Patient Preference Study

Acute leukemia patient’s preferences for treatment outcomes

Background

Obtaining a better understanding of the treatment outcomes that matter to people living with acute leukaemia can be useful to guide drug discovery, inform the appraisal of new treatments, and the development of managed access or outcomes-based payment schemes. To date, however, little quantitative preference research has been conducted in this context. This study could help to inform future treatment reimbursement decisions.

Aims

The overall aim of this study is to better understand the aspects of treatment that matter most to different groups of acute leukemia patients. This evidence will help inform the evaluation of treatments now and in the future.

More specifically, the objectives are to:

  • Better understand relative importance of different aspects of leukemia treatments and their outcomes to individuals with acute leukemia
  • Better understand and characterise the heterogeneity in patients’ preferences based on observed patient characteristics.
  • Support future health technology assessments and promote access to future treatment options, based on the aspects that matter most to patients.

Study approach

Methods

We will administer a discrete choice experiment (DCE) to elicit the preferences of people with acute leukaemia. DCEs involve presenting respondents with a set of choice tasks. Each choice task will involve the respondent choosing between two treatment options (or an opt-out option), which will be described using a set of attributes (treatment characteristics) with varying levels. In order to develop the attributes and levels of the DCE, qualitative research will be conducted with patients to understand the treatment characteristics of greatest importance. Once the attributes and levels are finalised, an experimental design will be produced alongside a broader online survey, which will be pilot tested and then subsequently launched.

Patient profile

Age: 18+ y.o

All acute leukemias

ALAN is running one study in patients who are relapsed/ refractory and another study in newly diagnosed patients.

Countries

  • UK
  • US
  • EU4

 

Outcomes

The data generated by the choice experiments will be analysed using statistical models, to estimate the impact of changes in attribute levels on respondent utility or satisfaction.

We will also explore how the utility of different attribute levels differs by respondent characteristics and disease experience (preference heterogeneity).

The primary deliverable will be a written report for the study sponsor, which will form the basis of a manuscript for submission to a peer-reviewed journal.

 

We are working with OHE on these studies.

For more information, please visit OHE – Health Economics and Outcomes Research