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Focus on access to medicines: Bloodwise’s work on Health Technology Appraisals

The Bloodwise logo. Bloodwise appears in black text against a white background
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04 Apr 2018

​A round-up of the recent submissions our Policy Team have made to Health Technology Appraisals on behalf of blood cancer patients

A variety of tablets

An important part of the work our Policy Team do is to make submissions on behalf of blood cancer patients for Health Technology Appraisals (HTA’s). These are assessments by NICE (National Institute of Clinical Excellence) in England and equivalent bodies in the rest of the UK to decide whether new and existing medicines and treatments should be recommended for use within the NHS.

The recommendations made are based on a review of the clinical evidence (how effective is the drug?) and economic evidence (does it represent value for money?). The pharmaceutical companies who manufacture the drugs submit this evidence and there is also input from independent clinical experts. The role of patients and patient groups in this process is vital because, without this input, the decision makers would have no insight into the real effect of these often life-changing treatments on those living with these conditions.

In the last few months we have contributed to the following HTAs on behalf of blood cancer patients.

NICE consultation on updated technology appraisal process guide

In November we submitted a response to a NICE Consultation on streamlining their approvals process. This is necessary because of the ever increasing rate of new medicines being produced and the need to improve access for patients where such treatments may significantly improve their quality of life – and, in many cases, save their lives.

NICE propose that with new processes in place they hope to have capacity to publish 75 appraisals a year, a significant increase on current capacity. While Bloodwise supports such a proposal in theory, the initial consultation included proposals to reduce patient attendance at committee meetings. We strongly opposed this, together with many other patient groups, and we were pleased to learn in the new year that NICE had agreed to abandon this proposal.

The focus is now on allowing for expert advice to be sought, engagement on the technical aspects, and commercial interactions before committees meet, as a means of reducing the number of occasions where more than one committee meeting is required for the majority of appraisals. In our recent response to phase 2 of the consultation, we supported the drive to increase the number of appraisals and to improve efficiency. We confirmed that while we definitely want the patient voice to be enhanced, there has to be recognition that the process needs to be easily understandable and genuinely responsive for this to happen. In addition to our own response to the consultation, we also collaborated with other members of rare cancers coalition, Cancer 52, to produce a joint response. Read the joint response we wrote with Cancer 52.

Brentuximab vedotin for Hodgkin lymphoma

In early January, we made a submission to NICE in relation to the review of their previous recommendation that brentuximab vedotin should be available to patients within the Cancer Drugs Fund. This would be for treatment of CD30-positive Hodgkin lymphoma in adults who have relapsed after at least two previous therapies and cannot have a stem cell transplant or multi-agent chemotherapy.

With help from our patient ambassadors, who kindly shared their experiences of this condition and their treatment, we strongly argued that this drug is a step-change in the treatment of Hodgkin lymphoma and should continue to be available to patients on the NHS. The committee meeting on this has just taken place so we expect a final decision very soon.

Arsenic trioxide for the treatment of APL

We made a submission to the All Wales Medicines Strategy Group in February for the appraisal of arsenic trioxide for the induction of remission, and consolidation in adult patients with newly diagnosed low-to-intermediate risk acute promyelocytic leukaemia (APL) in combination with all trans retinoic acid (ATRA). This treatment is acknowledged by clinicians and patients to be a significant step forward in the treatment of APL for two reasons: it removes the need for highly toxic chemotherapy: and, above all, because the relapse rate following treatment is so much lower than that following traditional chemo. Therefore, it was very important to us that we made this submission on behalf of patients with APL. We also had the opportunity to make a late submission to the NICE appraisal for the same treatment and indication profile and expect the committee’s decision on this imminently.

Midostaurin for treatment of AML

We made a submission to the Scottish Medicines Consortium in early March for the appraisal of midostaurin for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FLT3 mutation positive in combination with standard chemotherapy drugs daunorubicin and cytarabine. Despite the difficulties in tracking down patients who had experience of this treatment through our ambassadors, we were fortunate to make contact with a patient in the US through Facebook who matched the exact indication and was in remission following this treatment.

CAR-T cell therapies: work in progress

We are now starting to work on submissions to NICE for appraisals of two different versions of an exciting new treatment, CAR T-cell therapy. The two versions (produced by different pharmaceutical companies) being assessed are: Tisagenlecleucel-T, for treating both diffuse large B-cell lymphoma in adults and B-cell lymphoblastic leukaemia in 3-25 year olds; and Axicabtagene ciloleucel, also for diffuse large B-cell lymphoma as well as mediastinal B-cell lymphoma and follicular lymphoma.

The treatment works by using patients’ own white blood cells to fight cancer. The patient’s T cells are extracted through a filtration process and reprogrammed, then reintroduced to the patient’s bloodstream where they seek and destroy cancerous cells. Clinical trials have proved to be very successful so far.

Bloodwise believes that CAR-T cell therapy will make a significant difference to people with blood cancer in the future and so it is essential that we make these submissions and support the therapies being made available on the NHS. The difficulty, as is often the case with these submissions, is finding patients to contribute their accounts of treatment particularly as the clinical trials are so recent and the majority of them have taken place outside the UK.

If you think you can help or have any contacts in the blood cancer community who you believe might have relevant experience of these treatments or even if you have not had the treatment but are able to share your experience of these conditions, please do get in touch with our Policy Officer, Charlotte Cherry on ccherry@bloodwise.org.uk or 020 7504 2229.