Multiple Myeloma

Multiple myeloma (MM) is the second most common blood cancer. There are various drugs available for MM. Yet, MM is considered an incurable disease and only half of patients live longer than five years.

Numerous clinical trials are currently investigating the benefits and risks of upcoming treatment classes such as CAR-T immunotherapies and bi-specific T-cell engaging immunotherapies. These are often associated with high benefits but also severe risks. In decision-making regarding these therapies, having information from patient preference studies on how patients trade off between their benefits and risks may be extremely valuable. 

The clinical objective of this study is to understand how patients trade off between the benefits and risks of multiple myeloma treatments. The methodological objectives are to: 1) compare two preference methods and 2) investigate whether and how patient preferences may be influenced by patient characteristics (e.g. treatment and disease experience).

The study consists of two phases: 1) group discussions with MM patients to find out which treatment characteristics they find most important, 2) online questionnaires with MM patients to quantify the trade-offs they are willing to make between hypothetical treatments that vary with respect to these characteristics. 

Fact sheet

Therapeutic area Multiple Myeloma
Study led by  KU Leuven
PREFER leads team Rosanne Janssens
Isabelle Huys
MPLC decision point of interest Drug development
Marketing authorisation
HTA/Reimbursement
PREFER case study acronym KUL - MM
Clinical objectives Identify patient-relevant benefit-risk attributes of MM treatments (including upcoming treatments such as immunotherapy)

Quantify trade-offs for benefit-risk attributes of MM treatments (including upcoming treatments such as immunotherapy)
Patients from Belgium
Romania
Spain
Finland
Methods in Qualitative study Focus group discussions using the Nominal Group Technique
Methods in Quantitative study Discrete Choice Experiment (DCE)
Swing Weighting
End-date qualitative data commection January 2020
End-date quantitative data collection Apri