What is the value of treatment effects according to Multiple Myeloma patients?


Patient preference studies provide evidence about what treatment effects are important to patients, how important they are to patients, and quantify the tradeoffs patients are willing to make between treatment effects. Understanding patient preferences may be especially valuable in the case of multiple myeloma, where the existence of treatments with different effects raises uncertainty about the value of these treatments effects to patients.

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However, there is uncertainty about how patient preference studies should be designed, conducted and analysed to inform decision-making across the drug life cycle, including drug development decisions by industry, benefit-risk assessment by regulators and health technology assessment (HTA) by HTA bodies and payers. This case study may guide stakeholders (researchers, regulators, payers, patient organisations, clinicians) in conducting or evaluating preference studies and contributes to the debate regarding best practices for preference studies aiming to inform drug decision-making.

Right now, PREFER is planning a preference survey that will collect data from patients to determine how they weigh the relative importance of treatment effects . We are also looking at preference heterogeneity, which means how preferences could be influenced by things like the patients’ quality of life, how they are affected by their disease, and demographic characteristics such as their age, gender, education.

We will use two different preference methods in this study: Discrete Choice Experiments (often called DCE), and Swing Weighting. This will allow us to see the differences in results between different methods, and help build knowledge for the PREFER recommendations on when and how to integrate patient preferences in medical product decision-making. Initially, we had planned to end our data collection in April this year, but there have been some delays because of the Covid-19 pandemic. We now expect data collection to end in December 2020, so keep an eye out for results from this study in 2021!

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By Rosanne Janssens & Josepine Fernow

Last modified: 2021-11-10