Scotland’s Cancer Strategy and Drug Approval Updates

Last updated: 19th June 2023

The Cancer Strategy for Scotland 2023-33 sets out the Scottish Government’s aims for ensuring patients receive the best treatment and support throughout their cancer journey, and improving outcomes and survival for cancer patients.

This new cancer strategy represents a chance to improve on previous cancer plans and ensure that the issues facing blood cancer patients are adequately considered.

“We are pleased to see that the Scottish Government has published their ten-year Cancer Strategy. We are encouraged that the strategy recognises the specific difficulty of tracking the progress and improvements in diagnosing blood cancers earlier, with the need to use different measurements like emergency presentations as a measurement for cancers that cannot be staged. However, the strategy fails to recognise other areas where the experience of leukaemia patients differs from those with solid tumours, particularly when it comes to addressing the unique needs of patients on Watch and Wait” – Zack Pemberton-Whiteley, Chief Executive at Leukaemia Care.

How was Leukaemia Care involved in the drafting of this strategy?

The Scottish Government consulted with Leukaemia Care and other members of the Scottish Cancer Coalition, which brings together a number of different cancer charities in Scotland. We reviewed draft versions of the strategy and raised any concerns we had. 

We highlighted some of the key challenges we have been campaigning in our Left to #WatchWaitWorry and #SpotLeukaemia campaigns. These include:

• Ensuring more patients are diagnosed earlier, as earlier diagnosis is linked to better survival for patients.
• Better recognition of the need to support people who are on active monitoring or Watch and Wait. Current planning for cancer care focuses heavily on those immediately pre treatment, during treatment and immediately after, leaving many leukaemia patients without support.
• Better access to clinical nurse specialists (CNSs) for all leukaemia patients, as leukaemia patients were less likely to report access to this support than those with solid tumour cancers.

To create our response, we used evidence from our Living with Leukaemia survey, our patient advisory panels and our campaigns, all of which help give us a picture of what it’s like to live with a diagnosis of a leukaemia, an MPN or MDS.

Has the strategy acknowledged and addressed any blood cancer issues?

We are pleased to see that there have been improvements in the latest Scottish cancer strategy, particularly when it comes to diagnosing blood cancers earlier.

Leukaemia, MPNs, and MDS cannot be staged like tumorous cancers. The non-specific nature of blood cancer symptoms and the relative rarity of leukaemia can make it difficult for both patients and healthcare professionals to recognise the potential cause as leukaemia. This can potentially lead to a patient presenting as an emergency instead – e.g. in Accident and Emergency – as their symptoms become more severe. Being diagnosed via A&E has been linked to reduced survival.

The strategy recognises that not all cancers can be conventionally staged, including leukaemia. This document acknowledges that emergency presentation data is needed in order to track progress and improvements in the earlier diagnosis of leukaemia. We are pleased to see this particular concern that we frequently highlight in our #SpotLeukaemia campaign has been acknowledged. 

We are also encouraged to see a commitment in the Scottish Cancer Strategy to routinely publish emergency presentation data through Public Health Scotland. This data will be vital in reducing the early diagnosis inequality gap between stageable and non-stageable cancers, and improving survival rates.

We also called on the Scottish Government to raise awareness of the non-specific symptoms of leukaemia, and to encourage people to get their symptoms checked. We are pleased to see this commitment included in Scotland’s cancer strategy, with a focus in also supporting primary care professionals in referring patients presenting with non-specific symptoms.

How can the strategy be improved?

There is still work to be done, particular in relation to our Left to #WatchWaitWorry campaign and ensuring adequate support for chronic lymphocytic (CLL) patients on Watch and Wait.

The Cancer Strategy for Scotland 2023-33 has failed to mention or make any reference to ‘active monitoring’ and ‘Watch and Wait’, or the important role clinical nurse specialists (CNSs) play in supporting CLL patients. This is particularly concerning as there are approximately 13,000 people living in the UK with CLL on watch and wait. Additionally, Watch and Wait is not just an issue for CLL patients, but also for other blood cancer types, as well as prostate cancer. 

Overall, workforce and the pressure that a reducing number of staff puts on the NHS in Scotland remains a concern for us. The Scottish Government must take urgent action on this, to be able to achieve what they have set out in this cancer strategy in growing the number of NHS workforce training places and ensuring that all professionals are doing the work they are best placed to do.  

The Scottish Government has committed to completing a workforce review  of key professions in Scotland’s cancer services by 2026. We will continue to advocate for the Scottish Government to train more clinical nurse specialists, who provide invaluable support for leukaemia patients.

How will Leukaemia Care continue to encourage the Scottish Government to address any shortcomings in the strategy?

The cancer strategy is broken up into 3-year stages, known as Action Plans. Each stage focuses on specific cancer types and issues. The first 2023-26 Action Plan focuses on cancers that are the largest burden and have poorer survival, such as lung, liver, and pancreatic cancer.

Leukaemia Care will continue to work with the Scottish Government to ensure that the issues facing all blood cancer patients to be addressed in future Action Plans.

Are there cancer plans for England, Wales, and Northern Ireland?

