How to do a patient preference study

PREFER uses a step-wise approach to designing and conducting patient preference studies. In our approach, communicating with patients about the study before, during and after is crucial.

Designing a study

• Defining the research question: The research question consists of 3 parts: 1) The reason for asking for patient preferences, 2) The situation to be investigated, for instance which aspects of a disease or treatment matter to patients, or what is the level of risk patients are willing to take for a particular level of benefit, 3) The type of disease that will be studied.
• Defining the “sample”: Determining which patients are best suited to answer the research question. In this activity, different criteria need to be set up for defining which type of patient participant would be most appropriate to answer the defined research question.
• Choosing the method: Identifying if one, or several methods will be used to explore patient preferences. Methods can be qualitative or quantitative. Qualitative methods build on interviews or group discussions and address the question of why patients value different treatment attributes. Quantitative methods are survey-based and address the question of “How much” patients prefer one attribute over another, and the minimum acceptable benefit and the maximal acceptable risk they would need to receive in order to find a treatment acceptable.
• Designing the “instrument”: defining the questions and description of the attributes. Attributes are the different features of a treatment or health states that matter to patients and can be weighed against each other, for example the risk of a specific side effect may be weighed against the benefits of a treatment.

Conducting a study

• Recruiting participants based on how the “sample” was defined when the study was designed. Patients will be invited to participate if they meet the inclusion criteria.
• Piloting the study, which means running a small-scale version of the study to see if patients understand the questions. After the pilot test, researchers will fine-tune the questions in response to the feedback received in the pilot.
• Collecting information (data) for instance through recording interviews or collecting responses to surveys.
• Analyzing and interpreting the information (data) using different kinds of tools, statistics for survey data, or looking for common themes that appear for instance in interviews.

Communicating with patients

Prior, during, as well as after a patient preference study, communication with patients is crucial. Researchers need to explain the context and aim to patients who are invited to participate in a patient preference study. In addition, communication regarding the study results and use of the results is important. In PREFER, our goal is that pharma companies (that make the medicines), regulators (that approve medicines), Health Technology Assessment Bodies and Payers (that decide about whether medicines are paid for by the health care system) use the results of such studies to inform the decisions that they need to make on the development, approval and reimbursement of medicines. In this workshop, we will focus on communication of results of a patient preference study back to patient participants after the study is finished.

We want to make sure that study results are communicated back to the patient participants in a manner and timeframe that is useful and meaningful to them. This can be done in different ways and it’s important to understand what ways might be preferred by, and work best for patient participants.

Organising the work

In order to make all these activities happen, a clear organisation is necessary. This includes management of staff and expertise, involving patients, following good practices, applying for ethics approval, managing financial resources, and time management. Ensuring that patient involvement is happening in a correct way may include identifying what level of involvement is needed from patient researchers, time management, finding a meeting location that involved patients can get to or access (in person or online), providing involved patients with compensation for their time and effort, preparing information to inform possible patient researchers on the study and patient involvement needs, finding potential patient researchers to be involved in the study, and establishing written agreements with patient researchers on expectations, arrangements and working procedures.