What are patient preferences - Isabelle Huys | PREFER webinar

IMI PREFER

29 Apr 202110:04

Summary

TLDRThe transcript discusses 'PREFER', a public-private partnership focusing on patient preferences in healthcare. It involves pharmaceutical companies, academic institutions, and agencies to measure and implement patient preferences in decision-making processes. The project, initiated in 2016, has conducted case studies across various diseases and aims to draft recommendations by March 2022. It differentiates patient preferences from patient-reported outcomes and highlights the importance of understanding patient values and trade-offs in medical interventions.

Takeaways

🤝 PREFER is a public-private partnership that involves collaboration between various stakeholders, including pharmaceutical companies, academic institutions, health technology agencies, and patient organizations.
🔍 The primary goal of PREFER is to guide industry, regulatory authorities, and reimbursement agencies in decision-making by incorporating patient preferences into medical product development and assessment.
📈 PREFER aims to develop evidence-based recommendations by analyzing insights gathered from patient preference methodologies, which began with a focus on understanding these methodologies through interviews and focus group discussions.
📚 The project has been conducting case studies in various disease areas to apply and analyze patient preference methodologies, some of which have been completed and are under analysis.
🏥 PREFER is also involved in an IMI (Innovative Medicines Initiative) qualification procedure to gain approval for the patient preference study at the EU level.
🌐 Despite challenges during the COVID-19 pandemic, PREFER has continued its case studies and aims to draft recommendations by the end of March 2022.
📊 Patient preferences are distinct from patient-reported outcomes, focusing on the qualitative and quantitative assessments of the desirability and acceptability of specific health intervention alternatives.
🔑 The term 'attributes' is crucial in patient preference studies, referring to the characteristics and features of health interventions or disease states that patients consider important.
🛠️ Patient preference studies are structured in a multi-step process, including design, conduct, and communication phases, and typically involve both qualitative and quantitative research methods.
📈 The quantitative phase of patient preference studies aims to quantify the relative importance of attributes, determine acceptable risk levels, and understand patient trade-offs.
📊 Patient preference studies can inform decision-making across the medical product life cycle, from discovery to post-marketing authorization, in areas such as clinical trial endpoint selection, benefit-risk assessments, and labeling.

Q & A

What is the purpose of the PREFER project?
-The PREFER project aims to guide industry, regulatory authorities, HTA bodies, and reimbursement agencies in their decisions on how to measure and implement patient preferences in medical product decision-making, with the ultimate goal of developing evidence-based recommendations based on patient insights.
What kind of entities does the PREFER project involve?
-PREFER is a public-private partnership that includes large pharmaceutical companies, smaller companies, academic institutions, health technology agencies, patient organizations, and other stakeholders.
When did the PREFER project start and what was its initial focus?
-The PREFER project started in 2016 with a focus on identifying patient preference methodologies through interviews and focus group discussions to gain insights into stakeholders' needs.
What is the significance of involving patient preferences in medical decision-making?
-Involving patient preferences is significant as it ensures that the patient's view is considered in decisions related to clinical trial endpoints, benefit-risk assessments, labeling, and early access, among other aspects of the medical product lifecycle.
How are patient preferences different from patient-reported outcomes?
-Patient-reported outcomes are data collected on a patient's health status directly from the patient, without comparison to alternatives. In contrast, patient preferences refer to qualitative and quantitative assessments of the relative desirability and acceptability of specific alternatives or choices among different attributes of health interventions.
What are some examples of attributes that might be considered in patient preference studies?
-Examples of attributes include benefits such as pain relief, risks such as hair loss, long-term health safety risks, and other aspects like the availability of information, which are important for stakeholders to make informed decisions.
What are patient preference-sensitive decisions and why are they important?
-Patient preference-sensitive decisions are situations where the patient's view is particularly needed, such as when there is uncertainty or no clear superior treatment option, and understanding the patient's tolerance for risk or uncertainty is crucial.
How does the PREFER project plan to draft its recommendations?
-The PREFER project aims to draft its recommendations by the end of March 2022, based on the insights gained from conducting several case studies in different disease areas and analyzing the results.
What are the typical steps involved in setting up a patient preference study?
-Setting up a patient preference study typically involves a design step, a conduct step where the study is executed, and a communication step where the results are shared. These studies usually consist of both a qualitative phase to explore what patients find important and a quantitative phase to quantify the relative importance of attributes.
How does the PREFER project address the heterogeneity among patients in its studies?
-The project addresses patient heterogeneity by targeting a larger group of patients in the quantitative phase of the study to reach significant results and understand how preferences differ according to various patient characteristics.
What challenges did the PREFER project face during the COVID-19 pandemic?
-During the COVID-19 pandemic, the PREFER project faced difficulties in continuing its case studies. However, they managed to adapt and continue most of their studies, aiming to maintain their progress towards drafting recommendations.

Outlines

00:00

🤝 Public-Private Partnership in Patient Preferences

The first paragraph introduces 'PREFER' as a public-private partnership that involves various stakeholders, including pharmaceutical companies, academic institutions, and patient organizations. The goal of PREFER is to develop methodologies for measuring and implementing patient preferences in decision-making processes. The initiative aims to guide industry, regulatory authorities, and reimbursement agencies in their decisions regarding medical product development. The project, which began in 2016, has conducted interviews and focus groups to gather insights into patient preference methodologies and is currently working on case studies across different disease areas. The ultimate aim is to inform medical product decision-making with evidence-based recommendations derived from patient insights.

