What are patient preferences - Isabelle Huys | PREFER webinar
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
- π€ PREFER is a public-private partnership that involves collaboration between various stakeholders, including pharmaceutical companies, academic institutions, health technology agencies, and patient organizations.
- π 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.
- π 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.
- π 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.
- π₯ PREFER is also involved in an IMI (Innovative Medicines Initiative) qualification procedure to gain approval for the patient preference study at the EU level.
- π Despite challenges during the COVID-19 pandemic, PREFER has continued its case studies and aims to draft recommendations by the end of March 2022.
- π 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.
- π 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.
- π οΈ 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.
- π The quantitative phase of patient preference studies aims to quantify the relative importance of attributes, determine acceptable risk levels, and understand patient trade-offs.
- π 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
π€ 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.
π 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
π‘PREFER
π‘Patient Preferences
π‘Patient-Reported Outcomes (PRO)
π‘Health Technology Agencies
π‘Qualitative and Quantitative Assessments
π‘Discrete Choice Experiment (DCE)
π‘Patient Heterogeneity
π‘Patient Preference Sensitive Decisions
π‘Methodologies
π‘Evidence-Based Recommendations
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.
Transcripts
i think i will just start with giving a
few words about what is prefer
i am i prefer so i am i prefer is a
public private partnership
between profits and non-profits entities
or
institutions where we have the industry
like large pharmaceutical companies but
also smaller companies that collaborate
with academic institutions health
technology
agency agencies patient organizations
in order to find out methodologies about
how to
measure and how to implement
methodologies
on patient preferences and how these
patient preferences can be measured
but also implemented in decision making
and the ultimate aim of prefer
is to guide industry regulatory
authorities hda bodies reimbursement
agencies
with their decisions on in different
institutions that they have and to guide
them on
how to measure patient preferences how
they can be assessed and how they can be
used
to inform medical product decision
making
with the aim also for them to further
develop
evidence-based recommendations based on
what
the insights of clifford reveal
river is a project that has started in
2016
with an uh work package that uh focused
on page finding out more about
patient preference methodologies
we did a lot of interviews a lot of
focus group
discussions and reveals therefore lots
of insights about
stakeholders and what are their needs we
will hear much about this
later by rosanna
after that work we selected certain
patient preference
methodologies that were further on used
into concrete case studies prefer as
conducting and has conducted
several case studies in different
disease areas
and these the case studies are still
going on and certain have been
have been finalized already and are at
the other point of
analysis at the same time
we have been involved in an imnita
qualification procedure
um which is also a procedure that is
going on
in order to involve also
the to get also the approval
for this email for this patient
preference study at the ima unita
level and as we are in
kovitz time indeed we had a very
difficult time also for
for prefer however we managed to
continue our case studies
most of the case studies and we aim to
draft our recommendations
by the end of march 2022
but what are these patient preferences
in the end so there are different
concepts that circulate
which are all related to the term
patient preferences
think about patient reported outcomes
people sometimes confuse patient
preferences with patient reported
outcomes
but patient report outcomes are more
data that
are collected on health status of a
particular patient directly from these
patients so
without any comparison with the
alternatives
whereas patient preferences as we use
and prefer
are more referred to qualitative and
quantitative assessments
of the relative desirability and
acceptability to patients
of specific alternatives or choices
among the outcomes or other attributes
that differ among
alternative health interventions and
here the term
attributes is a particular important one
so it's really about what is the
relative desirability and acceptability
of patients
of different attributes that are related
to a particular health intervention
and attributes are characteristics
features
related to these interventions or
disease states this can be
benefits related to that for instance
the relief of
pain or risks related to particular
health intervention for instance the
loss of hair
risk of having a loss of hair or
uncertainties related to it
for instance what are the long-term
health safety risks that are related to
a particular treatment
or even other um aspects which
we are in need of for instance certain
times of information that is or is not
available and all these aspects
are important for different stakeholders
to take decisions
across the work life cycle
here you see these different
stakeholders that can make use
of patient preferences in their decision
making context
for instance at the level of discovery
at the level of
pre-clinical developments also during
clinical development marketing
authorization
uh think of decisions at the european
residence agency or at the fda
health technology agencies or
reimbursement agencies here you see
the kce which is a partner in our
projects
or at the post-marketing authorization
level
and what type of decisions are these
then well it can be about decisions
related to defining the relative
clinical trial endpoint selections or
about the patient reported outcome
measures development
itself it can also be decisions which
are related to benefit risk assessments
or labeling or early access
and on a general level um patient
preferences might inform
decision making especially those
decisions that our patient preference
sensitive so we call them patient
preference
sensitive situations or patient
preference sensitive decisions
and what are then patient preference
sensitive
decisions or situations these are
situations where there's really a need
for the patient's view for instance
about
the most important attributes benefits
risks
most of administrations of a particular
disease
of a particular medicinal product that
might be one situation
another situation that can be when
multiple treatment options
exist and no option is clearly superior
for all the patients that we have in our
minds
and then it might also be a preference
sensitive situation so a situation where
patient preferences might really have
value
where there is really an uncertainty or
where the other op
where there is indeed evidence that
support one option over the other
and whether that is uncertain or very
valuable
and where there is really a need to
understand what is the patient's
tolerance
for that uncertainty and lastly there is
also a situation where there might be a
need to investigate further the
heterogeneity
among different patients views in a
population
and so we need to take care of about
these situations
and especially there their generation of
information about patient preferences
valuable and how do we do this we do
this
based on or using or setting up patient
preference
studies so patient preference studies
are studies that are characteristically
or typically set up
in different steps there is a design
step of a patient reference study there
is a conduct step so the
the point where we conduct a study
execute a study
and then we have the the point where we
communicate about the results
uh of this patient preference study and
indeed
all of all these aspects need to be
organized adequately
and that's also something which is which
need to be taken into account
so patient preference studies typically
consists of a qualitative phase
and a quantitative phase and we think of
when we talk about a qualitative phase
patient preferences make use of
qualitative methods
literature reviews focus groups
interviews depending on the setup
depending on the
type of population a group of population
that we need to target
and within at a qualitative level we
want to investigate
what do patients find important
treatment attributes and not only what
but also
why at the moment we have that this
may inform the more quantitative phase
of the study which might be a survey
discrete choice experiment or a swing
waiting
type of exercise where we want to
investigate
okay how much do patients now value
these attributes and what are the
trade-offs that these patients are
willing to make between these attributes
and how do preferences differ according
to the different characteristics that
all these patients have in our sample
what is the patient heterogeneity so for
this quantitative part
the aim is really to quantify to give
patient preference weights
relative importance of the attributes
determination
what is the maximal acceptable risk or
the minimum acceptable benefits
that patients need in order to accept a
certain
risk for instance and for that we also
target a larger group of patients
compared to the
qualitative part in order to reach
significant results
so this is actually in a nutshell what
are patient preference
studies how you can set them up it's
something which we in prefer
work further out and which we you can
also
hear in subsequent webinars in the
future
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