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Health and Technology Appraisals – the importance of patient involvement

written by

Leukaemia Care, Charity

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Treatments provided by the NHS in England, are often subject to approval for use by NICE (the National Institute for Health and Care Excellence) and your patient voice and experience is hugely vital within this decision making.

In a process called a health and technology appraisal (HTA), new drugs and treatments are assessed by NICE (England) or another body before they are routinely provided by the NHS. The HTA committee considers both clinical evidence and economic evidence to assess whether the new treatment or technology should be recommended for use.

This involves assessing how clinically effective the new treatment is, how it compares to the best currently available treatment and whether it improves patient’s quality of life in respect to survival, side-effects, length of hospitalisation and other similar factors. This is all incorporated into deciding whether the treatment represents good value for money.

Clinical trial data is vitally important in providing quantitative evidence that the treatment offers greater benefits for patients than the current standard of care. Evidence from patients and their carers who have experience with the treatment prior to the HTA can also help to prove the clinical effectiveness of a new treatment.

We often use statistics from our patient survey to provide evidence on the patient experience of living with leukaemia. We also explain how current treatments impact patient’s quality of life and highlight the advantages and disadvantages of the new treatment being assessed. 

The importance of patient involvement within the HTA process has been very clearly highlighted in a recent HTA in which we have been involved with.

In August 2017, NICE’s initial decision was to not recommend the use of inotuzumab ozogamicin for the treatment of acute lymphoblastic leukaemia (ALL) in patients who had relapsed, or not responded to therapy. Despite the clinical trial data demonstrating clinical effectiveness, the cost of the treatment was deemed to be too high as the drug was licensed for up-to six rounds of use.

However, UK practice is to use inotuzumab ozogamicin to enable patients to have a possibly curative stem cell transplant and, in this setting, a maximum of three rounds of treatment would be used. This would offer lower cost for treatment, than considered by the HTA committee.

In the appeals process, two patients and a carer whom had experience with inotuzumab ozogamicin represented Leukaemia Care to provide evidence that the treatment provides a life-saving option and crucially, in each case, required no more than two rounds of treatment. The evidence provided by these three members was invaluable in ensuring the appeal was successful and the HTA has now returned to the committee for a second assessment.

At Leukaemia Care, we are involved in providing the patient voice to many other HTAs for new leukaemia treatments. In order for us to do this, we need your help to produce the evidence. The patients, carers, friends and relatives who have experience of living with, and being treated for, a leukaemia. Particularly, if you are involved in clinical trials or are accessing treatment through the Cancer Drugs Fund.

If you would like to help, please fill out our short HTA Patients Comments Form, which can be accessed here: