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Myeloma UK/NICE collaborative project: NICE publishes opinion on patient preference data

Head of Patient Advocacy, Shelagh McKinlay shares her thoughts on a perspective from NICE on the use of patient preference data.

Patient advocacy news, Research news // 19th February 2020

Myeloma UK has a strong track record of communicating the patient voice to decision makers in the UK’s drug approval bodies to ensure that patients have access to the right treatment at the right time. We do this by submitting evidence informed by patients’ opinions on treatments and by attending meetings with drug approval bodies alongside patients themselves.

Our research, however, has shown that patients often have distinct opinions when it comes to their treatment and care. In particular, patients can value the benefits and risk of treatment differently so a patient’s definition of the right treatment can differ a lot. Their opinion will depend on a whole range of factors including age, family circumstances, how many treatments they have had, or the level of side effects experienced.

One way to capture patient opinions on new treatments is to run patient preference studies. These studies tell us not just what is important to patients, but what aspects of their treatment and care, as well as what benefits, are most important to them; and what “trade-offs” patients are prepared to make when thinking about the advantages and disadvantages of treatment.

To understand how patient preference data could be used in approving new treatments for use on the NHS (known as a health technology assessment or HTA) Myeloma UK funded a two-year project in collaboration with a research team at the National Institute of Health and Care Excellence (NICE).

The project team found that there was “clear scope for better use of patient preference data within HTA” and recommended that further work including pilots be carried out to identify how patient preference data can be applied in drug approval decision making.

This month, NICE published their opinion on the use of patient preference data in HTA decision making.

They recognised the potential in the use of patient preference studies to inform decision making throughout the drug development and approval processes.

This included:

  • In early drug development to help identify trial endpoints
  • In assessing benefits and risks, particularly in regulatory approval (approving licenses for drugs)
  • in HTA processes to inform decisions on “reimbursement” (deciding on the value of a drug and whether it should be approved for use on the NHS)

Although NICE do see patient preference data forming part of the decision making process alongside other types of evidence, they do not see patient preference data being used as part of the calculations used to determine whether a treatment is considered cost-effective.

Shelagh McKinlay, Head of Patient Advocacy at Myeloma UK said:

“It is great to see a large health approval body like NICE highlight the importance of involving patients and capturing patient preference data in the drug development and approval processes.

We are encouraged that NICE see patient preference data as offering valuable insights to health technology assessment bodies. There is broad consensus that the system must do better in understanding what is important to patients and the decisions they must make about treatment benefits in “the real world”. Without understanding what matters most to patients, how can we truly understand the value of a treatment?

We believe there are many ways in which NICE could apply greater flexibility to ensure that patient preference data carries due weight in the decision making process.  We hope that the potential of patient preference data can be looked at through the ongoing NICE methods review.

And it is also worth emphasising the transformative potential of patient preference data if it is applied as early as possible in the drug development process; not just in identifying trial endpoints but in helping industry to understand the gap between treatments that are desired by patients and what is currently in development.”

Myeloma UK will continue to advocate for myeloma patients to make sure they are involved, and their voice is heard when decisions are being made about their health and care. We work to strike a balance between harnessing big data through the development of digital tools and finding new ways to involve and connect with individual patients, giving them the opportunity to talk about living well with myeloma. This combination of statistical and first-hand evidence gives real weight to the patient experience and to policymakers’ understanding of the impact a treatment can have. This approach is a cornerstone of our commitment to putting patients at the heart of everything we do.