05:01

📊 Patient Preference Studies and Their Role in Decision Making

The second paragraph delves into the importance of patient preferences in the context of decision-making across various stages of the medical product life cycle, from discovery to post-marketing authorization. It discusses the types of decisions influenced by patient preferences, such as clinical trial endpoint selections, benefit-risk assessments, and labeling. The paragraph also defines 'patient preference-sensitive' decisions, which are situations where the patient's perspective is crucial due to the presence of multiple treatment options, uncertainty, or the need to understand patient tolerance for risk. The process of conducting patient preference studies is outlined, emphasizing the need for a well-organized approach that includes qualitative and quantitative phases. The qualitative phase focuses on understanding what patients find important, while the quantitative phase aims to quantify patient preferences and identify trade-offs they are willing to make.

Mindmap

Keywords

💡PREFER

PREFER stands for 'Patient Preferences in Healthcare Decision-making.' It is a public-private partnership involving various stakeholders such as pharmaceutical companies, academic institutions, health technology agencies, and patient organizations. The primary aim of PREFER is to guide industry, regulatory authorities, and reimbursement agencies in decision-making by incorporating patient preferences. This initiative is crucial as it ensures that the patient's voice is considered in healthcare decisions, which is a central theme of the video.

💡Patient Preferences

Patient preferences refer to the qualitative and quantitative assessments of the relative desirability and acceptability of specific alternatives or choices among outcomes or other attributes that differ among alternative health interventions. In the context of the video, patient preferences are critical in guiding decisions across the healthcare product life cycle, from discovery to post-marketing authorization. The video emphasizes the importance of understanding these preferences to inform medical product decision-making.

💡Patient-Reported Outcomes (PRO)

Patient-Reported Outcomes (PRO) are data collected directly from patients about their health status without any comparison with alternatives. While PROs are an important aspect of patient-centered care, they are distinct from patient preferences, which involve evaluating the relative desirability of different treatment attributes. The video script clarifies this distinction to highlight the unique role of patient preferences in decision-making.

💡Health Technology Agencies

Health technology agencies are organizations that evaluate the effectiveness, safety, and economic aspects of healthcare interventions. In the video, these agencies are mentioned as key stakeholders that can utilize patient preferences to inform their decisions on healthcare policies and reimbursement. The inclusion of patient preferences in their assessments can lead to more patient-centered healthcare practices.

💡Qualitative and Quantitative Assessments

Qualitative and quantitative assessments are research methods used to understand patient preferences. Qualitative methods, such as interviews and focus groups, explore 'what' and 'why' patients find certain treatment attributes important. Quantitative methods, like surveys and discrete choice experiments, measure 'how much' patients value these attributes and their willingness to make trade-offs. The video underscores the importance of both approaches in conducting patient preference studies.

💡Discrete Choice Experiment (DCE)

A Discrete Choice Experiment (DCE) is a quantitative method used to elicit patient preferences by presenting them with hypothetical choices between different treatment options, each with varying attributes and levels. DCEs help determine the relative importance of treatment attributes and patient trade-offs, which is a significant part of understanding patient preferences as discussed in the video.

💡Patient Heterogeneity

Patient heterogeneity refers to the variability in patient preferences due to different characteristics among patients. Recognizing and accounting for this heterogeneity is essential in patient preference studies to ensure that the preferences of diverse patient populations are represented. The video highlights the importance of capturing this diversity to inform decision-making that is sensitive to the needs of all patients.

💡Patient Preference Sensitive Decisions

Patient preference sensitive decisions are situations where incorporating patient preferences is particularly valuable. These include decisions about the most important treatment attributes, when multiple treatment options exist without a clear superior choice, or when there is uncertainty about the best option. The video emphasizes the role of patient preferences in guiding such decisions to ensure they align with patient values and priorities.

💡Methodologies

Methodologies in the context of the video refer to the systematic approaches and techniques used to measure and implement patient preferences. These methodologies are crucial for conducting patient preference studies and ensuring that the insights gained are robust and reliable. The video discusses the development and application of these methodologies as a key aspect of the PREFER project.

💡Evidence-Based Recommendations

Evidence-based recommendations are decisions or guidelines that are informed by scientific evidence, including patient preferences. The video mentions that the ultimate aim of PREFER is to develop such recommendations, which can help various stakeholders, including regulatory authorities and healthcare providers, make more informed decisions that consider the voice of the patient.

Highlights

PREFER is a public-private partnership involving industry, academia, and patient organizations.

The aim of PREFER is to guide industry, regulatory authorities, and reimbursement agencies on measuring and implementing patient preferences.

PREFER conducts case studies in various disease areas to understand patient preferences.

Patient preferences are distinct from patient-reported outcomes, focusing on the relative desirability and acceptability of health intervention attributes.

Attributes of health interventions include benefits, risks, and other features that patients consider important.

Patient preferences can inform decision-making across the work life cycle, from discovery to post-marketing authorization.

Patient preference-sensitive decisions are situations where the patient's view is crucial, such as when multiple treatment options exist.

Patient preference studies are conducted in two phases: qualitative to understand what patients value and why, and quantitative to measure the relative importance of these attributes.

Qualitative methods like literature reviews, focus groups, and interviews are used to explore patient preferences.

Quantitative methods such as surveys and discrete choice experiments help to quantify patient preferences and trade-offs.

PREFER's work includes an EMA qualification procedure to gain approval for patient preference studies at the regulatory level.

Despite challenges during the COVID-19 pandemic, PREFER managed to continue its case studies.

PREFER aims to draft recommendations by the end of March 2022 based on its findings.

Patient preferences can influence decisions related to clinical trial endpoint selections and benefit-risk assessments.

Understanding patient heterogeneity is crucial for generating valuable information about patient preferences.

PREFER's work will be further elaborated in subsequent webinars, providing insights into patient preference studies.