Multiple Myeloma

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 (MM), where the existence of treatments with different effects raises uncertainty about the value of these treatments and their effects to patients.

However, uncertainty exists on 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.

The objectives of this study are:

  • To determine the relative importance of MM treatment effects according to MM patients
  • To compare two methods for quantifying patient preferences: ‘swing weighting’ (SW) and ‘discrete choice experiment’ (DCE)
  • To investigate preference heterogeneity; how preferences may be influenced by patient characteristics, namely clinical and demographic characteristics and quality of life
  • To investigate the internal validity of responses to DCE and SW questions
  • To assess participants’ self-reported comprehension and overall evaluation of the DCE and SW questions

The study consists of two phases:

  1. A qualitative phase: Focus group discussions using the ‘nominal group technique’ (NGT) with approximately 6 MM patients each, in Belgium, Romania, Finland and Spain, that ask patients to reach consensus on the treatment effects they find most important when considering treatment options.
  2. A quantitative phase: An online survey among MM patients in Belgium, Romania, Finland and Spain that quantifies the trade-offs they are willing to make between hypothetical treatments that vary with respect to these effects.

Research team

  • Ana Vallejo (Myeloma Patients Europe)
  • Ananda Plate (Myeloma Patients Europe)
  • Anneleen Vanhellemont (KU/UZ Leuven)
  • Chris De Ronne (CMP Vlaanderen)
  • Daniel Coriu (Clinica de Hematologie)
  • Diana Preda (Clinica de Hematologie)
  • Elena Cabezudo (Hospital de Sant Joan Despí Moisés Broggi. Consorci Sanitari Integral/ICO-Hospitalet)
  • Hilde Stevens (I3h, Université libre de Bruxelles (ULB))
  • Isabelle Huys (KU Leuven)
  • Kate Morgan (Myeloma Patients Europe)
  • Martina Vandebroek (KU Leuven)
  • Michel Delforge (KU/UZ Leuven)
  • Raija Silvennoinen (Helsinki University Hospital)
  • Rosanne Janssens (KU Leuven)
  • Ruxandra Irimia (Clinica de Hematologie)
  • Steven Simoens (KU Leuven)
  • Sorina Badelita (Clinica de Hematologie)

Fact sheet

Therapeutic area Multiple Myeloma (MM)
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
  • To determine the relative importance of MM treatment effects according to MM patients.
  • To investigate preference heterogeneity; how preferences may be influenced by patient characteristics, namely clinical and demographic characteristics and quality of life.
Patients from Belgium
Romania
Spain
Finland
Methods in Qualitative study Focus group discussions using the "nominal group technique" (NGT)
Methods in Quantitative study Discrete Choice Experiment (DCE)
Swing Weighting
End-date qualitative data collection January 2020
End-date quantitative data collection December 2020