The UK Government is currently drafting a Major Conditions Strategy for England. We will be consulting with the UK Government in the drafting stages of this strategy to ensure that England’s strategy reflects the improvements seen in other UK nations’ cancer strategies when it comes to considering blood cancer issues.

Wales recently published their Cancer Improvement plan 2023-26 which can be read here. We are pleased that the Welsh cancer plan recognises the need for all cancer patients to have access to a key worker, so we will be campaigning for all leukaemia patients to have access to a CNS.

Northern Ireland have also recently published their Cancer Strategy for Northern Ireland 2022-32 which can be read here. This is currently the only strategy amongst the four UK nations to mention the needs of CLL patients on active monitoring and the important role CNSs have in providing care to this group of patients. We welcome this and hope other UK nations follow suit in acknowledging the needs of patients on active monitoring. 

Last updated: 10th July 2023

The SMC has decided not to approve Besremi, also known as ropeginterferon alfa-2b, for use on the NHS in Scotland. This treatment is a monotherapy, intended to treat adult patients with polycythaemia vera (PV), without symptoms of an enlarged spleen.

Who are the SMC and what do they do?

The SMC considers and advises on all new medicines for NHS Scotland, on evidence of clinical (how well the drug works in patients) and cost-effectiveness (whether it is better value for money than alternatives). The SMC will make its decision based on the evidence submitted by the pharmaceutical company making the drug, healthcare professionals, and also request input from patient groups such as Leukaemia Care. 

The company manufacturing the medicine provides evidence of why the medicine is effective both in how well it works and the cost to NHS Scotland compared to current treatments. Patient groups are involved in representing the views of patients by talking about any potential impact this new medicine will have on patients and carers. The healthcare professionals advise on any potential benefits of this new medicine compared to the current treatment landscape for this set of patients. Collectively, this helps the SMC make its final decision.

Why did the SMC make this decision?

The main clinical trial evidence came from two clinical studies, PROUD-PV and CONTINUATION-PV, which compared ropeginterferon alfa-2b against the existing treatment hydroxycarbamide (HU), the first treatment option given in Scotland-based clinical practice as it is relatively low cost and widely available. 

The SMC decided to reject ropeginterferon alfa-2b as they were not satisfied with the evidence from the clinical trials that ropeginterferon alfa-2b would be a more effective alternative to HU, particularly for patients who have not undergone treatment before, and those who only had a partial response to HU.

The SMC were not satisfied with the cost of ropeginterferon alfa-2b when compared to the potential health benefits of the treatment. There was further concern with the high cost of ropeginterferon alfa-2b in comparison to existing treatments such as HU and pegasys, which cost less than ropeginterferon alfa-2b. 

We are disappointed to see this treatment has not been approved. When the SMC asked us for our input, we stated that while there are a number of other treatment options in this setting, having a licensed alternative, such as ropeginterferon alfa-2b, would be a step forward. The licensing process looks at whether a drug is safe and effective in a particular group of patients, in this case PV. There are currently no licensed medicines in this setting for PV patients. 

Last updated: 10th July 2023

Leukaemia Care campaigns for the Scottish Medicines Consortium (SMC) to approve belumosudil (Rezurock) on the NHS in Scotland.

Leukaemia Care today welcomes the news that the Scottish Medicines Consortium (SMC) has approved the use of belumosudil (Rezurock) on the NHS in Scotland. It is available for a period of 3 years initially, whilst more data on how well it works is collected. It will be used for patients aged 12 and over with chronic graft-versus-host disease (cGvHD) who have received at least two prior lines of systemic (whole body) treatment.

Once more data on the treatment has been collected, the use of belumosudil will be reviewed. We will report if access to the treatment changes in the future.

How was Leukaemia Care involved in the decision to approve this treatment?

Leukaemia Care worked alongside the charity Anthony Nolan to gather data, patient experience and put together a written response to the SMC before making their decision. We focused on why belumosudil could be beneficial for patients when compared to the existing treatments currently available in Scotland.

We highlighted that many patients that are due to receive their third therapy for GVHD are likely to be battling multiple, significant side effects already, such as severe inflammation of their eyes and skin.

As such, we argued that a quick and effective third line therapy is needed to reduce the most significant side effects and control the impact and severity of GvHD. As some patients currently continue to be referred onwards to 4th and 5th line therapies, belumosudil holds the potential of resolving or managing a patient’s advanced chronic GvHD without further treatment interventions.

What is Graft versus Host Disease?

Graft versus Host Disease (GvHD) happens when a particular type of white blood cell, called T cells, in the donated stem cells or bone marrow begin to attack the patients’ own body cells. This happens because the donated cells (the graft) see the patients’ own body cells (the host) as foreign and start to attack them.

Chronic GvHD tends to develop more slowly than acute GVHD. Chronic GvHD symptoms can often include skin rashes, mouth sores, dry eyes, liver inflammation, the development of scar tissue in the skin and joints, and damage to the lungs.

Although many patients with chronic GvHD experience mild to moderate symptoms, a small number of patients can develop more severe symptoms.

