first of its kind, and should reassure parents and children with FOP that this is an absolute priority. For too long, rare diseases, because numbers are low, have not had the significance, priority and attention that more common diseases with lots of campaigners and patients have had. The framework will push this to the top of the agenda.

The second reassurance I can give is that funding is available; there is £1 billion per year for clinical research across the board. Just because it is a rare disease does not exclude FOP from these funds. From a practical point of view, it does make research harder, as my hon. Friend the Member for South West Bedfordshire (Andrew Selous) highlighted. However, this does not mean that FOP researchers cannot apply for these funds; there are other criteria that are applied to low-volume scenarios.

Thirdly, I want to reassure Members that clinical research is happening. There are one or two studies that have taken off in this area; often that is the catalyst that needs to happen. I am hearing from colleagues across the House, who have constituents who are affected, that there is a desire to do more research. Very often, this desire is what is needed more than anything to find the researchers who want to do the research and have research questions—whether those are about diagnosis, treatment, or, ultimately, a cure. The funding is there to help support that, and there is practical help and support to bring those studies to fruition. Let me reassure colleagues that, as the Minister, I believe that research is the answer to many of the questions that have been asked today. I am very aware of how distressing this condition is, and the impact that it has on both the quantity and quality of a young person’s life. The Government are committed to ensuring that all rare diseases get better access to the resources that are there. With particular regard to FOP, I am sure that we can work with colleagues across the House to deliver answers to some of the questions they have asked today.