How patients’ views helped inform the approval of a new option for conditioning treatment before a stem cell transplant

July 6, 2020
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After the news that the National Institute for Health and Care Excellence (NICE) have recommended treosulfan (Trecondi) as an option for conditioning treatment before a stem cell transplant in England, Charlotte from our Policy and Public Affairs team shares what this means for patients and how Anthony Nolan promoted the views and experiences of patients to help make this happen.

Conditioning treatment

Treosulfan (Trecondi) has been approved this week by NICE, for use in the NHS in England, Wales (1) and Northern Ireland as an option for conditioning treatment before stem cell transplant for people with blood cancer who would benefit from a reduced intensity regimen. This type of conditioning therapy is an alternative for patients who cannot tolerate full intensity conditioning treatment, such as older patients or those with multiple health conditions.

Before a patient undergoes a stem cell transplant, their immune system will be treated and prepared to make way for new cells, and this is called conditioning. There is no universally agreed conditioning regimen for patients that require a stem cell transplant. That is why Anthony Nolan is funding clinical trials through the IMPACT Partnership to determine which conditioning regimes are the most appropriate for individual patient groups.

There are different intensities of conditioning which are suitable for different people. The best regimen for patients will be determined by their doctor: considering their diagnosis and remission status, alongside their age, if they have any other conditions and their donor.

When making room for the donor stem cells, conditioning treatments can cause side effects. Patients told us that the introduction of any conditioning regimen with reduced toxicity and side-effects would be welcomed as a benefit for future patients.

Understanding patient experience

NICE asked Anthony Nolan, as the UK’s leading stem cell transplant charity, to make a submission and represent the views and experiences of stem cell transplant patients during the appraisal of this new conditioning drug. As the NHS has limited resources, they can only fund drugs that are proven to be clinically-effective: according to research and trials, and cost-effective: based on whether the benefit to patients is big enough.

To ensure that we could represent patients effectively, we launched a survey on patients’ experiences of conditioning treatment and its side effects which was shared with our Patients and Families Network. There were 20 responses, primarily from patients located in England, however there was also patient representation from Wales and Scotland.

We were also asked to nominate a patient expert to help provide verbal evidence of their own experience to the Committee, to help clarify issues and respond to questions from the patient perspective. On top of this, we discussed the use of conditioning regimens with medical professionals to support the submission.

Patient experience

Patients told us that they understood the reason why they were undergoing conditioning treatment prior to transplant, and as such accepted it as a necessary part of their transplant journey.

However, despite the understanding of the need for this treatment, actual experience of conditioning was a shock for many patients.

‘I found this part of the treatment pretty tough. I knew the reason for it, so I prepared mentally but the physical shock is quite hard.’

The conditioning regimen, for many patients, was more challenging than their previous experiences of chemotherapy. A response highlighted that the ‘very strong nausea and vomiting’ they experienced from their reduced intensity conditioning regimen was significantly worse than previous experience of chemotherapy.

In over half of the responses, at least three different side effects were reported. The most commonly reported were; extreme fatigue, nausea and vomiting, diarrhoea, loss of appetite, constipation, mucositis, ulcers and hair loss.

Patients told us that these side effects impacted their quality of life, their independence, including their ability to look after themselves, and their emotional health and wellbeing.

‘It was a worrying time and a struggle emotionally’

Patients also reported that the experience of these side effects were challenging for their family and carers too.

‘My family found it difficult to cope and support me during this period. It caused a lot of stress because they didn't understand or know when things would improve.’

However, importantly, the responses showed that patients understood the role of conditioning as necessary to their transplant and that while the side effects experienced were not insignificant, they did not regret undergoing their transplant.

Using this evidence

We used these insights in the written submission we sent to NICE which provided background knowledge of stem cell transplant as a treatment, the role of conditioning and the way it is experienced by patients.

Our Head of Policy and Public Affairs attended the Committee meeting to talk through our survey and the findings, alongside Alan, the patient representative, who sat on the Committee panel with us to represent his own personal experience of conditioning treatment before transplant.

Our patient representative

Lieutenant Colonel Alan Tindale MBE was diagnosed with Acute Lymphoblastic Leukaemia (ALL) in 2013 and received an allogeneic stem cell transplant in 2014. Alan told the committee about the ups and downs he experienced during his treatment and recovery, including the psychological impact of this.

I wasn’t prepared for how difficult the process would be and the impact that has on your life and the lives of those around you.

Alan shared how his conditioning treatment made him ‘feel poorly instantly’ and how, although this was a necessary step to prepare for the transplant, feeling so unwell left him feeling ‘like he had taken a huge backward step’ due to the side effects. He told the Committee how important it is to patients to have the right conditioning treatment for them and that any reduction in associated side effects would be very welcomed by patients.

Alan reflected positively on his experience as a patient expert saying:

I appreciated this opportunity to put a patient’s perspective across and it felt as if I was part of a significant development by doing so.

The outcome

As a result of the appraisal, Treosulfan (Trecondi) has been recommended by NICE as an option for conditioning treatment before a stem cell transplant for people with blood cancer who would benefit from a reduced intensity regimen. This enables more choice for clinicians choosing the right conditioning regimen for patients.

The recommendation takes all the relevant current evidence into account. We would like to see this decision reviewed in the future to ensure that any new evidence, which may extend access to new patient groups, is considered by NICE.

NICE said our participation with Alan, and written submissions were very useful and an important resource for them to understand the patient experience of transplant, as well as the context that treosulfan, as a conditioning treatment, should be seen in.

This appraisal covered blood cancer, however, it is also available for patients with blood disorders following a decision by NICE not to go through the full appraisal process.

Looking forward

NICE are responsible for ensuring a medicine is both clinically and cost effective for the NHS, and they ask for a range of evidence from the drug company to prove this. However, the insights we provided to NICE, about patient experience of a drug, and the effect it has on their quality of life, are as important. This evidence shouldn’t be an additional ‘nice to have’, it should be an integral part of appraising a drug, in the same way its cost is.

We would encourage NICE, as part of the ongoing review of their methods and processes, to ensure that they adapt these to ensure that gathering detailed patient experience information is a priority. This will allow a more complete discussion on the benefits of a medicine, including how it can impact the quality of a patient’s life.

While we aren’t participating in any appraisals at the moment keep an eye out for opportunities to work with us to represent the patient perspective in the future.

1. The Welsh ministers have issued directions to the NHS in Wales on implementing NICE technology appraisal guidance. When a NICE technology appraisal recommends the use of a drug or treatment, or other technology, the NHS in Wales must usually provide funding and resources for it within two months of the first publication of the final appraisal document. The Northern Irish authorities will, in the main, follow NICE decisions, however, Scotland have their own appraisal process carried out by the Scottish Medicine Consortium.