International Thalassaemia Day

Bambos Charalambous Excerpts

Wednesday 27th April 2022 Westminster Hall

[Stewart Hosie (in the Chair)]

I will call Bambos Charalambous to move the motion and then the Minister to respond. There will not be an opportunity for the Member in charge to wind up, as is the convention in 30-minute debates.

[Bambos Charalambous (Enfield, Southgate) (Lab)]

I beg to move,

That this House has considered International Thalassaemia Day 2022.

It is a pleasure to serve under your chairmanship, Mr Hosie.

International Thalassaemia Day is on 8 May, and this year’s message is “Be Aware. Share. Care.” The first part of the message is about raising awareness, so what is thalassaemia? Thalassaemias are inherited blood disorders that lower, alter or stop the production of haemoglobin in the blood. That leads to anaemia, which might be severe or life-threatening if not managed appropriately. There are several types of thalassaemia, depending on the severity of the mutation inherited. The most severe type is beta thalassaemia major, or transfusion-dependent thalassaemia, followed by beta thalassaemia intermedia and haemoglobin H disease.

Those living with transfusion-dependent thalassaemia receive blood transfusions every three weeks for life and daily iron chelation therapy. All patients develop secondary conditions and complications due to thalassaemia and iron overload. Sadly, they develop conditions such as diabetes, heart failure, osteoporosis and liver failure. Patients therefore have to spend a lot of time in hospital, whether it is for blood transfusions for thalassemia, or for the treatment and monitoring of secondary conditions.

Thalassaemia is not transmitted by transfusion, infection, environmental conditions or other factors, but is recessively inherited. It is more prevalent in individuals with Caribbean, South American, African, Mediterranean, south Asian, south-east Asian and middle eastern ancestry. Due to migration over centuries, it is found throughout the world, and it is estimated that there are 100 million people worldwide with a thalassaemia trait who are asymptomatic.

The prevalence of thalassaemia varies across different regions in the UK. Data published in 2020 by the National Haemoglobinopathy Registry—the NHR—indicates that there were more than 900 people living with beta thalassaemia major in the UK, 238 living with beta thalassaemia intermedia, 280 with beta thalassaemia/Hb E disease, and 300 with haemoglobin H disease.

The majority of patients with thalassaemia in the UK come from a British Pakistani or British Asian community. Each year, around 20 to 30 couples in the UK are identified as being at high risk of having a baby with a form of thalassaemia. My constituency of Enfield, Southgate has the highest number of people with thalassaemia in the UK and is home to the UK Thalassaemia Society, which campaigns for greater awareness and better health outcomes for people with thalassaemia. It has also given me advice and shared its findings for this debate.

The second part of the message for International Thalassaemia Day is about sharing—sharing essential information and knowledge to support the best health and social care outcomes for people with thalassaemia. The Department of Health and Social Care published its UK rare diseases framework last year, which acknowledged a number of challenges and set out the Government’s four priorities, which include increasing awareness of rare diseases among healthcare professionals, better co-ordination of care, and improving access to specialist care treatments and drugs, all of which I and the thalassaemia community very much support.

I want to focus on the last point about improving access to specialist care treatments and drugs. Thalassaemia is a rare disease and there are very few treatments for the condition. Some gene therapies have been developed, but have often not been able to progress beyond the National Institute for Health and Care Excellence’s criteria because either the formula for quality-adjusted life years is loaded against people with rare diseases or there is a smaller sample of people upon whom the gene therapy trials have been conducted. That is primarily because people with rare diseases are often few in number, and that limits who the therapy can be trialled on.

I note that NICE has done its methods and processes review, but I ask the Minister to ensure that the highly specialised technology and standard technology appraisal pathways are both fit for purpose for people with rare diseases, and that the uncertainty of cost effectiveness due to small population sizes is a serious consideration for NICE in assessing the appraisal of new gene-therapy technologies. Gene therapy and other technologies for people with rare diseases are literally a matter of life and death, and much more work needs to be done by the Government to ensure that people with rare diseases are not disadvantaged by the bureaucratic processes that fail to take into account the unique nature and impact of rare diseases on those who have to live with them.

The final part of the message is about care. This is about the experience of people with thalassaemia when receiving healthcare. I have met a number of people with thalassaemia who have shared their experiences with me. They require regular blood transfusions, and they told me about their pain and suffering following transfusions and how debilitating that can be. I have also read testimonies of people with thalassaemia who have experienced differing levels of treatment by health professionals. Because the condition tends to be extremely specific to a particular ethnic group in the UK and there are very small numbers of patients, the UKTS has found a huge disparity in services throughout the country with regard to the accessibility of thalassaemia care.

[Mr Pat McFadden (Wolverhampton South East) (Lab)]

I am grateful to my hon. Friend for the speech he is making. On care, I do not know if he has had a chance to read the report from the sickle cell and thalassaemia all-party parliamentary group entitled, “No One’s Listening”. Sickle cell is not exactly the same thing as thalassaemia. There are differences, but there are similarities too in people’s experiences. Does he share my hope that that report will serve as a turning point to win a resolve for better treatment and greater understanding of these conditions, all the way from the Department of Health and Social Care through to the decision makers in the NHS?

--- Later in debate ---

[Bambos Charalambous]

My right hon. Friend makes an excellent point. I am aware of the report and have read it. He is absolutely right that much more needs to be done to ensure that people with thalassaemia, sickle cell and other rare diseases get the treatment they need. It is also about better training for health professionals to identify the symptoms of thalassaemia, sickle cell and other such conditions, so that people with thalassaemia are not disadvantaged when they come into contact with health professionals for the first time.

According to the UK Thalassaemia Society, the experience of their members is that the UK’s thalassaemia services are under-resourced, underdeveloped and understaffed, even compared with the treatment received by those with other blood disorders. Part of the work that the UK Thalassaemia Society has been involved in over several years is to review sickle cell and thalassaemia units throughout the country and all aspects that make up the patient pathway, from emergency care to in-patient and out-patient services. There appears to be evidence of health inequalities between the treatment of patients with thalassaemia and patients with other conditions.

As we know, thalassaemia particularly impacts specific ethnic groups, such as the British Asian population in the UK, and patients have reported to the UK Thalassaemia Society that they often feel that their ethnicity is linked to below standard treatment, and they have on occasion reported distressing instances of overt racism in connection with their treatment. That has sometimes severely impacted patients’ mental health, with patients describing feeling defeated and, in the worst instances, not wanting to live any more.

The UKTS has found that patients and families in some cases are afraid to talk to the managers and nurses involved about the instances described as they are fearful that they will receive worse treatment and be stereotyped further. As a result, they have chosen to suffer in silence. That is obviously extremely concerning, and I will happily share more information about that with the Minister outside the debate. I am sure that, like me, the Minister will find it entirely unacceptable that the ethnicity of patients in any way affects treatment or, in the worst cases, leads to deeply offensive racism.

It is worth noting that thalassaemia affects many ethnicities, though predominantly those of Asian heritage, and the average life span is considerably lower in the Asian population than in the Mediterranean population. That may be for a variety of reasons; however, there is certainly worry among members of the UK Thalassaemia Society that the racial disparity they encounter may have an impact on their health outcomes. Again, I would be grateful if the Minister took that concern on board and raised it with health professionals.

I hope this debate will help raise awareness of thalassaemia and the particular difficulties that patients face. I hope the Minister will tell us how she will work to improve equal access to care and equal health outcomes for this community. How does she believe we can tackle the explicit and implicit discriminatory attitudes that still exist in healthcare settings? I thank the UK Thalassaemia Society, Genetic Alliance UK and the Royal College of Pathologists for providing me with information for the debate. I hope the Minister will take my points into consideration and re-evaluate the Government’s position on thalassaemia and rare diseases, to see what more can be done to improve health outcomes for people with thalassaemia and to raise awareness of the condition among health professionals and the wider public.