Why is the approval of this treatment important for patients?

Before the approval of belumosudil, the only other treatment recommended in Scotland for managing chronic GvHD for patients who have received two prior lines of therapy was Extracorporeal Photopheresis (ECP). ECP involves removing blood from the patient, treating the blood with chemicals to remove the white blood cells causing the GvHD, and then returning this blood to the patient.

ECP can take up to 4 hours and can also require patients to attend hospital twice a week every two weeks. This process can last 6 months or longer and with only 2 health centres in Scotland that can provide this treatment, patients may have to travel long distances to receive ECP.

Belumosudil is a new treatment that is likely to improve patients’ experience of treatment and quality of life, due to its convenience and the option to possibly take it at home.

Belumosudil comes in the form of a tablet that patients take once a day. Patients we spoke to expressed a preference for oral ‘at home’ treatments that allowed them to spend more time away from the  hospital. This treatment could be of benefit to patients’ well-being by allowing them to spend more time with family, friends, and continue a normal social life.

This new treatment could also remove the financial burden on patients and their families, who have to take time off work to travel with the patient to hospital appointments if they are receiving ECP.

Clinical trials have suggested that belumosudil is well tolerated in comparison to existing treatments available in Scotland. Patients we spoke to highlighted the extremely unpleasant side effects of many of the currently available treatments and wanted new treatments that have less serious and unpleasant side effects.

Last updated: 10th July 2023

Leukaemia Care campaigns for the Scottish Medicines Consortium (SMC) to approve oral azacitidine on the NHS in Scotland for adults with acute myeloid leukaemia (AML).

Today, Leukaemia Care welcomes the decision by the Scottish Medicines Consortium (SMC) to approve oral azacitidine for use on the NHS in Scotland. This is a treatment for acute myeloid leukaemia (AML) in adults. Oral azacitidine will be used specifically as a maintenance treatment after induction therapy.

What’s the news and how was Leukaemia Care involved? 

Following the approval of oral azacitidine in England in 2022, we are pleased to announce that the Scottish Medicines Consortium (SMC) have also now decided to approve this treatment for use in Scotland. The brand name for oral azacitidine is Onureg and it is produced by Celgene.

Similar to our involvement in the NICE (National Institute for Health and Care Excellence) process, Leukaemia Care also submitted a written response to the SMC representing the views of AML patients. A submission is created by drawing on experiences and data, and advocating for equity of access for Scottish patients.

We are very pleased that patients in Scotland now have equal access to oral azacitidine as those in England, Wales, and Northern Ireland.

What is maintenance treatment?

Oral azacitidine has now been approved for use on the NHS in Scotland as maintenance treatment after induction therapy for adult AML patients. Induction therapy includes treatments such as chemotherapy or radiotherapy and aims to put a patient’s AML into remission. This is done by using high doses of treatment over a short period of time.

Maintenance treatment is a long-term and usually less intensive treatment. It is given after the leukaemia is in remission, to prolong the remission and hopefully prevent a relapse from happening. This is what oral azacitidine will now be given to patients for. A transplant might be an option if a clinician is worried about relapse, but transplants are not suitable for everyone.

This is important as before this there was no maintenance treatment for the vast majority of AML patients in Scotland. A maintenance treatment called midostaurin is only available for a minority of patients whose condition was caused by a rare genetic mutation.

Which patients can access this treatment?

Oral azacitidine is recommended by the SMC as an option for maintenance treatment for adults with AML who:

– Are in complete remission, or complete remission with incomplete blood count recovery, after induction therapy with or without consolidation treatment, AND
– Who cannot have or do not want a haematopoietic stem cell transplant

We argued that those who cannot have a transplant may not be eligible for reasons out of their control and for non-clinical reasons, such as financial circumstances or challenges with getting a transplant donor. Therefore, there needs to be an alternative for people, whatever their reason for not having a transplant.

Why is the approval of this treatment important for patients?

Relapse rates in AML are common, at around 50%. Relapse is a scary and daunting prospect for many as it leads to lower chances of overall survival. It also has a negative impact on patients’ quality of life, e.g., their mental health.

Therefore, a treatment which could prevent relapse after initial remission would be in patients’ best interests. The approval of oral azacitidine fills this gap and provides a much needed treatment option, especially for those who do not want or can’t have a stem cell transplant. These are people who would be left with few other options if they were to relapse, so preventing relapse if possible is preferred.

We are very pleased that patients in Scotland now have equal access to oral azacitidine as those in England, Wales, and Northern Ireland.

What are the other benefits of the treatment to patients?

A patient with personal experience of having AML and a different, subcutaneous injection, version of azacitidine stated that:

“I personally benefited from azacitidine in sub cutaneous form and thought it was important for the committee to hear from a patient’s perspective the numerous advantages of having it available in an oral form.”

The SMC has approved azacitidine in oral form, which means it is taken as a tablet. Survey data has shown us that oral treatments are favoured by many patients for their convenience, as they reduce travel time to and from hospital, reduce financial burden of travel and spending time in hospital and allow patients to spend more time with friends and family. Oral tablets are also often preferred as they are less invasive.