Results from the PREFER Project Show a Need for Inclusion of QoL-Related Treatment Outcomes in MM Drug Development

New and complex treatments for multiple myeloma (MM) are being developed that have varying efficacy and side-effect profiles, which create uncertainties for patients compared with currently available therapies. Stakeholders involved in drug development see the potential benefit in using patient preference studies to inform decisions; however, more robust patient-preference data are needed to aid in MM drug development and evaluation.¹

At the 2022 ISPOR (International Society for Pharmacoeconomics and Outcomes Research) meeting, Janssens and colleagues presented the results of a study of patient preferences among those with MM, which highlighted results from the Patient Preferences in Benefit-Risk Assessment during the Drug Life Cycle (PREFER) project.² The PREFER project was designed to investigate how and when it is best to include patient preferences in decision making during drug development and to recommend best-practices for drug developers, regulatory authorities, health technology assessment bodies, and payers.²

This was a mixed-methods patient preference study that used qualitative and quantitative phases.² The qualitative phase was conducted initially to understand key attributes (symptoms, side-effects, and efficacy outcomes and uncertainties) most important to patients. It was done in 3 steps consisting of literature reviews, focus group discussions with patients, and a thematic analysis involving multistakeholder discussions.^1,2 The key attributes and severity levels (from mild to severe) discovered in the qualitative phase of the study were used to develop patient surveys in the quantitative phase.^1,2 The goal of the quantitative phase was to gain insight into the relative value of these key attributes to patients, understand how preferences differ among patients (preference heterogeneity), and test and compare 2 types of surveys: Discrete Choice Experiment and Swing Weighting.²

Overall, 393 patients from 21 countries participated.³ The patient population was heterogeneous regarding years of diagnosis and prior therapies. Results from the quantitative phase showed that life expectancy was most important to patients with the most and least prior therapies.³ However, patients with intermediary treatment experiences noted that quality of life-related attributes, such as pain and mobility problems, were most important.³ Depending on a patient’s side-effect and symptom experience, researchers found substantial heterogeneity in patient preferences.³

This study suggests that there is a need for inclusion of quality of life-related disease and treatment outcomes in MM drug development. For preference studies to be successful, it is important to incorporate a qualitative portion to ensure appropriate attribute selection and to involve clinicians to ensure that the attributes are clinically plausible and formatted accurately. In addition, it is important to obtain input from patients and patient organizations to develop and pilot the survey. Substantial internal review is also important, including external peer-review by experts in the field. Finally, a detailed description of participants’ backgrounds and how the attribute weights are statistically influenced by background variables is necessary.

Sources

1. Janssens R, Lang T, Vallejo A, et al. Patient preferences for multiple myeloma treatments: a multinational qualitative study. Front Med (Lausanne). 2021 Jul;8:686165.
2. Janssens R. Patient preferences for multiple myeloma (MM) treatment: A mixed-methods patient preference study using discrete choice experiment and swing weighting. Presented at: ISPOR; May 15-18 2022; Washington, DC. Accessed May 31, 2022.
3. Janssens R. Patient preferences for multiple myeloma treatment: A stated preference survey using discrete choice experiment and swing weighting. Abstract presented at: ISPOR; May 15-18 2022; Washington, DC. www.ispor.org/heor-resources/presentations-database/presentation-paper/intl2022-3472/14046/patient-preferences-for-multiple-myeloma-treatment-a-stated-preference-survey-using-discrete-choice-experiment-and-swing-weighting. Accessed May 31, 2022.