New research supports better integration of patient voice in health technology assessments

Research news // 1st July 2019

Myeloma UK’s strategy is to make sure that the patient voice is used to inform decision making to benefit patients. This includes decisions around the availability of treatments, so evidence for the value of treatments can considered in ways which also matter to patients.

From 2016 to 2018, NICE (the National Institute for Health and Care Excellence) and Myeloma UK collaborated on a piece of research to understand more about the way that organisations such as NICE can use insights from patients in their decision making about new medicines. Myeloma UK funded the project and the Science Policy and Research Team at NICE conducted the study, with input from myeloma patients and family members and other stakeholders.

NICE is an organisation which provides national guidance and advice to improve health and social care in the UK. Its role is to improve outcomes for people using the NHS and other public health and social care services. NICE do this by producing guidelines (based on evidence from research) on treatments and care for conditions such as myeloma. NICE also produces quality standards for those providing and commissioning health and social care services.

The aim of our project was to understand more about the way patient preference data can be collected and used in decisions about new medicines and treatments. ‘Patient preferences’ is a term used to describe the data elicited by methods that capture patients’ preferences for hypothetical or actual treatment attributes.

Patient perspectives are currently captured by expert patients taking part in appraisal meetings, where the costs, benefits, and risks of potential new treatments are discussed. Although patients are listened to as part of these meetings, patient perspectives are captured without systematic or agreed methodologies and so patient input is hard to quantify (compared to other numerical evidence such as clinical trial data).

This study was therefore important as we wanted to know more about the quantitative methods (methodologies) that can be used to elicit patient preference data, so that Myeloma UK and other organisations can conduct studies that will be impactful and meaningful as part of appraisal processes.

The Science Policy and Research Team at NICE undertook a two-year research project to see how these methods work and how NICE might utilise them. This study examining the ways that patient preference data could be used in decisions about new medicines had three stages:

  1. The first was to look at the literature and current research activity relating to patient preferences
  2. Researchers then held a workshop with patients and family members to understand more about what aspects of patient experience should be considered by researchers and by NICE
  3. An evaluative workshop was then held with researchers, health care professionals, policy makers, and other charities to discuss findings of the study so far

Findings from the research are as follows. A type of study called a ‘discrete choice experiment’ was found to be a promising method for measuring patient preferences. Our research suggests that discrete choice experiments (DCEs) could be very helpful to NICE in some situations. Examples include when several, but very different treatment options for the same condition exist, such as a choice between taking a drug or an operation, or a choice between taking a pill daily versus receiving an injection monthly.

Patient preference studies might also provide important insights in conditions where a person’s experience of the condition, and what treatments they might prefer, can differ a lot from person to person. People who have been living with the condition for a long time, for example, might have different preferences for treatment options than people who are newly diagnosed.

This NICE project also suggested that there is a clear scope for better use of quantitative patient preference studies. Findings demonstrated clearly how myeloma impacts the lives of patients and how important it is that these preferences are captured and used to ensure new treatments reflect the needs and preferences of patients and family members living with myeloma and other conditions.

Although this research project has now ended, NICE remains interested in how patient preferences studies might help its work. Recently, the NICE Scientific Advice team have piloted providing scientific advice for a patient preferences study. Companies who are interested in performing these studies can get in touch with the NICE Scientific Advice team for more information

You can read the full report here.

You can also find out more about NICE here or read our Health Technology Assessment information leaflet.